Tue May 10
2:00 PM - 3:00 PM
On-Demand Podium Session
View anytime on this date, after 2:00pm, until June 17. Applications of Discrete Choice Experiments for Patient Preference Elicitation
On-demand
Moderator
Ellen Janssen, PhD
The Janssen Pharmaceutical Companies of J&J, Baltimore, MD, USA
P53: Patient Preferences for Multiple Myeloma Treatment: A Stated Preference Survey Using Discrete Choice Experiment and Swing Weighting
2:15PM - 2:30PM
Janssens R 1 , Lang T2 , Vallejo A2 , Galinsky J2 , Morgan K2 , Plate A2 , Verschueren M3 , Schoefs E1 , Vanhellemont A4 , Delforge M4 , Hellem Schjesvold F5 , Cabezudo E6 , Vandebroek M3 , Stevens H7 , Simoens S1 , Huys I1 1 KU Leuven, Leuven, VBR, Belgium, 2 Myeloma Patients Europe, Brussels, Belgium, 3 KU Leuven, Leuven, Belgium, 4 University Hospitals Leuven, Leuven, Belgium, 5 Oslo University Hospital, Oslo, Norway, 6 H. Moises Broggi / ICO-Hospitalet, Barcelona, Spain, 7 Université libre de Bruxelles, Brussels, Belgium
OBJECTIVES: Patient preferences have become an important focus for inclusion in drug development and evaluation. Understanding patient preferences is especially valuable in Multiple Myeloma (MM), where the rapid development of treatments with diverging benefit-risk profiles raises uncertainty about what matters most to patients. This study aimed to investigate which treatment attributes (side-effects, symptoms, efficacy outcomes, uncertainties) are most important to MM patients.
METHODS: A preference survey incorporating Discrete Choice Experiment (DCE) and Swing Weighting (SW) was widely disseminated through the European MM patient population. The survey was developed using a qualitative study during which MM patients (n=24) reached consensus on the attributes and levels included in the survey. MM patients and patient organizations provided extensive feedback during the survey development and piloting.
RESULTS: 393 MM patients across 21 countries participated. Patients were heterogeneous regarding years since diagnosis (M: 6) and prior therapies (M: 3). While life expectancy was most important to patients with the most and least prior therapies, quality of life-related attributes such as pain and mobility problems were most important to participants with intermediary treatment experience. Significant preference heterogeneity was revealed depending on participants’ side-effect and symptom experience. Participants highlighted the difficulty of trading-off between life expectancy and quality of life, and between physical and mental health. Patients demanded psychological support to cope with their symptoms, side-effects and uncertainties.
CONCLUSIONS: Preferences elicited from patients reveal the need for the systematic inclusion and prioritization of quality of life-related treatment outcomes by drug developers, regulators, Health Technology Assessment bodies, and healthcare providers in MM drug development, evidence generation, evaluation, and clinical practice. In order for patients to make informed choices in preference studies, researchers should involve patients and patient organizations during the selection of the attributes, levels, and explanations, how the preference questions are asked, the visuals and survey technology.
P56: Patient Preferences in Multiple Myeloma: A Discrete Choice Experiment
2:00PM - 2:15PM
Tervonen T1 , Duenas A2 , Collacott H2 , Lam A3 , Gries KS3 , Carson R3 , Trevor N4 , Krucien N2 , He J 3 1 Evidera, London, UK; University Medical Center Groningen, London, UK, 2 Evidera, London, LON, UK, 3 Janssen Global Services, LLC, Raritan, NJ, USA, 4 Janssen-Cilag Ltd, High Wycombe, BKM, UK
OBJECTIVES
: This study assessed the preferences of patients with multiple myeloma (MM) for treatment attributes and evaluated the impact of health-related quality of life (HRQoL) on those preferences.
METHODS
: Patients in the UK, France, and Germany with physician-confirmed transplant eligible (TE) or transplant ineligible (TIE) newly diagnosed MM (NDMM) or relapsed/refractory MM (RRMM) completed a discrete choice experiment (DCE). HRQoL was assessed using the EuroQoL Five Dimension (EQ-5D) questionnaire. Preferences for four benefit attributes (increased life expectancy, increased time to relapse, reduced pain, and reduced fatigue), three administration attributes (method of administration, frequency of administration, and monitoring), and one risk attribute (risk of severe infection) were analyzed with a multinominal logit model. Differences between subgroups were also analyzed.
RESULTS
: 300 patients completed the DCE (TE NDMM, n=108; TIE NDMM, n=105; RRMM, n=87). Median age was 68 years; most common symptoms were fatigue (69%), bone pain (63%), and sleepiness/tiredness (55%). Median EQ-5D score was 0.8 (IQR 0.7-0.9), with no differences by disease stage. Patients most valued reduced pain and fatigue and increased life expectancy. In general, patients favored shorter injection/monitoring time and less frequent administration. Patients were willing to make trade-offs, such as accepting a reduction of ≥ 2.7 years of life expectancy for no pain vs extreme pain and ≥ 2.0 years for no fatigue vs constant fatigue. Patients with lower EQ-5D scores were willing to trade more years of life expectancy than those with higher scores for reductions in pain or fatigue. Disease stage (NDMM vs RRMM), country, and age did not affect preferences.
CONCLUSIONS
: Patients with MM valued treatments that reduced pain and fatigue and were willing to trade life expectancy for better quality of life. HRQoL influenced patient preferences, suggesting that this is an important attribute that should be considered as part of the treatment decision-making process.
P55: A Discrete Choice Experiment of Patient-Informed Preferences for Major Depressive Disorder Treatment
2:30PM - 2:45PM
Dosreis S 1 , Amill-Rosario A1 , Ali C2 , Elonge E3 , Xie R4 , Chapman R5 , Slejko JF1 1 University of Maryland School of Pharmacy, Baltimore, MD, USA, 2 University of Maryland School of Pharmacy, Sandy Spring, MD, USA, 3 University of Maryland Baltimore, Baltimore, MD, USA, 4 The Innovation and Value Initiative, Newton, MA, USA, 5 The Innovation and Value Initiative, Alexandria, VA, USA
OBJECTIVE : Major depressive disorder (MDD) affects 10% of US adults, of which 30% experience moderate/severe impairment despite treatment. While value assessment often relies on clinically derived measures, how MDD treatment impacts individuals’ daily lives and what matters most to them is also relevant for value assessment. The study aimed to assess preferences for patient-informed attributes of MDD treatment.
METHODS
: A discrete choice experiment (DCE), developed with meaningful stakeholder engagement, was administered to 150 community-dwelling adults aged 18 and older diagnosed with MDD. We excluded post-partum depression and comorbid bipolar, psychosis, and cognitive disorders. A diverse sample from across the US was recruited via ResearchMatch.org. The DCE had six attributes, each with three levels: treatment modality, time to effect, days of hopefulness, improvement in productivity, impact on relationships, and out-of-pocket costs. An orthogonal array design with 100 D-efficiency yielded six choice tasks. A conditional logit model generated preference weights for each attribute level. The relative attribute importance is the proportion each attribute contributes to the total variance in preference weights.
RESULTS
: Participants were 40 years-old on average, 62% male, 28% female, and 10% non-binary/transgender. Race/ethnicity demographics reflected 45% White, 15% Black, 19% Latino, 13% Asian, and 8% mixed race. The relative attribute importance, in rank order, is out-of-pocket costs (30%), social relationships (29%), hopeful days (18%), treatment modality (9%), productivity (7%), and time to treatment effect (7%). Participants preferred treatment modalities that included medication, psychotherapy, and other services (e.g., brain stimulation); improved social relations; hopefulness six-days per week, and
< $90 USD monthly out-of-pocket costs.
CONCLUSION : Among individuals with MDD, social and life impact outcomes are preferred over time to treatment effect or productivity, which are more commonly used in economic evaluation. To reflect patient value more completely, these attributes should be considered in value assessments as well.
P54: Patient Preferences for Treatment of BCG-Unresponsive Non-Muscle Invasive Bladder Cancer: A Discrete Choice Experiment
2:45PM - 3:00PM
Collacott H 1 , Rentz A2 , Krucien N1 , Heidenreich S1 , Ghatnekar O3 1 Evidera, London, LON, UK, 2 Evidera, Bethesda, MD, USA, 3 Ferring International PharmaScience Center, Copenhagen, Denmark
OBJECTIVES:
First-line treatments for high-risk non-muscle invasive bladder cancer (NMIBC) include transurethral resection of the bladder tumour and Bacillus Calmette-Guerin (BCG) intravesical therapy. If BCG therapy fails, patients are offered bladder removal surgery (radical cystectomy (RC)), a major surgical intervention with impact on quality of life. This study quantifies the trade-offs patients are willing to make when choosing between RC and an alternative medical treatment. METHODS:
An online discrete choice experiment was completed by adults in the UK, France, Germany and Canada who were, or had been, treated with BCG therapy, or have undergone RC for NMIBC. Participants repeatedly chose between immediate RC and two hypothetical medical treatments that varied in: time-to-RC; risk of progressing to MIBC (muscle invasive bladder cancer) while on treatment; risk of serious side effects (SAEs); and administration. Preferences were analysed with an error-component logit model. Relative attribute importance (RAI) scores and minimum acceptable time to RC were obtained to explore patients’ treatment priorities.
RESULTS:
The study included 107 patients (64% male, average age 63 years, 93% diagnosed with NMIBC 1-5 years before; 82% Eastern Cooperative Oncology Group performance status ≤2). Delay in time to RC had the largest influence on treatment preferences (RAI = 55.0%), followed by reducing the risk of progressing to MIBC while on treatment (RAI = 25.1%), and less frequent administrations (RAI 11.6%). Risk of SAEs (RAI = 8.3%) was the least important attribute. Patients were willing to accept a 20% increase in the risk of progressing to MIBC for an additional 2.3-years until RC, and a 10% increase in the risk of SAEs for an additional 0.8-years until RC.
CONCLUSIONS:
Patients treated with BCG for NMIBC placed a high importance on delaying RC and were willing to accept increased risks of both SAEs and progression to MIBC for prolonged bladder preservation.
Real-World Evidence for Comparative Effectiveness, Safety and Adherence Evaluations
On-demand
Moderator
Laura Bozzi, MS, PhD
Janssen of J&J, Titusville, NJ, USA
Laura Bozzi, MS, PhD, is Manager of Benefit-Risk Assessment in the Global R&D Epidemiology Department at Janssen Pharmaceuticals of Johnson & Johnson in New Jersey. Laura started at Janssen in July 2021 and her role focuses on structured benefit-risk assessment to support regulatory submissions across therapeutic areas. Additionally, Laura leads patient preference studies to inform benefit-risk assessment and support internal decision-making. Prior to joining Janssen, Laura completed her doctorate at the University of Maryland, Baltimore (UMB) in the Pharmaceutical Health Services Research program where she received a R36 Health Services Research Dissertation Grant from the Agency for Health Research and Quality. As a graduate research assistant at UMB, she was apart of the Patient-Driven Values in Healthcare Evaluation (PAVE) Center where her research consisted of the design, implementation, and analysis of discrete choice experiments to understand patient preferences as it pertains to treatment decisions in the areas of COPD and Major Depressive Disorder. Additionally, she has served project coordinator for PAVE, developing high level strategy for engaging internal and external stakeholders (e.g. non-profit organizations, patient advocated, and industry partners) on PCORI, AHRQ, and PhRMA funding and research opportunities. She obtained her Bachelor of Science degree in Biological Sciences from the University of Connecticut and her Master of Science degree in Epidemiology & Genetics from UMB.
P74: Real-World Comparative Effectiveness of Pegfilgrastim Biosimilars Versus Originator
2:15PM - 2:30PM
Wang CY 1 , Park H1 , Heldermon CD2 , Vouri SM1 , Brown JD1 1 Center for Drug Evaluation & Safety, Department of Pharmaceutical Outcomes and Policy, Gainesville, FL, USA, 2 University of Florida, College of Medicine, Gainesville, FL, USA
OBJECTIVES:
Real-world evidence on the clinical effectiveness of pegfilgrastim biosimilars are limited. This study compared the incidence of febrile neutropenia (FN) between pegfilgrastim biosimilars (pegfilgrastim-jmdb, pegfilgrastim-cbqv) and originator users.
METHODS:
A retrospective cohort study using 2019 IBM MarketScan Commercial and Medicare Supplemental database was conducted in adult patients with cancer initiating myelosuppressive chemotherapy courses. At least 2 cancer diagnoses (at least 7 days apart within +/- 30 days of the initiation) were required. The following exclusion criteria were applied: (1) ≥2 solid cancers; (2) acute myeloid leukemia; (3) autologous peripheral blood progenitor cell collection; (4) bone marrow transplantation; (5) using pegfilgrastim on-body-injector/with unknown route; (6) using more than 1 granulocyte-colony stimulating factors product. Cumulative incidence of FN associated hospitalization was measured by ICD-10 diagnosis codes (neutropenia, fever, or infection) in the first cycle. After 1:1 propensity score (PS) matching, we compare FN risk between biosimilars and originator users using equivalence (with a margin of 6%) and superiority hypothesis tests.
RESULTS:
A total of 2,045 patients were included (445 used pegfilgrastim-jmdb, 636 used pegfilgrastim-cbqv, and 964 used pegfilgrastim originator). After PS matching, 13 out of 445 originator users and 17 out of 445 pegfilgrastim-jmdb users developed FN (risk difference was 0.9%; p-value was 0.4575 for superiority test indicating no difference ;p-value was <0.0001 for equivalence test indicating statistical equivalence). After PS matching, 14 out of 633 originator users and 16 out of 633 pegfilgrastim-cbqv users developed FN (risk difference was 0.32%; p-value was 0.7117 for superiority test indicating no difference; p-value was <0.0001 for equivalence test indicating statistical equivalence).
CONCLUSIONS:
In this real-world study of patients with cancer receiving myelosuppressive chemotherapy, there was no difference in FN risk between patients receiving pegfilgrastim originator and biosimilars in the first cycle.
P76: Comparative Adherence Trajectories across Oral Disease-Modifying Agents in Multiple Sclerosis
2:30PM - 2:45PM
Earla JR 1 , Hutton GJ2 , Aparasu RR3 1 University of Houston, College of Pharmacy, Fords, NJ, USA, 2 Baylor College of Medicine Medical Center, McNair Campus, Houston, TX, USA, 3 University of Houston, College of Pharmacy, Houston, TX, USA
OBJECTIVES:
The oral Disease-Modifying Agents (DMAs) such as fingolimod (FIN), teriflunomide (TER), and dimethyl fumarate (DMF) are convenient alternatives to injectable DMAs for Multiple Sclerosis (MS). However, there is limited evidence regarding the comparative adherence patterns across different oral DMAs. This study compared the adherence trajectories between FIN, TER, and DMF users with MS.
METHODS:
A retrospective longitudinal study was conducted involving adults (≥18 years) identified with MS (ICD-9/10-CM:340/G35 and a DMA prescription) from the 2015–2019 IBM MarketScan Commercial Claims Database. Patients were classified as incident FIN-, TER- or DMF-users based on the index DMA with one year of washout period. The DMA adherence trajectories based on Proportion Days Covered (PDC) were examined using Group-Based Trajectory Modeling (GBTM) during the one year after the treatment initiation. Generalized boosting models (GBM)-based Inverse Probability Treatment Weights (IPTW) were incorporated in multinomial logistic regression to assess the comparative adherence trajectories across oral DMAs with FIN group as reference category.
RESULTS:
The study cohort consisted of 1,913 MS patients who were initiated with FIN (24.2%,n=462), TER (23.9%,n=468), and DMF (51.9%,n=993) during 2016–2018. The adherence rate (PDC≥0.8) among FIN, TER, and DMF users was found to be 70.8%(n=327), 59.6%(n=273), and 61.0%(n=606), respectively. The GBTM grouped study subjects into three adherence trajectories – Complete Adherers-59.1%, Slow Decliners-22.6% and Rapid Discontinuers-18.3%. The multinomial logistic regression model involving GBM-based IPTW revealed that TER (adjusted odds ratio [aOR]-2.32, 95% CI:1.57-3.42) and DMF (aOR-2.50, 95% CI:1.62-3.88) users had higher odds to be rapid discontinuers relative to FIN users. In addition, TER users are more likely (aOR-1.50, 95% CI:1.06-2.13) to be slow decliners compared to FIN.
CONCLUSIONS:
Teriflunomide and dimethyl fumarate were associated with poorer adherence trajectories than fingolimod. More research is needed to evaluate the clinical implications of these adherence trajectories of oral DMAs for the management of MS.
P73: Real-World Effectiveness and Safety of Non-Vitamin K Antagonist Oral Anticoagulants (NOAC) in Patients with Non-Valvular Atrial Fibrillation (NVAF): A Retrospective Cohort Study in Singapore
2:00PM - 2:15PM
Foo W1 , Hui T 2 , Ong SKB1 , Ng KH1 1 Agency for Care Effectiveness, Ministry of Health, Singapore, Singapore, 2 Agency for Care Effectiveness, Ministry of Health, Singapore, 01, Singapore
OBJECTIVES:
Non-vitamin K antagonist oral anticoagulants (NOACs) are increasingly being used over warfarin for stroke prevention in non-valvular atrial fibrillation (NVAF) patients. However, there are limited studies on clinical outcomes of NOAC in a multi-ethnic Asian country such as Singapore. This study aims to compare the real-world effectiveness and safety between NOAC and warfarin in NVAF patients and assess the potential long-term impact on the healthcare system in Singapore.
METHODS:
Using government’s linked national health record databases, patients aged 18 years and above initiated on NOAC or warfarin for NVAF from 2015 to 2018 were included in this retrospective cohort study. Patient’s demographics, comorbidities, and comedications were balanced using propensity score matching. Primary efficacy and safety endpoints were stroke or systematic embolism (SE) and intracranial haemorrhage (ICH) or gastrointestinal (GI) bleeding, respectively. Effect size was estimated using hazard ratios (HRs) with 95% confidence intervals (CIs) from Cox regression. A Markov model was used to extrapolate the number of deaths and hospitalisations avoided with NOAC initiation.
RESULTS:
After propensity score matching, 3,315 comparable patients in each cohort remained. Over a median follow-up period of 2.0 years, NOAC users were less likely to develop a stroke or SE than warfarin users (HR 0.83; 95% CI 0.70-0.99). NOAC initiation was associated with a lower incidence rate of any ICH or GI bleed (HR 0.77; 95% CI 0.66-0.91) and all-cause mortality (HR 0.81; 95% CI 0.70-0.94). NOAC initiation was estimated to avoid 1,270 deaths, 622 stroke/SE hospitalisations and 1,311 ICH/GI bleed hospitalisations, which could contribute to $28 million saved over 10 years.
CONCLUSION : Consistent with clinical trials and other real-world findings, this large cohort study confirmed that NOAC reduced risk of stroke or SE, ICH/GI bleed and avoid all-cause mortality, and has potential to bring about cost savings to the healthcare system.
P75: Impact of High-Dose Vs Standard-Dose Influenza Vaccine on Respiratory Hospitalizations Among Adults
2:45PM - 3:00PM
Bianchini M 1 , Wright GC2 , Anderson HD2 , Perraillon MC2 , Lindrooth RC2 1 University of Colorado Anschutz, Denver, CO, USA, 2 University of Colorado Anschutz, Aurora, CO, USA
OBJECTIVES:
The high-dose (HD) influenza vaccine is approved for use in adults ages ≥65 with evidence for reducing influenza infections and hospitalizations. The objective of this study was to determine the impact of the HD vs standard-dose (SD) influenza vaccine on hospitalizations among adults ages 50-80.
METHODS:
A fuzzy regression discontinuity design was used to estimate the causal effect of the HD vaccine on respiratory hospitalizations. This design takes advantage of the discontinuity in likelihood of receiving the HD vaccine at age 65 to compare the outcomes of adults immediately above and below age 65. Data was extracted from IQVIA claims database for adults with an insurance claim for a HD or SD vaccine during the 2012-2018 influenza seasons (September-April). Outcomes and covariates were identified using International Classification of Diseases codes. The primary outcome was respiratory-related hospitalization. Covariates included demographics, comorbidities, and history of receiving the HD vaccine.
RESULTS:
The study included 384,180 individuals. The HD vaccine was used in 0.3% of adults 50-64 and 52% of adults 65 and older. Receipt of the HD vaccine decreased the probability of respiratory-related hospitalization by 0.5% points (95% CI -0.8%, -0.2%) compared to the SD vaccine (p=0.002) for adults who received HD because it was approved for their age group. Results were robust to various model specifications and sensitivity tests. CONCLUSIONS:
The HD vaccine reduced the rate of respiratory-related hospitalization compared to the SD vaccine among adults who received HD vaccine because of their age. The results suggest that extending approval to adults ages 50-64 would reduce respiratory-related hospitalizations among those who become newly eligible.
HEOR Studies in Medication Management
On-demand
P70: Evaluation of Cost and Health Utilization Outcomes of Medicare Beneficiaries Enrolled in a Medication Synchronization Program
2:00PM - 2:15PM
Waghmare P
OBJECTIVES:
Medication synchronization (med-sync) aligns patients’ chronic medications to a predetermined routine pickup date at a community pharmacy. An appointment-based model (ABM) med-sync service includes a comprehensive medication review or other clinical appointment at the pharmacy. We compared the cost and healthcare utilization outcomes of Medicare beneficiaries enrolled in an ABM med-sync program to beneficiaries not enrolled in such a program.
METHODS:
This retrospective cohort study included Medicare beneficiaries obtaining medications from pharmacies providing ABM med-sync. Medicare inpatient, outpatient, emergency, and pharmacy claims data from 2014 to 2016 were used to create med-sync (n=13,193) and non-med-sync (n=156,987) cohorts. All patients were followed longitudinally for 12 months before and after a 2015 index/enrollment date. Baseline characteristics including age, gender, race, geographical region, income-based enrollment status, copayment, and urban residence were utilized to create a logistic regression model for propensity score matching. A 1:1 greedy nearest neighbor matching algorithm was adapted for sequentially matching both cohorts. Difference-in-differences (DID) was used to compare mean changes in costs and utilization outcomes between cohorts.
RESULTS:
After matching, 13,193 beneficiaries in each cohort were used for analysis. Mean outpatient, emergency, pharmacy, and total costs increased before and after enrollment for both cohorts. No significant DID in costs were observed between cohorts. Healthcare utilization mean DID were significantly greater in the non-med-sync cohort compared to the med-sync cohort for outpatient visits (DID: 1.17, p<0.0001), emergency department visits (DID: 0.03, p=0.0372) and pharmacy fills (DID: 1.93, p<0.0001). There was no significant DID for inpatient visits between cohorts.
CONCLUSION: Outpatient, emergency, and pharmacy utilizations changes were significantly higher in the non-med-sync cohort compared to the med-sync cohort in the 12-months after enrollment. Lower pharmacy utilization could be due to optimization of therapy during medication reviews of ABM med-sync.
P69: Assessing Pharmacist-Administered Influenza Vaccinations and the Potential Impact of State Policies on Vaccination Rates: A Claims Data Analysis
2:15PM - 2:30PM
Tran J
Objective: Pharmacists have been permitted to immunize in all US states for over a decade, historically under enabling laws and varying requirements such as collaborative practice agreements (CPAs). This study aimed to characterize trends in influenza vaccinations over time and evaluate whether changes in policies related to CPAs impacted annual influenza vaccination rates. Methods: Real-world influenza vaccination trends were observed using IBM MarketScan administrative claims data for adults with employer-sponsored commercial plans for 2010 to 2019. We identified states with relevant policy changes using the National Association of Boards of Pharmacy (NABP) Surveys of Pharmacy Laws. Difference-in-differences analyses evaluated the effect of adding (Nebraska in 2017) or removing (Illinois in 2011, Montana in 2014) a CPA requirement on state annual influenza vaccination rates in the overall outpatient setting (i.e., pharmacy, physician offices, outpatient clinics) and in pharmacy only. These states were compared to similar states without CPA-related changes during this time period. Results : We identified 263,817 pharmacist-administered influenza vaccines in 2010, accounting for 9.1% of outpatient influenza vaccinations identified, and this reached 1.27 million by 2019, or 37.0% of outpatient influenza vaccinations. No significant effects of CPA-related policy changes were observed with vaccinations administered at pharmacies. However, the odds of outpatient influenza vaccination were 4% lower after Nebraska added CPA requirements (p=0.005) and 10% higher after Montana removed CPA requirements (p<0.001). No effects were seen in Illinois. Conclusion : The number and proportion of influenza vaccinations at pharmacies increased between 2010 to 2019 in the MarketScan commercial population. Although we found CPA-related changes impacted outpatient influenza vaccinations in two states, they did not affect vaccinations specifically administered in the pharmacy. Our study suggests CPAs may no longer have an enabling effect as vaccinations at pharmacies have become more common. Further research is needed to better understand their impact on outpatient vaccinations.
P71: Inpatient and Outpatient Medical Costs, Hospitalizations, and Length of Stay Associated with Adherence to Non-cycled Oral Antineoplastics Among Oncology Patients
2:30PM - 2:45PM
Staskon F
OBJECTIVES:
Increasing utilization of non-cycled antineoplastic therapies for a variety of cancers has generated interest in applying a proportion of days covered (PDC) adherence metric, and examining costs and health outcomes associated with adherent PDC (≥80%).
METHODS:
This retrospective study used MarketScan Commercial Claims and Encounters databases from 2017--2019. PDC was calculated for 2018 or 2019 utilizing non-cycled medications (monthly dosages for 31 products) from 15 therapeutic categories. New therapy/diagnosis indications were inferred from the 2017 files. Continuous enrollment was required as were ICD oncology diagnosis codes for those 18—65 years of age. Sample exclusion criteria were deaths, hospice care, inpatient transplant services, and females on fertility therapy. Models examined adherence level (PDC≥80% or not) and 9 covariates, with interaction terms, for significant associations on medical costs (inpatient and outpatient), hospitalizations, and length of days stay (LOS). Finally, for patients meeting 2019 PDC criteria, combined yearly results were modeled for associations.
RESULTS:
Of the 5,694 patients meeting 2018 PDC criteria, 2,034 retained a 2019 PDC, with adherence at 73.2% in 2018 and 67.9% in both years. In 2018, 34.8% were new to therapy/diagnosis and a mean age was 52.4 years. In 2018, 16.2% were hospitalized with LOS=9.5, and 24.1% were hospitalized in both years with LOS=8.8. Models for 2018 found significant effects favoring the adherent cohort with reduced medical costs (-$9,600, p<.0001), odds of hospitalization (0.72, p<.0007), and oncology-related LOS (-1, p<.02). Results for combined years indicated adherence effects for reduced medical costs (-$9,356, p<.0001), odds for hospitalization (0.6, p<.0002), and oncology-related LOS (-2.9, p<.0007). Significant covariate interactions are discussed in the poster.
CONCLUSION: Remaining adherent to oral antineoplastic therapy was associated with lower medical costs, fewer hospitalizations, and a shorter LOS, even across multiple years. These reductions were dependent on comorbidities, new to therapy/diagnosis, or metropolitan area.
P72: Designing a Value-Based Formulary for Kaiser Permanente Washington: A Hypothetical Case Study of Diabetes Mellitus Medications
2:45PM - 3:00PM
Chen Y 1 , Loucks AR2 , Sullivan SD1 , Pearson SD3 , Martin P2 , Yeung K2 1 Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, University of Washington, Seattle, WA, USA, 2 Kaiser Permanente Washington, Seattle, WA, USA, 3 Institute for Clinical and Economic Review, Boston, MA, USA
OBJECTIVES:
Value-based formulary (VBF) design sets lower copayments for pharmaceuticals with a high assessed value and higher copayments for drugs with a low assessed value. Previous VBFs focused on reducing cost-sharing without matching each drug's copayment to its assessed value. Our aim is to outline a novel VBF design for Kaiser Permanente Washington (KPWA) beneficiaries and estimate possible spending changes.
METHODS:
Formulary design exercise involved consultation with multiple stakeholders including the P&T chair, formulary design manager, clinical, and health economics experts. We designed a 4-tier VBF with exclusions based on incremental cost-effectiveness ratios (ICERs): Tier 1: ICER ≤$50,000, with zero cost-sharing. Tier 2: ICER ≤$100,000, with cost-sharing of $30. Tier 3: ICER ≤$150,000, with cost-sharing of 10% and no ability to apply coupons. Tier 4: ICER ≤$200,000, with cost-sharing of 30% and no coupons. Excluded drugs: ICER >$200,000 or dominated. We assessed value of each drug primarily using cost-effectiveness analysis from available Institute for Clinical and Economic Review evidence reports. We aligned drug prices with KPWA prices for 23 diabetes mellitus (DM) medicines to derive plan-specific incremental cost per QALY gained estimates. Using pharmacy claims (2019-2020), we identified a cohort of 40,150 beneficiaries who were on the included DM medicines. We forecasted future health plan spending and out-of-pocket (OOP) costs with VBF, using published own-price elasticity estimates.
RESULTS:
The average age of the cohort was 55, and 51% of them were female. When compared to the traditional formulary, the VBF is estimated to reduce total annual health plan spending by 10%, saving $87 in annual spending per member and $91 in annual OOP spending per member. Sensitivity analyses using various price elasticity values showed declines in all spending outcomes.
CONCLUSIONS:
Designing a VBF in a U.S. employer-based health plan has the potential to save money for both the plan’s and the patient's OOP expenses.
Cost Effectiveness Analysis in Oncology Studies
On-demand
Moderator
Koen Degeling, PhD, MSc, BSc
Lumen Value & Access – a Healthcare Consultancy Group Company, New York, NY, USA
Dr Koen Degeling is a Research Scientist, Health Economic Modelling & Advanced Analytics at Lumen Value & Access, a Healthcare Consultancy Group company. He was trained as an Industrial Engineer specializing in Healthcare Technology and Management and holds a PhD in Advanced Health Economic Modelling from the University of Twente in the Netherlands. Prior to joining Lumen Value & Access, Koen worked on real-world data-driven health economic and health services research projects at the Cancer Health Services Research department of the University of Melbourne in Australia, where he continues to be involved as an honorary fellow. He is an active ISPOR member and currently serves on the Editorial Advisory Board for Values & Outcomes Spotlight and ISPOR New Professionals Steering Committee, is involved in several short courses and workshops, and has served as global chair, committee co-chair, and chapter president within the ISPOR Student Network.
P64: Cost-Utility Analysis Comparing Mastectomy and Lumpectomy in Early Stage Breast Cancer Surgeries in Sweden - a Life-Time Approach Using Markov Modelling
2:00PM - 2:15PM
Pham PD
Background: Breast cancer is the most common cancer type among Swedish women. Although the treatments of breast cancer has developed dramatically over years, there lacks health economics assessments on these treatments, especially cost-utility analysis. This study performed a cost-utility analysis comparing three available surgical procedures, including mastectomy, lumpectomy without irradiation and lumpectomy with irradiation in Swedish context. Methods: A 6-state Markov model with 20-year time horizon was used to investigate the cost-utility of three alternatives. Transition probabilities parameters were obtained based on the best available evidence in Sweden and similar contexts. Both healthcare and societal perspectives were considered in cost estimation. An individual dataset from Federation of Swedish county councils and municipalities (SKL) was used to calculate costs. Quality-adjusted life years (QALYs) were used to calculate utility, based on a study in a similar context of Norway due to the lack of utility evidence in Sweden. Deterministic univariate and multivariate sensitivity analysis were performed to handle uncertainties. Results: Lumpectomy with irradiation dominated two other options and was the most cost-effective treatment option for early stage breast cancer in Sweden. Moving from mastectomy to lumpectomy without irradiation, the incremental cost-effective ratio (ICER) was 153,350 SEK in healthcare perspective and 32,017 SEK in societal perspective, per QALY gained. This ICER should still be considered as cost-effective in Swedish context. Conclusions: This study revealed the cost and utilities in life-time approach of three surgical procedures in early stage breast cancer treatments in Sweden, namely mastectomy, lumpectomy without irradiation and lumpectomy with irradiation. The results showed that lumpectomy with irradiation appeared to be the most cost-effective option during 20-year follow-up in both healthcare and societal perspectives. Future studies should be expanded to estimate the more reliable parameters in Sweden to build up the consistency of our findings.
P61: Cost Utility Analysis of Circulating Tumour DNA Guided Adjuvant Chemotherapy in Stage II Colon Cancer
2:15PM - 2:30PM
To YH 1 , Degeling K2 , Kosmider S3 , Wong R1 , Lee M1 , Dunn C1 , Gard G1 , Jalali A1 , Wong V1 , IJzerman M2 , Gibbs P1 , Tie J1 1 Walter and Eliza Hall Institute of Medical Research, Parkville, VIC, Australia, 2 The University of Melbourne, Melbourne, VIC, Australia, 3 Western Health, Footscray, VIC, Australia
Background: There is currently potential overuse of adjuvant chemotherapy (AC) in patients with stage II colon cancer (CC) given the uncertain survival benefit in unselected patients. A circulating tumour DNA (ctDNA) approach has the ability to improve patient selection for AC, defining patients who may benefit from treatment (ctDNA positive) and those who will not (ctDNA negative). This study aimed to estimate the health and economic impact of ctDNA-guided prescription of AC for stage II CC. Methods: A cost-utility analysis was performed comparing ctDNA-guided AC prescription for stage II CC to standard of care (SOC), where 22.6% of SOC patients received AC, all ctDNA-positive patients (8.7%) received AC and all ctDNA-negative patients (91.3%) did not. A third preference-sensitive ctDNA strategy was included where 6.8% of ctDNA-negative patients would receive AC to reflect potential non-compliance. A state-transition model was populated utilising a landmark cohort study investigating the prognostic value of ctDNA and clinical registries. The analysis employed an Australian payer perspective and lifetime horizon. Extensive scenario and probabilistic analyses quantified model uncertainty. Results: Compared to SOC, the ctDNA and preference-sensitive ctDNA strategies increased quality-adjusted life years (QALYs) by 0.20 (95% confidence interval -0.40 to 0.81) and 0.19 (-0.40 to 0.78), and resulted in incremental costs of AUD - 4,215 (-17,651 to 9,216) and AUD - 2,450 (-15,472 to 10,570), respectively. ctDNA remained cost-effective at a willingness-to-pay of AUD 20,000 per QALY gained throughout most scenario analyses in which the proportion of ctDNA-positive patients cured by AC and compliance to a ctDNA negative test results were decreased. Conclusions : ctDNA-guided AC is a potentially cost-effective strategy to improve patient selection for adjuvant chemotherapy in resected stage II colon cancer. Expanding the analysis with results from ongoing randomised clinical studies will be important to reduce uncertainty in the model.
P62: Cost-Effectiveness of Atezolizumab Plus Cobimetinib and Vemurafenib in the Treatment of BRAF V600 Mutation-Positive Metastatic Melanoma
2:45PM - 3:00PM
Cai C 1 , Yunusa I2 , Tarhini A3 1 University of South Carolina, Columbia , SC, USA, 2 University of South Carolina, Columbia, SC, USA, 3 University of South Florida Morsani College of Medicine, Tampa, FL, USA
OBJECTIVES:
To evaluate the cost-effectiveness of atezolizumab and vemurafenib plus cobimetinib vs. vemurafenib plus cobimetinib alone in patients with newly diagnosed unresectable BRAF V600 mutated metastatic melanoma from the US healthcare perspective.
METHODS:
This economic evaluation study used a three-state partitioned survival model to assess the cost-effectiveness of the triplet combination of PD-L1 inhibitor atezolizumab plus BRAF inhibitor vemurafenib plus MEK inhibitor cobimetinib. The observed overall survival curves and progression free survival curves were digitized from the IMspire150 trial (January 2017 – April 2018) and the long-term survivals (over a life-time horizon) beyond the end of the study were extrapolated using seven different survival models. Life-years (LYs) gained and quality-adjusted life-years (QALYs), costs, and incremental cost-effectiveness ratio (ICER) expressed as cost per LYs and per QALYs saved were estimated. The cost and health preference data were collected from a literature review.
RESULTS:
Adding atezolizumab to vemurafenib and cobimetinib provided an additional 3.267 QALYs compared to doublet regimen of vemurafenib plus cobimetinib, at an ICER of $271,669/QALY, which is not considered cost-effective at the willingness-to-pay (WTP) threshold of $150,000/QALY. However, the scenario analyses found that atezolizumab combined with vemurafenib plus cobimetinib could be cost-effective at a 20-year (ICER, $121,432/QALY) and 30-years ($98,092/QALY) time horizons when both strategies were stopped after two years of treatments, and over a lifetime horizon ($122,220/QALY) when immunotherapy was stopped after two years of treatment.
CONCLUSIONS:
Combining immunotherapy with targeted therapies can lead to significant survival benefit for patients with BRAF V600 mutated advanced melanoma but was not cost-effective at a WTP value of $150 000/QALY. Adding the immunotherapy to the targeted therapies could be cost-effective over a lifetime horizon if long-lasting immunotherapeutic effect was sustainable and immunotherapy was stopped after 2 years in the absence of disease progression.
P63: Cost Effectiveness of Cabozantinib Plus Nivolumab As First-Line Treatment for Renal Cell Carcinoma
2:30PM - 2:45PM
Marciniak A 1 , Gultyaev D2 , Obrzut G3 , Mollon P1 , Wallace JF4 1 Ipsen, Boulogne-Billancourt, France, 2 Certara, Loerrach, BW, Germany, 3 Certara, Krakow, Poland, 4 Exelixis, Inc., Alameda, CA, USA
OBJECTIVE
The combination of cabozantinib/nivolumab (CaboNivo) was approved in 2021 for the first-line (1L) treatment of adults with advanced renal cell carcinoma (aRCC) based on the results of the CheckMate 9ER trial (NCT03141177). We conducted a cost-effectiveness analysis of CaboNivo versus alternative 1L aRCC therapies.
METHODS :
A global cost-effectiveness model was developed in Microsoft Excel® using a partitioned survival model and deterministic and probabilistic frameworks to estimate treatment-specific effectiveness (life years, quality-adjusted life years [QALYs]) and costs. The model comprised three mutually-exclusive health states: progression free, progressed disease and death. Under a lifetime horizon (50-years), patients entered the model in the progression-free state and received 1L CaboNivo or an alternative tyrosine kinase inhibitor (TKI; cabozantinib, pazopanib, temsirolimus, tivozanib, sorafenib, sunitinib) or combination (axitinib/avelumab, axitinib/pembrolizumab, ipilimumab/nivolumab, lenvatinib/pembrolizumab). Treatment effect estimates (overall survival, progression-free survival) were derived from a network meta-analysis using published data for all treatment alternatives, adjusted for the prognostic profile of the source populations. Following 1L treatment discontinuation (progressed-disease state), patients received subsequent therapy, with treatment distribution derived from clinical studies. Adverse events were incorporated as one-off events during the state associated with cost and utility decrement. Health state utilities were derived from EQ-5D data from the CheckMate 9ER trial. France was used as the base-case.
RESULTS:
CaboNivo’s effectiveness was similar to that of ipilimumab/nivolumab (7.4 life years and 5.4 QALYs for both) and it was more effective than alternative TKIs (life-year range, 5.1–6.2; QALY range, 3.8–4.6) and combinations (life-year range, 6.3–7.1; QALY range, 4.7–5.2). CaboNivo was more costly than all TKI monotherapies (total cost EUR 248,369 vs EUR 54,278–174,112), but similar to other combination approaches (total cost EUR 187,276–387,010).
CONCLUSIONS:
Cost Effectiveness Analysis of Emerging Therapies/Technologies
On-demand
Moderator
Vijay GC, PhD
University of York, Heslington, York, YOR, United Kingdom
P65: Cost-Effectiveness of Every Two Month Cabotegravir Long-Acting (CAB-LA) Compared with Daily Oral Emtricitabine (FTC)/Tenofovir Disoproxil Fumarate (TDF) for PRE-Exposure Prophylaxis (PREP) to Prevent HIV-1 Infection in the United States
2:00PM - 2:15PM
Davis A 1 , Brogan A2 , Mellott CE1 , Fraysse J3 , Oglesby A4 1 RTI Health Solutions, Research Triangle Park, NC, USA, 2 RTI Health Solutions, San Diego, CA, USA, 3 ViiV Healthcare, Amsterdam, Netherlands, 4 ViiV Healthcare, RTP, NC, USA
OBJECTIVE: CAB-LA administered every two months was recently approved in the US as PrEP for individuals at risk of acquiring HIV-1 infection based on the HPTN 083 and 084 studies, which both demonstrated superior reduction in HIV-1 acquisition compared with daily oral FTC/TDF in men who have sex with men (MSM), transgender women (TGW), and cisgender women. A decision-analytic model was developed to assess the lifetime cost-effectiveness of CAB-LA compared with FTC/TDF for HIV-1 PrEP in the US.
METHODS:
The modeled population included individuals aged ≥18 years eligible to receive PrEP. Individuals entered the Markov model receiving either CAB-LA or FTC/TDF and could continue initial PrEP or choose alternate PrEP options or no PrEP over time. Efficacy was taken from the HPTN 083 and 084 clinical trials and adjusted for adherence and persistence. If HIV seroconversion occurred, individuals discontinued PrEP and were assumed to receive antiretroviral treatment and other associated HIV-related care for life. Secondary HIV-1 transmission to partners could also occur and, rarely, PrEP-related breakthrough resistance. Utility decrements and costs for PrEP and HIV-related care were obtained from published sources. The model estimated lifetime costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs) from a healthcare sector perspective with costs and health outcomes discounted at 3% annually. Sensitivity analysis was conducted to assess the impact of varying population subgroups, the comparator, high-risk duration, and PrEP adherence and persistence.
RESULTS:
The model estimated that CAB-LA prevented more primary and secondary HIV-1 infections than FTC/TDF and yielded 0.21 fewer QALYs lost. Additional lifetime costs were $7,853, resulting in an incremental cost of $37,333 per QALY gained. For all scenarios analyzed, CAB-LA generally remained cost-effective or cost-saving with all ICERs at or below $107,693.
CONCLUSIONS:
For populations at risk for HIV-1, CAB-LA for PrEP represents a cost-effective alternative to daily oral FTC/TDF.
P66: Cost-Effectiveness of the Chest Pain Choice Decision Aid Versus Usual Care Among Low-Risk Chest Pain Patients Presenting to the Emergency Department
2:30PM - 2:45PM
Dhatt H 1 , Hurwitz JT2 , Axon D2 , Warholak T2 , Slack M2 , Grizzle AJ2 1 University of Arizona (time of research) | Janssen (current), Scottsdale, AZ, USA, 2 University of Arizona, Tucson, AZ, USA
OBJECTIVES: This study aimed to evaluate the cost-effectiveness of outpatient follow-up after using the Chest Pain Choice decision aid (CPC-DA) compared to usual care in low-risk chest pain patients who presented to the emergency department (ED).
METHODS: A decision analytic model compared costs and effectiveness of the CPC-DA and usual care from a payer perspective. Effectiveness was based on published randomized controlled trial data comparing the CPC-DA with usual care across six EDs in the United States. Patients (N=898) presenting with chest pain and considered for admission to an observation unit for cardiac testing were included in the original trial. The model assessed 30-day probability of events and costs for outpatient follow up, ED-Observation Unit (ED-OU) admission, and cardiac events in and out of hospital. The measure of effect was defined as patients discharged to outpatient follow-up with no cardiac events. Costs reflect 2021 US dollars, and the incremental cost-effectiveness ratio (ICER) represents the cost per admission averted to ED-OU without cardiac events. One-way deterministic sensitivity analysis and probabilistic sensitivity analysis using Monte Carlo simulation were conducted.
RESULTS: The CPC-DA group yielded an expected cost of $3,917.58 and effect of 0.59 (i.e., 59% discharged to outpatient follow-up without a cardiac event), while the usual care group had an expected cost of $4,676.28 and effect of 0.40. The incremental cost of -$758.69 and effect of 0.19 indicates that outpatient follow-up stemming from CPC-DA is a dominant strategy compared to that from usual care. These findings were robust to sensitivity analyses.
CONCLUSIONS: The CPC-DA, designed to help patients and clinicians make a shared and informed decision regarding whether to be admitted to the ED-OU or discharged to outpatient follow-up for further cardiac testing and evaluation, can be cost-effective compared to usual care among low-risk chest pain patients presenting to the ED.
P68: Cost-Effectiveness of Ofatumumab in Comparison with Teriflunomide for Relapsing Multiple Sclerosis Treatment in China: From Both Healthcare System and Societal Perspectives
2:45PM - 3:00PM
Li H1 , Jia Y 2 , HU M1 1 Fudan University, Shanghai, China, 2 Fudan University, Shanghai, 31, China
OBJECTIVES:
Multiple Sclerosis (MS) imposes a long-term medical and financial burden on patients and families, resulting in substantial social burden due to its disabling outcomes. Disease-modifying treatments (DMT) have been recommended as the standard therapy in controlling disease progression, of which teriflunomide is one of the most common DMT drugs in China, and ofatumumab is the only highly effective DMT available in China. Data on the cost-effectiveness of ofatumumab are limited. This study aimed to perform an economic evaluation of ofatumumab versus teriflunomide from healthcare system and societal perspectives.
METHODS:
An 11-states Markov model, based on patient's Expanded Disability Status Scale score, was constructed to simulate a hypothetical cohort of 1,000 patients. Patient demographics were derived from the ASCLEPIOS clinical trial. The time horizon was average life expectancy. The transition probability of states, clinical effectiveness, safety data, and utility data were obtained from the literature and meta-analysis. Direct and indirect costs were calculated by combining resource utilization captured in the 2021 China MS Disease Burden study, and unit expenditure was derived from the Shanghai Healthcare Commission Information Center. Costs and quality-adjusted life-years (QALYs) were discounted by 5% annually. The incremental cost-effectiveness ratio (ICER) was calculated and a threshold of 1 to 3 times GDP per capita in China was applied.
RESULTS:
From the healthcare system perspective, compared with teriflunomide, the incremental cost of treating with ofatumumab was 95,961 RMB (14,874 USD), and patients treated with ofatumumab gained 0.685 QALYs. The ICER was 140,038 RMB/QALY (21,706 USD/QALY). From the societal perspective, ofatumumab was dominant, with increased QALYs and lower lifetime costs.
CONCLUSIONS:
From the healthcare system perspective, ofatumumab is cost-effective at the threshold of 2-times GDP per capita in China. When considering productivity loss, ofatumumab can reduce social burden and be both clinically superior and cost saving.
P67: Short-Term Cost-Effectiveness of a Remote Robotic Mechanical Thrombectomy System
2:15PM - 2:30PM
Boltyenkov A1 , Sanmartin M 2 , Sangha K3 , Wang J4 , Bastani M4 , Katz JM4 , Pandya A5 , Sanelli PC4 1 Siemens Healthineers, Newark, DE, USA, 2 Siemens Healthineers, Malvern, PA, USA, 3 Siemens Healthineers, Roseville, CA, USA, 4 Northwell Health, Manhasset, NY, USA, 5 Harvard University, Boston, MA, USA
OBJECTIVES:
Mechanical thrombectomy (MT) is a treatment for acute ischemic stroke (AIS). The clinical effect of MT for AIS patients suffering a large-vessel occlusion, is highly time-dependent. Patients admitted to a primary stroke center (PSC) currently must be transferred to a comprehensive stroke center (CSC) for a MT. A remote robotic MT system has the potential to disrupt the acute stroke care pathway by allowing the performance of MT at a PSC by an interventional radiologist physically located in a CSC.
METHODS:
We developed a discrete event simulation (DES) model to simulate the stroke treatment of AIS patients to evaluate the short term costs and quality-adjusted life-years (QALY) within the first 90 days. Cost components included in the model were acute care costs and treatment procedure costs. No long term or chronic care costs were included. We performed probabilistic sensitivity analyses (PSA) to assess the uncertainty of model results.
RESULTS:
Baseline results indicated that in the short term remote robotic MT is both more costly (by $1,259) and more effective (by 0.01 QALY) than patient transfer from PSC to CSC for MT procedure, corresponding to incremental cost-effectiveness ratio (ICER) of $148,202
. In the 10,000 1-st order simulation trials the remote robotic MT strategy led to 2% (31/1521) fewer deaths, 1.8% (62/3533) more functionally independent patients (modified Rankin Score (mRS) 0-2 at 90 days), and 0.6% (31/4946) fewer functionally dependent patients (mRS 3-5 at 90 days). Robotic MT was cost-effective in 5% and 53% of PSA iterations using cost-effectiveness threshold of $100,000/QALY and $150,000/QALY, respectively.
CONCLUSIONS:
Remote robotic MT saves lives, increases the number of patients with functional independence, and decreases the number of patients with functional dependence. If only the short term cost and health effects are considered, the ICER is above the $100,000 threshold, but below the $150,000 threshold.
Analytic Studies in Patient-Centered Research
On-demand
Moderator
Elisabeth Oehrlein, PhD, MS
Applied Patient Experience, LLC, Washington, DC, USA
Elisabeth M. Oehrlein, Ph.D., MS, is Assistant Vice President, Research & Programs, at the National Health Council, joining the organization in July 2018. Dr. Oehrlein is a mixed-methods researcher with expertise in value/health technology assessment, outcomes research, and patient-focused medical product development. Her research interests include patient journey/experience mapping and applying patient experiences when developing real-world research to ensure studies reflect the “real world” as closely as possible. She is an active member of HTAi’s Patient and Citizen Involvement Group, as well as the International Society for Pharmacoeconomics and Outcomes Research, where she holds leadership roles in the Patient-Centered and Real-World Evidence Special Interest Groups. She has published widely in medical, economic, and health policy journals and serves as an Associate Editor of Value in Health.
Dr. Oehrlein holds a BA from Franklin & Marshall College, an MS in Epidemiology from the University of Maryland School of Medicine’s Department of Epidemiology and Human Genetics, and a Ph.D. in Pharmaceutical Health Services Research from the University of Maryland School of Pharmacy.
P52: Mapping CHU9D Utility Scores from the PEDSQLTM for Children with Chronic Conditions in an Ethnically Diverse and Deprived Metropolitan Population
2:15PM - 2:30PM
Soley-Bori M 1 , Kelly C2 , Lingam R3 , Forman J4 , Cecil L4 , Newham J5 , Wolfe I4 , Fox-Rushby J6 1 King's College London, LONDON, LON, UK, 2 Queen's University Belfast, Belfast, UK, 3 UNSW, South Wales, NSW, Australia, 4 King's College London, London, HI, UK, 5 Northumbria University, Newcastle upon Tyne, UK, 6 King's College London, London, UK
OBJECTIVES: The Child Health Utility 9D (CHU9D) is a paediatric preference-based questionnaire to measure health related quality of life (HRQoL) commonly used in cost-utility analyses. When not available, mapping algorithms between the Paediatric Quality of Life InventoryTM (PedsQL) into the CHU9D exist. Current mappings are based on populations with limited age ranges and medical conditions and, thus, require further external validation. This study externally validates the most recent PedsQL to CHU9D mappings and develops new mappings based on a sample with a wide age range (0 to 16 years of age) and chronic conditions (asthma, eczema, or constipation).
METHODS: Data from the Children and Young People’s Health Partnership (CYPHP) Evelina London Model of Care, a new integrated paediatric healthcare delivery model, was used (N=1,775 participants). PedsQL responses were imputed into existing mappings and predicted CHU9D values obtained and compared to observed values based on goodness of fit measures (R-squared, mean absolute errors-MAE, mean squared errors, and the percentage of observations with an absolute error smaller than 0.05). To develop new mapping algorithms, the study sample was randomly divided into estimation (80%) and validation (20%) groups.
RESULTS: Existing mappings show an acceptable performance in the CYPHP sample. All MAEs (lower values indicate better fit) are between 0.056 (5-7 years, dimension score) and 0.11 (multimorbidity, total score) and, thus, within the lower bound of previously published estimates, which ranged from 0.074 to 0.230. The new CYPHP mappings perform better for the study sample compared to previous versions.
CONCLUSIONS: The new CYPHP mapping algorithms can predict CHU9D scores from PedsQL scores with good accuracy. These algorithms outperformed those also externally validated in this study. The CYPHP mappings are particularly relevant for samples with children and young people with chronic conditions living in deprived and urban settings. Further validation in an external sample is needed.
P51: Osteoarthritis Affecting Health-Related Quality of Life Among Older Patients
2:30PM - 2:45PM
Basu S1 , Vivek V1 , Kathe N2 , Agrawal N 3 1 Complete HEOR Solutions (CHEORS), North Wales, PA, USA, 2 Complete HEOR Solutions (CHEORS), North Wales, CA, USA, 3 Complete HEOR Solutions (CHEORS), Philadelphia, PA, USA
OBJECTIVES:
Osteoarthritis is a common subtype of musculoskeletal disorder (MSD) among older adults. It is associated with increased swelling, inflammation, decreased range of motion, and joint instability with significant implications on health-related quality of life (HRQoL). This study compared HRQoL of patients with osteoarthritis and other types of MSDs
. METHODS:
The nationally representative study sample was selected from the 2011-2019 Medical Expenditure Panel Survey (MEPS). This cross-sectional study compared the Physical Component Summary (PCS) and Mental Component Summary (MCS) scores of osteoarthritis patients (ICD10 M15-M19 and ICD9 715-716) and other MSDs patients 50 years and older of age.
An inverse probability of treatment weighted (IPTW) regression analysis was conducted to control for sociodemographic and clinical factors to assess the marginal HRQoL burden associated with osteoarthritis compared to other MSDs. RESULTS:
The
study included 19,677 unweighted patients representing 214 million osteoarthritis patients and 46,114 unweighted patients representing 507 million other MSD patients. Most of the patients with osteoarthritis were non-Hispanic white (69.6%), female (62.4%),
and working in white-collar jobs (45.5%). The mean PCS and MCS were 36.17 (SE:0.19) and 46.60 (SE:0.19), respectively, in osteoarthritis patients, while in the other MSD patients, the PCS and MCS scores were 40.28 (SE:0.17) and 46.50 (SE:0.16), respectively. The IPTW regression analysis found that osteoarthritis patients had a 4.87 (SE:0.30) unit lower PCS score compared to patients with other MSD (43.03 (SE:0.20)). H owever, the MCS score did not differ between osteoarthritis patients and other MSD patients. CONCLUSIONS:
P49: Predictors of Symptom Worsening or Improvement Using Remote Patient Reported Outcomes (PRO) Technology
2:00PM - 2:15PM
Wujcik D 1 , Dudley W2 1 Carevive Systems, Inc, FRANKLIN, TN, USA, 2 University of North Carolina, Greensboro, Greensboro, NC, USA
OBJECTIVES: Symptom burden for patients with cancer is substantial, yet proactive symptom reporting and management can decrease hospitalizations/ED visits and improve QOL. This research aims to identify symptom patterns and predictors leading to an automated alert of severe symptoms (Worsening) and symptom resolution after the alert (Improvement) in patients using remote symptom management technology.
METHODS: Patients undergoing cancer treatment completed weekly surveys using remote reporting technology with PRO-CTCAE derived symptom assessments. Consensus scores are calculated and severe reports trigger automated care team alerts. Pain trajectories of Worsening and Improving were examined using cluster analysis to extract patient subgroups followed by multinomial logistic regression predictors of cluster membership. Regression analyses were also employed to examine change scores (Worsening = Alert - Pre-Alert and Improvement = Alert – Post Alert).
RESULTS
: Data from 328 patients, over an average 10.5 (SD=8.8) weeks, showed 270 (82%) experienced symptoms that generated at least one alert. Patients were 86% female, 73% white, and average age 58.65 (SD=13.26). Fitness was captured using a modified geriatric assessment (mGA) with 67% Fit; 21% Intermediate, 12% Frail. Symptoms generating alerts were pain (.60), insomnia (.40), constipation (.37), decreased appetite (.36), fatigue (.34), nausea (.31), neuropathy (.27), and diarrhea (.22). Three subgroups patterns of change trajectories were identified using two-step cluster analysis. Cluster membership was not predicted by age, gender, race, or MGA. Regression analyses of Worsening showed that age was a significant positive predictor of worsening - older patients had sharper trajectories of worsening prior to the trigger. mGA was a significant predictor of Improvement with more frail and non-white patients showing sharper Improvement.
CONCLUSIONS
: The results may be used to identify at risk patients who will benefit from intervention at less severe reports of symptoms and those who improve more quickly. Further studies will expand understanding of change in PRO's and implications for patient care.
P50: Comparison of the EQ-5D-5L and Aqol-8D in a Cohort of People with Idiopathic Pulmonary Fibrosis in Australia
2:45PM - 3:00PM
Cox I 1 , Campbell JA2 , de Graaff B2 , Otahal P3 , Corte TJ4 , Moodley Y5 , Hopkins P6 , Macansh S7 , Walters HE2 , Palmer AJ2 1 Menzies Institute for Medical Research, University of Tasmania, Hobart, TAS, Australia, 2 University of Tasmania, Hobart, TAS, Australia, 3 University of Tasmania, Hobart, NSW, Australia, 4 The University of Sydney, Sydney, NSW, Australia, 5 The University of Western Australia, Perth, NSW, Australia, 6 University of Queensland, Brisbane, NSW, Australia, 7 Lung Foundation Australia, Camperdown, NSW, Australia
Background Idiopathic pulmonary fibrosis (IPF) is a debilitating chronic lung disease with a high symptom burden, which has a substantial impact on health-related quality of life (HRQoL). Our study aimed to assess the performance of the EuroQol five-dimension (EQ-5D-5L) and the Assessment of Quality of Life- eight-dimension (AQoL-8D) questionnaires in measuring HRQoL as utility values (HSUVs) in an Australian IPF cohort. Methods Data were collected from participants of the Australian IPF Registry (AIPFR) using self-administered surveys which included the EQ-5D-5L and the AQoL-8D. Clinical data and disease specific HRQoL instruments were collected from the AIPFR. Performance of the two instruments was evaluated based on questionnaire practicality, agreement between the two instruments and test performance (internal and construct validity). Results Overall completion rates for the EQ-5D-5L and AQoL-8D were 96% and 85% respectively. Mean (median) HSUVs were 0.65 (0.70) and 0.69 (0.72) for the EQ-5D-5L and AQoL-8D respectively. There was reasonable agreement between the two instruments based on the Bland-Altman plot mean difference (-0.04) and intraclass correlation coefficient (0.84), however there were some differences. A larger range of values was observed with the EQ-5D-5L when compared to the AQOL-8D (-0.57-1.00 vs 0.16-1.00). The EQ-5D-5L had a greater divergent sensitivity and efficacy in relation to assessing HSUVs between clinical groupings. The AQoL-8D however had a higher sensitivity to measure psychosocial aspects of HRQoL in IPF. Conclusion The EQ-5D-5L exhibited better performance when compared to AQoL-8D in persons with IPF. This may be attributable to the high symptom burden which is physically debilitating to which the EQ-5D-5L may be more sensitive.
Applying Disparate Data Sources and Methods for Evaluating Real-World Evidence
On-demand
Moderator
Anna Hung, PharmD, PhD, MS
Duke University School of Medicine, Durham, NC, USA
Anna Hung, PharmD, PhD, MS is a pharmacist and health services researcher interested in payer and patient decision making related to pharmacy benefits. Her methodological research interests include health care cost evaluations, quasi-experimental study designs, and stated preference research.
P57: Reasons for Treatment Changes in Children and Adolescents with Attention-Deficit/Hyperactivity Disorder (ADHD): A Chart Review Study
2:15PM - 2:30PM
Schein J 1 , Cloutier M2 , Gauthier-Loiselle M2 , Bungay R2 , Guérin A2 , Childress A3 1 Otsuka Pharmaceutical Development & Commercialization, Inc., Princeton, NJ, USA, 2 Analysis Group, Inc., Montreal, QC, Canada, 3 Center for Psychiatry and Behavioral Medicine, Las Vegas, NV, USA
Objective: To examine the reasons underlying treatment changes among pediatric patients with ADHD. Methods: Data were obtained through online medical chart abstraction (August–September, 2021). Eligible patients with ADHD had initiated a treatment regimen at ages 6–17 and within 1–5 years of chart abstraction. Reasons contributing to treatment discontinuation were analyzed for a randomly selected treatment episode. ADHD/treatment-related complication rate was also described. Physicians’ perspective on adherence to ADHD treatment was assessed through an online survey. Results were reported overall and among children (ages 6–12) and adolescents (ages 13–17), separately. Results: A total of 156 physicians abstracted 434 patient charts (235 children; 199 adolescents). Mean patient age was 11.3 years and 68.7% were male. Treatment regimens analyzed included stimulants (83.2%), nonstimulants (11.3%), and combination therapy (5.1%); average treatment duration was 23.3 months. Among patients who discontinued treatment (N=83), inadequate/suboptimal symptom management (60.2%) was the most common reason for discontinuing treatment, while 25.3% reported a treatment discontinuation due to ADHD/treatment-related complications. The most common ADHD/treatment-related complications leading to treatment discontinuation were anxiety (19.0%), insomnia/sleep disturbances (19.0%), and emotional impulsivity (19.0%). Overall, 42.4% of patients had ≥1 documented ADHD/treatment-related complication, and this proportion reached 54.5% among patients receiving combination therapy. Insomnia/sleep disturbance was the most common ADHD/treatment-related complication and occurred in 9.7% of patients. Notably, 75.5% of patients reported the experience or fear of complications had a negative impact on their adherence to ADHD treatment. Physicians reported taking actions toward patients’ non-adherence by further educating patients (81.0%), closer monitoring (59.9%), and changing the prescribed ADHD medication (38.1%). Results were similar among children and adolescents. Conclusion: Lack of effectiveness and ADHD/treatment-related complications are important reasons for treatment changes among children and adolescents with ADHD, highlighting the need for more effective and tolerable treatments to mitigate the burden of ADHD.
P60: The Association between Statin Use and Severe Outcomes in COVID-19 Infected Beneficiaries Enrolled in Mississippi Medicaid
2:45PM - 3:00PM
Rong Y 1 , Pittman E2 , Ramachandran S3 , Bentley J3 , Banahan III B4 , Kirby T5 , Smith D5 , Bhattacharya K6 1 University of Mississippi, Amherst, MA, USA, 2 University of Mississippi, University, MS, USA, 3 University of Mississippi, Oxford, MS, USA, 4 Center for Pharmaceutical Marketing and Management, University of Mississippi, University, MS, USA, 5 Office of the Governor, Mississippi Division of Medicaid, Jackson, MS, USA, 6 Center for Pharmaceutical Marketing and Management, University, MS, USA
Objective: Previous research suggests that the use of statin is associated with lower risk of severe outcomes among patients infected with the SARS-CoV-2 virus, but this has not been evaluated in a low-income Medicaid population. This study assessed the association between antecedent statin use and COVID-related hospitalization and all-cause death in Medicaid-enrolled patients with COVID-19. Methods: Beneficiaries with antecedent statin use were identified based on records of statin prescription in the 90-day prior to the index date through pharmacy claims. Outcomes were all-cause mortality and hospitalization in 30 days, 60 days, and 90 days post index. Propensity score matching was performed to match statin users with non-users 1:1, based on age, sex, race, comorbidities, and medication use. Multivariable conditional logistic regression was conducted, adjusting for index month, cancer diagnosis and long-term care residency, to estimate the association of statin use with outcomes of interests. Results: A total of 10,792 beneficiaries met the inclusion criteria with 1,415 (13.1%) statin users. A total of 2,214 beneficiaries were included in the matched cohort. Unadjusted rates of COVID-related hospitalization and all-cause death in the matched cohort were 21.9% and 4.8% for statin users, and 21.5% and 7.4% for statin non-users, respectively. Multivariable logistic regression showed no significant difference in odds of hospitalization for matched statin users and non-users. Statin users have lower odds of mortality within 30 days (OR:0.51, 95%CI:0.32-0.83), 60 days (OR:0.56, 95%CI:0.37-0.85) and 90 days (OR:0.55, 95%CI:0.37-0.82) after diagnosis of COVID-19. Conclusion: COVID-19 beneficiaries with antecedent statin use might have lower odds of death after COVID infection.
P58: Measuring the Real-World Impact of Subsidy Decision on Clinical Practice and Patient Outcomes: Post-Subsidy Evaluation of Sofosbuvir/Velpatasvir for Treating Chronic Hepatitis C
2:00PM - 2:15PM
Luo XS 1 , Ong SKB1 , Tan CK2 , Lin L1 , Ng KH1 1 Agency for Care Effectiveness, Ministry of Health, Singapore, Singapore, 2 Department of Gastroenterology and Hepatology, Singapore General Hospital, Singapore, Singapore
OBJECTIVES
: Sofosbuvir/velpatasvir was recommended for subsidy to treat chronic hepatitis C in Singapore in 2018. This study measured the impact of subsidy listing on clinical practice and patient outcomes. Specifically, we looked at (1) pre- and post-subsidy changes in utilisation and prescribing pattern, (2) real-world clinical effectiveness and safety outcomes, and (3) potential long-term impact on population health and the healthcare system.
METHODS:
Utilisation trends and prescribing patterns were assessed using aggregated drug utilisation data from public hospitals' dispensing systems and clinical data from the national electronic health record database. A single-cohort prospective study was conducted to evaluate sustained virological response rate 12 weeks post-treatment (SVR12) and adverse events (AEs). A Markov model was used to extrapolate the number of disease progressive cases and deaths avoided and estimate cost savings to the healthcare system.
RESULTS:
Usage of sofosbuvir/velpatasvir increased sharply since its subsidy listing and dropped subsequently, whereas utilisation of comparator drugs remained low. Prescribing rate of sofosbuvir/velpatasvir increased from 13.7% to 90.2%, and 39.1% of patients previously on peg-interferon and ribavirin switched to sofosbuvir/velpatasvir post-subsidy. In the cohort study, 365 out of 375 patients (97.3% [95% confidence interval: 95.1%-98.6%]) achieved SVR12. AEs were reported in 3.7% of patients. No serious AE was reported. Based on projected number of patients over the next four years, 184 and 287 cases progressing to decompensated cirrhosis and hepatocellular carcinoma respectively, and 370 hepatitis C-associated deaths were estimated to have been averted. Overall cost savings to the system was projected to exceed SGD$39million over five years.
CONCLUSIONS:
The subsidy decision had led to increased accessibility to patients and intended changes in clinical practice. Sofosbuvir/velpatasvir was clinically effective and safe in the real-world, with a potential to bring about significant cost savings to the healthcare system by averting progression to liver complications and liver-related deaths.
