Mon 22 Nov
16:00 - 17:00
On-Demand Podium Sessions
View anytime on this date, after 16:00, until January 2. Impact of the COVID-19 Pandemic: Healthcare Utilisation and Outcomes
Live
This session covers the latest research on impacts of COVID-19 on healthcare utilisation and outcomes using different data sources.
Moderator
Roisin Adams, MPharm, MSc, PhD
National Centre for Pharmacoeconomics, Dublin, Ireland
Dr. Roisin Adams is Head of HTA Strategy and External engagement for the NCPE. Dr. Adams led the HTA team for a number
of years before being seconded to the HSE to lead a new unit tasked with overseeing and managing high cost drugs in acute hospitals. Dr. Adams co-chairs the HTA domain of the Beneluxa initiative and oversees the EUNetHTA work of the NCPE. She also is a Director on the Board of the International Horizon Scanning Initiative. She has been awarded a number of grants from the Health Research Board and held advisory positions for Department of Health, the Health Information and Quality Authority and policy direction at EU level. In recent years she has led the COVID-19 specific assessments for the NCPE.
P22: COVID-19 Pandemic Impacts Volume of Evaluation & Management (E&M) Telehealth Visits within Community Oncology Practices
4:15PM - 4:30PM
Karhade M 1 , Mohammad N2 , Robert N3 , Wu N4 , Heller B5 , Alwardt S6 , Neubauer M6 , Smith H6 , Moore L7 1 Ontada, Houston, TX, USA, 2 Ontada, Cypress, TX, USA, 3 Ontada, Irving, TX, USA, 4 US Oncology Network, The Woodlands, TX, USA, 5 Southern Cancer Center, Mobile, AL, USA, 6 Ontada, The Woodlands, TX, USA, 7 Ontada, Shaker Heights, OH, USA
OBJECTIVES: The USA declared the COVID-19 pandemic a national emergency on 03/13/20. On 03/17/20, CMS expanded telehealth rules, allowing Medicare to cover telehealth visits like regular visits. This study aims to analyze the utilization of Evaluation & Management (E&M) telehealth options in community oncology pre and post pandemic. METHODS: Deidentified patient visits data were obtained from iKnowMed electronic health records between 01/01/18 to 05/24/2021 from 20 US Oncology practices. A combination of patient MRN and date was used as an identifier to report number of visits for all measures. Patient visits with modifiers –GT, –95, and –GQ were classified as telehealth visits. Visit dates without modifiers were defined as non-telehealth (in-office) visits. E&M visits were defined based on standard CPT codes. RESULTS: A total of 5,914,125 unique E&M patient visits were analyzed during the study period. Between Jan-2018 and Mar-2020 (pre-COVID-19), E&M visits rose from 30,000/week to 36,000/week (20%). Fewer than 0.01% of these visits were telehealth. By April 12, 2020, overall E&M visits had dropped 35%, but the telehealth visits had risen to 16%. Since then, the overall E&M visit count remained approximately 5% lower as compared to the pre-COVID-19 trend, and telehealth visits averaged approximately 6% thereafter. Corresponding to the 2nd wave, in Dec-2020 the telehealth proportion rose again to 10%. As of 05/23/2021, telehealth E&M visits represented approximately 5% of the total E&M visits within US Oncology practices. CONCLUSIONS: This study provides a timeline of how COVID-19 has impacted E&M visits and telehealth utilization among community oncology practices. The pandemic has led to an increase in E&M telehealth visits that may remain post pandemic. Continued research is necessary to monitor telehealth utilization and its impact on the quality of care, provider finances, and future of community oncology considering rising vaccination rates, CDC guidance, and public sentiment.
P21: Social Distancing and Trends in Influenza Hospitalization during the COVID-19 Outbreak: A Difference-in-Difference Analysis of German Claims Data
4:00PM - 4:15PM
Pacis S 1 , Maywald U2 , Wilke T3 , Ghiani M4 1 Cytel Inc, Berlin, BE, Germany, 2 AOK PLUS, Dresden, Germany, 3 IPAM e.V., Wismar, Germany, 4 IPAM, University of Wismar, Berlin, BE, Germany
OBJECTIVES : As COVID-19 spread worldwide, indicators of influenza activity in the Northern Hemisphere began to decline by mid-to-late February. In Germany, federal lockdown measures were introduced to contain the outbreak on 22/03/2020 (week 12). We used claims data from AOK PLUS, a regional sickness fund covering around half the population in Saxony and Thuringia (6.2 million inhabitants), to examine the trend of influenza hospitalizations in 2020 compared to 2019. METHODS : Using data from 01/01/2019 to 31/05/2020 (weeks 1-22), influenza hospitalizations were identified using ICD-10-GM codes J10-J11. We estimated changes in the number of influenza hospitalizations using a “difference-in-differences” model including variables for age group (<18, 18-44, 45-64, 65-79, 80+), gender, week, year, and outbreak status (interaction variable between year 2020 and week 12 or later). Adjusted incidence rate ratios (aIRRs) were estimated using Poisson regression with heteroskedasticity-robust standard errors. RESULTS : During weeks 1-22, we observed 5,174 influenza hospitalizations in 2019 and 2020. Influenza hospitalizations in 2020 showed similar trends until week 12 and then showed a relative decline compared to 2019. The average number of influenza hospitalizations per week during weeks 12-22 significantly decreased in 2020 compared to 2019 (1.6 vs. 5.2; aIRR: 0.45; 95% CI: 0.34-0.59; p<0.001). When stratified by age group, all groups except age 18-44 had a similar decrease in average influenza hospitalizations per week in 2020 compared to 2019, with large relative declines in patients age 80+ (2.2 vs. 5.8; aIRR: 0.36; 95% CI: 0.28-0.46; p<0.001) and children <18 (1.8 vs. 8.0; aIRR: 0.38; 95% CI 0.32-0.46; p<0.001). CONCLUSIONS : The number of influenza hospitalizations saw a relative faster decline in 2020 compared to 2019 after the introduction of federal lockdown measures in Germany, possibly due to the effectiveness of non-pharmaceutical interventions like social distancing and the use of facemasks.
P24: Telehealth Access and Use by the U.S. Medicare Population during the Pandemic
4:45PM - 5:00PM
Swenson T Des Moines University, Des Moines, IA, USA
OBJECTIVES: Telehealth access and reimbursement varied by payer and regionally prior to COVID-19. and its limited availability expanded in response to the pandemic. The health behavioral response by older adults to COVID-19 has varied over time with the geographic spread of the pandemic and affected access and utilization of medical services. The purpose of this paper is to examine changes in access to telemedicine in 2020 in response to the pandemic for the U.S. Medicare population. METHODS: The first two waves in June and October 2020 of the rapid response survey fielded by the Centers for Medicare and Medicaid Services (CMS) to track and monitor the effects of the pandemic within the U.S. Medicare population. With a panel sample size of 9686 Medicare beneficiaries, the calculated statistics use replicate weights to adjust for the complex survey sample design and balanced repeated replication using Fay’s adjustment of 0.3 for variance estimation. RESULTS: Nearly 45 percent of the Medicare population reported use of a telehealth appointment between June and October of 2020. The likelihood of using telemedicine increased for those with chronic conditions, such as depression, and for those with higher incomes and education. Medical practices were more likely to encourage telehealth visits for Medicare patients between March and June with 57 percent of the Medicare population reporting that their usual provider offered a telemedicine appointment to replace a regular office visit during the spring and 48% reporting the suggested telemedicine replacement from July through October 2020. Overall access to telehealth increased from 60% to 64% but varied by race/ethnicity, gender, Census regions, and rural status. CONCLUSIONS: Access to telemedicine services expanded for the U.S. Medicare population during the pandemic but usage varied by chronic disease status, socioeconomic and demographic factors, and geography.
P23: Change in Healthcare Utilisation and Inpatient Mortality in Patients Hospitalised with Heart Failure during the Coronavirus Pandemic in England: A Retrospective Cross-Sectional Study Utilising HES
4:30PM - 4:45PM
Jones D , Barham L Learna Ltd in partnership with the University of South Wales, Royston, UK
OBJECTIVES: This study quantifies change in healthcare utilisation and inpatient mortality of all adult patients hospitalised with Heart Failure in England during three coronavirus national lockdowns compared to the same time period in the previous year. METHODS: A retrospective cross-sectional study using the Hospital Episode Statistics (HES) database was conducted. All adults admitted to an English hospital with a primary diagnosis of I110 Hypertensive heart disease with (congestive) heart failure, I255 Ischaemic cardiomyopathy, I420 Dilated cardiomyopathy, I429 Cardiomyopathy unspecified, I500 Congestive heart failure, I501 Left ventricular failure and I509 Heart failure unspecified between 1st March 2019 and 28th February 2021 were included. Admissions, bed days and inpatient deaths of patients admitted between 1st March 2020 and 28th February 2021 (during pandemic) was compared with patients admitted between 1st March 2019 and 29th February 2020 (prior to pandemic). The difference in event count was used to test national changes and a P-value of ≤0.05 was used to test significance RESULTS: There were 140,035 heart failure admissions in the observational period, 64,770 during the pandemic and 75,265 prior to the pandemic, all data were analysed. There were reductions in admissions (69,555 vs 80,715, P<0.0000000000), bed days (586,430 vs 753,985, P=0.0000000000) and inpatient deaths (7,650 vs 8,305, P=0.0000002154) during the pandemic. CONCLUSIONS: There were significantly fewer admissions, bed days and inpatient deaths for patients admitted with heart failure during the coronavirus pandemic. Interpretation of this change is challenging as this may reflect unmet health needs as patients ‘put off’ seeking care. Further research is required to analyse the change in out of hospital healthcare utilisation, deaths in other settings and to explore potential for excess and latent morbidity and mortality that may result from reduced access to hospital services during the pandemic.
Methods and Controversies in the Evaluation of Oncology Products
Live
The evaluation of innovative oncology products often depends on evidence from a small number of landmark clinical trials. These trials often present challenges of short follow-up and lack of power to measure differences in overall survival. This session discusses recent work to handle these challenges and explore the degree of uncertainty around benefits and costs. Among the work presented are novel methods for survival prediction that synthesize clinical data (from trials and external sources) with expert opinion, validation of extrapolation assumptions, comparison of structural modelling methods, and decision makers' willingness to accept surrogate endpoints.
Moderator
Oriana Ciani, PhD
SDA Bocconi School of Management, Milan, MI, Italy
Oriana Ciani is Associate Professor of Practice at SDA Bocconi in Milan. She holds a MSc in Biomedical Engineering from Politecnico di Milano and postgraduate degree in Healthcare Management from Bocconi University. She received her PhD from the University of Exeter with a thesis focusing on the evaluation of surrogate end points. She has been 2020 Fulbright Research Scholar at Yale School of Medicine and Yale School of Public Health. Oriana's research interests are centred on the use of evidence synthesis techniques to inform policy decisions, health technology assessment (HTA) and healthcare policies evaluation.
P52: Surrogate Survival Endpoints: Are They Sufficient to Support Access?
4:45PM - 5:00PM
Doolub N1 , Dacosta RFD2 , Grosvenor A3 , Wang GD4 , Macaulay R 3 1 Precision Advisors, London, LON, UK, 2 PrecisionADVISORS, London, UK, 3 Precision Advisors, London, UK, 4 PrecisionAdvisors, London, UK
Immuno-oncology therapies (IOs) have revolutionised metastatic cancer management over the last decade. These are now being investigated in earlier stages of cancer, where surrogate survival endpoints such as relapse-free survival (RFS), disease-free survival (DFS), and pathological complete response (pCR) are needed to bridge the evidence gap prior to maturation of overall survival (OS) data. This research assesses surrogate survival endpoints in supporting access/reimbursement in early-stage cancers. Publicly accessible assessments of early-stage cancer therapies by seven HTA-bodies (HAS, G-BA, Medicinrådet, NICE, SMC, PBAC, CADTH) up to May 2021 were extracted. 52 assessments of 11 drug:indication pairings were identified, which utilised four surrogate primary endpoints (RFS, DFS, invasive DFS, pCR). 30 were fully reimbursed, 11 restricted reimbursement, 11 not reimbursed. Payer support was defined as being considered clinically/patient-relevant and/or a valid surrogate to OS. RFS was the best-supported surrogate endpoint by HTA bodies (7/7), followed by DFS (5/7) and IDFS (5/7), while pCR (2/7) was least well-supported. NICE and SMC were the most supporting of access using surrogate endpoints, followed by G-BA, while CADTH was least supporting. Assessments were most positive where the magnitude of the surrogate benefit were greater/showed a superior benefit-risk and where early data showed a likely long-term OS benefit. However, some assessments from NICE/G-BA were conditional and contingent on more mature follow-up data being provided, and PBAC imposed a flow-on restriction limiting retreatment with IOs as later line(s) of therapy. Several IOs already approved and reimbursed in the metastatic setting are being investigated in the early-stage setting using surrogate survival endpoints. These surrogate endpoints have been widely utilised for regulatory approval, and as shown here, HTA bodies are supportive of some surrogate endpoints based on their patient relevance and/or their validated OS correlation.