P59: Comparative Efficacy and Safety of Ozanimod and Ponesimod for Relapsing Multiple Sclerosis: A Matching-Adjusted Indirect Comparison
2:30PM - 2:45PM
Swallow E 1 , Pham T2 , Patterson-Lomba O1 , Yin L3 , Gomez-Lievano A1 , Liu J1 , Tencer T4 , Gupte-Singh K5 1 Analysis Group Inc., Boston, MA, USA, 2 Bristol Myers Squibb, Brooklyn, NY, USA, 3 Analysis Group Inc., Los Angeles, CA, USA, 4 Bristol Myers Squibb, San Diego, CA, USA, 5 Bristol Myers Squibb, Princeton, NJ, USA
Objectives: Ozanimod and ponesimod are both sphingosine 1-phosphate receptor modulators approved by the FDA for treatment of relapsing forms of multiple sclerosis (MS). Without head-to-head trials between these 2 treatments, we performed a matching-adjusted indirect comparison (MAIC) of efficacy and safety outcomes between ozanimod and ponesimod for MS. Methods: A MAIC was used to compare efficacy and safety of ozanimod and ponesimod at 2 years, including annualized relapse rate (ARR), % change from baseline in brain volume loss (BVL), rates of any treatment-emergent adverse events (TEAEs), serious AEs, AEs leading to discontinuation, and other safety endpoints. Individual patient-level data were obtained for ozanimod from the RADIANCE-B trial; aggregate-level patient data were obtained for ponesimod from the OPTIMUM trial. The MAIC was not anchored owing to lack of a common comparator across trials. The following characteristics were matched between trial populations: age, % female, time since MS symptom onset, relapses in prior year, Expanded Disability Status Scale score, disease-modifying therapies received within prior 2 years, absence of gadolinium-enhancing T1 lesions, and % of patients from Eastern Europe. Results: After matching, key baseline characteristics were balanced between patients receiving ozanimod and ponesimod. Compared with ponesimod, ozanimod had a numerically lower mean ARR (rate ratio: 0.80 [95% CI: 0.57, 1.10]) and was associated with a significant reduction in BVL (% change difference: 0.20 [0.05, 0.36]). Additionally, ozanimod was associated with a significantly lower risk of TEAEs (risk difference: −11.9% [–16.8%, –7.0%]), AEs leading to discontinuation (−6.1% [−8.9%, −3.4%]), and lymphocyte count <0.2 K/μL (−2.3% [−4.2%, −0.5%]). There were no statistically significant differences in other safety endpoints. Conclusion: The MAIC showed that ozanimod is more efficacious in preserving brain volume compared with ponesimod but comparable in ARR and has a favorable safety profile.
Sun May 15
7:00 AM - 5:00 PM
ISPOR 2022 Registration Hours
In-person
The ISPOR Registration Desk will be open for in-person participants.
8:00 AM - 12:00 PM
Short Course Morning Session
Risk-Sharing/Performance-Based Arrangements for Drugs and Other Medical Products
In-person
Level: Intermediate
Track: Health Policy & Regulatory
This Short Course requires an additional, separate registration fee. Visit the ISPOR 2022 registration page to sign up and learn more.
During the recent years, Managed Entry Agreements (MEAs) have become instrumental in ensuring the access of the innovative medicines. This course is designed for health care professionals (including public decision-makers, academia and industry) involved in pricing and reimbursement decisions who are wishing to understand the applicability and technical aspects of managed entry agreements (MEAs) in countries with severe economic constraints and explicit cost-effectiveness criterion. The topic will be introduced with key features of pricing and reimbursement systems in Central-Eastern European countries to understand why special methods are needed to facilitate evidence-based reimbursement policies of new health technologies. Faculty will present an economic model to explain the methodology and implications of managed entry agreements in cost-effectiveness and budget impact analysis. Participants will then have the opportunity to apply what they have learned through a hands-on exercise on making pricing and reimbursement decisions. A decision algorithm will be presented to support evidence and value-based policy decisions of high-cost new technologies in CEE countries. A series of password protected economic models will add more and more complexity to a pragmatic case study on a new pharmaceutical product in oncology. To close the course faculty will lead a discussion on the applicability of a pragmatic decision tool illustrating the pros and cons of different managed entry agreements and their usefulness in CEE settings. Participants who wish to gain hands-on experience must bring their laptops with Microsoft Excel for Windows installed.
***Registrants will receive a digital course book. Copyright, Trademark and Confidentiality Policies apply.***
Faculty Member
Josh J. Carlson, PhD
University of Washington, Seattle, WA, USA
Lou Garrison, PhD
University of Washington, Seattle, WA, USA
Lou Garrison, PhD, is Professor Emeritus in The Comparative Health Outcomes, Policy, and Economics Institute in the School of Pharmacy at the University of Washington, where he joined the faculty in 2004. Prior to this, he has worked in non-profit policy research (13 years) and the pharmaceutical industry (12 years).
Dr. Garrison received a PhD in Economics from Stanford University. He has more than 180 peer-reviewed publications. His research interests include a wide range of national and international health policy issues.
Dr. Garrison was elected as ISPOR President for 2016-2017. He is currently serving as co-chair of ISPOR’s Policy Outlook Committee for the Health Science Policy Council.
Adrian Towse, MA, MPhil
Office of Health Economics, London, United Kingdom
Professor Adrian Towse is director emeritus and senior research fellow of the Office of Health Economics in the UK. Adrian’s current research includes incentives for new drugs and vaccines to tackle Antimicrobial Resistance, the use of 'risk-sharing' arrangements between healthcare payers and pharmaceutical companies, including value-based pricing approaches; the economics of pharmacogenetics for healthcare payers and the pharmaceutical industry; economic issues that affect both R&D for and access to treatments for diseases prevalent in the developing world; the economics of medical negligence; and measuring productivity in healthcare.
A visiting professor at the London School of Economics and a senior researcher at the Nuffield Department of Population Health at the University of Oxford, Adrian also has been a visiting professor at the University of York. For ten years, he served as the non-executive director of the Oxford Radcliffe Hospitals NHS Trust, one of the UK’s largest hospitals. Adrian was president of ISPOR, for the 2014-15 term.
Adrian joined the OHE in 1993 and served as director for 25 years. He holds an MA (Hons) in Politics, Philosophy and Economics from Keble College, Oxford; an MPhil in Management Studies from Nuffield College, Oxford, and the Oxford Centre for Management Studies; and is a member of the Chartered Institute of Management Accountants.
Market Access & Value Assessment of Medical Devices
In-person
Level: Intermediate
Track: Health Policy & Regulatory
This Short Course requires an additional, separate registration fee. Visit the ISPOR 2022 registration page to sign up and learn more.
This course is designed for those with an intermediate knowledge of medical devices and their market access pathways. The focus will be on understanding the areas of the US and EU value drivers of medical devices, the stakeholder organizations necessary to engage to obtain medical device funding/reimbursement and adoption, and the healthcare system pathways through which medical devices can be implemented. The course will also include a primer on the health economics and outcomes research methods typically used for demonstrating medical device value.
Market access for medical devices is an evolving, multi-faceted, and multi-stakeholder journey that requires dedicated knowledge. Learning about the challenges and opportunities of launching medical devices is key, since the requirements from stakeholders in terms of demonstrating clinical and economic benefits of medical devices has risen.
This course will attempt to demystify the medical device landscape and help all stakeholders ensure that appropriate patients benefit from innovation in the medical device space through improving participants’ awareness of marketplace trends and needs to demonstrate clinical and economic value.
***Registrants will receive a digital course book. Copyright, Trademark and Confidentiality Policies apply.***
Faculty Member
Richard Charter, MSc
Alira Health, Basel, BS, Switzerland
Stephen Hull, MHS
Hull Associates LLC, Rockland, MA, USA
Stephen Hull is principal and founder of Hull Associates LLC, a specialized global reimbursement strategy firm focused on medical device, diagnostic and biotech technologies. Founded in 2007, Hull Associates has market access and reimbursement experts in all major product areas, and is dedicated to helping clients achieve market access and reimbursement success. With 40+ seasoned partners worldwide, Hull Associates LLC develops and executes strategies for reimbursement and product launches in the US and major global markets, including the Americas; Asia Pacific; Northern & Eastern Europe; The Middle East; and Western Europe.
Prior to forming Hull Associates, he served as senior vice president for Global Reimbursement at AdvaMed, in Washington, D.C., the leading US-based medical technology trade association. Internationally, he designed and launched numerous joint advocacy campaigns, with a primary focus on reimbursement issues, working with sister associations in the major overseas markets. Before his trade association work, Stephen was a principal at Covance Health Economics and Outcomes Services, where he executed numerous private and public payor reimbursement and market strategies.
Belinda A. Mohr, Ph.D.
Philips, Phoenix, AZ, USA
Belinda A. Mohr is Senior Manager of Health Economics and Outcomes Research at Philips. She has conducted several economic evaluations to demonstrate the value of medical devices to multiple stakeholders. Her work has been disseminated in peer-reviewed publications, podium and poster presentations, reimbursement submissions, and sales and marketing tools. Prior to joining Philips, Dr. Mohr was a Clinical Strategist and Health Economist for W. L. Gore & Associates, Inc. focused on generating economic evidence for Gore's products. She also worked as an Economist at the U.S. Food and Drug Administration focused on cost-benefit analyses of proposed FDA regulations. She holds a Ph.D. in Economics from the University of California, Santa Barbara.
Introduction to the Design & Database Analysis of Observational Studies of Treatment Effects Using Retrospective Data Sources
In-person
Level: Introductory
Track: Study Approaches
This Short Course requires an additional, separate registration fee. Visit the ISPOR 2022 registration page to sign up and learn more. Retrospective studies require strong principles of epidemiologic study design and complex analytical methods to adjust for bias and confounding. This course will provide an overview of the structures of commonly encountered retrospective data sources with a focus on large administrative data, as well as highlight design and measurement issues investigators face when developing a protocol using retrospective observational data. Approaches to measure and control for patient mix, including patient comorbidity and the use of restriction and stratification, will be presented. Linear multivariable regression, logistic regression, and propensity scoring analytic techniques will be presented and include examples using SAS code that can later be used by participants. This course is an introductory course designed to prepare participants to take intermediate and advanced observational research courses.
***Registrants will receive a digital course book. Copyright, Trademark and Confidentiality Policies apply.***
Faculty Member
Bradley C. Martin, PharmD, PhD, RPh
University of Arkansas for Medical Sciences College of Pharmacy, Little Rock, AR, USA
Dr. Bradley Martin is currently professor and was the founding head of the Pharmaceutical Evaluation and Policy (PEP) Division at the University of Arkansas for Medical Sciences College of Pharmacy. The Pharmaceutical Evaluation and Policy Division offers graduate and professional instruction and conducts research in pharmacoeconomics, patient reported outcomes, pharmaceutical economics, and large health claims data base analysis. Dr. Martin received his PharmD from the University of Illinois and earned his PhD in Pharmacy Care Administration from the University of Georgia. Dr. Martin’s research efforts have focused on conducting retrospective observational comparative effectiveness and economic analyses using large administrative data sets and national health surveys. He conducts policy analyses, and develops cost effectiveness models and has contributed to the understanding of the opioid epidemic. Dr. Martin has over 90 peer-reviewed manuscripts published, which have been collectively cited over 4000 times, and his work has been funded by NIH, AHRQ, VA, and a variety of research foundations as well as partnerships with the pharmaceutical industry. Dr. Martin is contributing to national policy research on opioids and health care financing and has recently lead an international effort to improve the conduct and reporting of CER observational research organized by three national associations: ISPOR, NPC, AMCP.
Cost-Effectiveness Analysis Alongside Clinical Trials
In-person
Level: Introductory
Track: Economic Evaluation
This Short Course requires an additional, separate registration fee. Visit the ISPOR 2022 registration page to sign up and learn more.
The growing number of prospective clinical/economic trials reflects both widespread interest in economic information for new technologies and the regulatory and reimbursement requirements of many countries that now consider evidence of economic value along with clinical efficacy. This course will present the design, conduct, and reporting of cost-effectiveness analyses alongside clinical trials based on, in part, "Good Research Practices for Cost-Effectiveness Analysis alongside Clinical Trials: The ISPOR RCT-CEA Task Force Reports". Trial design, selecting data elements, database design and management, analysis, and reporting of results will all be presented. Trials designed to evaluate effectiveness (rather than efficacy), as well as clinical outcome measures, will also be discussed, including how to obtain health resource use and health state utilities directly from study subjects and economic data collection fully integrated into the study. Analyses guided by an analysis plan and hypotheses, an incremental analysis using an intention to treat approach, characterization of uncertainty, and standards for reporting results will be presented. Familiarity with economic evaluations will be helpful.
***Registrants will receive a digital course book. Copyright, Trademark and Confidentiality Policies apply.***
Faculty Member
Federico Augustovski, MSc, PhD, MD
Institute for Clinical Effectiveness and Health Policy (IECS), Buenos Aires, B, Argentina
Federico Augustovski, MD, MSc, PhD, is the current director of Health Economic Evaluations and Technology Assessment at the Institute for Clinical Effectiveness and Health Policy (IECS), an independent non-profit organization affiliated to the University of Buenos Aires, a CONICET (National Scientific and Technical Research Council) center, and one of the few INAHTA Health Technology Assessments agencies in Latin America. Federico is the director of the WHO Collaborating Centre in Health Technology Assessment and Economic Evaluations at IECS. He is also the founding editor-in-chief for Latin America section of Value in Health Regional Issues, the ISPOR peer-reviewed journal for Latin America, Asia, and Central & Eastern Europe, Western Asia, and Africa. He is the director of the PAHO affiliated PROVAC Center of Excellence for decision making in vaccines. Federico leads a multidisciplinary team devoted to clinical and economic evaluations of new and existing preventive, diagnostic, and therapeutic technologies that provides research, education, and technical support with public and private health decision makers in Latin America. He is a professor of Public Health at the School of Public Health of the University of Buenos Aires, where he teaches courses for graduate and postgraduate students in Decision Sciences; Patient-Reported Outcomes Development in Health, as well as Health Economic Evaluations.
Federico earned his MD with honors at the University of Buenos Aires and is a specialist in family medicine. He practiced family medicine and was a staff physician for more than 20 years at the Family and Community Medicine Division of the Hospital Italiano de Buenos Aires. He received his MSc in epidemiology (Harvard School of Public Health). He was a European Union Scholar in health economics at the Centre for Health Economics at the University of York in the UK. His research production concentrates in health technology assessments and health economic evaluations methods and applications. He has published more than 70 PubMed-indexed papers.
Federico has served and serves ISPOR in several capacities during the past 10 years. Among other commitments, he was the first Latin American director on the board of directors, the founder and first president of the Argentine local chapter, the first chair of the Latin American Consortium, chair of the Research Excellence Award, president of Buenos Aires 2013 Regional meeting, member of the Health Science and Policy Council and Vision 2020 teams, as well as several Task Forces.
Shelby Reed, PhD, RPh
Duke Clinical Research Institute, Durham, NC, USA
Shelby D. Reed, PhD, is Professor in the Departments of Population Health Sciences and Medicine at Duke University’s School of Medicine. She is the director of the Center for Informing Health Decisions and Therapeutic Area leader for Population Health Sciences at the Duke Clinical Research Institute. She also is core faculty at the Duke-Margolis Center for Health Policy. Dr. Reed has over 20 years of experience in economic evaluation, health services research and health policy. Her research portfolio includes a broad array of trial‐based and model‐based cost‐effectiveness analyses of new and existing medical diagnostics, drugs, devices and patient‐centered interventions. For the past several years, she has increasingly dedicated her efforts to the field of stated‐preference research. In 2016, she co-founded the Preference Evaluation Research (PrefER) Group at the DCRI, and she currently serves as its director. She and the group are frequently sought to conduct stated-preference studies to inform regulatory decisions, health policy, care delivery, value assessment and clinical decision making with applied projects spanning a wide range of therapeutic areas.
Dr. Reed has published more than 200 manuscripts in peer‐reviewed journals. She was the first recipient of ISPOR's Bernie O’Brien New Investigator Award in 2005. She served on two ISPOR Task Force groups to develop recommendations for conducting economic evaluations alongside clinical trials and recommendations to address transferability of multinational economic evaluations. She recently served as a guest editor for a themed issue in Value in Health on Patient‐Focused Benefit‐Risk Analysis to Support Regulatory Decision‐Making. She served on the editorial boards for Health Services Research (2016-2020) and Value in Health (2013-2021). She served as President for ISPOR in 2017-2018, and she currently is Past-Chair of the Society’s Health Science Policy Council.
8:00 AM - 5:00 PM
Full Day Short Courses
Introduction to Health Economics and Outcomes Research
In-person
Level: Introductory
Track: Economic Evaluation
This Short Course requires an additional, separate registration fee. Visit the ISPOR 2022 registration page to sign up and learn more.
This course is designed to teach clinicians and new researchers how to incorporate health economics into study design and data analysis. Participants will first review the basic principles and concepts of health economic evaluations, then discuss how to collect and calculate the costs of different alternatives, determine the economic impact of clinical outcomes, and how to identify, track, and assign costs to different types of healthcare resources used. Different health economic models and techniques will be demonstrated including cost-minimization, cost-effectiveness, cost-benefit, cost-utility, and budget impact analysis. Decision analysis, sensitivity analysis, and discounting will all be demonstrated and practiced. This course is suitable for those with little or no experience with health economics.
***Registrants will receive a digital course book. Copyright, Trademark and Confidentiality Policies apply.***
Faculty Member
Lorne E. Basskin, PharmD
UNC Health, Cary, NC, USA
Lorne Basskin received his Bachelor's degree in business at the University of Toronto and worked as an accountant with a national firm and subsequently developed his own consulting business. He later received his PharmD from the University of the Pacific and completed a post-doctoral residency with Valley Medical Center in Seattle in 1995. Dr. Basskin went on to serve as an Associate Professor in Clinical Practice at two different schools of pharmacy in the United States, and was the Director of Post-Graduate and Continuing Education there for seven years. In 2002, Dr. Basskin started his own Medical Education firm, specializing in education for pharmacists and other health care professionals. From 2005 to 2011 he held various positions with HealthSouth Corporation, a for-profit group of 100 rehabilitation and long-term acute care hospitals. Most recently he served as their National Director of Pharmacy Clinical and Information Services and was involved in implementing the Cerner system of electronic medical records throughout the network. After a two year period with Wingate University College of Pharmacy as their Regional Dean, he moved to Asheville, NC, where he carries on a consulting practice specializing in education about health care technology and outcomes research.
Dr. Basskin has written and spoken extensively on the topics of Pharmacoeconomics and Outcomes Research, and published a book on the topic in 1998. He has over 40 peer-reviewed publications and has made over 120 presentations on both clinical and research related matters. He is on the Editorial Board of several National Pharmacy and Medical publications and serves as a peer reviewer for several professional organizations. He has written continuing education programs including that of diabetes, asthma, and Medicare reimbursement. Additionally, he has conducted over 100 one and two day workshops on the meaning and use of pharmacoeconomics for health care decision makers and health care researchers.
12:00 PM - 1:00 PM
Break
In-person
Coffee Service; Lunch on Own
1:00 PM - 5:00 PM
Short Course Afternoon Session
A Health Economics Approach to US Value Assessment Frameworks
In-person
Level: Introductory
Track: Health Policy & Regulatory
This Short Course requires an additional, separate registration fee. Visit the ISPOR 2022 registration page to sign up and learn more.
This short course will focus on the recent ISPOR Special Task Force Report, “A Health Economics Approach to US Value Frameworks.” It will begin with an overview of recent US value assessment frameworks, with emphasis on the importance of perspective and decision context in the construction and use of value frameworks. It will then review how a health economics approach from a societal or health plan perspective leads to use of cost-effectiveness analysis (CEA) to help guide efficient resource allocation.
There will be in-depth discussion of how measuring some aspects of the value of health benefits could augment the standard cost-per-quality-adjusted-life-year metric for CEA. Elements such as value of insurance, value of “hope,” real option value, severity of illness, and several others, have the potential to better capture how patients and/or society value the benefits of some treatments; each one is based on some research findings and some case examples will be shown.
The course will then review how budget considerations, cost-effectiveness thresholds, and opportunity costs enter CEA-based decision-making. Next faculty will review broader approaches to cost-benefit aggregation and value-based decision-making, including extended CEA, augmented CEA (introduced by this Report), and multi-criteria decision analysis (MCDA), with an overview of issues and new approaches to MCDA. It then discusses the strengths and weaknesses of recent US value assessment frameworks from this health economic perspective and closes with a review of the high-level recommendations of this Special Task Force.
***Registrants will receive a digital course book. Copyright, Trademark and Confidentiality Policies apply.***
Faculty Member
Lou Garrison, PhD
University of Washington, Seattle, WA, USA
Lou Garrison, PhD, is Professor Emeritus in The Comparative Health Outcomes, Policy, and Economics Institute in the School of Pharmacy at the University of Washington, where he joined the faculty in 2004. Prior to this, he has worked in non-profit policy research (13 years) and the pharmaceutical industry (12 years).
Dr. Garrison received a PhD in Economics from Stanford University. He has more than 180 peer-reviewed publications. His research interests include a wide range of national and international health policy issues.
Dr. Garrison was elected as ISPOR President for 2016-2017. He is currently serving as co-chair of ISPOR’s Policy Outlook Committee for the Health Science Policy Council.
Charles Phelps, MBA, PhD
University of Rochester, Rochester, NY, USA
Charles E Phelps, PhD, a health economist, has developed key models of cost-effectiveness analysis that provide the intellectual foundations for its practice. He was given the Victor R Fuchs Award for Lifetime Achievement in the Field of Health Economics in 2019, and has been a member of the National Academy of Medicine since 1991. His leading textbook, Health Economics is now in its 6th Edition. His recent interests have expanded to the use of multi-criteria decision analysis (MCDA), particularly in its proper use when the “decision-maker” is a group.
Richard Willke, PhD
ISPOR, Lawrenceville, NJ, USA
Dick became ISPOR’s first chief science officer in April 2016, following nearly 25 years in the pharmaceutical industry with Pfizer and its legacy companies. In his CSO role at ISPOR, Dick’s responsibilities are to develop, lead, support, and direct strategic initiatives related to research, scientific, and content priorities to accomplish the organization’s mission to promote health economics and outcomes research excellence to improve decision making for health globally. While with Pfizer, his final position was Vice President, Outcomes & Evidence, lead for Cardiovascular /Metabolic, Inflammation & Immunology, the last in a succession of HEOR group lead roles. He received a PhD in economics from Johns Hopkins University in 1982, concentrating in econometrics and labor economics. Prior to joining Pfizer’s legacy company Upjohn in 1991, he was a member of the economics faculty at Ohio State University as well as a senior economist at the American Medical Association Center for Health Policy Research.
Dick has served on the ISPOR Board of Directors (2007-09), was chair of the ISPOR Institutional Council in 2010, and was co-chair of the ISPOR Good Research Practices Task Force on Cost-Effectiveness Analysis in Randomized Clinical Trials in 2003-2005 as well as its 2014-15 reprise to revise and update that Report. He has co-taught many ISPOR short courses on this topic as well as on “Transferability of Cost-Effectiveness Data between Countries.” He was also a member of the Health Outcomes Committee of PhRMA from 1998-2009, having been its chair from 2002-2004. He has served as a co-editor for Value in Health, on the editorial board for Farmeconomia, on AHRQ, NIH, and PCORI project review study sections, and is a member of the Ohio State University Economics Advisory Board.
Prior to joining industry, Dr Willke served as department director in the Center of Health Policy Research at the American Medical Association and held research and teaching positions at The Ohio State University.
Dr Willke earned a PhD and MA in economics from Johns Hopkins University. He has authored more than 80 scholarly publications that examine the science and methodologies of health economics and outcomes research.
Introduction to Machine Learning Methods
In-person
Level: Intermediate
Track: Methodological & Statistical Research
This Short Course requires an additional, separate registration fee. Visit the ISPOR 2022 registration page to sign up and learn more.
Healthcare data are often available to payers and health care systems in real time, but are massive, high dimensional, and complex. Machine learning merges statistics, computer science, artificial intelligence, and information theory and offers powerful computational tools to enhance the extraction of useful information from complex healthcare data, build highly interpretable models, and make accurate predictions. This course gives an overview of basic machine learning concepts and provides an introduction to a few commonly used machine learning techniques and their practical applications in healthcare and pharmaceutical outcomes research. Participants will be introduced to foundational principles and concepts of statistical machine learning, then be provided with several specific machine learning techniques and their applications in health and pharmaceutical outcomes research. Different machine learning approaches using R will be demonstrated including tree-based methods, penalized regression, and neural networks analysis, as well as techniques for dimension reduction/feature selection. Participants will have hands-on practical experiences with machine learning and gain experience interpreting and evaluating the results and prediction performance that comes from machine learning modeling.
Distinguishing prediction modeling from research on real-world data meant for causal inference in pharmacoepidemiology will be also presented and discussed. This is an entry-level course but is designed for those with some familiarity with traditional statistical modeling techniques (eg, linear regression, logistic regression).
***Registrants will receive a digital course book. Copyright, Trademark and Confidentiality Policies apply.***
Faculty Member
Weihsuan Lo-Ciganic, PhD, MSPharm, MS
University of Florida, Gainesville, FL, USA
Dr. Wei-Hsuan Jenny Lo-Ciganic is a pharmacoepidemiologist and associate professor in the Department of Pharmaceutical Outcomes and Policy at the University of Florida College of Pharmacy. Her research agenda focuses on drug safety and addiction. Dr. Lo-Ciganic has extensive experience applying advanced predictive analytics including machine learning and trajectory modeling with large healthcare datasets. She conducts research to develop risk prediction algorithms and tools, and practical intervention applications for use in real-world settings to improve health outcomes and patient care. She is also a core faculty member in the Center for Drug Evaluation and Safety (CoDES) at the University of Florida College of Pharmacy.
John D. Seeger, PharmD, DrPH
Optum, Boston, MA, USA
Dr. John Seeger is a pharmacoepidemiologist and chief scientific officer at Optum Epidemiology. He is also adjunct assistant professor in Epidemiology at the Harvard School of Public Health. Dr. Seeger has conducted dozens of studies that have addressed regulatory drug safety issues across a wide range of drugs and disease conditions. Most of this work has involved the use of health insurance claims databases as platforms for pharmacoepidemiology, so Dr. Seeger's methodological expertise focuses on research issues encountered in such settings. He has worked extensively with propensity scores and related methods that mitigate confounding by collapsing covariates. Additionally, he is a co-instructor in courses on propensity scores and is the past president of the International Society for Pharmacoepidemiology (ISPE).
Hao Helen Zhang, PhD
University of Arizona, Tucson, AZ, USA
Dr. Hao Helen Zhang is a statistician and professor in mathematics at the University of Arizona. Dr. Zhang’s research agenda includes statistical machine learning, high-dimensional data analysis, nonparametric smoothing, and biomedical data analyses. She has more than 15 years of research and teaching experiences in machine learning for predictive data analytics and is co-author of the textbook Principles and Theory for Data Mining and Machine Learning. Additionally, she serves as a faculty member of the University of Arizona Statistics Graduate Interdisciplinary Program, and is associate editor of the Journal of the American Statistical Association, Journal of Royal Statistical Society, Journal of Computational and Graphical Statistics, and Statistical Analysis and Data Mining, and as the Editor-in-chief of the journal STAT. She is a fellow of the American Statistical Association, fellow of the Institute of Mathematical Statistics, and the 2019 IMS Medallion Lecturer.
Early-Stage Health Technology Assessment (HTA)
In-person
Level: Introductory
Track: Health Technology Assessment
This Short Course requires an additional, separate registration fee. Visit the ISPOR 2022 registration page to sign up and learn more.
As the cost of bringing a new health technology to market continues to climb, more and more firms, developers, and investors are searching for tools to prioritize their efforts on the technologies with the greatest potential for clinical impact and market viability. While health economic analysis has long been established as a necessity to inform decision making for market access and reimbursement, it is increasingly being used at earlier stages of product development for healthcare and life sciences to increase the access rate of R&E and efficiently prioritize data collection. The number of available methods for this field has continued to expand.
This course aims to demystify the objectives of early-stage health technology assessment and the methods of translational health economics. Students in the course will gain a thorough understanding of available methods for early-stage technology assessment, the specific challenges and solutions, and a clear sense of how to implement this in the complexity of health technology development, funding, regulation, pricing, and reimbursement. The course will utilize real-world examples and students will have the opportunity to strategize about the creation of a research plan for their purposes.
***Registrants will receive a digital course book. Copyright, Trademark and Confidentiality Policies apply.***
Faculty Member
William Canestaro, PhD, MSc
Washington Research Foundation, Seattle, WA, USA
Erik Landaas, PhD, MPH
Cook Medical, Bloomington, IN, USA
Lotte Steuten, PhD, MSc
Office of Health Economics, London, LON, United Kingdom
Lotte Steuten, PhD, is Vice-President and Head of Consulting at the Office of Health Economics and Honorary Visiting Professor at City, University of London, UK. After graduating cum laude with her PhD from Maastricht University, the Netherlands, Lotte has been active in the HEOR field for over 15 years in various academic roles and executive functions (non-profit and for-profit).
To inform better decisions based on independent research and analysis, Lotte worked effectively with pharmaceutical industry, technology assessors, payers and policy makers, (academic) researchers, clinical and patient representatives as well as capital investors. Notably, her international career-path, including the UK, the US and the Netherlands, has provided her with deep insights in the fundamental differences as well as commonalities between the role of HEOR in different healthcare systems, cultural and societal values.
Over the past decade, Lotte has been leading diverse teams; developing strategies and delivering on program or company missions, values and objectives; acquiring funding and managing budgets; and been responsible for executive decision-making as well as legal and fiduciary matters. In her current role at the Office of Health Economics she is responsible for the research-led consulting program, making sure to maintaining OHE's stellar reputation for objective, innovative and high-quality research and analysis globally, as well as meeting its charitable objectives. In addition, she is a professor at City, University of London, contributing to their Master and PhD programs. Before joining OHE, Lotte worked as an Associate Member at the Fred Hutch Cancer Research Institute and Associate Professor at the University of Washington, Seattle (US), where she currently holds affiliate appointments.
Stated Preference Methods
In-person
Level: Introductory
Track: Patient-Centered Research
This Short Course requires an additional, separate registration fee. Visit the ISPOR 2022 registration page to sign up and learn more. Stated-preferences methods describe a range of survey techniques that can be used to study the priorities and preferences of patients and other stakeholders in health. These methods include a range of experimental and non-experimental methods that can be used to study how health and healthcare is valued and experienced. The literature applying these methods in health has grown dramatically in the last 2 decades, and methods such as discrete-choice experiments, conjoint analysis, best-worst scaling, and other tradeoff techniques are now considered essential tools in outcomes research. This course will provide an overview of stated-preference methods used today and will highlight both good research practices and strategies to make studies more relevant and useful in decision making. Participants in this course will learn about a variety of methods, how these methods can best be used in health, and good practices to develop, analyze, interpret, and disseminate and apply these methods in health. Detailed case studies of empirical examples will be used to illustrate concepts and methods.
***Registrants will receive a digital course book. Copyright, Trademark and Confidentiality Policies apply.***
Faculty Member
John Bridges, PhD
Ohio State University College of Medicine, Columbus, OH, USA
John F.P. Bridges PhD is a professor in the Departments of Biomedical Informatics and Surgery within the Ohio State University College of Medicine. Working at the intersection of medicine and the social sciences, John advances and applies methods to incorporate the priorities and preferences of patients and other stakeholders in medical decision making. John was founding editor of The Patient – Patient Centered Outcomes Research (since 2008) and serves on the editorial boards of Pharmacoeconomics (since 2006), Expert Review of Pharmacoeconomics and Outcomes Research (since 2007), and International Journal of Technology Assessment in Health Care (since 2008). Within the ISPOR he was the founding chair of the Conjoint Analysis Working Group and the Conjoint Analysis Task Force that produced several reports on good research practices for stated-preference methods. He received ISPOR’s Bernie O’Brien New Investigator Award in 2006 and ISPOR’s Distinguished Service Award in 2011. He is the author of over 200 articles and a frequent speaker on the art and science of using stated-preference methods and engaging patient organizations in decision making. John is currently affiliated with Ohio State University Comprehensive Cancer Center’s Cancer Control Program, the Center for the Advancement of Team Science, Analytics and Systems Thinking in Health Systems Research and Implementation Science (CATALYST), and the Center for Surgical Health Assessment, Research and Policy (SHARP). He is also an adjunct professor within the Department of Health Behavior and Society at the John Hopkins Bloomberg School of Public Health.
Deborah Marshall, PhD, BSc
University of Calgary, Calgary, AB, Canada
Deborah is a professor at Cumming School of Medicine, University of Calgary and Arthur J.E. Child Chair in Rheumatology Outcomes Research and former Canada Research chair, Health Services and Systems Research. Her research program focuses on the measurement of preferences, cost-effectiveness analysis, and simulation modeling of health services and interventions. Deborah has over 20 years of research experience in health technology assessment agencies, academic institutions, and industry settings in Canada, US, and Europe. She is a founding co-investigator of the Patient and Community Engagement Research (PaCER) Program and co-leads the economics and stated preferences research platforms for the Canada-Netherlands Personalized Medicine Network in Childhood Arthritis and Rheumatic Disease (UCAN CANDU).
Deborah is an active member of the ISPOR as the past-president of the Board of Directors, and co-author on the three “ISPOR Task Force Reports for Good Research Practice – Checklist for Conjoint Analysis in Health, Conjoint Analysis Experimental Design and Statistical Methods for the Analysis of Discrete-Choice Experiments.”
Budget Impact Analysis I: A 6-Step Approach
In-person
Level: Introductory
Track: Economic Evaluation
This Short Course requires an additional, separate registration fee. Visit the ISPOR 2022 registration page to sign up and learn more.
This course will describe the methods used to estimate the budget impact of a new health care technology and will present six basic steps for estimating budget impact: (1) estimating the target population; (2) selecting a time horizon; (3) identifying current and projected treatment mix; (4) estimating current and future drug costs; (5) estimating change in disease-related costs; and (6) estimating and presenting changes in annual budget impact and health outcomes. Both static and dynamic methods for estimating the budget and health impact of adding a new drug to a health plan formulary will be presented. These six steps will be illustrated using actual budget impact models. This course is designed for those with some experience with pharmacoeconomic analysis.
***Registrants will receive a digital course book. Copyright, Trademark and Confidentiality Policies apply.***
Faculty Member
Ashley Davis, PhD
RTI Health Solutions, Research Triangle Park, NC, USA
Ashley Davis, PhD, is a Director of Health Economics at RTI-HS. Dr. Davis uses analytical methodologies to evaluate the clinical and economic value of upcoming pharmaceutical products and changes to health care policies. She has developed cost effectiveness, budget-impact, dynamic transmission, and population models, and has broad experience with a wide variety of mathematical modeling techniques, including Markov and stochastic models, simulation, statistical analysis, linear and nonlinear programming, and robust optimization. Dr. Davis has extensive experience programming models and user-friendly interfaces using Visual Basic for Applications in Excel and is proficient with other software packages, including MATLAB, C++, Java, and SAS. Dr. Davis has developed models and analyses in the areas of HIV, hepatitis C, cystic fibrosis, herpes zoster, influenza, Ebola virus, pneumococcal disease, respiratory syncytial virus, severe asthma, chronic obstructive pulmonary disease, eosinophilic esophagitis, spinal surgery, and organ transplantation.
Her research has been presented at various professional conferences and published in several peer-reviewed journals, including Pharmacoeconomics, PLoS One, HIV Clinical Trials, Clinical Journal of the American Society of Nephrology, Medical Decision Making, Transplantation, Institute for Operations Research and the Management Sciences (INFORMS), the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), and the American Transplant Congress.
Stephanie R. Earnshaw, PhD, MS
RTI Health Solutions, Research Triangle Park, NC, USA
Stephanie Earnshaw is senior vice president of Health Economics at RTI Health Solutions (RTI HS). She received her PhD in Industrial Engineering at North Carolina State University and has been with RTI-HS for over 19 years. She has presented workshops, distance learning, and short courses on decision-analytic modeling techniques for pharmaceutical companies and organizations such as ISPOR, the Academy of Managed Care Pharmacy (AMCP), and the Centers for Disease Control and Prevention (CDC). Dr. Earnshaw currently serves on the ISPOR Board of Directors, and she has held an Adjunct Faculty appointment at the University of North Carolina’s Eshelman School of Pharmacy, Division of Pharmaceutical Outcomes and Policy. She is also one of the lead authors of the newly published book “Budget-Impact Analysis of Health Care Interventions: A Practical Guide.”
Dr. Earnshaw’s research focus is in applying decision-analysis techniques to industry-related issues and health care problems. Her areas of specialization include mathematical programming (constrained optimization), network optimization, and Markov, simulation, and other state transition modeling. She has developed innovative mathematical models using these methods to determine pricing strategy, predict clinical outcomes, and allocate resources. In addition, she continues her support for the pharmaceutical, biotechnology, and diagnostic and medical device industry by developing budget-impact and cost-effectiveness models for their health technologies. Therapeutic areas include cardiovascular disease, gastrointestinal disorders, respiratory disease, transplantation, infectious disease, osteoporosis, vaccines, and oncology. She is a member of ISPOR and the Institute for Operations Research and the Management Sciences. She has presented her work at professional conferences and has published in several peer-reviewed journals.
C. Daniel Mullins, PhD
University of Maryland, School of Pharmacy, Baltimore, MD, USA
C. Daniel Mullins, PhD is a professor and chair of the Pharmaceutical Health Services Research Department at the University of Maryland School of Pharmacy. He received his BS in Economics from M.I.T. and his PhD in Economics from Duke University. His research and teaching focus on comparative effectiveness research (CER), patient-centered outcomes research (PCOR), pharmacoeconomics, and health disparities research. He directs the University of Maryland PATient-centered Involvement in Evaluating effectiveNess of TreatmentS (PATIENTS) Program, which received a University of Maryland Baltimore Champion of Excellence Award. He has received funding as a Principal Investigator from the NIH/NIA, NIH/NHLBI, AHRQ, and the Patient-Centered Outcomes Research Institute (PCORI) and was the Shared Resources Core Director for the NIH-sponsored University of Maryland Center for Health Disparities Research, Training, and Outreach. He previously served as a Regular Member of the AHRQ HSR and the NCI-J Study Sections and currently serves as a Regular Member for the AHRQ HCRT Study Section. He also has chaired PCORI Study Sections.
In addition to his work on federal grants, Professor Mullins is co-editor-in-chief for Value in Health and is author/co-author of approximately 200 peer-reviewed articles and book chapters on pharmacoeconomics, outcomes research, and pharmaceutical policy and health disparities research in journals such as The American Journal of Managed Care, The American Journal of Public Health, Cancer, Chest, Health Affairs, Health Services Research, JAMA, Journal of Clinical Oncology, Pharmacoeconomics, and Social Science & Medicine. He has received an Outstanding Service Award from the Drug Information Association (DIA) and two Service Awards from the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). In 2007, he received the Dr. Patricia Sokolove Outstanding Mentor Award from the University of Maryland, Baltimore campus-wide Graduate Student Association. In 2013, he was the recipient of the Dr. Daniel D. Savage Memorial Science Award, the Association of Black Cardiologists’ most prestigious annual award. Also, in 2013, he was awarded a University System of Maryland Wilson H. Elkins Professorship. In 2014, he and Robin Newhouse were named co-researchers of the year for the University of Maryland Baltimore campus.
Developing Decision-Grade Real-World Evidence
In-person
Level: Intermediate
Track: Real World Data & Information Systems
This Short Course requires an additional, separate registration fee. Visit the ISPOR 2022 registration page to sign up and learn more. In this course, participants will be guided through a hands-on analysis of real-world data to develop decision-grade real-world evidence (RWE) that could be used to support an indication expansion. The first section of the course focuses on what makes RWE “decision-grade.” We will review the most recent RWE frameworks and guidelines set by regulatory agencies and professional organizations, and we will examine case studies in which these guidelines were used in regulatory and HTA approval. The second half of the course is an active workshop where participants will use principles from the first half of the course to execute a decision-grade RWE study. Participants will be guided step-by-step in using a software platform that will enable them to work within a longitudinal US insurance claims database with anonymized patients. After the study has been executed, we will discuss how these results could be communicated to decision makers. Participants should come with a laptop with Google ChromeTM installed.
PREREQUISITE: Students are expected to be familiar with relevant concepts and methodologies for analyzing real-world data, but this course does not require specific programming skills.
***Registrants will receive a digital course book. Copyright, Trademark and Confidentiality Policies apply.***
Faculty Member
Jeremy Rassen, ScD
Aetion, Inc., New York, NY, USA
Dr. Jeremy A. Rassen, ScD, is co-founder and chief scientific officer at Aetion, Inc., a company that provides software to evaluate the effectiveness, safety, and value of medical treatments. At Aetion, Dr. Rassen leads the scientific effort around designing methodology for obtaining and communicating medical evidence from real-world data.
Dr. Rassen was formerly an assistant professor of Medicine at the Brigham and Women's Hospital and Harvard Medical School, where he focused on methodology for improved validity and reach of pharmacoepidemiology and comparative effectiveness research, including research into propensity score and instrumental variable methods. Before coming to the Brigham and Women’s Hospital, Dr. Rassen worked in Silicon Valley in numerous computer and software companies, including Hewlett-Packard and Epiphany, Inc. His focus was on high-performance software for the creation and analysis of large marketing databases.
Dr. Rassen received his Bachelor’s degree from Harvard College and his Doctorate degree in Epidemiology from the Harvard School of Public Health.
Sebastian Schneeweiss, MD, ScD
Harvard Medical School and Harvard School of Public Health, Boston, MA, USA
Shirley Wang, PhD
Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA
Dr. Wang is an Associate Professor at Brigham and Women’s Hospital, Harvard Medical School. She has led 2 joint task forces between ISPOR and the International Society of Pharmacoepidemiology (ISPE) focused on real-world evidence for healthcare decision-making. Dr. Wang directs the REPEAT Initiative, a non-profit program with projects aimed at improving transparency, reproducibility and robustness of evidence from healthcare databases and co-leads RCT-DUPLICATE, a series of projects designed to inform when and how real-world data analyses can draw causal conclusions.
Mon May 16
6:30 AM - 5:30 PM
ISPOR 2022 Registration Hours
In-person
The ISPOR Registration Desk will be open for in-person participants.
7:15 AM - 8:15 AM
Educational Symposium
Can Value-Based Care Exist Without Value-Based Research
In-person & Virtual
The concept of value-based care continues to play a significant role in healthcare policy discussions. But can value-based care ever be fully realized without value-based research? All too often clinical trial outcomes are statistically significant but clinically meaningless. Even when clinical trials are clinically relevant, the populations studied are not representative or the control arms at time of study design no longer represent the standard of care. Experts from Cardinal Health and industry weigh in on the critical importance of research in driving improved quality and outcomes for patients.
Sponsor
Cardinal Health
Moderators
Bruce Feinberg, DO
Cardinal Health Specialty Solutions, ATLANTA, GA, USA
Bruce Feinberg, D.O., is Vice President, Clinical Affairs and Chief Medical Officer for Cardinal Health Specialty Solutions. He is nationally recognized for his expertise in specialty oncology and the business of specialty healthcare. Dr. Feinberg has been instrumental in the development of clinical pathways that aim to control costs, improve quality, and increase predictability, all of which are key factors in developing a sustainable approach for caring for patients with high-cost diseases. A highly sought-after researcher and speaker on healthcare policy, value based care and real-world evidence research, Dr. Feinberg has over 200 publications in peer-review; and he is also the author of the bestselling Breast Cancer Answers and its follow-up book, Colon Cancer Answers.
Speakers
Ali McBride, PharmD, MS
BMS, Summit, NJ, USA
Kristin Savill, PhD
Cardinal Health, El Dorado Hills, CA, USA
Kristin M. Zimmerman Savill, PhD
Principal Scientist, Real-World Evidence & Insights
Kristin Zimmerman Savill, PhD is a Principal Scientist in the Real-World Evidence & Insights division of Cardinal Health Specialty Solutions and serves as Lead Scientist/Principal Investigator on oncology real-world evidence studies. As a scientific leader in real-world evidence research, she aims to generate strong scientific evidence to support clinical, development, and regulatory decision-making. Dr. Savill has nearly two decades of experience in oncology research, including 11+ years in the biopharma industry. Her research has focused on evaluating treatment patterns and clinical outcomes in real-world settings, factors that mediate clinical outcome, novel therapeutic strategies, and biological pathways that play a key role in tumorigenesis and survival. Dr. Savill has expertise in the design, conduct, analysis, and interpretation of cancer-related research studies. Her publication and presentation history includes manuscripts in peer-reviewed journals; abstracts, posters, and oral presentations at national and international scientific conferences; and scientific magazine articles. Prior to joining Cardinal Health, Dr. Savill served as a Scientific Manager in the Development Sciences division at Genentech where she played an integral role in biomarker strategy for oncology programs. She completed her postdoctoral training in the Translational Oncology department at Genentech and holds a PhD in Biochemistry from the University of Sussex in the UK as well as a BSc in Psychobiology from UCLA.
Scott Swain, PhD, MPH
Cardinal Health Specialty Solutions, Dublin, OH, USA
8:30 AM - 9:45 AM
Welcome & Plenary Session 1
HTA on the Run
In recent years, health authorities have been experimenting with regulatory and reimbursement models to reconcile the tension between the accelerating pace of emerging healthcare technologies and the traditionally slow, cautious procedures that regulate their introduction into practice. The pandemic has fueled this interest by testing healthcare systems globally, intensifying the need for timely introduction of vaccines, biologicals, diagnostics, telehealth and digital health solutions. These factors have forced health technology assessment to occur ‘on the run’. This plenary will feature three case studies presenting unique solutions to these challenges: 1) experiences from the Digital Health Applications (DiGA) fast track approval/reimbursement process implemented through the German Social Code; 2) insights from the Breakthrough Devices Program (BDP) in the US, which allows greater reliance on post-marketing data for medical devices and 3) an overview of the FDA Total Product Lifecycle Advisory Program, a menu of tools and services to get stakeholder input on evidence requirements for clinical development that is expected to provide new and enhanced pathways of regulatory and reimbursement approval in coming years. The technical and policy challenges faced during this unique time period will be highlighted, and the case studies will provide critical learnings about how to accelerate the introduction of new products and assess their impact on patients’ lives.
*Speakers to be added as confirmed!
Moderators
Ran D. Balicer, MD, PhD, MPH
Clalit Health Services, Tel Aviv, Israel
Speakers
Noa Dagan, MD, PhD, MPH
Clalit Health Services, Tel Aviv-Yafo, Israel
Noa Dagan is a public health physician and researcher. She holds an MD and an MPH from the Hebrew University, and a PhD in Computer Science from Ben-Gurion University. She completed her postdoc in the Department of Biomedical Informatics at Harvard Medical School.
Dr. Dagan heads the AI-driven Medicine Department at Clalit Innovation (Clalit is Israel's largest healthcare organization, covering over 50% of the Israeli population). Her responsibilities include the development and implementation of digital healthcare solutions to promote preventive, proactive and personalized medicine.
Dr. Dagan is also the co-director of the Digital Health Lab at the Department of Software and Information Systems Engineering in Ben-Gurion University.
Douglas Kelly, MD
Food and Drug Administration, Silver Spring, MD, USA
Simon Reif, PhD
ZEW, Mannheim, Germany
Dr. Simon Reif is head of the Health Care Markets and Health Policy research group at ZEW – Leibniz Centre for European Economic Research in Mannheim. He previously worked at FAU Erlangen-Nürnberg, RWI – Leibniz Institute for Economic Research, and dmac – Digital Health Application Centre. He has also been affiliated with the Evidence-Based-Economics Graduate Program of the Elite Network Bavaria and visited the Center for Health Economics at the University of York in 2017.
He received his PhD from FAU in 2018 and prior to that, obtained a Master's degree from LMU Munich in 2014 and undergraduate degrees from FAU in 2012 and the University of Hull in 2011. His areas of research focus on health economics and applied econometrics, with a particular interest in behavioral and institutional aspects of medical service provision. Current research projects deal with the design of hospital payment systems, regional access to health care, and strategies for the evaluation of digital health applications.
Joseph Ross, MD, MHS
Yale University, New Haven, CT, USA
Joseph S. Ross, MD, MHS, is a Professor of Medicine (General Medicine) and of Public Health (Health Policy and Management) at the Yale School of Medicine. His research examines the use and delivery of higher quality care and issues related to pharmaceutical and medical device regulation, evidence development, postmarket surveillance, and clinical adoption. Dr. Ross co-directs the Yale-Mayo Clinic Center for Excellence in Regulatory Science and Innovation (CERSI), the Yale Open Data Access (YODA) Project, and leads collaborations with the National Evaluation System for health Technology (NEST).
Welcome Remarks
Welcome Remarks
CEO Remarks
Nancy Berg, ISPOR CEO & Executive Director
Presidential Address
Isao Kamae, DrPH, MD, 2021-2022 President
Program Committee Co-Chair Welcome
Eberechukwu Onukwugha, PhD, ISPOR 2022 Program Committee Co-Chair
9:45 AM - 10:15 AM
Coffee Break
In-person
9:45 AM - 1:15 PM
In-Person and Virtual Poster Session 1
Live
In-person presenters will be with their posters from 12:15 – 1:15PM.
9:45 AM - 7:00 PM
Poster Viewing & Exhibit Hall Open
In-person & Virtual
10:15 AM - 11:15 AM
Concurrent Breakout Session 1
Health Technology Assessment of Treatments and Diagnostics for COVID-19: Best-Practice Guidance
Virtual
Level: Intermediate
PURPOSE
: This workshop will present recent best-practice guidance developed within the ‘Next Generation HTA’ project (HTx) to support health technology assessment (HTA) agencies in evaluating diagnostics and treatments for COVID-19. Panellists, all involved in the development process, will discuss the challenges facing the HTA of COVID-19 technologies and the key recommendations to address these challenges. Attendees will learn how the guidance can support HTA-based decision making during the pandemic.
DESCRIPTION
: HTA was not at the forefront of healthcare decision making about tests and treatments for COVID-19 in response to the pandemic, as governments prioritised urgent action. Now, HTA agencies are tasked with assessing the value of a growing number of COVID-19 technologies. HTx best-practice guidance has been developed, with input from the ISPOR HTA Council, to provide a pragmatic, consistent framework to approach these assessments. Dalia Dawoud will introduce the HTx project, and present the rationale for developing guidance to support the HTA of technologies for COVID-19 (12`). Jamie Elvidge will describe how a multi-stakeholder policy sandbox approach was used the develop the guidance, and will summarise the key recommendations (12`). Brian O’Rourke will outline the COVID-related challenges reported by HTA agencies, and consider the benefits, practicality, and implications of the guidance (12`). Neil Bertelsen will highlight the continued need for broad stakeholder engagement despite the challenging pandemic context, and present a tiered approach to gaining insights from relevant patient and citizen groups (12`). There will be audience polling and Q&A (12`). This workshop will be of interest to all HTA stakeholders.
Discussion Leaders
Dalia Dawoud, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Dalia Dawoud, PhD, is Senior Scientific Adviser at the National Institute for Health and Care Excellence (NICE). She holds MSc in Economic Evaluation in Health Care from City University London and PhD in pharmaceutical policy and economics from King’s College London.
She has long experience in using economic evaluation in clinical guidelines development and health technology assessment (HTA), gained through working on NICE Clinical Guidelines as well as technology appraisals. Dalia’s research interests are focused on the advanced methods of evidence synthesis and use in economic models and the use of real-world evidence to inform drug development and health care decision making. Dalia currently has overall responsibility of overseeing the delivery of NICE allocated tasks on a portfolio of IMI and Horizon 2020 funded research projects including EHDEN and HTx. She is widely published in the field of pharmaceutical policy and pharmacoeconomics. She also serves as Associate Editor for ISPOR journal Value in Health and as Associate Editor for Pharmacoeconomics and Outcomes Research for Elsevier’s journal Research in Social and Administrative Pharmacy. Dalia also holds adjunct position as Associate Professor at the Faculty of Pharmacy, Cairo University.
Discussants
Neil Bertelsen, MSc, MBA
Neil Bertelsen Consulting, Berlin, Germany
Neil Bertelsen has over 25 years of experience bringing the patient voice to health care decision makers and communicating the science of health care to patients in a way that truly informs their own personal health choices. Neil is passionate about bringing the patient experience and perspectives to decision-makers including industry and health technology assessment (HTA) bodies.
Neil is the past chair of ‘Health Technology Assessment International’s (HTAi) Patient and Citizen Involvement Interest Group’, an international group whose remit is to work closely with HTA organizations and patient organizations to bring patient involvement processes and patient insights and experiences into decision making process. Neil is currently on the steering committee of this group.
Neil is also a board member of PFMD (Patient Focused Medicines Development), an international consortium of stakeholders working towards more integrated inclusion of the patient voice across the medicine lifecycle.
Neil works directly with the patient advocacy community, the industry, and authorities such as HTA agencies to facilitate collaborations and co-creation of approaches to improve access to healthcare and better provision of care. As a facilitator of meetings and advisory boards, Neil has global experience working with multiple stakeholder on demanding issues that require a coordinated response.
Jamie Elvidge, MSc
National Institute for Health and Care Excellence, Manchester, United Kingdom
Jamie Elvidge, MSc is a health economist and scientific adviser in the Science Evidence & Analytics Directorate at NICE, UK. As part of the NICE Research team, he collaborates on a range of HEOR research projects. This includes HTx, a Horizon 2020 (EU) project to support patient-centred, societally oriented, real-time HTA decision-making.
Jamie has worked at NICE for 5 years, including as a modeller (Clinical Guidelines) and adviser (Technology Appraisals). He has over 10 years' experience in HEOR, including consultancy and academia. He holds an MSc in health economics (University of York).
Brian O'Rourke, PharmD
ISPOR HTA Council, Ottawa, ON, Canada
Dr. Brian O’Rourke served as the President and Chief Executive Officer of CADTH from 2009-2020. He joined CADTH following a distinguished career as a Pharmacist and Health Care Executive with the Canadian military. With over 40 years of experience in health care, Brian is a leading expert in the science and practice of health technology assessment (HTA) and served as the Board Chair for the International Network of Agencies for Health Technology Assessment from 2014 to 2018. He has a Bachelor of Science in Pharmacy from Dalhousie University and a Doctor of Pharmacy from the University of Toronto. Dr. O’Rourke continues to play an active role in the global HTA community. He is Chair of the Health Technology Assessment Steering Committee and a member of the Scientific Advisory Council at the Centre for Innovation in Regulatory Science (CIRS). He is also Chair of the Health Technology Assessment Council of the Professional Society for Health Economics and Outcomes Research (ISPOR). In November 2020, Dr. O’Rourke was appointed Colonel Commandant (Honorary) of the Royal Canadian Medical Service.
How to Apply Machine Learning to Health Economics and Outcomes Research: Findings from the ISPOR Machine Learning Task Force
In-person
Level: Intermediate
PURPOSE: ISPOR convened a Task Force to establish best-practices for applications of machine learning in health economics and outcomes research (HEOR). First, we introduce machine learning that can support HEOR. Second, we review the PALISADE Checklist to guide machine learning applications in HEOR. Third, we provide case studies where machine learning could be useful over traditional HEOR approaches.
DESCRIPTION: Advances in machine learning offer tremendous potential benefits to patients. The Task Force identified five methodological areas where machine learning could enhance accuracy of HEOR findings that we will review: (1) improve cohort selection—identifying samples with greater specificity based on inclusion criteria; (2) identifying independent predictors and covariates of health outcomes that extend beyond covariates identified in the literature; (3) improve predictive analytics of health outcomes, including those that are high cost or life threatening; (4) improvements in causal inference using targeted maximum likelihood estimation or double-debiased estimation to produce reliable evidence more quickly and eliminating the need for costly, time-consuming randomized controlled trials; and (5) be applied to development of economic models to reduce structural, parameter and sampling uncertainty in cost-effectiveness analysis. Furthermore, we examine whether machine learning offers consistently interpretable and transparent solutions to healthcare analytics. Then we will review the PALISADE Checklist, which the Task Force developed as a guide for key considerations that machine learning can offer in balance with the need for transparency in findings that differentiate patient care in the future. Finally, we will provide handouts for a series of case studies with applications to the Checklist so that the audience can determine if machine learning provides enhancement over traditional HEOR methods. We will engage the audience directly in the last step using Poll Everywhere to solicit feedback from attendees regarding remaining gaps in the PALISADE Checklist and any barriers to adopting of machine learning in HEOR.
Discussion Leaders
William Padula, PhD, MS, MSc
University of Southern California, Los Angeles, CA, USA
William Padula, PhD is assistant professor of pharmaceutical & Health Economics at the University of Southern California School of Pharmacy, and a Fellow in the Leonard D. Schaeffer Center for Health Policy & Economics. His research interests include medical cost-effectiveness analysis and applications of machine learning to health economics and outcomes research. He was the 2021 recipient of ISPOR’s Bernie O’Brien New Investigator Award, and Is an Associate Editor for Value in Health.
Discussants
Blythe Adamson, PhD, MPH
Flatiron Health, New York, NY, USA
Blythe Adamson is Principal Scientist in Machine Learning at Flatiron Health and Founder of Infectious Economics. She uses health economics, math, epidemiology, and data science to research and identify high-value medicines in development. Her research includes dynamic transmission modeling of infectious diseases, electronic medical records analysis in oncology, and cost-effectiveness studies to inform policy. Dr. Adamson received her PhD in Pharmacoeconomics and Masters in Public Health in Epidemiology from the University of Washington in Seattle. She served on the White House COVID Task Force in 2020. Prior to Flatiron, she worked on the development of HIV vaccines at Fred Hutchinson Cancer Research Center and informed Gates Foundation investing decisions with the Institute for Disease Modeling at Global Good.
William H. Crown, PhD
Brandeis University, Waltham, MA, USA
Dr.Crown is a Distinguished Research Scientist in the Heller School of Social Policy and Management, Brandeis University. He is an internationally recognized expert in real world data analysis, focusing upon research designs and statistical methods for drawing causal inferences from transactional health care datasets such as medical claims and electronic health records. Dr. Crown was 2013-14 President of ISPOR and currently co-chairs the ISPOR Task Force on Machine Learning. He is particularly interested in the intersection of machine learning and causal inference methods, as well as transparency in the conduct and reporting of empirical health care research.
David Vanness, PhD
Pennsylvania State University, University Park, PA, USA
Systematic Approaches to Assessing Physician Decision-Making Practices and Treatment Preferences
Virtual
Level: Foundational
PURPOSE: With an increasingly competitive landscape for novel therapies, understanding how physicians make choices between available alternatives has become critical for pharmaceutical manufacturers, payer organizations and regulatory agencies alike. Whether and how quickly new therapies are adopted by physicians is often unclear upon market launch, and there is both a lack of available data on physician preferences driving clinical decision-making and inattention to the broader clinical ecosystem facilitating or inhibiting uptake. With the FDA Patient-Focused Drug Develop program, there has been an emphasis on incorporating patient voices and preferences into value assessment for some therapies, and some of the methods developed to elicit patient preferences can be applied to understand provider decision-making as well.
DESCRIPTION: This workshop will introduce various mixed-methods research techniques used to systematically elicit physician decision-making practices and criteria used to select between treatment alternatives. Dr. Batt will provide an introduction, along with an overview of key areas where understanding physician deliberations and decision-making around treatment is critical for payers, manufacturers and health care systems (10 minutes). Dr. May-Slater will outline how qualitative research methods such as physician interviews, focus groups, and implementation sciences approaches can be used to inform quantitative approaches to elicit physician preferences. She will also discuss discrete choice experiments (DCE) as ways to assess how physicians trade-off between different treatment attributes in making treatment recommendations (20 minutes). Dr. Graf will discuss the use of expert elicitation methods such as the Delphi, Sheffield Expert Elicitation Framework (SHELF) and Multi-Criteria Decision-Making (MCDA) frameworks to understand how physicians interpret emerging evidence and incorporate novel therapeutic options into treatment recommendations (20 minutes). Drs. Batt and May-Slater will then facilitate an interactive exercise, asking workshop participants to brainstorm how provider preferences can be used to inform decision-making for two rare disease case studies (10 minutes).
Discussion Leaders
Katharine Batt, MD, MSc
Independent Consultant, Raleigh, NC, USA
Katharine Batt is a healthcare consultant and hematologist with specialties in hemophilia, bleeding/clotting disorders, thrombosis, hematologic malignancies and healthcare architecture and design. She has consulted on a range of clinical and health economic studies in hematology, including ones in rare and ultra-rare disease.
Discussants
Batul Electricwala, PhD
Novartis, Farmwood, NJ, USA
Marlon Graf, PhD
PRECISIONheor, Los Angeles, CA, USA
Marlon Graf is a Senior Research Economist at PRECISIONheor, with expertise in applied econometrics and mixed-methods research. He has conducted qualitative and quantitative analyses on many policy issues, including alcohol and crime control, innovation, technology and economic growth, skills development and workforce training, as well as financial decision-making and higher education finance. Dr. Graf holds a B.Sc. in business administration from the University of Mannheim (Germany), an MPP from UCLA, and a PhD in policy analysis from the Pardee RAND Graduate School.
Suepattra G. May, PhD, MPH
Precision Health Economics, Los Angeles, CA, USA
SEE Sense: Tools for Conducting Structured Expert Elicitation to Support Healthcare Decision Making
Virtual
Level: Intermediate
PURPOSE: To conduct a live demonstration of resources developed to support structured expert elicitation (SEE) in healthcare decision making, using case studies as examples.
DESCRIPTION:
In a landscape of accelerated approvals, a less mature evidence base and increasing reliance on real world evidence, obtaining judgements from clinical experts is becoming increasingly important to inform healthcare decision-making (HCDM). In the past, expert elicitation has generally been unstructured in nature, but growing demand has led to a desire to improve the robustness of the methods used. For example, NICE recently released its new draft manual for health technology assessments (HTA), which states a clear preference for SEE when gathering information from experts. Globally, SEE is starting to be used more widely, and it is expected that this form of elicitation will be increasingly used to inform economic analyses. In light of this anticipated change, we will provide an interactive demonstration of resources to support SEE in HCDM from two case studies, and introduce ongoing work to create an online repository of user-friendly open-source materials for SEE. Laura Bojke and Dawn Lee will provide a general introduction to SEE (15 minutes). Laura Bojke will discuss the SEE process and its role in HCDM. Dawn Lee will provide an overview of currently available resources for conducting SEE and highlight any practical challenges. Dina Jankovic will provide information on the development of the online repository (15 minutes). Dina Jankovic and James Horscroft will then conduct a live demonstration of the elicitation tools, giving real-life examples using recent case studies, and providing tips on how these tools can best be used (15 minutes). Polls will be used to seek input to inform the final contents of the online repository, followed by a Q&A session and discussion on application of the resources (15 minutes).
Discussion Leaders
Dawn Lee, MMath, MSc
BresMed Health Solutions Ltd., Sheffield, DBY, United Kingdom
Discussants
Laura Bojke, PhD
University of York, York, United Kingdom
Laura is a Professor of Health Economics from the Centre for Health Economics (CHE), University of York, UK. Laura Bojke has over 20 years experience in economic evaluation. Laura has worked on a wide range of applied and methodological projects, within pharmacoeconomics and public health. She has gained extensive cost-effectiveness modelling experience through her work as part of the evidence review group for NICE and worked on a number of projects involving the use of expert elicited data within decision analytic models.
James Horscroft, PhD
Lumanity, Sheffield, United Kingdom
James has 6 years of experience in providing health economics and outcomes research support for health technologies. Most of his work focuses on health technology assessment strategic planning and gathering evidence from experts, for example, via structured expert elicitation. He has experience in designing and conducting qualitative and quantitative expert elicitation/opinion studies, including the Sheffield Elicitation Framework, Delphi panels, and burden of illness studies. James graduated from a collaborative PhD in Physiology between the University of Cambridge and Zealand Pharma in 2016.
Dina Jankovic, PhD
University of York, York, United Kingdom
Dina Jankovic is a Research Fellow at the Centre for Health Economics, University of York, UK. Dina has worked on a range of applied cost-effectiveness analyses and methodological research, with special interest in using expert elicitation to support healthcare decision-making.
Podium Session 1
Tackling Reimbursement Challenges to Fair Access to Medicine
In-person
Moderator
Soumana Chamoun Nasser, PharmD, MHE
Lebanese American University, Byblos, Lebanon
Soumana Chamoun Nasser earned her BS degree in pharmacy from Massachusetts College of Pharmacy and Allied Sciences in 1998; her PharmD degree from Rhode Island University; and a Master in Health Economics & Pharmacoeconomics from University of Pompeu Fabra, Barcelona. She worked as pharmacist at New England Medical Center, then at Massachusetts General Hospital, in Boston. In 2003, she joined the School of pharmacy at the Lebanese American University in Lebanon. She is a Clinical Associate Professor, chairperson of the Pharmacy Practice department, and the president of ISPOR Lebanon chapter. Her teaching and research are in the area of pharmacy practice and health economics.
P3: An Assessment of Barriers to Fair Access to Cost-Effective Drugs
10:15AM - 10:30AM
Beinfeld M
Objectives: The extent to which insurance coverage provides fair access to pharmaceuticals has not been evaluated systematically. In 2020, ICER developed a set of appropriateness criteria addressing dimensions of fair access including cost sharing, step therapy, and clinical eligibility. In this research, we evaluated the degree to which coverage policies from leading US insurers are concordant with these fair access criteria for a set of fairly-priced drugs. Methods: We identified 28 drugs that were deemed cost effective (below $150K per QALY or evLYG) by ICER between 2015 and 2020. We leveraged the MMIT Analytics Market Access Database to determine the 15 US commercial formularies with the most covered lives and obtain tiering and prior authorization information. We evaluated the rate of concordance of prior authorization criteria against fair access criteria in four domains: cost sharing, clinical eligibility, step therapy, and provider qualifications. Results: Overall concordance with the fair access criteria across all drugs and payers was high: 254/332 (77%) of available policies met cost sharing, 290/302 (96%) met clinical eligibility, 313/317 (99%) met step therapy, and 311/311 (100%) met the prescriber restrictions criteria. Among the criteria assessed, variation in concordance across drugs was highest for cost sharing, ranging from 54% for emcizumab to 100% for insulin degludec and rivaroxaban. Variation across formularies was notable, particularly between three-tier formularies versus those with four or more tiers. While concordance with the step therapy criteria was high, we identified significant variation in step therapy requirements. Approximately 25% of policies were not available for analysis, a major limitation of this research. Conclusions: While overall concordance with the fair access criteria was high, we identified important barriers to patient access, particularly in the domains of cost sharing and step therapy. This review represents a tool for dialogue and action to achieve fair access.
P4: What Can the US Learn from Regulatory Decisions and Health Technology Assessments of New Drugs in Other Countries?
11:00AM - 11:15AM
Pham C 1 , Le K1 , Draves M2 , Seoane-Vazquez E3 1 Kaiser Permanente, Downey, CA, USA, 2 Kaiser Permanente, Oakland, CA, USA, 3 Chapman University School of Pharmacy, Irvine, CA, USA
OBJECTIVES To evaluate regulatory decisions and health technology assessments (HTA) in Australia, Canada, and England of new drugs approved by the US Food and Drug Administration (FDA) in 2017-2020 and estimate the US treatment cost per drug.
METHODS Information on new drugs approved by the FDA in 2017-2020 was extracted from Drugs@FDA and analyzed against regulatory decisions in Australia, Canada, and England. Reimbursement recommendations were collected from the Australian Pharmaceutical Benefits Advisory Committee (PBAC), the Canadian Agency for Drugs and Technologies in Health (CADTH), and the United Kingdom National Institute for Health and Care Excellence (NICE). For drugs not recommended by an international regulatory or HTA agency, US treatment costs per year were estimated from FDA product labeling and Wholesale Acquisition Cost (WAC) listed in Redbook (IBM Micromedex). Regulatory information, HTA reports, and WAC were current as of July 2021. Descriptive statistics were conducted to assess approval concordance, reasons for negative HTA recommendations, and characteristics of drugs receiving negative assessments.
RESULTS The FDA approved 206 new drugs in 2017-2020, of which 72% were granted marketing authorization by at least one other regulatory agency at a median delay of 10.1 months following US approval. Conversely, 4 drugs (abaloparatide, betrixaban, emapalumab, pexidartinib) were refused marketing authorization due to unfavorable benefit-to-risk assessments. 40% of FDA-approved drugs evaluated by PBAC, CADTH, or NICE received negative reimbursement recommendations due to uncertainty of clinical benefit and/or unacceptably high price. Approximately half of the drugs were for oncology indications and most were approved by the FDA through expedited review pathways and/or granted Orphan Drug designation. The average US treatment cost of new FDA drug approvals receiving negative appraisals from international agencies was $150,817/year.
CONCLUSIONS Review of drug approvals and HTA agency recommendations in other countries can provide evidence to support clinical decision-making of new drugs by US health systems and payers.
P2: Faster Access to Innovative Therapies with Risk-Sharing Agreements - Cancer Medication Reimbursement Decisions from January 2012 to November 2021 in Finland
10:45AM - 11:00AM
Ihalmo P1 , Väätäinen S2 , Soini E 3 , Vandorou C4 , Mankinen P2 , Nevalainen E2 , Price M4 1 Janssen-Cilag Oy, Espoo, Finland, 2 ESiOR Oy, Kuopio, Finland, 3 ESiOR Oy, Kuopio, 15, Finland, 4 Janssen EMEA, High Wycombe, UK
OBJECTIVES: Innovative therapies usually lack long-term trial outcomes data and real-world effectiveness evidence at the time of market authorization. Due to these uncertainties their public reimbursement poses challenges for payer and market authorization holder alike. Risk-sharing agreements have been proposed to diminish these challenges. Introduced in 2017, the Conditional Reimbursement System (CRS) enabled confidential agreements with the Pharmaceuticals Pricing Board (PPB) in Finland. The CRS presents possibilities for innovative pricing models, and potentially gives access to medications that might not become available for patients otherwise. Since 2017, around 50 medications have been reimbursed through the CRS. We studied how the CRS has affected reimbursement access to innovative oncological and hematological medicines in Finland.
METHODS: The reimbursement application and decision data provided by PPB (01/2011-11/2021: 50 oral medications, 237 applications, 209 decisions, 24 ongoing processes) were analyzed through the PICOSTEPS assessment framework. The control period prior to CRS was set to 2012-2016; CRS was considered as being implemented from 2018 onwards, with 2017 considered a transition year. Outcomes included changes in reimbursement dossier approval rates and time to reimbursement between these time frames.
RESULTS: The average approval rates for the first submitted dossier increased from 35.1% in 2012-2016 to 89.3% in 2018-2021. Average time to reimbursement was shortened by 46.6% (i.e., 1 year, from 794 to 424 days) after the CRS was permitted. Compared to 2015-2016, reimbursement was obtained 1.9 years faster after CRS was fully implemented. Typically, only one application process is needed to achieve reimbursement nowadays, while previously, on average, two applications were needed (range: one to four).
CONCLUSIONS: Patient access to new cancer medicines has been drastically improved by the introduction of a flexible and innovative pricing and reimbursement model. CRS has improved access and decreased the assessment burden for new hematological and oncological medicines in Finland.
P1: Estimating US National Prescription Drug Savings from Applying Value-Based Price Caps
10:30AM - 10:45AM
Yeung K 1 , Bloudek L2 , Ding Y3 , Sullivan SD2 1 Kaiser Permanente Washington Health Research Institute, Seattle, WA, USA, 2 University of Washington, Seattle, WA, USA, 3 Agency for Healthcare Research and Quality, Rockville, MD, USA
OBJECTIVES: Drug price negotiation is being considered by the United States Congress. The National Academy of Medicine recommends that negotiated drug prices be based on the value provided by the drugs. One way to inform value-based drug pricing is to cap prices at the level necessary to achieve a given cost-effectiveness threshold. Such value-based prices (VBPs) are recorded in evidence reviews produced by the Institute for Clinical and Economic Review (ICER). The objective of this study is to estimate annual US national drug spending changes if drug prices were capped at ICER VBPs.
METHODS: We obtained drug- and indication-specific VBPs from ICER evidence reports from 2015 to 2020. We merged in drug-specific sales in 2020, as reported in manufacturer 10-Q financial filings collected by SSR Health. We calculated total drug spending before and after applying VBP caps of $100,000/quality-adjusted life-years (QALY) and $150,000/QALY. For drugs with multiple ICER-reported VBPs (due to multiple indications or multiple dosage forms), we used the highest drug-specific VBP, and lowest drug-specific VBP. We tested the change in spending before and after applying VBP using the Wilcoxon signed-rank test. VBPs were inflated to 2020 US dollars.
RESULTS: There were 73 unique drugs with manufacturer-reported sales data in 2020 that also had ICER VBPs. These drugs represented $110.4 billion in total annual US drug spending. Applying the $150,000/QALY cap resulted in reductions in total annual drug spending of $4.3 billion and $18.6 billion using the highest and lowest VBP, respectively. Applying the $100,000/QALY cap resulted in reductions in total drug spending of $36.1 billion and $44.2 billion, using the highest and lowest VBP, respectively. All changes were statistically significant at p <0.01.
CONCLUSIONS: Applying VBP caps even for this small number of ICER-reviewed drugs could result in substantial US national drug savings.
Development and Methods for Quality-of-Life, Health Utilities and Patient-Centered Measures
Virtual
Moderator
Meng Li, PhD, ScM
University of Texas MD Anderson Cancer Center, Houston, TX, USA
P5: Anchor-Based Thresholds for Meaningful within-Patient Change in the Phase 3 Trial to Evaluate Tislelizumab for the Treatment of 2/3L NSCLC
10:15AM - 10:30AM
Iaconangelo C1 , McManus S 2 , Serrano D1 , Podger L3 , MA Y4 , Zhan L5 , Tang B6 , Barnes G7 1 OPEN Health Group, Bethesda, MD, USA, 2 OPEN Health Group, Atlanta, GA, USA, 3 OPEN Health Group, London, UK, 4 BeiGene, Inc, Beijing, China, 5 BeiGene, Ltd., Emeryville, CA, USA, 6 BeiGene USA, Emeryville, CA, USA, 7 BeiGene, Ltd., Blue Bell, PA, USA
OBJECTIVES: Although recent FDA oncology guidance has emphasized the importance of an anchor-based approach for estimating thresholds of meaningful within-patient change (MWPC), there is little published work reporting results based on the implementation of this approach for legacy instruments. This research applies the anchor-based approach for estimating the MWPC thresholds for EORTC QLQ-C30 and EORTC QLQ-LC13.
METHODS: Data (N=805) from the BGB-A317-303 Phase 3 clinical trial (NCT03358875) evaluating tislelizumab for the treatment of 2/3L non-small cell lung cancer (NSCLC) was analysed. The EORTC QLQ-C30 global health/QoL status scale treatment yielding 5 anchor groups: “Deteriorated, 2 or more categories,” “Deteriorated, 1 category,” “Maintained,” “Improved, 1 category,” and “Improved, 2 or more categories.” Change from baseline at Cycle 5 Day 1 (CFBLC5D1) scores were computed for each domain. MWPC thresholds were estimated as mean and median CFBLC5D1 for each domain stratified by anchor level.
RESULTS: Due to space constraints, only the results of the QLQ-LC13 score of “Pain in Arm or Shoulder” are presented here. Fourteen (3.2%) patients were in the anchor group “Deteriorated, 2 or more categories,” and the mean and median change scores were 14.29 and 16.67, respectively. These thresholds corresponded to approximately 1/2 category change on the raw scale. Additionally, 30 (6.8%) patients were in the anchor group “Improved, 2 or more categories” and the mean and median change scores were -15.56 and 0.00, respectively. Thresholds for all domains will be presented.
CONCLUSIONS: To satisfy the FDA requirement that MWPC thresholds have a minimum value of a 1-point change on a domain score, any EORTC transformed score threshold must be 33.33 points of change or greater. Thresholds acceptable under current regulatory framework must, therefore, routinely exceed the historic EORTC thresholds of 5- to 10-point change.
P7: Elicitation of Health State Utilities Associated Progression from BCG-Unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC) Health States
10:45AM - 11:00AM
Cooper O1 , Rentz A2 , Piglowska N1 , Smith C3 , Jakobsen J4 , Catto J5 , Niegisch G6 , Ghatnekar O4 , Swinburn P 1 1 Evidera, London, UK, 2 Evidera, Bethesda, MD, USA, 3 Evidera, London, LON, UK, 4 Ferring International PharmaScience Center, Copenhagen, Denmark, 5 University of Sheffield, Sheffield, UK, 6 Heinrich-Heine-Universität, Düsseldorf, Germany
Objectives : This study was conducted to elicit utility values for the treatment of NMIBC, with the aim to understand preferences for different stages of bladder cancer for use in a cost-effectiveness model. Methods : Phase I developed and tested NMIBC health states, involving interviews with three clinicians, a patient advisor, and 30 members of the general population (as a pilot stage of the time trade-off [TTO] exercise). Interview findings were used to finalise the health states. Phase II involved utility elicitation; vignette-based TTO utility interviews conducted in-person with a sample of participants from the general population in London, United Kingdom (UK). Four chronic and one path hypothetical health states described different NMIBC scenarios. Participants were asked to rank each of the four chronic health states, followed by the TTO valuation exercise for all health states. Descriptions differed in terms of NMIBC symptoms’ severity, impacts, and mode and frequency of administration. Results : Mean age of participants (n=202) was 46; 46% were female. Mean (standard deviation [SD]) EQ-5D-5L Visual Analogue Scale (VAS) and index scores were 83.2 (12.3) and 0.89 (0.18), respectively, indicating a healthy sample. Mean utilities were 0.781 for “No high-grade recurrence”, 0.586 for “High-grade recurrence”, 0.572 for “>1-year post cystectomy”, and 0.283 for “MIBC with metastatic disease”. The path health state of “First year post cystectomy” had a mean utility of 0.288. Pairwise comparisons found statistically significant differences between utilities (p < 0.001), except the comparison between high-grade recurrence and >1-year post cystectomy (p = 0.524). There were significant differences in health state utility scores by age and employment status. Conclusions : These results demonstrate that the general public highly value delaying radical cystectomy as a treatment alternative for bladder cancer. The health state with bladder-sparing therapy elicited a distinctly higher value compared to the other health states.
P8: Patient Prioritisation of Items for the New Patient-Reported Impact of Dermatological Diseases (PRIDD) Measure: A Delphi Study
11:00AM - 11:15AM
Pattinson R 1 , Trialonis-Suthakharan N2 , Tahmasebi Gandomkari N2 , Valencia López MJ2 , Austin J3 , Augustin M4 , Bundy C5 1 Cardiff University, Ottawa, ON, ON, Canada, 2 University Medical Center Hamburg (UKE), Hamburg, Germany, 3 International Alliance of Dermatology Patient Organizations, Ottawa, ON, Canada, 4 University Medical Center Hamburg, Hamburg, UK, 5 Cardiff University, Cardiff, VGL, UK
OBJECTIVES: The Global Research on the Impact of Dermatological Diseases (GRIDD) team is developing the new Patient-Reported Impact of Dermatological Diseases (PRIDD) measure, a patient-reported outcome measure of the impact of dermatological conditions on the patient’s life. We developed a conceptual framework through a qualitative interview study with 68 patients globally to derive impact items. This study aimed to seek consensus from patients on which items to prioritise for inclusion in PRIDD.
METHODS: We conducted a modified Delphi study, consisting of two rounds, starting with outcomes from the previous interview study in lieu of an idea generation round. Adults (≥ 18 years) worldwide living with a dermatological condition were recruited through the International Alliance of Dermatology Patient Organizations’ membership network. The survey consisted of a demographics questionnaire and 263 potential impact items and was translated into German, Spanish, French, Arabic and Chinese. Quantitative data were collected using Likert-type ranking scales and analysed against a priori consensus criteria. Qualitative data were collected using free-text responses that provided participants with an opportunity to provide additional feedback, and a Framework Analysis conducted.
RESULTS: 1154 people representing 90 dermatological conditions across 66 countries participated. Items were either removed, edited or added according to the consensus thresholds and qualitative feedback . The results generated the first draft of PRIDD, consisting of 27 items across five domains: physical, psychological, social, financial and daily life.
CONCLUSIONS: This Delphi study informed the item reduction process and resulted in the first draft of PRIDD. The data triangulated and refined the conceptual framework of impact. This new measure can inform policy and clinical practice by identifying what people with dermatological conditions from around the world consider to be the most important issues impacting their lives. PRIDD has since been pilot tested with patients and is currently undergoing psychometric testing.
P6: Development of Subjective Financial Distress Questionnaire (SFDQ) for Measuring Financial Toxicity Among Cancer Patients Undergoing Radiation Therapy
10:30AM - 10:45AM
Dar M 1 , Chauhan R2 , Murti K3 , Trivedi V2 , Dhingra S4 1 National Institute of Pharmaceutical Education and Research (NIPER), Hajipur, Patna, BR, India, 2 Mahavir Cancer Sansthan and Research Centre (MCSRC), 801505, Patna, India, 3 NIPER Hajipur, HAJIPUR, India, 4 National Institute of Pharmaceutical Education and Research (NIPER), Hajipur, 844102, Bihar, Patna, India
OBJECTIVES: The screening of cancer patients at risk of developing financial toxicity is essential for better clinical outcomes and to alleviate the financial burden on cancer survivors. The aim of this study was to develop an instrument for the assessment of financial toxicity among radiation oncology patients.
METHODS: The items for scale development were generated through literature review, expert opinion and patient interviews. The underlying factor structure of the preliminary scale was evaluated by Exploratory Factor Analysis (EFA) and Confirmatory Factor Analysis (CFA). Factors were extracted based on Kaiser Criterion (eigenvalues˃1) and the items with factor loadings ˃0.5 were retained. The reliability of the final scale was assessed using Cronbach’s alpha coefficient.
RESULTS: A pool of 47 items was generated of which 17 items were selected for scale development. The preliminary 17-item instrument was administered to 142 Head & Neck cancer patients. Of these 85% were male, 65% were diagnosed with oral cavity cancers and 95% belonged to rural areas. EFA was performed on 17 item preliminary scale using principal component analysis. Three items with factor loadings ˂0.5 were removed and the remaining 14 items loaded onto three factors explaining 62.0% of the total variance in EFA. The 14 items with factor loadings of ˃0.5 (range 0.60-0.86) were retained. The results of CFA showed that Chi-square goodness of fit test [χ2 (71) = 91.42, p ˃0.001] was not significant and the χ 2 /df = 1.28 suggesting that the three factor CFA model adequately fits the data. The values of other model fit indices, RMSEA (0.045), SRMR (0.014), GFI (0.92), CFI (0.98), and TLI (0.97) also support a good model fit. The Cronbach’s α of the final 14-item scale was 0.87 demonstrating excellent reliability.
CONCLUSIONS: The SFDQ captures and integrates all the relevant domains of financial toxicity and showed excellent validity and reliability in factor-analysis.
Qualitative and Quantitative Evidence on the Patient-Caregiver Dyad
In-person
Moderator
Julia F. Slejko, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
Julia F. Slejko, PhD is an Associate Professor of Pharmaceutical Health Services Research at the University of Maryland School of Pharmacy and is Co-Director of the Patient-Driven Values in Healthcare Evaluation (PAVE) Center. Dr Slejko’s research is focused on innovative approaches for decision-analytic modeling for economic and health outcomes assessments. She holds a BA in Molecular, Cellular, and Developmental Biology from the University of Colorado Boulder. During her PhD training, she focused on pharmacoeconomics at the University of Colorado School of Pharmacy Center for Pharmaceutical Outcomes Research. Her postdoctoral training was completed at the Pharmaceutical Outcomes Research and Policy Program in the University of Washington School of Pharmacy. Prior to her PhD training, she had a 7-year career in drug discovery at Array BioPharma. Dr Slejko is co-lead of ISPOR’s Women in HEOR initiative and currently Co-Chair Elect of the ISPOR Faculty Advisor Council.
P37: A Comparison of the Newly Developed EQ-HWB-S to the ASCOT and EQ-5D-5L as an Outcome Measure for Caregivers
10:15AM - 10:30AM
Kuharic M
OBJECTIVES: The EQ-HWB-S was developed as a measure of health and well-being for patients and for carers/caregivers. This study aims to examine the properties of the EQ-HWB-S in relation to the ASCOT and compare the discriminative ability of EQ-HWB-S, EQ-5D-5L, and ASCOT based on caregiver status and caregiver burden.
METHODS: An online panel of US-based respondents completed a survey that included the EQ-HWB Experimental Version, EQ-5D-5L, and ASCOT. In addition to item-level analysis, the EQ-HWB-S was scored using a non-preference-based scoring approach. We examined distribution of responses, strength of correlation between related items/constructs, and conducted known group comparisons based on caregiver status (yes/no) and caregiver burden (<20 or ≥20 hours spent per week providing care).
RESULTS: The dataset included a total of 903 respondents (carers=193; non-carers=710). 92 respondents reported low-caregiver burden while 91 high-caregiver burden in the last 7 days. Strong associations (rs > 0.5) were found between conceptually overlapping dimensions ASCOT and EQ-HWB-S items: “Control” and “No control” or related dimensions (“Social situation” and “Lonely”/”Unsupported”). The EQ-HWB-S summary score tended to better discriminate than ASCOT index score based on caregiver status (F-ratio: 1.40, 95% Cl 0.51-5.89) and caregiver burden (F-ratio: 5.81, 95% Cl 0.71-7.83). EQ-HWB-S summary score also tended to be able to better discriminate than EQ-5D-5L index score across caregiver status (F-ratio: 2.30, 95% Cl 0.81-15.84) and burden (F-ratio: 5.81, 95% Cl 1.45-31.52).
CONCLUSIONS: Initial evidence supports the validity of the EQ-HWB/EQ-HWB-s as an outcome measure for caregivers. All measures (EQ-HWB-S, ASCOT, EQ-5D-5L) demonstrated discriminative ability among people who provide caregiving. The EQ-HWB-S tended to outperform both the ASCOT and EQ-5D, and highlights its future potential to be used as a measure in economic evaluation across sectors.
P39: The Humanistic Burden of Transfusion-Dependent Lower-Risk Myelodysplastic Syndromes on Patients and Caregivers: Qualitative Research Findings
10:45AM - 11:00AM
Díez Campelo M1 , Yucel A2 , Lord-Bessen J2 , Wayser G3 , Beusterien K 4 , DeCongelio M3 , Bulkley A3 , Iraca TA5 , Hassan AA5 , Hughes C2 , Park S6 1 Hospital Universitario de Salamanca, Salamanca, Spain, 2 Bristol Myers Squibb, Princeton, NJ, USA, 3 Cerner Enviza, Malvern, PA, USA, 4 Cerner Enviza, Washington, DC, USA, 5 MDS Foundation, Inc, Yardville, NJ, USA, 6 Centre Hospitalier Universitaire de Grenoble, Grenoble, France
OBJECTIVES:
This study elicited humanistic burden of transfusion-dependent (TD) lower-risk (LR) myelodysplastic syndromes (MDS) on patients and their caregivers.
METHODS:
US patients and caregivers were interviewed separately via telephone; patients were TD LR-MDS and received ≥2 red blood cell transfusions (RBCTs) in the past 4 months. Interviews assessed impacts of living with MDS and treatment burden from both patient and caregiver perspectives. Transcripts were coded to identify key themes.
RESULTS:
8 patients and 8 caregivers (including 5 patient-caregiver pairs) were interviewed (median ages 73.5 [range, 58–87] and 63.0 [range, 30–85], respectively). Events leading to diagnosis included routine blood work and symptomatology, with the former event having a longer time to diagnosis (medians 4.5 years [range, 2 months–8 years] and 1 month [range, 1 month–1 month], respectively). Nearly all patients felt they had not received specific MDS information from physicians at diagnosis; frequently, patients were monitored and not treated following initial diagnosis. Patients and caregivers reported fatigue, shortness of breath, and dizziness as primary symptoms. Patients focused on impacts of MDS/RBCTs on emotions, social events, travel, physical activities, and relationships. Within pairs, caregivers appeared more likely to recognize patient-identified impacts on emotional health, e.g., depression, versus physical activity. RBCTs impose significant burden on patients and caregivers, who reported transfusions every 1 to 6 weeks, taking a median of 5.8 hours (range, 5.2–7.0), including travel, chair time, etc. Some expressed concerns about iron overload risk and potential major organ damage. Caregiver impacts included limiting physical and social events, less time with friends and family, and being unable to work.
CONCLUSIONS:
MDS has wide-ranging impacts on physical, emotional, and social well-being of patients and caregivers, with significant burden imposed by transfusion. Reducing transfusion dependence may alleviate the humanistic burden of TD LR-MDS on patients and caregivers.
P40: Cross-Sectional Assessment of Tardive Dyskinesia Work Productivity and Activity Impairment in Patients and Caregivers
10:30AM - 10:45AM
Jain R1 , Ayyagari R2 , Goldschmidt D3 , King S3 , Leo S 4 1 Texas Tech University School of Medicine–Permian Basin, Midland, TX, USA, 2 Analysis Group Inc., Boston, MA, USA, 3 Analysis Group, Inc., Boston, MA, USA, 4 Teva Branded Pharmaceutical Products R&D, Inc., Global Health Economics and Outcomes Research, Hoboken, NJ, USA
Objectives: Tardive dyskinesia (TD) is an iatrogenic, hyperkinetic movement disorder with substantial impact on patients and caregivers. This study assessed the impact of TD on the professional lives of patients and caregivers in the United States. Methods: Patients with TD and caregivers of patients with TD provided responses regarding the impact of TD movements/caregiving on their work productivity and, in the case of caregivers, on the patients’ work productivity using the Work Productivity and Activity Impairment Questionnaire. Data are summarized descriptively overall and by underlying disease (schizophrenia, major depressive disorder [MDD], and bipolar disorder). Results: Overall, 269 patients (mean [SD] age, 40.6 years [9.9]; 57.6% bachelor’s/graduate degree; 71.7% employed) and 162 caregivers (40.0 years [9.8]; 74.7% bachelor’s/graduate degree; 84.0% employed) were surveyed. In addition, caregivers provided information for their patient (62.6 years [15.4]; 52.5% bachelor’s/graduate degree; 4.9% employed). Among patients who were employed (59.9% full-time, 11.5% part-time, 3.3% self-employed), TD movements caused them to miss 29.1% of work time (ie, absenteeism), experience 68.4% impairment while working (ie, presenteeism), and have 73.5% overall work impairment. Among employed caregivers, providing care for a patient with TD caused them 13.8% absenteeism, 44.0% presenteeism, and 49.5% overall work impairment. Patients with underlying schizophrenia experienced the greatest impact on work productivity measures, and caregivers for patients with underlying MDD reported the greatest impact on their work productivity. Conclusions: These results demonstrate that TD imposes a substantial burden on patients’ and caregivers’ professional lives. This analysis indicates a greater burden than previously observed for patients with certain behavioral health diseases (and unknown TD status), suggesting that TD likely has an incremental impact on work productivity. These results should be taken into consideration when modeling the societal burden of TD.
Concurrent Breakout Session 1
What Is the Preferred Approach to US Drug Pricing Reforms: International Reference Pricing or Value Based Pricing?
In-person & Virtual
ISSUE: The call for drug pricing reform in the United States has amplified over the past two administrations and legislation has been drafted, though not passed. IRP and VBP based on cost-effectiveness are leading the proposed reform conversations, however, it is unclear how much impact these reforms would have on actual drug pricing, patient access and affordability, value and innovation. Using an analysis comparing IRP and VBP for 23 drugs across 28 indications as a baseline understanding of the approaches, the panel will debate the results and their preferred approaches to reforms in the US.
OVERVIEW: The moderator will provide context on the current US pricing reform discussion outlining the current policy proposals, including IRP and VBP. Margaret Labban will share results from a recent analysis comparing IRP vs. VBP to set the stage for the debate. Each panelist will briefly present their perspective on US drug pricing reform. The moderator will facilitate the debate on what US drug pricing reform should look like in order to facilitate patient access and affordability without stifling innovation. The moderator will incorporate questions from the audience to enrich the debate.
Moderators
Ashley Jaksa, MPH
Aetion, Inc., Boston, MA, USA
Nirosha Mahendraratnam Lederer, PhD, MSPH
Aetion, Washington, DC, USA
Nirosha Mahendraratnam Lederer, PhD is Head of US Federal Government and Senior Director of Real-World Evidence Strategy at Aetion. In this role, she leads partnership opportunities with the US federal government and advises clients on real-world evidence trends. Dr. Lederer has over 15 years of research experience using real-world data to support high-value decision-making in the US healthcare system. Before joining Aetion, she led the real-world evidence portfolio at the Duke Margolis Center for Health Policy including developing policies and strategies for increasing the usability and acceptance of RWD and RWE for regulatory and payment decision-making. She previously served as Subject Matter Expert in Patient-Focused Drug Development at the US FDA Oncology Center of Excellence where she supported the use and evaluation of novel drug development tools. Prior to FDA, Dr. Lederer worked at Avalere Health as Manager of Health Economics and Outcomes Research / Evidence-based Medicine Policy. In this capacity, she created global evidence generation programs as well as led policy development and government engagement strategies for Fortune 100 healthcare companies. Earlier, Dr. Lederer served on Capitol Hill with the House Committee on Ways and Means Subcommittee on Health during the passage of the Affordable Care Act.
She received her PhD in Health Outcomes and Policy from the UNC Eshelman School of Pharmacy. She also received her MSPH in Health Policy and Management from the Johns Hopkins Bloomberg School of Public Health and BA in Public Health from the Johns Hopkins University.
Panelists
Randy Burkholder, BA
PhRMA, Washington, DC, USA
Marianne Hamilton Lopez, PhD, MPA
Duke-Margolis Center for Health Policy, Washington, DC, USA
Margaret Labban, PhD
Global Pricing Innovation, London, United Kingdom
Margaret Labban, PhD is the Pricing and Market Access Solutions Manager at Global Pricing Innovations (GPI), delivering strategic solutions and products to pharmaceutical clients. As part of this role, Dr. Labban is the subject matter expert on the commercial team, and oversees all thought leadership activities at GPI. She has over 15 years of experience leading successful research projects across a range of disciplines, including 9 years focused on pricing, reimbursement and market access for the Life Sciences sector. Her areas of specialty include pricing data and analytics, global drug pricing policies and quantitative assessment of major reforms, and market access strategies. She has extensive experience working cross-functionally with health economists, data scientists and consultants on data-driven insights and thought leadership pieces. Prior to joining GPI, Dr. Labban was a Product Manager at IHS Markit (now part of S&P Global), working within the commercial team in the Life Sciences business to develop product enhancements, thought leadership activities and cross-disciplinary projects. Dr. Labban holds a doctorate degree in Neuroscience from McGill University, along with a graduate degree in Business Administration from the John Molson School of Business, Canada.
No Label, No Interest: How Should Sponsors Integrate Patient Experience Data Not Intended for Labeling in New Drug Applications?
In-person
ISSUE: PFDD guidance from FDA has supported the incorporation of the patients’ voice throughout drug development – to improve study designs, justify endpoints, understand priorities and preferences, and provide information on their experiences and outcomes. The FDA has a clear pathway for considering clinical outcome assessment (COA) data in drug labeling, but whether and how the other data generated from patients can be shared with FDA, and to what end, remains unclear. For example, a sponsor may conduct patient focus groups to inform study design and incorporate the resulting information in protocols that improves patient retention or recruitment. Is this appropriate data to share with FDA to demonstrate the patient-focused nature of the development program? Sponsors are increasingly submitting such data to the FDA in a consolidated “Patient Experience Dossier” in a marketing application submission which can become large and overwhelming. It may be more appropriate to identify when this information should be submitted, or whether the information should be integrated throughout the application. These are open questions that will be debated during this Issue Panel.
OVERVIEW: Joy Whitsett will moderate the issue panel, providing a brief presentation of PED and standard timings for submission of data to FDA. Each panelist will thereafter be asked what PED should be shared with FDA, and when and how to share in a way that both meets needs and expectations. Panelists will respond based on their role – Robyn Bent as an FDA recipient of PED, Robyn Carson as a Sponsor that has incorporated patient-focused drug development into product pipelines, and Bellinda King-Kallimanis as a patient advocate. The panel will also discuss how PED can be used by regulators and manufacturers to make patient-focused drug development a reality and offer reasons for and against a single “Patient Experience Dossier” in the NDA submission.
Moderators
Joy Whitsett, BS
IQVIA, New York, NY, USA
As Regulatory Director for the Patient Centered Solutions team at IQVIA, Joy provides leadership in integration of the patient voice into effective data-driven regulatory strategies. Joy has a broad industry background as a regulatory and quality professional. She has held leadership positions in multiple drug development programs with a consistent focus on the patient in all aspects, including heading up regulatory programs with patient experience data as a primary measure of effectiveness, establishment of quality systems in drug product manufacturing and oversight of clinical compliance for late-stage trials.
Panelists
Robyn Bent, MS
Food and Drug Administration, Silver Spring, MD, USA
Robyn Bent joined the US FDA in 2019 as the director of the Patient-Focused Drug Development (PFDD) Program in the Center for Drug Evaluation and Research (CDER). PFDD is an effort to systematically obtain patient input and facilitate the incorporation of meaningful patient input into drug development and regulatory decision making. Prior to joining FDA, Robyn held several positions at the National Institutes of Health. Captain Bent has extensive experience in clinical trial design, conduct, and oversight. Robyn earned her Bachelor of Science in Nursing from The Catholic University of America and her Master of Science degree from the George Washington University.
Robyn Carson, MPH
AbbVie, Madison, NJ, USA
Robyn T. Carson, MPH, is Vice President & Head of Patient-Centered Outcomes Research at AbbVie where she leads a team focused on integrating the patient voice through generation of patient experience data (PED) and ensuring deployment of best practices for clinical outcome assessment (COA) development, validation, and implementation across therapeutic areas. Robyn has driven patient-centered research in the pharmaceutical industry for 16 years where she has held roles of increasing responsibility at Pfizer, Forest Labs, Allergan and AbbVie.
Ms. Carson has made significant contributions to major product approvals and launches, as well as development of multiple novel patient-reported outcome (PRO) instruments and innovative real-world research platforms. In addition, Robyn has led key departmental and enterprise-wide initiatives related to patient-focused drug development (PFDD), patient-centricity, and equity, equality, diversity, and inclusion. Throughout her career, Robyn has been a dedicated mentor of junior researchers with a track-record of building highly productive teams who deliver high-quality and impactful research.
Robyn has also been a leader within the C-Path PRO Consortium since 2008, co-leading the IBS and FD Working Groups (WG). Robyn is a seasoned researcher with over 30 peer-reviewed articles and 100 research presentations. She has been an invited speaker to represent industry on PFDD, PED, COAs, and advancing novel methodologies for patient-centric outcomes research.
Prior to joining the pharmaceutical industry, Robyn conducted research at Columbia University, NYC Department of Health & Mental Hygiene, and served as a Research Fellow at the National Cancer Institute. Robyn holds a MPH in Epidemiology from Columbia University.
Bellinda L. King-Kallimanis, PhD
LUNGevity, Bethesda, MD, USA
10:15 AM - 11:30 AM
Signal Series
An Introduction to Cost Analysis Methods Using Large/administrative Claims Databases
Medicare’s Maximum Fair Prices for the First 10 Negotiated Drugs and Anticipated Cost Savings
New Analytical Approaches to 21st Century Challenges
In-person & Virtual
We live in a connected world of coupled dynamic systems. Epidemic dynamics, economic contagions, network transmission of poor information, human emotions and behaviours are producing concurrent and mutually amplifying shocks to global systems. The standard approaches of economics are inadequate to explain, to forecast, or to control this complex system. New science, modelling and analysis tools are essential to understand these coupled nonlinear systems and to inform policy.
This session is envisioning and discussing the approaches needed to analyse the many, often irrational-seeming, behaviours that are generated by the myriad interactions of billions of people, firms and institutions locally or globally, in small groups or as nations, at timescales ranging from nanoseconds (as in computer trading) to millennia (as in evolution).
Building on the expertise and capacity of an inter-disciplinary panel of neuroscientists, engineers and economists, this ISPOR Signal event will discuss innovative approaches for understanding the complexity of people, human-made systems and our economies and societies and how to ensure their adaptability, recovery and resilience. This involves applying insights and methods from behavioural neuroscience, contagion dynamics, network science, econophysics, and mathematics to policy questions spanning public health, economics, inequality, conflict, and other volatile dynamics.
Hosts
Laura Pizzi, PharmD, MPH, RPh
Rutgers University, Piscataway, NJ, USA
Dr. Laura Pizzi is Associate Chief Science Officer for ISPOR and Professor at Rutgers University in the schools of pharmacy and public health. For the past 25 years, she has led interdisciplinary teams of methodologists, statisticians, and clinicians to design and conduct economic analyses on healthcare interventions and is a frequent author, speaker, and mentor on the topic.
At ISPOR, she provides leadership to the organization’s scientific strategy and initiatives, including content planning and oversight of the Special Interest Groups, Patient Council and roundtables, the ISPOR Competency Framework workgroup, Publications Council, Institutional Council, and Digital Health Strategy. She also liaises with the Student Network and Faculty Advisor Council to support their scientific needs.
Speakers
Harris Eyre, MD, PhD
Organisation for Economic Cooperation and Development (OECD)-PRODEO Institute Neuroscience-inspired Policy Initiative, -, CA, USA
Harris co-leads the Organisation for Economic Co-Operation and Development (OECD)-PRODEO Institute Neuroscience-inspired Policy Initiative (aka NIPI) supported by the Meadows Institute in North America. His work centers on advancing innovative and transformative economic, policy and investment approaches to the global brain crisis driven by mental health challenges, addiction, neurodegenerative diseases, and other treatable and preventable brain health conditions.
Informed by his broad and diverse base of experience, Harris is advancing a coordinated set of strategies centered on the fact that our brains are arguably our biggest assets as individuals and communities. Brain Capital is a novel economic measure which integrates brain health and brain skills.
Harris operates in the pursuit of boosting brain health and brain skills. He achieves this by partnering with a wide range of brain health innovators to advance convergence between neuroscience, medicine, business, economics, technology, diplomacy, social sciences and the arts.
Across his career, he has operated as a physician, scientist, entrepreneur, executive services provider, author, new economic and finance thinker, and neuroscience diplomat. He also currently serves as President of PRODEO, a brain health technology executive services group. His work has been written up in Neuron, Forbes, the Financial Times, STAT and the Financial Post. He is on the Strategic Advisory Board of the Heka Fund, on the Founding Strategic Committee of Brain Health Nexus of Cohen Veterans Bioscience, a member of the Champion’s Cabinet of the Davos Alzheimer’s Collaborative, a member of Project Value Alzheimer’s Europe, and a scientific advisor to the Tropical Brain and Mind Foundation.
He maintains advisory or adjunct roles with the American Psychological Association, the Global Brain Health Institute, the Texas Medical Center, the American Association of Geriatric Psychiatry, Baylor College of Medicine, BrainLat, the Brookings Institution, and IMPACT at Deakin University.
He is an alumnus of Forbes 30 Under 30 and the Fulbright Scholar program. In 2021, he was awarded an EB1A Greencard, an honor typically reserved for Nobel and Pulitzer Prize winners. Harris has authored 120+ papers. He co-edited the book ‘Convergence Mental Health’ (Oxford Press).
Julian Karaguesian
McGill University, Montreal, QC, Canada
Benjamin Trump, PhD
U.S. Army Engineer Research and Development Center (ERDC), Boston, MA, USA
11:15 AM - 1:15 PM
Lunch
In-person
11:45 AM - 12:45 PM
Educational Symposium
Advances in the Development and Application of Real-World Evidence: Learnings from the US and China
In-person & Virtual
Major strides have been made to improve the availability and applicability of real-world data (RWD). However, generating reliable and timely real-world evidence (RWE) is a multifaceted process facing many challenges globally. This symposium will introduce several creative approaches to generating high-value RWD, showcase patient-centric data innovations, and discuss the application of RWE to support regulatory submissions and payer negotiations. First, to augment patient-centric care, an increasing number of patient support programs (PSPs) have been implemented by pharmaceutical companies. The presenters will demonstrate how to generate high-quality RWE that leverage PSPs. Both early evidence and long-term RWE in patients’ quality-of-life and clinical benefits post product launch can be reliably developed through this approach to demonstrate the real-world value of innovative treatments. Second, two case studies will be presented to illustrate how to generate high-quality RWE in China, where RWE research has traditionally faced many challenges. In one example, researchers developed and validated algorithms to generate high-quality RWD in order to study complex patient journey in a hematologic condition based on China’s National Longitudinal Cohort of Hematological Diseases (NICHE). In another example, researchers applied a creative design of a multi-center physician survey to generate reliable RWE to support China’s National Reimbursement Drug List negotiation. Lastly, the presenters will discuss considerations in the creation of a historical control arm to support regulatory approval. We hope this symposium can introduce these recent creative solutions in generating and applying RWD to the audience and stimulate discussions to further advancing the RWE research and methodologies.
Sponsor
Analysis Group
Moderators
Eric Wu, PhD
Analysis Group, Inc., Boston, MA, USA
Dr. Eric Wu, a Managing Principal at Analysis Group, is a health economist with expertise in health economics and outcomes research (HEOR), market access, and scientific evidence strategy. He has conducted research in more than 30 countries on behalf of pharmaceutical and medical device companies, payers, providers, and government agencies. Dr. Wu has contributed to over 200 publications across dozens of therapeutic areas, including regenerative therapies (gene and stem cell), rare and ultra-rare diseases, biologics, and immuno-oncology. He spends a significant portion of his practice developing new scientific methods to address challenges in health care research. Dr. Wu has developed client-focused solutions based on the use of artificial intelligence (AI), medical big data, real-world evidence, and innovative comparative-effectiveness research methodologies.
Speakers
L J Wei, PhD
Harvard School of Public Health, Boston, CA, USA
Min Yang, MD, PhD
Analysis Group, Boston, MA, USA
Dr. Min Yang, a Vice President at Analysis Group, specializes in health economics and outcomes research (HEOR). She works closely with pharmaceutical, biotech, and device companies to develop HEOR strategies. Dr. Yang also designs and conducts studies from pipeline product development through product launch, post-market research, and biosimilar evaluation. She has extensive experience with clinical trial data, health insurance claims databases, electronic medical records, and primary surveys for evidence generation through both clinical trials and real-world data with conventional and innovative methodologies. Dr. Yang is an expert in clinical outcome assessments – such as patient-reported outcomes (PRO) and clinician-reported outcomes (ClinRO) – as well as health preference research. She has supported the development and validation of multiple PROs and ClinROs, as well as generated evidence for regulatory submission.
A frequent collaborator with academic experts and clinical key opinion leaders, Dr. Yang’s research has been published in many peer-reviewed journals and presented at numerous clinical and economic research conferences. In recognition for her work in women’s health, she has received the American College of Obstetricians and Gynecologists (ACOG)/Bayer Healthcare Pharmaceuticals Research Award. In addition to Dr. Yang’s HEOR credentials, she is a licensed oncology surgeon in China and a guest lecturer at the University of Texas at Austin College of Pharmacy. Prior to joining Analysis Group, Dr. Yang was a senior scientist at QualityMetric (now part of Optum, Inc.)
Jia Zhong, ScD
Analysis Group, Inc., Boston, MA, USA
Dr. Jia Zhong, a Manager at Analysis Group, is an epidemiologist who specializes in clinical health economics and outcomes research (HEOR), clinical trials, and large-scale epidemiological studies. She has more than 10 years’ research experience in disease areas such as pulmonary diseases, diabetes, cardiovascular diseases, oncology, rare diseases, and immunology. Dr. Zhong’s expertise includes comparative efficacy and safety, individualized medicine, and longitudinal analysis. Her recent work includes leading large-scale prospective cohort studies, evaluating the comparative efficacy of immuno-oncology treatments for regulatory submissions, quantifying direct and indirect resource utilization and costs, and evaluating real-world effectiveness using patient reported outcomes (PROs). Her research has been published in Nature Immunology, Immunity, Proceedings of the National Academy of Sciences, Circulation, Circulation Research, Hypertension, and the International Journal of Epidemiology, among other publications. Prior to joining Analysis Group, Dr. Zhong was a research scientist at the Columbia University Mailman School of Public Health, where she developed data-based therapeutic target identification frameworks.
Discussion Groups
New this Year – Discussion Groups! Discussion Groups are facilitated conversations between conference attendees and select conference speakers. Held in the new, dynamic Discussion Lounge in the ISPOR Exhibit Hall, these discussions are intended to be highly interactive, collaborative, and promote the exchange of ideas in a peer-to-peer setting. Machine Learning Discussion Group
In-person
Moderator
William Padula, PhD, MS, MSc
University of Southern California, Los Angeles, CA, USA
William Padula, PhD is assistant professor of pharmaceutical & Health Economics at the University of Southern California School of Pharmacy, and a Fellow in the Leonard D. Schaeffer Center for Health Policy & Economics. His research interests include medical cost-effectiveness analysis and applications of machine learning to health economics and outcomes research. He was the 2021 recipient of ISPOR’s Bernie O’Brien New Investigator Award, and Is an Associate Editor for Value in Health.
ISPOR Forums
High-Cost Drugs: Experiences and Barriers to Access in Latin America
In-person
The COVID-19 pandemic has placed enormous strain on the Latin America region. Even after more than 2 years of the pandemic, healthcare systems in the region still face significant challenges, particularly related to economic crisis. In this difficult context, the demand for high-cost drugs continues, and the approaches to provide access vary considerably in different countries across the region. This forum will present some current initiatives from the region to address this issue. Dr. Stephen Stephani, Head of Oncology, UNIMED Central RS will present from a clinician perspective, discussing the current cancer care situation in Latin America, key challenges for patient access to oncology medicines, particularly financial toxicity, and explore some strategies on how to bend the increasing cost curve in the region. Professor Diego Rosselli of Pontificia Universidad Javeriana will discuss about the financing mechanisms for high-cost drugs in Colombia, access, barriers, and factors influencing access to innovative drugs. He will also share Colombian experience on how to enhance patient access by using real-world data to evaluate access to cancer care and implementation of value-based pricing for medicines. Dr. César Cruz, HTA Director of National Health Council will share Mexico’s experience from the last 10 years in consolidated purchase of medicines, medical devices, and other supplies for health, and will discuss about high-cost drugs plans and the government involvement in pay for performance.
Moderators
Yajaira Bastardo, PhD
Central University of Venezuela, Caracas, Venezuela
Diego Rosselli, MD, MEd, MHP
Pontificia Universidad Javeriana, Bogota, Colombia
Diego Rosselli is a Colombian neurologist trained at the London Institute of Psychiatry. He began his academic career as a neuroscience teacher. After obtaining his Master’s degree in Education in Harvard in 1993, he joined the Colombian Ministry of Health as Director for Scientific and Technological Development in charge of performing health technology assessments. He then obtained a second master's degree in Health Policy at the London School of Economics. Since then (1997), he has been teaching Health Economics at the Universidad Javeriana, in Bogotá. Rosselli has authored 7 books and more than 100 scientific articles in many aspects of clinical epidemiology, real-world evidence and health economics. He has lectured in pharmacoeconomics in 17 Latin American countries. He is currently the 2018-2020 Chair of the ISPOR Latin America Consortium Executive Committee and Colombia Chapter past president.
Speakers
Cesar Alberto Cruz Santiago, MSc, PhD, MD
National Health Council, Mexico City, Mexico
Stephen Stefani, MD, MBA
UNIMED Central RS, Porto Alegre, Brazil
Measuring Health Preferences of Multiple Stakeholders: Issues and Challenges
In-person
Preference information can inform and facilitate decision making in a wide variety of contexts including benefit-risk assessment, economic analyses (eg, cost-effectiveness analysis, cost-benefit analysis), clinical product development (eg, target product profile development, clinical trial design, etc.), and shared decision-making. Many decisions could be informed by the study of the preferences across multiple stakeholders, including patients, health care providers, caregivers, parents, children, and/or payers.
The Health Preference Research Special Interest Group-sponsored forum will be organized into three parts. First, we will describe motives for measuring preferences from multiple stakeholders in the context of different types of health decision-making. A range of health-related decision contexts that are relevant in various jurisdictions will be included, including, for example, economic analyses and benefit-risk assessment. The rationale for multiple sets of preferences may be related to the theoretical foundations for determining whose preferences to measure, and stakeholders’ roles in treatment decisions or the capabilities of the stakeholder (age, decision-making capacity/executive function). Second, we will discuss some of the survey design and measurement issues that must be considered when designing survey instruments that are both appropriate for a particular study population and clinically relevant. Reading level, terminology, presentation of quantitative information, and survey complexity should be adapted for the study population. The implications for these adaptations for the relevance of the preference results and the comparability of the results between stakeholder populations will be discussed. Third, we will debate how decision makers may interpret studies indicating either concordant or discordant preferences between different stakeholders and discuss how results might be interpreted to inform decision making. We will illustrate using examples of studies measuring preferences from multiple stakeholders. Finally, we look forward to finding out what you think via polling throughout the forum and the Q&A session.
Moderators
Juan Gonzalez, PhD
Duke Clinical Research Institute, Cary, NC, USA
Dr. Gonzalez is an Associate Professor in the Department of Population Health Sciences at the Duke University School of Medicine. He is an expert in the design of stated-preference survey instruments and the use of advanced statistical tools to analyze stated-preference data. His research has focused on two main areas: 1) transparency in benefit-risk evaluations of medical interventions, and 2) elicitation of health preferences from multiple stakeholders to support shared decision making.
Dr. Gonzalez Co-led the first FDA-sponsored preference study. The study was highlighted in FDA’s recent precedent-setting guidance for submitting patient-preference evidence to inform regulatory benefit-risk evaluations of new medical devices. More recently, Dr. Gonzalez collaborated with the Medical Devices Innovation Consortium (MDIC) to prepare the first catalog of preference-elicitation methods suitable for benefit-risk assessments of medical devices. The catalog was part of the Patient-Centered Benefit-Risk Assessment Framework developed by MDIC. As a core group member of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Conjoint Analysis Task Force, Dr. Gonzalez helped draft good-practice recommendations for statistical analysis, interpretation, and reporting of health preference data. Dr. Gonzalez is the current Chair of the ISPOR special interest group on stated-preference research.
Speakers
John Bridges, PhD
Ohio State University College of Medicine, Columbus, OH, USA
John F.P. Bridges PhD is a professor in the Departments of Biomedical Informatics and Surgery within the Ohio State University College of Medicine. Working at the intersection of medicine and the social sciences, John advances and applies methods to incorporate the priorities and preferences of patients and other stakeholders in medical decision making. John was founding editor of The Patient – Patient Centered Outcomes Research (since 2008) and serves on the editorial boards of Pharmacoeconomics (since 2006), Expert Review of Pharmacoeconomics and Outcomes Research (since 2007), and International Journal of Technology Assessment in Health Care (since 2008). Within the ISPOR he was the founding chair of the Conjoint Analysis Working Group and the Conjoint Analysis Task Force that produced several reports on good research practices for stated-preference methods. He received ISPOR’s Bernie O’Brien New Investigator Award in 2006 and ISPOR’s Distinguished Service Award in 2011. He is the author of over 200 articles and a frequent speaker on the art and science of using stated-preference methods and engaging patient organizations in decision making. John is currently affiliated with Ohio State University Comprehensive Cancer Center’s Cancer Control Program, the Center for the Advancement of Team Science, Analytics and Systems Thinking in Health Systems Research and Implementation Science (CATALYST), and the Center for Surgical Health Assessment, Research and Policy (SHARP). He is also an adjunct professor within the Department of Health Behavior and Society at the John Hopkins Bloomberg School of Public Health.
Eric Andrew Finkelstein, PhD, MHA
Duke-NUS Medical School, Durham, NC, USA
Christine Poulos, PhD
RTI Health Solutions, Research Triangle Park, NC, USA
Christine Poulos, PhD is Senior Economist and Vice President of Health Preference Assessment at RTI Health Solutions, Research Triangle Park, NC, USA. She has more than 20 years of academic, research, and government experience in health and environmental economics. She is an expert in the design of stated preference surveys and she has extensive experience in applying stated preference methods to measuring the benefit-risk preferences of patients and other health care decision makers. Her most recent work has applied these studies to support decision making throughout the clinical product development cycle for treatments for conditions including severe emphysema, Alzheimer’s’ disease, multiple sclerosis, endometriosis, type 2 diabetes, and infectious diseases. She is currently Chair for the Health Preference Research Special Interest Group of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Her research has been published in numerous health outcomes and medical journals.
Open Source In Precision Medicine: The Perfect Fit?
In-person
Precision medicine promises to provide cost-effective health care solutions to stratified patient populations. A hallmark of the precision medicine approach is mapping patients’ clinical pathways to outcomes. Researchers have used flexible software environments, such as R, and discrete event simulation (DES) to reflect individual patient trajectories. Researchers may adapt these models for different biomarkers, therapies, and prevention strategies for specific diseases. Open-source modeling (OSM), which shares underlying code alongside transparent reporting, can support the iterative development of models and application to other populations. The adoption of OSM for economic evaluation may alleviate the challenge of developing complex decision models in precision medicine while raising new concerns.
The OSM Special Interest Group (SIG) has selected leaders in the field to explore the value of OSM in precision medicine. The session will consider whether precision medicine constitutes the ideal context to further open-source initiatives. Chris Sampson will introduce the topic, outlining the value of OSM practices and some perceived challenges to their adoption. Susan Snyder will provide an overview of the complexities of economic evaluation in precision medicine, describing her work developing a generic pharmacogenomic cost-effectiveness model. Deborah Marshall will use applied examples to discuss the value of open-source practices for data federation to inform analysis and modeling of clinical pathways and treatment trajectories in genomic medicine. Erik Koffijberg will share practical insights from his work using DES, including the importance of transparent reporting of methods, modular approaches to modeling, and how this can contribute to sustainable research practices. The forum will provide an opportunity to discuss the intersection of these two critical developments in decision modeling methods and practice, exploring technical aspects and strategic questions. Each speaker will present one polling question to facilitate audience participation, and at least 20 minutes of the session will be reserved for a moderated discussion.
Moderators
Christopher Sampson, PhD
Office of Health Economics, London, LON, United Kingdom
Chris is an experienced researcher with more than 10 years working in the field of health economics, focusing on applied studies in a range of clinical contexts. His primary research interests are in economic evaluation in health care, including health technology assessment policy, the development of new methods for cost-effectiveness analysis, and the valuation of health. Chris is known for his expertise in the collection and analysis of patient-reported outcomes and resource-use data and methodological developments in the measurement of health-related quality of life.
Speakers
Koen Degeling, PhD, MSc, BSc
Lumen Value & Access – a Healthcare Consultancy Group Company, New York, NY, USA
Dr Koen Degeling is a Research Scientist, Health Economic Modelling & Advanced Analytics at Lumen Value & Access, a Healthcare Consultancy Group company. He was trained as an Industrial Engineer specializing in Healthcare Technology and Management and holds a PhD in Advanced Health Economic Modelling from the University of Twente in the Netherlands. Prior to joining Lumen Value & Access, Koen worked on real-world data-driven health economic and health services research projects at the Cancer Health Services Research department of the University of Melbourne in Australia, where he continues to be involved as an honorary fellow. He is an active ISPOR member and currently serves on the Editorial Advisory Board for Values & Outcomes Spotlight and ISPOR New Professionals Steering Committee, is involved in several short courses and workshops, and has served as global chair, committee co-chair, and chapter president within the ISPOR Student Network.
Deborah Marshall, PhD, BSc
University of Calgary, Calgary, AB, Canada
Deborah is a professor at Cumming School of Medicine, University of Calgary and Arthur J.E. Child Chair in Rheumatology Outcomes Research and former Canada Research chair, Health Services and Systems Research. Her research program focuses on the measurement of preferences, cost-effectiveness analysis, and simulation modeling of health services and interventions. Deborah has over 20 years of research experience in health technology assessment agencies, academic institutions, and industry settings in Canada, US, and Europe. She is a founding co-investigator of the Patient and Community Engagement Research (PaCER) Program and co-leads the economics and stated preferences research platforms for the Canada-Netherlands Personalized Medicine Network in Childhood Arthritis and Rheumatic Disease (UCAN CANDU).
Deborah is an active member of the ISPOR as the past-president of the Board of Directors, and co-author on the three “ISPOR Task Force Reports for Good Research Practice – Checklist for Conjoint Analysis in Health, Conjoint Analysis Experimental Design and Statistical Methods for the Analysis of Discrete-Choice Experiments.”
Susan Snyder, PhD, MBA
Georgia State University, Atlanta, GA, USA
12:15 PM - 1:15 PM
Poster Session 1 Poster Tours
New this Year – Poster Tours! ISPOR has curated collections of research posters for you within each of the poster sessions. Each tour will feature high impact abstracts within a specific topical area and will include a tour guide as well as the poster authors to share their work and engage in discussions with you. Visit the Learning Formats page for more information. Poster Tour: Cardiovascular Disorders
In-person
Posters featured in this tour:
CO13: The Effect of Preoperative Chest Physiotherapy on Oxygenation and Lung Functions Among Cardiac Surgery Patients: A Randomized Controlled Study
EE199: Cost-Effectiveness of Lisinopril and Carvedilol for Prevention of Trastuzumab-Induced Cardiotoxicity in US Adults with Early-Stage Breast Cancer
EE310: Cost-Effectiveness of Initiating Antihypertensive Therapy with Single-Pill Combinations Versus Monotherapy in US Adults
HSD119: Persistence to Treatment with Initially Prescribed Antiplatelet Agents for the Onset of Acute Coronary Syndrome
MT3: Completeness and Concordance between Electronic Medical Records (EMR) and Submitted Insurance Claims in German Hospitals Utilizing Percutaneous Left Ventricular Assist Devices (PLVADS): Considerations for Outcomes Research
SA45: The Effect of Team-Based Care Strategies on Systolic Blood Pressure at Six Months: A Meta-Analysis
Poster Tour: Real-World Evidence
In-person
Posters highlighted in this poster tour:
CO21: Patient Demographics, Clinical Characteristics, Treatment Patterns, and Survival Outcomes Associated with First-Line Treated Un-Resectable Advanced, Metastatic and Recurrent Esophageal Squamous Cell Carcinoma in the U.S.
CO94: Changes in Demographic and Clinical Characteristics of Patients with Type 2 Diabetes (T2DM) Initiating Subcutaneous Semaglutide
EE203: Clinical and Economic Outcomes of Patients with Multiple Rib Fractures Treated Operatively Vs. Non–Operatively — a US Hospital Database Analysis
EPH2: Adverse Outcomes Associated with Concurrent Gabapentin, Opioid, and Benzodiazepine Utilization: A Nested Case-Control Study
HSD3: Choosing Wisely? Use of Intensity Modulated Radiation Therapy Following Lumpectomy in Early-Stage Breast Cancer: A Seer-Medicare Analysis
HSD41:2015 American Thyroid Association Guidelines and Outcomes for Patients with Thyroid Cancer
12:45 PM - 1:15 PM
HEOR Theater
Real-World Data for Comparative Effectiveness Research
In-person
This HEOR Theater will:
Leverage real-world data to generate evidence around comparative effectiveness, including RWE methods and reducing bias
Understand special considerations when using RWD for CER for regulators
Examine case examples of how large real-world datasets can generate more powerful comparative effectiveness insights
Sponsor
OM1
Speaker
Jess Paulus, ScD
OM1, Dedham, MA, USA
1:30 PM - 2:30 PM
Concurrent Breakout Session 2
Challenges in Value Assessment of Technologies for COVID-19: A Health Economics Perspective
In-person
Level: Intermediate
PURPOSE: This workshop will outline issues for the value assessment of interventions for COVID-19, including modelling challenges and the appropriateness of existing approaches. Attendees should gain insight into the key difficulties and potential ways forward.
DESCRIPTION: Early in the pandemic, economic evaluation took a back seat in healthcare decision making. Value assessments were highly uncertain, informed by scant clinical evidence, and were heterogenous, with inconsistent modelling approaches in the rapidly evolving pandemic context. As more technologies enter the decision space, health economists must address these challenges and consider whether existing principles for economic evaluation remain appropriate. Much work is already underway to do this.
In this workshop, Melanie Whittington will reflect on the experience of value assessment in a pandemic, and introduce the various workstreams that discussants will present in more detail (10`). Yumi Asukai will present findings from an expert panel of health economists, which discussed the principles of economic evaluation, including the importance of non-health benefits and implications for cost-effectiveness thresholds (12`). Dalia Dawoud will present the key barriers to modelling, identified through a roundtable workshop of modellers and a “living” systematic review of evaluations, and recommendations from the HTx best-practice guidance to overcome them (12`). Andrew Briggs will consider the challenges in modelling the long-term effects and complications of COVID-19, and future directions to do so appropriately, as part of a whole-disease model (12`). Audience Q&A will include real-time polling about modelling during a pandemic (14`). This workshop will be of interest to health economists and other HTA stakeholders.
Discussion Leaders
Melanie Whittington, PhD
Institute for Clinical and Economic Review, Boston, MA, USA
Dr. Whittington is the Director of Health Economics at the Institute for Clinical and Economic Review (ICER). In her role, she leads the cost-effectiveness analyses and potential budget impact analyses within ICER reviews, directs the content within the Interactive Modeler™, and advances the field of health economics by developing innovative methods with applications for value assessment.
Discussants
Yumi Asukai, MSc
Gilead, London, United Kingdom
Yumi is a health economist specialising in economic modelling and evaluations for health technology appraisal. She received her BA in Political Science from Stanford University, before making the move to study health economics at the London School of Economics and the London School of Hygiene and Tropical Medicine. She has since held roles at IMS Health (now IQVIA) delivering a broad range of HEOR projects and at GSK heading the global economic modelling function. She is currently Senior Director at Gilead Sciences, leading economic modelling at the Centre of Excellence in HEOR.
Andrew Briggs, DPhil
London School of Hygiene & Tropical Medicine, London, LON, United Kingdom
Andrew is a professor of Health Economics and the London School of Hygiene & Tropical Medicine. Previously, he held the William R Lindsay Chair in Health Economics at the University of Glasgow. Following a sabbatical at Memorial Sloan Kettering Cancer Center, New York in 2016/17 he remains a visiting investigator, collaborating with Dr. Peter Bach on value frameworks for oncology medications.
Andrew has expertise in all areas of health economic evaluation -- he has published over 200 articles in the peer-reviewed literature. He has particularly focused on statistical methods for cost-effectiveness analysis. This includes statistical methods for estimation of parameters for cost-effectiveness models as well as statistical analysis of cost-effectiveness alongside clinical trials. He also has a more general interest in epidemiological methods, in particular the use of prognostic scoring methods for predicting health outcomes and the relationship with heterogeneity in cost-effectiveness.
Andrew took a leadership role as co-chair of the Joint Society for Medical Decision Making (SMDM) and ISPOR Task Force on Modelling Methods. The Task Force, which was responsible for producing a set of seven papers covering all aspects of modeling methods applied to medical decision making and health technology assessment. He is also the author of two successful textbooks, one published by OUP entitled Decision Modelling for Health Economic Evaluation, and another published by Wiley entitled Statistical Methods for Cost-Effectiveness Analysis.
In addition to his academic activities, Andrew is also director & principal of Avalon Health Economics, a small consultancy company based in New Jersey, USA, which focuses on providing support to companies bringing products to market in both US and ex-US.
Dalia Dawoud, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Dalia Dawoud, PhD, is Senior Scientific Adviser at the National Institute for Health and Care Excellence (NICE). She holds MSc in Economic Evaluation in Health Care from City University London and PhD in pharmaceutical policy and economics from King’s College London.
She has long experience in using economic evaluation in clinical guidelines development and health technology assessment (HTA), gained through working on NICE Clinical Guidelines as well as technology appraisals. Dalia’s research interests are focused on the advanced methods of evidence synthesis and use in economic models and the use of real-world evidence to inform drug development and health care decision making. Dalia currently has overall responsibility of overseeing the delivery of NICE allocated tasks on a portfolio of IMI and Horizon 2020 funded research projects including EHDEN and HTx. She is widely published in the field of pharmaceutical policy and pharmacoeconomics. She also serves as Associate Editor for ISPOR journal Value in Health and as Associate Editor for Pharmacoeconomics and Outcomes Research for Elsevier’s journal Research in Social and Administrative Pharmacy. Dalia also holds adjunct position as Associate Professor at the Faculty of Pharmacy, Cairo University.
Optimizing the Use of Patient-Reported Outcomes in Clinical Trials to Inform Decision-Making: The PROTEUS Consortium
In-person
Level: Intermediate
PURPOSE: With the growing emphasis on patient-centeredness, patient-reported outcomes (PROs) are taking on increasing importance in research studies, clinical care, and policy-making. The PROTEUS Consortium (P R O T E U S
DESCRIPTION: The workshop will have four 12-minute presentations, followed by an interactive discussion with audience polling and questions-and-answers, and will be relevant to industry and academic researchers, policy-makers, and methodologists. Dr. Snyder will describe how PROTEUS and the Tools can be used to inform PRO methods for protocol writing, measure selection, analysis, reporting, and application in practice. She will also introduce the resources (e.g., checklists, web tutorials, handbook) that have been developed to facilitate dissemination and implementation of the PRO methodologic Tools. Ms. Nelsen will provide an industry perspective on how the PROTEUS Tools and resources can help address the challenges of incorporating PROs in clinical trials. Drs. Bhatnagar and Jonsson will discuss the evaluation of PRO data from the regulatory and health technology assessment perspectives, respectively, and how high-quality PRO data, as promoted by the PROTEUS Tools and resources, can inform decision-making. The workshop will conclude with a 12-minute interactive session with audience polling regarding the usefulness of the current PROTEUS Tools and resources and priorities for future resource development, as well as questions from the audience. After the session, attendees will be better positioned to conduct rigorous PRO research, thereby producing PRO data decision-makers can use to promote patient-centered care.
Discussion Leaders
Claire Snyder, MHS, PhD
Johns Hopkins School of Medicine, Baltimore, MD, USA
Claire Snyder, PhD, is Professor of Medicine, Oncology, and Health Policy & Management at the Johns Hopkins Schools of Medicine and Public Health. Dr. Snyder’s research focuses on the quality of cancer care. Along with Dr. Michael Brundage, she leads the PROTEUS Consortium (Patient-Reported Outcomes Tools: Engaging Users & Stakeholders). She is a past president of the International Society for Quality of Life Research. Previously, Dr. Snyder worked at the U.S. National Cancer Institute. She began her career at Covance Health Economics and Outcomes Services Inc. Dr. Snyder has BA from Duke University and a PhD from Johns Hopkins.
Discussants
Vishal Bhatnagar, MD
Oncology Center of Excellence, U.S. Food and Drug Administration, Silver Spring, MD, USA
Vishal Bhatnagar, MD, is a medical oncologist/hematologist and the Associate Director for Patient Outcomes in the OCE. His interests include patient reported outcomes, patient preference and incorporation of patient experience in oncology trials. His work focuses on the operational management of the OCE’s Patient-Focused Drug Development program. Additionally, Dr. Bhatnagar has a strong clinical interest in multiple myeloma and has previously served as an Office of Hematology and Oncology multiple myeloma scientific liaison. Dr. Bhatnagar received his BA in Political Science and his medical degree at the George Washington University. He completed his internal medicine residency and hematology/oncology fellowship at the University of Maryland.
Pall Jonsson, BSc, PhD
National Institute for Health and Care Excellence (NICE), Manchester, LAN, United Kingdom
Pall Jonsson is Programme Director at the National Institute for Health and Care Excellence (NICE) where he heads up Data and Analytics. His team has a strategic role in ensuring NICE is at the forefront of harnessing new and emerging opportunities for using real world data to inform NICE’s guidance to the health and care sectors.
Before joining the Data and Analytics team, he was Associate Director for Science Policy and Research, responsible for NICE’s portfolio of international research projects in areas such as big data and real-world evidence. Pall has a PhD in bioinformatics from the University College London. Prior to joining NICE, he worked in academia, biotech and the pharmaceutical industry.
Linda Nelsen, MHS
GSK, Collegeville, PA, USA
Linda Nelsen leads Oncology Patient Centered Outcomes at GlaxoSmithKline with responsibility for development of innovative patient centered outcome strategies and demonstrating value of PROs across the oncology portfolio. Ms Nelsen has over 20 years experience in PRO development across oncology, respiratory and other disease areas. She is active in external initiatives, including past Industry co-Director of the Critical Path Institutes’ PRO Consortium. Linda has a master’s degree in Epidemiology from the Johns Hopkins University.
Spotlight Session
Improving the Acceptability of RWE: What's New in 2022?
In-person & Virtual
As real-world evidence becomes an increasingly important basis for health care decision making, the standards and expectations for what constitutes a high-quality RWE study are becoming better defined. Major regulatory agencies such as FDA and EMA are establishing data quality standards and processes via recent guidances and data source-building efforts such as DARWIN EU. For researchers, new resources to help write and register RWD protocols are becoming available to provide more pathways to improve the transparency of the study process. This session will provide more details and insights on these new developments from speakers closely involved in them.
Moderators
Richard Willke, PhD
ISPOR, Lawrenceville, NJ, USA
Dick became ISPOR’s first chief science officer in April 2016, following nearly 25 years in the pharmaceutical industry with Pfizer and its legacy companies. In his CSO role at ISPOR, Dick’s responsibilities are to develop, lead, support, and direct strategic initiatives related to research, scientific, and content priorities to accomplish the organization’s mission to promote health economics and outcomes research excellence to improve decision making for health globally. While with Pfizer, his final position was Vice President, Outcomes & Evidence, lead for Cardiovascular /Metabolic, Inflammation & Immunology, the last in a succession of HEOR group lead roles. He received a PhD in economics from Johns Hopkins University in 1982, concentrating in econometrics and labor economics. Prior to joining Pfizer’s legacy company Upjohn in 1991, he was a member of the economics faculty at Ohio State University as well as a senior economist at the American Medical Association Center for Health Policy Research.
Dick has served on the ISPOR Board of Directors (2007-09), was chair of the ISPOR Institutional Council in 2010, and was co-chair of the ISPOR Good Research Practices Task Force on Cost-Effectiveness Analysis in Randomized Clinical Trials in 2003-2005 as well as its 2014-15 reprise to revise and update that Report. He has co-taught many ISPOR short courses on this topic as well as on “Transferability of Cost-Effectiveness Data between Countries.” He was also a member of the Health Outcomes Committee of PhRMA from 1998-2009, having been its chair from 2002-2004. He has served as a co-editor for Value in Health, on the editorial board for Farmeconomia, on AHRQ, NIH, and PCORI project review study sections, and is a member of the Ohio State University Economics Advisory Board.
Prior to joining industry, Dr Willke served as department director in the Center of Health Policy Research at the American Medical Association and held research and teaching positions at The Ohio State University.
Dr Willke earned a PhD and MA in economics from Johns Hopkins University. He has authored more than 80 scholarly publications that examine the science and methodologies of health economics and outcomes research.
Panelists
John Concato, MD, MS, MPH
Food and Drug Administration, Silver Spring, MD, USA
John Concato, MD, MS, MPH, is an Associate Director of the Office of Medical Policy, Center for Drug Evaluation and Research, FDA. His responsibilities related to real-world evidence (RWE) include developing internal Agency processes, interacting with stakeholders, coordinating demonstration projects and guidance development, and serving as Chair of the RWE Subcommittee. He joined FDA in 2019 after a career at Yale School of Medicine and the US Department of Veterans Affairs, where he was a clinician, educator, independent investigator, research center director, and Professor of Medicine.