P49: Accuracy of Life Year Gains Predictions for CAR-T Therapy in the Long Term: An Analysis for Axicabtagene Ciloleucel in Refractory Large B-Cell Lymphoma
4:00PM - 4:15PM
Porteous A 1 , Gregori D1 , Hilton B2 1 Costello Medical, London, UK, 2 Costello Medical, Cambridge, UK
OBJECTIVES: Survival profiles for chimeric antigen receptor T cell (CAR-T) therapies commonly exhibit a plateau that may indicate a cure. This leads to challenges predicting long-term outcomes for novel CAR-T therapies when trial data are immature. This study retrospectively analysed the accuracy of overall survival (OS) extrapolations from interim data cuts in predicting realised long-term life years (LYs) for axicabtagene ciloleucel in patients with refractory large B-cell lymphoma. METHODS: Published OS data for axicabtagene ciloleucel from successive data cuts of ZUMA-1 (median follow-ups of 15.4, 27.1 and 51.1 months) were digitised. Standard parametric, spline (1–2 knots; normal, odds and hazard) and mixture cure models (MCMs) were fitted to the first two data cuts. Statistical fit was tested using Akaike and Bayesian information criteria (AIC and BIC). Cumulative LYs were estimated for each model over a 58-month time horizon, corresponding to the longest duration of published OS data. These projected LYs were then compared to realised LYs over this period. RESULTS: At the earliest data cut, MCMs provided the best predictions of realised LYs, with a mean absolute difference of 6.8% between predicted and realised LYs across MCM models (range: 0.5%–10.7%). Standard parametric extrapolations considerably underestimated realised LYs (mean absolute difference: 19.2%; range: 9.9%–28.0%), whilst spline models offered a mean absolute difference of 8.3% (range: 1.4%–13.2%). Similar findings were observed for extrapolations based on the second data cut (representing 11.7 months additional follow-up), but differences between model classes were less pronounced. CONCLUSIONS: MCMs may offer the best predictions of long-term survival for CAR-T therapies, particularly when only short-term data are available. Standard parametric models may be inappropriate to predict survival when extrapolating immature data, failing to capture the plateau in survival typical of CAR-T therapies. Further research is required to determine whether these findings are generalisable across CAR-T therapies and indications.
P50: Competing Risk and Multistate Model Compared to Partitioned Survival Model in Metastatic Non-Small Cell Lung Cancer
4:15PM - 4:30PM
Le Mezo A 1 , Kandel M2 , Caillon M3 , Chauny JV4 , Borget I5 1 Master 2 Market-Access et Evaluation Médico-Economique, Université Paris-Saclay, Sèvres, France, 2 IQVIA France, La Défense Cedex, France, 3 Amgen SAS, Boulogne Billancourt, 75, France, 4 Amgen SAS, Boulogne Billancourt, France, 5 Department of Biostatistics and Epidemiology, Gustave Roussy, Paris-Saclay University, Villejuif, France; Oncostat; GRADES, Paris-Saclay University, Châtenay-Malabry, France U1018, Inserm, Paris-Saclay University, “Ligue Contre le Cancer” labeled team, Chatenay-Malabry, 92, France
OBJECTIVES : Partitioned survival analysis (PartSA) is commonly used for economic evaluations in oncology. We compare PartSA to a competing risk semi-markov multi-state model (MSM) and investigate differences in estimated cost-effectiveness in metastatic non-small cell lung cancer from a French perspective. METHODS : To populate both models, pooled data from Checkmate 017 and 057 was digitized to reconstruct patient-level data from overall survival, progression-free survival and post-progression survival Kaplan Meier curves. Models consisted of three states: progression-free, progressed disease and death. Statistical analysis was performed under R to fit parametric survival models for the PartSA and to estimate state transitions for the MSM (package “mstate”). Costs and utilities were integrated in the model using values from nivolumab’s French HTA submission. Outcomes were discounted at 2,5% per annum over a 7-year time horizon. RESULTS : Both models produced similar results during trial period (15 months) but showed discrepancies during extrapolation, inducing different costs and quality-adjusted life years (QALYs) over time. Incremental life years gained (LYG) of nivolumab versus docetaxel were 1,11 LYG (PartSA) and 0,88 LYG (MSM). However, both incremental cost effectiveness ratios (ICER) were similar, 88 979€/QALY (PartSA) and 90 148€/QALY (MSM). Scenarios assessing different parametric extrapolations produced an average of 112 496€/QALY (PartSA) and 113 085€/QALY (MSM) with coefficients of variation (CV) estimated at 19,0% (PartSA) and 16,3% (MSM). Probabilistic ICER results were similar between models with CV estimated at 21,8% (PartSA) and 23,2%(MSM). CONCLUSIONS : This study provides new evidence on structural uncertainty. The MSM produced similar results to the PartSA for a 15 months follow-up. Ideally, both models should be tested to validate extrapolations and to ensure that the structural uncertainty is limited. However, when the time horizon is short (<10 years), the impact of the model choice on the ICER seems limited.
P51: Blended Survival Curves: A New Approach to Extrapolation for Time-to-Event Clinical Trial Data in Health Technology Assessment
4:30PM - 4:45PM
Che Z 1 , Baio G2 , Green N2 1 University College London, London, UK, 2 University College London, London, LON, UK
OBJECTIVES: Survival extrapolation is generally required in the cost-effectiveness analysis to estimate the survival benefit of a new intervention, due to the limited duration of randomized controlled trials (RCTs). Current techniques of extrapolation often assume constant treatment effect beyond the observed period in the RCT, which is implausible and highly influential in survival estimates for resource allocation decisions. The objective of this study is to develop a novel methodology based on “blending” survival curves as a possible solution. METHODS: We mixed a flexible Cox semi-parametric model conducted in Bayesian setting to fit the observed data and a parametric model either by prior assumptions or external data on the long-term expected behavior of the underlying survival curves. The two are “blended” into a single survival curve that is equivalent to the Cox model over the follow-up intervals and gradually approaching to the parametric model over the extrapolation period based on a weight function. The weight function and mixing area of the blended curve control the way the internal and external data sources influence the estimated survival. RESULTS: A 4-year follow-up RCT of rituximab in combination with fludarabine and cyclophosphamide (RFC) v. fludarabine and cyclophosphamide alone (FC) for the first-line treatment of chronic lymphocytic leukemia is used to illustrate the method. Two kinds of prior information, registry data and summary of clinical knowledge were respectively used for the long-term estimate. CONCLUSIONS: Long-term extrapolation with various assumptions of treatment effect may give significantly different estimated mean survival gains. The blending process allows a consideration of plausible scenarios, abandoning the over-optimistic constant treatment effect and provides sufficient flexibility. Not only internal but also external validity could be carefully considered since a wide range of external evidence can be used to inform the long-term estimate, including hard data from real world and clinical expert opinion.
Access to Care/Real World Evidence to Inform Decision Making
This session covers the use of real world evidence to inform decision making within innovative therapies, metastatic colorectal cancer, statin therapy and atopic dermatitis.
Moderator
Olga Ovcinnikova, MSc
MSD UK, Chorleywood, United Kingdom
Over 12 years of working in pharmaceutical industry, health economics consultancies and public health centers across Europe. I have experience across a wide range of therapeutic areas including oncology, vaccines, GI, mental health and blood disorders.
P2: Estimation of Metastatic Colorectal Cancer Patients Carrying BRAF Mutation Potentially Eligible to Targeted Therapy: A Real-World Evidence Study in Italy
4:15PM - 4:30PM
Degli Espositi L 1 , Sangiorgi D2 , Andretta M3 , Bacca M4 , Barbieri A5 , Bartolini F6 , Cavaliere A7 , Chinellato A8 , Ciaccia A9 , Cillo MR10 , Citraro R11 , Costantini A12 , De Francesco A11 , Enieri N8 , Ferrante F13 , Gentile S14 , Lavalle A14 , Mancini D4 , Mensurati M15 , Moscogiuri R16 , Pastorello M17 , Procacci C18 , Re D19 , Santoleri F12 , Serao Creazzola S20 , Tegon M8 , Ubertazzo L21 , Vercellone A22 , Perrone V23 1 CliCon S.r.l. Health, Economics & Outcomes Research, Bologna, Italy, 2 CliCon S.r.l. Health, Economics & Outcomes Research, Bologna, BO, Italy, 3 Azienda ULSS 8 Berica, Vicenza, Italy, 4 ASL Brindisi, Brindisi, Italy, 5 ASL Vercelli, Vercelli, Italy, 6 USL Umbria 2, Terni, Italy, 7 ASL Viterbo, Viterbo, Italy, 8 Azienda ULSS 3 Serenissima, Mestre (VE), Italy, 9 ASL Foggia, Foggia, Italy, 10 ASL Salerno, Salerno, Italy, 11 Azienda ospedaliero-universitaria Mater Domini, Catanzaro, Italy, 12 ASL Pescara, Pescara, Italy, 13 ASL Frosinone, Frosinone, Italy, 14 Direzione Generale per la Salute Regione Molise, Campobasso, Italy, 15 ASL Roma 3, Roma, Italy, 16 ASL Taranto, Taranto, Italy, 17 ASP Palermo, Palermo, Italy, 18 ASL BAT, Trani, Italy, 19 ASL Teramo, Teramo, Italy, 20 ASL Napoli 1, Napoli, Italy, 21 ASL Roma 4, Civitavecchia (RM), Italy, 22 ASL Napoli 3 SUD, Torre del Greco, Italy, 23 CliCon S.r.l. Health, Economics & Outcomes Research, Ravenna, RA, Italy
OBJECTIVES : Cancer treatments represent one of the most expensive items for the National Health System. In a limited-resource system, the introduction of costly and innovative oncological therapies makes it necessary to balance the innovation and the access to treatments based on patient eligibility. The study aimed to evaluate the possibility of identifying metastatic colorectal cancer (mCRC) patients carrying BRAF-gene mutation, potentially eligible to targeted therapy, by linking administrative and pathological anatomy (PA) databases. METHODS : A retrospective study was conducted across 2013-2019 in a sample of Italian Entities, using the data-linkage between administrative and PA databases. Data were reported per million of health-assisted individuals. CRC and mCRC patients [diagnosed by at least one hospitalization for CRC or mCRC (ICD-9-CM codes 153-154 and 196-197-198, respectively)], were screened. Mutational status of mCRC patients was identified by BRAF genetic test (procedure codes: 91.30.3/91.36.5/91.29.3/91.29.4). Data-linkage of these data with those from the administrative databases allowed the identification of mCRC patients carrying BRAF mutation (BRAF+ ). RESULTS : Overall, 4,666 CRC patients were identified, with an incidence (2019) estimated of 0.7/1,000 of health-assisted individuals. Among them, mCRC accounted for the 39% (N=1,818) of patients. The 50% (N=915) of mCRC patients had an outpatient test for BRAF. After the data-linkage between administrative and PA databases, 83% (N=765) of them performed the BRAF test, and 107 patients (14% of patients with BRAF test reported) were BRAF+ . CONCLUSIONS : These results reported an epidemiological scenario of CRC and mCRC-BRAF+ Italian patients in line with published data, showing that our methodology could be a supportive tool to identify eligible patients for targeted therapy. Furthermore, the use of PA database would allow to quantify patients with a specific genetic profile required to access to innovative therapies, thus enabling to estimate health-costs and to plan the pharmaceutical expenditure in a perspective of economic sustainability.