Xavier Kurz, MD PhD
European Medicines Agency, Amsterdam, Netherlands
Xavier Kurz graduated in 1982 as a Medical Doctor at the University of Liege, Belgium. He specialised in Tropical Medicine and worked for several years in public health projects in Africa and Asia. He obtained a MSc (1991) and a PhD (1997) in Epidemiology and Biostatistics at McGill University, Montreal, Canada. He then joined the Department of Pharmacology of the University of Liege and the Belgian Centre for Pharmacovigilance (Ministry of Health) as scientific expert. He joined the European Medicines Agency (EMA) on 1st September 2005. He is currently Head of Data Analytics within the Data Analysis and Methods Task Force.
Shirley Wang, PhD
Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA
Dr. Wang is an Associate Professor at Brigham and Women’s Hospital, Harvard Medical School. She has led 2 joint task forces between ISPOR and the International Society of Pharmacoepidemiology (ISPE) focused on real-world evidence for healthcare decision-making. Dr. Wang directs the REPEAT Initiative, a non-profit program with projects aimed at improving transparency, reproducibility and robustness of evidence from healthcare databases and co-leads RCT-DUPLICATE, a series of projects designed to inform when and how real-world data analyses can draw causal conclusions.
Concurrent Breakout Session 2
Let Them in or Build a Wall? Transporting Inferences Across Borders
In-person
ISSUE: Health technology assessment is increasingly reliant on a combination of evidence generated in a controlled setting (with and without randomization) and in the real world. Despite advancements in methods facilitating the synthesis of data from different sources, decision makers (reimbursement bodies, clinicians, manufacturers, etc) are consistently challenged with determining how far results extend to populations not included in the initial study(ies). Transporting inferences across jurisidictions requires decision makers to assume that the patient characteristics and experiences in the study population and the target population are comparable. The effect of unmeasured confounders (potentially related to treatment patterns or the treatment setting) is a source of uncertainty for clinicians, manufacturers and HTA / reimbursement decision makers. Before “importing” RWE collected in another population, it is important to consider the impact of differences in patient characteristics, treatment guidelines, clinical practice and patient preferences.
OVERVIEW: This interactive panel will provide perspectives across geographies and stakeholders. The moderator will begin the panel by framing the topic of transportability before baselining important elements of consideration when assessing exchangeability of inferences from one population to another. This will be followed by each panelist presenting their perspectives on the transportability of RWE. The moderator will then ask panelists to describe how they would recommend HEOR studies to fill global evidence gaps when RWE isn’t accessible/available in every country of interest. Initially from the perspective of a manufacturer tasked with planning a global real world evidence generation program designed to supplement traditional clinical regulatory label-enabling programs, intended to inform access decisions in multiple markets. Two other panelists will represent the perspectives of important stakeholders within the HTA process, a technical / methodological advisor and a treating physician. The issue panel will conclude with audience Q&A
Moderators
Blythe Adamson, PhD, MPH
Flatiron Health, New York, NY, USA
Blythe Adamson is Principal Scientist in Machine Learning at Flatiron Health and Founder of Infectious Economics. She uses health economics, math, epidemiology, and data science to research and identify high-value medicines in development. Her research includes dynamic transmission modeling of infectious diseases, electronic medical records analysis in oncology, and cost-effectiveness studies to inform policy. Dr. Adamson received her PhD in Pharmacoeconomics and Masters in Public Health in Epidemiology from the University of Washington in Seattle. She served on the White House COVID Task Force in 2020. Prior to Flatiron, she worked on the development of HIV vaccines at Fred Hutchinson Cancer Research Center and informed Gates Foundation investing decisions with the Institute for Disease Modeling at Global Good.
Panelists
Nick Latimer, PhD
ScHARR, University of Sheffield, Sheffield, DBY, United Kingdom
Nick is a Professor of Health Economics at the University of Sheffield, and a Yorkshire Cancer Research Senior Fellow. He is a member of NICE Technology Appraisal Committee B and of the NICE Decision Support Unit (DSU). Nick’s research focuses on survival analysis in economic evaluations. He has authored four DSU technical support documents related to survival analysis, treatment switching, and partitioned survival models. His current work involves investigating the use of cancer registry datasets to estimate the comparative effectiveness of cancer treatments used in clinical practice.
Joshua Ray, MSc
F. Hoffmann-La Roche, Basel, BS, Switzerland
Joshua Ray, BA, MSc has over 15 years of experience developing economic evaluations and statistical approaches to support HTA and reimbursement decision making.
Vivek Subbiah, Doctor of Medicine
MD Anderson Cancer Center, Houston, TX, USA
Medical Director of the Clinical Center for Targeted Therapy (Phase I Program). Executive Director of Oncology Research, University of Texas MD Anderson Cancer Network
How Can a Robust Real-World Data Infrastructure Advance a More Patient-Centered Approach to Drug Development?
In-person & Virtual
ISSUE: New opportunities for patient engagement in value assessment and regulatory activities have revealed insights on which outcomes are important to diverse patients. Decision-makers are increasingly aware of the incongruity between representativeness of those enrolled, what matters to patients, and data collected in clinical trials. In parallel, real-world evidence (RWE) has attained a larger role in drug development and value assessment, with examples of RWE being used to support regulatory approval and measure treatment outcomes most important to patients.
Despite the volume of data and broad recognition of the critical role that real world data (RWD) can play in supplementing evidence from clinical trials, the extent to which the US data infrastructure supports analyses of diverse populations on patient-centered outcomes is unclear. To answer this question, a closer look at the current state of RWD generation and the infrastructure that supports its collection and dissemination may be appropriate.
OVERVIEW: The panel will open with introductions and discussion of the extent to which the current data infrastructure may or may not facilitate a crucial next step in patient-focused drug development (panelist 1), learning how diseases and drugs affect diverse patient populations (panelist 2), and measuring outcomes that matter to patients (panelist 3) (10min each; 30min total). The panel will then debate what investments and improvements are needed to ensure a data infrastructure in the US that can generate evidence that is both germane and of sufficient quality to support drug development and reimbursement for diverse patient populations (20 min). The panel will close with audience polling on several key questions and audience questions for the panel (10 min). Stakeholders interested in use of RWD to support drug development, including industry, RWD vendors, regulators, and public and private payers should attend.
Moderators
Richard Chapman, PhD, MS
The Innovation and Value Initiative, Alexandria, VA, USA
Dr. Chapman is the Chief Science Officer for the Innovation and Value Initiative (IVI), a nonprofit research organization whose mission is to advance the science, practice, and use of value assessment in healthcare to make it more meaningful to those who receive, provide, and pay for care. Prior to that, Dr. Chapman was Director of Health Economics at the Institute for Clinical and Economic Review, where he led development of economic evaluations assessing the potential cost-effectiveness and budgetary impact of clinical interventions.
Panelists
Tzuyung Douglas Kou, PhD, MPH, MA
Bristol-Myers Squibb, Lawrenceville, NJ, USA
Dr. Tzuyung Douglas Kou is the Senior Director of Epidemiology and Therapeutic Area Head for Immunology, Cardiovascular, and Fibrosis at Bristol-Myers Squibb (BMS). Dr. Kou has more than 15 years of experience in the pharmaceutical industry, designing and conducting clinical and drug safety research using various real-world data (RWD) resources and innovative analytical platforms.
In his most recent safety role at BMS, he drove the regulatory and data generation strategy resulting in a FDA submission using real world evidence (RWE) for approval of new indication. He has had extensive direct interactions, including numerous face-to-face meetings and written correspondence, with several regulatory agencies and health authorities around the world, such as the FDA, EMA, and PMDA, to negotiate the design and content of enhanced pharmacovigilance and pharmacoepidemiology studies that leverage RWE. He has partnered with the leaders of various academic and governmental registries and data repositories to understand their capabilities and limitations, and subsequently maximize their use to answer important safety-related questions about the use of specific drugs in the real world.
Elisabeth Oehrlein, PhD, MS
Applied Patient Experience, LLC, Washington, DC, USA
Elisabeth M. Oehrlein, Ph.D., MS, is Assistant Vice President, Research & Programs, at the National Health Council, joining the organization in July 2018. Dr. Oehrlein is a mixed-methods researcher with expertise in value/health technology assessment, outcomes research, and patient-focused medical product development. Her research interests include patient journey/experience mapping and applying patient experiences when developing real-world research to ensure studies reflect the “real world” as closely as possible. She is an active member of HTAi’s Patient and Citizen Involvement Group, as well as the International Society for Pharmacoeconomics and Outcomes Research, where she holds leadership roles in the Patient-Centered and Real-World Evidence Special Interest Groups. She has published widely in medical, economic, and health policy journals and serves as an Associate Editor of Value in Health.
Dr. Oehrlein holds a BA from Franklin & Marshall College, an MS in Epidemiology from the University of Maryland School of Medicine’s Department of Epidemiology and Human Genetics, and a Ph.D. in Pharmaceutical Health Services Research from the University of Maryland School of Pharmacy.
Mark Trusheim, MS, BS
Massachusetts Institute of Technology, Cambridge, MA, USA
Mark Trusheim, MS, BS, Strategic Director, NEWDIGS and Visiting Scientist MIT, Boston, MA
Mark Trusheim is Strategic Director, MIT NEWDIGS where he also co-leads the Financing and reimbursement of Cures in the US (FoCUS) Project; and a Visiting Scientist at the MIT Sloan School of Management. Through MIT he has also served as a Special Government Employee for the FDA’s Office of the Commissioner.
Mark’s research focuses on the economics of biomedical innovation, especially precision financing for patient access, precision medicine, adaptive pathways, platform trials and digital health advances.
Prior to MIT, his career spanned big data at Kenan Systems, marketing at Searle Pharmaceuticals, eHealth as Vice President of Monsanto Health Solutions, genomics as President of Cereon Genomics, and policy as the President of the Massachusetts Biotechnology Council.
He holds degrees in Chemistry from Stanford University and Management from MIT.
Defining and Operationalizing No-Value Care
Virtual
ISSUE: Addressing inefficient spending – such as the pervasive delivery of low-value and potentially harmful care – is critically important for a resilient and prepared health system. However, many current low-value care definitions identify low-value care within the healthcare system by operationalizing what is considered low-value through situational context. This results in a level of subjectivity where emphasizing different elements of the definition can impact the determination of low-value. A potential solution is for policymakers to first address no-value care. A no-value care definition could serve as the first step to help limit inefficient spending, prevent harmful care delivery, and create a more resilient value-driven healthcare system. This session will discuss the need for a more concrete definition of “no-value care”, what types of services would fall into this category, how often these services appear as having no or little cost effectiveness evidence and the financial implications of adopting a more concrete, no-value care definition.
OVERVIEW: Overview provided by Mark Fendrick approximately 15 minutes. Low-value care has been the topic of discussion for decades in the United States. In 2012, the discussion became more focused with the introduction of the Choosing Wisely campaign whereby specialty society collaborators identified medical tests and services that could be deemed unnecessary to help spur conversation about what is appropriate and necessary treatment. Since then, several studies have examined how best to define and measure low-value care. These definitions, however, are often unactionable as most definitions are not concrete. This session will discuss the need for a more concrete definition of “no-value care”, what types of services would fall into this category, how often these services appear as having no or little cost effectiveness evidence and the financial implications of adopting a more concrete, no-value care definition.
Moderators
A. Mark Fendrick, MD
Departments of Internal Medicine; Center for Value-Based Insurance Design; University of Michigan, Ann Arbor, MI, USA
A. Mark Fendrick, M.D. is a Professor of Internal Medicine and a Professor of Health Management and Policy at the University of Michigan. Dr. Fendrick conceptualized and coined the term Value-Based Insurance Design (V-BID) and currently directs the V-BID Center at the University of Michigan [www.vbidcenter.org], the leading advocate for development, implementation, and evaluation of innovative health benefit plans. His research focuses on how clinician payment and consumer engagement initiatives impact access to care, quality of care, health care disparities, and health care costs. Dr. Fendrick is an elected member of National Academy of Medicine (formerly IOM), serves on the Medicare Coverage Advisory Committee, and has been invited to present testimony before the U.S. Senate Committee on Health, Education, Labor and Pensions, the U.S. House of Representatives Ways and Means Subcommittee on Health, and the U.S. Senate Committee on Armed Services Subcommittee on Personnel. Dr. Fendrick is the co-editor in chief of the American Journal of Managed Care and is an editorial board member for 3 additional peer-reviewed publications. He remains clinically active in the practice of general internal medicine.
Panelists
Beth Beaudin-Seiler, PhD
Altarum, Ann Arbor, MI, USA
Dr. Beaudin-Seiler is an experienced Senior Analyst in the Applied Research and Analytics practice area at Altarum. Dr. Beaudin-Seiler is experienced in both quantitative and qualitative health-related research projects. She has extensive experience conducting literature reviews; conducting key informant interviews for both focus groups and one-on-one interviews; and is highly proficient in analyzing qualitative data. Her qualitative work includes topic areas such as defining and measuring low-value care; defining safety-net hospitals; and practicality of low-value care tools to visualize waste within health care systems. Dr. Beaudin-Seiler has experience in survey development, fielding and analysis of quantitative data in the Consumer Healthcare Experience State Survey (CHESS) where advocates from several states have utilized this data to inform key decisionmakers on out-of-network billing practices, surprise billing prevalence and other affordability issues in their respective states. She is also the manager of the Research Consortium for Health Care Value Assessment.
Peter I Neumann, ScD
Tufts Medical Center, Boston, MA, USA
Peter J. Neumann, Sc.D., is Director of the Center for the Evaluation of Value and Risk in Health (CEVR) at the Institute for Clinical Research and Health Policy Studies at Tufts Medical Center, and Professor of Medicine at Tufts University School of Medicine. He is the founder and director of the Cost-Effectiveness Registry, a comprehensive database of cost-effectiveness analyses in health care. Dr. Neumann has written widely on the role of clinical and economic evidence in pharmaceutical decision making and on regulatory and reimbursement issues in health care.
Podium Session 2
Evidence on Treatment Access, Disparities and Social Determinants of Health
In-person
Moderator
Stacey Kowal, BS, MSc
Genentech, Inc., Alameda, CA, USA
I am a global health economics and outcomes research (HEOR) expert with more than 15 years of experience in healthcare research and team leadership, including study conceptualization and execution, team building/staff development, external stakeholder relationship cultivation, and business unit operations. As a researcher, I’m most passionate about using economic modeling and real world evidence to understand how new innovations impact patients and their families, as well as broader stakeholders in the healthcare system. My current research uses a range of HEOR tools (economic modeling, real world data, patient reported outcomes) to identify and advance methods to improve our ability to measure and communicate value as it relates to health equity, precision medicine, health policy and health technology assessment.
P10: Parental Vaccine Sentiment and Decision-Making Factors for Pediatric COVID-19 Vaccinations
1:45PM - 2:00PM
Wu B 1 , Almeida R2 , Eslami N3 , Garfield S3 1 Ernst & Young, Doylestown, PA, USA, 2 Ernst & Young, Redwood City, CA, USA, 3 Ernst & Young, Boston, MA, USA
OBJECTIVES:
This nationwide study focused on how parents perceive COVID-19 vaccinations and their behaviors related to vaccinating their children. The study looked at how parents are considering vaccination, who can most influence their perspectives, how a parent’s own vaccination status can impact decision-making as well as what messages would make them more likely to vaccinate their children.
METHODS
: A national online survey was fielded to American adults via email invitation from a nationally recognized survey panel provider during four periods: in May 2021 (
Wave 1 ) prior to Emergency Use Authorization (EUA) of the vaccine for 12–15-year-old individuals, and follow-up surveys conducted in July (
Wave 2 ), September (
Wave 3 ), and December (
Wave 4) 2021. Each survey collected approximately 2,000 responses, and respondents were allowed into the survey if they had at least one child between the ages of 12 and 17 for Waves 1-3 and at least one child aged 5 or older in Wave 4. The survey sample was aligned to the racial and ethnic composition of Americans and controlled for urbanicity.
RESULTS:
Vaccine perceptions differed by the race/ethnicity of respondents, among mothers/fathers, across geographic areas, and by education level. Top concerns parents have about the vaccine include long-term and short-term side effects and limited testing of the vaccine. Pediatricians play a significant role in influencing parents’ decision on whether to vaccinate their children for COVID-19, and most parents prefer to get their children vaccinated at their pediatrician’s office. Wave 4 results indicated that parents with children under 12 are more uncomfortable with vaccinating their children than those with children ages 12 and older.
CONCLUSION: For those trying to increase vaccination among the pediatric population, understanding the concerns of parents, finding trusted voices, getting messages out via accessible channels, and supporting parents in making vaccination decisions will remain critical.
P12: Development of the Medication Access Screening Tool (RXMAST) in Patients with Chronic Diseases: A Qualitative Study
1:30PM - 1:45PM
Brown CM1 , Vohra Y 1 , Thurman W2 , Moczygemba L1 , McNabb B3 , Tucker A3 , Patterson L4 , Fain J5 1 The University of Texas at Austin College of Pharmacy, Texas Center for Health Outcomes Research and Education (TxCORE), Austin, TX, USA, 2 The University of Texas at Austin, College of Nursing, Austin, TX, USA, 3 Community Pharmacy Enhanced Services Network- Texas, Austin, TX, USA, 4 Hale Center Clinical Pharmacy, Hale Center, TX, USA, 5 Pieratt's Pharmacy, Giddings , TX, USA
Objective: Disparity populations disproportionately experience negative social determinants of health (SDoH) that limit their access to needed medications and medical care. This study aimed to develop a comprehensive understanding of patient experiences with medication access that are grounded in SDoH that drive suboptimal medication use. Methods: Patients aged 18 years and above, with at least two chronic conditions and prescribed at least two chronic medications were recruited from four independent pharmacy sites in Texas to participate in 1-hour focus groups (FGs). A total of four FGs were conducted, and data saturation was achieved. A moderator guide containing SDoH issues identified by Pharmacy Quality Alliance’s Access to Care framework, pharmacy case studies, and a targeted literature search was used. The FG data were transcribed and coded by two researchers working independently to identify themes. Codes and themes were discussed until consensus was reached, and salient medication access issues were identified. Results: 31 patients participated in the FGs. 64.5% (N=20) were female and the mean age was 64.6 years (range: 25-91). A majority was white (51.6%, N=16) followed by African American (29%, N=9) and all (100%, N=31) had at least graduated high school. Most had two chronic conditions (41.9%, N=13) and were on at least five prescribed medications (58.1%, N=18). A total of eight (8) salient medication access issues were identified: forgetfulness, high medication burden, lack of care coordination, high medications costs, transportation issues, social isolation, deteriorating mental health, and use of complimentary and integrative medicine (CIM). Conclusion: Patients with chronic diseases reported several SDoH-related issues, including some that are psychosocial (social isolation) and culturally embedded (CIM), that negatively impact medication access. Salient issues identified will be used in the development of the RxMAST that can be used by pharmacies to screen patients for medication access problems and provide customizable SDoH solutions.
P9: Social Determinants of Health and Disruptive Life Events Among Patients with Schizophrenia or Bipolar Disorder
2:15PM - 2:30PM
Nau CL1 , Hong BD1 , Padilla A1 , Waters H 2 , Houle CR3 , Penfold RB4 , Rossom R5 , Braciszewski JM6 1 Kaiser Permanente Southern California, Pasadena, CA, USA, 2 Otsuka Pharmaceutical Development & Commercialization Inc., Marco Island, FL, USA, 3 Partnership for Health Analytic Research, LLC, Beverly Hills, CA, USA, 4 Kaiser Permanente, Seattle, WA, USA, 5 HealthPartners Institute, Minnesota, MN, USA, 6 Henry Ford Health System, Detroit, MI, USA
Objective: To assess risk of disruptive life events (DLEs) and the effects of social determinants of health (SDoH) on DLEs in patients with schizophrenia (SCZ) or bipolar I disorder (BP-I). Methods: Multi-site retrospective, cohort study using electronic medical record data from Kaiser Permanente Southern California and Henry Ford Health System matched to DLE data (address changes, judgements, lien filings, bankruptcy, arrests) from TransUnion. Adults with SCZ or BP-I were matched to major depressive disorder (MDD) and general health (GH) controls based on date of incident or first prevalent diagnosis and demographic characteristics. Generalized estimating equation regression models assessed impact of covariates on outcomes. Results: Samples included 15,634 patients with SCZ and 29,380 patients with BP-I matched to MDD controls and 16,095 patients with SCZ and 29,850 patients with BP-I matched to GH controls. Differences in DLEs were found between patients with SCZ and BP-I and their control groups. Comparing patients with SCZ and BP-I to their matched controls, the likelihood of experiencing DLEs differed by race, age, and gender. Compared to white patients, Asian patients were less likely to experience any DLE, while Black patients were more likely to experience any DLE. Patients currently under age 65 (vs. > 65) were more likely to move, have judgements, declare bankruptcy, and have been arrested. Odds of having a lien were less for patients 18-44 years but more for those 45-64 years (both vs. > 65). Findings for gender were mixed based on comparator cohort. Conclusions: Further research is warranted to understand the role of social determinants of health on the occurrence of DLEs among patients with mental illness to develop policies and clinical pathways that help improve equitable access to care.
P11: Racial Disparity in Treatments for Tibial Fracture Using Propensity-Score Matched Cohorts in a Medicaid Database
2:00PM - 2:15PM
Vanderkarr M 1 , Ruppenkamp J1 , Parikh A2 , Vanderkarr M3 , Holy C4 , Sparks C5 1 Johnson & Johnson, New Brunswick, NJ, USA, 2 DePuy Synthes, Inc., West Chester, PA, USA, 3 DePuy Synthes, Inc., Bay Village, OH, USA, 4 Johnson & Johnson Co., New Brunswick, NJ, USA, 5 DePuy Synthes, West Chester, PA, USA
Objectives: Racial disparities in elective treatments have been documented but is less known within urgent care. Our study compared healthcare utilization in surgically-treated tibial fracture cases among race/ethnic groups. Methods: Patients within IBM® MarketScan® Medicaid database with tibial fracture and surgical intervention (date of surgery = index) from 10/1/2015 to 12/31/2020 and ≥ 6 months of continuous enrollment pre-index were identified. Exclusion criteria included: polytrauma, amputation at index, fractures pre-index, race other than Black or White. Variables included patient demographics, comorbidities (defined by Elixhauser Index (EI)), fracture types (open vs closed, Gustilo I-II vs III), procedural characteristics (surgery type, implant type used). Black vs White patients were matched using propensity scores on age, gender, surgical procedures, fracture type, and comorbidities. Outcomes included post-surgical treatment (pain medication use, reoperations) and complications (infection, nonunion) up to 2 years post-index. Chi-square tests and survival analyses (Kaplan-Meier and Cox regression) were conducted. Results: 6,280 matched patients (3,140 White, 3,140 Black) were analyzed (48.2% female, average age 40.3 (standard deviation (SD): 18.7), 17.7% <19 years, average EI 2.2 (SD: 2.6), closed fracture: 70.5%, 59.0% shaft and 28.6% comminuted fractures). 52.1% were treated with internal fixation, 19.6% external fixation and 25.4% intramedullary nails. Blacks were significantly less likely to have a reoperation (hazard ratio: 0.88, 95%CI: 0.80-0.97, p= 0.01) but complication rates were not statistically different between Black and White patients. Whites were significantly more likely to be prescribed antibiotics, strong opiates, and antidepressants (Patients with ≥ 1 prescription at 12-months, for antibiotics: 66.7% White, 54.6% Black, difference: 12.1%, p<0.001; for antidepressants: 41.9% White, 29.0% Black, difference: 12.9%, p<0.001; for strong opiates: 10.4% White, 6.4% Black, difference: 4.0%, p<0.001). Conclusions: Although complication rates were similar between Black and White patients, treatments, such as reoperation and medication prescriptions, differed.
Emerging Methods in Economic Evaluations
Virtual
Moderator
Asrul Akmal Shafie, PhD
Universiti Sains Malaysia, Minden, Penang, Malaysia
P47: Integrating a Polygenic Risk Score for Coronary Artery Disease As a Risk Enhancing Factor in the Pooled Cohort Equation: A Cost-Effectiveness Analysis Study
2:00PM - 2:15PM
Mujwara D 1 , Henno G2 , Vernon ST3 , Peng S4 , Domenico PD1 , Schroeder B4 , Busby GB1 , Figtree GA5 , Bottà G1 1 Allelica, New York, NY, USA, 2 Pacific Biosciences, Menlo Park, CA, USA, 3 Royal North Shore Hospital, Sydney, NSW, Australia, 4 Illumina Inc, San Diego, CA, USA, 5 University of Sydney, Sydney, NSW, Australia
OBJECTIVES: Polygenic risk score for coronary artery disease (CAD-PRS) improves precision in determining the risk of atherosclerotic cardiovascular disease (ASCVD) but health benefits and healthcare costs associated with CAD-PRS are unknown. We examined the cost-effectiveness of including CAD-PRS as a risk-enhancing-factor in the pooled-cohort-equation (PCE)—the standard-of-care for determining the risk of ASCVD—versus the PCE-alone.
METHODS: We applied a Markov model on a 40-year-old cohort with borderline/intermediate 10-year-risk (5-to-<20%) of ASCVD to project health benefits and costs of identifying individuals in the top quintile of the CAD-PRS distribution, who have a 2-fold increased risk of CAD versus the reminder of the distribution and are therefore eligible for statin prevention therapy but remain unidentified by the PCE-alone. Outcomes included: adverse events (CAD and ischemic stroke) averted, quality-adjusted life-years (QALYs) gained, incremental costs and incremental cost-effectiveness ratio (ICER). Parameter inputs came from the literature and costs reflected a healthcare perspective. Deterministic and probabilistic sensitivity analyses examined parameter uncertainty. Future annual costs and QALYs were discounted at 3%.
RESULTS: PCE+CAD-PRS had higher mean QALYs (0.003, 0.011) and lower mean costs ($40, $180) per-person screened, and averted adverse events (29, 50) in a cohort of 10,000 individuals versus the PCE-alone per time horizon (5-year, 10-year), respectively. Risk of CAD, statin effectiveness and CAD treatment costs had the largest impact on the ICER, but the ICER values remained below the $50,000 willingness-to-pay threshold except when the risk was ≤6% in the 5-year time horizon. Probabilistic sensitivity analysis results indicated that PCE+CAD-PRS was cost-effective with a probability of 94% and 99% at the $50,000 willingness-to-pay threshold in the 5-year and 10-year time horizon, respectively.
CONCLUSIONS: Implementing CAD-PRS as a risk-enhancing-factor in the PCE to determine the risk of ASCVD reduced the mean cost per person screened, improved QALYs and averted future events of CAD and ischemic stroke compared to the PCE-alone.
P46: Missing Data and Missed Opportunities: A Potential Solution to Data Gaps for Societal Perspective Economic Evaluations
1:30PM - 1:45PM
Richardson M 1 , Whittington M2 , Campbell J3 1 Institute for Clinical and Economic Review, London, ON, Canada, 2 Institute for Clinical and Economic Review, Liberty, MO, USA, 3 Institute for Clinical and Economic Review, Hingham, MA, USA
OBJECTIVES: To explore the relationship between incremental non-healthcare sector societal costs and incremental quality adjusted life years (QALYs) in published ICER economic evaluations.
METHODS: ICER drug reviews conducted between May 2019 and November 2021 were identified. Data including condition, intervention(s), comparator(s), perspective of analysis, and incremental QALYs and costs were extracted for each comparison and perspective undertaken (i.e., healthcare sector and societal). Details regarding the domains included in the societal perspective were summarized descriptively, and the relationship between incremental non-healthcare sector societal costs and incremental QALYs were assessed graphically and for fit.
RESULTS: Twenty ICER reviews were included in the analysis. Of the 17 reviews where a societal perspective analysis was undertaken, 9 were excluded because: a) incremental costs from the societal perspective were higher than incremental costs from the health system perspective (n=1), b) treatments were less costly and equally, or more, effective (n=3), c) QALY gains were marginal (i.e., <0.015; n=1), or d) insufficient data was available due to rounding (n=4). For the 8 remaining reviews (28 pairwise comparisons versus usual care), a linear relationship (R2 = 0.8126) was observed between the incremental non-healthcare sector societal cost savings and incremental QALYs. Our results suggest that for every additional lifetime discounted QALY gained, incremental non-healthcare societal cost savings increased by $12,000 (2021 USD) on average (range: $5,600 to $108,000). Our analysis is limited by the small number of reviews included in our analysis.
CONCLUSIONS: Economic modelers could assume this relationship holds for analyses that lack societal level information. This estimate offers a placeholder value to update once additional data are available. Future research should explore this relationship in other reviews and assess the variability in findings according to the characteristics of the condition such as severity and mean age of onset.
P45: Visualizing Joint Costs and Effects for Multiple Alternatives within Cost Effectiveness Analyses: Cost-Disutility Versus Traditional Cost-Effectiveness Planes in an Economic Evaluation of Opioid Use Disorder Treatments
2:15PM - 2:30PM
Beaulieu E 1 , Rittenhouse B2 1 Harvard Medical School and Massachusetts General Hospital, Boston, MA, USA, 2 MCPHS University, Winchester, MA, USA
OBJECTIVES: Multiple options exist to visually present the joint costs and effects of multiple alternatives within a cost effectiveness analysis (CEA). The most common method to characterize treatment alternatives’ costs and effectiveness is the cost effectiveness plane. We present an application of an alternative presentation method introduced by Eckermann to plot alternatives’ costs and effectiveness: the cost disutility (C-DU) plane. We compare the C-DU visualization to the traditional cost effectiveness plane using a CEA on pharmacological treatments for opioid use disorder (OUD). Technical and economic efficiencies may also be calculated using the C-DU plane, quantifying divergence from optimality for suboptimal treatments.
METHODS: Using a CEA comparing clinic-based methadone (MC), office-based methadone (MO), and office-base buprenorphine (BO) to treat OUD patients, we present the joint cost and effectiveness of the competing alternatives in conventional cost-effectiveness space as well as C-DU space. In C-DU space, each of three alternatives is plotted with respective costs relative to the lowest-cost alternative on the y-axis and with respective disutility relative to the effectiveness of the most-effective alternative on the x-axis. Via additional analyses enabled by the C-DU plane, we calculate technical and economic efficiencies for each alternative.
RESULTS: Both planes are presented. We quantified technical efficiency of MC, MO, and BO respectively at 1, 1, and 0.322. Applying a willingness to pay threshold of $7,000 per additional patient retained in six-month treatment, we quantified economic efficiency of MC, MO, and BO respectively at 0.814, 1, and 0.301.
CONCLUSIONS: The C-DU plane offers advantages over the conventional cost-effectiveness plane by removing potential ambiguities regarding interpretation dependent on quadrant. The C-DU plane offers additional insight in comparing alternatives by enabling the quantification of technical and economic efficiency for each alternative. The C-DU plane can thus be a valuable supplemental visualization tool within CEA reporting.
P48: Are Health Technology Assessment (HTA) Bodies Responding to the Assessment Challenges Posed By Cell and Gene Therapies?
1:45PM - 2:00PM
Fornaro G1 , Drummond M 2 , Ciani O3 , Jommi C4 1 SDA Bocconi School of Management, Milan, Italy, 2 University of York, York, YOR, UK, 3 SDA Bocconi School of Management, Milan, MI, Italy, 4 SDA Bocconi School of Management, Bocconi University, Milano, Italy
OBJECTIVES: Cell and gene therapies have characteristics that pose additional challenges for HTA. The objectives of this research were to assess how HTA bodies are responding to these challenges and to identify where further initiatives are required.
METHODS: HTA reports and supporting documents were analysed for 9 cell and gene therapies in 10 indications from the US (ICER), Canada (CADTH), England (NICE), Scotland (SMC), France (HAS) and Italy (AIFA). Data were extracted using a recently published checklist for assessing gene therapies that specifies 19 key characteristics.1
RESULTS: In total 31 HTA reports were analysed. The key clinical characteristics of cell and gene therapies were widely considered in the reports: surrogate endpoint (84% of reports), rare disease (71%), serious condition (94%), single arm trial (100%), pediatric population (100%), adverse consequences (100%), size of clinical trial (100%), length of clinical trial (97%), extrapolation to long-term outcomes (65%). The consideration of key economic characteristics and other key aspects of the economic evaluation was more variable: serious disease (94%), value to caregivers (45%), insurance value (0%), scientific spillovers (10%), lack of alternatives (71%), substantial improvement in life expectancy (65%), discounting (74%), different discount rates (55%), uncertainty (100%), alternative payment models (29%).
CONCLUSIONS: HTA bodies are considering many of the key characteristics of cell and gene therapies, although this response is variable. The main areas where further initiatives are required include the validation of surrogate endpoints, the criteria for accepting single arm clinical studies, the assessment of family and scientific spillovers, estimating insurance value, exploring scenarios using different discount rates and the use of alternative payment models to reduce uncertainty.
Reference 1. Drummond
et al Value in Health 2019; 22(6):661-668
Concurrent Breakout Session 2
Multi-Stakeholder Survey on Use of Digital Health Tools Transforming the Process for Development and Use of Medical Device Technologies
Virtual
Level: Foundational
The learning objectives of this session are: to provide an overview of the survey project, and introduction to the stakeholder roles and their use of digital health technologies, to review the methodology for analyzing the data collected and results of the survey, and to discuss the survey findings regarding stakeholder’s successes, challenges, and opportunities of integrating digital health technologies to improve patient experiences.
The rapid increase in availability and use of digital health technologies – accelerated by the onset of the COVID-19 pandemic – has created opportunities for dramatically transforming the process for development and use of medical technologies. MDIC launched an effort to survey key stakeholders across the medical device ecosystem including patients, providers, hospital systems, medical device manufacturers, med tech, researchers, and payers, with the goal of identifying challenges, opportunities, and successes for leveraging digital health technologies and information to advance innovation. The session will include the following presentations:
An overview of the survey project and results (Dohse, 20 min presentation with slides). Discussion of challenges and opportunities from the industry perspective (industry person, 10 min). FDA-CDRH’s response to the survey and broader perspective on patient-generated health data and use of digital health technologies (Saha, 10 min). The session will be concluded by a Q&A discussion between audience and speakers (15 min). This session will be of interest to professionals interested in digital health technologies and the healthcare ecosystem. The session will benefit medical device and pharma sponsors and regulators.
Speakers
Heather Colvin, PhD
Johnson & Johnson, Washington, DC, USA
Heather M. Colvin is the Director of Evidence and Outcomes Policy for Johnson & Johnson MedTech where she works to advance the adoption of innovative evidence approaches including real-world evidence, the science of patient input, to improve diversity and inclusion in clinical evidence. Prior to joining Johnson & Johnson, she worked at the Duke-Margolis Center for Health Policy, the Brookings Institution, the Institute of Medicine (now the National Academy of Medicine), HHS’ Office of Global Health Affairs and the National Committee for Quality Assurance. She holds a Master of Public Policy from Georgetown University and a Bachelor of Arts in Cultural Anthropology from George Mason University.
Anindita Saha, BS
U.S. Food and Drug Administration, Silver Spring, MD, USA
Anindita (Annie) Saha is the Assistant Director for the Digital Health Center of Excellence (DHCoE) at the Food and Drug Administration (FDA) Center for Devices and Radiological Health (CDRH). Ms. Saha is leading the development of partnerships, regulatory science, strategic planning, and operations for the DHCoE to empower digital health stakeholders in advancing healthcare. Additionally, Annie helped incubate and continues to support CDRH’s patient science and engagement efforts to advance the science and adoption of patient input as evidence, including patient preference information (PPI), clinical outcome assessments (COAs) including patient-reported outcomes (PROs), and patient-generated health data (PGHD). These efforts include researching the use of digital health technologies to capture the patient perspective
Discussion Leaders
Heidi Dohse, BA, NBCHWC
Tour de Heart, Hailey, ID, USA
Heidi Dohse is passionate about improving patient outcomes and uses her athletic events to inspire people living with heart issues. She partners with physicians and researchers worldwide to provide insights regarding the patient experience and ideas for engagement. In addition, she travels globally, educating audiences on digital health, data, and healthcare. Heidi recently retired from her position as Sr. Program Manager for Google Cloud Healthcare & Life Sciences organization to focus on her non-profit organization Tour de Heart.
She is a member of the MDIC Science of Patient Input working group, selected as a member of the NESTcc Active Surveillance Methodology Working Group, and Heart Rhythm Society’s Cardiovascular Digital Health Journal editorial board.
Dealing with Disability in Health Technology Assessment (HTA)
Virtual
Level: Intermediate
PURPOSE: The U.S. Affordable Care Act (ACA) prohibits use of Quality-Adjusted Life Year (QALY)-based models that “discount the value of a life of a disabled person.” This Panel will present three methods intended to resolve this problem, discuss their strengths and weaknesses, and then engage in a general discussion (including extensive audience participation) directed towards finding Cost-Effectiveness Analysis (CEA) methods that do not violate the ACA constraints and which could expand the use of CEA around the world. DESCRIPTION. The Equal Value of Lives (EVL) method values any improvement in Life Expectancy (LE) at a counterfactual Quality of Life (QoL) value of 1, replacing a presumed lower QoL value due to disability. Scholars argue that this approach under-values interventions that both improve LE and QoL. The Healthy Years in Total (HYT) model makes the same adjustment for LE extensions, but employs counterfactual QoL gains to assess an intervention’s full value. In contrast, the Generalized Risk-Adjusted Cost-Effectiveness (GRACE) model does not alter assumed quantities of LE or QoL for disabled individuals, but instead modifies the value assigned to improvements in QoL and LE, using a utility-maximizing model of value that incorporates diminishing returns to QoL. In Europe, WTP thresholds are being adjusted using severity of illness, with the UK set to adopt specified weights as in the Netherlands. Presenters will discuss how these approaches differ, the data needed to implement them, and to what extent they resolve the ACA’s prohibition against using QALY-based methods that discriminate against disabled persons. The panel will then include extensive Q&A audience participation, guided by the Discussion Leader. Any stakeholder who has an interest in understanding how HTA models incorporate permanent disability and/or resolving the Affordable Care Act ban on use of QALYs that discriminate against disabled people should attend.
Panelists
Anirban Basu, PhD. MS
University of Washington, Seattle, WA, USA
Anirban Basu is a Professor of Health Economics and the Stergachis Family Endowed Director of The CHOICE Institute at the University of Washington, Seattle. He holds joint appointments with the Departments of Health Services and Economics at UW, is a Faculty Research Fellow at the US National Bureau of Economic Research, and an elected Fellow of the American Statistical Association. His work sits at the intersection of microeconomics, statistics, and health policy. His research focuses on understanding the economic value of health care through scientific disciplines of applied economic theory, comparative and cost-effectiveness analyses, causal inference methods, program evaluation, and outcomes research. He served on the Second Panel on Cost-effectiveness Analysis in Health and Medicine and serves on the Editorial Advisory Board for Value in Health Journal. He received his master's in Biostatistics from UNC-Chapel Hill and a PhD in Public Policy Studies from the University of Chicago.
Susan Griffin, PhD
University of York, York, United Kingdom
Darius Lakdawalla, PhD
USC Leonard D. Schaeffer Center for Health Policy and Economics, Los Angeles, CA, USA
Darius Lakdawalla is a widely published, award-winning researcher and a leading authority on health economics and health policy. He holds the Quintiles Chair in Pharmaceutical Development and Regulatory Innovation at the University of Southern California, where he sits on the faculties of the School of Pharmacy, the Sol Price School of Public Policy, and the Leonard D. Schaeffer Center for Health Policy and Economics, one of the nation’s premier health policy research centers. His research has focused primarily on the economics of risks to health, the value and determinants of medical innovation, the economics of health insurance markets, and the industrial organization of healthcare markets.
Discussion Leaders
Charles Phelps, MBA, PhD
University of Rochester, Rochester, NY, USA
Charles E Phelps, PhD, a health economist, has developed key models of cost-effectiveness analysis that provide the intellectual foundations for its practice. He was given the Victor R Fuchs Award for Lifetime Achievement in the Field of Health Economics in 2019, and has been a member of the National Academy of Medicine since 1991. His leading textbook, Health Economics is now in its 6th Edition. His recent interests have expanded to the use of multi-criteria decision analysis (MCDA), particularly in its proper use when the “decision-maker” is a group.
2:30 PM - 3:00 PM
Coffee Break
In-person
3:00 PM - 4:00 PM
Educational Symposium
Digging Deeper – Uncovering Expanded Patient Insights through Linked Data
In-person & Virtual
Rich clinical insights into real-world treatment patterns and healthcare utilization are within reach. Closed administrative claims data are routinely used in HEOR research to assess areas such as Patient Adherence, Health Care Resource Utilization, and the Patient Journey. The nature of claims data ensures that activities associated with a patient’s care are captured, but analyses of the data are limited to information recorded for the purposes of reimbursement – e.g., patient demographics, diagnosis and procedure codes, and pharmacy fills. This can restrict the insights to what occurred, with limited visibility into clinical information that may have influenced treatment decisions and healthcare utilization. Analyses of patient behavior and outcomes also are limited to the population as a whole – or segmented by variables available in, or proxy clinical measures derived from, claims data. Patient registries are purpose-built to capture clinical measures directly from physicians and patients including disease duration for chronic illnesses and disease activity based on validated composite outcome measures incorporating physical exam findings and patient reported outcomes, but may not reflect the full spectrum of healthcare encounters. Linking claims data with the clinical data available in patient registry data provides an additional level of depth that is not typically available today. This symposium will focus on the benefits that can be gained from leveraging multiple linked data sources. Panelists will discuss the attributes of both data sources and the power of the combined data, including specific examples of how additional insights have been derived from the unique combination of claims and patient registry data.
Sponsor
CorEvitas
Speaker
Scott Henderson, MS
CorEvitas, Albany, NY, USA
Scott B Robinson, MA, MPH
Inovalon, Washington, DC, USA
Heather Von Allmen, BA
CorEvitas, Albany, NY, USA
Discussion Groups
New this Year – Discussion Groups! Discussion Groups are facilitated conversations between conference attendees and select conference speakers. Held in the new, dynamic Discussion Lounge in the ISPOR Exhibit Hall, these discussions are intended to be highly interactive, collaborative, and promote the exchange of ideas in a peer-to-peer setting. Value Assessment of Technologies Discussion Group
In-person
Moderator
Melanie Whittington, PhD
Institute for Clinical and Economic Review, Boston, MA, USA
Dr. Whittington is the Director of Health Economics at the Institute for Clinical and Economic Review (ICER). In her role, she leads the cost-effectiveness analyses and potential budget impact analyses within ICER reviews, directs the content within the Interactive Modeler™, and advances the field of health economics by developing innovative methods with applications for value assessment.
ISPOR Forums
Making the Case for PGHD: Proving that Collection and Application of Patient-Generated Health Data Can Be Rigorous and Vital to Improving HTA
In-person
This forum, geared to all stakeholders with an interest in using real world evidence in Health Technology Assessment (HTA), will focus on the ongoing efforts to solidify Patient-Generated Health Data (PGHD) as a key input into HTA processes. In this panel, we are exploring one type of PGHD; survey or registry data collected directly from patients and/or caregivers or by patient research and advocacy groups, outside of clinical trials.
The panel will explore methods to best capture, analyze, and apply PGHD in value assessment. In many countries, patients have been involved in the HTA process for years, sharing their personal stories to help provide context and deeper meaning to the clinical trial, claims, electronic medical record, and other data sources being reviewed for medical product evaluation. More recently, given the explosion of “big data” across all sectors of healthcare, patient communities have become increasingly interested in the use of PGHD to tell the longitudinal story of disease experience and therapeutic management. Those steeped in HTA methodology have been concerned however about how relevant and rigorous PGHD is or can be in the evaluation process.
The panelists have held multi-stakeholder discussions (including patients, researchers, HTA bodies, industry, and policymakers) prior to this forum will lend insights from those rich discussions. The first two speakers will present promising practices and case studies from their regional roundtables (North America and European perspectives) outlining the use of PGHD in HTA, highlighting the gaps that PGHD can fill, and exploring the barriers they are still working to overcome. The panel will also discuss a potential template for capturing and using PGHD in the HTA process. The remainder of the session will be a facilitated discussion between audience members and panelists, discussing concerns, opportunities, and ideas for more routinely embedding PGHD into the HTA process.
Moderators
Mary Suz Schrandt, JD
ExPPect, Arlington, VA, USA
Speakers
Cat Davis Ahmed, MBA
Family Heart Foundation, Pasadena, CA, USA
Cat Davis Ahmed is Vice President for Policy and Outreach for the Family Heart Foundation, where she works with individuals living with, or at risk for, early cardiovascular disease due to inherited lipid disorders (Familial Hypercholesterolemia and elevated Lipoprotein(a)) and the medical professionals who treat them. She is a coauthor on publications in the Journal of the American College of Cardiology, Circulation, and Atherosclerosis. Cat is a member of the American Heart Association’s Atherosclerosis, Hypertension, and Obesity in the Young Committee of the Council on Cardiovascular Disease in the Young and Co-Investigator on the NIH-funded “Identification Methods, Patient Activation, and Cascade Testing for FH” (IMPACT-FH) study to improve detection of, and family screening for, FH. She speaks at national medical conferences, including the American Heart Association Scientific Sessions, ISPOR, and the Global Cardiovascular Clinical Trialists Forum. As someone who has FH herself, she knows first-hand the impact the disorder can have on individuals and families. The Family Heart Foundation is a non-profit, patient-centered, research and advocacy organization dedicated to increasing the rate of early diagnosis and encouraging proactive treatment of inherited lipid disorders in order to prevent premature heart disease. Cat holds a BA from Union College and an MBA from the Yale School of Management.
Kayleigh Majercak, MS
University of Maryland Baltimore, Baltimore, MD, USA
Kayleigh Majercak is a researcher within the Pharmaceutical Health Services Research department at the University of Maryland Baltimore (UMB). Her research interests include patient-centered outcomes research, patient-reported outcomes, patient experience data, and mixed-methods research. In her previous role, Kayleigh worked as a data analytics consultant at Humana, Inc. Using claims data, her work focused on informing market leaders with population-level analytics to support Humana’s Bold Goal Communities’ initiative towards improving members’ health. At UMB, her current research and the focus of her PhD dissertation is co-developing a disease-agnostic survey template to fill the patient-centered evidence gaps in US value assessment. Kayleigh is active in ISPOR and a fellow with the National Health Council. Kayleigh graduated with a Bachelor of Science in Pharmaceutical Sciences degree from The University of Toledo and earned her Master of Science degree in pharmaceutical evaluation and policy from the University of Arkansas for Medical Sciences.
Derick Mitchell, PhD
IPPOSI, Dublin, Ireland
Performance Outcome Assessment Emerging Good Practices Task Force: Final Recommendations
In-person
A performance outcome (PerfO) assessment is a type of clinical outcome assessment (COA) based on measurement of standardized task(s) actively undertaken by a patient according to a set of instructions and administered by a trained individual or completed by the patient independently. PerfO assessments reflect physical (eg, mobility), cognitive (eg, working memory), sensory (eg, visual acuity), and other functional skills (eg, instrumental activities of daily living). Such assessments require special attention due to the nature of the concepts of interest for measurement, and in the execution and interpretation of those assessments. For instance, because PerfO assessments require completion of a defined task, confirmation of content validity must include linking that task to a real-world activity or behavior that is meaningful in a person’s daily life. This task force’s recommendations build upon earlier ISPOR task force reports covering other COA types. The goal is to improve the evaluation and documentation of content validity and other measurement properties, including reliability, construct validity, and ability to detect change for PerfO assessments.
The forum will cover the task force’s final report on emerging good measurement practice recommendations for evaluating and/or developing PerfO assessments for use in capturing clinical benefit in treatment trials. Task force members will discuss: 1. Identifying the concept of interest and determining when a PerfO assessment is the optimal approach to measuring it 2. Considerations when identifying, selecting, or modifying existing PerfO assessments or developing new ones 3. Unique challenges when evaluating content validity and other measurement properties of PerfO assessments 4. Importance of conducting a pilot evaluation of PerfO assessments in the target population Attendees will evaluate the final good practice recommendations. This feedback will form the basis of an interactive discussion with polling between the panel and the audience around the practical application of the task force’s recommendations.
Moderators
Stephen Joel Coons, PhD
Critical Path Institute, Tucson, AZ, USA
Speakers
Michelle Campbell, PhD
US Food and Drug Administration, Silver Spring, MD, USA
Christopher Edgar, MSc, PhD
Cogstate, New Haven, CT, USA
Dr. Chris Edgar has an extensive background in drug development and clinical trials methodology, and is an expert in the development, validation ,and application of clinical outcome assessments (COAs). He has held positions as scientific director at Cognitive Drug Research, senior clinical lead at Bracket/UBC, principal scientist at Roche, and is currently Chief Science Officer at Cogstate. Dr. Edgar has worked across all phases of clinical development and in multiple therapy areas, specializing in neuroscience, cognition and performance-based outcome assessments.
Elektra Papadopoulos, MD, MPH
AbbVie Inc., North Chicago, IL, USA
Building Diversity in the HEOR Workforce: A Call for Input and Action
In-person
ISPOR’s Strategic Plan 2020 incorporated core Organizational Values, including embracing diversity of perspectives; ISPOR’s Diversity Policy codified that commitment and specified the types of diversity sought in our membership and activities.
Hemant Phatak (Institutional Council) will open the session by introducing the speakers and forum objectives. Laura Pizzi (ISPOR leadership) will identify the organization’s existing resources to support different aspects of diversity (e.g., Women in HEOR, Leadership Development Initiative, Low- and Middle-Income Country initiative) and summarize the Institutional Council’s Workforce Diversity initiative. Annesha White (Faculty Advisor Council) will present a faculty advisor’s perspective, discussing models such as diversity mentor-focused fellowships and targeted academic programming. Olamide Olujohungbe (Student Network) will present the student perspective by reflecting on her recent experience in attaining a HEOR fellowship in terms of mentorship and supports that are meaningful for new professionals. Uchenna Iloeje (seasoned HEOR industry professional) will discuss the importance of data to inform diversity efforts and that these data should not only focus on new professionals but also mid- and senior-career professionals. The session will close by sharing recommendations and Implementation actions and inviting participants to give feedback to on these ideas and their prioritization.
Moderators
Hemant Phatak, PhD
Accleron Pharma, Cambridge, MA, USA
Speakers
Uchenna Iloeje, MD, MPH
SpringWorks Therapeutics, Stamford, CT, USA
Dr. Uche Iloeje is Vice President, and Head Global Medical Affairs at SpringWorks Therapeutics, a clinical stage biopharmaceutical company based in Stamford Connecticut. He is a Nigerian born and trained physician graduating from The University of Nigeria in 1987. He specialized in Internal Medicine at the University of Connecticut, Critical Care Medicine at the University of Pittsburgh, and Epidemiology and Environmental Health at Yale University. He has been in the pharmaceutical industry for over 2 decades, working in several companies, with experience in health economics and outcomes research, epidemiology, clinical development, medical affairs and payer evidence. Dr. Iloeje has authored or co-authored over 50 peer reviewed articles in high impact journals including, but not limited to JAMA, Hepatology, Gastroenterology, American Journal of Epidemiology, Diabetes Obesity and Metabolism, American Journal of Cardiology, Clinical Infectious Diseases, HIV Medicine, Journal of the National Cancer Institute, Journal of Clinical Oncology, and Pharmacoeconomics.
Olamide Olujohungbe, BS
University of Maryland College of Pharmacy, Baltimore, MD, USA
Olamide C. Olujohungbe is a 4th year PharmD candidate at the University of Maryland School of Pharmacy. Upon graduation this May, she will be pursuing a fellowship in Health Economics and Outcomes Research (HEOR) with Rutgers University in conjunction with Bausch Health. Last summer, she completed an Advanced Pharmacy Practice Experience (APPE) with Merck Pharmaceuticals in their HEOR department which impacted her decision to gain further industry experience within HEOR.
Laura Pizzi, PharmD, MPH, RPh
Rutgers University, Piscataway, NJ, USA
Dr. Laura Pizzi is Associate Chief Science Officer for ISPOR and Professor at Rutgers University in the schools of pharmacy and public health. For the past 25 years, she has led interdisciplinary teams of methodologists, statisticians, and clinicians to design and conduct economic analyses on healthcare interventions and is a frequent author, speaker, and mentor on the topic.
At ISPOR, she provides leadership to the organization’s scientific strategy and initiatives, including content planning and oversight of the Special Interest Groups, Patient Council and roundtables, the ISPOR Competency Framework workgroup, Publications Council, Institutional Council, and Digital Health Strategy. She also liaises with the Student Network and Faculty Advisor Council to support their scientific needs.
Annesha White, PharmD, MS, PhD
University of North Texas Health Sciences Center, Fort Worth, TX, USA
Annesha White, PharmD, MS, PhD is a Senior Associate Dean for Assessment and Associate Professor at the University of North Texas Health Science Center College of Pharmacy. Dr. White has worked on funded projects with the Florida Medicaid program including studies on COX-2 Inhibitors, Hepatitis C and End Stage Renal Disease. She also worked at the Government Accountability Office (the investigative arm of Congress), to examine Pharmacy Benefit Managers impact on Health Plans, Enrollees, and Pharmacies. Dr. White's primary research interests include the design of studies to address issues in the health services research arena. Areas of focus include Medicare, Geriatrics, Managed Care, Pharmacoeconomics and Outcomes Research. Her research has included a focus on a variety of disease states, such as heart disease, chronic pain and end stage renal disease with the goal of providing care that is balanced in quality and cost. Her research involves a team approach to care examining the various aspects of the health care system and how entities can join together to enhance efforts. Dr. White has published many peer-reviewed articles, a textbook entitled Introduction to the Pharmacy Profession and serves as a referee for journals such as the Journal of Managed Care Pharmacy.
3:00 PM - 6:30 PM
In-Person and Virtual Poster Session 2
Live
In-person presenters will be with their posters from 5:30 – 6:30PM.
3:45 PM - 4:15 PM
HEOR Theater
Utilizing RWE and HEOR Throughout the Product Lifecycle: From Product Positioning to Market Access and Reimbursement
In-person
The role of real-world evidence (RWE) and health economics and outcomes research (HEOR) for market access and reimbursement is well established, but there is an increasing interest in utilizing RWE and HEOR earlier in the product lifecycle, adopting a more proactive approach towards supporting product development and evidence generation. This HEOR theater will open with an industry perspective on remaining challenges throughout the product development process, followed by presentations and a discussion on how an integrated and lifecycle approach towards RWE, HEOR, and market access and reimbursement can address these challenges.
Sponsor
Lumen Value & Access
Speaker
Kathy Belk, BA
Lumen Value & Access – a Healthcare Consultancy Group Company, New York City, NY, USA
Kathy oversees the HEOR, RWE, and modeling teams at HCG. She is a Health Services Researcher with more than 20 years of experience managing large, administrative & EMR datasets, as well as utilizing real-world data for clinical & economical research.
She has extensive experience leading data-driven projects for pharmaceutical & medical device companies in areas such as comparative effectiveness, market access, patient services, outcomes research & predictive modeling using both retrospective & prospective research designs. Her experience spans multiple clinical areas including oncology, chronic diseases, & rare diseases. Many of these studies have been presented at national & international scientific conferences & published in peer-reviewed journals.
Additionally, Kathy has participated in several national & hospital-specific pilot projects targeting quality measurement and improvement as well hospital payment strategy, including serving as the analytics lead for a pay-for-performance demonstration project with the Centers for Medicare & Medicaid Services.
Koen Degeling, PhD, MSc, BSc
Lumen Value & Access – a Healthcare Consultancy Group Company, New York, NY, USA
Dr Koen Degeling is a Research Scientist, Health Economic Modelling & Advanced Analytics at Lumen Value & Access, a Healthcare Consultancy Group company. He was trained as an Industrial Engineer specializing in Healthcare Technology and Management and holds a PhD in Advanced Health Economic Modelling from the University of Twente in the Netherlands. Prior to joining Lumen Value & Access, Koen worked on real-world data-driven health economic and health services research projects at the Cancer Health Services Research department of the University of Melbourne in Australia, where he continues to be involved as an honorary fellow. He is an active ISPOR member and currently serves on the Editorial Advisory Board for Values & Outcomes Spotlight and ISPOR New Professionals Steering Committee, is involved in several short courses and workshops, and has served as global chair, committee co-chair, and chapter president within the ISPOR Student Network.
Ramiro Gilardino, MD, MHS, MSc
Lumen Value & Access - A Healthcare Consultancy Group Co., New York, NY, USA
Dr. Ramiro Gilardino brings 13 years of experience in health economics, outcomes research, and global health policy in pharmaceutical, medical devices companies, and not-for-profit organizations both at the regional and global reach.
He developed customer-centered value propositions, registry studies, claims analysis, among other health economics tactics supporting access to medical technologies. He also was engaged in health policy matters, including health technology assessment policies, patient engagement, and value assessment, where he worked among decision-makers in emerging markets in LATAM and CEMEA.
He is deeply committed to delivering global health innovation for access to technologies while sustaining health systems.
Lucinda Orsini, DPM, MPH
COMPASS Pathways, Skillman, NJ, USA
4:00 PM - 4:30 PM
Break
In-person
4:30 PM - 5:30 PM
Concurrent Breakout Session 3
Artificial Intelligence in HEOR: A Student Roundtable Panel
Virtual
Level: Foundational
Highlighted as one of ISPOR’s Top HEOR Trends of 2022-2023, artificial intelligence (AI) has been significant in changing the scope of healthcare through its vast capability in data analysis. Student attendees are invited to listen in on this panel-style session to learn about the impact of AI in the field of HEOR and ask questions from three expert panelists. During this virtual session, the panelists will explore the history of AI in HEOR, current applications of AI, and the future impact of AI in HEOR.
Moderators
Shrey Gohil, B. Pharm.
University of Houston, Houston, TX, USA
Panelists
Chris Cameron, MSc, PhD
EVERSANA, Sydney, NS, Canada
William H. Crown, PhD
Brandeis University, Waltham, MA, USA
Dr.Crown is a Distinguished Research Scientist in the Heller School of Social Policy and Management, Brandeis University. He is an internationally recognized expert in real world data analysis, focusing upon research designs and statistical methods for drawing causal inferences from transactional health care datasets such as medical claims and electronic health records. Dr. Crown was 2013-14 President of ISPOR and currently co-chairs the ISPOR Task Force on Machine Learning. He is particularly interested in the intersection of machine learning and causal inference methods, as well as transparency in the conduct and reporting of empirical health care research.
William Padula, PhD, MS, MSc
University of Southern California, Los Angeles, CA, USA
William Padula, PhD is assistant professor of pharmaceutical & Health Economics at the University of Southern California School of Pharmacy, and a Fellow in the Leonard D. Schaeffer Center for Health Policy & Economics. His research interests include medical cost-effectiveness analysis and applications of machine learning to health economics and outcomes research. He was the 2021 recipient of ISPOR’s Bernie O’Brien New Investigator Award, and Is an Associate Editor for Value in Health.
Podium Session 3
HEOR Studies in Alzheimer's Disease and Related Dementias
Virtual
Moderator
Maciej Niewada, MD, PhD
Medical University of Warsaw and HealthQuest, Warsaw, Poland
Background:
1991-1997 Medical University of Białystok/Medical University of Warsaw (MD)
1995-2000 Warsaw School of Economics (MSc in Economics);
Professional experience:
since 1997 Medical University of Warsaw, Professor in Department of Clinical and Experimental Pharmacology – specialty: clinical pharmacology
2000-2006 Institute of Psychiatry and Neurology, Professor Assistant in 2nd Neurological Department – clinical specialty: neurology
2000-2004 Medical University of Warsaw - Ph.D. on cost of stroke from societal perspective
2013 - Thesis presented to achieve a habilitation qualification – Hospital Stroke Registry in Poland – analysis of three editions in 2001-2008. Patients clinical characteristic, therapeutic management and prognosis in ischaemic and hemorrhagic stroke.
since 2009 – CEO in Healthquest – consulting company focusing on market access and reimbursement application (more info: http://healthquest.pl/12,About.html )
Co-author of Polish guidelines on heath technology assessment adopted by The Agency for Health Technology Assessment in Poland (AHTAPol).
Founder member and current Past President of the Polish Pharmacoeconomic Society (ISPOR Poland Chapter).
Co-author of over hundred health technology assessments reports (including technologies dedicated for neurology and psychiatry), costing and epidemiological studies (i.e. diabetes, cardiovascular diseases, etc.), quality of life and utility studies (http://www.researchgate.net/profile/Maciej_Niewada ). Apart from pharma industry cooperating with Ministry of Health and National Health Fund as an external advisor.
P19: Utilization of Low-Value and High-Value Care and Its Association with Cognitive Impairment
5:15PM - 5:30PM
Barthold D1 , Jiang S 1 , Fendrick AM2 , Phelan E1 , Thielke S1 , Borson S3 , Basu A1 1 University of Washington, Seattle, WA, USA, 2 Departments of Internal Medicine; Center for Value-Based Insurance Design; University of Michigan, Ann Arbor, MI, USA, 3 University of Southern California, Los Angeles, CA, USA
OBJECTIVES: Utilization of low-value (LV) care and under-utilization of high-value (HV) care by people with cognitive impairment (both mild cognitive impairment (MCI) and Alzheimer’s disease and related dementias (ADRD)) could have large negative consequences for patient health, health system waste, and societal welfare. We aimed to assess the association of cognitive impairment (CI) and utilization of high and low-value care.
METHODS: Health and Retirement Study (HRS) data linked to Medicare claims (1996-2018), with CI measured using the modified telephone interview for cognitive status (mTICS) to identify patients with MCI and ADRD. In the 12 months following cognitive assessments, we examined claims for screening of colorectal cancer (CRC, HV for age <75), CRC (LV for age >85), prostate cancer (PC, LV for age>70), cervical cancer (CC, LV for age>65). We identified age, sex, race, socioeconomic status, comorbidities, and HRS wave as confounders. We examined the association between CI and utilization of HV and LV care by logistic regression in a cross-sectional manner. We then assessed changes in utilization after changes in CI status using individual fixed effect models in a longitudinal analysis. Lastly, we evaluated the association between utilization and duration of CI.
RESULTS: Cross-sectional analyses showed that compared to those with normal cognitive function, people with ADRD were less likely to receive LV CRC screening (OR=0.74, p=0.008), LV CC screening (OR=0.64, p<0.001), and LV PC screening (OR=0.69, p<0.001); HV CRC screening was less likely with weak significance (OR=0.73, p=0.055). Analyses of longitudinal CI showed utilization of these services did not change by within-individual changes in CI or duration of CI.
CONCLUSIONS: People with ADRD are less likely to get screening, regardless of its value, compared to people without CI. Changes in CI or CI duration did not affect the utilization of CRC, PC, and CC screening services.
P18: Rural–Urban Differences in Provider Types Diagnosing and Prescribing for Patients with Alzheimer's Disease
5:00PM - 5:15PM
Park J
OBJECTIVES:
Although roughly 20% of the US population lives in rural areas, only 9% of US physicians practice in rural communities. Importantly, the lack of access to specialists has been identified as the primary reason for worse health outcomes in rural patients with chronic conditions than their urban counterparts. This study aims to identify and compare the provider types diagnosing and prescribing for patients with Alzheimer’s disease (AD) in rural and urban settings.
METHODS:
We conducted a retrospective cohort study in IBM MarketScan
® to identify provider types including physician specialties, nurse practitioners, and physician assistants diagnosing and prescribing for patients with AD. Neurologists were classified as AD specialists for analysis. Patients newly diagnosed with AD and AD prescriptions written between 2016 and 2019 were analyzed. Two-sample z-tests were used to compare the proportion of specialists and non-physician providers diagnosing and prescribing for patients with AD in both settings.
RESULTS:
In total, 9,964 patients with AD and 103,067 AD prescriptions were included in our analyses. 16.96% of patients were diagnosed and 16.54% of prescriptions were written in rural settings. In rural areas, a significantly lower proportion of prescriptions were written by specialists (95% CI: -0.059, -0.046), while a significantly higher proportion of prescriptions were written by non-physicians (95% CI: 0.020, 0.028). Similarly, a significantly lower proportion of patients in rural settings were diagnosed by specialists (95% CI: -0.054, -0.019). The proportion of patients in rural and urban settings diagnosed by non-physicians did not significantly differ (95% CI: -0.014, 0.011).
CONCLUSION: Patients with AD in urban areas are more likely to be diagnosed by or receive prescriptions from AD specialists than patients in rural areas. Care patterns are expected to evolve with disease-modifying treatment approval—thus, proactive and strategic efforts to safeguard access to AD specialists for rural patients will be necessary.
P20: Risk of Antimuscarinic Initiation with Cholinesterase Inhibitor Use in Patients with Alzheimer's Disease
4:45PM - 5:00PM
Chikermane S 1 , Holmes HM2 , Sherer JT3 , Aparasu RR3 1 University of Houston, Houston, TX, USA, 2 University of Texas Health Science Center, Houston, TX, USA, 3 University of Houston, College of Pharmacy, Houston, TX, USA
OBJECTIVE: Antimuscarinic use in Alzheimer’s disease (AD) is a significant concern as it can worsen cognition due to its anticholinergic effects. This study evaluated the risk of antimuscarinic initiation with the use of individual Cholinesterase Inhibitors (CHEIs) in patients with AD.
METHODS:
This nested case-control study used 2013-2015 Medicare data involving patients
> 65 years with AD and without antimuscarinic use in 2013. Cases included those with antimuscarinic use in 2014-2015. Incidence density sampling was used to identify controls without antimuscarinic use, and variable-ratio matching on age was used to match cases and controls. CHEI use was categorized as current use (event-30 days), recent use (event-31 days to event-90 days), and past use (event-91 days to event-180 days). Covariates were identified using the Andersen Behavioral Model involving predisposing, enabling, and need factors during baseline and the 6-month period before the event date. Conditional logistic regression was performed to identify the association between the use of CHEIs and the risk of antimuscarinic initiation.
RESULTS:
The study included 1,909 cases and 9,064 controls; most were 80 years or older (70.2%), female (74.4%), non-Hispanic White (79.4%), had 1-2 Elixhauser comorbidities (40.6%), and were mildly frail (51.8%). After adjusting for potential confounders, current use of CHEI was associated with a 62% (Odds Ratio (OR) = 1.62, 95% CI: 1.18-2.21;p<0.01) increased risk of antimuscarinic use compared to non-current use. Current use of donepezil was associated with a 48% (OR=1.48, 95% CI: 1.03-2.12;p=0.04) increased risk, and current use of rivastigmine was associated with a 171% (OR=2.71, 95% CI: 1.46-5.03;p<0.01) increased risk of antimuscarinic use compared to those who were not current users.
CONCLUSION: The study found an increased risk of antimuscarinic initiation with the current use of CHEIs, mainly with donepezil and rivastigmine. Concerted efforts are needed to manage prescribing cascades in older adults with AD.