P4: Switching, Persistence and Adherence to Statin Therapy: A Retrospective Cohort Study Using the Australian National Pharmacy Data
4:30PM - 4:45PM
Talic S 1 , Marquina C1 , Zomer E1 , Lybrand S2 , Liew D3 , Ademi Z1 1 Monash University, Melbourne, Australia, 2 Amgen Australia, North Ryde, Australia, 3 Monash University, Melbourne, VIC, Australia
OBJECTIVES: Statins are widely prescribed for the primary and secondary prevention of cardiovascular disease, but their effectiveness is dependent on the level of adherence and persistence. This study aimed to explore switching, adherence and persistence among the Australian general population with newly dispensed statins. METHODS: A retrospective cohort study was conducted using a random sample of data from the Australian national prescription claims data. Switching, adherence to and persistence with statins were assessed for people starting statins from 1 January 2015 to 31 December 2019. Cox proportional hazard models were used to compare outcomes between different statins. RESULTS: A cohort of 141,062 people dispensed statins and followed over a median duration of 2.5 years were included. Of the cohort, 29.3% switched statin intensity, 28.4% switched statin type, 3.7% switched to ezetimibe and in 2.7%, ezetimibe was added as combination therapy during the study period. Overall, 58.8% discontinued statins based on the 90-day gap criteria, of whom 55.2% restarted. The proportion of people non-adherent was 24.0% at 6 months to 49.0% at 5 years. People on low and moderate intensity statins were more likely to discontinue compared to those on high-intensity statins HR 1.20, 95% confidence interval [CI] 1.09–1.31), (HR 1.28, 95%CI 1.14–1.42), respectively. Compared to maintaining same statin type and intensity, switching statins, which includes up-titration (HR 0.77, 95%CI 0.70 to 0.86) was associated with less likelihood of discontinuation after reinitiation. CONCLUSIONS: Long-term persistence and adherence to statins remains generally poor among Australians, which limits the effectiveness of these medicines and the consequent health impact they may provide for individuals. Switching between statins is prevalent in one third of statin users, although any clinical benefit of the observed switching trend is unknown. This, combined with the high volume of statin prescriptions, highlights the need for better strategies to address poor persistence and adherence.
P1: Data-Linkage Between Administrative and Pathological Anatomy Databases for the Identification of Lung Cancer Patients Eligible to Innovative Therapies
4:00PM - 4:15PM
Degli Espositi L1 , Sangiorgi D 2 , Andretta M3 , Bacca M4 , Barbieri A5 , Bartolini F6 , Cavaliere A7 , Chinellato A8 , Ciaccia A9 , Cillo MR10 , Citraro R11 , Costantini A12 , De Francesco A11 , Ferrante F13 , Gentile S14 , Lavalle A14 , Mancini D4 , Mensurati M15 , Moscogiuri R16 , Pastorello M17 , Procacci C18 , Re D19 , Santoleri F12 , Serao Creazzola S20 , Ubertazzo L21 , Vercellone A22 , Perrone V1 1 CliCon S.r.l. Health, Economics & Outcomes Research, Bologna, Italy, 2 CliCon S.r.l. Health, Economics & Outcomes Research, Bologna, BO, Italy, 3 Azienda ULSS 8 Berica, Vicenza, Italy, 4 ASL Brindisi, Brindisi, Italy, 5 ASL Vercelli, Vercelli, Italy, 6 USL Umbria 2, Terni, Italy, 7 ASL Viterbo, Viterbo, Italy, 8 Azienda ULSS 3 Serenissima, Mestre (VE), Italy, 9 ASL Foggia, Foggia, Italy, 10 ASL Salerno, Salerno, Italy, 11 Azienda ospedaliero-universitaria Mater Domini, Catanzaro, Italy, 12 ASL Pescara, Pescara, Italy, 13 ASL Frosinone, Frosinone, Italy, 14 Direzione Generale per la Salute Regione Molise, Campobasso, Italy, 15 ASL Roma 3, Roma, Italy, 16 ASL Taranto, Taranto, Italy, 17 ASP Palermo, Palermo, Italy, 18 ASL BAT, Andria (BT), Italy, 19 ASL Teramo, Teramo, Italy, 20 ASL Napoli 1, Napoli, Italy, 21 ASL Roma 4, Roma, Italy, 22 ASL Napoli 3 SUD, Torre del Greco, Italy
OBJECTIVES : The introduction of innovative and costly oncological therapies highlights the need for healthcare system to balance the innovation with the access to therapies based on patient eligibility. This study aimed to identify, through real-world data from administrative and pathological anatomy (PA) databases, patients with metastatic lung cancer carrying specific tumor markers, potentially eligible to innovative immunotherapy treatments. METHODS : The data-linkage between administrative and PA databases on a pool of Italian local Healthcare Entities (LHEs) was performed. Data were reported per million of health-assisted individuals. All patients diagnosed for lung cancer (ICD-9-CM code:162) from 2013-2019 were included. Metastatic disease has been diagnosed by using ICD-9-CM codes 196-197-198. The data integration with PA data-set was carried out to evaluate morphologic characteristics (M-80023; M-80413; M-80423) and the level of PD-L1 expression, required for the eligibility to specific immunotherapies. RESULTS : Overall, 4,387 lung cancer patients were included, with annual incidence of 0.7/1,000 health-assisted individuals for year 2019. Among them, 37% (N=1,625) presented metastasis, in line with published evidence indicating around 30-40% of lung cancer to present metastasis. Further analyses were performed in metastatic patients with a record in the PA database (N=365). The 87% (N=317) of them had non-small cell lung cancer, accordingly to the literature estimation, and 79% of patients were potentially eligible for immunotherapy since showed positivity to PD-L1 TPS ≥1%. CONCLUSIONS : The present study results are in line with epidemiological data reported by AIRTUM (incidence 0.7/1,000) and with international literature and showed how the applied methodology could represent a valuable tool for identifying patients eligible to new therapies. The use of PA data-set could allow the detection of patients with specific genetic profiles and their access to innovative medications. The quantification of potentially eligible patients would also allow budget impact estimation needed to plan the pharmaceutical spending by LHE.
Evaluating Individuals and Patients Preferences: Discrete Choice Experiments and Beyond
This session illustrates how discrete choice experiments (DCE) can be used to better understand individuals' or patients' preferences and discusses how alternative methods such as the probabilistic threshold technique may be better to estimate maximum acceptable risk.
Moderator
Anne E Spencer, BSc, MPhil, PhD
University of Exeter, Exeter, United Kingdom
P19: Assessing Heterogeneity in MAR: Methods and Models Beyond DCE
4:30PM - 4:45PM
Janssen E 1 , DiSantostefano R1 , Falahee M2 , Simons G2 , Englbrecht M3 , Radawski C4 , Raza K2 , Hauber B5 , Veldwijk J6 1 Janssen R&D, Titusville, NJ, USA, 2 University of Birmingham, Birmingham, UK, 3 Freelance, Eckental, Germany, 4 Eli Lily & Company, Indianapolis, IN, USA, 5 Pfizer, New York, NY, USA, 6 Erasmus University Rotterdam, Rotterdam, Netherlands
OBJECTIVES : Discrete choice experiments (DCEs) are robust stated-preference methods frequently used to estimate maximum acceptable risk (MAR) as a secondary outcome. However, DCEs provide sample-level estimates and explaining preference heterogeneity for MARs based on participant characteristics can be difficult. The study objective was to compare the capability of a DCE and a probabilistic threshold technique (PTT) to identify preference heterogeneity among MARs for preventive rheumatoid arthritis (RA) treatment. METHODS : Participants from 3 countries (United Kingdom (UK), Germany, and Romania, n = 2959) completed a DCE and PTT in random order. Participants made choices between treatments that reduced chance of developing RA but increased chance of three risks (mild and serious side effects, serious infection). For the PTT, interval regressions estimated MARs that accounted for age, education, numeracy, literacy, and RA family history. For the DCE, random parameters logit (RPL) models were used to calculate MARs for subgroups in which heterogeneity was identified in the PTT. RESULTS : The PTT identified preference heterogeneity for numeracy, literacy, and family history. Regarding these characteristics, the PTT identified statistically significantly different MARs (p<0.05) for at least one risk in at least two countries. The DCE identified preference heterogeneity for the chance of serious infection between UK participants with low vs. high numeracy (p<0.05). Using the DCE, no statistically different MARs were identified for other combinations of participant characteristics, risks, or countries. CONCLUSIONS : The PTT identified preference heterogeneity in MARs for more participant characteristics by directly incorporating participant characteristics in the regression model. When attempting to estimate MARs, PTT may partially overcome challenges with stratified DCE models, particularly if analyses such as latent class analysis are not feasible or desirable. Further research is needed to confirm the findings in this case studies and to explore which method most accurately identify true underlying preference heterogeneity are needed.
P17: Preference of Rheumatoid Arthritis Patients for Tapering Biologics: A Discrete Choice Experiment
4:00PM - 4:15PM
Suz Jack C 1 , Stamp L2 , Treharne G1 , Marra C1 1 University of Otago, Dunedin, New Zealand, 2 University of Otago, Christchurch, New Zealand
OBJECTIVES : Tapering of biologics is a safe and feasible approach in the long-term management of rheumatoid arthritis (RA) patients who are in remission. However, the appeal of tapering strategies needs to be balanced against the risks of disrupting patients’ disease control. The aim of this study was to measure the preferences of RA patients and their risk-benefit trade-offs in relation to biologic tapering. METHODS: A web-based discrete choice experiment (DCE) was employed. Seven attributes (identified via focus groups and a systematic review) of varying levels describing three hypothetical choice were presented: frequency of treatment, chances of known adverse effects, chances of regaining disease control and healthcare service-related features. DCE data were analysed using mixed logit model to estimate the preference weights for key treatment features and to quantify trade-offs between the attributes. RESULTS: A total of 142 complete responses were analysed. Mean age was 60.3 years with an average disease duration of 20.8 years Frequency of biologic treatment was the most important attribute, followed by the chance of flare upon tapering. Time to see the rheumatology team after a flare was ranked the least important among the seven attributes. On average, participants were willing to accept between 25.3% to 50.2% increase in chance of disease flare in exchange for reducing the frequency of biologic treatment, chance of serious infection and chance of skin cancer. CONCLUSIONS: This study provides evidence that RA patients’ preference for tapering biologics are most influenced by the frequency of treatment and chance of flare. For these attributes, they are willing to accept a greater chance of flare in exchange for treatment benefits in the form of a reduction in biologic dosing and potential risk of serious infection and skin cancer associated with long-term biologic use. These findings have implications for clinical practice and policy making about tapering.
P18: Patient Preferences for Attributes of a Multi-Cancer Early Detection Test: A Discrete Choice Experiment (DCE) Quantitative Pilot Study
4:15PM - 4:30PM
Gelhorn H1 , Ross M 1 , Kansal AR2 , Fung E2 , Seiden M3 , Chung KC2 1 Evidera, Bethesda, MD, USA, 2 GRAIL, Inc., Menlo Park, CA, USA, 3 McKesson, The Woodlands, TX, USA
OBJECTIVES : Early cancer detection and intervention can significantly improve patient outcomes and reduce mortality rates. Evidence shows that emerging blood-based multi-cancer early detection (MCED) tests can detect a variety of cancer types across stages and provide a predicted cancer signal origin with high specificity. However, little is known about patients’ preferences for MCED tests. This study aimed to quantify preferences for attributes of blood-based MCED tests among the US general population aged 50-80 years. METHODS : A DCE consisting of five attributes (true positives, false negatives, false positives, likelihood of the cancer type unknown [e.g., inaccurate cancer signal origin], and number of cancers tested for) was administered online to US general population members to elicit preferences to quantitatively pilot test the DCE. Data were analyzed using an error-component multinomial logit model and relative attribute importance (RAI) was obtained. RESULTS : Participants (N=303) were 62.0% male (n=188), mean age 68.2 years (SD=6.4). RAI indicated that the rank order of attribute importance was false negatives (35.7%), true positives (27.6%), false positives (17.3%), number of cancers tested for (16.8%), and cancer type unknown (2.7%). Attributes related to improved test accuracy were important and participants strongly preferred screenings that tested for more cancer types (all p < 0.05). Preferences were non-significant for the likelihood of cancer type unknown attribute levels. Overall, 71.9% of participants reported that they would prefer to receive the MCED test in addition to their currently recommended cancer screenings. CONCLUSIONS : Participants’ preferences were strongly driven by the desire for a screening test with fewer false negatives and more true positives, with these 2 attributes comprising 63.3% of the RAI. False positive results and number of types of cancer tested for also impacted preferences but were of lower importance. The majority of participants preferred adding a MCED test to supplement current cancer screenings.
P20: Evaluating Preferences and the Effect of Altruism on COVID-19 Vaccine Decisions: A Discrete Choice Experiment
4:45PM - 5:00PM
Kelley M 1 , Drabo EF2 , Gong CL3 1 University of Southern California, Los Angeles, CA, USA, 2 Johns Hopkins Bloomberg School of Public Health, Department of Health Policy and Management, Baltimore, MD, USA, 3 Children's Hospital Los Angeles, Los Angeles, CA, USA
OBJECTIVES : To elucidate how Americans value COVID-19 vaccine characteristics, and determine whether their willingness to vaccinate is altered by the framing of the vaccination decision as altruistic or not. METHODS : We conducted a discrete choice experiment (DCE) with Amazon MTurk participants randomized into a control group with standard DCE questions, versus a treatment group with questions framed altruistically. The survey consisted of demographic questions, an altruism index, and a DCE of 12 choice tasks with 3 profiles (Vaccine A, Vaccine B, and No Vaccination). Vaccine attributes included number of doses, efficacy in preventing infection, risk of severe disease, severe side effect type, risk of severe side effect, and subsidy. We estimated preference weights using multinomial logit models, controlling for framing, sex, age, political party, health status, race/ethnicity, and altruism score. RESULTS : Sample included 2,014 respondents (control with no framing, n=1,037; altruism framing, n=977). Respondents preferred COVID-19 vaccines with allergic reactions vs neurological disorder as side effects (OR: 1.32; P <0.01), higher efficacy (OR: 1.03; P < 0.01), higher subsidies (OR: 1.00; P < 0.01), lower risk of side effects (OR: 0.99; P < 0.01), and lower risk of severe disease (OR: 0.99; P < 0.01). Preferences for single- vs double-dose formulations did not significantly differ (P > 0.01). Respondents with higher baseline altruism scores were more likely to prefer vaccination compared to those with lower altruism scores (RR: 1.83; P < 0.01). However, framing neither significantly affected preferences for vaccination nor modified the effect of baseline altruism on these preferences for vaccination. CONCLUSIONS : Preferences were strongest for vaccines with less severe side effects, suggesting that innovators should prioritize COVID-19 vaccines with these characteristics. More altruistic individuals were more likely to vaccinate, but framing did not modulate vaccination decisions, implying its limited nudging effects for vaccination.
Using Real World Data to Assess Patient Outcomes
Live
This session explores the use of real world data to assess patient outcomes drawing from examples in Herpes Zoster, Type-2 Diabetes, non-small cell lung cancer and psoriasis.
Moderator
Laura Mccullagh, PhD
National Centre for Pharmacoeconomics, Dublin, Ireland
Dr Laura McCullagh is Head of Research, a Chief I Pharmacist and a member of the Senior Management Team within the National Centre for Pharmacoeconomics (NCPE), Ireland. Dr McCullagh is a lead health technology assessor within the NCPE. The NCPE is affiliated with Trinity College Dublin; roles here include various teaching and research commitments. Previous roles have included Associate Professor of Pharmacoeconomics & Health Technology Assessment, Trinity College Dublin, Senior Medicines Information Pharmacist (Oxford Radcliffe Trust, UK). Dr McCullagh holds a PhD (Trinity College Dublin) for research which focused on HTA within the Irish Healthcare Setting.
P67: Analysis of the Pharmaco-Utilization and Healthcare Costs in Pediatric Psoriatic Patients: A Real-World Retrospective Study Among the Italian Population
4:30PM - 4:45PM
Perrone V1 , Losi S2 , Sabatino S3 , Mezzetti M4 , Dovizio M 1 , Veronesi C1 , Degli Espositi L1 1 CliCon S.r.l. Health, Economics & Outcomes Research, Bologna, Italy, 2 Eli Lilly Italy S.p.A, Sesto Fiorentino, Italy, 3 Eli Lilly Italy S.p.A, Sesto Fiorentino (FI), Italy, 4 Eli Lilly Italy S.p.A., Roma, Italy
OBJECTIVES: The objective of the study was to assess the profile of pediatric patients with psoriasis (PSO) in Italy, in terms of treatment patterns, pharmaco-utilization, and healthcare costs. METHODS: A retrospective study was conducted using the administrative databases of selected Italian health departments, covering around 22% of the population. Patients (<18 years) affected by PSO [diagnosed by at least one PSO hospitalization and/or an active PSO exemption code and/or topical antipsoriatics prescription] during 01/2010-10/2019, were included. The first occurrence of ≥1 inclusion criterion was considered index-date. During follow-up (≥12 months from index-date), the pharmaco-utilization (as treatment lines and discontinuation), and healthcare cost/patient [for drugs, outpatients specialistic services (OSS), and hospitalizations] were assessed in biological users under the age of 18 years during the follow-up. RESULTS: Overall, 7,077 patients were included (mean age 12.6 years, male 47%). During follow-up, 29.6% were prescribed systemic corticosteroids, 4.2% used conventional systemic therapies (cyclosporine/methotrexate/acitretin/dimethylfumarate), and 40.2% (index-date excluded) used topical antipsoriatics. Eighty-five patients (1.2%) had a biological prescription during follow-up, 61 of which were under 18 throughout follow-up. Among them, 42.6% were prescribed etanercept, 55.7% adalimumab, and the remaining ustekinumab. During available follow-up, 47.5% and 24.6% of biologic-treated patients maintained their therapy with adalimumab and etanercept, respectively. During the first-year follow-up, 39.7% of biological-treated patients interrupted (>90 days of treatment-gap) their treatment. Mean annual healthcare cost/patient for biological utilizers under 18 throughout follow-up(N=61) was 6,830€ (5,422€ for drugs, 986€ for OSS, and 422€ for hospitalizations). CONCLUSIONS: This Italian real-world study reported the pharmaco-utilization and costs for pediatric PSO patients in clinical practice. A low proportion of pediatric patients currently treated with biological therapies was found, with less than half of patients maintaining treatment. Furthermore, their management was associated with a high economic burden. The optimization of treatment for pediatric PSO patients is still an unmet need.
P66: Epidemiology of Non-Small Cell Lung Cancer (NSCLC) by Histology and Disease Stage in Western Europe (WE): Population-Level Projections 2021-2026
4:15PM - 4:30PM
Kanas G 1 , Kalilani L2 , Durbin L3 , Clark O3 , Nersesyan K3 , Keeven K3 , Giove TJ4 , Chao J5 , Aziez A6 , Stojadinovic A7 , Hogea C8 1 Kantar Health, Dublin, CA, USA, 2 GlaxoSmithKline, Durham, NC, USA, 3 Kantar Health, New York, NY, USA, 4 GlaxoSmithKline, Mississauga, ON, Canada, 5 GlaxoSmithKline, Collegeville, PA, USA, 6 GlaxoSmithKline, Zug, Switzerland, 7 GlaxoSmithKline, Upper Providence, PA, USA, 8 GlaxoSmithKline, Philadelphia, PA, USA
OBJECTIVES: NSCLC accounts for approximately 85% of all lung cancers. Population-level projections are critical to further anticipate disease burden and address unmet needs. METHODS: We obtained the historic, annual age- and sex-specific incidence of lung cancer from country-specific registries in WE (France, Germany, Italy, Spain, and the United Kingdom). Histology data from the IARC’s Cancer Incidence in Five Continents (volume XI) were used to estimate total number of patients with NSCLC and by histology (non-squamous [NSQ] and squamous [SQ]). Assuming that observed trends will continue, the most recent country-, age-, and sex-specific incidence of NSCLC by histology were estimated and multiplied by the respective projected country populations to estimate annual number of cases from 2021 through 2026. Stage-specific distribution of NSCLC by histology according to the American Joint Committee on Cancer 8th edition staging system from the Surveillance, Epidemiology, and End Results program for the year 2018 was applied. RESULTS: Total number of new NSQ NSCLC cases for the five countries is projected to increase from 162,789 in 2021 to 174,177 in 2026. NSQ stage IIIB/C increases from 9,735 to 10,417 and stage IV from 77,585 to 83,013 cases. The total number of new SQ NSCLC cases is projected to increase from 43,187 to 46,286 over the same period. SQ stage IIIB/C increases from 5,994 to 6,424 and stage IV from 13,423 to 14,386 cases. CONCLUSIONS: Newly diagnosed cases for advanced/metastatic NSCLC among all five WE countries is projected to rise 7.0% for NSQ and 7.2% for SQ NSCLC between 2021 and 2026, with most new cases being advanced/metastatic (stage IIIB/C-IV) patients. The 5-year overall survival rate for NSCLC remains ~30% for stage III and ~6% for stage IV. Coupled with projected increases in case numbers, this underlines the continued need for novel therapies and synergistic combinations to treat NSCLC.
P68: Cardiovascular Events and Mortality in Type-2 Diabetic Patients under Second-Line Treatment with Hypoglycemic Agents: An Italian Real-World Study
4:45PM - 5:00PM
Degli Espositi L 1 , Sangiorgi D2 , Nappi C3 , Andretta M4 , Barbieri A5 , Citraro R6 , Bartolini F7 , Cavaliere A8 , Ciaccia A9 , Chinellato A10 , Cillo MR11 , Dell'Orco S12 , Ferrante F13 , Gentile S14 , Procacci C15 , Re D16 , Ubertazzo L17 , Vercellone A18 , Perrone V2 1 CliCon S.r.l. Health, Economics & Outcomes Research, Ravenna, Italy, 2 CliCon S.r.l. Health, Economics & Outcomes Research, Bologna, BO, Italy, 3 CliCon S.r.l. Health, Economics & Outcomes Research, Bologna, Italy, 4 Azienda ULSS 8 Berica, Vicenza, Italy, 5 ASL Vercelli, Vercelli, Italy, 6 Azienda ospedaliero-universitaria Mater Domini, Catanzaro, Italy, 7 USL Umbria 2, Terni, Italy, 8 ASL Viterbo, Viterbo, Italy, 9 ASL Foggia, Foggia, Italy, 10 Azienda ULSS 3 Serenissima, Mestre (VE), Italy, 11 ASL Salerno, Salerno, Italy, 12 ASL Roma 6, Albano Laziale, Italy, 13 ASL Frosinone, Frosinone, Italy, 14 Direzione Generale per la Salute Regione Molise, Campobasso, Italy, 15 ASL BAT, Trani, Italy, 16 ASL Teramo, Teramo, Italy, 17 ASL Roma 4, Civitavecchia (RM), Italy, 18 ASL Napoli 3 SUD, Torre del Greco, Italy
OBJECTIVES: To evaluate the incidence and risk of major adverse cardiovascular events (MACE) and all-cause mortality in type-2 diabetic patients with metformin failure in second-line treatment with hypoglycemic drugs, using real-world data in Italy. METHODS: A retrospective study was conducted using administrative databases from a sample of Italian Local Health Units. During 01/2015-12/2017, all adult type-2 diabetic patients who failed metformin therapy and in second-line with hypoglycemic drugs: sulfonylurea (SULF), dipeptidyl peptidase inhibitors (i-DPP-4), glucagon-like-peptide-1 (GLP-1), or inhibitors of the sodium/glucose-2 co-transporter (SGLT-2i), alone or combined with metformin, were included. Index-date corresponded to 1st prescription date; patients were followed-up from index-date to end of 2019. The Propensity Score Matching (PSM) methodology was applied to abate covariates variability among cohorts. RESULTS: Among the 18,818 patients included (mean age 65.6 years, 57.5% male), 40.3% had SULF as second-line, 36.1% i-DPP-4, 15.3% SGLT-2i, 8.3% GLP-1. After PSM, the incidence rate per 100 person-years of MACE was 3.7 for SULF patients, 3.1 for i-DPP-4 patients, 2.7 for SGTL-2i patients, and 2.6 for GLP-1a patients. All-cause death incidence rate per 100 person-years was higher in SULF cohort (1.4) than DPP-4 (1.3), SGLT2-i (0.7) and GLP-1 (0.9) (p= 0.001). Compared to SULF patients, SGLT-2i and GLP-1 had a 26% (p-value=0.013) and 30%(p-value=0.003) lower risk of MACE events, respectively. SGLT-2i and GLP-1 patients were also associated to a lower risk (SGLT-2i:52%, p-value=0.001; GLP-1:40%, p-value=0.012) of all-cause death rather than SULF cohort. CONCLUSIONS: This study provides an evaluation from real-world data of the clinical profile of patients in second-line with hypoglycemic agents after metformin failure. Our findings showed that in matched cohorts, lower incidences and risks of MACE and all-cause death were observed among GLP-1 and SGLT2-i patients than in SULF ones. These data suggest that the evaluation of clinical outcomes with therapeutic interventions might support health-decision making, to also improve prescriptive appropriateness.