Emerging Evidence on SARS-COV2 Vaccine Utilization and Outcomes
In-person
P14: Identification of COVID-19 Vaccine Hesitancy Predictors
5:00PM - 5:15PM
Saldarriaga E
Background. Only 65.4% of the eligible population is vaccinated against COVID-19. Vaccine hesitancy is a complex phenomenon that plays an important role in explaining the low vaccination rates. Having a good understanding of the factors that drive hesitancy can improve our capacity to address it. In this study I use regularized regression to identify the most important predictors of vaccine hesitancy. Methods. The CDC estimated the proportion of people (18 and over) hesitant or unsure to take the COVID-19 vaccine at the county level; May 2021, data available at HHS-ASPE. The prediction set included demographic variables (age, race, and education), the CDC social vulnerability index which measures the relative stress people is under in a community, and the proportion of votes the Republican party received in the 2020 presidential election collected by MIT as a proxy of political affiliation at the community level. I fitted a LASSO regression model, implemented via leave-one-out cross-validation to find the penalization that minimizes the mean average error (MAE) and excludes variables without explanatory power. I used bootstrap with one-thousand iterations to estimate coefficients’ confidence intervals. Results. The final dataset included 3,111 counties. At optimum, the MAE was 2.7%, which denotes average prediction error of vaccine hesitancy. Among the most important drivers of hesitancy were proportion of people with some years of college (Coef: 12%; 95%CI: 7%, 16%), less than high-school diploma (10%; 5%, 15%), proportion of Black/African American (8%; 7%, 10%), and political affiliation (7%; 6%, 9%). The variables that reduce hesitancy were proportion of Asian population(-26%; -33%, -21%), people aged 65 and more (-21%; -26%, -13%), college graduates (-19%; -22%, -16%), and males (-17%; -25%, -9%). Conclusions. The model demonstrated good prediction properties. These results can help in deepen our understanding of the drivers of vaccine hesitancy, especially in acknowledging its multifactorial nature.
P16: COVID-19 Vaccination Strategies Considering a Vaccine Adapted to a New Variant of Concern: A Decision-Analytic Modeling Study
4:30PM - 4:45PM
Jahn B 1 , Sroczynski G1 , Bicher M2 , Rippinger C3 , Mühlberger N1 , Santamaria J1 , Urach C3 , Popper N4 , Siebert U5 1 UMIT - University for Health Sciences, Medical Informatics and Technology, Institute of Public Health, Medical Decision Making and Health Technology Assessment, Hall i.T., Austria, 2 dwh GmbH, dwh simulation-services; TU Wien, Institute for Information Systems Engineering, Vienna, Austria, 3 dwh GmbH, dwh simulation services, Vienna, Austria, 4 dwh GmbH, dwh simluation services; TU Wien, Institute for Information Systems and Engineering; DEXHELPP, Association for Decision Support Health Policy and Planning, Vienna, Austria, 5 UMIT - University for Health Sciences, Medical Informatics and Technology, Institute of Public Health, Medical Decision Making and HTA/ONCOTYROL - Center for Personalized, Austria; Harvard T.H.Chan School of Public Health, Dept. Health Policy & Management, Boston, MA, USA
Background: Several SARS-CoV-2 variants have developed requiring adaptations of current vaccines. Using a flexible concurrent disease model, we aim to identify optimal vaccination strategies for a COVID-19 vaccine adapted to a hypothetical variant of concern (VoC) focusing on age-specific prioritization during a period with still unvaccinated age groups and initially limited vaccination doses.Methods: A dynamic agent‐based population model for Austria was extended to a concurrent infectious disease model. A hypothetical, new VoC affects the current pandemic. The parameters for the variant’s infectivity, virulence, susceptibility to the current vaccine and initial vaccination coverage when the VoC is detected were varied in 81 scenarios. Evaluated vaccination strategies are: 1) re-vaccination of the elderly with the VoC-vaccine only, 2) vaccination of the unvaccinated with the VoC-vaccine only, 3) providing the VoC-vaccine and the current vaccine to the unvaccinated, and 4) providing VoC-vaccine to elderly and current vaccine to unvaccinated compared to 5) continuing with the current vaccine, only to minimize COVID‐19‐related hospitalizations and deaths. A time horizon of ten months was considered. Results: Prioritization of vaccination depends on target outcomes, combinations of VoC characteristics and initial vaccine coverage. For example, at a 75% reduced effectiveness of the current vaccine for the VoC: Minimizing hospitalizations are achieved by selecting strategy 1 followed by 3 (1 followed by 2) considering increased VoC-infectivity of 0% or 33% (66%) independent of vaccination coverage. To minimize deaths, strategy 2 is preferred followed by strategy 4 independent of increased VoC-infectivity when initially only individuals age 80+ are vaccinated. For an initial vaccination coverage of individuals age 65+, strategy 1 is preferred followed by 2 (increased 33% or 66% VoC-infectivity). Conclusion: Our study provides a flexible vaccination-decision basis for a partially vaccinated population with occurring VoC considering availability of VoC-adapted vaccine only or in addition to the current vaccines.
P13: Risk of COVID-19 Vaccine Breakthrough Infection in Fully Vaccinated Patients with Multiple Comorbid Conditions in the United States
5:15PM - 5:30PM
Liu M 1 , Wong AC2 , McPheeters JT2 , Dixon R3 , Madge V4 , Basra G4 , Sachdev A4 , Veeranki P2 1 Optum, sammamish, WA, USA, 2 Optum, Eden Prairie, MN, USA, 3 Optum, Athens, GA, USA, 4 Optum Global Solution India, Delhi, DL, India
OBJECTIVES:
Patients with multiple comorbid conditions are at increased risk of COVID-19 infection. Vaccines have been proven to be highly effective against COVID-19, however their effectiveness in patients with multiple comorbid conditions might be curtailed due to compromised immune function, increased viral replication
, and induced immunosenescence, subsequently leading to breakthrough infections (BIs). This study assessed the association of multiple comorbid conditions with COVID-19 BIs in fully vaccinated adults.
METHODS
: Using Optum Research Database, a large administrative claims database, adult enrollees with complete vaccination status were identified between December 1, 2020 and May 31, 2021. COVID-19 BIs was defined using ICD-10 diagnosis codes for COVID-19 ≥14 days of completing vaccination. Patients with multiple comorbid conditions were identified using ICD-10 diagnosis codes, and categorized into 0, 1, 2, 3+ groups. Logistic regression models were used to estimate the association between COVID-19 BIs and multiple comorbid conditions, separately, and categories of comorbid conditions adjusting for patient demographics.
RESULTS
: The study included 1,520,838 fully vaccinated adults. Mean age was 47.6 years (±14.5) with 53.1% of females, and most patients resided in Midwest (34%) and Southern regions (33.7%). About 1.1% of patients reported COVID-19 BIs. Patients with HIV/Immunodeficiency (1.59%), asthma (1.56%) and stroke (1.4%) were more likely to have BIs. Compared to patients with no comorbid conditions, patients with 1, 2 and 3+ comorbid conditions were significantly associated with 1.3 (95% confidence interval (CI): 1.25-1.36), 1.5 (95% CI: 1.42-1.59) and 2.26 (95% CI: 2.13-2.41) times increased odds of COVID-19 BIs after adjusted for patient demographics and lifestyle factors.
CONCLUSIONS
: The study provides real-world evidence of role of multiple comorbidities in increased risk of COVID-19 BIs among fully vaccinated individuals. As new SARS-CoV variants emerge, continued detection, prevention, and management efforts of comorbid conditions is important to sustain high effectiveness of COVID-19 vaccinations and reduce BIs.
P15: Drivers of Health Disparities and Consequences for COVID-19 Vaccine Choices: Modelling Health Preference Heterogeneity Among Underserved Populations
4:45PM - 5:00PM
Van Den Broek-Altenburg E 1 , Benson J2 , Atherly A1 , Hess S3 1 University of Vermont, Burlington, VT, USA, 2 University of Vermont, Burlington, Netherlands, 3 University of Leeds, Leeds, UK
OBJECTIVES: Reducing the extra burden COVID-19 is having on people already facing disparities is among national priorities for the COVID-19 vaccine rollout. The objective of this study was to identify key factors underlying the disparities in COVID-19 vaccination.
METHODS: We gathered longitudinal data from a representative sample of the U.S. population of the four largest U.S. states. We used a discrete choice experiment asking respondents to imagine a situation where a number of vaccines for COVID-19 had been developed. They were then faced with six scenarios where in each task, two possible vaccines were described with seven attributes. We estimated five sets of choice models and compared model fit, including a simple multinomial logit, nested logit (NL) grouping together all vaccine options, NL with socio-demographic effects, latent class (LC) with purely random heterogeneity and LC with the same socio-demographic effects as the NL models.
RESULTS: Overall, we found that individuals who identify as Black had lower rates of vaccine hesitancy than those who identify as White. This was true overall, by latent class and within latent class. This suggests that, Blacks are not universally more vaccine hesitant. Combining the respondents who would not consider a vaccine (17%) with those who would consider one but ultimately choose not to vaccinate (11%), our findings indicate that more than 1 in 4 (28%) persons will not be willing to vaccinate. The no-vaccine rate is highest in Whites and lowest in Blacks.
CONCLUSIONS: Lower rates of vaccination among Black Americans do not reflect lower rates of racially motivated vaccine hesitancy. Instead, these lower rates reflect a higher proportion of Blacks among groups with vaccine hesitancy – lower income and lower educated individuals. To reduce racial disparities in vaccination rates, it will be necessary to address vaccine hesitancy more broadly in disadvantaged populations.
Concurrent Breakout Session 3
Using Real-World Data to Provide Insights in the COVID-19 Crisis: Challenges and Opportunities
In-person
Level: Intermediate
PURPOSE: The Covid-19 pandemic has disrupted all aspects of healthcare delivery and generated controversy in matters related to disease prevention, treatment options, and even reporting of events. At the same time, the pandemic has shown that drugs and vaccines can be developed, approved, and marketed in one year, instead of the usual 10-15 years. This may put greater importance on and increases the value of real-world data (RWD). The objective of this workshop is to highlight the unique opportunities and challenges of using RWD during the Covid-19 pandemic. Particular attention will be given to the use of electronic health records (EHR) and health insurance claims as alternative or additional data sources compared to data used by local and central governments.
DESCRIPTION: This workshop will start by giving a broad overview of the different types of RWD that can be used for Covid-19 research (10 min). This is followed by discussing the unique challenges of building fit-for-purpose real-world datasets from health insurance claims or EHRs of Covid-19 patients (20 min). The next 10 minutes will be used to review some of the regulatory aspects of RWD in the Covid-19 era. Next, an overview of the opportunities of using RWD for Covid-19 research are discussed. The audience will select through real-time polling what use cases of RWD in Covid-19 research should be presented (15 min), with topics including for example the natural history of Covid-19; uptake and effectiveness of Covid-19 vaccines in the post-marketing setting; medical cost related to the Covid-19 crisis; consequences of the pandemic for the execution of clinical trials of non-Covid-19 drugs; and under-utilization of routine healthcare services. The last 5 minutes will be used to discuss any other challenges and opportunities in the use of RWD for Covid-19 research that are put forward by the audience.
Discussion Leaders
Brian Buysse, PhD
Syneos Health, Farnborough, CS, United Kingdom
Brian Buysse, PhD is a Senior Director Epidemiology at Syneos Health, a company he joined in 2017 and where he designs and oversees the scientific conduct of real-world research. Prior to that, he has held positions as epidemiologist, statistician, and lead statistician at IQVIA in the UK (previously IMS Health, 2015-2017), and had a 12-year long academic career in the Netherlands (2003-2006), the United States (2007) and Germany (2008-2014). He is currently based on Spain.
Discussants
Tara Isherwood, MSc, BSc
Syneos Health, Maidenhead, WNM, United Kingdom
Tabassum Khan, MD, MPH
Komodo Health, Atlanta, GA, USA
Jennifer Stacey, BS
TriNetX Inc., Cambridge, MA, USA
Jennifer Stacey has 20 years of clinical research experience ranging from biomarker and target discovery to strategic conduction of drug development programs. By leveraging big data with health analytics and informatics, she has provided consultative trial planning, competitive intelligence, and custom RWE project work for over 100 pharmaceutical and contract research organizations. Prior to joining TriNetX, Jennifer held scientific positions at Cell Signaling Technology, Citeline, and inVentiv Health. She received her BS in Biology/Pre-Medicine from St. Lawrence University.
Is Open-Source Model Development Sustainable?
Virtual
Level: Intermediate
PURPOSE: How can development, use, and maintenance of open-source models be encouraged in the absence of financial (and other) incentives around intellectual property? This workshop will consider alternatives and financial approaches in the context of open-source modelling, as well as advantages and disadvantages of each.
DESCRIPTION: Open-source economic models have been slow to gain ground. While few stakeholders are explicitly opposed to open-source modelling, there remain several barriers to adoption. One is the financial aspect of developing and sustaining these models. A closely related one is the ownership of the intellectual property. The discussants will present the opportunities and challenges of developing and maintaining open-source models. Due to their nature as a public good, it can be difficult to create financial incentives to encourage the development and adoption of these models. Dr. Chapman (10 min.) will moderate the discussion and provide a brief background of the issue, as well as pose key questions to the discussants. Dr. Arnold (10 min.) will discuss the history around the call for health economic models to be available as open-source, and learnings from the recently-conducted ISPOR Open-Source Model SIG survey around barriers to their development. Dr. Caro (10 min.) will discuss the opportunities and challenges of building these models and making them freely available based on examples from his experience. Dr. Sadatsafavi (10 min.) will discuss the concepts of transparency versus accessibility, potential solutions to preserve intellectual property of model developers while enabling stakeholders to gain access to the output of such models, and the need for a common standard for interrogating models in addition to simply making their code available. Time will be reserved for audience questions.
Those who develop models, use them in decision making, or fund their development will benefit from attending.
Discussion Leaders
Richard Chapman, PhD, MS
The Innovation and Value Initiative, Alexandria, VA, USA
Dr. Chapman is the Chief Science Officer for the Innovation and Value Initiative (IVI), a nonprofit research organization whose mission is to advance the science, practice, and use of value assessment in healthcare to make it more meaningful to those who receive, provide, and pay for care. Prior to that, Dr. Chapman was Director of Health Economics at the Institute for Clinical and Economic Review, where he led development of economic evaluations assessing the potential cost-effectiveness and budgetary impact of clinical interventions.
Discussants
Renée Arnold, PharmD, RPh
National Institutes of Health, New York, NY, USA
Renée JG Arnold is currently Entrepreneur-in-Residence (EIR), HEOR, NHLBI (NIH) and EIR, Biohealth Innovation, Inc, New York, NY; Adjunct Full Professor, Master of Public Health Program, Department of Environmental Medicine and Public Health, Icahn School of Medicine at Mount Sinai, New York, NY, USA, where she has developed and teaches the pharmacoeconomics coursework. She is also president & CEO of Arnold Consultancy & Technology, LLC where she oversees outcomes research and develops affiliated software for pharmaceutical and federal government programs. Her special interest in evidence-based health derives from her research that deals with use of technology to collect and/or model real-world data for use in rational decision making by healthcare practitioners and policy makers. Dr. Arnold completed her undergraduate training at the University of Maryland and received her Doctor of Pharmacy degree from the University of Southern California in Los Angeles. She also completed a one-year post-doctoral residency at University Hospital in San Diego/University of California at San Francisco School of Pharmacy. Dr. Arnold was previously vice president of HEOR at Quorum Consulting, Inc./Navigant Consulting; principal of IMS Health (IQVIA); and president and co-founder of Pharmacon International, Inc. Center for Health Outcomes Excellence.
Dr. Arnold is a founding member of ISPOR and is the head of both the ISPOR Distance Learning Program, as well as the head of the newly-developed Open Source Models Special Interest Group. She is an author/co-author of numerous articles, book chapters, and books in the areas of pharmacology, pharmacoeconomics, and cost-containment strategies.
J. Jaime Caro, MDCM, FACP, FRCPC
McGill University, London School of Economics, Evidera, Bethesda, MD, USA
Jaime Caro, Chief Scientist at Evidera and Professor of Epidemiology and of Medicine at McGill University and Professor in Practice at London School of Economics. He pioneered the use of DES, developed the Simulated Treatment Comparison approach and proposed the efficiency frontier as an alternative to cost/QALY. Recently, he has developed a new modelling technique, DICE simulation, tailored to problems in Health Technology Assessment.
Mohsen Sadatsafavi, MD, PhD
University of British Columbia, Vancouver, Canada
Mohsen Sadatsafavi, MD, PhD, is an epidemiologist and health economist and Associate Professor at The University of British Columbia - Faculty of Pharmaceutical Sciences. He leads the Respiratory Evaluation Sciences Program (http://resp.core.ubc.ca), a program of outcomes research dedicated to asthma and Chronic Obstructive Pulmonary Disease. He is interested in applying principles of Decision Theory at both clinical and policy levels. For his work, he has received salary awards from The Canadian Institutes of Health Research, Michael Smith Foundation for Health Research, Association of Faculties of Pharmaceutical Sciences of Canada, and the Canadian Society for Pharmaceutical Sciences.
In or Out? Challenges and Opportunities to the Collection of Patient Preference Information Within Clinical Trials
In-person & Virtual
Level: Intermediate
PURPOSE: To discuss opportunities, challenges, and approaches to collecting patient preference information in clinical trials.
DESCRIPTION: New treatments are increasingly evaluated in a patient-centred manner in which benefit-risk assessments combine clinical data with patient preference information (PPI). For example, the FDA has issued specific guidance on PPI, and the EMA issued a positive opinion on PPI qualification. To ensure regulators can consider PPI, studies need to rise to the level of valid scientific evidence. For example, in most cases, the study population in the PPI study should reflect as closely as possible the proposed indicated population. While most patient-preference studies are constrained in their ability to comply with clinical recruitment standards, including PPI in clinical trials seems an intuitive response to such concerns. Preference elicitation within the trial population can also aid in exploring preference heterogeneity based on robust clinical data. However, the decision to collect PPI in a trial requires careful consideration of multiple factors, including but not limited to the alignment of instrument development and protocol timelines, managing the cognitive burden imposed on patients, legal/ethical requirements and opportunity costs. Dr. Ng will discuss how PPI collected in clinical trials can support regulatory benefit-risk assessments. Dr. Katz will draw on her experience using PPI in clinical trials to inform regulatory benefit-risk assessments from an industry perspective. Dr. Danyliv will discuss the role that PPI collected in trials can take in HTAs. Dr. Heidenreich will cover the operational perspective, with a focus on best practice. To enhance the discussion, session leaders will use interactive polling to elicit facilitators and barriers experienced by session participants.
Discussion Leaders
Sebastian Heidenreich, MSc, PhD
Evidera, London, LON, United Kingdom
Discussants
Andrii Danyliv, PhD
Novartis Pharma AG, Basel, BS, Switzerland
Andriy Danyliv works as the Head of HEOR Innovation at Novartis. He graduated as PhD in Health Service Research at the Maastricht University in 2014 and after several years in health economics research moved to work in HEOR for pharma industry. In academia Andriy worked on research in the areas applying stated preferences for the assessment of patient payments, economic evaluation of gestational diabetes programs, quality of primary care. At Novartis, he is looking for and piloting novel methods and approaches in HEOR space, and the use of patient preferences in HTA is one of the key interests. Andriy is involved in IMI PREFER and ISPOR Task Force on the use of patient preferences for decision making.
Eva Katz, PhD, MPH
Johnson & Johnson Inc, Raritan, NJ, USA
Xinyi Ng, MS, PhD
FDA, Silver Spring, MD, USA
Xinyi Ng, PhD is a visiting scientist with the Analytics Benefit-Risk Assessment (ABRA) team with the Office of Biostatistics and Pharmacovigilance, CBER, FDA. She leads patient preference information (PPI) studies within CBER and is part of the science of patient input (SPI) working group, which aims to advance the use of patient experience data, including PPI, to inform regulatory science. Prior to joining the FDA, she was a scientist in a HEOR consulting firm and have experience conducting PPI studies. She has a PhD in Pharmaceutical Health Services Research from the University of Maryland Baltimore.
Measuring Health Equity: Lessons from an Integrated Healthcare System
In-person
Level: Foundational
PURPOSE: To define health equity and discuss and practice methods for measurement.
DESCRIPTION: Recently health equity has become a high priority for healthcare delivery systems across the U.S., especially as the COVID-19 pandemic has exposed long-standing inequities among racially, socially, and economically minoritized and marginalized populations. However, we struggle as a society to solve these issues, in part, because we lack appropriate methods to quantify the inequities and identify drivers – both necessary for the development of viable solutions and the monitoring of impact.
Understanding health outcomes in these underserved population sub-groups requires innovative analytics and dynamic application of health and population data. Use of proxy indicators, such as quality of life and mortality, to measure health disparities has been limited and rarely implemented at a population-level. The members of this panel designed, developed, and implemented a Health Equity Index (HEI) and a COVID-19 Vaccine Equity Index (CVEI), using analytic methods intended to enable healthcare systems to identify and prioritize the remediation of health disparities. These two metrics are somewhat atypically designed to compare a subgroup to the ideal version of itself, rather than comparing it to a reference subgroup which often represents a majority (
e.g. non-Hispanic White individuals). Because of this nuance, we are able to capture equity issues in an intersection of multiple types of subgroups such as low income, uninsured, housing insecure, etc... It is our hope that these tools will be adapted for other conditions and used by any institution striving to address health inequities. In this workshop, we will share our measurement methodology in several topic areas, and we will distribute a (simulated) dataset to provide hands on experience in using our measurement approaches. In sharing this work, we hope to begin to address the pressing need for methods to identify and quantify health inequities.
Discussion Leaders
Alice Pressman, PhD, MS
Precision Medicine Group, Lafayette, CA, USA
Alice Pressman, PhD, MS, a Vice President at PRECISIONheor, is an epidemiologist and biostatistician specializing in evaluation methodology, clinical trials methods, health equity/disparities, health systems evaluation, and chronic pain research. For the past 30+ years, she has served as a research investigator and biostatistician for clinical and epidemiologic research studies in diverse settings including academia, community-based research institutes, healthcare systems, and most recently, a healthcare consulting firm. She holds a master’s degree in statistics, and a PhD in epidemiology from the University of Wisconsin and University of California, Berkeley, respectively.
Discussants
Kristen Azar, RN, MSN/MPH, FAHA
Sutter Health, Palo Alto, CA, USA
Kristen Azar serves as the Scientific Medical Director for the Sutter Health Institute for Advancing Health Equity (IAHE) and leads an experienced, multidisciplinary group of clinicians, researchers and administrators. The team is focused on using resources and data within Sutter’s integrated network to incubate innovative solutions to eliminate health disparities and advance the science of health equity. Over more than a decade, she has led numerous published studies in the areas of health services research, healthcare disparities and health equity.
Azar holds graduate degrees from Johns Hopkins in nursing and public health. She completed undergraduate studies at the University of California, Berkeley and obtained a Bachelor of Science in Nursing from Johns Hopkins University. She is a member of the Sigma Theta Tau International Honor Society of Nursing, as well as the Delta Omega Honor Society for Public Health. Additionally, she was named a Fellow of the American Heart Association in 2018. Currently, she is a PhD candidate at the University of California San Francisco in Epidemiology and Translational Science.
Azar joined Sutter Health’s research enterprise in 2009. Her research is aimed at identifying and addressing health disparities and examining the impact of social determinants of health on chronic disease risk and management. She has diverse clinical experience as a public health and preventive cardiology nurse in and outside the U.S., and has worked with racially, ethnically and socioeconomically diverse populations.
As Scientific Medical Director for the Sutter Health IAHE, Azar is a recognized health equity leader and received a 2021 Nurse Leader Award for her work in health equity, from the Association of California Nurse Leaders. Under her leadership, with support from its advisors and essential partners, IAHE has made significant progress in developing a multi-year strategic plan, cultivating partnerships with industry, academia and community groups, identifying opportunities for fundraising and joining the national dialogue around health equity. The Institute, established in December 2020, achieved many accomplishments in its inaugural year. The Institute team authored 16 publications in prominent journals, led three health system-wide initiative, produced impactful thought leadership, and garnered significant media interest for their work, including efforts to bring more equitable care to individuals impacted by COVID-19.
Stephen Lockhart, MD, PhD
Sutter Health Institute for Advancing Health Equity, Oakland, CA, USA
Stephen H. Lockhart, M.D., PhD is the Founding Director Emeritus of the Sutter Health Institute for Advancing Health Equity, and prior to retirement, served as Vice President and Chief Medical Officer for Sutter Health. He is a Rhodes Scholar, with a master's degree in economics from Oxford University, and M.D. and Ph.D. degrees from Cornell. He serves on the California Surgeon General’s Advisory Council focused on the study of adverse childhood experiences and served on California Gov. Jerry Brown’s Advisory Committee on Precision Medicine. Dr. Lockhart served on the Board of Directors (including six years as Chair) of NatureBridge. Dr. Lockhart is a current or past board member of ECRI (a healthcare safety nonprofit), the National Parks Conservation Association, REI, the David & Lucile Packard Foundation, the E.O. Wilson Biodiversity Foundation, and is chair of Parks California.
Making Your Key Messages Heard and Understood: Strategies and Methods for Effective Communication of Scientific Information to Non-Technical Audiences
In-person
Level: Foundational
PURPOSE: Much of health economics and outcomes research (HEOR) involves generating a range of evidence to substantiate the burden of illness and the benefits of new treatments. Communications of that work, such as those presented at this conference, are largely scientific. However, HEOR evidence is increasingly communicated to diverse audiences, including clinicians, policy makers, and patients. This workshop will discuss the importance of effectively communicating technical information to a range of audiences and practical methods for doing so.
DESCRIPTION: Novel communication strategies are required to make key messages about HEOR evidence compelling to less technical audiences and inspire them to take action. This goal is different from that of scientific publications, which is to convey sufficient methodologic detail so the results can be replicated. For diverse audiences to value the information presented, the focus of the communication should be the interpretation and relevance of facts and figures rather than the facts and figures themselves. Additionally, the decreasing frequency of face-to-face interactions heightens the need for simple and interactive communications where the cognitive effort required to understand their key messages is relatively low. In this presentation, users will learn (1) evidence-based principles for the effective communication of technical information; (2) practical methods derived from design and user-experience expertise to make communications targeted and efficient; and (3) case studies and outcomes of real-world communication strategies employed by a pharmaceutical manufacturer. The audience will participate in live demonstrations of alternative communication strategies and how they differ in effectiveness. Participants will also be encouraged to discuss their own communication challenges and mitigation strategies.
Discussion Leaders
Sonya J. Snedecor, PhD
OPEN Health, Bethesda, MD, USA
Discussants
Ali Alobaidi, PharmD, MS
AbbVie Inc., North Chicago, IL, USA
Dr. Ali Alobaidi is Associate Director in the Global Health Economics and Outcomes Research (HEOR) Strategy Team at AbbVie Inc. Ali leads global evidence generation efforts to strengthen differentiation and value demonstration of neurology products to treat patients with advanced Parkinson’s Disease (PD). Prior to this role, Ali held positions of increasing responsibilities including HEOR Manager and Post-doctoral Fellow. Ali received his MS degree with focus in Pharmacoepidemiology and Health Outcomes Research and Doctor of Pharmacy (PharmD) degree from the University of Illinois at Chicago.
How Much Weight Should be Placed on Additional Value Elements in Health Technology Assessment?
In-person & Virtual
ISSUE: Health technology assessment (HTA) organizations support direct or indirect pricing and reimbursement negotiations using systematic methods that estimate the value of health interventions. Current HTA relies on clinical evidence generated for regulatory approval to inform comparative- and cost-effectiveness calculations. However, there have been recent calls to include additional value elements more explicitly (e.g., real-option value, value of hope, scientific spillovers, insurance value, etc.) that may quantify the wider impact interventions have on patients and society . These recent calls have generated suggested alternatives and supplements to current HTA practice. However, without understanding the weight society places on additional value elements, the return on investment for evidence generation on additional value elements is unclear.
OVERVIEW: This global HTA panel will explore the question: how much weight should be placed on additional patient and societal value elements in reference to current HTA practice? Brett McQueen will introduce the topic by presenting a background on current HTA practice and emerging alternatives and supplements to incorporate additional value elements. Jon Campbell will provide an overview of current practices at the Institute for Clinical and Economic Review and summarize how the organization is addressing calls to include additional value elements in their assessments. Jacoline Bouvy will provide a similar overview from the perspective of an HTA body . Zoltán Kaló will provide an overview from the perspective of lower income countries. Core to the issue, each panelist will opine on the weight HTA bodies in their own settings should use for additional value elements. Differences in opinion will be explored and debated. Finally, we will engage the audience by asking how much weight they would place on additional value elements while also fielding questions for the panelists.
Moderators
Robert McQueen, PhD
University of Colorado, Aurora, CO, USA
R. Brett McQueen is an Assistant Professor at the University of Colorado (CU) Skaggs School of Pharmacy and Pharmaceutical Sciences, and member in the Center for Pharmaceutical Outcomes Research. His research interests include decision-analytic modeling applications and methodology, applied microeconometrics in health, and novel value assessment methods. Brett has current funding in micro-costing health interventions, evaluating performance-based risk sharing agreements, estimating patient and payer preferences for various pharmaceuticals, and novel value assessment methods. He is the course director for “Pharmaceutical Economics and Policy Analysis” in the Pharmaceutical Outcomes Research PhD program at CU.
Panelists
Jon Campbell, PhD
Institute for Clinical and Economic Review, Hingham, MA, USA
Jon (Jonathan D.) Campbell is Senior Vice President for Health Economics at the Institute for Clinical and Economic Review (ICER). Jon joined ICER’s senior management team as a leader in value assessment methods and application; he oversees the growth of ICER’s health economics efforts and leads the continued innovation of ICER’s value assessment methodology. Additionally, Jon continues to build bridges within the global health economics community through engagement with ICER’s Health Economic Council and through leadership and participation in health technology assessment societies and agencies. Further, Jon seeks creative value assessment solutions for ICER’s diverse stakeholders by prioritizing improved outcomes for patients.
Jon is an affiliate faculty member at Tufts University School of Medicine in the Center for the Evaluation of Value and Risk in Health. Jon is an author of over 100 peer-reviewed manuscripts in the field of value assessment as well as an author on many ICER assessments. Jon is a former ICER Health Economics Council member and five-year external collaborator through his former role as Associate Professor with tenure at the University of Colorado Anschutz Medical Campus. He holds graduate training degrees in pharmaceutical outcomes research (PhD) and biostatistics (MS) from the University of Washington. He graduated with a BA in mathematics and chemistry from St. Olaf College.
Jon also enjoys playing most racket-related sports. He grew up playing pickleball in the Seattle area and is seeking opportunities to spread the pickleball love in the Boston area.
Zoltan Kalo, PhD
1) Semmelweis University; 2) Syreon Research Institute, Budapest, Hungary
Zoltán Kaló is a professor of Health Economics at the Center for Health Technology Assessment of Semmelweis University in Budapest, Hungary. Before moving to Semmelweis University in July 2019 he was the founder and co-director of an international master program in Health Policy, Planning, and Financing at Eötvös Loránd University (ELTE).
Dr. Kaló is also the founder and leader of Syreon Research Institute, an international research corporation specializing in health policy, health economic modeling, and technology assessment.
He has 25 years of international experience in academia and industry, specializing in health systems design, HTA implementation, health economics and outcomes research, patient access, and pricing policies of healthcare technologies.
Dr. Kaló serves as a policy advisor to public decision makers and global healthcare corporations. He is a Scientific Committee member of the Innovative Medicines Initiative 2 Joint Undertaking (IMI2 JU). He was a director of ISPOR between 2012-2014, and the chair of ISPOR Central and Eastern European Network Executive Committee between 2013-2015.
Sam Roberts, DPhil
National Institute for Health and Care Excellence (NICE), London, MA, United Kingdom
5:30 PM - 6:30 PM
Poster Session 2 Poster Tours
New this Year – Poster Tours! ISPOR has curated collections of research posters for you within each of the poster sessions. Each tour will feature high impact abstracts within a specific topical area and will include a tour guide as well as the poster authors to share their work and engage in discussions with you. Visit the Learning Formats page for more information. Poster Tour: LMIC Focused Work
In-person
Posters featured in this tour:
EE4: Cost-Effectiveness Analysis of Trastuzumab Emtansine in the Treatment of Patients with HER2+ EARLY Breast Cancer (EBC) and Residual Disease after Neoadjuvant Treatment in Colombia
EE117: Estimating Economic and Disease Burden of Snakebite in South East Asia: A Decision Analytic Model
EE327: Surgical Sites Infection: A Micro-Costing Approach from the Brazilian Private Healthcare System Perspective
EE448: Cost-Utility Analysis of Benralizumab in Severe Eosinophilic Asthma in Panama
EPH93: Treatment Patterns of Triple Negative Breast Cancer in Brazilian Private Healthcare Setting: a Claim Database Study
Poster Tour: Diabetes/Endocrine/Metabolic Disorders
In-person
Posters featured in this tour:
EE116: Implications of Utilizing Survey Versus Administrative Claims Data to Evaluate the Cost of Diabetes Among Medicare Beneficiaries with Cancer: A Methods Study
EE205: Cost-Effectiveness Analysis of Empagliflozin Versus Albiglutide Among Individuals with Type 2 Diabetes in the US
EE366: Contrasting Three HbA1c Progression Equations Using the IQVIA Core Diabetes Model
HPR31: Impact of Telehealth Use during the COVID-19 Pandemic on Glycemic Control and Other Biomarkers Among Type 2 Diabetes Patients
RWD38: Early Treatment Modification for Type 2 Diabetes in a US Real-World Population: Impact on Glycemic Control, Diabetic Complications, and Healthcare Utilization
5:30 PM - 7:00 PM
ISPOR Welcome Reception (Exhibit Hall)
In-person
6:00 PM - 6:15 PM
Special Student Session
Health Care Spending Guiding Principles: A Checklist for Assessing Health Care Spending Estimates and Policies for Patient-centered Care
In-person
The continued rise in health care spending and patient out-of-pocket (OOP) costs in the United States has led to an intense debate among policymakers and other health care stakeholders on managing increasing costs. Numerous factors contribute to increased spending and OOP costs, including an aging population; structural, administrative and process complexity in the health system; innovative methods for disease prevention; improved speed and accuracy of diagnoses; adoption of novel treatments; health care prices and component costs; and benefit design.
The Health Care Spending Guiding Principles are intended to guide a rigorous, evidence-based evaluation and discussion of health care spending estimates and policies. They aim to improve health care spending efficiency and maximize patient health. These principles can be used to
guide and evaluate health spending analyses’ methodological rigor, limitations and alignment with patient-centered care, and
guide and evaluate health spending policy, including identification of tradeoffs, risks of unintended consequences and implications for patient-centered care. Users can leverage the results of their analyses to inform policymakers, media and the general public.
Sponsor
National Pharmaceutical Council
Moderator
Michael Ciarametaro, MBA
National Pharmaceutical Council, Washington, DC, USA
Michael Ciarametaro serves as the National Pharmaceutical Council's vice president of research. In this position, Mr. Ciarametaro conducts health policy research focused on addressing rising health spending and better aligning stakeholder incentives with value.
Mr. Ciarametaro has 20 years of experience creating value in the health care system through policy, research and leadership. His cross-disciplinary expertise spans multiple roles (policy researcher, strategic value and market access consultant, financial analyst, operations and process designer) and multiple areas of the health care ecosystem (health policy research organization, biopharmaceutical industry, private payers, public payers). For the past six-plus years, Mr. Ciarametaro has been focused on health policy research with an emphasis on addressing rising health spending and better aligning stakeholder incentives with value.
Mr. Ciarametaro holds a Bachelor of Science from the University of Virginia and received his MBA from George Mason University.
Tue May 17
7:30 AM - 5:30 PM
ISPOR 2022 Registration Hours
In-person
The ISPOR Registration Desk will be open for in-person participants.
8:30 AM - 9:45 AM
Plenary Session 2
Welcome Remarks
Welcome Remarks
CEO Welcome
Nancy Berg, ISPOR CEO & Executive Director
Incoming President Address
Jan Hansen, PhD, 2021-2022 President-Elect
ISPOR Science Office Update
Richard Willke, PhD, Chief Science Officer
Program Committee Co-Chair Welcome
Ran Balicer, MD, PhD, MPH, ISPOR 2022 Program Committee Co-Chair
Can Big Data Analytics Deliver on the Promises of Personalized Medicine for All? Unpacking the Health Equity Considerations
Successful application of big data analytics to personalized medicine often involves probabilistic risk assessment models to apply individual patient characteristics to treatment decisions. However, these models have been criticized for insufficient “contextual specificity” and for their vulnerability to incorrect interpretations of the predictive model results. Contextual specificity reflects the rich diversity of individuals who interact with the healthcare system as characterized by demographics, cultures, lifestyles, preferences, socio-economic status, and genetic endowments. Moreover, the reality is that many datasets available to healthcare systems, researchers, payers, and regulatory decision makers do not fully reflect all groups or experiences within healthcare systems. In addition, studies using healthcare data do not always interpret race and ethnicity within the context of the often-racialized environment in which healthcare utilization occurs. What are the tradeoffs associated with including race as a covariate in prediction models? Considering the measures of race available in healthcare system data, are these data ‘fit for purpose’ when the goal is to guide healthcare delivery in racially, ethnically, and culturally diverse patient populations? How can HEOR inform and guide the use of healthcare system data while advancing health equity goals? This session will discuss how we can appropriately utilize healthcare data for probabilistic risk assessment as well as illustrate HEOR’s evolving and increasing role in addressing these issues.
*Speakers to be added as confirmed!
Moderators
Ebere Onukwugha, BSc, MSc, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
Eberechukwu Onukwugha, PhD is an Associate Professor in the Department of Pharmaceutical Health Services Research and is the Executive Director of Pharmaceutical Research Computing at the University of Maryland School of Pharmacy. She received a Master of Science in agricultural and applied economics as well as a Doctor of Philosophy in economics (concentration: econometrics) from Virginia Tech. Dr. Onukwugha was a recipient of the PhRMA Foundation’s Post-Doctoral Fellowship in health economics and outcomes research. Dr. Onukwugha examines the costs and health outcomes associated with health-related decisions as well as the institutional and environmental context framing these decisions.
Speakers
Bob Darin, MBA
Blue Health Intelligence, Chicago, IL, USA
Interim Chief Executive Officer
Bob Darin leads Blue Health Intelligence’s® (BHI®) efforts to leverage the healthcare industry’s most comprehensive data assets to drive improvements in health outcomes and advance value-based care. Darin is a national expert in the application of analytics and data at scale to unlock value in healthcare. His background includes innovative leadership in managed care, pharmacy and pharmacy benefits management, hospitals, life sciences, and real-world evidence. He has developed and deployed end-to-end data and analytics strategies that have resulted in improved patient outcomes, reduced hospitalizations, and cost for high risk/high need patients, lowering out of pocket costs to patients, and increasing transparency for patients, payors, and physicians. Darin has also led multiple initiatives to reduce inequities in healthcare by identifying and eliminating bias in advanced analytics/AI algorithms.
Before joining BHI in 2022, Darin spent nine years as Senior Vice President at CVS Health and held positions of Chief Analytics Officer and Chief Data Officer. He also has held executive positions at Cardinal Health, Bupa/ Health Dialog, and HealthBenchmarks, Inc. (now part of IQVIA), and he began his healthcare career at Blue Cross and Blue Shield Association.
Darin holds an honors Master of Business Administration degree in analytic finance from the University of Chicago, Graduate School of Business, and received a magna cum laude degree in economics from Harvard College.
Charles Manski, PhD
Northwestern University, Chicago, IL, USA
CHARLES F. MANSKI is Board of Trustees Professor in Economics at Northwestern University. He received his B.S. and Ph.D. in economics from M. I. T. in 1970 and 1973. His research spans econometrics, judgment and decision, and analysis of public policy. His books include Patient Care under Uncertainty (Princeton, 2019) and Public Policy in an Uncertain World (Harvard, 2013). He is an elected Member of the National Academy of Sciences. He is an elected Fellow of the American Academy of Arts and Sciences, the Econometric Society, the American Statistical Association, the American Association for the Advancement of Science, and Corresponding Fellow of the British Academy.
George Mensah, MD, FACC
National Heart, Lung, and Blood Institute, Washington, DC, USA
Michael Pencina, PhD
Duke University School of Medicine, Durham, NC, USA
Michael Pencina, PhD, is Vice Dean for Data Science for Duke University School of Medicine, Professor of Biostatistics & Bioinformatics, and Director of Duke AI Health. He leads quantitative science for training, laboratory, clinical and data science missions. He is an international expert in risk prediction model development and evaluation; novel, efficient clinical study design; and machine learning for medical decision support. Recognized as a Highly Cited Researcher from 2014-21, he serves as Deputy Editor for Statistics, JAMA-Cardiology, and Associate Editor, Statistics in Medicine.
9:45 AM - 10:15 AM
Coffee Break
In-person
9:45 AM - 1:15 PM
In-Person and Virtual Poster Session 3
Live
In-person presenters will be with their posters from 12:15 – 1:15PM.
9:45 AM - 7:00 PM
Poster Viewing & Exhibit Hall Open
In-person & Virtual
In-person/Virtual
10:15 AM - 11:15 AM
Podium Session 4
Machine Learning in HEOR
In-person
Moderator
William Padula, PhD, MS, MSc
University of Southern California, Los Angeles, CA, USA
William Padula, PhD is assistant professor of pharmaceutical & Health Economics at the University of Southern California School of Pharmacy, and a Fellow in the Leonard D. Schaeffer Center for Health Policy & Economics. His research interests include medical cost-effectiveness analysis and applications of machine learning to health economics and outcomes research. He was the 2021 recipient of ISPOR’s Bernie O’Brien New Investigator Award, and Is an Associate Editor for Value in Health.
P24: Predictors of Major Adverse Cardiovascular Events Among Type 2 Diabetes Mellitus Patients: A Machine Learning Time-to-Event Analysis
11:00AM - 11:15AM
Icten Z 1 , Friedman M2 , Menzin J1 1 Panalgo, Boston, MA, USA, 2 Panalgo LLC, Boston, MA, USA
OBJECTIVES:
Cardiovascular disease (CVD) is the leading cause of mortality in type 2 diabetes mellitus (T2DM). Understanding the predictors of major adverse cardiovascular events (MACE) may help improve treatment for these patients. This study sought to identify predictors of MACE using machine learning (ML). METHODS:
Patients ≥50 years of age with ≥1 T2DM diagnosis were identified using the Optum
® Integrated Electronic Health Records and Claims database between 01/01/2008 and 12/31/2020. The index date was assigned based on a randomly selected T2DM encounter, allowing for a 12-month baseline. Patients with prior CVD events were excluded. MACE was comprised of an inpatient or emergency department visit with myocardial infarction or stroke or CVD-related death. Patients were followed until the earliest of a MACE event, non-CVD death, end of enrollment or data availability. Baseline features included demographics, comorbidities, medication, procedure and healthcare resource utilization measures with and without selected literature-derived labs/vitals. Three-fold cross validation was used to tune and evaluate regularized Cox proportional hazards regression (RegCox), random survival forest and gradient boosting survival models. The best model was selected using the C-index.
RESULTS:
We identified 113,550 patients (mean age=64.0; females=55.8%) followed for a median (IQR) duration of 627 (293-1,209) days, with 3,854 (3.4%) of patients experiencing the MACE event. The best performing model was the RegCox with labs/vitals (C-index=73.7%) although exclusion of labs/vitals had little effect on model performance (C-index=73.1%). Features associated with increasing MACE risk included older age, higher Charlson score, systolic blood pressure, total cholesterol, number of comorbidities, lower HDL, along with use of furosemide and insulin glargine. Commercial insurance, being from the West, being female and having never smoked were associated with decreasing risk.
CONCLUSIONS:
Our study identified several key predictors of MACE among T2DM patients. These findings can support implementation of strategies to prevent CVD events among these patients.
P23: Machine Learning Approaches to Evaluate Treatment Switching in Patients with Multiple Sclerosis: Analyses of Electronic Medical Records
10:45AM - 11:00AM
Li J 1 , Huang Y2 , Aparasu RR1 1 University of Houston, College of Pharmacy, Houston, TX, USA, 2 University of Houston, College of Pharmacy, houston, TX, USA
OBJECTIVES: Disease-Modifying Agents (DMA) are often switched to another agent for effectiveness and safety considerations in Multiple Sclerosis (MS). This study developed and compared the prediction models for DMA switching among MS patients using machine learning (ML) algorithms.
METHODS: This was a retrospective longitudinal study using the TriNetX data from a federated electronic medical records network. Adults with ≥1 DMA and ≥1 MS diagnosis were identified (September 2010-May 2017), and the earliest DMA date was assigned as the index date. Patients were required to have ≥1 outpatient visit and ≥1 prescription in 12 months pre- and 24 months post-index. Switching was defined as receiving different DMA prescription than the index prescription during the follow-up. Logistic Regression (LR), Least Absolute Shrinkage and Selection Operator regression (LASSO), Random Forests (RF), and Extreme Gradient Boosting (XGBoost) were used to develop prediction models with 72 baseline variables. Models were trained using 70% of the randomly split data with the up-sampling method. Models' performance were evaluated using the Area Under the Curves (AUC), accuracy, recall, and F-1 score.
RESULTS: This study identified 7,258 eligible MS patients with ≥1 DMA, and 16.0% of them switched to another DMA within 2 years. The RF model achieved the best model performance with an AUC of 0.65 (accuracy 61%, recall 60%, and F1 score 72%). Other models were comparable: XGBoost model AUC of 0.63 (accuracy 63%, recall 56%, and F1 score 34%), the LR model AUC of 0.63 (accuracy 63%, recall 61%, and F1 score 72%), and the LASSO model AUC of 0.63 (accuracy 61%, recall 57%, and F1 score 33%).
CONCLUSIONS: The RF model provided the best performance for predicting the treatment switch in MS patients based on most performance metrics. More work is needed to understand the role of ML approaches in optimal treatment selection to provide individualized care.
P22: Electronic Medical Record Risk Modeling of Heart Failure Among Patients with Type 2 Diabetes
10:30AM - 10:45AM
Hong D 1 , Fort D2 , Shi L1 , Price-Haywood E2 1 Tulane University School of Public Health and Tropical Medicine, New Orleans, LA, USA, 2 Ochsner Health, New Orleans, LA, USA
OBJECTIVES: To optimize population health management tool, health systems are interested to locally develop risk prediction models for heart failure (HF) from their electronic health records. Further, machine learning methods can be used to transform general predictive models into the locally tailored models that are better suited to local populations. This study aimed to compare the performance of the tailored model to the two HF risk equations: the Risk Equations for Complications Of type 2 Diabetes (RECODe) risk equation and the Building, Relating, Assessing, and Validating Outcomes (BRAVO) risk equation.
METHODS: We conducted a retrospective study within Ochsner Health System (Louisiana’s largest integrated delivery health system, n=6,245, 2013-2017). The patients were required to have two outpatient diagnoses of T2DM recorded in separate months or a diagnosis recorded during an inpatient encounter. The baseline clinical data were limited to 180 days before the index T2DM diagnosis. The outcome was defined as at least one hospitalization with ICD-9/10-CM codes diagnoses of HF (402.11, 402.01, 402.91, 428, I50) after index T2DM. The Ochsner HF risk model employed Cox proportional hazards models, followed by LASSO regression to select predictor variables from demographic characteristics, clinical variables, medications, and biomarkers. Model discrimination was used to compare the two HF risk equations: RECODe and BRAVO risk equations.
RESULTS: Among the statistically significant factors in the Ochsner (n=11), RECODe (n=14), and BRAVO (n=9), age, blood pressure, and HbA1c were included across all three risk equations. The Ochsner heart failure risk equation had high internal discrimination (C-statistics 0.858, 95% confidence interval (CI): 0.840-0.877). The Ochsner HF risk equation had better discrimination than BRAVO (C-statistics 0.743, 95% CI 0.715-0.772) and RECODe (C-statistics 0.525, 95% CI: 0.490-0.560).
CONCLUSIONS: While the BRAVO risk equation required the fewest variables, a tailored risk equation further improved risk stratification of HF for patients with T2DM.
P21: A Scoping Review of the Use of Machine Learning in Health Economics and Outcomes Research: Part 1 - Data from Wearable Devices
10:15AM - 10:30AM
Lee W 1 , Schwartz N2 , Bansal A2 , Khor S2 , Hammarlund N1 , Basu A2 , Devine B2 1 University of Washington, SEATTLE, WA, USA, 2 University of Washington, Seattle, WA, USA
OBJECTIVES: With the emerging use of machine learning (ML) techniques, there has been particular interest in utilizing data from wearable devices for health economics and outcomes research (HEOR). We aimed to understand the emerging patterns of how ML has been applied to data collected from wearables in HEOR.
METHODS: We identified studies published in PubMed between January 2016 through March 2021. Studies that included at least one HEOR-related MeSH term, applied an ML technique and used wearable data were eligible for inclusion. Two reviewers abstracted information on ML application types, data on which ML was applied, and specific ML methods used. Extracted data were analyzed using descriptive analyses.
RESULTS: A total of 148 studies were identified from PubMed, among which 33 studies met the inclusion criteria. Between 2016-2021, there has been an increase over time in the number of ML studies using wearable data. ML has been more frequently used for monitoring events in real time (79%) than to predict future events (21%). There has been a wide range of outcomes examined, ranging from general physical or mental health (45%) to more disease-specific outcomes such as disease incidence (18%) and disease progression (15%). ML has also been used for treatment-related outcomes such as treatment adherence (9%), treatment outcomes (9%), and healthcare resource utilization (3%). Data for ML models were more often derived from wearable devices with specific medical purposes (58%) than those without (42%). Studies have used tree-based methods (e.g., random forests and boosting) most frequently (25%).
CONCLUSIONS: There has been a wide range of applications of ML to wearable data, from predicting general health outcomes to disease- or treatment-specific health outcomes. Both medical and non-medical wearable devices have been used as a data source, showing the potential for providing rich data for ML studies in HEOR.
Concurrent Breakout Session 4
Engaging Black Americans in Outcomes Research Studies Amidst a Challenging COVID Operating Environment: Experience From an Ongoing Vaccine Hesitancy Study in Philadelphia
In-person
Level: Intermediate
This session will discuss the impacts of the ongoing pandemic on engaging Black Americans in outcomes research. An ongoing study will be used as a platform to illustrate lessons learned; this study is specifically aimed at testing a vaccine hesitancy intervention delivered to older Black Philadelphians. Tarlan Namvar will moderate the session, starting off by highlighting the pandemic’s impacts on outcomes study recruitment with focus on minority populations and briefly introducing the Motivating Older Adults to Trust Vaccines and Their Effects (MOTIVATE) study as a case example for discussion (15 minutes); Katherine Prioli will discuss methodological considerations posed by the pandemic in terms of virtual deployment of study consent, data collection, and associated Institutional Review Board implications (15 minutes); and Keyerra Charles will discuss community outreach strategies used to engage the older Black population in outcomes research through senior centers and other community venues (e.g., churches and residential facilities) involved in the MOTIVATE study as well as previous studies. She will close by sharing insights on how to build trust in outcomes research among potential participants, which is essential to their participation as well as ultimately developing the scientific evidence needed to inform healthcare decisions and improve health equity (20 minutes). In the remaining 15 minutes, the audience will be encouraged to share their experiences with a goal of identifying a short list of best practices. Polling will be used to ascertain the audience’s current involvement in the topic as well as to identify the major recruitment challenges experienced by participants in the COVID era. Academia and industry researchers, health technology assessment experts, and patient advocates who are involved in designing, conducting, and/or evaluating outcomes research studies of older Black Americans will benefit from attending this session.
Panelists
Keyerra Charles, MPH, CHES
Center in the Park Senior Center, Philadelphia, PA, USA
Roland Lucas
Center in the Park Senior Center, Philadelphia, PA, USA
Katherine Prioli, MS
Rutgers University, Piscataway, NJ, USA
Discussion Leaders
Tarlan Namvar, PharmD
Rutgers University, Piscataway, NJ, USA
A highly motivated PharmD by training. Currently, working as a Health Economics and Outcome Research fellow at HOPE program/Rutgers University affiliated with Novo Nordisk Inc. Collaborating with cross-functional teams in strategic planning, and demonstrating value propositions through pipeline, launch, and in line products. Looking forward to meeting with HEOR professionals and learn more about their expertise.
Could Gene Therapy Value-Based Purchasing Become Common Thanks to the New CMS Rule on Medicaid Best Price?
In-person
Level: Intermediate
PURPOSE: Explore the use of alternative payment approaches managing financial risk associated with curative therapies, explain how a 2021 CMS rule could lead to widespread adoption of Value-Based Purchasing Arrangements (VBPs), detail remaining challenges, and discuss how those challenges can be overcome by a combination of clever implementation and eventually, legislative enhancements to the Medicaid Drug Rebate Program.
DESCRIPTION: Durable, potentially curative cell and gene therapies with short-term treatment regimens promise to transform care for some conditions. However, by their very nature, they create significant financial risk from their up-front costs and uncertain long-term efficacy. Precision financing approaches, such as milestone contracts, warranties, and annuities, are a key tool to address this challenge. However, these financing tools present both challenges and opportunities. More importantly, understanding which precision tools to use when is critical for payers looking to manage the financial risk associated with these transformative therapies.
One of the biggest challenges is the 1990 Medicaid “best price” (MBP) rule, which ensures that the Medicaid program receives discounts at least as large as commercial payers. Payment approaches have evolved over the years and this rule now hinders the adoption of these innovative new payment models to support patient access and payer sustainability. CMS is implementing a new rule for tiered-MBP reporting based on performance. It remains unclear to what extent the new rule will enable VBPs and other payment innovations. Even as tiered-MBP enable more aggressive risk sharing for orphan therapies other challenges remain. Implementation mechanics remain unclear, burdens on states to track outcomes may curtail state participation and some issues such as unit pricing remain unaddressed. This panel will bring both quantitative analysis and practical experience perspectives on the issues. Audience will be polled on most important challenges and solutions at the beginning and end of the workshop with time for Q&A.
Discussion Leaders
Mark Trusheim, MS, BS
Massachusetts Institute of Technology, Cambridge, MA, USA
Mark Trusheim, MS, BS, Strategic Director, NEWDIGS and Visiting Scientist MIT, Boston, MA
Mark Trusheim is Strategic Director, MIT NEWDIGS where he also co-leads the Financing and reimbursement of Cures in the US (FoCUS) Project; and a Visiting Scientist at the MIT Sloan School of Management. Through MIT he has also served as a Special Government Employee for the FDA’s Office of the Commissioner.
Mark’s research focuses on the economics of biomedical innovation, especially precision financing for patient access, precision medicine, adaptive pathways, platform trials and digital health advances.
Prior to MIT, his career spanned big data at Kenan Systems, marketing at Searle Pharmaceuticals, eHealth as Vice President of Monsanto Health Solutions, genomics as President of Cereon Genomics, and policy as the President of the Massachusetts Biotechnology Council.
He holds degrees in Chemistry from Stanford University and Management from MIT.
Discussants
Michael Ciarametaro, MBA
National Pharmaceutical Council, Washington, DC, USA
Michael Ciarametaro serves as the National Pharmaceutical Council's vice president of research. In this position, Mr. Ciarametaro conducts health policy research focused on addressing rising health spending and better aligning stakeholder incentives with value.
Mr. Ciarametaro has 20 years of experience creating value in the health care system through policy, research and leadership. His cross-disciplinary expertise spans multiple roles (policy researcher, strategic value and market access consultant, financial analyst, operations and process designer) and multiple areas of the health care ecosystem (health policy research organization, biopharmaceutical industry, private payers, public payers). For the past six-plus years, Mr. Ciarametaro has been focused on health policy research with an emphasis on addressing rising health spending and better aligning stakeholder incentives with value.
Mr. Ciarametaro holds a Bachelor of Science from the University of Virginia and received his MBA from George Mason University.
Dorothy Hoffman, MPP
Pfizer, New York, NY, USA
Dorothy Hoffman is External Engagement and Access Policy Lead in Pfizer’s Healthcare Innovation Center. Dorothy is responsible for leading Pfizer’s Enterprise strategy on access policy to enable value-based healthcare, digital health, and equitable access to health.
Dorothy has over 18 years of experience in the pharmaceutical and managed care industries. Previously, she was Vice President of Prescription Drug Policy at UnitedHealth Group. Dorothy’s team was responsible for implementing the company’s pharmacy care services transformation and growth initiative with federal and state health programs.
Dorothy started her career at Eli Lilly and Company where she held multiple roles of increasing impact and responsibility in Corporate HQ, the US affiliate and in European country and regional offices. Dorothy led Lilly’s US and International Public Policy teams and created the first-of-its-kind innovative payer partnership on value-based contracts.
Dorothy has a Masters in Public Policy from the University of Denver and a certificate in Design Thinking from Stanford. She lives in the NYC area with her husband and son.
Gail Ryan, PharmD
Point32Health, Harvard Pilgrim Health Care & Tufts Health Plan, Hopkinton, MA, USA
Gail Ryan is the Director of Pharmaceutical Transformation at Point32Health, a regional health services company serving over 2 million lives and an industry leader in innovation and value-based contracting. As a clinical pharmacist with 20 years of managed care experience, Gail’s practice has spanned the pharmacy benefit, medical drug policy, trend analysis, cost management initiatives and clinical innovation. In her current role and through partnerships with multi-stakeholders, she is driving equitable access to high-quality, transformative therapies while addressing emerging trends, challenges, and financial risk.
Are We Patient and Care-Partner Centric Enough in Early Alzheimer’s Disease Clinical Trials? The Potential Limitations of Using Legacy Clinical Outcome Assessments (COA) in the New Era of Alzheimer’s Disease Drug Development
Virtual
Level: Foundational
PURPOSE: To evaluate current clinical outcomes assessment (COA) measures and strategies in mild cognitive impairment (MCI) and early Alzheimer’s Disease (AD) and explore whether the use of legacy AD measures in the new era of early AD drug development is limiting our ability to identify patient relevant meaningful benefit.
DESCRIPTION: The recent paradigm shift in AD drug development, from later stage symptomatic AD to disease modifying monoclonal antibody (MAB) therapies in MCI and early AD, has received a lot of attention: good and bad. With a lot of focus on biomarkers and price, the patient relevance of these new treatments appears to have been lost, or lost in translation, as legacy definitions of meaningful change and difference have been applied to this new context.
Fit for purpose COAs in MCI and early AD clinical trials should measure patient relevant concepts, using appropriate item wording and response options, reported by an appropriately informed respondent, to be able to define and appropriately interpret patient benefit. This workshop will explore whether we can effectively evaluate the patient relevant meaningful benefit of new MAB therapies with the legacy AD COAs, typically being used in clinical trials. This workshop will include presentations to address: What do we know about what matters most to MCI and early AD patients and their care-partners? How well do current COA used in MCI and early AD trials address concepts that matter in MCI and early AD? How can we currently evaluate patient relevant meaningful benefit in MCI and early AD and what should we consider for the future? How can we leverage COA trial data to support patient, care-partner and payer relevant long-term treatment benefits. The content of this workshop will be of interest to clinicians, payers, regulators, study sponsors, COA scientists, health economists and patient associations in the AD field.
Discussion Leaders
John Harrison, BSc (Hons), PhD, PhD
Vrije Universiteit Amsterdam, Amsterdam, Netherlands
In brief: John is an acknowledged cognition expert whose principal professional interest is in helping people understand, maintain and enhance their cognitive skills.
Professor John Harrison is an expert psychologist with a special interest in cognition. John is Principal Consultant at Metis Cognition, a psychology practice established to advise with the selection and successful integration of cognitive testing into therapeutic development programs. He is Associate Professor with the AUmc Alzheimer Center and Visiting Professor at King’s College London. He holds Chartered Psychologist status and has authored/co-authored more than 80 books and scientific articles.
Discussants
Sarah Acaster, MSc
Acaster Lloyd Consulting Ltd, London, LON, United Kingdom
William Herring, PhD
RTI Health Solutions, Research Triangle Park, NC, USA
Will Herring, PhD, is an Executive Director in the Health Economics group at RTI Health Solutions with expertise in cost-effectiveness and value-based pricing modeling for chronic progressive diseases and for conditions affecting older adults. He has presented research on economic modeling and value assessment challenges for Alzheimer’s disease in multiple international settings and is a member of the steering committee for the International Pharmaco-Economic Collaboration for Alzheimer’s Disease (IPECAD) Modelling Group. He is Affiliated to Research at the Karolinska Institutet and is a member of the ISPOR Open-Source Modeling Special Interest Group.
Russ Paulsen, MA
UsAgainstAlzheimer’s, Washington, DC, USA
Russ Paulsen is a nationally recognized nonprofit leader and innovator. Programs and teams with his leadership have saved hundreds of lives, helped rebuild and heal communities across the nation, and brought cutting-edge technology to public health and human services.
Since he joined UsAgainstAlzheimer’s, the team built a web and telephone platform that uses cutting-edge technology to deliver information tailored to where a specific user is on their Alzheimer’s journey, received its first-ever funding from CDC, and assembled a 100-organization coalition on Alzheimer’s prevention. All functions and programs report to Russ.
His team at the American Red Cross built a campaign to reduce deaths from home fires that has saved more than 800 lives since its launch and made 900,000 high-risk homes safer through smoke alarms and fire escape plans. More than 1.6 million kids have learned about fire safety through classroom presentations and an award-winning videogame his team designed.
And in the aftermath of Hurricane Katrina, in addition to rebuilding thousands of homes along the Gulf Coast, Russ’s team helped rebuild the mental health system of coastal Louisiana and Mississippi through targeted grants and a system of treatment reimbursement that created an economic incentive for small providers to return.
Russ’s career in helping began with the 1989 earthquake in the San Francisco Bay Area, when he felt compelled to drop what he was doing and volunteer.
He and his family live in Bethesda, Maryland.
Conceptual Issues in the Valuation of Health in Children
Virtual
Level: Intermediate
PURPOSE
: To clarify key conceptual issues in the valuation of health in children through a participatory workshop, and so enable ISPOR delegates to develop their own views on how the health of children should be valued.
DESCRIPTION
: The valuation of health in children poses conceptual issues that can be organised into two categories: (1) those related to generating utility values; and (2) those related to how QALYs should be valued depending on the age of the recipient. Such issues are faced by researchers seeking to design valuation studies and by policymakers seeking to make methodological recommendations. Discussion leader SM will summarise ongoing debates about how the health of children should be valued, explain how clarifying the conceptual issues will enable more informed discussion and decision-making, and field a live poll to gauge participants' understanding of these issues. [
10 min ] Discussant EL will explore whose preferences we should elicit to value child health states, and from which perspective those preferences should be elicited. He will describe the factors that might influence choices of valuation source and perspective, and seek the audience’s views on which approaches are feasible and appropriate. [
15 min ] Discussant TP will show how empirical research can be used to investigate whether a QALY provided to a child is valued differently to a QALY provided to an adult. She will administer a live person-trade-off preference elicitation exercise seeking to understand views on priority-setting, and will comment on how the audience’s preferences compare with those reported in the existing empirical literature. [
20 min ] Discussant KS will provide the perspective of a policymaker, reflect on the results from the audience interactive elements and propose an agenda for further research. [
15 min ]
Discussion Leaders
Simon McNamara, PhD
BresMed, Sheffield, YOR, United Kingdom
Director of Consulting at Lumanity (previously known as BresMed) and Honorary Research Fellow at the University of Sheffield. PhD in Health Economics. Co-investigator on three EuroQol funded HRQoL-focused projects. Thirteen years experience working in HEOR.
Discussants
Ernest Law, PharmD, PhD
Pfizer, New York, NY, USA
Ernest is a Director of Health Economics & Outcomes Research on the Global Immunology & Inflammation Patient & Health Impact team at Pfizer. His research interests include health preferences, patient-reported outcomes, patient-centered and comparative effectiveness research, and shared decision-making. Ernest is involved in conducting early- and late-phase clinical trials, real-world studies, and economic evaluations, currently focusing on autoimmune diseases in both adult and adolescent populations. He is a member of the Euroqol Group, an international network of scientists dedicated to the science of health measurement, valuation, and evolving the EQ-5D family of instruments. Ernest holds a Doctor of Pharmacy (PharmD) from the University of British Columbia and obtained his Ph.D. from the University of Illinois at Chicago.
Tessa Peasgood, PhD
University of Melbourne, Melbourne, VIC, Australia
Tessa Peasgood is a Senior Lecture in Health Economics at the School of Population and Global Health, University of Melbourne. She specialises in the measurement and valuation of health and wellbeing.
Koonal Shah, PhD
National Institute for Health and Care Excellence (NICE), London, LON, United Kingdom
Member of NICE's Science Policy and Research team. Actively involved in develop NICE's methodological position on the valuation of health in children. Honorary Research Fellow at the University of Sheffield.
Lessons Learned From Health Technology Assessment Pilot Projects to Assess Real-World Evidence’s Usefulness in Reassessments of Effectiveness and Value
In-person & Virtual
Level: Intermediate
PURPOSE: To identify and understand key learnings and challenges from two pilot projects conducted by Health Technology Assessment (HTA) agencies using real-world evidence to update prior health technology assessments. To determine best practices for RWE use in HTA assessments based on demonstration projects and audience feedback
DESCRIPTION: HTA agencies traditionally use real-world evidence (RWE) as economic modeling inputs for one-time HTA assessment after regulatory approval. More recently, HTA agencies are considerings shifting toward assessing drugs at multiple timepoints throughout the product’s lifecycle. To better understand the role of RWE in serial HTA assessments, ICER and NICE conducted pilot projects to update prior HTA assessments. Key learnings from these pilot projects will inform future RWE guidances and best practices.
An overview of the current RWE guidances for HTA assessments and an introduction to the pilot projects will be provided (Ashley Jaksa). ICER and NICE representatives will provide in-depth examination of the pilot projects and key learnings from each study. Speakers will also explore tenets of RWE best practices and guidances based on the pilot studies (Jon Campbell and Seamus Kent). The industry representative will share experiences with conducting RWE within their institution and discuss collaborative approaches to providing this evidence to payers for future assessments (Gregory Daniel). The speakers will share insights and lessons learned from each demonstration project that will eventually inform institutional best practices and future guidances on RWE use in HTAs. The speakers will solicit public comments and immediate feedback by conducting real-time polling to key learnings. The audience is expected to participate and provide their perspective and experience regarding RWE best practices.
Discussion Leaders
Ashley Jaksa, MPH
Aetion, Inc., Boston, MA, USA
Discussants
Jon Campbell, PhD
Institute for Clinical and Economic Review, Hingham, MA, USA
Jon (Jonathan D.) Campbell is Senior Vice President for Health Economics at the Institute for Clinical and Economic Review (ICER). Jon joined ICER’s senior management team as a leader in value assessment methods and application; he oversees the growth of ICER’s health economics efforts and leads the continued innovation of ICER’s value assessment methodology. Additionally, Jon continues to build bridges within the global health economics community through engagement with ICER’s Health Economic Council and through leadership and participation in health technology assessment societies and agencies. Further, Jon seeks creative value assessment solutions for ICER’s diverse stakeholders by prioritizing improved outcomes for patients.
Jon is an affiliate faculty member at Tufts University School of Medicine in the Center for the Evaluation of Value and Risk in Health. Jon is an author of over 100 peer-reviewed manuscripts in the field of value assessment as well as an author on many ICER assessments. Jon is a former ICER Health Economics Council member and five-year external collaborator through his former role as Associate Professor with tenure at the University of Colorado Anschutz Medical Campus. He holds graduate training degrees in pharmaceutical outcomes research (PhD) and biostatistics (MS) from the University of Washington. He graduated with a BA in mathematics and chemistry from St. Olaf College.
Jon also enjoys playing most racket-related sports. He grew up playing pickleball in the Seattle area and is seeking opportunities to spread the pickleball love in the Boston area.
Gregory Daniel, PHD, MPH
Eli Lilly, Washington DC, DC, USA
Seamus Kent, PhD
National Institute for Health and Care Excellence (NICE), London, LON, United Kingdom
Seamus is a Senior Adviser in Data & Analytics at NICE. He is leading the development of a framework for the use of data and analytics in the development of NICE guidance.
Applying Advanced Real-World Evidence to HEOR: What, Why, and How?