P65: Health Care Resource Utilization and Potential Disease Deterioration After Herpes Zoster Incidence in Patients with Underlying Conditions: A Retrospective Cohort Study Based on German Claims Data, 2007-2018
4:00PM - 4:15PM
Witte J 1 , Batram M2 , Schwarz M3 , Hain JJ3 , Ultsch B4 , Steinmann M1 , Bhavsar AB5 , Greiner W1 1 Department for Health Economics and Health Care Management, Bielefeld University, Bielefeld, Germany, 2 Department for Econometrics, Bielefeld University, Bielefeld, Germany, 3 GSK, Munich, Germany, 4 GSK, Munich, BY, Germany, 5 GSK- Vaccines, Wavre, WBR, Belgium
OBJECTIVES: This study aimed to investigate potential disease deterioration for underlying conditions (UCs: chronic obstructive pulmonary disease, diabetes mellitus type 1 or 2, depression, coronary heart disease, chronic heart failure (CHF), and rheumatoid arthritis) after an acute herpes zoster (HZ) using health care resource utilization (HCRU) and disease-specific worsening indicators. METHODS: Analyses were based on claims data, representing 13% of the German statutory health insurance population (corresponding to 87% of the entire German population). Patients aged ≥18 years with UCs were included when an incident HZ-diagnosis (defined by International Classification of Diseases and prescription of an antiviral drug) was observed between 2008-2016. Patients were matched to controls with the same UC but without HZ in the observational period using propensity scores. HCRU was analyzed for four quarters prior and eight quarters after acute HZ. Regression models were used to identify potential excess HCRU and disease-specific worsening indicators in the post-acute quarters. RESULTS: Over the observational period, HCRU data from 172,093 HZ patients, matched to 172,093 controls, were analyzed. Matching achieved almost perfect balance, as there was no significant difference for 12 out of 13 matching variables. Excess costs were observed in the index quarter of acute HZ for all UCs (range: €92-€259). A significant increase in post-acute excess costs was observed for patients with CHF only, amounting to 8.56% (152€) and 6.61% (114€) in the first and second quarter after HZ-incidence, respectively. Disease-specific worsening indicators were observed only for patients with depression who showed somatoform disorders excess diagnosis and excess antidepressant use in post-acute quarters. CONCLUSIONS: HZ-incidence is associated with a significant increase in excess HCRU in the index quarter of HZ-diagnosis for patients with UCs. The present results suggest that deterioration of UCs more than 6 months after incident HZ is relevant for patients with CHF and depression.
Is Social Media Information Useful to Understand Patient Experiences and the Burden of Disease?
Live
Social media is increasingly used as a source of information in health technology appraisals. This session illustrates how this data can be used to understand the patient journey, quality of life, unmet needs, and experiences with available treatments.
Moderator
Lynda Doward, Master of Research
RTI Health Solutions, Manchester, United Kingdom
P34: Use of Social Media Listening (SML) Methods for Understanding the Patient Experience of Chronic Disease: A Scoping Review
4:15PM - 4:30PM
Trevisan F 1 , Clifford S2 , Cooper V2 1 Sprout Health Solutions, Pinner, UK, 2 Sprout Health Solutions, Los Angeles, CA, USA
OBJECTIVES: To conduct a scoping review of the literature related to the use of social media listening (SML) methods for understanding patients’ experiences of chronic disease. METHODS: Methodological guidance for conducting scoping reviews was followed. PubMed, Medline and EMBASE databases were searched using terms for social media listening, patient experience and quality of life. Primary research studies and systematic reviews that investigated the use of SML for understanding the patient experience of health or healthcare were included. Titles and abstracts were reviewed by two reviewers. One reviewer extracted data from full text articles. Quality of data extraction was assessed and verified by a second reviewer. A descriptive synthesis was performed with a focus on summarizing information related to the type of social media platforms, methods, data extraction tools and outcomes reported. RESULTS: The literature search identified 53 studies; 14 met the inclusion criteria and were included in the full-text review and data extraction. Social media platforms included Twitter, Facebook, HealthUnlocked, and disease-specific online communities. Methods used by researchers conducting SML included keyword frequency analysis, identification of patient reported terms for functional status, qualitative content analysis, identification of frequently asked questions and modelling of the patient experience. Outcomes included identification of symptoms experienced by and of importance to patients (including physical, psychological and cognitive symptoms), experience of medical treatment and procedures, social relationships and support, financial difficulties, information on the temporal patterns of symptoms experienced by patients and identification of racial and ethnic disparities in patient experience of disease. CONCLUSIONS: The findings highlight the potential value of SML as a method for capturing the patient voice and understanding patient experiences of chronic disease. SML has the potential to complement traditional methods, reduce patient burden and include the experience of people who may not take part in formal research studies.
P33: Patient Experiences and Insights on Chronic Ocular Pain from a Social Media Listening Study
4:00PM - 4:15PM
Goel K1 , Parashar N2 , Aasaithambi S2 , Verma H2 , O'Brien P 3 , Sloesen B4 1 Novartis Healthcare Pvt. Ltd., Hyderabad, AP, India, 2 Novartis Healthcare Pvt. Ltd., Hyderabad, India, 3 Novartis Business Services Center, Limerick, Ireland, 4 Novartis Pharma NV, Vilvoorde, Belgium
Objective: To identify the perceived causes for chronic ocular pain (COP) (≥3 months pain duration), its impact on quality of life (QoL) and understand the patient journey from social media posts. Methods: In this retrospective study, publicly available social media conversations were identified from searches triaged by a combination of automated relevancy keyword algorithm and manual review, and subsequently analyzed post anonymizing for COP content. Twitter, forums, and other (Facebook, Blogs, etc.) platforms were leveraged for the time period February 2020 to February 2021. Results: A total of 464 (UK=208, US=175, Canada=65 and Australia=16) patient/caregiver conversations on COP were identified. Top discussion points were symptoms (62%) and causes of COP (58%). Ocular factors (including dry eye disease, thyroid/Graves’ disease, and ocular surgeries) contributed to ~46% of causes identified, while non-ocular factors (including migraine, COVID, and side-effects/withdrawal of medications) contributed to ~54%. The most commonly mentioned symptoms (555) were headache/head pressures (96), dry/gritty eyes (67), light sensitivity (34), insomnia (29), and redness/pink eyes (28). Symptoms impacted all aspects of patients’ QoL: physical day-to-day activities such as reading, driving, and sleeping; emotional wellbeing such as depression/hopelessness, frustration/anger, fear, and suicidal thoughts; functional wellbeing such as difficulty at work/study place, reduced productivity or having to quit their job; social impacts such as being irritated around people, and having a less active social life. Eye drops (58/140 mentions) are the most commonly mentioned treatment option. Common coping strategies mentioned were blue-light filter glasses/eyeglasses (17), and hot compresses (11). Key unmet needs mentioned by patients were failed, improper, delayed diagnosis (62), and lack of effective treatments or appropriate management (30). Conclusion: Insights from this study reported patients’ experiences, concerns, and the adverse impact on overall QoL. The results can help in better understanding the patients’ perspective, which can be considered during drug development.
P36: Unmet Needs of Caregivers in Locally Advanced or Metastatic Bladder Cancer from Social Media in the US
4:30PM - 4:45PM
Renner S1 , Loussikian P1 , Marrel A2 , Barbier V2 , Foulquié P1 , Mebarki A1 , Schück S1 , Bharmal M 3 1 Kap Code, Paris, France, 2 Icon, Lyon, France, 3 EMD Serono, Billerica, MA, USA
OBJECTIVES: Bladder cancer (BC) is the sixth most common cancer in the US; the prognosis for patients with locally advanced or metastatic BC is very poor. Few studies have assessed its burden on caregivers. This study aimed to characterize difficulties and unmet needs of caregivers for patients with locally advanced or metastatic BC as reported on social media. METHODS: US caregiver testimonials were collected from social media posts between January 2015 and April 2021 using specific terms for locally advanced or metastatic BC. These were qualitatively analyzed to identify caregiver difficulties and unmet need until saturation. RESULTS: Of 1214 testimonials from 679 caregivers on 72 social media sources, 423 were randomly selected and analyzed until saturation. From those that reported age-related data (<15% of testimonials), most caregivers were women (83.2%) with a mean age of 35.4 years, whereas the reported mean age of patients was 67.2 years. A total of 177 testimonials that expressed ≥1 caregiver- or patient-centered difficulty were identified and classified into a list of 36 types of challenges. The main difficulties related to the caregivers’ psychological impact throughout the patient journey (26%), the desire to share experiences among peers/support groups (15.8%), and the fear and management of patients’ adverse events (12.4%). Other major difficulties expressed included the specific psychological burden of end-of-life support or grief work (10.2%), the daily impact of being a caregiver (relocation, time consumption; 9.6%), stress due to screening and diagnostic delay (7.3%), and the change in relationships between patients and caregivers (5.1%). CONCLUSIONS: Qualitative analysis of social media testimonials from caregivers of patients with BC in the US provided insights on the substantial psychological impact and burden of care on them. Future research may explore BC caregiver well-being and quality of life as outcomes in quantitative studies.
Informing the Decision-Making Process in Real Time
Live
This session illustrates the potential of real world data information to inform the decision-making process.
Moderator
Michael Barry, MB, FRCPI, PhD
National Centre for Pharmacoeconomics, Dublin, Ireland
P29: Reimbursement Outcomes for Combination Therapies vs Monotherapies in Lung Cancer and Multiple Myeloma in the Top Five European Markets
4:00PM - 4:15PM
Izmirlieva MA 1 , Reinaud F2 , Taiyeb M3 , Ando G1 1 GlobalData, London, UK, 2 GlobalData, Paris, France, 3 GlobalData, Bangalore, India
Objectives: Theoretically, combination therapies would face greater difficulty in demonstrating cost effectiveness because the backbone therapy is often priced close to the relevant country’s cost-effectiveness threshold. Unless the backbone therapy’s cost is reduced, the combination may not be cost-effective even if the add-on therapy is priced at zero. We set out to verify it this is true in practice by assessing reimbursement outcomes for combination therapies vs monotherapies in lung cancer and multiple myeloma. Methods: Reimbursement status and level of reimbursement for all drugs in lung cancer and multiple myeloma, which were first priced between 1 January 2011 and 31 December 2020, were assessed in France, Germany, Italy, Spain and the United Kingdom using data from the IHS Markit POLI database. The reimbursement status review was supplemented by Amélioration du Service Médical Rendu (ASMR) ratings in France, Federal Joint Committee (G-BA) ratings in Germany and NICE guidance in the UK to assess the likely pressure on prices for those combination therapies that gained reimbursement. Results: In lung cancer combination therapies were more likely to be rejected for reimbursement compared to monotherapies. Across the five countries, 20 out of the 56 combination therapy presentations (equivalent to 35.7%) were rejected for reimbursement compared to 11.4% (31 out of 271) for monotherapy presentations. In multiple myeloma, 5.3% of combination therapy presentations (7 out of 132) were rejected for reimbursement, while every single monotherapy was approved for reimbursement. Combination therapies also had less favourable ASMR and G-BA ratings. Conclusions: This review of reimbursement decisions and cost-effectiveness assessment outcomes for drugs approved over a 10-year period in the top five European markets confirms that combination therapies in lung cancer and multiple myeloma face greater difficulty in demonstrating cost-effectiveness compared to monotherapies. Even when approved for reimbursement, combination therapies are subject to greater pressure on prices.
P31: Impact of COVID-19 on HTA/PRMA of Medicinal Products in Europe: A Payer Perspective
4:30PM - 4:45PM
Mycka J1 , Dellamano R2 , Lobb W1 , Dalal N 3 , Dellamano L2 , Pereira E1 1 Medical Marketing Economics LLC (MME), Montclair, NJ, USA, 2 ValueVector, Milan, Italy, 3 Medical Marketing Economics LLC (MME), St Albans, NJ, USA
OBJECTIVES: Assess payer perceptions of COVID-19 pandemic’s impact on health systems, focusing on HTA, pricing, reimbursement and market access (PRMA) of new, branded medicines in the EU4 and UK.
METHODS: In June 2021, MME Advisors conducted a virtual, national payer / advisor board with representatives from France (2), Germany (2), Italy (1), Spain (1), and the UK (2) - to discuss key topics within the pandemic’s context, such as:
Disruption to healthcare systems HTA impact: backlog, re-prioritization, framework PRMA impact: net price pressure, conditional pricing/RWE and time to market Differences and similarities within oncology, rare diseases, ATMPs and general medicines RESULTS: Unlike the significant disruptions seen during the height of the pandemic in 2020, payers saw impact ranging from moderate (Italy) to high (Spain) as of June 2021. Disruption by disease state varied: oncology was highly disrupted everywhere but Germany. Most payers did not anticipate shifts in long term priorities or budget cuts to healthcare post pandemic. HTA impact was minimal, with no need to re-prioritize by therapy area or alter plans to adjust frameworks. Likelihood of stricter HTA criteria varied with payers in Italy anticipating more scrutiny for oncology and in Germany for rare diseases/ATMPs. While time to market was expected to remain mostly stable, delays anticipated in Spain. Majority of payers anticipated increasing pressure on drugs’ net prices; however, they were divided on increases in conditional pricing/RWE.
CONCLUSIONS: Perceived COVID-19 impact varied by country based on infrastructure and adaptability. Germany less impacted, whereas in other markets (e.g., Spain) COVID-19 seemed to have accelerated changes, rather than drive PRMA policy. Given the importance of healthcare, overall budget cuts were not anticipated, although the need to deploy funds to diverse areas (e.g., healthcare worker salaries, hospital capacity) could complicate future scenarios, especially for high-cost therapies. Therefore, continued monitoring is warranted.
P30: Potential Impacts of the New MHRA Policy for Biosimilar Approval for the Industry and Patients
4:15PM - 4:30PM
Ribeiro A , Walker A, Walsh K Lifescience Dynamics Ltd, London, UK
OBJECTIVES: To understand the potential impact of the new MHRA guidance for biosimilar licensing in the UK and other key markets, in terms of accelerating biosimilar development, time to market, prescribing limits (e.g. automatic substitution) and, patient access to biologics. METHODS: We reviewed the MHRA guidance on biosimilar licensing, alongside that from the FDA/EMA, and country-specific guidance on biosimilar use. We also analysed FDA/EMA approvals for biosimilars approved without confirmatory efficacy trials. Finally, a virtual iAdBoard was organised with payers/KOLs from France, the UK and US to capture different perceptions on the new policy and downstream impacts on biosimilar access. RESULTS: The MHRA has discontinued the requirement for biosimilars to undergo confirmatory efficacy trials as a licensing condition. Although the new policy was celebrated by the biosimilar industry, one must note the FDA and EMA do not explicitly state a Phase 3 trial requirement for biosimilar approval, and to date, two pegfilgrastim biosimilars have been approved without a Phase 3 trial (Udenyca, Nyvepria), given their robust chemical characterization and Phase 1 trials’ results. Additionally, the new MHRA guidance contemplates exceptions where comparative trials are required, leaving uncertainty around for how many biosimilars, particularly monoclonal antibodies, chemical characterization plus PK/PD trials will suffice. The virtual iAdboard revealed payer differences in opinion regarding impacts on biosimilar development timelines (vs agreement on economic viability), and in future policies encouraging biosimilar uptake, with EU payers more receptive to the change than US counterparts, but concerned with backlash from HCPs. CONCLUSIONS: If the FDA/EMA endorse the MHRA decision, in the future, a strong CMC/Phase 1 package could replace Phase 3 studies for biosimilar licensing. However, it is yet unclear whether this abbreviated data package will result in faster times to market or if it will have negative impacts on prescribing freedom and patient access to biosimilars.
P32: Price Analysis of Cancer Therapies for the Treatment of Patients with Unresectable Hepatocellular Carcinoma
4:45PM - 5:00PM
Williams A 1 , Anderson N2 , Gwon YG3 , Wifler W2 1 Boston Scientific, Marlborough, MA, USA, 2 Boston Scientific, Maple Grove, MN, USA, 3 Boston Scientific, Kuala Lumpur, MN, Malaysia
OBJECTIVES: The price of cancer therapy for the treatment of adult US patients with hepatocellular carcinoma (HCC) remains unknown. This study estimated the price of systemic therapies (ST) compared to selective internal radiotherapy (SIRT) for the treatment of HCC from the payer and provider perspectives. METHODS: The National Comprehensive Cancer Network (NCCN) Guidelines were used as a framework to model the treatment strategies for HCC. The associated drug prices as of May 2021 for ST and SIRT were obtained from the IBM Micromedex Redbook (Average Wholesale Price [AWP]), (Wholesale Acquisition Cost [WAC]), Medicare’s Average Sale Price (ASP), and Decision Resources Group (DRG) ASP. Clinical parameters, such as treatment duration and FDA-recommended daily dose (DDD), were obtained from randomized controlled trials and FDA-approved labels. The total price/DDD was calculated for each treatment therapy and treatment duration over a short-term (<12 months) horizon. Sensitivity analysis was conducted to explore the impact of treatment duration uncertainty on model results. Because drug rebates are unknown, these price estimates did not account for drug rebates or patient assistance programs negotiated directly with manufacturers by Pharmacy Benefit Managers. RESULTS: 11 STs and 3 SIRTs were included in our analysis. The median price/DDD of ST varied by perspective: Medicare ASP: $97,466 (IQR: $341-$205,393); Provider WAC: $123,322 (IQR: $18,475-$305,615); Provider AWP: $186,389 (IQR: $22,170-$366,738). The median price for SIRT was estimated; Medicare ASP: $21,877 (IQR: $21,877-$22,269) and Provider ASP: $21,873 (IQR: $21,316–$21,873). The price differences are greater than SIRT when considering patients who progress through first-line and second-line ST. CONCLUSIONS: The price of cancer therapy for HCC varies widely by payer-provider perspective. The availability of alternative cancer therapies, such as locoregional (non-surgical) approaches, may offer clinical meaningful benefit and reduce the total costs of HCC care from the payer-provider perspectives.
Development and Measurement of Health Utilities
Live
This session presents interesting new research in health utility estimation. Studies include the estimation of the EQ-5D-5L value set for Italy, an exploration of the association between EQ-5D utility and migraine frequency, and a vignette valuation study for eye disease. The association between health state utility and work productivity is also discussed, informing the debate about the extent to which wider societal benefits of health technologies are associated with health utilities.
Moderator
Sorrel Wolowacz, PhD
RTI Health Solutions, Manchester, United Kingdom
Sorrel Wolowacz, PhD, is Head of European Health Economics at RTI-HS, with 20 years of experience in health economics research and consulting. Her research focuses primarily on economic modelling, health utility estimation, observational studies, and health technology appraisal submissions. Dr. Wolowacz is a member of the editorial board for the Journal of Comparative Effectiveness Research and was co-chair of the ISPOR Good Research Practices Task Force addressing Measurement of Health State Utility Values for Economic Models in Clinical Studies and for the ISPOR Working Group for Oncology Health Economic Modeling.
P10: Evaluating the Correlation between Monthly Migraine Days and Quality-of-Life: Utility Analyses to Inform a Japanese Cost-Effectiveness Model for Fremanezumab in Migraine
4:00PM - 4:15PM
Peterse E 1 , Bennison C2 , Paris J3 , Chatterjee A2 , Wang X4 , Kojima Y4 , Yamato K4 1 OPEN Health, Rotterdam, Netherlands, 2 OPEN Health, York, UK, 3 OPEN Health, Kent, KEN, UK, 4 Otsuka Pharmaceutical Co., Ltd., Tokyo, Japan
OBJECTIVES : A cost-effectiveness model (CEM) for fremanezumab in migraine prevention from a Japanese public healthcare payer perspective has been developed. To inform health state specific utilities, we analyzed the correlation between the number of monthly migraine days (MMD) and a patient’s quality-of-life using data from Japanese-Korean trials. METHODS : The health states in the CEM are defined by the number of MMD, ranging from 0 to 28. Data from three Japanese-Korean clinical trials (406-102-00001, 406-102-00002, 406-102-00003) was analyzed. MSQoL (migraine specific quality of life) values measured in the trials were mapped to EQ-5D-3L utility values using a previously published mapping algorithm. To account for the repeated nature of the data, linear mixed effects models were fitted to the EQ-5D-3L values. MMD, MMD at baseline, treatment arm (monthly injection, quarterly injection, placebo), scheduled visits (month 1, month 2 etc.), age, sex, prior migraine medication and country were explored as covariates. The final model was selected based on the Akaike information criterion (AIC) value using forward and backward selection. RESULTS : In total, 3743 utility values from 970 patients were included in our analyses. The mean observed utility value was 0.83 for patients with 0 MMD and 0.51 for patients with 28 MMD. Fremanezumab decreased the number of MMD, thereby increasing a patient’s quality-of-life. The variables MMD, baseline MMD, scheduled visits and country were included in the final model. The regression coefficient for MMD was -0.01 (p<0.001), demonstrating that, after adjusting for baseline MMD, schedule visits and country, utility decreased by 0.01 for every day increase in MMD. CONCLUSIONS : There was a strong correlation between the number of MMD and quality-of-life in patients with migraine. Estimates derived from the linear mixed-effects model can be used to inform health-state specific utilities in the Japanese cost-effectiveness model for fremanezumab in migraine prevention.
P12: Are Gains in Health Utility Associated with Gains in Work Productivity and Role Functioning in Chronic Diseases? A Systematic Literature Review
4:30PM - 4:45PM
Aggio D 1 , Williams A2 , McNamara L3 , Lloyd A1 1 Acaster Lloyd Consulting Ltd., London, UK, 2 Kyowa Kirin International, Marlow, BKM, UK, 3 Kyowa Kirin Ltd., Galashiels, UK
OBJECTIVES : Disease experience for people living with chronic diseases has changed dramatically with improvements in health utility. It remains unclear, however, the extent to which improvements in health utility leads to gains in work productivity and role functioning. This systematic literature review aimed to explore the relationship between health utility and work productivity or role functioning across chronic diseases. METHODS : Diseases selected were chronic and severe (based on health utility weights in range 0.50 to 0.70). Records from a structured search conducted in MEDLINE, Embase and PsycINFO were reviewed against inclusion criteria and assessed for study quality/relevance. Articles published from 2000 – February 2021 and available in English were considered. Studies included a measure of health utility (e.g., EQ-5D) and productivity or role function (e.g., employment status, presenteeism and absenteeism). Study quality was assessed in terms of design, analysis approach, missing data and evidence of bias. RESULTS : The search identified 876 records; 244 underwent full review, and 34 of the highest quality studies were extracted. Only 4 longitudinal studies were identified. Studies included different diseases including multiple sclerosis, rheumatoid arthritis, and stroke. Weighted mean health utilities of 0.79 were observed for employed (full/part time) people with a chronic disease, compared with 0.71 for part time employed, 0.61 for those unemployed/not in work, and 0.62 for those incapable of working. These associations held in studies controlling for potential confounders (e.g., age, symptom severity etc). Values corresponded to approximately a 5% increase in employment per 0.1 unit increase in health utility value. CONCLUSIONS : There is limited longitudinal research among people with chronic diseases exploring how changes in health utility may lead to changes in work productivity and role functioning. However, the findings suggest that amongst people with a chronic and severe disease, better health states are expected to be associated with higher productivity.
P11: Development of an EQ-5D-5L Value Set for Italy Using Videoconferencing Administered Personal Interviews: Reporting on the Feasibility of a New Mode of Administration for Valuation Studies
4:15PM - 4:30PM
Finch AP 1 , Meregaglia M2 , Ciani O2 , Roudijk B3 , Fattore G4 , Jommi C5 1 EuroQol Research Foundation, Amsterdam, Netherlands, 2 SDA Bocconi School of Management, Milan, Italy, 3 EuroQoL Research Group, Rotterdam, Netherlands, 4 Bocconi University, Milano, Italy, 5 SDA Bocconi School of Management, Bocconi University, Milano, Italy
OBJECTIVES : To derive an Italian value set for the EQ-5D-5L using videoconferencing interviews and to determine the feasibility of this mode of administration. METHODS : Preferences were collected using the EQ-VT V2. Two valuation methods were employed, composite time trade-off (cTTO) and discrete choice experiment (DCE). The target sample size was 1,000 participants. Participants were recruited using a market research company with experience in quantitative and qualitative data collection. Videoconferencing administered interviews were conducted by 11 interviewers selected among PhD students, researchers, and other academic affiliates. A pilot of 199 interviews was employed to assess the technical, operational and protocol feasibility of videoconferencing interviews. Standard QC parameters were used to monitor interviewers’ performance during the data collection. To inform the modelling choices, GLS, Tobit, Logit, Probit and Hybrid models were fitted to the data, with different error specifications. Models were compared in terms of monotonicity of coefficients, statistical significance, and theoretical considerations. RESULTS : 1182 videoconferencing interviews were completed between October 2020 and February 2021, including 199 feasibility pilot interviews. Dropouts and technical problems occurred in less than 5% of the interviews, and all interviewers complied with the protocol as well as showing significant improvements in QC parameters. The results suggested videoconferencing was a feasible mode of administration. The final sample was representative of the Italian general population for age, gender, and education as recorded in 2019 by ISTAT. Among the models tested, the Hybrid Tobit heteroscedastic model without constant was selected for the derivation of the tariff. In the selected model, coefficients for all dimensions levels were statistically significant and monotonically decreasing. Values ranged from -0.571 for the PITS state to 1 for health state 11111. CONCLUSIONS : An Italian societal value set for the EQ-5D-5L was developed. This can be used for economic evaluations and decision making in Italy. Videoconferencing appeared feasible for valuation interviews.
Two Years of COVID-19: What Has Been the Global Impact on HRQoL and Clinical Outcomes?
Live
This session illustrates recent evidence about the impact of COVID19 on patients' health related quality of life and clinical outcomes.