In-person
Level: Intermediate
PURPOSE: Panelists will discuss the importance of high-validity, real-world evidence and its ever-increasing role in the decision-making process for payers, regulators, and providers.
DESCRIPTION: In an initial 15-minute presentation, each panelist will take 5 minutes to share their institutions’ experience with advanced real-world evidence. In a 25-minute discussion, panel members will respond to questions posed by moderator Dan Riskin, CEO of Verantos and Adjunct Professor of Surgery and Biomedical Informatics Research at Stanford University. Kara Kilpatrick, Director of Observational Research at Amgen, will discuss Amgen’s early results in applying high-validity real-world evidence to asthma. Dr. Arthur Garan, Director of Advanced Heart Failure and Transplant Cardiology, Cardiovascular Disease at Harvard’s Beth Israel Deaconess Medical Center, will discuss their engagement in real-world evidence and how it’s influencing care delivery. Finally, Dr. Steve Kymes, Lundbecks’ Director of Health Economics and Outcomes Research, will discuss advanced real-world evidence efforts in migraine and how advanced techniques are implemented at the time of product launch. We invite attendees to converse with the moderator and panelists in a 15-minute open Q+A following this discussion.
Discussion Leaders
Dan Riskin, MD, MBA, FACS
Verantos, Menlo Park, CA, USA
Dan Riskin is Founder and Chief Executive Officer of Verantos and previously held positions as Founder and CEO of Health Fidelity and Special Projects Consultant at Apple. He is Adjunct Professor of Surgery and Adjunct Professor of Biomedical Informatics Research at Stanford University.
Dan is an expert in healthcare artificial intelligence and successful serial entrepreneur. Products he has developed and commercialized influence the care of millions of patients annually. He has spoken on medical technology at FDA, CMS, NASA, DARPA, NSF, and NIH. His contributions in data-driven healthcare have been featured in Forbes, The Wall Street Journal, and other leading media. Dan served on the Obama Healthcare Policy Committee and testified before Congress on the 21st Century Cures Initiative.
Dan’s medical credentials include a MD from Boston University, residency in surgery at UCLA, and fellowship in critical care and acute care surgery at Stanford University. He is board-certified in four specialties, including surgery, critical care, palliative care, and clinical informatics. His business training includes a MBA with a focus in bioinformatics from the Massachusetts Institute of Technology.
Discussants
Steven Kymes, PhD, MHA
Lundbeck LLC, Deerfield, IL, USA
Keri Monda, PhD
Amgen Inc, Thousand Oaks, CA, USA
Keri Monda is Executive Director of Observational Research in the Center for Observational Research at Amgen, Inc. Prior to her time at Amgen, Dr. Monda was on faculty in the Department of Epidemiology at the University of North Carolina at Chapel Hill, where she also did her pre- and post-doctoral training in cardiovascular and genetic epidemiology.
Arthur Garan Reshad, MD, MS
Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA, USA
Arthur G. Reshad earned his M.D. from Columbia University and completed his residency with New York Presbyterian Hospital. Arthur Garan is affiliated with Beth Israel Deaconess Medical Center, an Associate Professor at Harvard Medical School, and specializes in Advanced Heart Failure and Transplant Cardiology and Cardiovascular Disease.
Severity Shortfall: Graceful or Awkward? Contextual or Continuous?
Virtual
ISSUE: Evidence indicates public preference to prioritise health gains for severe health problems. The panel will debate how to operationalise this. Should it be an automatic adjustment or one contextual factor for deliberation? Do we have an agreed, measurable definition of severity that enables us to do this? How do we address opportunity cost? Perspectives will come from a leading HTA body, an academic working on severity, and a patient advocate representing severe diseases with other key attributes that matter.
OVERVIEW: The panelists perspective will be as follows:
Adrian Towse will moderate the session and set out the approaches being taken by different HTA bodies and alternative measures of severity in the literature. He will note a trend to incorporating severity adjustments directly into thresholds.
Steve Pearson (ICER) will provide an HTA approach, arguing that severity is relevant to value but that ICER’s current “contextual consideration” is appropriate, given disagreement over the most appropriate conceptualization of severity, the importance of other factors that impact value, and of the opportunity cost for health systems of higher prices.
Chuck Phelps will provide an academic perspective, arguing severity can be operationalised in continuous manner, as described in the GRACE framework. A continuous approach provides transparency and consistency.
Durhane Wong-Rieger will provide a patient perspective, arguing that “severity” should be at least ONE modifier but that begs the question of how to measure severity across conditions in a way that is acceptable. A highly contextualized approach is necessary if no one solution that fits all diseases, treatments, and payer scenarios. Following a brief introduction to the issue by the moderator (4 mins), each panellist will speak for 12 minutes and we will allow 20 minutes for audience interaction.
Moderators
Adrian Towse, MA, MPhil
Office of Health Economics, London, United Kingdom
Professor Adrian Towse is director emeritus and senior research fellow of the Office of Health Economics in the UK. Adrian’s current research includes incentives for new drugs and vaccines to tackle Antimicrobial Resistance, the use of 'risk-sharing' arrangements between healthcare payers and pharmaceutical companies, including value-based pricing approaches; the economics of pharmacogenetics for healthcare payers and the pharmaceutical industry; economic issues that affect both R&D for and access to treatments for diseases prevalent in the developing world; the economics of medical negligence; and measuring productivity in healthcare.
A visiting professor at the London School of Economics and a senior researcher at the Nuffield Department of Population Health at the University of Oxford, Adrian also has been a visiting professor at the University of York. For ten years, he served as the non-executive director of the Oxford Radcliffe Hospitals NHS Trust, one of the UK’s largest hospitals. Adrian was president of ISPOR, for the 2014-15 term.
Adrian joined the OHE in 1993 and served as director for 25 years. He holds an MA (Hons) in Politics, Philosophy and Economics from Keble College, Oxford; an MPhil in Management Studies from Nuffield College, Oxford, and the Oxford Centre for Management Studies; and is a member of the Chartered Institute of Management Accountants.
Panelists
Steven D. Pearson, MD, MSc
Institute for Clinical and Economic Review, Boston, MA, USA
Steven D. Pearson, MD, MSc is the Founder and President of the Institute for Clinical and Economic Review (ICER), an independent non-profit organization that evaluates the evidence on the value of medical tests, treatments, and delivery system innovations to encourage collaborative efforts to improve patient care and control costs. Dr. Pearson is also a Lecturer in the Department of Population Medicine at Harvard Medical School. He received his medical degree from UCSF, completed an internal medicine residency and research fellowship at Brigham and Women’s Hospital, and obtained a Master of Science Degree in Health Policy and Management at the Harvard School of Public Health.
Charles Phelps, MBA, PhD
University of Rochester, Rochester, NY, USA
Charles E Phelps, PhD, a health economist, has developed key models of cost-effectiveness analysis that provide the intellectual foundations for its practice. He was given the Victor R Fuchs Award for Lifetime Achievement in the Field of Health Economics in 2019, and has been a member of the National Academy of Medicine since 1991. His leading textbook, Health Economics is now in its 6th Edition. His recent interests have expanded to the use of multi-criteria decision analysis (MCDA), particularly in its proper use when the “decision-maker” is a group.
Durhane Wong-Rieger, MA PhD
Canadian Organization for Rare Disorders, Toronto, ON, Canada
Durhane Wong Rieger, PhD, is the President & CEO of the Canadian Organization for Rare Disorders (CORD). She is also the President & CEO of the Institute for Optimizing Health Outcomes (Canada), Chair of the Consumer Advocare Network and Chair of Canadian Heart Patient Alliance.
10:15 AM - 11:30 AM
Spotlight Session
Emerging Methods in Real World Analyses involving Social Determinants of Health
In-person & Virtual
Social determinants of health (SDOH) are increasingly being recognized as important contributors to healthcare costs and outcomes that must be recognized and addressed. Drawing upon the public health discipline, the first speaker will present a conceptual framework of SDOH constructs and data sources commonly used to analyze them. Speaker 2 will discuss methods currently being applied to understanding and accounting for the relationship between SDOH and health outcomes in real world data. The speakers will be followed by a discussant who will reflect upon lessons learned and explore future directions for furthering HEOR methods in this area.
Moderators
Newell McElwee, PharmD, MSPH
Boehringer Ingelheim Pharmaceuticals, Inc., Ridgefield, CT, USA
Newell is Vice President, Health Economics and Outcomes Research at Boehringer Ingelheim Pharmaceuticals, Inc. He is a recognized leader in HEOR with 30 years experience in the pharmaceutical industry. Prior to coming to Boehringer Ingelheim, Newell led outcomes research groups at Pfizer and Merck. He has been actively engaged with a number of outcomes research related organizations, is a past ISPOR Board member and currently chairs the ISPOR Health Science Policy Council.
Newell received his PharmD from Mercer University and his MSPH (epidemiology) from the University of Utah. He completed a clinical pharmacy residency at Osteopathic Medical Center of Texas and a post-doctoral fellowship in clinical pharmacology and toxicology at the University of Utah Medical Center
Panelists
Karen Hacker, MD, MPH
CDC, Atlanta , GA, USA
Rachel Harrington, PhD
The National Committee for Quality Assurance (NCQA), Washington, DC, USA
Rachel Harrington is a Research Scientist at the National Committee for Quality Assurance, where she leads a team focused on evolving approaches to health equity in quality measurement. This includes the Equity in HEDIS initiative, and a project to develop an equity-focused quality measurement framework for applications in Medicaid. Rachel also serves as the scientific lead for measures of medication use in older adults and risk-adjusted measures of resource utilization. She represents NCQA on a number of external panels, including the National Quality Forum Risk Adjustment Guidance technical expert panel. Before joining NCQA, Dr. Harrington was a fellow at the University of Illinois at Chicago Institute for Health Research and Policy, where her research focused on healthcare quality among complex pediatric populations. Prior to that, she spent over 10 years in the pharmaceutical industry, including roles in health economics and outcomes research. Dr. Harrington holds a Ph.D. in Pharmacy Systems, Outcomes and Policy from the University of Illinois at Chicago.
James Love-Koh, BA, PGDip, MSc, PhD
National Institute for Health and Care Excellence (NICE), Manchester, LAN, United Kingdom
11:15 AM - 1:15 PM
Lunch
In-person
11:45 AM - 12:45 PM
Discussion Groups
New this Year – Discussion Groups! Discussion Groups are facilitated conversations between conference attendees and select conference speakers. Held in the new, dynamic Discussion Lounge in the ISPOR Exhibit Hall, these discussions are intended to be highly interactive, collaborative, and promote the exchange of ideas in a peer-to-peer setting. Health Technology Assessment Discussion Group
In-person
Moderator
Ashley Jaksa, MPH
Aetion, Inc., Boston, MA, USA
Educational Symposium
Can Pharmaceutical Pricing Move Beyond Cost/QALY for Value Consideration?
In-person & Virtual
Current economic evaluation studies may underestimate the value of innovative cell and gene therapies in rare diseases, impacting on Health Technology Assessment (HTA) frameworks, market access and pricing decisions. This symposium will explore additional add-on benefits of gene therapies apart from quality-adjusted life years (QALYs) that may be considered, how these can be quantified, and their role in influencing economic modelling. Using case studies of value assessment frameworks, panelists will discuss the value of pricing factors beyond cost and QALY considerations for gene therapies.
Sponsor
Novartis Gene Therapies
Moderators
Omar Dabbous, MD, MPH
Novartis Gene Therapies, Inc., Bannockburn, IL, USA
Speakers
Michael Drummond, MCom, DPhil
Centre for Health Economics, University of York, York, United Kingdom
Michael Drummond, BSc, MCom, DPhil is professor of Health Economics and former Director of the Centre for Health Economics at the University of York. His particular field of interest is in the economic evaluation of health care treatments and programmes. He has undertaken evaluations in a wide range of medical fields including care of the elderly, neonatal intensive care, immunization programmes, services for people with AIDS, eye health care and pharmaceuticals. He is the author of two major textbooks and more than 700 scientific papers, and has acted as a consultant to the World Health Organization and the European Union. He has been President of the International Society of Technology Assessment in Health Care, and the International Society for Pharmacoeconomics and Outcomes Research. In October 2010 he was made a member of the National Academy of Medicine in the USA. He has advised several governments on the assessment of health technologies and chaired one of the Guideline Review Panels for the National Institute for Health and Care Excellence (NICE) in the UK. He is currently Co-Editor-in-Chief of Value in Health and has been awarded 3 honorary doctorates, from City University (London), Erasmus University (Rotterdam) and the University of Lisbon.
Sean Sullivan, BScPharm, PhD
University of Washington, Seattle, WA, USA
ISPOR Forums
The Evolution of Biosimilar Markets: Key Elements for Long-Term Sustainability of the Healthcare Ecosystem
In-person
Biosimilars are biological agents developed to be highly similar and therapeutically equivalent to originator biologics. Their main development driver is their potential to bring market competition. By fostering price competition, biosimilars promote quality care while managing spending. This is fundamental to support healthcare ecosystem’s long-term sustainability. However, the literature indicates that the level of competition achieved following biosimilars market availability varies by country and by molecule, and that biosimilars contribution to attaining price reductions and extended patients’ access to treatments is also variable. This variability is indicative of the divergent implementation of biosimilar policies across health systems, and of the different capacity of these policies to support favorable environments for the market launch and adoption of biosimilars. There is a need to map policies concerning biosimilars and to identify elements of these policies that affect the long-term sustainability of off-patent biologic and biosimilar markets.
This Forum addresses topics of interest for the ISPOR Biosimilars Special Interest Group and will follow the structure of a panel. Cate Lockhart will introduce the session and provide the US perspective on elements that may undermine the long-term sustainability of off-patent biologic and biosimilar markets. Murray Aitken will share insights from IQVIA on building frameworks to evaluate sustainability risks. Jackie Vanderpuye-Orgle will discuss elements needed to reach consensus on long-term sustainability objectives. Anna Hyde will comment on these sustainability objectives from the patients’ advocate perspective. Each speaker (10 minutes allocated per presentation) will raise a question to be addressed by the audience via a real-time polling system. After the speakers’ presentations, Teresa Barcina will moderate a Q&A session that will be structured around the answers of the audience to the poll questions.
Moderators
Teresa Barcina, PharmD
KU Leuven, Leuven, VBR, Belgium
Teresa Barcina Lacosta is a PhD researcher in Pharmaceutical Sciences at KU Leuven. Her PhD project explores market dynamics and sustainability of off-patent biologics and biosimilars in Europe. This project is supported by the KU Leuven MABEL Fund, which is dedicated to the analysis of the market environment of biologics following exclusivity loss.
Speakers
Murray Aitken, MBA
IQVIA Institute for Human Data Science and the London School of Economics and Political Science, New York, NY, USA
Murray Aitken is a senior vice president of IQVIA and Executive Director of the IQVIA Institute for Human Data Science. The Institute undertakes independent research for publication, drawing upon the resources of IQVIA, and focuses on improving understanding of critical healthcare issues around the world, including the role of medicines in patient care, the disruptive impact of technology, productivity in research and development, and the value of information in improving decision-making.
In his role, Murray directs the research agenda and co-authors reports, while also engaging externally with a broad range of healthcare decision-makers in the public and private sectors. Reports by the Institute are widely cited by policy-makers, referenced in peer-reviewed research, and covered by the media.
Anna Hyde, MA
Arthritis Foundation, Silver Spring, MD, USA
Anna Hyde is the Vice President of Advocacy and Access at the Arthritis Foundation. She oversees both the federal and state legislative programs, in addition to grassroots engagement. Her focus is to raise the visibility of arthritis as a public health priority, build support for federal and state legislation that ensures access to affordable, high-quality health care, and enhance patient engagement in the policy-making process. Anna previously served as Senior Director of Advocacy and Access, managing the federal affairs portfolio and overseeing the state advocacy team.
Prior to joining the Arthritis Foundation in 2014, Anna worked as Senior Manager for Federal Affairs at the American Congress of Obstetricians and Gynecologists, where she managed a portfolio of issues including appropriations, physician workforce, and health IT. She began her health policy career as a Congressional Fellow for Energy and Commerce Committee members, where she drafted legislation and staffed Committee activities. Anna received a BA in History from Southern Methodist University, and taught junior high and high school history before moving to Washington D.C. in 2007 to pursue an MA in Political Science from American University.
Cate Lockhart, PharmD, PhD
Biologics and Biosimilars Collective Intelligence Consortium, Asheville, NC, USA
Cate Lockhart, MS, PharmD, PhD is the Executive Director of the Biologics and Biosimilars Collective Intelligence Consortium (BBCIC.org) where she is responsible for both the business and research programs of this large, multi-stakeholder research collaboration. Previously she was the HEOR expert at Omeros Corp in addition to part-time work with BBCIC as Research Team Coordinator. She also conducted several projects in health economics and outcomes research as an independent consultant. Prior to that, Dr. Lockhart was Associate Clinical Director at Strategic Pharmacy Innovations. She has done extensive research, medical writing and consulting in multiple disease states and therapeutic areas, producing clinical and economic reviews, pharmacoeconomic models and AMCP dossiers to support formulary decisions and value assessments of pharmaceutical products across a variety of therapeutic areas. Cate has a unique breadth of formal training and practical experience and knowledge in basic science, managed care pharmacy, clinical analyses and quantitative sciences including outcomes research, health economics and epidemiology. Cate has three undergraduate degrees: B.S. in Electrical Engineering, B.S. in Visual Communications, and B.F.A. in Theatre Arts. She completed three advanced degrees from the University of Washington in Seattle: a PharmD, an M.S. in Health Economics and Outcomes Research, and a Ph.D. in Pharmaceutical Sciences. She has a U.S. Patent, “Calibration Method and System for PET Scanners.”
Jacqueline Vanderpuye-Orgle, PhD
Parexel, Los Angeles, CA, USA
Dr Jackie Vanderpuye-Orgle is a Vice President and the Global Head of Advanced Analytics at Parexel. As a health economist, she has over 20 years of progressive experience in the application of econometric principles and statistical analysis to both clinical and real-world data. In her role at Parexel, Jackie is responsible for providing technical expertise in parametric modelling, meta-analysis, machine learning/AI, and other advanced methods. She has also held research and consulting roles at Amgen, Precision Health Economics, Endurance Reinsurance, Analysis Group, and the World Bank. Jackie has a PhD from Cornell University and is currently past-Chair of the ISPOR Biosimilars SIG.
ISPOR Task Force on Emerging Good Practice in Quantitative Benefit-Risk Assessment: Final Recommendations
In-person
Decisions throughout the medical product lifecycle are informed by benefit-risk assessment (BRA), including marketing authorization and surveillance, health technology assessment, and clinical decisions. All BRAs involve examination of the decision context, specification of benefits and risks for treatment options, and judgement about the acceptability of benefit-risk trade-offs within the decision context. In some cases, the benefit-risk balance of a product is unclear and quantitative BRA (qBRA) can add value and supplement a qualitative, descriptive BRA. Such analyses integrate trade-off preference data with evidence on product performances using an evaluation framework (e.g., multi-criteria decision analysis), to perform an overall assessment of individual benefit-risk profiles. The ISPOR Task Force on emerging good practices in qBRA developed guidance on how to conduct and report a scientifically rigorous qBRA.
In this Forum, we will present the Task Force’s final recommendations. To begin, we will briefly review the history of qBRA, including recent examples of use by regulators (Lackey – 15 mins). Then the task force will present our final recommendations for conducting and reporting qBRA in five steps: formulating the research question, developing a model, eliciting preferences, conducting analysis, and communicating results, together with a case study (Ho – 15 mins). Following this, we will describe how the qBRA is practically implemented in industry (Mauer – 15 mins). Through polling we will gather audience reactions to the recommendations and discuss future areas of research related to qBRA (Tervonen – 15 mins). Researchers conducting qBRA as well as decision-makers utilizing the results of qBRA will benefit from attending.
Moderators
Tommi Tervonen, PhD
Evidera, London, UK; University Medical Center Groningen, London, LON, United Kingdom
Tommi Tervonen is Director of Patient Preferences and a Senior Research Scientist with the Patient-Centered Research team at Evidera. His research focuses on the development of preference elicitation and benefit-risk assessment methods, as well as their application in the health domain.
Speakers
Martin Ho, MS
Google, San Francisco, CA, USA
Leila Lackey, D.Env
Food and Drug Administration, Rockville, MD, USA
Dr. Lackey is a decision analyst at FDA’s Center for Drug Evaluation and Research. With a background in public health, Dr. Lackey is part of the team that supports regulatory benefit-risk assessment and decision-making at the Center through the use of structured quantitative and qualitative techniques.
Jonathan Mauer, RPh, MBA
Pfizer, New York, NY, USA
Nutrition Economics – Are We Ready For A New Approach? Opportunities To Advance the Science
In-person
The Nutrition Economics Special Interest Group is dedicated to characterizing the economic and health outcomes of nutrition and advancing the scientific methodologies and approaches for studying nutrition economics more broadly. Over the past several years, the SIG has completed two collaborative papers: the first was a scoping review of MN terminology and regulations in Europe and the United States, which found extensive inconsistencies across countries in terminology used and a dearth of economic evaluations to inform the adoption of FSMP. The second paper took a broad look at coverage and reimbursement policies for FSMP across 14 countries in four continents. Despite the importance of medical nutrition (MN) for the management of malnutrition and nutrition-related disorders and conditions, little is known about regulatory policies governing reimbursement and coverage of Food for Special Medical Purposes (FSMP) across the world. A number of issues were identified, including (1) limited reimbursement in the outpatient and community settings; (2) variation in the conditions eligible for reimbursement across countries; (3) lack of health technology assessment to inform coverage and reimbursement decisions; and (4) a disconnect between clinical guidelines and coverage.
This Forum session will highlight the key findings from the most recent report, delve into the coverage and reimbursement issues identified in the review, and highlight opportunities for future collaborations related to the economics of MN and non-MN to improve societal health. The session will wrap up with a discussion about key SIG initiatives for the coming year.
Speaker
Amarsinh M Desai, PhD, MS, B.Pharm, D.Pharm
Nestle Health Science, Bridgewater, NJ, USA
Amarsinh Desai, PhD is an Health Economist and presently US Market Access Lead with Nestle Health Science. In his current role, he leads value demonstration, evidence generation and HEOR activities for Nestle medical nutrition products and programs.
Before joining Nestle, Dr. Desai was working as an Associate Health Economist at ICON plc, a clinical research organization, where he supported Global Health Technology Assessment – Health Economics, Reimbursement & Outcomes (GHTA – HERO) team with health economics projects.
He graduated with PhD in Health Outcomes from University of Cincinnati, Master of Science in Drug Regulatory Affairs, and has background in Pharmacy. His expertise and experience include conduct of observational studies with variety of data sources, health economic evaluations and evidence synthesis in wide variety of therapeutic areas.
Amarsinh Desai held positions such as President for International Society for Pharmacoeconomics & Outcomes Research (ISPOR) and Vice-President for International Society for Pharmacoepidemiology (ISPE) student chapters. He received several awards for distinguished services and finalist poster awards at International conferences and World Congress.
Karen Freijer, PhD
Erasmus University, Rotterdam, Netherlands
Tricia Johnson, PhD
Rush University, Oak Park, IL, USA
Tricia Johnson is Professor and Economist in the Department of Health Systems Management and co-leads the Health Equity Research Group at Rush University in Chicago, USA. She is one of the few human milk economists in the world, and her current research evaluates the short and long-term economic consequences of breastmilk and nutrition strategies for premature infants. She is Multiple Principal Investigator of a randomized controlled trial that tests an economic intervention aimed at reducing racial and ethnic gaps in the provision of mother’s own milk for very preterm infants in the neonatal intensive care unit. Dr. Johnson is Chair-Elect of ISPOR’s Nutrition Economics Special Interest Group.
Moreno Perugini, MS, MBA, BSc
Aimmune, Oak Park, IL, USA
Moreno is currently Senior Vice President Global Medical Affairs and HEOR at Aimmune Therapeutics, a Nestle Health Science Company. In this role, he leads a team of senior experts to increase medical education, KOL engagement, evidence generation and HEOR across the company therapeutic areas.
Prior to Nestle Health Science, Moreno was VP Health Technology Assessment for Sanofi - Aventis with the responsibility for assessing the clinical and economic evidence needed to successfully commercialize new technologies worldwide. Prior to his time with Sanofi, Moreno held roles of increasing responsibility across Evidence Generation, Market Access, and Early Commercialization in AbbVie, Lilly, Novartis and Pfizer.
Moreno’s interest are Health Policy, Decision Making, Modeling and Reimbursement across the different HealthCare systems.
Moreno holds an MBA from UCLA - Bocconi, a Master in Public Health from University Pompeu Fabra, a Master in Applied Mathematics from Roma 3 University. He is certified in Regulatory and Pharmaceutical Pricing.
12:15 PM - 1:15 PM
Poster Session 3 Poster Tours
New this Year – Poster Tours! ISPOR has curated collections of research posters for you within each of the poster sessions. Each tour will feature high impact abstracts within a specific topical area and will include a tour guide as well as the poster authors to share their work and engage in discussions with you. Visit the Learning Formats page for more information. Poster Tour: Infectious Disease (Non-Vaccine)
In-person
Posters featured in this tour:
CO75: Predictors of Value-Based Outcomes in Sepsis: An Analysis Using Electronic Health Record and Administrative Claims Data
EE238: Healthcare Costs of COVID-19 Versus Flu and Pneumonia - a Payer Perspective
EE211: Cost of Long COVID Following Severe Disease - a US Healthcare Database Analysis
EE260: Early Albumin Infusion May Reduce Intensive Care Unit (ICU) Cost in Cirrhotic Patients with Spontaneous Bacterial Peritonitis (SBP): A Cross-Sectional Study
EPH120: Comparing the Effectiveness and Cost-Effectiveness of SARS-COV-2 Screening Strategies Using Rapid Antigen Tests in a Residential College Campus
EPH98: A Systematic Review and Meta-Analysis for Risk Factor Profiles in Patients with Resistant Acinetobacter Baumannii Infection Relative to Control Patients
Poster Tour: Student Research Spotlight
In-person
Posters featured in this tour:
EE113: Comparing Partitioned Survival and State Transition Modeling Approaches: A Case Study of Osimertinib Versus Pemetrexed-Platinum
EE277: Cost-Effectiveness Analysis of National Smoking Cessation Service Among Chronic Obstructive Pulmonary Disease (COPD) Patients in Thailand
EE311: Cost-Effectiveness Analysis of Ruxolitinib vs Best Available Therapy for the Treatment of Myelofibrosis in the United States
EE342: Compression Therapy with Early Endovenous Ablation in Venous Leg Ulceration in the U.S.: A Cost-Effectiveness Analysis
EE44: Cost-Effectiveness of Pembrolizumab in First-Line Treatment of PD-L1 Positive Persistent, Recurrent, or Metastatic Cervical Cancer
SA12: Can We Predicting Trial Failure Among Older Adult-Specific Clinical Trials Using Trial-Level Factors?
12:45 PM - 1:15 PM
HEOR Theater
Comparing Registry and Electronic Health Record (EHR) Data for Real-World Evidence Generation: Heart Failure as a Case Study
In-person
Generating real-world evidence (RWE) of patients with heart failure requires clinical data elements not commonly found in administrative databases. This presentation compares and contrasts the view of patients with heart failure from the perspective of the PINNACLE® Registry, a large cardiovascular disease registry developed by the American College of Cardiology, with those identified in the Practice Fusion ambulatory electronic health record (EHR) database. Both data sources represent large collections of real-world data (RWD) across the United States, but from different perspectives; where PINNACLE primarily represents cardiologists and related specialists, Practice Fusion contains primary care physicians and specialists in ambulatory, community-based healthcare practices. Differences between these types of data sources have deep implications for identifying key patient populations within RWD sources in terms of both understanding heart failure management, as well as potential recruitment for observational research or clinical trials.
Sponsor
Veradigm
Speaker
Machaon Bonafede, PhD, MPH
Veradigm Life Sciences, Brentwood, NH, USA
Nam Nguyen, MS, MBA
Veradigm Life Sciences, Houston, TX, USA
1:30 PM - 2:30 PM
Concurrent Breakout Session 5
Do We Really Need Another Preference Study? Using Benefit-Transfer Methods to Increase Use of Patient-Preference Data in Decision Making
In-person
Level: Intermediate
PURPOSE: Time and cost of de-novo, stated-preference survey development, data collection, and analysis often are barriers to incorporating patient preferences in decision making. In some therapeutic areas, there is enough preference data to begin evaluating and using previous studies to predict values in a new context. This workshop will focus on how to leverage existing preference evidence obtained in one or more treatment contexts to impute or “transfer” health-improvement benefits to answer different questions. Participants will learn how to assess the robustness of preference evidence bases to support benefit transfers and what statistical methods are available to conduct principled and transparent benefit transfers.
DESCRIPTION: Workshop attendees will obtain a working knowledge of available methods for conducting valid benefit transfers. The workshop will review a) how benefit-transfer methods have extensively been used in non-health applied economics, b) how familiar biostatistical tools can be adapted to benefit-transfer applications, and c) how multiple strategies were used in a recent health application. Dr. Johnson will chair the session and introduce the topic in the context of the extensive benefit-transfer literature in environmental economics (10 min.), Dr. Groothuis-Oudshoorn will show how established statistical methods for estimation, attribution, and prediction can apply to benefit transfers (15 min.), and Dr. Gonzalez will illustrate how these methods were applied to evaluate the preference evidence base in psoriasis and to derive consensus values and functions to predict maximum acceptable treatment risk for given therapeutic benefits and to evaluate the impact of cross-study factors (15 min.). Audience participation will include identifying problems and solutions for a hypothetical case study in inflammatory bowel disease (20 min). This interactive and informative workshop will be valuable to researchers, clinicians, and industry analysts who are interested making patient-preference evidence available for a wider range of decision-making applications.
Discussion Leaders
Reed Johnson, PhD
Duke Clinical Research Institute, Durham, NC, USA
F. Reed Johnson, PhD, has more than 45 years of academic and research experience in health and environmental economics. He currently is Professor in the Departments of Population Health Sciences and Medicine, Duke School of Medicine, and the Duke Clinical Research Institute. He led the first FDA-sponsored study to quantify patients’ willingness to accept benefit-risk tradeoffs for new health technologies. In 2018 the International Society for Pharmacoeconomics and Outcomes Research awarded him the Donabedian Outcomes Research Lifetime Achievement Award.
Discussants
Marco Boeri, PhD
RTI Health Solutions, Belfast, United Kingdom
Juan Gonzalez, PhD
Duke Clinical Research Institute, Cary, NC, USA
Dr. Gonzalez is an Associate Professor in the Department of Population Health Sciences at the Duke University School of Medicine. He is an expert in the design of stated-preference survey instruments and the use of advanced statistical tools to analyze stated-preference data. His research has focused on two main areas: 1) transparency in benefit-risk evaluations of medical interventions, and 2) elicitation of health preferences from multiple stakeholders to support shared decision making.
Dr. Gonzalez Co-led the first FDA-sponsored preference study. The study was highlighted in FDA’s recent precedent-setting guidance for submitting patient-preference evidence to inform regulatory benefit-risk evaluations of new medical devices. More recently, Dr. Gonzalez collaborated with the Medical Devices Innovation Consortium (MDIC) to prepare the first catalog of preference-elicitation methods suitable for benefit-risk assessments of medical devices. The catalog was part of the Patient-Centered Benefit-Risk Assessment Framework developed by MDIC. As a core group member of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Conjoint Analysis Task Force, Dr. Gonzalez helped draft good-practice recommendations for statistical analysis, interpretation, and reporting of health preference data. Dr. Gonzalez is the current Chair of the ISPOR special interest group on stated-preference research.
Best Practices for Causal Study Designs Using Real-World Data
In-person
Level: Intermediate
PURPOSE: This workshop provides step-by-step guidance on the conduct of causal inference studies using observational real-world data (RWD), highlighting practical considerations, and illustrated with case study examples. Participants will use real-time voting to share their own experiences and take part in a knowledge assessment.
DESCRIPTION: Causal inference in observational research is growing more important, driven by the need for generalizable and rapidly delivered real-world evidence (RWE) to inform regulatory, payer, and patient/provider decision-making. Existing methodological literature on this topic – from the new-user, active comparator design to the adjustment of time-varying confounders – is rich but can be complex and daunting to navigate. It is important that appropriate measures to mitigate bias are incorporated both into the design and the analysis of causal inference studies using RWD.
Workshop participants with intermediate-level subject knowledge will be familiarized with concepts needed to successfully design causal inference studies using RWD. Dr Grabner will initiate the workshop highlighting the underlying need for explicit consideration of causal study design principles, introduce the best-practices documents that the authors have developed, and solicit feedback from participants on their level of comfort with causal methods and interpretations using RWD (~15-20 minutes). Afterwards, Dr Gangan will present step-by-step guidance in designing a causal RWD study, including selecting an estimand, creating a directed acyclic graph, identifying biases and corresponding solutions, and conducting sensitivity analyses (~15-20 minutes). Next, Dr dosReis will present case study examples of specific causal design/analytic issues and how they were addressed (or not addressed). Dr dosReis will also conduct the second part of the knowledge assessment and provide workshop conclusions (~15-20 minutes). Besides real-time voting, there will be time for audience questions. In addition, the best-practices documents (consisting of a step-by-step guide to causal study design and a glossary of key terms) will be available to all participants.
Discussion Leaders
Michael Grabner, PhD
HealthCore, Inc., Wilmington, DE, USA
Dr. Grabner has over ten years of experience conducting HEOR using a variety of study designs, data sources, and statistical methods. As a Principal Scientist at HealthCore, he is responsible for developing research solutions for internal and external clients, ensuring the scientific appropriateness and integrity of study goals, and for the dissemination of research findings. His research focuses on the comparative effectiveness and cost effectiveness of medical treatments with an emphasis in diabetes, autoimmune disease, and oncology.
Discussants
Susan Dosreis, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
Susan dosReis, PhD is a professor and Vice Chair of Research in the Department of Pharmaceutical Health Services Research at the University of Maryland School of Pharmacy. She has expertise in stated preference methods to evaluate trade-offs of medication benefits and risks. As Director of the Patient-Driven Values in Healthcare Evaluation (PAVE) Center, she is applying novel methods to better understand treatment decision-making from a variety of patient groups, and in particular the underserved communities.
Nilesh Gangan, PhD
HealthCore, Inc., Wilmington, DE, USA
Nilesh Gangan is a research scientist trained in HEOR and Real-World Evidence (RWE) generation. His expertise includes leading and designing retrospective database, patient-reported outcomes, health economic modeling studies and has research experience across different therapeutic areas. He enjoys generating new research ideas, designing, and managing studies to answer different healthcare questions.
The Empowered Patient: Driving Individual and Population Health with Data & Technology
In-person & Virtual
Level: Foundational
Purpose: The objectives of this session are to educate the audience on the challenges standing in the way of equipping patients with their own health data and the manifold benefits of an empowered patient at both the individual and population levels.
Description: O
ver 80% of Americans support increased access to personal health information, according to a recent Pew survey. Patients are playing an increasingly active role in their healthcare experiences and changing the dynamic between the patient, the physician, the payer, and the life sciences industry. And yet the healthcare system still lacks the infrastructure for patients to comprehensively access, understand, and make use of their own data. How can we break down the healthcare system’s siloes to give patients full control of their health information? What are the advantages and implications of patient-controlled data? In this conversation, two digital health experts will discuss their efforts to enhance patient engagement and the role of data and technology to impact patient behaviors that can lead to improved health outcomes, increased satisfaction and care delivery efficiency, reduced costs, better quality of care, and patient safety. The conversation will also draw attention to the role patients can play in advancing high quality research, powered by real-world data, to drive a deeper understanding of health and disease in the real world. Using their work together in multiple sclerosis research as a case study, PicnicHealth CEO Noga Leviner and Komodo Health President Web Sun will detail their joint conviction on the power of reliable, comprehensive real-world data to transform the industry’s approach to studying complex disease. The conversation will spotlight the challenges of building a comprehensive patient history, emerging approaches and benefits to tackling health data at both the patient level and the population level.
Speakers
Dan Drozd, MD, MSc Epidemiology
PicnicHealth, San Francisco, CA, USA
Tabassum Khan, MD, MPH
Komodo Health, Atlanta, GA, USA
Discussion Leaders
Ivy Weng, MD
Komodo Health, New York City, NY, USA
What Statistics and Analytics Resources Are Available to HEOR Professionals?
Virtual
Level: Intermediate
Purpose : The objectives of this session are to: 1) identify training tools and resources for gaining the statistical knowledge and skills necessary to conduct and report health economics and outcomes research; 2) discuss gaps in training tools and resources. Description : The ISPOR Health Economics and Outcomes Research (HEOR) Competencies Framework (CF) is comprised of 41 competencies grouped into 13 topic domains. In prior sessions, we highlighted the ‘Statistics and Analytics’ competency, the sole category within the ‘Methodological and Statistical Research’ topic domain. Focusing on the ‘Statistics and Analytics’ competency, we pre-specified subtopics and mapped them to available tools and resources. The pre-specified subtopics included: study design; regression models; extract-transform-load; data; statistical inference; big data methods; prediction models; statistical programs; emerging interfaces; risk adjustment methods; communicating results (visualization). The list of subtopics was developed by the study team, reviewed by experts in the HEOR field, and then updated based on their input. We developed data collection tools and utilized them to systematically extract information about training resources available in the published literature or provided through ISPOR webinars and Short Courses. During this session, we will describe our search process then present our findings from the review of published literature (12 minutes, Onukwugha), ISPOR webinars (12 minutes, Karcher), and ISPOR Short Courses (12 minutes, Faries). Presenters will characterize training resources based on the identified subtopics addressed, recommendations provided, and links to supplemental training tools. We will identify gaps in the available training tools and resources by describing the extent to which they covered the range of pre-specified subtopics (4 minutes, Onukwugha). The session will conclude with a discussion (15 minutes) during which we will seek feedback from the audience on approaches to address the identified gaps and guidance on the next competency category that should be examined.
Speakers
Douglas Faries, PhD
Eli Lilly and Company, Indianapolis, IN, USA
Doug Faries has a PhD in Statistics from Oklahoma State University and is currently a Senior Research Fellow in Real World Analytics & Access at Lilly. In this role, Doug is involved with the design and analysis of observational research including comparative effectiveness and he leads the development of real-world analytical capabilities for the business. He is active in the statistical community with over 150 peer-reviewed manuscripts and his research interests are in causal inference and unmeasured confounding.
Helene Karcher, PhD
Novartis Pharma AG, Basel, Switzerland
Discussion Leaders
Ebere Onukwugha, BSc, MSc, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
Eberechukwu Onukwugha, PhD is an Associate Professor in the Department of Pharmaceutical Health Services Research and is the Executive Director of Pharmaceutical Research Computing at the University of Maryland School of Pharmacy. She received a Master of Science in agricultural and applied economics as well as a Doctor of Philosophy in economics (concentration: econometrics) from Virginia Tech. Dr. Onukwugha was a recipient of the PhRMA Foundation’s Post-Doctoral Fellowship in health economics and outcomes research. Dr. Onukwugha examines the costs and health outcomes associated with health-related decisions as well as the institutional and environmental context framing these decisions.
Podium Session 5
Addressing the Impact of Social Determinants and Disparities on Health Outcomes
Virtual
Moderator
Mehtap Tatar, MD, PhD
Polar Health Economics & Policy Consultancy, Ankara, Turkey
P28: Socioeconomic Disparities in Ischemic Stroke Care: Imaging and Acute Treatment Utilization from a Comprehensive Stroke Center
2:00PM - 2:15PM
Wang J 1 , Boltyenkov A2 , Katz JM1 , O'Hara J3 , Gribko M1 , Sanelli PC1 1 Northwell Health, Manhasset, NY, USA, 2 Siemens Healthcare GmbH, Erlangen, Germany, 3 Feinstein Institutes for Medical Research, Manhasset, NY, USA
Objective: We analyzed whether socioeconomic factors affected utilization of stroke neuroimaging and acute treatment at a comprehensive stroke center. Methods: Retrospective study of consecutive acute ischemic stroke patients at a metropolitan comprehensive stroke center from 2012-2020 was performed. Differences in neuroimaging (CTA, CTP, MRI, MRA) and treatment (intravenous thrombolysis – IVT, endovascular thrombectomy – EVT) utilization were evaluated based on socioeconomic factors of age, sex, race, insurance-type, and household income. Chi-square tests were used for bivariate analyses. A multivariable logistic regression model was fit to determine associations between socioeconomic factors and neuroimaging and treatment utilization while controlling for medical comorbidities and stroke-specific factors. Results: The cohort comprised 6,140 consecutive AIS discharges from our institution during the study period. It had 47.6% female; 59.5% white, 16.6% Black, and 10.6% Asian; with 65.2% having private insurance, 52.7% Medicare, 12.9% Medicaid, and 2.1% uninsured. The mean (SD) age was 70.5 (15.1) years, NIHSS 7.4 (7.9), and Last-Known-Well to Admission 18.2 (31.1) hours. The most common comorbidities were hypertension (69.1%), dyslipidemia (40.9%), obesity (35.2%), and diabetes mellitus (30.8%), while 18.5% had prior stroke. 80+ year-old patients had lower CTA (OR=0.62 [0.51-0.75]) and EVT utilization (OR=0.53 [0.39-0.73]), while female patients had lower CTA utilization (OR=0.78 [0.65-0.93]), but equal treatment. All races had equivalent CTA, CTP, and MRA utilization, and odds of acute stroke treatment. While privately insured patients had higher utilization of MRA (OR=1.24 [1.04-1.49]) and EVT (OR=1.62 [1.20-2.20]), uninsured patients had higher MRI utilization (OR=1.64 [1.07-2.50]). Conclusions: Patients 80 years old and older had lower utilization of both CTA and EVT, while female patients had less CTA utilization, but equal acute stroke treatment. All races had equal utilization of acute stroke treatment and all other neuroimaging modalities. We found significantly fewer disparities in stroke imaging or acute treatment utilization than prior studies.
P27: Addressing Health Barriers Using Social Determinant of Health Data
1:30PM - 1:45PM
Kaur G
Objective: Blue Cross and Blue Shield of Louisiana (BCBSLA) seeks to improve health outcomes for its members and is gaining better insight on persistent challenges affecting them, such as chronic conditions, utilization practices and medication adherence. One approach BCBSLA is using is incorporating Social Determinants of Health (SDOH) data into existing workflows to better understand the health barriers that impact their communities. Methods: Representing hundreds of disparate social factors, this information comes from multiple sources, including government agencies and private companies. Examples include the CDC’s Social Vulnerability Index, the Robert Wood Johnson Foundation, 500 Cities: Census Tract-Level Data, Data, and Smart Location Database. Disparate data was normalized and processed to create a final set compatible with the current BCBSLA enterprise-wide data warehouse. SDOH data was then incorporated into existing programs and predictive models. The first effort using the new data resource was to understand the gaps in childhood immunization status and behavioral health. Results: SDOH data enabled BCBSLA to identify census tracts in parishes that were below the state’s overall immunization rate and assign potential reasons for the observed inequities. Overall, Louisiana CIS Combination 2 (DTaP, IPV, MMR, HiB, HepB, VZV) immunization rate is 74.28%. However, 45% of parishes were below the state immunization rate with the majority being socioeconomically poor. For census tracts with no SDOH barriers, immunization rates were higher and primarily driven by provider intervention. Similarly, BCBSLA used SDOH data to identify social barriers within parishes where prevalence of chronic conditions is high and where behavioral health prevalence rates need to be addressed. Conclusions: By augmenting existing analytics with SDOH data, BCBSLA can incorporate this newly generated evidence into interventions that can contribute to BCBSLA improving the effectiveness of care for its members.
P26: Unintended Consequences of the CDC’s 2016 Guideline for Prescribing Opioids for Chronic Pain for the Individuals with Sickle Cell Disease
2:15PM - 2:30PM
Kang HA 1 , Wang B2 , Barner JC1 , Ataga KI3 , Mignacca RC4 1 The University of Texas at Austin, Austin, TX, USA, 2 The University of Texas at El Paso, El Paso, TX, USA, 3 The University of Tennessee Health Science Center, Memphis, TN, USA, 4 Children’s Blood and Cancer Center at Dell Children's Hospital, Austin, TX, USA
OBJECTIVES:
Because the Centers for Disease Control (CDC) Guideline for Prescribing Opioids for Chronic Pain released in March 2016 was not intended to restrict opioid therapy to patients with sickle cell disease (SCD), we assessed whether the guideline resulted in a shift in prescribing level and negative health outcomes among this population. METHODS:
This retrospective cohort study employed an interrupted time series analysis using the IBM® MarketScan® Commercial Database from 1/1/2011 to 12/31/2019. Patients who were
≥1 years old, had ≥ 3 SCD diagnoses within 5 years, and no cancer diagnosis were included. Monthly-measured opioid use outcomes included: opioid prescription rate, mean total morphine milligram equivalents (MME) per patient, mean daily MME per opioid prescription, and mean number of days supplied per opioid prescription. Monthly rates of vaso-occlusive crisis (VOC)-related emergency department (ED) visits and hospitalizations were measured as health outcomes. Segmented regressions (breakpoint: March 2016) were conducted for the all outcomes and the regression coefficients were used to estimate the changes of trends before and after the guideline release.
RESULTS:
The cohort included 14,979 SCD patients with a mean age of 25.9±16.9 years and 56.9% female. Compared to the pre-guideline trend, the following changes were observed after the guideline release: a significant decrease in the opioid prescription rate (–0.29 prescriptions/100 person-month, P<0.001), amount of opioid prescribed (–141.0 MME/person-month, P=0.001; –10.1 MME/prescription-month, P<0.001), and number of days supplied per prescription (-0.05 days/month, P<0.001). However, following release of the guideline, there was a significant increase in the VOC-related hospitalization rate (+0.16 hospitalizations/100 person-month, p=0.001), although no significant change in the VOC-related ED visit rate was observed.
CONCLUSIONS:
Release of the CDC guideline was associated with a decrease in opioid prescribing practices and unfavorable health outcomes in patients with SCD. The guidelines may have an unintended negative impact on this population.
P25: Income and Equity Effect of Modelled Sugar Tax and Fruit and Vegetable Subsidy Program across Different Income Groups in Canada
1:45PM - 2:00PM
Liu S
Objectives: Food taxes and subsidies have been implemented in many countries, and they have effectively improved the dietary patterns of populations and reduced disease risks, enhanced quality of life, and saved healthcare costs. However, the equity consequences of these interventions are not well understood. This study aims to assess the impact of sugar tax and fruit and vegetable subsidy programs on future health care spending for chronic disease management and treatment by income quintiles in Canada. Methods: Using proportional multi-state life table-based Markov models, we simulated the changes in disability-adjusted life years (DALYs), healthcare costs, and food expenditure after implementing a CAD$0.75/100g free sugar content tax on sugary foods and beverages and a 20% subsidy on fruits and vegetables for five income quintiles, over a lifetime of the 2015 Canadian adult population. Model parameters were from the previous studies, Statistic Canada, and other available datasets. Results: The simulated tax and subsidy programs could avert 12.19 million DALYs and save CAD$123.06 billion in direct healthcare costs in a lifetime period. The two lowest income quintiles would gain greater health benefits, with 45.30% of averted DALYs and 46.59% of healthcare cost offsets. The estimated annual sugar tax revenue and food subsidy were CAD$4.43 billion and CAD$7.65 billion, respectively. The lowest income quintiles would pay the highest proportion of income in sugar tax (0.81%, CAD$119.34/person/year), while they would gain the highest proportion of income in fruit and vegetable subsidy (1.30%, CAD$193.71/person/year) making their net gain the highest among income groups. Conclusion: The simulation study shows for Canada that lower groups would gain greater health benefits from simulated tax and subsidy programs. Although the sugar tax was financially regressive, the fruit and vegetable subsidy could compensate for the tax burden of disadvantaged groups and improve population health and achieve health and financial equity.
Concurrent Breakout Session 5
Paying for Digital Therapeutics (DTx): What Evidence Is Needed?
In-person
ISSUE: There is a disconnect between digital innovators, payers, and patients as it relates to the value offered by DTx (software or software-based products intended to help prevent, treat, or manage a disease or medical condition). Payers seek adequate evidence in comparison to other interventions. In the digital era, manufacturers contest that evidence standards such as RCTs are not practical while patients request access to DTx to improve quality of life. What is feasible to expedite DTx coverage decision making and improve patient care?
OVERVIEW: Biopharma companies have embraced DTx and studies have shown to improve outcomes from DTx, either alone or in conjunction with conventional protocols. While there is some optimism in DTx circles that COVID-19 will accelerate acceptance and use of these promising treatment options, the DTx sector as a whole is still in the early stages of development and the needs for payers to evaluate these products is not well understood. Each panelist will present information from current practice and survey results (15 mins each) to debate:
What role does regulatory approval for DTx play? How can we measure, analyze and interpret improvements from DTx? What are the competencies needed for evaluation? How could RWE improve certainty for payers? How can evidence be communicated between payers and manufacturers? What are the issues around implementation for healthcare systems? An interactive discussion around these topics with participants will follow the presentations. Participants from payers, DTx manufacturers and HTA bodies will benefit from attending. The feedback collected will provide opportunities to define further research questions and examine recommendations for the next steps with a new focus on the challenges of DTx solutions.
Moderators
Anita Burrell, MA, MBA
Anita Burrell Consulting, Flemington, NJ, USA
Anita has over 23 years’ experience of leadership in the pharmaceutical industry in a wide variety of roles. She joined the industry as a pioneer in the field of Health Economics for Burroughs Wellcome and moved to New Jersey in 2001 to lead Global Health Economics and Outcomes Research for Aventis. Following the Sanofi merger in 2004 she moved to Paris to assume the role of Head, Global Health Economics, Reimbursement and Pricing for successful market access of products. On her return to the USA, she led Aubagio, the oral MS therapy through submission and approval in over 30 markets worldwide and was responsible for the strategic commercial execution on the Sanofi diabetes portfolio
As the principal of Anita Burrell Consulting LLC, Anita helps companies understand market dynamics and payer behaviour in the top pharmaceutical markets, she has developed predictive models to inform health technology assessment submissions, and enables clients to develop relevant value evidence development and payer communication for major markets.
Anita holds a BA (Hons) in Economics from the University of Stirling, a MA in Economics from Dalhousie University, and an MBA from Kingston University.
Panelists
Vyishali Dharbhamalla, PharmD
Academy of Managed Care Pharmacy, Alexandria, VA, USA
Vyishali Dharbhamalla, PharmD, RPh, is the Senior Manager, Professional Affairs, at the Academy of Managed Care Pharmacy (AMCP). In her role, she works on advancing AMCP’s strategic priorities and thought leadership work which includes working with advisory groups and partnership forums. She is currently working on two areas of the strategic priorities, health disparities and quality. She also works with two AMCP committees, precepts students, and engages with external stakeholders.
Dr. Dharbhamalla earned a bachelor of sciences, bachelor of arts, and a doctorate in pharmacy from The Ohio State University. She completed a Leadership and Association Management Fellowship at the Ohio Pharmacists Association.
Jennifer Goldsack, MS, MBA
DiMe, Boston, MA, USA
Jen Goldsack is the Executive Director of the Digital Medicine Society (DiMe). Previously, Jen spent several years at the Clinical Trials Transformation Initiative (CTTI) where she led development and implementation several projects within CTTI’s Mobile Program and was the operational co-lead on the first randomized clinical trial using FDA’s Sentinel System. Jen spent five years working in research at the Hospital of the University of Pennsylvania, first in Outcomes Research in the Department of Surgery and later in the Department of Medicine. More recently, she helped launch the Value Institute, a pragmatic research and innovation center embedded in a large academic medical center in Delaware. Jen earned her master’s degree in chemistry from the University of Oxford, England, her masters in the history and sociology of medicine from the University of Pennsylvania, and her MBA from the George Washington University. Additionally, she is a certified Lean Six Sigma Green Belt and a Certified Professional in Healthcare Quality. Ms Goldsack is a retired athlete, formerly a Pan American Games Champion, Olympian and World Championship silver medalist.
Zachary Zalewski, PhD, JD
Avalere Health, Naples, FL, USA
As a member of Avalere’s team, Zach brings a holistic perspective in supporting clients through the FDA regulatory and policy environments. Trained in genetics, he has broad technical knowledge and expertise in molecular biology and related disciplines. From his legal studies, he has experience with statutory interpretation, regulatory analysis, and case law.
Zach has a JD from Case Western Reserve University with a health law concentration, a PhD in molecular and human genetics from Baylor College of Medicine, and a dual major BS in microbiology and history from Michigan State University. Prior to joining Avalere and while in law school, Zach clerked at a Cleveland intellectual property law firm and spent a semester at The Hastings Center, a nonpartisan bioethics research institute.
Surrogate Endpoints Under Attack: Is It Still Worth Performing Surrogacy Validation? Lessons from NSCLC
In-person
ISSUE: The use of surrogate endpoints for overall survival has been advocated as a way to accelerate drug development, approval, and reimbursement decisions, particularly for treatments for smaller populations or diseases where death rates are lower and take longer to occur. Thus, their use could facilitate addressing unmet medical needs in a timely manner. Mitigating the impact of recent regulatory requirements to change Phase 3 trial designs in oncology, such as crossover, has also been given as a rationale for using surrogate endpoints. For NSCLC, hyper-progression on immune-oncology treatments and crossover have been identified as potential factors leading to different results in the surrogacy validation process. Yet, a comprehensive framework and methods to understanding and validating surrogacy in oncology are currently lacking. Recent scientific and public debates show that surrogate endpoints are under attack, and a more rigorous and consistent evaluation is desired to provide greater confidence in their use and pave the way for more effective clinical development and care decisions.
OVERVIEW: Silvia Paddock moderates the session and sets the scene by presenting the challenges of surrogacy validation in oncology and summarizing its particularities in NSCLC. Dalia Dawoud provides an update on recent requirements for surrogacy validation at NICE and the implications for future HTAs. Advanced statistical methods can reduce the uncertainty around predictions from the surrogate to the final outcome. Billy Amzal illustrates this for NSCLC and shows that progression-free survival can serve as potential surrogate for overall survival. He also discusses options to transfer the findings into a real-world setting. Jeff Allen provides an overview of the potential benefits of using surrogate endpoints for people living with cancer and recent initiatives on surrogacy validation in NSCLC at Friends of Cancer Research. The audience and panelists reflect on the presentations in 15 minutes.
Moderators
Silvia Paddock, PhD
PricewaterhouseCoopers AG, Zurich, Switzerland
2021-current: Senior Manager at PricewaterhouseCoopers AG, Zurich, Switzerland
2019-2021: Senior Manager at Certara, Germany
2016-2021: Independent Consultant (Paddock Science)
2012-2016: Senior Associate, Rose Li and Associates (RLA), Bethesda, USA
2010-2012: Independent Consultant (Paddock Science)
2008-2009: Associate Professor, Department of Neuroscience, Karolinska Institute, Stockholm, Sweden
2006-2008: Assistant Professor, Department of Neuroscience, Karolinska Institute, Stockholm, Sweden
2003-2005: Visiting Postdoctoral Fellow, National Institute of Mental Health, National Institutes of Health, Bethesda, MD
Panelists
Jeff Allen, PhD
Friends of Cancer Research, Washington, DC, USA
Jeff Allen, Ph.D. serves as the President and CEO of Friends of Cancer Research (Friends). For over 25 years, Friends has created unique scientific partnerships, accelerated policy change, and supported groundbreaking research to deliver new therapies to patients quickly and safely. As a key thought leader on issues related to the U.S. Food and Drug Administration, healthcare, and regulatory policy, he is regularly published in prestigious medical journals and policy publications and has contributed his expertise to the legislative process on multiple occasions.
Billy Amzal, MSc, MPH, PhD
Quinten Health, PARIS 05, 75, France
Billy Amzal graduated from Ecole Polytechnique, from AgroParisTech and holds a PhD in Decision Mathematics from Paris-Dauphine University which was awarded by the International Society of Bayesian Analysis and by the International Biometrics Society. Over the last 20 years, Billy has developed predictive analytics tools to inform and support strategic decision making in healthcare. Prior to joining Quinten Health as CEO, Billy developed predictive and impactful tools within big pharma companies and public health agencies. He then led consultancy teams at Certara focusing on RWD and decision analytics as senior VP, acted as statistical expert for public Health Authorities (EFSA, ANSES, WHO), and taught pharmaco-economics at CNAM university in Paris.
Billy authored more than 100 scientific publications in international journals.
He conceived and led hundreds of disease modeling projects supported 100s of HEOR model submissions to HTAs and real-world database studies. He created Quinten Health, the leading company in RW simulation and disease modeling.
Dalia Dawoud, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Dalia Dawoud, PhD, is Senior Scientific Adviser at the National Institute for Health and Care Excellence (NICE). She holds MSc in Economic Evaluation in Health Care from City University London and PhD in pharmaceutical policy and economics from King’s College London.
She has long experience in using economic evaluation in clinical guidelines development and health technology assessment (HTA), gained through working on NICE Clinical Guidelines as well as technology appraisals. Dalia’s research interests are focused on the advanced methods of evidence synthesis and use in economic models and the use of real-world evidence to inform drug development and health care decision making. Dalia currently has overall responsibility of overseeing the delivery of NICE allocated tasks on a portfolio of IMI and Horizon 2020 funded research projects including EHDEN and HTx. She is widely published in the field of pharmaceutical policy and pharmacoeconomics. She also serves as Associate Editor for ISPOR journal Value in Health and as Associate Editor for Pharmacoeconomics and Outcomes Research for Elsevier’s journal Research in Social and Administrative Pharmacy. Dalia also holds adjunct position as Associate Professor at the Faculty of Pharmacy, Cairo University.
Standing Up Computable Phenotypes for Generating Real World Evidence: What Are Computable Phenotypes and Can They Really Be Standardized and Reused?
In-person & Virtual
ISSUE:
Computable phenotypes are definitions of clinical conditions, outcomes and exposures that can be implemented in real-world data sources, such as electronic health record (EHR) data and/or medical claims data. The September 2021 FDA draft guidance on “Real World Data: Assessing Electronic Health Records and Medical Claims Data To Support Regulatory Decision-Making for Drug and Biological Products” recommended that standardized computable phenotypes be utilized in real world evidence (RWE) studies for regulatory decision-making. The use of publicly available and reusable computable phenotypes can increase the transparency and efficiency of research in “real world” health system settings – and also increase the speed of dissemination of results and practice change. However, there are challenges in creating reusable phenotypes that can operate on heterogeneous EHR data with different levels of data quality, population representation, and data capture.
OVERVIEW:
In this interactive panel, each panelist (10 min each) will describe their experience with developing computable phenotypes for different use cases including health system research, PCORnet studies and FDA Sentinel system surveillance, and will share their different views on many questions including:
What is standardization in this context? Who owns the standardization process and who will fund it? How can data heterogeneity across sites/sources be systematically identified and addressed in a manner that assures comparability across sites/sources? What are some key issues in validation, sharing and reuse of computable phenotypes developed with artificial intelligence? What are the costs associated with standardizing computable phenotypes? Will the resources required create a barrier for lower-resourced health provider organizations to participate? Can standardization be implemented in a way that avoids perpetuating disparities/inequities? Then the panel will invite the audience to join the discussion and debate the issues. At the end of the session, we will discuss possible next steps and how the ISPOR community can help address these important questions.
Moderators
Elise Berliner, PhD
Cerner Enviza, Kansas City, MO, USA
Panelists
David Carrell, PhD
Kaiser Permanent Washington, SEATTLE, WA, USA
Kevin Haynes, PharmD, MSCE, FISPE
Janssen Research and Development, LLC, Titusville, NJ, USA
Rachel Richesson, PhD, MPH, FACMI
University of Michigan Medical School, Ann Arbor, MI, USA
Technology and Machine-Learning Enabled Systematic Reviews: How Can They Be Leveraged to Support Global Health Technology Assessments?
Virtual
ISSUE: There have been significant advances in technology and machine learning methods to support systematic reviews. Indeed, the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) for reporting systematic reviews has recognized the application of automation tools within their recently updated 2020 updated guideline. However, many health technology assessment (HTA) agencies have not yet issued formal guidance around the application and use of technology and machine-learning applications to support systematic reviews in submissions and technology appraisals. In the absence of such guidance, it is unclear whether use of emerging technologies and machine learning applications to support systematic reviews will be accepted by global HTA bodies. There is an urgent need to explore this issue from a multi-stakeholder perspective.
OVERVIEW: This panel will debate whether technology and machine-learning enabled systematic reviews can meet the evidence requirements of global HTA bodies. Dr. Chris Cameron will moderate the panel and provide an overview of the current landscape of technology and machine-learning applications, as well as HTA requirements on systematic reviews to support global HTA submissions. He will also pose key questions for the panelists to debate, including:
What are the opportunities with leveraging technology and machine-learning enabled systematic reviews for HTA submissions? What are the challenges with leveraging technology and machine-learning enabled systematic reviews for HTA submissions? Why might technology and machine-learning enabled systematic reviews be acceptable for publications, but not be considered well suited for HTA submissions? Which technology and machine-learning methods are most acceptable from an HTA perspective? International experts will each describe their unique perspective from technology/ML developer, HTA and academic perspective on technology and machine-learning enabled systematic reviews in support of HTA submissions. Target stakeholders include HTA agencies, academics, patient advocacy organizations, and pharma/biotech manufacturers.
Moderators
Chris Cameron, MSc, PhD
EVERSANA, Sydney, NS, Canada
Panelists
Candyce Hamel, MPH
Canadian Association of Radiologists, Ottawa, ON, Canada
Brian Hutton, Ph.D.
University of Ottawa, Ottawa, ON, Canada
Brian Hutton is a Senior Scientist at the Ottawa Hospital Research Institute in Ottawa, Canada and an Associate Professor in the University of Ottawa's School of Epidemiology and Public Health. He is also Director of the Knowledge Synthesis Group, and has interests in the areas of knowledge synthesis, network meta-analysis, clinical trials and real-world data.
Ian Stefanison, BS
Evidence Partners, Ottawa, ON, Canada
2:30 PM - 3:00 PM
Coffee Break
In-person
3:00 PM - 4:00 PM
ISPOR Forums
Collaboration on HTA Within the EU: What Does It Mean for CEE Countries?
In-person
The European Union (EU) has made significant steps recently towards a strong collaboration on Health Technology Assessment (HTA). The ultimate goal is a permanent framework for joint work, that includes joint scientific consultations, and joint clinical assessments among other potential areas of collaboration. This can potentially bring significant changes to the everyday practice of HTA institutions in EU member states and beyond. Members of the ISPOR Central and Eastern Europe (CEE) Consortium agreed that this timely topic is definitely worth discussing, as it would impact the work of all stakeholders who are connected to the local HTA processes in one way or another.
In this session, Bertalan Németh, Chair of the ISPOR CEE Consortium will provide an overview of the new HTA regulation in EU. Gergő Merész from the Hungarian HTA Office will present his view on how these milestones were achieved and what are the perceived benefits and limitations of the changes. This will be followed by Oresta Pinyazhko, Director of the HTA Department of Ukraine who will present her views in the impact on daily HTA practice from a European but non-EU perspective. Maciej Niewada from an independent researcher perspective will explain how this new situation can affect stakeholders other than HTA agencies.
Moderators
Zoltan Kalo, PhD
1) Semmelweis University; 2) Syreon Research Institute, Budapest, Hungary
Zoltán Kaló is a professor of Health Economics at the Center for Health Technology Assessment of Semmelweis University in Budapest, Hungary. Before moving to Semmelweis University in July 2019 he was the founder and co-director of an international master program in Health Policy, Planning, and Financing at Eötvös Loránd University (ELTE).
Dr. Kaló is also the founder and leader of Syreon Research Institute, an international research corporation specializing in health policy, health economic modeling, and technology assessment.
He has 25 years of international experience in academia and industry, specializing in health systems design, HTA implementation, health economics and outcomes research, patient access, and pricing policies of healthcare technologies.
Dr. Kaló serves as a policy advisor to public decision makers and global healthcare corporations. He is a Scientific Committee member of the Innovative Medicines Initiative 2 Joint Undertaking (IMI2 JU). He was a director of ISPOR between 2012-2014, and the chair of ISPOR Central and Eastern European Network Executive Committee between 2013-2015.
Bertalan Németh, PhD
ISPOR CEE Consortium Executive Committee and Syreon Research Institute, Budapest, Hungary
Bertalan Németh PhD graduated from the Corvinus University of Budapest (MSc in Quantitative economics and Operation research), the Eötvös Loránd University (Pharmaceutical economics and drug policies), and the Semmelweis University School of PhD Studies. Between 2010 and 2015 he was a health economist at the Hungarian HTA office. Since August 2015 Bertalan has been a Senior Health Economist, and since 2019 a Principal Researcher at Syreon Research Institute. Bertalan is responsible for strategic consulting, and he is involved in various projects that model for economic evaluation in health, health technology assessment and health statistics as well. Bertalan is the Past President of ISPOR Hungary Chapter, and Chair of the ISPOR CEE Consortium. He was a participant in the international EUnetHTA project, the ISPOR HTA Roundtable Europe, and the Scientific Committee of the META Conference. Bertalan was also a faculty member of the global ISPOR HTA Training, and was the module leader of Health Technology Assessment for the MSc program at Eötvös Loránd University.
Speakers
Gergő Merész, MSc
National Instute of Pharmacy and Nutrition, Budapest, Hungary
Maciej Niewada, MD, PhD
Medical University of Warsaw and HealthQuest, Warsaw, Poland
Background:
1991-1997 Medical University of Białystok/Medical University of Warsaw (MD)
1995-2000 Warsaw School of Economics (MSc in Economics);
Professional experience:
since 1997 Medical University of Warsaw, Professor in Department of Clinical and Experimental Pharmacology – specialty: clinical pharmacology
2000-2006 Institute of Psychiatry and Neurology, Professor Assistant in 2nd Neurological Department – clinical specialty: neurology
2000-2004 Medical University of Warsaw - Ph.D. on cost of stroke from societal perspective
2013 - Thesis presented to achieve a habilitation qualification – Hospital Stroke Registry in Poland – analysis of three editions in 2001-2008. Patients clinical characteristic, therapeutic management and prognosis in ischaemic and hemorrhagic stroke.
since 2009 – CEO in Healthquest – consulting company focusing on market access and reimbursement application (more info: http://healthquest.pl/12,About.html )
Co-author of Polish guidelines on heath technology assessment adopted by The Agency for Health Technology Assessment in Poland (AHTAPol).
Founder member and current Past President of the Polish Pharmacoeconomic Society (ISPOR Poland Chapter).
Co-author of over hundred health technology assessments reports (including technologies dedicated for neurology and psychiatry), costing and epidemiological studies (i.e. diabetes, cardiovascular diseases, etc.), quality of life and utility studies (http://www.researchgate.net/profile/Maciej_Niewada ). Apart from pharma industry cooperating with Ministry of Health and National Health Fund as an external advisor.
Oresta Piniazhko, PhD
State Expert Center (SEC) of Ministry of Health, Kyiv, Ukraine
Oresta Piniazhko, PhD, Director of HTA Department at State Expert Centre of Ministry of Health, Ukraine.
Oresta is an experienced expert in HTA, pharmaceutical policy and implementation practitioner. She holds a PhD degree in Pharmacoeconomics and is currently holding a position of Director of HTA Department at the State Expert Center of the Ministry of Health of Ukraine, ensuring management and implementation of the best international practices of HTA into health care system of Ukraine since February 2019. Being a dynamic communicator she is also a President of Ukraine ISPOR Chapter since 2017 and before ISPOR Ukraine Students Network (2015-2017). Oresta is visiting lecturer at The Institute of Business Education of Vadym Hetman Kyiv National Economics University and senior lecturer at Danylo Halytskyi Lviv National Medical University.
How Can We Make the Study of Patient Preferences More Useful to Decision-Makers in Health? The Final Recommendations from the ISPOR Using Patient Preferences to Inform Decision Making Good Practices Task Force
In-person
There is growing interest in increasing the use and relevance of patient preferences studies among decision makers in health. Our task force focused on developing a new framework that would: 1) be applicable to the wide variety of preference methods; 2) identify key domains that would guide researchers and other stakeholders in making patient preference studies more useful to decision makers; and 3) detail important questions that would guide researchers conducting preference studies and those critically appraising them.
The framework spans 5 domains for conducting patient preference studies to increase the use and relevance of these studies to decision makers: i. The context – A complete understanding of how decision makers function, how decisions are made, and how studies on patient preferences may be received and used. ii. The purpose – A clear articulation of how the study on patient preferences responds to decision makers’ needs and how the findings are expected to inform decision making. iii. The population – A thorough consideration of who the findings of the study will be applied to (directly or indirectly) and the intended and unintended consequences for those people. iv. The method – A selection of method/s to study patient preferences that reflects the needs, wants, and capabilities of decision makers, patients, and other stakeholders. v. The impact – A proactive effort both to ensure that the study findings can inform decision making and to measure if and how they influenced decision makers and decision-making.
Speakers will discuss: 1) a detailed overview of the ISPOR Framework; 2) compare it to different preference elicitation methods’ frameworks/recommendations such as IMI PREFER; 3) it’s application to a decision-relevant case study and 4) the decision-maker’s perspective. Feedback on bridging the gap between methods and applications will be part of the polling and moderated Q&A session.
Moderators
John Bridges, PhD
Ohio State University College of Medicine, Columbus, OH, USA
John F.P. Bridges PhD is a professor in the Departments of Biomedical Informatics and Surgery within the Ohio State University College of Medicine. Working at the intersection of medicine and the social sciences, John advances and applies methods to incorporate the priorities and preferences of patients and other stakeholders in medical decision making. John was founding editor of The Patient – Patient Centered Outcomes Research (since 2008) and serves on the editorial boards of Pharmacoeconomics (since 2006), Expert Review of Pharmacoeconomics and Outcomes Research (since 2007), and International Journal of Technology Assessment in Health Care (since 2008). Within the ISPOR he was the founding chair of the Conjoint Analysis Working Group and the Conjoint Analysis Task Force that produced several reports on good research practices for stated-preference methods. He received ISPOR’s Bernie O’Brien New Investigator Award in 2006 and ISPOR’s Distinguished Service Award in 2011. He is the author of over 200 articles and a frequent speaker on the art and science of using stated-preference methods and engaging patient organizations in decision making. John is currently affiliated with Ohio State University Comprehensive Cancer Center’s Cancer Control Program, the Center for the Advancement of Team Science, Analytics and Systems Thinking in Health Systems Research and Implementation Science (CATALYST), and the Center for Surgical Health Assessment, Research and Policy (SHARP). He is also an adjunct professor within the Department of Health Behavior and Society at the John Hopkins Bloomberg School of Public Health.
Speakers
Sebastian Heidenreich, MSc, PhD
Evidera, London, LON, United Kingdom
Laura Lee Johnson, PhD
Food and Drug Administration, Silver Spring, MD, USA
Dr. Laura Lee Johnson is a division director in the Office of Biostatistics at the U.S. Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER) and the Office of Translational Sciences’ Patient Focused Drug Development liaison. Among her many activities Dr. Johnson serves on the FDA-NIH Interagency Clinical Outcome Assessments Working Group, the IMI PREFER Scientific Advisory Board, and co-directs the NIH Principles and Practice of Clinical Research course.
Deborah Marshall, PhD, BSc
University of Calgary, Calgary, AB, Canada
Deborah is a professor at Cumming School of Medicine, University of Calgary and Arthur J.E. Child Chair in Rheumatology Outcomes Research and former Canada Research chair, Health Services and Systems Research. Her research program focuses on the measurement of preferences, cost-effectiveness analysis, and simulation modeling of health services and interventions. Deborah has over 20 years of research experience in health technology assessment agencies, academic institutions, and industry settings in Canada, US, and Europe. She is a founding co-investigator of the Patient and Community Engagement Research (PaCER) Program and co-leads the economics and stated preferences research platforms for the Canada-Netherlands Personalized Medicine Network in Childhood Arthritis and Rheumatic Disease (UCAN CANDU).
Deborah is an active member of the ISPOR as the past-president of the Board of Directors, and co-author on the three “ISPOR Task Force Reports for Good Research Practice – Checklist for Conjoint Analysis in Health, Conjoint Analysis Experimental Design and Statistical Methods for the Analysis of Discrete-Choice Experiments.”
New Professionals Session
Things They Didn't Teach in Grad School: Survival Skills for New Professionals
In-person
Graduate school does a great job of teaching technical skills. However, early career success also depends on demonstrating competencies in soft skills like influencing, creative problem-solving, leadership behaviors, critical thinking, cross-functional work, and strategic decision making. Join the members of ISPOR’s New Professional Steering Committee for this panel-style session as they highlight key soft skills and effective ways to learn and develop these skills.
Moderators
Sanket Shah, MBBS, PhD
Novo Nordisk, Gaithersberg, MD, USA
Speakers
Mark Bounthavong, PharmD, PhD, MPH
University of California-San Diego, San Diego, CA, USA
Koen Degeling, PhD, MSc, BSc
Lumen Value & Access – a Healthcare Consultancy Group Company, New York, NY, USA
Dr Koen Degeling is a Research Scientist, Health Economic Modelling & Advanced Analytics at Lumen Value & Access, a Healthcare Consultancy Group company. He was trained as an Industrial Engineer specializing in Healthcare Technology and Management and holds a PhD in Advanced Health Economic Modelling from the University of Twente in the Netherlands. Prior to joining Lumen Value & Access, Koen worked on real-world data-driven health economic and health services research projects at the Cancer Health Services Research department of the University of Melbourne in Australia, where he continues to be involved as an honorary fellow. He is an active ISPOR member and currently serves on the Editorial Advisory Board for Values & Outcomes Spotlight and ISPOR New Professionals Steering Committee, is involved in several short courses and workshops, and has served as global chair, committee co-chair, and chapter president within the ISPOR Student Network.
Anna Hung, PharmD, PhD, MS
Duke University School of Medicine, Durham, NC, USA
Anna Hung, PharmD, PhD, MS is a pharmacist and health services researcher interested in payer and patient decision making related to pharmacy benefits. Her methodological research interests include health care cost evaluations, quasi-experimental study designs, and stated preference research.
Daniel Simmons, PharmD, MS
AstraZeneca Pharmaceuticals, Gaithersburg, MD, USA
Educational Symposium
Downstream Effects of Pandemic-Related Disruptions in Healthcare Utilization and Cost: Insights from Healthcare Claims and Survey Data
In-person & Virtual
Two years from the emergence of the COVID-19 pandemic in the US, we are looking to understand the implications of the pandemic on healthcare, both from a disease management and economic perspective. Numerous analyses have reported reductions in healthcare resource utilization, especially early in 2020. Moving forward, health outcomes researchers need to understand the potential impacts of this low utilization on the general population as well as medically complex patients, different socioeconomic groups, different geographic regions, and individuals with varying types of healthcare access regulated via their insurance coverage. Life sciences organizations looking for reliable insights on therapeutic areas of interest and data to support their products continue to turn to real-world evidence (RWE) for answers. However, the actionable insights that researchers can derive from RWE are subject to research methodologies, data availability, and other external factors –such as the COVID-19 pandemic.
Join experts from Watson Health and Johns Hopkins Bloomberg School of Public Health for a discussion on the implications of the pandemic, and considerations for your outcomes research practice. Backed by in-depth insights from closed-system claims data, supplemented with healthcare consumption survey data, researchers will discuss:
How delay of diagnosis or alterations in disease management due to the initial pandemic lockdown could have long-term health and economic implications
The potential impacts of variations such as payer or plan type, specific comorbidity burdens, socioeconomic status, and region of residence on patient outcomes and costs
Important factors that will need to be addressed to ensure post-pandemic health equity
Sponsor
IBM Watson Health
Moderators
Luke Boulanger, MA, MBA
IBM Watson Health, Cambridge, MA, USA
Speakers
G. Caleb Alexander, MD, MS
Johns Hopkins Bloomberg School of Public Health, Center for Drug Safety and Effectiveness, Baltimore, MD, USA
G. Caleb Alexander, MD, MS is a Professor of Epidemiology and Medicine at Johns Hopkins Bloomberg School of Public Health, where he serves as a founding co-Director of the Center for Drug Safety and Effectiveness and Principal Investigator of the Johns Hopkins Center of Excellence in Regulatory Science and Innovation (CERSI). He is a practicing general internist and pharmacoepidemiologist and is internationally recognized for his research examining prescription drug utilization, safety and effectiveness. Dr. Alexander is the author of over 375 scientific articles and book chapters, many using primary or secondary data to characterize the epidemiology of prescription opioid use in the United States as well as to evaluate the effect of federal and state regulatory and payment policies on opioid prescribing, dispensing and utilization. His work has also included a focus on the use and safety of pharmacologic treatments for cardiovascular disease, population-based patterns and determinants of pharmaceutical use, the impact of changes in pharmaceutical policy on pharmaceutical expenditures and utilization, and U.S. Food and Drug Administration regulatory communications and post-approval activities. Dr. Alexander received his B.A. cum laude from the University of Pennsylvania, an MD from Case Western Reserve University, and a Master of Science from the University of Chicago.
Brenna Brady, PhD
IBM Watson Health, Laurel, MD, USA
Brandi Hodor, BS
IBM Watson Health, Alexandria, KY, USA
Discussion Groups
New this Year – Discussion Groups! Discussion Groups are facilitated conversations between conference attendees and select conference speakers. Held in the new, dynamic Discussion Lounge in the ISPOR Exhibit Hall, these discussions are intended to be highly interactive, collaborative, and promote the exchange of ideas in a peer-to-peer setting. Health Equity Discussion Group
In-person
Open to all attendees; seating is limited. This open discussion facilitated by the Health Equity Special Interest Group (SIG) leaders will reflect on key points from the morning’s Plenary and the Spotlight sessions on addressing health equity in health economics and outcomes research. Participants are invited to share their thoughts and research experiences and explore ways to engage with the SIG to further these interests.
Moderator
Caroline Jacobsen, MPhil
Boston Scientific, Minneapolis, MN, USA
Stacey Kowal, BS, MSc
Genentech, Inc., Alameda, CA, USA
I am a global health economics and outcomes research (HEOR) expert with more than 15 years of experience in healthcare research and team leadership, including study conceptualization and execution, team building/staff development, external stakeholder relationship cultivation, and business unit operations. As a researcher, I’m most passionate about using economic modeling and real world evidence to understand how new innovations impact patients and their families, as well as broader stakeholders in the healthcare system. My current research uses a range of HEOR tools (economic modeling, real world data, patient reported outcomes) to identify and advance methods to improve our ability to measure and communicate value as it relates to health equity, precision medicine, health policy and health technology assessment.
3:00 PM - 6:30 PM
In-Person and Virtual Poster Session 4
Live
In-person presenters will be with their posters from 5:30 – 6:30PM.
3:45 PM - 4:15 PM
HEOR Theater
Transforming Real-World Data into Insights That Drive Value
In-person
The explosion of real-world data sources presents opportunities for new data like never before, but it also can be daunting to know which data are the right data for your specific needs. Starting with a clear understanding of the research question is key to any good product evidence generation strategy, and your data needs change throughout the lifecycle. A variety of data sources, including both trial and real-world data, are needed to inform strategic decisions. The use of a centralized data ecosystem and application of data science can support data mining or pooled analyses. By applying advanced scientific methodologies to generate and analyze data, insights can be derived to address multiple research questions, as well as identify evidence gaps, generate new hypotheses, and inform ongoing evidence strategies.
Join our panel of experts as they discuss:
How to successfully approach and implement a real-world data strategy
The value a data lake can provide in data alignment, transformation, and access across multiple stakeholders
How to make data smarter
The importance of real-world data reliability and integrity
Why data are just data without the right scientific study design and statistical analysis to produce actionable insights
Sponsor
Evidera, a PPD business
Speaker
Ariel Berger, MPH
Evidera, a PPD business, Waltham, MA, USA
Nikhil Desouza, PhD
Evidera, a PPD business, Wilmington, NC, USA
David L. Van Brunt, PhD, MS, BA
Evidera, a PPD business, Libertyvile, IL, USA
4:00 PM - 4:30 PM
Break
In-person
4:30 PM - 5:30 PM
Podium Session 6
Clinical Trial Methods and Applications in HEOR
Virtual
Moderator
Robert McQueen, PhD
University of Colorado, Aurora, CO, USA
R. Brett McQueen is an Assistant Professor at the University of Colorado (CU) Skaggs School of Pharmacy and Pharmaceutical Sciences, and member in the Center for Pharmaceutical Outcomes Research. His research interests include decision-analytic modeling applications and methodology, applied microeconometrics in health, and novel value assessment methods. Brett has current funding in micro-costing health interventions, evaluating performance-based risk sharing agreements, estimating patient and payer preferences for various pharmaceuticals, and novel value assessment methods. He is the course director for “Pharmaceutical Economics and Policy Analysis” in the Pharmaceutical Outcomes Research PhD program at CU.
P30: Combining Real-World and Randomized Controlled Trial Survival Data Using Bayesian Methods
4:30PM - 4:45PM
Xia Z 1 , Sheinson D2 1 University of Washington, Kirkland, WA, USA, 2 Genentech, LA JOLLA, CA, USA
OBJECTIVES: Randomized controlled trials (RCTs) are the gold standard for clinical evidence and are routinely incorporated into health economic models. However, there are practical and scientific reasons for why observational studies may be preferred to inform model extrapolation in health technology assessment (Zhao, 2016; Ligthelm, 2007; MacLehose, 2000; Ioannidis, 2005; Concato, 2000). This study explored a Bayesian framework for combining information from RCTs and real-world data (RWD) in health economic models, allowing for flexible weighting of the two data sources via prior information.
METHODS: Bayesian exponential survival models were fit to overall and progression-free survival data from a real-world cohort of alectinib- or crizotinib-treated ALK-positive (ALK+) advanced non-small cell lung cancer patients selected from the Flatiron Health electronic health record-derived deidentified nationwide longitudinal database. Posterior mean and 95% credible interval (CrI) estimates were generated for hazard ratios (HRs) and incremental cost-effectiveness ratios (ICERs) comparing alectinib versus crizotinib under different weightings of prior information from RCTs and different cut points of RWD accumulation.
RESULTS: The study included 463 ALK+ patients who were treated with alectinib or crizotinib. Estimated HRs ranged from 0.63 (95% CrI: 0.46-0.85) to 0.71 (0.61-0.83) using uninformative (i.e. not informed by RCT data, only by RWD) to strongly informative priors, while estimated ICERs were ~8% higher when using strongly informative versus uninformative priors. When using strong prior information, model estimates stabilized after 80 alectinib patients were accumulated from RWD. In contrast, when using an uninformative prior, model estimates stabilized after 183 alectinib patients were accumulated from RWD.
CONCLUSIONS: A Bayesian estimation framework allows for controlling the level of borrowing between RCT and RWD according to one’s prior belief. If sample size from RWD is small, adding prior information from RCTs can provide more stable estimates, whereas prior assumptions may be relaxed as more RWD is accumulated.
P29: Comparing G-Computation, Propensity Score-Based Weighting, and Targeted Maximum Likelihood Estimation for Analyzing Externally Controlled Trials with an Unmeasured Confounder: A Simulation Study
4:45PM - 5:00PM
Ren J 1 , Cislo P2 , Cappelleri JC3 , Hlavacek P2 , DiBonaventura M2 1 Pfizer Inc, Collegeville, PA, USA, 2 Pfizer Inc, New York, NY, USA, 3 Pfizer Inc., Groton, CT, USA
OBJECTIVES: In orphan and rare diseases, single-arm trials are common given the impracticability, if not impossibility, of randomized controlled trials. In these settings, an external control (EC) can be employed to compare against the single-arm trial to estimate treatment effects, though minimizing potential biases to interpret these effects is critical. We sought to compare different methods for causal inference in simulated data sets with measured (included from the model) and unmeasured (excluded) confounders.
METHODS: In the simulated data, three types of outcomes (continuous, binary, and time-to-event) were compared between trial and EC arms. Two measured baseline covariates (confounders) were unbalanced between the two arms. For each outcome, the two scenarios for relationship between unmeasured confounder and observed variables were: A) directly associated with one baseline covariate and the outcome; and B) directly associated with one baseline covariate, the outcome, and treatment assignment. In 3,000 random samples (100 or 200 patients per sample), we used g-computation, propensity score-based weighting, and targeted maximum likelihood estimation (TMLE) to estimate treatment effects based on observed outcomes and measured covariates.
RESULTS: In scenario A, the average estimates from all proposed methods were similar to the true effect (e.g. 1.14 to 1.19 vs 1.15 for the binary outcome), but g-computation had the smallest mean squared error (MSE), as well as a reasonable coverage (0.91-0.94) as measured by 95% confidence interval in different settings. In scenario B, most of results were similar with those in scenario A, except in one setting of continuous outcome where the estimates were more discrepant from the true effect.
CONCLUSIONS: Treatment effects estimated by g-computation, propensity score-based weighting, and TMLE appear to be reasonable for EC trials, even if confounders are not completely measured. Of the proposed approaches, evidence suggests that g-computation is a preferable approach producing relatively unbiased estimates.
P31: Feasibility of Using Oncology Specific Electronic Health Records (EHR) Data to Emulate Clinical Trial Inclusion and Exclusion Criteria
5:00PM - 5:15PM
Wilson T 1 , Dye J2 , Spark S1 , Bian J1 , Amirian ES1 , Espirito J1 , Robert N3 1 Ontada, The Woodlands, TX, USA, 2 Ontada, Atlanta, GA, USA, 3 Ontada, Irving, TX, USA
OBJECTIVES
: Prior studies have attempted to ascertain the feasibility of using EHR to emulate data elements necessary in clinical trials. However, few studies have focused on assessing the utility of both
structured and
unstructured (i.e., abstracted) data from
oncology specific EHR systems for such purposes. This study examined critical elements of recent oncology clinical trials to assess the degree to which these real-world data (RWD) can be reasonably used to retrospectively replicate the information needed in trials.
METHODS
: FDA approvals of oncology drugs in 2020 were identified and matched to trial data from the Aggregate Analysis of ClinicalTrials.gov (AACT) database (downloaded 12/17/2021). Inclusion and exclusion criteria in these trials were tabulated from AACT, and the degree to which they could be ascertained as structured data (available in a database, such as age, sex, diagnosis, stage, treatment, biomarkers, etc.) and/or unstructured data (such as free text fields, scanned documents, outside diagnostic reports, etc.) in an oncology specific EMR system were qualitatively assessed by experienced investigators.
RESULTS
: Among the 53 trials identified, 20 were phase III studies and had necessary information available in AACT. Of the 459 eligibility criteria reviewed, the median per study was 21 (range 7-51). Median inclusion was 8.5 (range 2-31) and exclusion was 12 (range 2-25). Overall, 81% of eligibility criteria was likely available in the oncology EHR (50% in structured and/or unstructured and 31% in unstructured only). Approximately 19% of criteria was not generally available in EHRs outside of a clinical trial (e.g., signed informed consent, agreements for use of birth control, etc.)
CONCLUSIONS
: Oncology-specific EHR data can be leveraged to emulate approximately 80% of inclusion/exclusion criteria of clinical trials. However, it is important to differentiate eligibility criteria that are available as part of real-world patient care versus those applied specifically for the purpose of a trial.
P32: Power Implications of Estimator Choice in Synthetic Control Arm Analyses: Results from a Simulation Comparing Average Treatment Effects on the Treated and Untreated Under Propensity Score Weighting
5:15PM - 5:30PM
Mackay E
OBJECTIVES: Synthetic control arm (SCA) methods are seeing increased use in comparative effectiveness research. SCAs compare single-arm trials against external control arms constructed from real-world data (RWD) where conducting randomized clinical trials is infeasible. In rare diseases sample sizes are generally severely limited in both single-arm trial and RWD, presenting design challenges. Often practitioners estimate the average treatment effect on the treated (ATT) via inverse probability of treatment weighting (IPTW). However, ATT may not always maximize power and, depending on cohort relative sample sizes, estimators like average treatment effect on the untreated (ATU) could outperform. Our objective is to compare power for these estimators across a range of scenarios in which SCA methods are typically applied.
METHODS: We compare ATT vs. ATU power under IPTW via simulation. We simulate survival data under an exponential survival model where the hazard rate depends on treatment and a binary prognostic variable which is imbalanced between cohorts. We estimate propensity scores conditional on the prognostic variable and compute ATT and ATU using IPTW via weighted Cox regression. We use two-sided tests with significance level 5% and robust standard errors. We vary sample sizes separately in each cohort and compare power for the ATT and ATU. We assume a hazard ratio of 0.6 favouring treatment in the single-arm trial.
RESULTS: With 100 patients in both trial and RWD, power is 53.7% for ATT vs. 57.8% for ATU. Increasing RWD sample size to 400, the gap widens to 79.4% vs. 71.9%. It widens even further to 71.5% vs. 87.1% with 400 trial patients and 100 in RWD.
CONCLUSIONS: The power simulation results demonstrate that selection of ATT vs. ATU estimators can have meaningful impacts on SCA power. When designing SCAs, estimator choices should consider anticipated relative sample sizes in single-arm trial and RWD to preserve power.
Economic Evaluation of Disease Burden Using Real-World Data
Virtual
Moderator
Xiao Xu, PhD
Yale University, New Haven, CT, USA
Xiao Xu, PhD, is an associate professor in the Department of Obstetrics, Gynecology and Reproductive Sciences at Yale School of Medicine. As a health economist and health services researcher, her work focuses on identifying factors that influence the quality and utilization of women’s health services. Her recent studies examined hospital and regional variation in healthcare delivery, costs, and patient outcomes; comparative effectiveness and cost effectiveness of health interventions; and health and healthcare disparities. Her research has been funded by the National Institutes of Health, Agency for Healthcare Research and Quality, and various research foundations.
P41: Utilization and Spending Trends in Medicare Part D Antidiabetic Drugs, 2015-2019
5:15PM - 5:30PM
Sistani F 1 , Shaya FT1 , Rodriguez de Bittner M2 1 University of Maryland Baltimore, Baltimore, MD, USA, 2 University of Maryland School of Pharmacy, Baltimore, MD, USA
Objectives: This study aims to highlight the substantial economic burden of Antidiabetics on Medicare Part D. Methods: We analyzed the de-identified data from Medicare Drug Spending Dashboard and utilization dataset from 2015 to 2019. We identified 10 classes of common antidiabetic medications and described the Medicare Part D total spending, utilization, and relative annual changes on those medications. Furthermore, we described the spending by manufacturers and compared the spending on generic and brand antidiabetics during this time frame. Results: Medicare Part D total spending on antidiabetics was $124 billion with 56% growth from 2015 to 2019. Approximately, 95% of this spending was on single-agent medications. While insulin remained the largest source of total spending on antidiabetics every year, non-insulin antidiabetics, such as Dipeptidyl peptidase 4 inhibitors (DPP4i), glucagon-like peptide-1 (GLP-1) receptor agonists, and sodium glucose co-transporter inhibitors (SGLT2i) respectively accounted for 12%, 17% and 6% of spending. Medicare Part D antidiabetics utilization increased from 83.2 million prescriptions in 2015 to 90.9 million prescriptions in 2019. While utilization of older agents such as metformin and sulfonylureas remained relatively constant over the study period, numbers of prescriptions for newer combination agents such as SGLT2i- DPP4i (1147% growth) and SGLT2i-metformin (457%) increased significantly over the same time frame. Most of Medicare Part D total spending on antidiabetics was on brand medications (96%) and the top 4 highest spending was on antidiabetics manufactured by Novo Nordisk, Sanofi-Aventis, Eli Lilly and Company, and Merck Sharp & Dohme Corp. Conclusions: Between 2015 and 2019, on average, $25 billion per year was spent by Medicare Part D on antidiabetics. Physicians’ adoption of newer brand antidiabetics, in part, plays a role in Medicare Part D spending. Physicians should work to ensure appropriate guideline-recommended treatment for patients with diabetes.
P42: Direct and Indirect Costs Associated with Major Depressive Disorder
4:30PM - 4:45PM
Culpepper L1 , Higa S2 , Martin A3 , Gillard P4 , Parikh M 5 , Harrington A4 1 Boston University School of Medicine, Westport, MA, USA, 2 AbbVie, Seattle, WA, USA, 3 Cerner Enviza, North Kansas City, Burlingame, CA, USA, 4 AbbVie, Irvine, CA, USA, 5 AbbVie, Madison, NJ, USA
OBJECTIVES:
Major depressive disorder (MDD) is a disabling mental health condition with significant economic burden. This study evaluated direct and indirect healthcare costs for patients with MDD and cost differences across MDD severity levels.
METHODS:
Adults (
≥ 18 years) with a self-reported physician diagnosis of depression on the 2019 National Health and Wellness Survey were stratified by disease severity using Patient Health Questionnaire (PHQ-9) scores: mild≤9, moderate=10-14, moderate-severe=15-19, severe≥20. The comparison population consisted of participants representing the general US population without MDD, bipolar I disorder, or schizophrenia. Outcomes included estimated direct costs associated with self-reported healthcare resource utilization (healthcare professional [HCP] visits, emergency room [ER] visits, hospitalizations) and estimated indirect costs associated with work productivity loss. Cost outcomes (reported as annualized mean estimates) were compared between MDD and general population cohorts, and across MDD severity subgroups via multivariate analyses, adjusting for key baseline differences.
RESULTS:
There were 10,710 participants in the MDD cohort (severity: mild=5905, moderate=2206, moderate-severe=1565, severe=1034). In contrast to the general population (N=52,687), adults with MDD had significantly higher 12-month total direct costs ($8814 vs $6072;
P <.001), driven by higher costs for HCP visits ($3571 vs $1662;
P <.001), ER visits ($559 vs $366;
P <.001), and hospitalizations ($4408 vs $3495;
P =.007). Total 12-month indirect costs were significantly greater in the MDD population versus the general population ($5425 vs $3085;
P <.001). Increased MDD severity was associated with significantly increased 12-month total direct costs (mild=$8220; moderate=$10,353; moderate-severe=$10,819; severe=$12,433; all
P <.05 vs mild) and total indirect costs (mild=$4490; moderate=$6537; moderate-severe=$7438; severe=$8797; all
P <.001 vs mild).
CONCLUSIONS:
Patients with MDD had significantly higher direct and indirect healthcare costs relative to the general population. Compared to patients with mild MDD, patients with greater severity had significantly higher total direct and indirect costs. Results underscore the need for effective MDD treatment regimens to decrease disease severity.
P43: The Economic Burden of Idiopathic Pulmonary Fibrosis in Australia
5:00PM - 5:15PM
Cox I 1 , de Graaff B2 , Ahmad H2 , Campbell JA2 , Otahal P3 , Corte TJ4 , Walters HE2 , Palmer AJ2 1 Menzies Institute for Medical Research, University of Tasmania, Hobart, TAS, Australia, 2 University of Tasmania, Hobart, TAS, Australia, 3 University of Tasmania, Hobart, NSW, Australia, 4 The University of Sydney, Sydney, NSW, Australia
Background Idiopathic pulmonary fibrosis (IPF) is form of interstitial lung disease which generally occurs in persons 60 years and older.It is characterised by a high symptom burden, and frequent encounters with health services. This study aimed to determine the economic burden of IPF in Australia with a focus on resource utilisation and associated direct costs. Methods Participants were recruited from the Australian IPF Registry (AIPFR) between August 2018 and December 2019. Data on resource utilisation and costs were collected via cost diaries and linked administrative data. Clinical data were collected from the AIPFR. Costing was performed from a partial societal perspective and a “bottom up” costing methodology was utilized focusing on direct medical and non-medical costs. Costs were standardized to 2021 Australian dollars ($). Results The average annual total direct costs per person with IPF was $31,655 (95% confidence interval: $27,723-$35,757). Extrapolating costs based on prevalence estimates, the total annual costs in Australia are projected to be $299 million (95% confidence interval: $262 million -$338 million) which is based on published prevalence for IPF. Costs were mainly driven by antifibrotic medication, hospital admissions and medications for comorbidities. Disease severity, comorbidities and antifibrotic medication all had varying impacts on resource utilisation and costs. Conclusion This study provides the first comprehensive analysis of IPF-related direct costs in Australia, identifies the key cost drivers and provides a framework for future health economic analyses. Additionally, it provided insight into the major cost drivers which include antifibrotic medication, hospital admissions and medications related to comorbidities. Our findings emphasise the importance of the appropriate management of comorbidities in the care of people with IPF as this was one of the main reasons for hospitalisations.
P44: Multiple Myeloma Total Direct Costs in Colombia: A National Cohort Study Based on Administrative Claims Databases
4:45PM - 5:00PM
Patiño Benavidez A1 , Buitrago G2 , Torres G 3 1 Universidad Nacional de Colombia, Bogotá, D.C., CUN, Colombia, 2 Universidad Nacional de Colombia, Hospital Universitario Nacional de Colombia, Bogotá D.C., Colombia, 3 Universidad Nacional de Colombia, BOGOTA, CUN, Colombia
Background: Multiple myeloma (MM) is associated with a substantial economic burden for health systems worldwide, however, evidence regarding this burden in middle-income countries is lacking. Colombia, a middle-income country, provides healthcare services included and not included in its Health Benefit Package (HBP and non-HBP services, respectively) to its population through several insurance regimes. Almost 48% of Colombian population receive healthcare services through the contributory regime. This study aims to describe the costs of healthcare services delivered to patients with MM affiliated with the contributory regime in 2018. Methods: We conducted a cost-of-illness study from the perspective of the Colombian Health System that included all direct costs generated by prevalent cases of MM during 2018. We identified patients with MM through a case-selection validated electronic algorithm. The costs of HBP services were calculated using the Capitation Sufficiency Database, an administrative database that contains patient level data on consumption of HBP services in the contributory regime. The costs of non-HBP services were calculated using aggregated data from the MIPRES database. Costs were deflated to 2020 Colombian pesos and were expressed in 2020 international dollars (INT$). Results: We identified 2,132 patients with MM. Total costs were INT$188 million, with 75%(INT$141.3 million) of these costs caused by consumption of non-HBP services. Median costs for women were INT$88,312 (25%-75% IQR INT$82,526 to INT$105,079), and for men were INT$89,208 (25%-75% IQR INT$82,598 to INT$105,057). Median costs for patients with Charlson Comorbidities Index score ≤2 were lower than median costs for patients with a CCI score ≥ 5 (INT$86,557, 25%-75% IQR INT$81,826 to INT$98,348 versus $INT92,366, 25%-75% IQR INT$84,480 to INT$110,671, respectively). Conclusions: MM poses a substantial economic burden for the Colombian Health System with Non-HBP services accounting for the largest proportion of direct costs of care. Higher CCI scores are associated with increased costs of care.
Concurrent Breakout Session 6
Combining Real-World and Clinical Trial Data to Study the Effectiveness of Thrombolytics for Treating Patients with COVID-19 Associated Acute Respiratory Distress Syndrome
In-person & Virtual
Level: Intermediate
PURPOSE: To demonstrate the challenges of conducting randomized controlled trials to study treatments for hospitalized patients with COVID-19 during the pandemic and illustrate how real-world data (RWD) can help address these challenges and supplement clinical trial results.
DESCRIPTION: Pandemic conditions present multiple barriers to the study of investigational drugs, including challenges in site selection and recruitment, identification of suitable control arms for comparison, and ability to detect small effects in noisy clinical outcome data[1] ,[2] ,[3] . An analysis of COVID-19 clinical trials showed that the majority of trials for COVID-19 therapeutics were not designed to yield actionable information due to low randomization rates and underpowered outcome data[4]
In this workshop, we will describe the design, operation, and analyses of a multicenter cohort study that combined data from the Phase 2a STARS study (NCT04357730) and data from the electronic health records of patients treated outside of the trial. We will share the challenges faced including operational hurdles and patient enrollment, the collaborative effort involved in clinical operations (i.e. team science), how to integrate real-world patients into study design elements such as inclusion/exclusion criteria and primary/secondary endpoints, and methodology used to incorporate RWD into the analysis and interpretation of results. [1] Jama 323.22 (2020): 2262-2263.
[2] The Lancet 395.10236 (2020): 1525-1527.
[3] Nature Reviews Drug Discovery 19.10 (2020): 662-664.
[4]
Discussion Leaders
Mitra Corral, BS, MPH, MS
Genentech, South San Francisco, CA, USA
Mitra Corral, MS, MPH has been a Principal Health Economist at Genentech. She earned her BS in Actuarial Science from New Jersey Institute of Technology; an MS in Statistics from Rutgers University; and an MPH in Epidemiology from University of Medicine & Dentistry of New Jersey. She has extensive experience in Health Economics and Outcomes Research having worked in Managed Care, Pharmaceutical and Medical device organizations.
Janice Wang, MD, FCCP
Northwell Health Department of Medicine, Port Washington, NY, USA
Dr. Janice Wang is Director of the Adult Cystic Fibrosis Center and Therapeutic Development Network Center in the Division of Pulmonary, Critical Care and Sleep Medicine at Northwell Health. She has led investigator-initiated and industry-sponsored clinical trials related to COVID-19 and cystic fibrosis therapeutics. These included the investigational use of tissue plasminogen activator in COVID-19 patients with acute respiratory distress syndrome, and the use of prophylactic versus therapeutic dose of anticoagulation for COVID-19 patients. She is also a Certified Principal Investigator from the Association of Clinical Research Professionals.
Discussants
Marquita Decker-Palmer, MD, MPH, PhD
Genentech Inc., South San Francisco, CA, USA
Physician, clinical and translational researcher with a focus in health economics and outcomes
Daniel Sheinson, PhD
Genentech, La Jolla, CA, USA
Rongrong Wang, MPH
Genentech, Inc., South San Francisco, CA, USA
Ms. Wang is an interdisciplinary data scientist with expertise in epidemiology, biostatistics, health and economics outcome research, and public health. She has over 7 years of experience in using real-world data and designing observational research studies to support the value and access of drug products as well as healthcare policy and innovation. Ms. Wang earned a bachelor’s degree in medicine from Sun-Yat Sen University and a master’s degree in Public Health from Yale University. She also obtained a Graduate Certificate in Data Mining and Application from Stanford University.
High Quality of Life Against All Odds? Evaluating Quality of Life Measurement Approaches in Degenerative Diseases Affecting Children and Young Adults
In-person & Virtual
Level: Intermediate
PURPOSE: The objective is to characterize issues around the assessment and valuation of health for children and young adults with degenerative diseases, delineate issues for HTA, and to generate discussion around best practice recommendations.
DESCRIPTION:
The estimation of QALYs for use in HTA relies on utility generated from HRQoL tools and the extent to which the HRQoL tools adequately capture the impact of the condition.
People with degenerative disease may place different weights on certain aspects of quality of life, because (1) they have never experienced a ‘full’ quality of life similar to healthy individuals, and (2) they experience health changes that lead to shifts in internal standards (i.e., ‘recalibration’), values (i.e., ‘reprioritization’) and conceptualization (i.e., ‘reconceptualization’) of key HRQoL domains as they are aware that their condition will deteriorate.
There is concern that patient-reported HRQoL impacts in degenerative diseases may be underreported due to adjusted expectations and disease state adaptation. This may mean that from the perspective of a caregiver/clinician (or other external observer,) the impact of the condition on HRQoL may be considered more significant.
There are further concerns the EQ-5D and other generic preference-based measures do not adequately capture the main health benefits of interventions and QALY impacts may lack sensitivity in circumstances where other measures and testimony suggest health impacts.
To address the workshop aims, we will include presentations and discussion that address:
The challenges faced with degenerative diseases and whether the domains adequately capture the impact of the condition.
An overview of patient vs. general public vs. alternative perspectives with reference to current HTA guidance and practice. Further consideration for how generic/ condition-specific measures and other evidence can be developed and used in combination to overcome challenges.
An overview of how HTA bodies have evaluated and addressed such approaches/concerns in past/how HTA decisions have been impacted.
Discussion Leaders
Andrew Lloyd, DPhil
Acaster Lloyd Consulting Ltd, London, United Kingdom
Andrew Lloyd is a Director of Acaster Lloyd Consulting Ltd. His background is in patient reported outcomes research, with a focus on patient preferences / utilities. Andrew was the VP of the PRO Group at ICON PLC. He previously held research posts at several universities within the UK. Andrew is an Honorary Professor at the London School of Hygiene and Tropical Medicine. He is a former co-editor of Value in Health. He is a board member of the EuroQol Group.
Discussants
Joel Iff, PharmD, PhD
Sarepta Therapeutics Inc, Cambridge, MA, USA
Antony Martin, BSc (Hons), MSc, PhD
QC Medica, York, NYK, United Kingdom
Antony has a background in Health Economics and completed his PhD at the University of Liverpool funded by the NIHR. He has extensive experience working with leading HEOR consultancies and in academia. He has also acted as a specialist advisor for NICE and NIHR HTA programmes.
Antony's research spans evidence generation and synthesis with a focus on patient-centred research and observational studies. He has experience working within a range of health areas including oncology and across several rare diseases. In addition, he publishes widely in scientific journals and often acts as a journal reviewer for HEOR studies.
Antony has developed a keen interest in health equity research and is motivated by promoting patient access to innovative therapies.
A. Simon Pickard, PhD
University of Illinois at Chicago, Chicago, IL, USA
A. Simon Pickard, PhD, is a Professor and Director of Graduate Studies in the Department of Pharmacy Systems, Outcomes and Policymat the University of Illinois Chicago College of Pharmacy. Dr. Pickard’s research focuses on evaluating the safety, effectiveness and value of health care interventions; improving drug-related outcomes through education (particularly opioids); and on the measurement and evaluation of quality of care and health outcomes. He has extensively contributed to the EuroQol Group, and served on the Executive Committee from 2006-2020 (Chair 2014-17).
National Institutes of Health Small Business and Commercialization Grants: You Too Can Access Funding
In-person
Level: Foundational
PURPOSE: The National Institutes of Health (NIH) comprises 27 components that support activities impacting health. The NIH’s National Heart, Lung, and Blood Institute (NHLBI) and the National Institute on Aging (NIA) promote the prevention and treatment of disease by supporting discovery research and translational activities to move innovations from bench to marketplace. The purpose of this workshop is to increase awareness of the NIH’s Small Business Innovation Research (SBIR), Small Business Technology Transfer (STTR), and proof of concept programs [NIH Centers for Accelerated Innovations (NCAI), Research Evaluation and Commercialization Hubs (REACH), and Catalyze] and how the science of HEOR supports innovation, fundraising and commercialization of technologies by entrepreneurs, small businesses, academics, scientists and consultants.
DESCRIPTION: Dr. Laura Pizzi will introduce the panelists and discuss the overall challenges and opportunities related to obtaining NIH funding for HEOR (5 minutes), Dr. Rousche will describe the different NIH programs and resources available to innovators (10 minutes), Dr. Arnold will describe the various areas of coaching (intellectual property protection, regulatory pathways, health economics and outcomes research, reimbursement, marketability, and investment) available to successful entrepreneurs within NHLBI’s Innovation Office (10 minutes), and Dr. Jutkowitz will provide suggestions on NIH grantsmanship and strategies towards attaining funding (15 minutes). Lastly, Dr. Arnold will lead a critique of an NIH awardee’s video pitch presentation at an event focused on helping companies find private sector partners and support (12 minutes), followed by a short audience Q&A (8 minutes). Through this workshop, audience members will gain an understanding of the NIH’s small business programs and product development resources, and how to access the NIH for non-dilutive funding.
Discussion Leaders
Laura Pizzi, PharmD, MPH, RPh
Rutgers University, Piscataway, NJ, USA
Dr. Laura Pizzi is Associate Chief Science Officer for ISPOR and Professor at Rutgers University in the schools of pharmacy and public health. For the past 25 years, she has led interdisciplinary teams of methodologists, statisticians, and clinicians to design and conduct economic analyses on healthcare interventions and is a frequent author, speaker, and mentor on the topic.
At ISPOR, she provides leadership to the organization’s scientific strategy and initiatives, including content planning and oversight of the Special Interest Groups, Patient Council and roundtables, the ISPOR Competency Framework workgroup, Publications Council, Institutional Council, and Digital Health Strategy. She also liaises with the Student Network and Faculty Advisor Council to support their scientific needs.
Discussants
Renée Arnold, PharmD, RPh
National Institutes of Health, New York, NY, USA
Renée JG Arnold is currently Entrepreneur-in-Residence (EIR), HEOR, NHLBI (NIH) and EIR, Biohealth Innovation, Inc, New York, NY; Adjunct Full Professor, Master of Public Health Program, Department of Environmental Medicine and Public Health, Icahn School of Medicine at Mount Sinai, New York, NY, USA, where she has developed and teaches the pharmacoeconomics coursework. She is also president & CEO of Arnold Consultancy & Technology, LLC where she oversees outcomes research and develops affiliated software for pharmaceutical and federal government programs. Her special interest in evidence-based health derives from her research that deals with use of technology to collect and/or model real-world data for use in rational decision making by healthcare practitioners and policy makers. Dr. Arnold completed her undergraduate training at the University of Maryland and received her Doctor of Pharmacy degree from the University of Southern California in Los Angeles. She also completed a one-year post-doctoral residency at University Hospital in San Diego/University of California at San Francisco School of Pharmacy. Dr. Arnold was previously vice president of HEOR at Quorum Consulting, Inc./Navigant Consulting; principal of IMS Health (IQVIA); and president and co-founder of Pharmacon International, Inc. Center for Health Outcomes Excellence.
Dr. Arnold is a founding member of ISPOR and is the head of both the ISPOR Distance Learning Program, as well as the head of the newly-developed Open Source Models Special Interest Group. She is an author/co-author of numerous articles, book chapters, and books in the areas of pharmacology, pharmacoeconomics, and cost-containment strategies.
Eric Jutkowitz, PhD
Brown University, Providenice, RI, USA
Kathleen Rousche, PhD
National Institutes of Health, Bethesda, MD, USA
Women in HEOR Session: Strengthening Communication Practices
In-person
Level: Foundational
Our keynote speaker will be Sheila Cort, Senior Director & Coach and Head of Taft ClearPoint at Taft Communications. She will focus on leading through your communications by strengthening your communication and presentation skills and executive presence, as well as providing essential tips for daily communications success. This session will be hosted by Julia F. Slejko, PhD co-lead of ISPOR’s Women in HEOR initiative and features interaction throughout and provides time for audience Q&A. The session is open to all ISPOR 2022 registrants. To learn more about ISPOR’s Women in HEOR initiative, the evidence for diversity as a performance issue, and the Society’s progress in its diversity initiatives, visit
www.ispor.org/womeninheor .
Moderators
Julia F. Slejko, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
Julia F. Slejko, PhD is an Associate Professor of Pharmaceutical Health Services Research at the University of Maryland School of Pharmacy and is Co-Director of the Patient-Driven Values in Healthcare Evaluation (PAVE) Center. Dr Slejko’s research is focused on innovative approaches for decision-analytic modeling for economic and health outcomes assessments. She holds a BA in Molecular, Cellular, and Developmental Biology from the University of Colorado Boulder. During her PhD training, she focused on pharmacoeconomics at the University of Colorado School of Pharmacy Center for Pharmaceutical Outcomes Research. Her postdoctoral training was completed at the Pharmaceutical Outcomes Research and Policy Program in the University of Washington School of Pharmacy. Prior to her PhD training, she had a 7-year career in drug discovery at Array BioPharma. Dr Slejko is co-lead of ISPOR’s Women in HEOR initiative and currently Co-Chair Elect of the ISPOR Faculty Advisor Council.
Discussion Leaders
Sheila Cort, BA
Taft Communications, Lawrenceville, NJ, USA
Getting Real with Real World Evidence for Regulatory Decision Making: Where Are We Going?
In-person
Level: Intermediate
Purpose: Explore opportunities and lessons learned for using real-world data (RWD) to generate real-world evidence (RWE) in the support of benefit:risk assessments for regulatory decisions. Overview/Description: Real World Data (RWD) may provide supplemental information when RCTs are not feasible, ethical, or include only a small number of patients (e.g. rare diseases, rare oncology subtypes, pediatrics). Generating RWE may enhance understanding of long-term outcomes, generate external controls to support single-arm trials, and support label expansion to new indications or biomarkers. While RWD holds great promise, it is subject to considerable set of unique biases and other limitations which are now highlighted even more during the massive health system related disruptions in the COVID-19 era. These disruptions could impact the design and interpretation of future studies using RWD sources and highlight the need to understand data capture and characterize RWD before using it to generate RWE. This workshop will focus on an evaluating current progress, lessons learned, and future opportunities for RWD utilization. Dr. Rivera will provide an overview of the current global regulatory landscape and demonstrate new applications of RWE to support regulatory decision making. Dr. Burcu will describe methodological challenges in the design of fit-for-purpose observational research. Dr. Lederer will provide an overview on the assessment of validity and performance of real-world endpoints and lessons learned. Dr. Oehrlein will illustrate the importance of patient perspective on the evolving use of RWE rising to meet public health challenge. There will be allocated 15 minutes of interactive panel discussions responding to audience questions and comments.
Speakers
Mehmet Burcu, PhD, MS
Merck & Co., Inc., Kenilworth, NJ, USA
Nirosha Mahendraratnam Lederer, PhD, MSPH
Aetion, Washington, DC, USA
Nirosha Mahendraratnam Lederer, PhD is Head of US Federal Government and Senior Director of Real-World Evidence Strategy at Aetion. In this role, she leads partnership opportunities with the US federal government and advises clients on real-world evidence trends. Dr. Lederer has over 15 years of research experience using real-world data to support high-value decision-making in the US healthcare system. Before joining Aetion, she led the real-world evidence portfolio at the Duke Margolis Center for Health Policy including developing policies and strategies for increasing the usability and acceptance of RWD and RWE for regulatory and payment decision-making. She previously served as Subject Matter Expert in Patient-Focused Drug Development at the US FDA Oncology Center of Excellence where she supported the use and evaluation of novel drug development tools. Prior to FDA, Dr. Lederer worked at Avalere Health as Manager of Health Economics and Outcomes Research / Evidence-based Medicine Policy. In this capacity, she created global evidence generation programs as well as led policy development and government engagement strategies for Fortune 100 healthcare companies. Earlier, Dr. Lederer served on Capitol Hill with the House Committee on Ways and Means Subcommittee on Health during the passage of the Affordable Care Act.
She received her PhD in Health Outcomes and Policy from the UNC Eshelman School of Pharmacy. She also received her MSPH in Health Policy and Management from the Johns Hopkins Bloomberg School of Public Health and BA in Public Health from the Johns Hopkins University.
Donna Rivera, PharmD, MSc
US Food and Drug Administration, Silver Spring, DC, USA
Discussion Leaders
Elisabeth Oehrlein, PhD, MS
Applied Patient Experience, LLC, Washington, DC, USA
Elisabeth M. Oehrlein, Ph.D., MS, is Assistant Vice President, Research & Programs, at the National Health Council, joining the organization in July 2018. Dr. Oehrlein is a mixed-methods researcher with expertise in value/health technology assessment, outcomes research, and patient-focused medical product development. Her research interests include patient journey/experience mapping and applying patient experiences when developing real-world research to ensure studies reflect the “real world” as closely as possible. She is an active member of HTAi’s Patient and Citizen Involvement Group, as well as the International Society for Pharmacoeconomics and Outcomes Research, where she holds leadership roles in the Patient-Centered and Real-World Evidence Special Interest Groups. She has published widely in medical, economic, and health policy journals and serves as an Associate Editor of Value in Health.
Dr. Oehrlein holds a BA from Franklin & Marshall College, an MS in Epidemiology from the University of Maryland School of Medicine’s Department of Epidemiology and Human Genetics, and a Ph.D. in Pharmaceutical Health Services Research from the University of Maryland School of Pharmacy.
How Can HTA Become Truly Participatory? Implementing the Guidance of the Joint HTAI – ISPOR Task Force Deliberative Processes for HTA
In-person
ISSUE: Deliberative processes are intended to improve the quality of recommendations and decision making by allowing for participatory decision making, drawing on different kinds of participants, at different points of a decision-making cycle based on a consideration of facts and values. The HTAi-ISPOR task force – in close collaboration with the wider HTA community – has developed guidance for a deliberative process from an HTA perspective and a minimum set of considerations on the use of deliberative processes in HTA. Don Husereau will moderate and provide an overview of issues encountered by the Joint Task Force. Steve Pearson will provide a perspective of what is required practically and Mary Schrandt, and Newell McElwee will describe perspectives of clinicians/HTA bodies, patients, and the life sciences sector.
OVERVIEW: HTA organizations continue to improve processes including engagement with stakeholders. However, most HTA processes are still consultative and very few truly engage stakeholders. Panelists will discuss whether and how HTA processes can evolve to be become more deliberative. They will reflect on guidance from the Task Force to discuss its practical implications? Key questions that will be addressed include: Can guidance for HTA be a step forward? What else is needed to facilitate better participation? What can we learn from other areas? This session will provide an overview of practical challenges faced by HTA bodies, patients, industry and the research community in developing and implementing truly participatory processes in HTA.
Moderators
Don Husereau, BScPharm, MSc
University of Ottawa, Ottawa, ON, Canada
DON HUSEREAU is an Adjunct Professor of Medicine at The University of Ottawa. He has expertise in health economics and health technology assessment and works with private and public sector life sciences organizations to help them understand the value of health technology and its implications for health and innovation policy.
Panelists
Newell McElwee, PharmD, MSPH
Boehringer Ingelheim Pharmaceuticals, Inc., Ridgefield, CT, USA
Newell is Vice President, Health Economics and Outcomes Research at Boehringer Ingelheim Pharmaceuticals, Inc. He is a recognized leader in HEOR with 30 years experience in the pharmaceutical industry. Prior to coming to Boehringer Ingelheim, Newell led outcomes research groups at Pfizer and Merck. He has been actively engaged with a number of outcomes research related organizations, is a past ISPOR Board member and currently chairs the ISPOR Health Science Policy Council.
Newell received his PharmD from Mercer University and his MSPH (epidemiology) from the University of Utah. He completed a clinical pharmacy residency at Osteopathic Medical Center of Texas and a post-doctoral fellowship in clinical pharmacology and toxicology at the University of Utah Medical Center
Mary Suz Schrandt, JD
ExPPect, Arlington, VA, USA
Yvette Venable, BA
Institute for Clinical and Economic Review, Watertown, MA, USA
Yvette is the Vice President of Patient Engagement for the Institute of Clinical and Economic Review in the United States. In this newly created role, she leads the organization’s enhanced patient engagement program, serves as a dedicated resource to the patient community, and ensures that patient voices are driving ICER’s thinking and decisions.
Throughout her 20+ year career, Yvette has been a champion of strengthening the patient voice in health policy decision-making and health technology assessment. She spent 15 years in Europe working in global public affairs, advocacy, and patient access within life sciences companies, and has also held healthcare communications and market development roles with global consultancies in the USA.
The Challenges of Bringing Cell & Gene Therapies to Emerging Markets
Virtual
ISSUE:
Despite their promising clinical benefits, cell and gene therapies face a myriad of unique issues to guarantee their access and funding: the science is complex, administration requires capability building, and therapies have a high cost, as the treatment is sold in a onetime payment per unit that potentially provides superior benefits to therapies administered continuously but paid throughout years. The high upfront cost paired with durable clinical benefits raises concerns about their sustainability in the health care systems’ traditional funding models. These barriers are more pronounced in emerging markets given the lack of infrastructure and capabilities required to overcome the existent hurdles and the financial impact caused by the reimbursement of these new technologies. As cell and gene therapies expand to emerging markets, there is an urgent need to identify practical and sustainable solutions to allow patients in these markets to access innovative therapies. OVERVIEW:
In this panel, experts from different perspectives and geographies ranging from the ex-MoH experience in Turkey to the discussion of innovative contracting in the Brazilian public health system, will share their perspectives and experience on the challenges that come with the access and funding of cell and gene therapies given the challenges associated with the upfront costs and potential long term clinical benefits of cell and gene therapies. Valeria Boers Trilles will provide a short overview of the current cell and gene therapies landscape in emerging markets and will set the stage by posing key questions for the panelists to debate: What are the key learnings from prior experience in achieving access and funding of high-cost, high-value therapies in emerging markets? Considering the specificities of the challenges faced by emerging markets and based on the European Union and United States experiences; what would be sustainable solutions to unlock access to these therapies in emerging markets?
Moderators
Valeria Boers-Trilles, MPhil
Trinity Life Sciences, San Francisco, CA, USA
Ms. Boers is a Director in Trinity’s Value, Access & Pricing practice. Prior to joining Trinity, Ms. Boers was part of the CBPartners team and focused her experience on pricing and market access engagements, proving strategic support to optimize value communication, product positioning, IRP-based launch sequence and pricing opportunity across a range of therapeutic areas, including oncology, immunology and gastroenterology. She also supported non-asset specific engagements to inform client’s internal strategic planning and organization design based on market dynamics.
Ms. Boers has experience with various primary research methodologies, including in-depth interviews, expert advisory boards, and mock payer negotiations.
Ms. Boers speaks Spanish with native fluency and has developed her expertise within the Latin America region, as well as Southern Europe.
Panelists
Renata Hauegen, PhD
(Former) Fiocruz/MoH Brazil, rio de janeiro, Brazil
Health Director at Prospectiva, in charge of strategic projects in Public Affairs and Value Access in Health. Holds a doctorate in public policy from the Institute of Economics at UFRJ whith a thesis on risk sharing agreements and its feasibility to the brazilian public health system Founded CURIe projects and studies in access to health. Used to be the manager of the health innovation and partnership sarea of Fiocruz's health technology development center. Also led the legal department of Farmanguinhos/Fiocruz She has acted as an advisor on international projects such as the Global Virome Project.
Guvenc Kockaya, MD, MSc, PhD
ECONiX, Istanbul, 34, Turkey
Dr. Guvenc Kockaya is a medical doctor and health economist. He has a career of more than 15 years in market access & health economics with government, academia, and private industry experience. He is the editor of the books titled as “Pharmaceutical Market Access in Emerging Markets” and “Pharmaceutical Market Access in Developed Markets” which are still in the Top 100 Best Sellers (International) in Amazon under “Pharmaceutical & Biotechnology Industry (Kindle Store)”. He is the founder of ECONiX which is an internal company that gives tailor-made consultancy services including but not limited to market access, health economics & outcome research, medical affairs, and business development for government and academic institutes, pharmaceuticals, medical devices, and healthcare services companies in Eastern Europe, Middle East, North Africa, and Western Asia countries with offices in Estonia, Tunisia, and Turkey.
Sean Nagle, MS, MPH
Novartis, Miami, FL, USA
Current Head of Global Value, Access and Policy for Cardio Renal Metabolic Franchise of Novartis. Former Head of Canada and Latin America Region Patient Access and Commercial Development. Has experience in US, EU, emerging markets in public, private and NGO sectors of health care. MS, MPH from Columbia University School of Public Health
5:30 PM - 6:30 PM
Poster Session 4 Poster Tours
New this Year – Poster Tours! ISPOR has curated collections of research posters for you within each of the poster sessions. Each tour will feature high impact abstracts within a specific topical area and will include a tour guide as well as the poster authors to share their work and engage in discussions with you. Visit the Learning Formats page for more information. Poster Tour: Rare and Orphan Diseases
In-person
Posters featured in this tour:
EE15: Economic Burden of Transfusion-Dependent Beta-Thalassemia in the United States
EE48: Cost-Effectiveness of Alternative Diagnostic Testing Pathways with Whole Exome Sequencing (WES) in a Rare Disease Patient Population: The Canadian Care-for-Rare SOLVE (SOLVE) Multi-Centre Observational Cohort
EE500: Comparison of Healthcare Resource Utilization and Costs in Patients with and without Bullous Pemphigoid: A Retrospective Analysis of US Health Insurance Claims Data
HSD67: The Burden of Systemic Glucocorticoid (GC) Use in Anti-Neutrophilic Cytoplasmic Autoantibody (ANCA)-Associated Vasculitis Patients
HSD85: Real-World Usage Patterns and Costs of IVIG Treatment in Adults with Generalized Myasthenia Gravis in the United States
PCR102: Leveraging Social Media for Patient Experience Insights in Rare Disease
Poster Tour: Oncology
In-person
Posters featured in this tour:
EE149: Predicting the Population Budget Impact of Current and New Listings for Colorectal Cancer: The PRIMCAT-CRC Model
EE308: Real-World Treatment Patterns and Costs in Relapsed and Refractory Diffuse Large B-Cell Lymphoma in the United States
EE359: US Cost-Effectiveness of Chimeric Antigen Receptor T-Cell (CAR T) Therapy for Patients with Relapsed or Refractory Large B-Cell Lymphoma (R/R LBCL), Considering Infusion Setting and Payor Claims Data
EE437: Healthcare Resource Utilization and Costs in Patients with Multiple Myeloma Who Received 1 to 3 Prior Lines of Therapy, Including a Proteasome Inhibitor, an Immunomodulatory Drug, and Exposed to (AND DISCONTINUED) Lenalidomide in the United States
HSD101: Lack of Concordance between Real-World Treatment Patterns and Clinical Guideline Recommendations For Metastatic Hormone-Sensitive Prostate Cancer (MHSPC) Patients
5:30 PM - 7:00 PM
Networking Social (Exhibit Hall)
In-person
Wed May 18
7:30 AM - 1:00 PM
ISPOR 2022 Registration Hours
In-person
The ISPOR Registration Desk will be open for in-person participants.
8:00 AM - 9:00 AM
Concurrent Breakout Session 7
Assessing Real Option Value in Health Technology Assessment of Drugs
In-person
Level: Foundational
PURPOSE: This workshop's objectives are to provide a pragmatic guide and recommendations to quantify real option value (ROV) in health technology assessment (HTA) of drugs. After the workshop, participants will be able to (1) understand how ROV is generated; (2) identify situations where ROV is relevant; and (3) estimate ROV.
DESCRIPTION: The assessment of the value delivered by drugs has become increasingly important with a rapid innovation of drugs, which is accompanied by increasing healthcare costs. As such, the recent recommendations of the ISPOR Special Task Force on Value Assessment Frameworks introduced several potential novel ways that treatments could deliver value. One novel value element is ROV which will be the focus of this workshop. In the first half of the workshop, we will walk the audience through 1) the definition of ROV, 2) the key types of ROV, and 3) mechanisms by which each type of ROV are generated. After this, we will use real-time polling to discuss real-world examples of ROV across different disease types and will assess the type and mechanism for each of them. In the second half, we will walk through the approach to quantifying ROV using a flow chart, providing the audience with the pragmatic guide to ROV estimation from an ex ante perspectives. Lastly, we will conduct a real-time demonstration of quantification steps using a hypothetical state-transition model, focusing on how key ROV parameters can be incorporated into the model and how the parameters affect the size of ROV. This interactive and informative workshop will be especially beneficial for researchers interested in assessing the value of drugs using the ROV component of an augmented value assessment framework.
Discussion Leaders
Meng Li, PhD, ScM
University of Texas MD Anderson Cancer Center, Houston, TX, USA
Discussants
Stacey Kowal, BS, MSc
Genentech, Inc., Alameda, CA, USA
I am a global health economics and outcomes research (HEOR) expert with more than 15 years of experience in healthcare research and team leadership, including study conceptualization and execution, team building/staff development, external stakeholder relationship cultivation, and business unit operations. As a researcher, I’m most passionate about using economic modeling and real world evidence to understand how new innovations impact patients and their families, as well as broader stakeholders in the healthcare system. My current research uses a range of HEOR tools (economic modeling, real world data, patient reported outcomes) to identify and advance methods to improve our ability to measure and communicate value as it relates to health equity, precision medicine, health policy and health technology assessment.
Woojung Lee, PharmD
University of Washington, Seattle, WA, USA
Woojung Lee is a 4th year PhD candidate at the Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute at the University of Washington. Before entering the PhD program, she received her PharmD from Seoul National University in Seoul, South Korea, in 2016 and BS in biology from Yonsei University in 2012. Woojung has worked on health economics and outcomes projects using real-world data analysis (e.g., electronic medical records and claims data), disease modeling, and systematic review. Her research has been around the use of real-world evidence in economic assessment of drugs, the value of cancer drugs and diagnostic testing, novel applications of machine learning in HEOR, medication experience in older adults and dementia prevention strategies. She is currently working on her dissertation on the challenges and the value of older adult-specific clinical trials, which is supported by the PhRMA Foundation.
David Veenstra, PhD, PharmD
University of Washington, Seattle, WA, USA
Dr. Veenstra is Professor and Associate Director of the Comparative Health Outcomes, Policy & Economics (CHOICE) Institute in the Department of Pharmacy at the University of Washington.
Dr. Veenstra’s primary research interests are the clinical, economic, and policy implications of using genomic information in healthcare. His major research projects include evaluation of the cost effectiveness of population-level genomic screening, pharmacogenomics in diverse populations, and evidence thresholds, research prioritization, and preferences for precision medicine.
Dr. Veenstra’s research has been funded through grants from the National Human Genome Research Institute, Centers for Disease Control, and the National Cancer Institute.
Dr. Veenstra has worked extensively with organizations such as the Academy of Managed Care Pharmacy (AMCP) and the Institute for Clinical and Economic Review (ICER) to further the practical application of cost-effectiveness analysis in managed care decision making.
Dr. Veenstra teaches courses in health economics and managed care and is an author of five book chapters and over 200 peer-reviewed publications.
The Role of Actuarial Science in US Manufacturers’ Potential Budget Impact and Cost-Effectiveness Value Propositions
In-person
Level: Foundational
The objective of this session is to introduce how actuarial modeling capabilities join with clinical knowledge to bring a unique and valuable perspective. Drug and device manufacturers must work within a complex reimbursement / payment structure in the US for their products. Our discussion will focus on the following topics (average 15min each), and conclude with an open question-and-answer period (10min): (1) Patient population modeling, or how actuarial science using real-world data can be used to organize cost models that estimate the disease burden and cost implications for a manufacturer’s various customer types. (2) Pricing strategy considerations, a) including how actuarial science can help stakeholders understand the impact of complex pricing structures such as rebates on various payers (Medicare, Medicaid, commercial, pharmacy benefit manager, employer), b) how alternative contracting methods such as risk-based contracts or value-based pricing are valued, and c) how regulatory and customer willingness factors impact pricing and budgets. (3) Product launch support, such as how actuarial science can be used to estimate profitability of patients taking / using intervention for payer value proposition and compare across various customer types This session will benefit drug and device manufacturers at the foundational and intermediate level.
Speakers
Tracy Margiott, FSA, MAAA
Milliman, Windsor, CT, USA
Tracy specializes in evaluating health benefit programs for insurance companies, employers, pharmacy benefit managers, Medicare plan sponsors, and government entities. She supports drug manufacturers with optimizing pricing and formulary placement from a payer perspective in Medicare and commercial markets. Tracy enjoys helping clients assess and understand strategic considerations in response to regulatory and market changes.
Discussion Leaders
David Williams, BA
Milliman, Windsor, CT, USA
David is a senior healthcare consultant in the Hartford office of Milliman. He joined the firm in 1999.
David has more than 40 years of experience in healthcare economics and finance. His clients include medical device manufacturers, employers, insurers, physicians and hospitals. He uses big data and data science to help his clients.
David started his career as the Director of Medical Economics with Kaiser Permanente and MedSpan.
When he isn’t working, he is reading Chinese history, listening to his family play music, or volunteering.
Approaches for Utilizing Patient Preference Information to Inform Clinical Trial Design
Virtual
Level: Intermediate
The objectives are to introduce a heart failure patient preference study; to introduce approaches for utilizing patient preference data to inform clinical trial designs; and to discuss FDA’s perspectives on these topics. Patient preference information (PPI) studies offer the opportunity for medical device sponsors, regulators, and other stakeholders to quantify what matters most to patients. Taking full advantage of this opportunity requires a series of thoughtful decisions in designing a patient preference study and subsequent use of PPI in clinical trial designs. The Medical Device Innovation Consortium undertook a series of case studies and activities, including measuring patient preferences in heart failure and interviews with experts across the medical device ecosystem, to identify lessons learned and approaches for utilizing PPI to inform clinical trial designs. The session will begin with an overview of MDIC, development of the Patient Preference Information in the Design of Clinical Trials Framework, and how lessons learned and approaches were developed (5 min, Liden). This will be followed by a summary of one of MDIC’s PPI case studies that involved six medical device companies and was focused on developing and conducting a study to understand patient preferences pertaining to heart failure devices (10 min, Reed). The session will continue with an overview of approaches for incorporating PPI in clinical trial design, including opportunities to engage with regulators, experts and patients, identifying relevant endpoints, appropriate ways to leverage PPI for use in statistical evaluations of trial data, and generalizability of the PPI study sample to the population of patients eligible for a device (20 min, Liden). In the final presentation, FDA-CDRH will provide perspectives on use of PPI (10 min, Tarver). The session will conclude with a discussion between audience and speakers (15 min). This session may benefit clinical trialists, medical device and pharmaceutical sponsors, and regulators.
Speakers
Shelby Reed, PhD, RPh
Duke Clinical Research Institute, Durham, NC, USA
Shelby D. Reed, PhD, is Professor in the Departments of Population Health Sciences and Medicine at Duke University’s School of Medicine. She is the director of the Center for Informing Health Decisions and Therapeutic Area leader for Population Health Sciences at the Duke Clinical Research Institute. She also is core faculty at the Duke-Margolis Center for Health Policy. Dr. Reed has over 20 years of experience in economic evaluation, health services research and health policy. Her research portfolio includes a broad array of trial‐based and model‐based cost‐effectiveness analyses of new and existing medical diagnostics, drugs, devices and patient‐centered interventions. For the past several years, she has increasingly dedicated her efforts to the field of stated‐preference research. In 2016, she co-founded the Preference Evaluation Research (PrefER) Group at the DCRI, and she currently serves as its director. She and the group are frequently sought to conduct stated-preference studies to inform regulatory decisions, health policy, care delivery, value assessment and clinical decision making with applied projects spanning a wide range of therapeutic areas.
Dr. Reed has published more than 200 manuscripts in peer‐reviewed journals. She was the first recipient of ISPOR's Bernie O’Brien New Investigator Award in 2005. She served on two ISPOR Task Force groups to develop recommendations for conducting economic evaluations alongside clinical trials and recommendations to address transferability of multinational economic evaluations. She recently served as a guest editor for a themed issue in Value in Health on Patient‐Focused Benefit‐Risk Analysis to Support Regulatory Decision‐Making. She served on the editorial boards for Health Services Research (2016-2020) and Value in Health (2013-2021). She served as President for ISPOR in 2017-2018, and she currently is Past-Chair of the Society’s Health Science Policy Council.