Moderator
Carla DeMuro, MS
RTI Health Solutions, Research Triangle Park, NC, USA
P61: COVID-19 Concerns Experienced by Pregnant and Postpartum Women and Their Influence on Health-Related Quality-of-Life
4:00PM - 4:15PM
Aytha Swathi P 1 , Regan A1 , Grinshteyn E1 , Nosek M1 , Gu NY2 1 University of San Francisco, Sacramento, CA, USA, 2 NYG Technologies, Santa Clarita, CA, USA
OBJECTIVES : Pregnant and postpartum women may be particularly susceptible to pandemic-induced anxiety/depression, which can adversely affect maternal and infant health. This study investigates the COVID-19 concerns experienced by pregnant and postpartum women and their Influence on health-related quality-of-life (HRQoL). METHODS: We conducted an online, national US survey (EuroQol grant: 260-2020RA) between May and June 2021. Respondents completed the EQ-5D-5L instrument and rated their level of concerns for their own health, their baby’s health, and their family’s health, being pregnant and giving birth during the pandemic. Women indicated whether they strongly agreed, agreed, neither agreed nor disagreed, disagreed or strongly disagreed with each concern. Respondents who indicated they “strongly agreed” were classified as having strong concerns. We used median regression to estimate the EQ-5D-5L utility and EQ-VAS scores by level of maternal concerns. RESULTS: Among 2,070 respondents, pregnant and postpartum women commonly expressed strong concerns about giving birth during the pandemic (44%; 95% CI 39%, 48%), the health of their baby (44%; 95% CI 40%, 48%), the health of their family (38%; 95% CI 34%, 42%), and being pregnant during the pandemic (38%; 95% CI 34%, 42%). Fewer respondents expressed strong concerns about their own health (27%; 95% CI 23%, 31%). Overall, there was no association between HRQoL measures and maternal concerns during COVID-19. Among women who gave birth during 2020 (n=536), each unit increase in concerns about being pregnant was associated a 0.02 decline in the EQ-5D-5L utility (95% CI -0.04, -0.01). No difference was observed in EQ5D-VAS scores ( ß=0.00; 95% CI -0.01, 0.01). No other differences in HRQoL were observed. CONCLUSIONS: Although overall, there was no relationship between maternal concerns during the COVID-19 pandemic and HRQoL, we did observe small declines in HRQoL associated with concern of being pregnant during the pandemic among women pregnant early in the pandemic.
P64: Evaluating the Impact of the COVID-19 Pandemic on Mortality after Myocardial Infarctions Hospitalization in Germany
4:45PM - 5:00PM
Krieger J 1 , Hardtstock F2 , Wilke T3 , Maywald U4 1 Cytel Inc, Berlin, Germany, 2 Cytel Inc, Wismar, Germany, 3 IPAM e.V., Wismar, Germany, 4 AOK PLUS, Dresden, Germany
OBJECTIVES: It was hypothesized that COVID-19 lockdown measures led to later admission of myocardial infarction (MI) patients to hospitals and, consequently, higher average case severity and mortality. The aim of this study was to compare MI-associated mortality between COVID-19 lockdown and pre-COVID-19 periods. METHODS: We used German claims data from continuously insured adults hospitalized with a MI (ICD-10 I21), and compared 30-days mortality for cases in March-May 2020 (first COVID lockdown in Germany) with March to May in 2017-2019. Multivariable logistic regression models were conducted to test for differences in mortality between pre-COVID and COVID months while controlling for patients’ age, sex, previous MIs (2-years baseline), and cardiovascular comorbidities. RESULTS: In 2020, we observed 758/612/712 MIs in March/April/May, which was fewer MIs than the average for the same months between 2017-2019 (March: 901; April: 716; May: 853). Over the observational years, there was a shift towards younger patients (average age 2017: 77; 2020: 74), and the proportion of women decreased (2017: 42.54%; 2020: 39.19%). The length of the index-hospitalization was significantly lower during the COVID-19 pandemic (March-May 2017-2019: 9.19 days; March-May 2020: 8.11 days; p<0.001). Furthermore, the number of deceased patients was lower during the COVID lockdown period (March 2017-2019: 16.53% vs. March 2020: 14.12%; April: 17.19% vs. 16.99%; May: 16.72% vs. 13.48%). However, regression models showed no significant difference between COVID and pre-COVID months except for May (OR [COVID vs. pre-COVID]: 0.73, p=0.014). CONCLUSIONS: Even if we cannot rule out the possibility of a higher MI-related mortality during COVID lockdown periods in non-hospitalized MI patients, we cannot confirm the hypothesis that hospitalized patients generally showed a higher mortality due to later admission to hospitals and thus more higher case severity at time of hospital admission.
P62: The Impact of the COVID-19 Pandemic on the Quality of Life of Patients with Melanoma – Findings from a UK Melanoma Registry
4:15PM - 4:30PM
Heinrich M1 , Mulgina D1 , Kudlac A1 , Ouyang C1 , Larkin M 2 , Ofori A3 1 Vitaccess, Oxford, LON, UK, 2 Vitaccess, London, LON, UK, 3 Vitaccess, London, UK
OBJECTIVES: The Melanoma UK digital registry, launched in collaboration with Melanoma UK in 2017, captures real-world patient-reported quality of life (QoL), symptoms, and side effects of melanoma treatment using a mobile application. 729 UK patients with melanoma have since joined the registry, with approximately 60% of participants in Stage 3 and 4 of the disease. Due to the COVID-19 pandemic, a national lockdown was announced in the UK in March 2020 with an instruction to stay at home, particularly for vulnerable people with health conditions. The impact of the fundamental changes in day-to-day living and the delivery of healthcare on people diagnosed with melanoma needs to be better understood. This study explored if the COVID-19 pandemic was associated with changes in Melanoma UK registry participants’ QoL. METHODS: QoL data, measured using EQ-5D and QLQ-C30 from 423 participants, were analysed. Data submitted between 03/2019 and 02/2020 were deemed pre-COVID-19 data; data submitted between 03/2020 and 03/2021 were considered as peri-pandemic data. A monthly average score was calculated for each domain of EQ-5D and QLQ-C30. T-tests were conducted to compare the individual domain scores calculated for the pre- and peri-pandemic periods. RESULTS: EQ-5D: Mobility ( p =0.03), self-care ( p =0.03), usual activities ( p =0.003), pain symptoms ( p =0.03), and health in general ( p =0.0004) improved during the pandemic. QLQ-C30: There was an observed improvement in physical ( p =0.02), role ( p =0.01), and social functioning ( p =0.003). Fatigue ( p =0.002) and pain symptoms ( p =0.05) decreased during the pandemic. Participants also reported experiencing less financial difficulty ( p =0.02) in comparison with the pre-pandemic period. No change in emotional well-being was observed. CONCLUSIONS: The QoL of patients with melanoma improved during the COVID-19 pandemic. Future research should involve qualitative interviews with melanoma patients and their caregivers to explore the mechanisms of this change, the role of support networks, and the impact on caregivers.
P63: Impact of COVID-19 on Health-Related Quality-of-Life in the United States, Sweden and Norway: A Cross-Country Comparison Using a Panel Survey
4:30PM - 4:45PM
Chen J 1 , Gong CL2 , Jiao X1 , Zawadzki NK1 , Persson U3 , Hay JW1 , Gu NY4 1 University of Southern California, Los Angeles, CA, USA, 2 Children's Hospital Los Angeles, Los Angeles, CA, USA, 3 The Swedish Institute for Health Economics (IHE), Lund, Sweden, 4 NYG Technologies, Santa Clarita, CA, USA
OBJECTIVES: To assess and compare the impact of the COVID-19 pandemic on health-related quality-of-life (HRQoL) in the United States, Sweden and Norway. METHODS: Two waves of web-based survey were conducted in April 2020 and January 2021 to collect demographic data, COVID-19 status, behavior and employment changes related to COVID-19 in each country (EuroQol Grant: 246-2020RA). EQ-5D-5L was used to assess health status of respondents. Results were compared between the two waves to measure changes in HRQoL. One-way ANOVA was used to detect significant differences between countries, and t-tests for differences between waves. RESULTS: We collected 2,734, 1,003 and 1,020 responses in Wave 1, and 2,252, 1,013 and 1,011 responses in Wave 2 for the US, Sweden, and Norway respectively. Corresponding mean (SD) EQ-VAS scores were 74.6 (±19.2), 68.7 (±21.4), and 69.2 (±20.8) in Wave 1 (p<0.001), and 76.4 (±18.6), 68.2 (±20.3), and 67.8 (±21.7) in Wave 2 (p<0.001). Between waves, only the VAS scores in the US were significantly different (p<0.001). Mean (SD) utility scores were 0.822 (±0.222), 0.768 (±0.260), and 0.808 (±0.248) in wave 1 (p<0.001), and 0.823 (±0.221), 0.783 (±0.237), and 0.777 (±0.271) in wave 2 (p<0.001); there were no significant differences between waves for all three countries. Anxiety/depression was consistently the most problematic EQ-5D-5L subdomain among Swedish and Americans (>50%), followed by pain/discomfort. >45% Norwegians also reported problems in anxiety/depression subdomain in both waves. The proportions reporting problems in anxiety/depression increased in wave 2 for Sweden and Norway, but decreased for the US. CONCLUSIONS: Population HRQoL in Sweden and Norway has been similar throughout the pandemic, while a rebound in population mean VAS was observed in the US. However, the large proportions reporting problems in anxiety/depression across waves in all 3 countries indicates that mental health issues resulting from the pandemic are a major concern.
The EQ-5D-5L in Practice in Europe and Beyond: Advantages and Limitations
Live
This session explores the pros and cons of assessing people's quality of life using the EQ-5D-5L scores. It shows to what extend it can provide valuable insights of the quality of life in the general population, but has limitations to assess quality of life in Alzheimer's disease. It shows how online platforms can be used for valuing health states, and its current use for clinical benefit assessment.
Moderator
Aureliano Paolo Finch, PhD
EuroQol Research Foundation, Amsterdam, Netherlands
Aureliano Finch is a scientist at the EuroQol Research Foundation, a member of the Descriptive System working group and coordinator of the EQ family of instruments valuation studies. His research mainly relates to the development and testing of preference based instruments of health and wellbeing. He applies both quantitative and qualitative methods for this purpose.
P59: To What Extent Is EQ-5D Used as a Tool for Clinical Outcome Assessment?
4:30PM - 4:45PM
Shaw C 1 , Longworth L1 , Bennett B2 , Ruane P3 , Watson C1 , Francis LE1 , Shaw J4 1 PHMR Ltd., London, UK, 2 Bristol-Myers Squibb, Uxbridge, UK, 3 PHMR Ltd., Newcastle upon Tyne, NBL, UK, 4 Bristol-Myers Squibb, Lawrenceville, NJ, USA
OBJECTIVES: The EQ-5D is widely used to inform economic evaluations of health technologies. However, the extent of its use for clinical outcome assessment (COA) is unclear. This review identified the prevalence with which EQ-5D data are evaluated by health authorities in clinical benefit assessments. METHODS: Drug technology assessments (TAs) published by HTA agencies in England, France, Germany and the US during the last 2 years were identified. Product labelling for drugs approved by the European Medicines Agency (EMA) and US Food and Drug Administration (FDA) over the last 5 years were also reviewed. Only documents reporting EQ-5D as a COA measure were included. RESULTS: EQ-5D data were reported for COA in 139 TAs with the majority reported for Germany (n=78) and the remainder for England (n=46), France (n=12) and the US (n=3). Visual analogue scale (VAS) scores were presented most frequently (n=111) followed by utility index scores (n=48) and dimension levels (n=1). The VAS accounted for 99% of EQ-5D reports in Germany. Minimally important differences (MIDs) were discussed in 51 TAs: 34% and 24% of VAS and index score reports, respectively. Three-hundred twenty drugs were approved by the EMA and 735 by the FDA, and among these 15 and 35, respectively, presented EQ-5D data for COA. All EQ-5D data submitted to the FDA were reported in supporting documentation. Index scores, VAS scores and dimension levels were cited for 32, 26 and 5 drugs, respectively. Discussion of MIDs was more frequent in EMA documents (35%) than FDA documents (11%). CONCLUSIONS: The EQ-5D has been used for COA in HTA submissions; most frequently the VAS in German TAs. EQ-5D was also used to support labelling claims in a minority of EMA and FDA decisions. No EQ-5D data were reported in FDA product labelling, which suggests that the data were not considered material.