Michelle Tarver, MD, PhD
U.S. Food and Drug Administration, Silver Spring, MD, USA
Dr. Michelle Tarver is the Deputy Director of the Office of Strategic Partnerships and Technology Innovation where she helps provide leadership for all scientific collaborative and emerging technology-related activities at the Center for Devices and Radiological Health (CDRH). Under her leadership, the office is advancing efforts to include diverse and underrepresented patient perspectives in the evaluation of medical devices through the Health of Women program, the Patient Science & Engagement Program, and the Pediatric and Special Populations program. She provides leadership and oversight for CDRH in matters relating to public health emergency preparedness and response activities involving medical devices. Not only does she assist in guiding work in standards development and implementation for medical device innovation and manufacturing, she also provides strategic vision for collecting, analyzing and integrating the patient perspective in the development, evaluation and surveillance of medical devices, including digital health technologies. As the lead of CDRH’s Strategic Priority on Collaborative Communities, she guides and facilitates collaboration in fostering the development and evaluation of innovative medical devices that can address unmet public health needs. She continues to advance the patient perspective as the Program Director for Patient Science in the Digital Health Center of Excellence. In addition to her prior experience in patient-focused efforts as the Director of the Patient Science and Engagement Program, Dr. Tarver has extensive experience in premarket and postmarket review of various medical devices, developing guidance documents and standards, and fostering internal and external collaborations.
Dr. Tarver attended Spelman College in Atlanta, GA where she received a B.S. in Biochemistry. She completed the M.D./Ph.D. program at The Johns Hopkins University Bloomberg School of Public Health (Ph.D. in clinical epidemiology) and The Johns Hopkins University School of Medicine. Following her internal medicine internship, she completed a residency in ophthalmology with fellowship training in ocular inflammation (uveitis) both at the Wilmer Eye Institute (Johns Hopkins). As an epidemiologist and board-certified ophthalmologist, she has worked on longitudinal epidemiological studies, clinical trials, registries, developing patient-reported outcome measures as well as surveys to capture patient preferences with medical devices. Her research has resulted in numerous peer-reviewed publications and published book chapters. As a dedicated clinician, she continues to evaluate and treat ophthalmology patients at Solomon Eye Associates in Bowie, MD.
Discussion Leaders
Barry Liden, Juris Doctor (JD)
Edwards Lifesciences, Garrett Park, MD, USA
How Best to Frame Cost-Effectiveness Thresholds for Policy Making in the US
In-person & Virtual
ISSUE: In cost-effectiveness research, an age-old debate surrounds whether to impose cost-effectiveness thresholds within or alongside cost-effectiveness findings to support value-based policy making. And, if imposed, what threshold(s)? The purpose of this issue panel is to debate cost-effectiveness threshold framing solutions with the goal of aiding health technology policy making for US health systems and society.
OVERVIEW: The expert panel will explore the question: how best to frame cost-effectiveness thresholds for policy making in the US? More specifically, the panelists and moderator will debate threshold framing solutions that are aligned with gains for health systems and society. Jon Campbell will introduce the topic of US cost-effectiveness thresholds and share current threshold solutions practiced within the Institute for Clinical and Economic Review. The panelists will share and debate threshold framing solutions with a focus on perspective (the health care system perspective with a fixed budget and k opportunity-cost thresholds vs. the societal perspective with v willingness-to-pay thresholds) and other considerations for modifying thresholds. Tara Lavelle will argue for threshold considerations consistent with the broader societal perspective. David Vanness will argue for threshold considerations based on health opportunity costs. Charles Phelps will share ideas for when (and how much) thresholds should vary by severity of untreated disease. Core to the issue, each panelist will opine on threshold framing solutions that aim to improve (i.e., provide a surplus to) US health systems and society rather than pricing technologies to achieve neutrality where the entire surplus is transferred to the suppliers. Differences in expert opinion will be explored and debated. Finally, we will poll the audience to test threshold framing solutions while protecting ten minutes for audience questions.
Moderators
Jon Campbell, PhD
Institute for Clinical and Economic Review, Hingham, MA, USA
Jon (Jonathan D.) Campbell is Senior Vice President for Health Economics at the Institute for Clinical and Economic Review (ICER). Jon joined ICER’s senior management team as a leader in value assessment methods and application; he oversees the growth of ICER’s health economics efforts and leads the continued innovation of ICER’s value assessment methodology. Additionally, Jon continues to build bridges within the global health economics community through engagement with ICER’s Health Economic Council and through leadership and participation in health technology assessment societies and agencies. Further, Jon seeks creative value assessment solutions for ICER’s diverse stakeholders by prioritizing improved outcomes for patients.
Jon is an affiliate faculty member at Tufts University School of Medicine in the Center for the Evaluation of Value and Risk in Health. Jon is an author of over 100 peer-reviewed manuscripts in the field of value assessment as well as an author on many ICER assessments. Jon is a former ICER Health Economics Council member and five-year external collaborator through his former role as Associate Professor with tenure at the University of Colorado Anschutz Medical Campus. He holds graduate training degrees in pharmaceutical outcomes research (PhD) and biostatistics (MS) from the University of Washington. He graduated with a BA in mathematics and chemistry from St. Olaf College.
Jon also enjoys playing most racket-related sports. He grew up playing pickleball in the Seattle area and is seeking opportunities to spread the pickleball love in the Boston area.
Panelists
Tara Lavelle, PhD
Tufts Medical Center, Boston, MA, USA
Tara Lavelle is an Assistant Professor at the Tufts Medical Center Institute for Clinical Research and Health Policy Studies and an Investigator at the Center for the Evaluation of Value and Risk in Health. Her research addresses a range of topics related to the cost and value of health care services delivered in the United States. Dr. Lavelle is particularly interested in advancing the use of comparative and cost-effectiveness research in vulnerable populations, including children. Previous work includes the development of novel methods for evaluating health and economic outcomes of childhood illness, and assessing preference-based quality of life outcomes in caregivers.
Dr. Lavelle earned her PhD in Health Policy with a concentration in Decision Science from Harvard University and was previously a postdoctoral research fellow in the Child Health Evaluation and Research Unit in the Division of General Pediatrics at the University of Michigan.
Charles Phelps, MBA, PhD
University of Rochester, Rochester, NY, USA
Charles E Phelps, PhD, a health economist, has developed key models of cost-effectiveness analysis that provide the intellectual foundations for its practice. He was given the Victor R Fuchs Award for Lifetime Achievement in the Field of Health Economics in 2019, and has been a member of the National Academy of Medicine since 1991. His leading textbook, Health Economics is now in its 6th Edition. His recent interests have expanded to the use of multi-criteria decision analysis (MCDA), particularly in its proper use when the “decision-maker” is a group.
David Vanness, PhD
Pennsylvania State University, University Park, PA, USA
Health Technology Assessment for Gene Therapies: Are Our Methods Fit for Purpose?
Virtual
ISSUE: GTx represents a new era of medicine, offering the potential for transformational benefits for patients, health systems and society. Health Technology Assessment (HTA) of GTx can be challenging for a variety of reasons, including clinical evidence generation; uncertainty; assessment of cost and affordability; and narrow value perspectives such as limitations of traditional quality of life measures.
The main objective of the panel will be to reconcile the various challenges facing value assessment for GTx, and put forward evidence-based recommendations to overcome these barriers. The panel will underscore the perspectives and priorities of patients, health economists, and industry, while highlighting policy and methodological changes that need to be made to current HTA practice and payer decision-making.
OVERVIEW: The moderator (Adrian Towse) will briefly introduce the topic, and summarize the findings of a literature review and expert roundtable programme. He will then pose key questions to the presenters, who will each speak for approximately 15 minutes, providing their diverse perspectives on the issues and key considerations.
Louis Garrison, Professor Emeritus, University of Washington will set out the economic arguments including the success points and sticking points on HTA reform for gene therapies in different countries. Bhash Parasuraman, Vice President, Pfizer will comment from an industry perspective, highlighting the urgent need for policy change to facilitate timely access to innovative gene therapies. Mark Skinner,
President/CEO, Institute for Policy Advancement, Ltd. who has previously led the World Federation of Hemophilia and National Hemophilia Foundation will provide a patient perspective. The panel will discuss which areas of HTA are in need of change and debate possible solutions such as outcomes-based pricing and other innovative payment schemes.
Moderators
Adrian Towse, MA, MPhil
Office of Health Economics, London, United Kingdom
Professor Adrian Towse is director emeritus and senior research fellow of the Office of Health Economics in the UK. Adrian’s current research includes incentives for new drugs and vaccines to tackle Antimicrobial Resistance, the use of 'risk-sharing' arrangements between healthcare payers and pharmaceutical companies, including value-based pricing approaches; the economics of pharmacogenetics for healthcare payers and the pharmaceutical industry; economic issues that affect both R&D for and access to treatments for diseases prevalent in the developing world; the economics of medical negligence; and measuring productivity in healthcare.
A visiting professor at the London School of Economics and a senior researcher at the Nuffield Department of Population Health at the University of Oxford, Adrian also has been a visiting professor at the University of York. For ten years, he served as the non-executive director of the Oxford Radcliffe Hospitals NHS Trust, one of the UK’s largest hospitals. Adrian was president of ISPOR, for the 2014-15 term.
Adrian joined the OHE in 1993 and served as director for 25 years. He holds an MA (Hons) in Politics, Philosophy and Economics from Keble College, Oxford; an MPhil in Management Studies from Nuffield College, Oxford, and the Oxford Centre for Management Studies; and is a member of the Chartered Institute of Management Accountants.
Panelists
Lou Garrison, PhD
University of Washington, Seattle, WA, USA
Lou Garrison, PhD, is Professor Emeritus in The Comparative Health Outcomes, Policy, and Economics Institute in the School of Pharmacy at the University of Washington, where he joined the faculty in 2004. Prior to this, he has worked in non-profit policy research (13 years) and the pharmaceutical industry (12 years).
Dr. Garrison received a PhD in Economics from Stanford University. He has more than 180 peer-reviewed publications. His research interests include a wide range of national and international health policy issues.
Dr. Garrison was elected as ISPOR President for 2016-2017. He is currently serving as co-chair of ISPOR’s Policy Outlook Committee for the Health Science Policy Council.
Patricia Schepman, PharmD, MS, PhD
Pfizer Inc., Cos Cob, CT, USA
Mark Skinner, JD
Institute for Policy Advancement, Ltd, Washington, D.C., DC, USA
Mark Skinner, Washington, DC, is President/CEO of the Institute for Policy Advancement Ltd, specializing in patient-centered outcomes research. He is an Assistant Professor in the Department of Health Research Methods, Evidence and Impact at McMaster University and the principal investigator for the Patient Reported Outcomes Burdens and Experiences (PROBE) global research study. He has previously led both the World Federation of Hemophilia and National Hemophilia Foundation, where he currently serves on the Medical and Scientific Advisory Council. He holds degrees in Public and Business Administration from Kansas State University and JD from Washburn University School of Law.
Is the Global Real-World Data (RWD) Supply-Chain Broken? Choosing between RWD Quality Versus Locality in Supporting Single Arm Submissions
In-person & Virtual
ISSUE: The growing number of highly specialized products (cell-based and gene therapies) and the focus on personalized medicine are rapidly expanding trends in the drug development landscape. These products often achieve regulatory approval based on single-arm trials due to ethical considerations or difficulty of recruiting patients, thus imposing a challenge to HTA bodies to conduct comparative effectiveness research. Few HTA submissions based on single-arm trials have been successful in convincingly establishing comparative effectiveness. During this assessment process, decision makers have shown preference for local data reflecting clinical practice. However, this preference can bring methodological challenges depending on the data local availability and quality and present operational difficulties when industry is planning for multiple submissions across different countries. Further, firm guidelines on what is considered convincing evidence is lacking, and the quality assessment criteria of a “good” external control arm differ substantially across HTA bodies.
OVERVIEW
: Several manufacturers have already supported their regulatory and HTA filings using single-arm trials while creating an external real-world arm (historical trial data and/or using real-world-evidence [RWE] sources) to establish comparative effectiveness. However, selecting the most appropriate source for this control arm remains challenging. When a RWE control arm is used, HTA authorities show a preference for local data. For manufacturers, it is challenging to ensure patient representation in a specific location while using “good” quality data and making efficiencies in resources. This panel will debate, from an industry, academic and HTA perspective (15 min each presentation) if a control arm based on a single RWE data source can be achievable and meet the requirements of different HTA bodies; whether quality or locality of RWE data is the main driver in HTA acceptance and whether current HTA guidance can inform decisions in this area. 10min discussion will follow. Stakeholders (industry, researchers, payers) will benefit from this panel.
Moderators
Grammati Sarri, PhD Msc DiDS
Cytel, London, LON, United Kingdom
The authors have a deep understanding on evidentiary requirements for preparing health technologies' submissions to health care decision-makers. Dr Sarri and Dr Freitag have previously worked for an HTA body (NICE) and several years of experience consulting pharmaceutical companies on how to best use different types of evidence to increase the value proposition of new technologies and positive launch of products. Dr Schmetz has several years of market access experience as a consultant.
Panelists
Luis G. Hernandez, PhD MPH MSc
Takeda Pharmaceuticals America, Inc., Westford, MA, USA
Luis Hernandez, PhD MPH MSc, is the Head of Global Health Economics, in the Global Oncology Patient Value, Policy, and Access, at Takeda Oncology, where he is responsible for generating health economics evidence to demonstrate the value of innovative and transformative medicines to accelerate access for patients across the world, sustainable pricing, and develop valuable partnerships to shape the environment. Dr. Hernandez has extensive experience in health economics and outcomes research, evidence generation planning, market access, and cross functional team leadership and management.
Prior to joining Takeda, Dr. Hernandez spent 13 years at Evidera (a business within Pharmaceutical Product Development, LLC, [PPD] a leading global contract research organization [CRO]) where he spearheaded health economics evidence generation and dissemination to support the optimal value proposition for pipeline, launch, and lifecycle assets. During his tenure, Dr. Hernandez held several leadership roles including Senior Director and Senior Scientist heading Evidera’s health economics modeling and simulation in Waltham, Massachusetts, USA.
Between 2009 and 2010, Dr. Hernandez was a full time professor of Probability, Statistics, Discrete Event Simulation, and Decision Analysis for undergraduate and graduate students at Universidad de los Andes (Bogota, Colombia) for the Industrial Engineering Department and the Executive MBA program.
Dr. Hernandez holds a PhD in Health Economics from the University of Groningen (the Netherlands), a Master’s in Public Health from Tufts University (Boston, Massachusetts, USA), and Master’s and Bachelor’s degrees in Industrial Engineering from Universidad de los Andes (Bogota, Colombia).
Seamus Kent, PhD
National Institute for Health and Care Excellence (NICE), London, LON, United Kingdom
Seamus is a Senior Adviser in Data & Analytics at NICE. He is leading the development of a framework for the use of data and analytics in the development of NICE guidance.
Kristian Thorlund, MSc, PhD
McMaster University, Hamilton, ON, Canada
What Is Value? How Can We Appropriately Assess the Value of Digital Health Technologies?
In-person
ISSUE: The purpose of this Issue Panel is to debate the appropriate approaches to value assessment of innovative digital health technologies (DHTs). The session will create a dialogue around the value of innovative DHTs: where value lies, for whom value is created, and an introduction into the future challenges of measuring these domains.
OVERVIEW: Due to the nature of the technology, innovative DHTs may not be able to adhere to traditional value assessment criteria, which creates a need for alternative valuation methods. For example, DHTs may not be able to produce evidence of effectiveness through randomized controlled trials (RCTs) and may need alternative avenues to prove effectiveness, such as through Real World Evidence. Additionally, DHTs may be presenting new aspects of value that are not adequately accounted for using current assessment criteria. For example, the need for secure data provenance is a new value construct unique to digital technologies.
This Issue Panel will explore how value of digital health technologies can be assessed through questions such as, “what kind of evidence do we need to see in a framework for a DHT before a health system can decide to reimburse it?” and “what are the new aspects of value that DHTs present?” Each panelist will have an opportunity to present their unique perspectives on these issues, including perspectives from industry, academic research, and an HTA advisory body. Additionally, the session will focus on the importance of multi-stakeholder involvement in defining value in a standardized value framework for broader technological and therapeutic area applicability, which is an important aspect of assessing value because what is valuable differs between stakeholder groups. Perspectives will be presented on how to facilitate multi-stakeholder collaboration to define value. Declaration: Funding will be provided by Sanofi.
Moderators
Panos Kanavos, PhD
London School of Economics and Political Science, London, United Kingdom
Dr Panos Kanavos is Associate Professor in International Health Policy in the Department of Health Policy at London School of Economics and Political Science, Deputy Director at LSE Health and Programme Director of the Medical Technology Research Group (MTRG).He has acted as an advisor to a number of international governmental and non-governmental organizations, including the European Commission, the European Parliament, the World Bank, the World Health Organization, the Organization for Economic Co-operation and Development (OECD), and Ministries of Health of over 28 transition, emerging and developing countries.
Panelists
Daniel Anggono, BSE
Sanofi, Bridgewater, NJ, USA
Federico Augustovski, MSc, PhD, MD
Institute for Clinical Effectiveness and Health Policy (IECS), Buenos Aires, B, Argentina
Federico Augustovski, MD, MSc, PhD, is the current director of Health Economic Evaluations and Technology Assessment at the Institute for Clinical Effectiveness and Health Policy (IECS), an independent non-profit organization affiliated to the University of Buenos Aires, a CONICET (National Scientific and Technical Research Council) center, and one of the few INAHTA Health Technology Assessments agencies in Latin America. Federico is the director of the WHO Collaborating Centre in Health Technology Assessment and Economic Evaluations at IECS. He is also the founding editor-in-chief for Latin America section of Value in Health Regional Issues, the ISPOR peer-reviewed journal for Latin America, Asia, and Central & Eastern Europe, Western Asia, and Africa. He is the director of the PAHO affiliated PROVAC Center of Excellence for decision making in vaccines. Federico leads a multidisciplinary team devoted to clinical and economic evaluations of new and existing preventive, diagnostic, and therapeutic technologies that provides research, education, and technical support with public and private health decision makers in Latin America. He is a professor of Public Health at the School of Public Health of the University of Buenos Aires, where he teaches courses for graduate and postgraduate students in Decision Sciences; Patient-Reported Outcomes Development in Health, as well as Health Economic Evaluations.
Federico earned his MD with honors at the University of Buenos Aires and is a specialist in family medicine. He practiced family medicine and was a staff physician for more than 20 years at the Family and Community Medicine Division of the Hospital Italiano de Buenos Aires. He received his MSc in epidemiology (Harvard School of Public Health). He was a European Union Scholar in health economics at the Centre for Health Economics at the University of York in the UK. His research production concentrates in health technology assessments and health economic evaluations methods and applications. He has published more than 70 PubMed-indexed papers.
Federico has served and serves ISPOR in several capacities during the past 10 years. Among other commitments, he was the first Latin American director on the board of directors, the founder and first president of the Argentine local chapter, the first chair of the Latin American Consortium, chair of the Research Excellence Award, president of Buenos Aires 2013 Regional meeting, member of the Health Science and Policy Council and Vision 2020 teams, as well as several Task Forces.
Harriet Unsworth, PhD
National Institute of Health and Care Excellence, Manchester, United Kingdom
Harriet Unsworth is a Technical Advisor in the Office for Digital Health at the National Institute for Health and Care Excellence (NICE), UK. She is an expert in the evaluation of digital and artificial intelligence technologies for health and care. She has an MSc in data science and artificial intelligence and a PhD in molecular biology.
9:00 AM - 10:00 AM
Poster Session 5 Poster Tours
New this Year – Poster Tours! ISPOR has curated collections of research posters for you within each of the poster sessions. Each tour will feature high impact abstracts within a specific topical area and will include a tour guide as well as the poster authors to share their work and engage in discussions with you. Visit the Learning Formats page for more information. Poster Tour: Vaccines
In-person
Posters featured in this tour:
EE174: Economic Evaluation of Seasonal Influenza Vaccination in Elderly and Health Workers: A Systematic Review and Meta-Analysis
EE188: COVID-19 Vaccine Hesitancy Among Early Adaptors in Jordan
EE204: Budgetary Impact of New Recommendations for Pneumococcal Vaccination of US Adults
EE335: The Hospitalization Burden of COVID-19 in Patients with NSCLC: Differential Impact of Vaccination
EE388: Public Health and Economic Implications of Increasing Access to Herpes Zoster Vaccination Rate in Community Pharmacies
EPH65: The Public Health Impact of Routine Vaccination in 16-40-Year-Old Females for the Prevention of Cytomegalovirus and Congenital Cytomegalovirus in the United States
Poster Tour: Mental Health
In-person
Posters featured in this tour:
CO110: Comparative Effectiveness of Sertraline, Fluoxetine Vs Escitalopram Among Adults with Depression in the United States
CO122: Patient Versus Caregiver and Clinician Reports of Cognitive Difficulties in Patients with Schizophrenia Switching to Long-Acting Injectable Antipsychotic Aripiprazole Lauroxil: A Post Hoc Analysis
EE9: Real-World Naloxone Prescription Trends, Costs, and Comorbidities in Commercial, Managed Medicare, and Medicaid Patients in the United States (2016-2019)
RWD66: Leveraging Laboratory Results from Multiple Data Sources to Re-Assess a Possible Association between Serum Uric Acid and Schizophrenia in Real World Practice Settings
SA15: Real-World Calibration of the Disease Recovery Evaluation and Modification (DREaM) Randomized Clinical Trial in Adult Medicaid Beneficiaries With Recent-Onset Schizophrenia
Coffee Break
In-person
9:00 AM - 12:45 PM
In-Person and Virtual Poster Session 5
Live
In-person presenters will be with their posters from 9:00 – 10:00AM.
Poster Viewing & Exhibit Hall Open
In-person
In-person/Virtual
10:00 AM - 11:00 AM
Podium Session 8
Studies on COVID-19 Healthcare Impacts
In-person
Moderator
William Padula, PhD, MS, MSc
University of Southern California, Los Angeles, CA, USA
William Padula, PhD is assistant professor of pharmaceutical & Health Economics at the University of Southern California School of Pharmacy, and a Fellow in the Leonard D. Schaeffer Center for Health Policy & Economics. His research interests include medical cost-effectiveness analysis and applications of machine learning to health economics and outcomes research. He was the 2021 recipient of ISPOR’s Bernie O’Brien New Investigator Award, and Is an Associate Editor for Value in Health.
P36: The Impact of COVID-19 Diagnosis on Healthcare Costs
10:45AM - 11:00AM
Morrow C
OBJECTIVES: The Covid-19 pandemic has disrupted the healthcare system and created high burden of disease. This research aims to measure the healthcare costs of commercially insured and Medicare patients with Covid-19 before and after diagnosis.
METHODS: All patients with a Covid-19 diagnosis between March 2020 and January 2021 were identified in the IBM® MarketScan® Commercial and Medicare Research Databases, using International Classification of Diseases (ICD) diagnosis code U07.1. Demographic information was measured on the date of diagnosis. Healthcare costs were measured in the 6 months prior to diagnosis and compared to healthcare costs measured in the 6 months after diagnosis for pediatric and adult commercially insured and Medicare patients. The databases provide detailed cost, use, and outcomes data for healthcare services performed in both inpatient and outpatient settings.
RESULTS: In this descriptive analysis, 786,238 patients (743,987 Commercial and 42,251 Medicare) were diagnosed with Covid-19 during the study period. Overall, 46% were male, and 90% were above the age of 18, with a mean age of 40. In the 6 months before a Covid-19 diagnosis, mean (SD) healthcare costs were $4,771 ($19,795) for Commercial and $11,622 ($22,665) for Medicare. In the 6 months following Covid-19 diagnosis, mean (SD) healthcare costs were $7,795 ($35,633) for Commercial and $20,221 ($35,162) for Medicare. Post-diagnosis mean healthcare spending was 1.6 times that of pre-diagnosis for Commercial and 1.7 times larger for Medicare. A similar trend is observed among the subset of pediatric patients.
CONCLUSIONS: Measuring change in baseline healthcare costs in a highly representative claims database is foundational in understanding the true economic burden of the Covid-19 pandemic. Further research in longitudinal claims data will illuminate burden of long Covid-19. Further research is needed to investigate disparities and the burdens of out-of-pocket spending and absenteeism/job loss.
P35: Risk of Psychiatric and Neurological Sequelae after the COVID-19 Infection: A Retrospective Cohort Study Using Claim Data
10:15AM - 10:30AM
Gaur A1 , Kukreja I2 , Mishra N3 , Roy A4 , Chopra A3 , Gupta A5 , Verma V 6 , Pandey S7 , Brooks L8 , Sulzicki M9 , Field S10 , Krebs B11 1 Optum, Gurugram, HR, India, 2 Optum Global Solutions, India, New Delhi, DL, India, 3 Optum Global Solutions, India, Gurugram, HR, India, 4 Optum, Gurgaon, HR, India, 5 Optum, Eden Prairie, MN, USA, 6 Optum Global Solutions, India, Gurgaon, HR, India, 7 Optum Global Solutions, India, noida, gautam buddha nagar, UP, India, 8 Optum, Basking Ridge, NJ, USA, 9 Optum, Trumbull, CT, USA, 10 Optum, Dallas, TX, USA, 11 Optum, Tucson, AZ, USA
Objective: Studies erstwhile have suggested that COVID-19 also had effects on brain health. In this study we intend to provide estimates of incidence rates, odd ratio, and risks of psychiatric and neurological sequelae in patients after a COVID-19 diagnosis. Method : This retrospective and observational study included patients diagnosed with COVID-19 infection between 1st March 2020 to 31st March 2021 with ICD-10-CM diagnosis recorded in the large deidentified database of US health insurance claims representing ~15% population. Only the patients having continuous eligibility between 6 months before (baseline period) to 6-month post (follow-up period) the first diagnosis of COVID-19 (index date) were included in study. Frequency of Psychiatric and Neurological ICD-10-CM diagnosis codes occurring during baseline period and during follow-up period were evaluated. ICD-10-CM were grouped in the disease buckets. For every disease bucket, we evaluated the risk estimates and odds ratios (ORs) of association with COVID-19. Results : Among 1,154,471 patients diagnosed with COVID-19, the estimated incidence of following neurological and psychiatric disorders from 14 to 180 days after index diagnosis were: 11.1% for Anxiety disorders, 8.4% for Mood disorders, 7.4% for Insomnia, 4.1% for Dementia, 4.0% for Substance misuse, 2.6% for other disorders of brain, 1.9% for Nerve disorders, and 0.7% for Psychotic disorders. Disorders showing higher absolute risk (AR) included Anxiety (OR 1.23, AR 13.5%), Mood disorder (OR 1.18, AR 9.8%), Insomnia (OR 1.25, AR 8.4%), Dementia (OR 1.40, AR 4.5%), Substance misuse (OR 0.98, AR 4.3%), and Other disorders of Brain (OR 1.62, AR 2.7%). Conclusion : The results provide evidence for substantial psychiatric and neurological morbidity in the 6 months after COVID-19 infection. Results also may provide guidance on prognosis, treatment decisions and patient counselling.
P33: Evaluating the Impact of the COVID-19 Pandemic on the Diagnosis and Staging of New Breast Cancer Cases: A Retrospective Analysis of Medical Chart Data from the United States
10:30AM - 10:45AM
Knapp R 1 , Hardtstock F1 , Wilke T2 , Boner B3 , Hurmiz C3 , McCracken A3 1 Cytel Inc., Berlin, Germany, 2 IPAM e.V., Wismar, Germany, 3 Guardian Research Network, Spartanburg, SC, USA
Objectives Our study sought to assess the impact of the COVID-19 pandemic and resultant lockdown measures on the staging of incident breast cancer diagnoses in the United States, using data from 2018-2021. Methods We conducted a retrospective analysis of medical chart data provided by Guardian Research Network. Adult patients were eligible for inclusion if they received an inpatient/outpatient breast cancer code [ICD-10 C50] from 01/01/2018-12/31/2020. Patients with pre-existing cancer diagnoses (C00-C96) recorded within two years were excluded. The number of new cancer cases was descriptively analyzed, alongside cancer stage at diagnosis for a subset of patients, by triangulating TNM measurements, physician staging assessments and C77-79 codes recorded within the index/subsequent quarter. Results Overall, 6,639 patients (mean age: 62.75; 99.16% female) were identified. Minor fluctuations in the average number of new diagnoses per quarter were observed over time (2018: 507.25; 2019: 580.75; 2020: 571.75). A drop in new diagnoses was detected during the second quarter of 2020 (412), however relatively stable numbers were observed during all other quarters in 2020 (mean: 625; range: 586-646). A total of 587 and 491 patients from 2019 and 2020 were included in follow-up cancer staging analyses. Differences in the distribution of new diagnoses by stage were consistent across most quarters in 2019 and 2020. Nonetheless, only 23.38% of patients received Stage 1 cancer diagnoses during the second quarter in 2020, as compared to 34.01% in 2019. The proportion of patients with stage 2/3 diagnoses was consistent across both years, while the share of stage 4 diagnoses was higher in the second quarter of 2020 (32.47% versus 22.45%). Conclusions The pandemic’s impact on breast cancer diagnoses was particularly pronounced during the second quarter of 2020, corresponding with fewer overall cases and more severe prognoses, with potential links to delayed care and/or constricted access to healthcare services.
P34: Identifying Interventions That Reduced COVID-19 Mortality in Long-Term Care Facilities: A Causal Inference Analysis
10:00AM - 10:15AM
Goldberg R
OBJECTIVES
: Because of a lack of experimental evidence to treat COVID-19, we applied causal inference (CI) analysis to longitudinal health record data of 4,091 long-term care high-risk patients with COVID-19 to ascertain those interventions that directly improved health outcomes.
METHODS
: COVID-19 patient data collected from January through October of 2020. Directed Acyclic Graphs (DAGs) were built to model assumed treatment cause and effect relationships and to eliminate bias from latent variables. Orthogonal Random Forests were used to generate individual heterogeneous treatment cause and effect models of all concurrent pharmacotherapy and a propensity score was calculated for each. Treatment-specific logistic-regression models were used to determine average treatment effects across the entire patient population. Mortality within 120 days of a Covid -19 diagnosis was the primary endpoint. Analysis was conducted using Python 3.9.0 in conjunction with the EconML and Scikit-Learn packages.
RESULTS
: Patients with higher body temperatures, oxygen saturation below 90% during their COVID-19 infection, and low platelet counts were at a significantly increased risk of death within 120 days. We determined that the average treatment effect across all COVID-19 positive residents on mortality risk was caused directly by omeprazole, enoxaparin cholecalciferol, apixaban, prednisone, sennosides and guaifenesin by showing counterfactually that mortality risk increased without those medicines.
CONCLUSIONS
: Causal inference combines graphical models of causal relationships with the calculation of counterfactuals (“what-if” questions) to identify actionable causal factors that lead to improved health. Additionally, as demonstrated, interventional models derived from causal inference can be used to extrapolate “findings across domains (i.e., settings, populations, environments) that differ both in their distributions and in their inherent causal characteristics.” Future application of causal inference should demonstrate that such models can dynamically update treatment to optimize health as patient personal and clinical conditions change.
Concurrent Breakout Session 8
HTA in a Public Crisis: Communication Challenges
Virtual
ISSUE: As a major public health crisis, the COVID-19 pandemic challenges healthcare systems and decision-makers, as important decisions relating to new health technologies have to be made under considerable uncertainty and pressure. At the same time, researchers are still themselves learning about the emerging condition. They find themselves in a “ doing while learning” position, where only partial information is available and initial positive results may be followed by negative ones, or early negative results are followed by other, more positive, results. Knowledge is constantly challenged, uncertainties are high and while seemingly a large amount of literature is available, its validity is often questionable . Despite all of these limitations, there is a significant pressure on HTA agencies are requested to provide rapid opinions on a range of questions: which populations could benefit the most from different interventions? Whom to vaccinate first? What role for monoclonal antibodies or antiviral treatments for people at different disease stages (with or without oxygen, hospitalized or not, pre-exposure or post-exposure prophylaxis etc.). What’s the right way to inform the public about the rationale of this work and consequent decisions?
OVERVIEW: The moderator will briefly introduce the issue (4 mins) and the panel will then debate the concerns of challenging HTA communication and on perspective on the questions set out above. Panelists will each speak for 10 minutes, providing their perspectives on the issues as well as the key considerations. 20 minutes will be reserved for audience discussion.
The panel will be of interest to those working in HTA, healthcare professionals and patient organisations
Moderators
Francois Houyez, Patient Advocate
European Organisation for Rare Diseases (EURORDIS), Paris, France
François Houÿez is Director of Treatment Information and Access at the European Organisation for Rare Diseases EURORDIS.
He has always been working as a patient advocate since the early 90s, first in the HIV/AIDS advocacy, and in rare diseases since 2003.
His experience with compassionate use programmes started in 1988.
He pioneered patient advocacy with the European Medicines Agency as part of the first patients’ delegation that engaged dialogue with the Agency back in 1996.
François is also a patient.
Panelists
Dalia Dawoud, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Dalia Dawoud, PhD, is Senior Scientific Adviser at the National Institute for Health and Care Excellence (NICE). She holds MSc in Economic Evaluation in Health Care from City University London and PhD in pharmaceutical policy and economics from King’s College London.
She has long experience in using economic evaluation in clinical guidelines development and health technology assessment (HTA), gained through working on NICE Clinical Guidelines as well as technology appraisals. Dalia’s research interests are focused on the advanced methods of evidence synthesis and use in economic models and the use of real-world evidence to inform drug development and health care decision making. Dalia currently has overall responsibility of overseeing the delivery of NICE allocated tasks on a portfolio of IMI and Horizon 2020 funded research projects including EHDEN and HTx. She is widely published in the field of pharmaceutical policy and pharmacoeconomics. She also serves as Associate Editor for ISPOR journal Value in Health and as Associate Editor for Pharmacoeconomics and Outcomes Research for Elsevier’s journal Research in Social and Administrative Pharmacy. Dalia also holds adjunct position as Associate Professor at the Faculty of Pharmacy, Cairo University.
Saskia Knies, PhD
Health Care Institute, Diemen, NH, Netherlands
Saskia Knies, PhD is senior advisor health economics and personalised medicine at the National Health Care Institute in het Netherlands (Zorginstituut Nederland; ZIN). In addition, she is the coordinator of the research network HTA in which ZIN collaborates with Erasmus University Rotterdam and Utrecht University. She holds a MSc in Health Science Research – specialisation Health Technology Assessment from Maastricht University and a PhD in health sciences also from Maastricht University. She has extensive experience in using economic evaluations in reimbursement decision making by working as a pharmacoeconomic assessor at ZIN. Saskia’s research interest are focused on HTA methods development to support healthcare decision making especially concerning economic evaluations, transferability of (health economic) evidence and on international comparisons. Saskia is also involved in the H2020 funded research project HTx. Her main task in all her work is building bridges between research especially methods development or policy research and the use of these methods in supporting decision making. Saskia has multiple publications in the field of health technology assessment and pharmacoeconomics. She has been actively involved in different ISPOR activities, among others faculty member of the short course on transferability, member of Value of Information task force, member of the HTA Roundtable Europe, reviewer for Value in Health and presenter in several panels and workshops. Saskia is also a guest senior researcher at the Erasmus School of Health Policy & Management (ESHPM) at Erasmus University Rotterdam.
Bertalan Németh, PhD
ISPOR CEE Consortium Executive Committee and Syreon Research Institute, Budapest, Hungary
Bertalan Németh PhD graduated from the Corvinus University of Budapest (MSc in Quantitative economics and Operation research), the Eötvös Loránd University (Pharmaceutical economics and drug policies), and the Semmelweis University School of PhD Studies. Between 2010 and 2015 he was a health economist at the Hungarian HTA office. Since August 2015 Bertalan has been a Senior Health Economist, and since 2019 a Principal Researcher at Syreon Research Institute. Bertalan is responsible for strategic consulting, and he is involved in various projects that model for economic evaluation in health, health technology assessment and health statistics as well. Bertalan is the Past President of ISPOR Hungary Chapter, and Chair of the ISPOR CEE Consortium. He was a participant in the international EUnetHTA project, the ISPOR HTA Roundtable Europe, and the Scientific Committee of the META Conference. Bertalan was also a faculty member of the global ISPOR HTA Training, and was the module leader of Health Technology Assessment for the MSc program at Eötvös Loránd University.
Relevance of Using International Real-World Data in Regulatory and HTA Decision Making
In-person
ISSUE: - International randomized controlled trials are common and regulators (such as the FDA) acknowledge the globalization of drug development and trial conduct. However, it is not always feasible to study large populations of patients within each country where the manufacturer is seeking access. This challenge is present in RCTs and real-world data studies. While country-specific RWD exists, there are often challenges in accessing this data for commercial purposes. The ideal situation is submitting within country data to demonstrate effectiveness, safety, and value, but regulatory and HTA agencies understand the limitations and barriers to access. These decision-makers have signaled receptivity to international RWD (i.e., RWE from outside their country) if clinical practice, care delivery, healthcare systems, and patient populations are similar and data are reliable and relevant. Currently, there is a lack of guidance on what constitutes sufficient similarity in healthcare systems. This panel will discuss the ideal settings to use international RWD and gaps in current guidance from decision-makers. Panelists will also debate a working definition of “similarity” between healthcare systems and criterion needed to demonstrate generalizability.
OVERVIEW: - The moderator will present an overview of the current decision-maker recommendations on using international RWD and identify gaps in the recommendations. Each panelist will briefly present their perspective on the topic and their recommendations for closing the gaps in current guidance. The moderator will facilitate the debate on what “similarity between healthcare systems” should look like and how this may vary (e.g., by disease condition). The moderator will also incorporate questions from the audience to enrich the debate.
Moderators
Ashley Jaksa, MPH
Aetion, Inc., Boston, MA, USA
Panelists
Rami Ben-Joseph, PhD
Jazz Pharmaceuticals, New York, NY, USA
Dr. Ben-Joseph is an accomplished leader with a proven track record of demonstrating the value of pharmaceuticals, medical devices, and diagnostic services in diverse therapeutic areas. Dr. Ben-Joseph has expertise in health outcomes, pharmacoeconomics, epidemiology, market-access, pricing and reimbursement of pharmaceuticals and medical devices. Dr. Ben-Joseph heads Big Data Real World Evidence at Jazz Pharmaceuticals.
Kelvin K.W. Chan, MD, MSc, PhD
Sunnybrook Odette Cancer Centre, Toronto, ON, Canada
Pall Jonsson, BSc, PhD
National Institute for Health and Care Excellence (NICE), Manchester, LAN, United Kingdom
Pall Jonsson is Programme Director at the National Institute for Health and Care Excellence (NICE) where he heads up Data and Analytics. His team has a strategic role in ensuring NICE is at the forefront of harnessing new and emerging opportunities for using real world data to inform NICE’s guidance to the health and care sectors.
Before joining the Data and Analytics team, he was Associate Director for Science Policy and Research, responsible for NICE’s portfolio of international research projects in areas such as big data and real-world evidence. Pall has a PhD in bioinformatics from the University College London. Prior to joining NICE, he worked in academia, biotech and the pharmaceutical industry.
Are We Capturing the Multidimensional Value of Therapies for Rare Diseases Through the QALY?
In-person
ISSUE: Are CEA and QALY methodology resulting in inaccurate valuation with unintended consequences of inappropriate recommendations? Is QALY scientific basis refutable in general, or for Rare Diseases (RDs), specifically? What are the alternatives?
OVERVIEW: Quality-adjusted life-years (QALYs) have been used since the 1980s as a standard health outcome measure for conducting cost-utility analyses. It is well-documented that different QALY estimates can be obtained by simply changing the utility assessment method and that most of the assumptions in Rare Disorders effectiveness and projections are inappropriate.
The panel will share the experiences and views of major stakeholders (patients, decision makers, experts and industry) about the challenges associated with current HTA practices in the use of QALY to assess the value of RDs care. Barriers and enablers for the incorporation of methodological alternatives or complementary sources of evidence in HTA processes will be also discussed.
Moderators
Alicia Granados, MD
Sanofi, Barcelona, Spain
Dr. Alicia Granados is serving at Sanofi Genzyme as Head of Global Rare disease Medical Scientific Advocacy. She joined Genzyme in 2011 as Global Head of HTA scientific strategy with special focus in providing strategic direction in evidence generation, with the aim of contributing to optimize both effectiveness and efficiency of integrate evidence generation plans.
Dr. Granados was responsible for the creation and direction of Catalan Agency for Health Technology Assessment and Research (CAHTAR) in 1991. She was a founding member of INAHTA, the first international HTA network; as well as the first HTA joint initiative in Europe, EUR-ASSESS, predecessor of EUnetHTA. She is also former President of the International Society for Technology Assessment in Health Care (ISTAHC) and the Chair of the Committee for the Creation of a new HTA Society: HTAi, becoming the first HTAi president in 2003. Since January 2022 she is Chair of HTAi Policy Forum Advisory Committee .
Dr. Granados has been temporary advisor of several UN agencies on Evidence Based Health Care, and Acting Regional Advisor of WHO European Office, leading the Health Evidence Network. She has been an Associate Professor of Medicine at the University of Barcelona and has more than 60 scientific and policy articles published. Dr. Granados is former President of Autonomous University of Barcelona’s Board of Trustees.
Alicia Granados MD, PhD PH. Universidad de Barcelona, Catalunya España
Panelists
Ariel Beresniak, MD, MPH, PhD
Data Mining International, Geneva, Switzerland
Ariel Beresniak is an international expert in modelling and decision making in life sciences. He graduated as a physician with a 5 years specialization in Public Health from the Faculty of Medicine at University of Marseilles (France). Following his medical studies, he obtained his Master’s degree in Economic research and his PhD in Applied Mathematics in Economics at the Claude Bernard University in Lyon (France). Subsequently, he obtained two certificates in Healthcare Evaluation at the Harvard School of Public Health (USA), followed by an Accreditation to Supervise Research (France).
Michael Drummond, MCom, DPhil
Centre for Health Economics, University of York, York, United Kingdom
Michael Drummond, BSc, MCom, DPhil is professor of Health Economics and former Director of the Centre for Health Economics at the University of York. His particular field of interest is in the economic evaluation of health care treatments and programmes. He has undertaken evaluations in a wide range of medical fields including care of the elderly, neonatal intensive care, immunization programmes, services for people with AIDS, eye health care and pharmaceuticals. He is the author of two major textbooks and more than 700 scientific papers, and has acted as a consultant to the World Health Organization and the European Union. He has been President of the International Society of Technology Assessment in Health Care, and the International Society for Pharmacoeconomics and Outcomes Research. In October 2010 he was made a member of the National Academy of Medicine in the USA. He has advised several governments on the assessment of health technologies and chaired one of the Guideline Review Panels for the National Institute for Health and Care Excellence (NICE) in the UK. He is currently Co-Editor-in-Chief of Value in Health and has been awarded 3 honorary doctorates, from City University (London), Erasmus University (Rotterdam) and the University of Lisbon.
Sheela Upadhyaya, MSc
NICE - National Institute for Health and Care Excellence, London, LON, United Kingdom
Sheela Upadhyaya is currently the Rare Disease Strategic Advisor at NICE having recently led the activities from the NICE team on the Accelerated Access Collaborative. Her passion to secure high quality care outcomes for patients with rare diseases developed over 15 years ago as a commissioner for the Highly Specialised services in the NHS and more recently she held the role of Associate Director Highly Specialised Technology program at NICE.
Sheela has participated in several European projects exploring the challenges in evaluating rare diseases. such as ORPH-VAL European Working Group for Value Assessment and Funding Processes in Rare Diseases. She is chair elect for the ISPOR Rare Disease Special Interest Group.
Durhane Wong-Rieger, MA PhD
Canadian Organization for Rare Disorders, Toronto, ON, Canada
Durhane Wong Rieger, PhD, is the President & CEO of the Canadian Organization for Rare Disorders (CORD). She is also the President & CEO of the Institute for Optimizing Health Outcomes (Canada), Chair of the Consumer Advocare Network and Chair of Canadian Heart Patient Alliance.
Advancing Research on Emotional Well-Being: Introducing a Family Well-Being Research Network
Virtual
Level: Foundational
PURPOSE To introduce the research community to the Family Well-being Research Network’s research resources and funding opportunities, to catalyze research through this and a larger NIH effort. DESCRIPTION This breakout session will present a newly-launched emotional well-being research network. FAM-NET, a collaboration of the Universities of Michigan, Pittsburgh, and Harvard, sponsored by NIH, brings together scholars investigating the interdependence of health and well-being within and among family members across the lifespan, from child well-being within a family to older adults’ well-being among family members and relatives. Emotional well-being, which includes life satisfaction, life purpose, and positive emotions, is a key public health target and an integral element of decision making. Likewise, family well-being includes the resilience and support of a family unit beyond its resources or function of its individual members. The measurement of family well-being requires both careful conceptualization of "family" and measures that are not merely the aggregation of a family's individual members’ well-being. Critical knowledge gaps exist in both understanding the research on the role of emotional well-being in health and in measuring well-being outcomes, which constrain the rigorous incorporation of emotional well-being into clinical and policy decisions. Both emotional well-being and family well-being can be influenced by policy decisions and are often considered when an individual makes decisions about healthcare. FAM-NET’s activities will foster transdisciplinary collaborations to create, launch, and sustain a new generation of well-being researchers and innovative research activities across the lifespan, with a special emphasis on measuring family well-being and child quality of life. We will describe our web-based resources designed to aid access to measurement tools and approaches, our research funding in the form of pilot project grants, and our mentorship activities through our Research Scholar Corps. We will invite attendees to become involved in FAM-NET and answer questions about our programs.
Panelists
Janel Hanmer, MD, PhD
University of Pittsburgh, Pittsburgh, PA, USA
Janel Hanmer, MD, PhD, is an Associate Professor of Medicine and the Assistant Dean of Medical Student Research at the University of Pittsburgh. Her research has established national normative values, evaluated mode of administration, and tested different methods to model multiple health conditions in health-related quality of life measures. Her recent work has developed the PROMIS-Preference (PROPr) score. In addition, as the Medical Director for Patient Reported Outcomes at UPMC, she evaluates the impact and use of patient reported outcomes in clinical settings.
Lisa A. Prosser, MS, PhD
University of Michigan, Ann Arbor, MI, USA
Discussion Leaders
Eve Wittenberg, PhD
Harvard School of Public Health, Boston, MA, USA
Three speakers will present this session, as the Principal Investigators of the FAM-NET Family Well-Being Research Network: Lisa Prosser, PhD, Janel Hanmer, MD, PhD, and Eve Wittenberg, PhD. Dr. Prosser is the Marilyn Fisher Blanch Research Professor of Pediatrics and the Director, Susan B. Meister Child Health Evaluation and Research Center, Department of Pediatrics at the University of Michigan, as well as holds administrative roles within the University. Dr. Hanmer is Associate Professor of Medicine and Medical Director of the University of Pittsburgh Medical Center Patient Reported Outcomes Center at UPMC. Dr. Wittenberg is Senior Research Scientist in the Center for Health Decision Science at the Harvard TH Chan School of Public Health. The PIs have complementary areas of expertise in outcomes measurement, including pediatric/child well-being, family and caregiver effects of illness, and psychometrics of quality of life/outcomes/well-being metrics. All have published extensively in the field of outcomes research and economic evaluation. This collaboration is a 4-year project funded by the National Institutes of Health as part of a broader effort to advance the science of measurement of emotional well-being.
Spotlight Session
Trial Emulation with RWD--Evidence on Feasibility, Challenges, and Opportunities
In-person & Virtual
Results of RWD-based comparative effectiveness or safety studies are now being considered by HTA agencies and other decision-making bodies. Yet there remain concerns about the validity of this type of evidence and questions of when to use it to inform decision-making. The target trial framework has begun to emerge as a preferred approach. Some major recent research efforts such as OPERAND and RCT-DUPLICATE emulated published randomized controlled trials using the target trial framework in an effort to understand when RWD-based studies can produce the same results. This session will include highlights of RCT-DUPLICATE and OPERAND and discussion of important learnings, advantages, and challenges of target trial emulation.
Moderators
Marc Berger, MD
Marc L. Berger, LLC, New York, NY, USA
Marc L. Berger, MD, is a semi-retired, part-time consultant and scientific advisor. Until July 2017, he was Vice President, Real World Data and Analytics (RWDnA) at Pfizer, Inc. Marc has held senior-level positions in industry including Executive Vice President and Senior Scientist at OptumInsight; Vice President, Global Health Outcomes at Eli Lilly and Company; and Vice President, Outcomes Research and Management at Merck & Co., Inc.
He currently serves as advisor to a number of pharmaceutical and health data analytics companies. Additionally, Marc is a Special Advisor for Real World Evidence to the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) contributing to its ongoing efforts and that of other organizations such as the Duke-Margolis Center for Health Policy to promote best practices in the creation of real-world evidence (RWE). Marc has written or co-written more than 130 peer-reviewed articles, book chapters, and other publications on a range of topics including health services research, outcomes research, health economics, and health policy. He received the Donabedian Lifetime Achievement Award from ISPOR in 2019.
Panelists
William H. Crown, PhD
Brandeis University, Waltham, MA, USA
Dr.Crown is a Distinguished Research Scientist in the Heller School of Social Policy and Management, Brandeis University. He is an internationally recognized expert in real world data analysis, focusing upon research designs and statistical methods for drawing causal inferences from transactional health care datasets such as medical claims and electronic health records. Dr. Crown was 2013-14 President of ISPOR and currently co-chairs the ISPOR Task Force on Machine Learning. He is particularly interested in the intersection of machine learning and causal inference methods, as well as transparency in the conduct and reporting of empirical health care research.
Seamus Kent, PhD
National Institute for Health and Care Excellence (NICE), London, LON, United Kingdom
Seamus is a Senior Adviser in Data & Analytics at NICE. He is leading the development of a framework for the use of data and analytics in the development of NICE guidance.
Shirley Wang, PhD
Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA
Dr. Wang is an Associate Professor at Brigham and Women’s Hospital, Harvard Medical School. She has led 2 joint task forces between ISPOR and the International Society of Pharmacoepidemiology (ISPE) focused on real-world evidence for healthcare decision-making. Dr. Wang directs the REPEAT Initiative, a non-profit program with projects aimed at improving transparency, reproducibility and robustness of evidence from healthcare databases and co-leads RCT-DUPLICATE, a series of projects designed to inform when and how real-world data analyses can draw causal conclusions.
Concurrent Breakout Session 8
The Role of Real-World Evidence in Regulatory Evaluation of Medical Devices: A Global Review
In-person
Level: Foundational
PURPOSE: To discuss the developments of using real-world evidence (RWE) to support regulatory evaluation on medical devices in US and China.
DESCRIPTION: Randomized controlled trials (RCT) has been considered gold standard to demonstrate the safety and efficacy of a medical device. However, practical limitations require alternative approaches to RCT and increased flexibility in trial design and statistical analysis. There are new developments in US (RWE guidelines) and China (free trade pilot zone) supporting the regulatory decisions for devices.
Dr. Du will discuss common issues in regulatory submissions using RWE in US. Quality of RWE varies by device type, sponsor experience and RWD sources. FDA assesses RWD relevance and reliability to determine whether RWE derived from particular RWD is qualified. Sponsor should clearly demonstrate its RWE fits for the regulatory purpose with acceptable quality. Dr. Shi will analyze the RWE test cases by the National Evaluation System for health Technology (NEST) and provide insights about how to efficiently consolidate RWE from clinical registries, EHRs, claims, and other sources to inform device development and evaluation, and to support regulatory decision-making throughout lifecycle. In China, the policy environment is evolving towards the use of RWE to support regulatory decision-making for devices. Recently, a unique opportunity has been introduced for devices to gain faster access by leveraging RWE generated in a free trade pilot zone in Bo’ao Lecheng, Hainan Province. A successful Bo’ao case will be shared where safety and effectiveness of XEN
® in Chinese patients with refractory glaucoma were collected and RWE were generated to enable the assessment of ethnic differences to treatment with XEN® and gain regulatory approval. With its continuing development, we anticipate more successful approvals of innovative devices and drugs. The workshop will benefit the audience to understand new developments, nuances, opportunities, and challenges in using RWE with regulatory pathways.
Discussion Leaders
Lizheng Shi, PhD, MA, MsPharm
Tulane University School of Public Health and Tropical Medicine, New Orleans, LA, USA
Lizheng Shi, PhD, MsPharm, MA, is Regents Professor and Interim Chair in the Department of Health Policy and Management at the School of Public Health and Tropical Medicine of Tulane University, and Clinical Faculty in Tulane’s Department of Medicine (Section of Endocrinology) and Department of Psychiatry. He is the founding director of Tulane’s Health Systems Analytics Research Center.
Discussants
Carol Bao, PhD
AbbVie, Inc., Long Grove, IL, USA
Carol Bao is the Vice President, HEOR Strategy for Neuroscience, Eye Care and General Medicine at AbbVie, Inc. She joined the company in 2008 as Manager, HEOR and has since led HEOR support for different therapeutic areas with increasing responsibilities for Immunology, Virology/Hepatology, Neuroscience and Specialty Products. Carol assumed her current role in November 2022. Prior to joining AbbVie, she was Senior Pharmacoeconomist at Abt Associates Inc. She has a Ph.D. in Economics from the University of Illinois at Chicago and a B.A. in Economics from Fudan University, China.
Dongyi (Tony) Du, MD, PhD
U.S. Food and Drug Administration, Silver Spring, MD, USA
Mei Yang, Ph.D.
Happy Life Technology, Short Hills, NJ, USA
Dr. Mei Yang is now the VP of iHS Global from Happy Life Technology, an affiliate of Yidu Tech Inc, which provides leading data science and real-world evidence generation for life science companies.
Mei received her PhD in Biostatistics from Boston University. She started her industry career in AbbVie, where she worked as the Global Health Economics and Outcomes Research (GHEOR) lead to support the launch of Humira in treating patients with inflammatory bowel disease. Subsequently, Mei worked in Daiichi Sankyo in the pain area, where she gained plenty experience on Patient Reported Outcomes (PROs) and Phase III clinical development. She joined Merck in 2015 as a Director of GHEOR in Merck, leading a cross-functional team to support late phase clinical development and drug launch in cardiovascular and diabetes area. She has developed a robust understanding of physicians, patients, payers, and their decision making systems; implemented innovative approaches to healthcare research and analytics; and became a results-oriented strategist and an expert in providing scientific insights and improving business impact through the utilization of value-based evidence and access strategies.
Since 2009, Mei has been focusing on real world evidence (RWE), health economics, and market access in the pharmaceutical industry and became a lead with deep insights into evolving health care delivery and reimbursement systems and the evidentiary needs of diverse customer groups across major global markets. Mei has published about 40 articles in high tier medical journals in various disease areas and is continuously publishing new researches. With over 11 years of experience in HEOR and RWE, Mei is devoted to this globally evolving environment and has broad interest in Big Data, AI technology, healthcare policy, and market access.
Engage for What? Rethinking the Purpose of Stakeholder Engagement in Healthcare Research and Health Technology Assessment (HTA)
In-person & Virtual
Level: Intermediate
PURPOSE
:
Identify gaps in existing stakeholder engagement efforts/models to inform healthcare research or HTA Highlight the importance of using the ultimate goals to guide the process of engagement Showcase some practical examples of how this might be done DESCRIPTION
: There have been increasing efforts to apply a stakeholder engagement approach to elicit inputs from diverse stakeholders in healthcare research and HTA in recent years. As the HEOR community continues to broaden our outreach to different stakeholders and develop our toolkit in stakeholder engagement, it is important to review our existing models of stakeholder engagement and ensure that such approaches are designed to be fit for purpose. In this workshop, Ms. Hyde will first discuss the importance of stakeholder engagement and introduce examples of existing models of stakeholder engagement (10 minutes). Ms. Valentine will share experiences and reflections from participating in stakeholder engagement efforts to inform healthcare research and HTA through the patient perspective (10 minutes). Dr. Concannon will then review the shortcomings of existing models for stakeholder engagement in HTA, and describe a fit-for-purpose approach to meaningful stakeholder engagement that can align engagement with research aims (15 minutes). Lastly, using IVI’s ongoing effort to develop an open-source economic model in major depressive disorder as an example, Ms. Bright will share key learnings on how meaningful stakeholder engagement might improve the relevance of economic models in HTA and illustrate how IVI plans to evaluate such impacts (15 minutes). Insights from the workshop are applicable across all stakeholders: researchers will gain insights into designing and using an effective stakeholder engagement approach in applying methods to inform HTA; other stakeholders including patient groups, innovators, and payers will gain insights on collaborating with researchers in the engagement process to ensure that stakeholder feedback can lead to meaningful engagement that will ultimately benefit those that receive care.
Discussion Leaders
Anna Hyde, MA
Arthritis Foundation, Silver Spring, MD, USA
Anna Hyde is the Vice President of Advocacy and Access at the Arthritis Foundation. She oversees both the federal and state legislative programs, in addition to grassroots engagement. Her focus is to raise the visibility of arthritis as a public health priority, build support for federal and state legislation that ensures access to affordable, high-quality health care, and enhance patient engagement in the policy-making process. Anna previously served as Senior Director of Advocacy and Access, managing the federal affairs portfolio and overseeing the state advocacy team.
Prior to joining the Arthritis Foundation in 2014, Anna worked as Senior Manager for Federal Affairs at the American Congress of Obstetricians and Gynecologists, where she managed a portfolio of issues including appropriations, physician workforce, and health IT. She began her health policy career as a Congressional Fellow for Energy and Commerce Committee members, where she drafted legislation and staffed Committee activities. Anna received a BA in History from Southern Methodist University, and taught junior high and high school history before moving to Washington D.C. in 2007 to pursue an MA in Political Science from American University.
Discussants
Jennifer Bright, MPA
Innovation and Value Initiative, Alexandria, VA, USA
Jennifer L. Bright, MPA is Chief Executive Officer of the Innovation and Value Initiative (IVI), a nonprofit research organization focused on improving patient-centered methods and practice in value assessment. Ms. Bright is also President of Momentum Health Strategies, a consultancy through which she advises clients on health policy, advocacy and patient engagement, business strategy, and organizational development and leadership. Ms. Bright is Board Chair-Elect of Mental Health America and an editor for the American Journal of Accountable Care.
Thomas Concannon, PhD
RAND Corporation, Boston, MA, USA
Thomas Concannon is a senior policy researcher at the RAND Corporation, assistant professor of medicine at Tufts University School of Medicine (2006-present), and Codirector of Stakeholder and Community Engagement at Tufts Clinical and Translational Science Institute (2015-present). For more than 25 years, Concannon has collaborated with patients, clinicians, and other stakeholders in health care and research. Concannon pursues two major research interests: (1) delivery, access, and use of specialty care, including in burns, cardiovascular, and orthopedic health services, (2) involvement of patients, clinicians, and other stakeholders in clinical and translational science. He is committed to improving the usefulness of research in policy decisions. He leads several large evaluations of PCORI, CMS, CMMI, and CDC programs and service delivery models. He has published frameworks aimed at improving the translation of bio-medical research through pragmatic and stakeholder-engaged study.
Concannon earned his Ph.D. in health policy at Harvard University and his M.A. in political science at McGill University.
Ashley Valentine, MRes
Sick Cells, Washington, DC, USA
Ashley co-founded Sick Cells with her brother, Marqus, in 2017 to educate, advocate, and raise awareness for sickle cell disease (SCD) through local, state, and federal legislative advocacy. Her past experience includes research analysis, data collection, grant writing, and business development. She conducted her Master's research on disparities in healthcare for people living with SCD, and continue to apply those skills to her work as the President of Sick Cells.
11:00 AM - 11:30 AM
Break
In-person
11:30 AM - 12:45 PM
Plenary Session 3
Closing Remarks
CEO Welcome
Nancy Berg, ISPOR CEO & Executive Director
Program Committee Co-Chair Welcome
Ran Balicer, MD, PhD, MPH, ISPOR 2022 Program Committee Co-Chair
Eberechukwu Onukwugha, PhD, ISPOR 2022 Program Committee Co-Chair
Speaker
Kat Bissett
ISPOR, Burke, VA, USA
The Patient (Finally) at the Center: How Can We Leverage Digital Data to Make Patient-Focused Adoption, Reimbursement and Management Decisions?
The increasing capture of patient-centric health data through clinical, transactional, and surveillance systems holds much promise in terms of tailoring diagnostic and treatment paradigms to the individual circumstances each patient is facing, whether in the form of comorbidities, stage of illness, or even socioeconomic factors. To be most efficient in healthcare systems, however, payers and health technology assessment bodies must also be able to use these data to move beyond evaluation at the level of the “average” patient and develop more customized approaches – can they? At the same time, more specific patient information comes with privacy concerns since many datasets in use or under development capture patient data without consent or input from patients about their uses. Patients and the larger advocacy community not only have their own views on the best use of their data, they may also be able to provide data through personal apps and other real-time data collection methods. In addition, some of these considerations may vary between higher- vs. lower-income countries. The latter may have fewer privacy protections but also the potential for outsized benefits from digital health applications. This session will consider how to make the best use of digital health data to target access to health technologies for the patients who would most benefit, while providing HTA organizations a clear opportunity to make direct use of real-world evidence, all in a way that will maintain appropriate patient engagement, privacy protections, and data governance.
Moderators
Daniel Ollendorf, PhD
Tufts Medical Center, Boston, MA, USA
Daniel Ollendorf, PhD is Director of Value Measurement and Global Health Initiatives at CEVR and Assistant Professor of Medicine at Tufts University School of Medicine. Dr. Ollendorf’s research interests include expanding the use of health technology assessment (HTA) and economic evaluation in low- and middle-income countries, as well as refinement and use of value assessment tools in the United States and other high-income settings. Prior to joining CEVR, Dr. Ollendorf was Chief Scientific Officer for the Institute for Clinical and Economic Review (ICER) for over 10 years, where he oversaw the broader HTA process and managed relationships with multiple stakeholders and research collaborators.
Dr. Ollendorf is non-resident Fellow in Global Health at the Center for Global Development, and currently serves as the Chair of the Health Technology Assessment International (HTAi) Global Policy Forum. He received his PhD in Clinical Epidemiology from the University of Amsterdam, and an MPH in Health Policy & Management/Epidemiology from Boston University.
Richard Willke, PhD
ISPOR, Lawrenceville, NJ, USA
Dick became ISPOR’s first chief science officer in April 2016, following nearly 25 years in the pharmaceutical industry with Pfizer and its legacy companies. In his CSO role at ISPOR, Dick’s responsibilities are to develop, lead, support, and direct strategic initiatives related to research, scientific, and content priorities to accomplish the organization’s mission to promote health economics and outcomes research excellence to improve decision making for health globally. While with Pfizer, his final position was Vice President, Outcomes & Evidence, lead for Cardiovascular /Metabolic, Inflammation & Immunology, the last in a succession of HEOR group lead roles. He received a PhD in economics from Johns Hopkins University in 1982, concentrating in econometrics and labor economics. Prior to joining Pfizer’s legacy company Upjohn in 1991, he was a member of the economics faculty at Ohio State University as well as a senior economist at the American Medical Association Center for Health Policy Research.
Dick has served on the ISPOR Board of Directors (2007-09), was chair of the ISPOR Institutional Council in 2010, and was co-chair of the ISPOR Good Research Practices Task Force on Cost-Effectiveness Analysis in Randomized Clinical Trials in 2003-2005 as well as its 2014-15 reprise to revise and update that Report. He has co-taught many ISPOR short courses on this topic as well as on “Transferability of Cost-Effectiveness Data between Countries.” He was also a member of the Health Outcomes Committee of PhRMA from 1998-2009, having been its chair from 2002-2004. He has served as a co-editor for Value in Health, on the editorial board for Farmeconomia, on AHRQ, NIH, and PCORI project review study sections, and is a member of the Ohio State University Economics Advisory Board.
Prior to joining industry, Dr Willke served as department director in the Center of Health Policy Research at the American Medical Association and held research and teaching positions at The Ohio State University.
Dr Willke earned a PhD and MA in economics from Johns Hopkins University. He has authored more than 80 scholarly publications that examine the science and methodologies of health economics and outcomes research.
Speakers
Anurag Agrawal, PhD
CSIR Institute of Genomics and Integrative Biology, Delhi, India
Professor Anurag Agrawal is an acclaimed Indian physician-scientist. His broader interests are in a new vision of health and healthcare seen through the lenses of emerging technologies such as genomics or artificial intelligence. He co-chaired the Lancet and Financial Times commission on Governing Digital Health Futures, chairs the SARS CoV-2 viral evolution advisory group at WHO, and serves on multiple national and international expert groups for emerging technologies.
Cat Davis Ahmed, MBA
Family Heart Foundation, Pasadena, CA, USA
Cat Davis Ahmed is Vice President for Policy and Outreach for the Family Heart Foundation, where she works with individuals living with, or at risk for, early cardiovascular disease due to inherited lipid disorders (Familial Hypercholesterolemia and elevated Lipoprotein(a)) and the medical professionals who treat them. She is a coauthor on publications in the Journal of the American College of Cardiology, Circulation, and Atherosclerosis. Cat is a member of the American Heart Association’s Atherosclerosis, Hypertension, and Obesity in the Young Committee of the Council on Cardiovascular Disease in the Young and Co-Investigator on the NIH-funded “Identification Methods, Patient Activation, and Cascade Testing for FH” (IMPACT-FH) study to improve detection of, and family screening for, FH. She speaks at national medical conferences, including the American Heart Association Scientific Sessions, ISPOR, and the Global Cardiovascular Clinical Trialists Forum. As someone who has FH herself, she knows first-hand the impact the disorder can have on individuals and families. The Family Heart Foundation is a non-profit, patient-centered, research and advocacy organization dedicated to increasing the rate of early diagnosis and encouraging proactive treatment of inherited lipid disorders in order to prevent premature heart disease. Cat holds a BA from Union College and an MBA from the Yale School of Management.
Khaled El Emam
University of Ottawa, Ottawa, ON, Canada
Dr. Khaled El Emam is the Canada Research Chair (Tier 1) in Medical AI at the University of Ottawa, where he is a Professor in the School of Epidemiology and Public Health. He is also a Senior Scientist at the Children’s Hospital of Eastern Ontario Research Institute and Director of the multi-disciplinary Electronic Health Information Laboratory, conducting research on privacy enhancing technologies to enable the sharing of health data for secondary purposes, including synthetic data generation and de-identification methods.
Khaled is a co-founder of Replica Analytics, a company that develops synthetic data generation technology, which was recently acquired by Aetion. As an entrepreneur, Khaled founded or co-founded six product and services companies involved with data management and data analytics, with some having successful exits. Prior to his academic roles, he was a Senior Research Officer at the National Research Council of Canada. He also served as the head of the Quantitative Methods Group at the Fraunhofer Institute in Kaiserslautern, Germany.
In 2003 and 2004, he was ranked as the top systems and software engineering scholar worldwide by the Journal of Systems and Software based on his research on measurement and quality evaluation and improvement. He held the Canada Research Chair in Electronic Health Information at the University of Ottawa from 2005 to 2015. Khaled has a PhD from the Department of Electrical and Electronics Engineering, King’s College, at the University of London, England.
Yvette Venable, BA
Institute for Clinical and Economic Review, Watertown, MA, USA
Yvette is the Vice President of Patient Engagement for the Institute of Clinical and Economic Review in the United States. In this newly created role, she leads the organization’s enhanced patient engagement program, serves as a dedicated resource to the patient community, and ensures that patient voices are driving ICER’s thinking and decisions.
Throughout her 20+ year career, Yvette has been a champion of strengthening the patient voice in health policy decision-making and health technology assessment. She spent 15 years in Europe working in global public affairs, advocacy, and patient access within life sciences companies, and has also held healthcare communications and market development roles with global consultancies in the USA.
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