P57: Mapping Quality of Life - Alzheimer's Disease (QOL-AD) Scores to EQ-5D-5L Utilities in the Ambar Trial Population
4:00PM - 4:15PM
Simons C1 , Gomez-Ulloa D 2 , Runken MC3 , Serrano D4 , Barnes B4 , Grifols C2 , Barcelo M2 , Podger L5 1 OPEN Health Group, York, NYK, UK, 2 Grifols, Sant Cugat del Vallès, Spain, 3 Grifols SSNA, Research Triangle Park, NC, USA, 4 OPEN Health Group, Bethesda, MD, USA, 5 OPEN Health Group, London, UK
OBJECTIVES: Health state utilities are required to model the cost/benefit of novel treatments in accordance with Health Technology Assessment guidelines. The AMBAR phase 2b/3 trial (NCT01561053) evaluated the efficacy and safety of plasma exchange with albumin replacement in mild-to-moderate Alzheimer’s disease (AD) patients. Health-related quality of life was measured through the disease-specific Quality of Life - Alzheimer’s disease (QoL-AD) questionnaire. EQ-5D-5L utilities were estimated from AMBAR QoL-AD data using a validated mapping algorithm developed by Rombach et al. (2020). METHODS: A multinomial logistic regression model was employed to generate mapped utility values for the placebo and pooled treatment arms for both patient and proxy-rated QoL-AD scores. The model included age and sex as covariates and was run including QoL-AD item 7 (marriage). The UK value set (crosswalk to EQ-5D-3L value set by van Hout et al. 2012) was applied. RESULTS: The analysis sample included 298 patients and 298 caregivers with complete baseline QoL-AD data (i.e., all 13-items completed). The mean mapped patient-rated EQ-5D-5L utility at final visit was 0.8132 for placebo and 0.8312 for pooled treatment. For proxy-raters, the mean mapped EQ-5D-5L utility at final visit was 0.7333 for placebo and 0.7235 for pooled treatment. The correlation between observed QoL-AD scores and mapped EQ-5D-5L utilities was 0.526 for patients and 0.552 for proxies, in line with the findings in Rombach et al. (2020). CONCLUSIONS: Mapped EQ-5D-5L utilities were estimated for the AMBAR population. The estimates were relatively consistent at final visit across treatment arms. However, the correlation between observed QoL-AD scores and mapped EQ-5D-5L scores remained moderate. This may indicate a general lack of conceptual overlap in the domains covered by the QoL-AD and the EQ-5D-5L, limiting the precision of a mapping approach and reinforcing the benefit of directly collecting utilities in AD trials for increased accuracy of estimates.
P60: A New Online Tool for Valuing Health States: Eliciting Personal Utility Functions for the EQ-5D-5L
4:45PM - 5:00PM
Schneider P 1 , van Hout B1 , Heisen M2 , Brazier JE1 , Devlin N3 1 University of Sheffield, Sheffield, UK, 2 Pharmerit International, Rotterdam, Netherlands, 3 University of Melbourne, Melbourne, Australia
OBJECTIVES: Standard health valuation methods, such as TTO or DCE are inefficient. They require data from hundreds if not thousands of participants to generate utility values (=value sets) for health descriptive systems. Here, we present the Online elicitation of Personal Utility Functions (OPUF) tool; a new type of online survey for valuing EQ-5D-5L health states using more efficient, compositional preference elicitation methods, which allows estimating value sets on the individual level. The objectives of this study are to report on the development of the tool, and to test the feasibility of using it to obtain individual-level value sets for the EQ-5D-5L. METHODS: We used an iterative design approach to adapt the PUF method, previously proposed by Devlin et al., for the EQ-5D-5L and for use as a standalone online tool. The valuation consists of three steps: level rating, dimension weighting, and anchoring. We conducted three iterative rounds of qualitative interviews to get feedback on the tasks and then piloted the tool in a sample of 50 participants from the UK.
RESULTS: On average, it took participants about 10 minutes to go through all tasks. Their responses indicated a good level of engagement. The two most important EQ-5D dimensions were Pain/Discomfort and Mobility. For 46 (92%) participants, we were able to construct a personal utility function. The results revealed that health state preferences differ considerably between individuals.
CONCLUSIONS: We successfully piloted the OPUF tool and showed that it can be used to derive a social as well as personal utility functions for the EQ-5D-5L. Even though the development of the online tool is in an early stage, there are potential avenues for further research. With some further abstraction, the OPUF tool could provide a modular software platform for creating valuation tools for any health descriptive system.
P58: EQ-5D-5L Population Norms for Italy
4:15PM - 4:30PM
Meregaglia M 1 , Finch AP2 , Malandrini F1 , Ciani O1 , Jommi C3 1 SDA Bocconi School of Management, Milan, Italy, 2 EuroQol Research Foundation, Amsterdam, Netherlands, 3 SDA Bocconi School of Management, Bocconi University, Milano, Italy
OBJECTIVES: The EQ-5D is a widely used instrument to measure patient-reported outcomes and health state utility values. This study aimed to report normative data for the EQ-5D-5L questionnaire in Italy based on a nationally representative sample. METHODS: This study is part of the EQ-5D-5L Italian valuation study. The target participants were a sample of the Italian adult population (aged 18 and above) and were recruited through a market research company. Eleven trained interviewers conducted one-to-one interviews using a videoconferencing software (Zoom) and a survey web application (LimeSurvey) between November 2020 and February 2021. The distribution of answers was estimated for the descriptive system of the EQ-5D-5L, and descriptive statistics were calculated for the visual analogue scale (EQ-VAS). Data analysis was performed using Stata (StataCorp). RESULTS: The sample was composed of 1,182 people and fully represented the Italian population (2020) in terms of age, gender, and geographical area. Mean age was 48.3; men were 48.7%. Half of the participants selected the two most common health states (i.e., ‘11111’ and ‘11112’). Mean VAS was 81.8, and steadily decreased with increasing age (from 87.0 in the 18-24 group to 75.1 among the over 75). In participants affected by chronic illness (39%), mean VAS dropped to 75.5. At least one problem (from slight to extreme) was reported by 12.1% of respondents for mobility, 4.2% for selfcare, 11.6% for usual activity, 43.3% for pain/discomfort and 41.2% for anxiety/depression. The frequency of problems generally increased with age, except for the last dimension, where 56% among the youngest reported complaints versus 30% of participants aged 75 and above. Moreover, self-reported anxiety/depression was far more common in women (49.7%) than in men (32.3%). CONCLUSIONS: EQ-5D-5L population norms provide useful insights into the health status of the Italian population and can be used as reference values for future surveys using the same tool.
Decision Modeling and Simulation
Live
This session covers studies using decision modeling and simulation to investigate the impact of different strategies to inform decision making.
Moderator
Linus Jönsson, MD, PhD
H. Lundbeck A/S, Copenhagen, Denmark
P5: Real-World Validation of the Implementation of Healthcare Capacity Optimization Measures Guided by the Simpli Tool: An Ophthalmology Proof of Concept in Portugal
4:00PM - 4:15PM
Mota M1 , Oliveira F1 , Moita Fidalgo R1 , Oliveira A1 , Franco C1 , Fernandes C1 , Martins I1 , Brito de Sá M1 , Silva JP1 , Fonte S1 , Perpetua P2 , Freitas R3 , Bandeiras C 4 1 Novartis Farma, Porto Salvo, Portugal, 2 Novartis Pharma Region Europe, Basel, Switzerland, 3 Novartis Pharma AG, Porto Salvo, Portugal, 4 Novartis Global Service Center, Dublin 4, D, Ireland
OBJECTIVES: Hospital services in ophthalmology face significant capacity constraints in Portugal. In 2020, SimPLI – Simulating Capacity Performance Leading to Impact was introduced to assist services in simulating the impact of measures to decrease the backlog of outpatient consultations and procedures. The aim of this work is to introduce the validation of the simulator outputs with the real-world production in a reference ophthalmology center in Portugal. METHODS: A spreadsheet-based simulator was developed for testing measures to accelerate the realization of delayed external consultations and outpatient surgeries. The baseline backlog is represented by the number of delayed procedures. The time to eliminate this backlog was calculated and compared with the output for scenarios where one or more measures were implemented. The number of consultations and procedures simulated at 2020 year-end (YE) was compared with real hospital production data for model validation. RESULTS: The implementation of 15-minute telemedicine consultations for follow-up appointments and the reduction in 10 minutes in the time of face-to-face consultations was predicted to increase the number of 1st consultations by 68% and the number of follow-up consultations by 77% in comparison with a scenario without any optimization measures. The time to solve outstanding consultations would be reduced by 7 months. In real practice in the same service, the application of the aforementioned measures was successful, with an additional 58% of 1st consultations and 85% follow-up appointments until 2020 year-end. In outpatient surgeries, the application of measures for capacity optimization reduced by 22% the number of patient lost to private hospitals, with considerable resulting savings. CONCLUSIONS: SimPLI is invaluable for planning the investment in efficient actions towards optimization of hospital capacity. The proof of concept demonstrates that the proposed measures were validated in real practice with improved provision of care in ophthalmology, eventually resulting in improved patient outcomes.
P6: Projecting COVID-19 Hospitalizations and Deaths Under Scenarios of Vaccination in Jefferson County, Kentucky
4:15PM - 4:30PM
Patel N University of Louisville, LOUISVILLE, KY, USA
OBJECTIVES : This report investigated the simulated effect of several vaccination scenarios on COVID hospitalizations and deaths in Jefferson County, Kentucky. Study Design: Eight scenarios were considered. First, it was assumed that the status quo scenario (~30,000 doses of Pfizer and Moderna vaccines distributed and administered every week) would continue without Johnson & Johnson’s vaccine. Then, three scenarios of the addition of Johnson & Johnson’s vaccine (10,000, 15,000, and 20,000 weakly) were considered. Next, an expansion over the status quo scenario (~40,000 doses of Pfizer per week) was considered with and without Johnson & Johnson’s vaccine scenarios. METHODS : An epidemic dynamics model (namely, a Susceptible-Exposed-Infectious Recovered (SEIR) model) is adopted and estimated in this study. In the model, transmission through different phases of the COVID-19 epidemic (susceptible, exposed, infectious, hospitalized, vaccinated, recovered, and dead) is regulated with transmission and clinical dynamics parameters. Key transmission parameters are the population, basic and effective reproduction factors, lengths of incubation periods, pre-infectiousness, infectiousness with and without symptoms, and vaccines’ efficacy rates and coverage. Key clinical dynamics parameters are hospitalization rate among the symptomatic, time from onset of severe symptoms to hospitalization, length of hospital stay, fatality rate among the hospitalized, recovery time among the hospitalized, the time from hospitalization to death. RESULTS : More intense vaccination than the status quo is expected to decrease hospitalizations and deaths in the next three months. However, the magnitude of the decrease in deaths is small, < 3 dozen. Importantly, it is expected that the COVID-19 infection continues to spread. Therefore, social distancing and other COVID-19 protection measures (for example, mask-wearing) must continue – should they be relaxed, a “during vaccination surge” will occur and should be expected in the late April-early May period. CONCLUSIONS : Implications for Policy or Practice are Social distancing and other COVID-19 protection measures (for example, mask-wearing) must continue.
P8: New Onset Cardiovascular Disease in Australia by Socioeconomic Groups: A Modelling Study
4:45PM - 5:00PM
Hastings K1 , Marquina C2 , Talic S2 , Zomer E2 , Morton J2 , Liew D1 , Ademi Z 2 1 Monash University, Melbourne, VIC, Australia, 2 Monash University, Melbourne, Australia
OBJECTIVES : To project incident cardiovascular disease (CVD) and related health economic outcomes in Australia by socioeconomic status between 2020 and 2029. METHODS : A dynamic population model was built to project the annual incidence new-onset CVD by quintile of socioeconomic disadvantage in Australians aged 40-90 years between 2020 and 2029 using the Pooled Cohort Equation (PCE). The model projected years of life lived, quality adjusted life years (QALYs), direct healthcare medical costs, and productivity losses due to new-onset CVD. All outcomes were discounted by 5% annually. RESULTS : Cardiovascular risk profiling using the PCE showed that 20% of the most disadvantaged quintile were considered at high risk of CVD, compared to 12% in the least disadvantaged quintile. From 2020 to 2029, the model projected 211,901 incident cardiovascular events would occur in the most disadvantaged quintile compared to 184,846 in the least disadvantaged. Acute healthcare costs in the most socioeconomically disadvantaged group were AU$ 206 million higher than in the least disadvantaged group, while the difference in societal costs was AU$ 820 million. Scenario analyses estimated that a 17% risk reduction in CVD would be needed in disadvantage quintiles 1-4 to achieve the same outcomes as the least disadvantaged quintile (quintile 5). CONCLUSIONS : The number of CV events and associated costs highlight the urgent need to implement scalable primary prevention interventions targeted at disadvantaged groups. This model provides a platform to assess which interventions are likely to yield more benefits in each socioeconomic group at the population level.
P7: Evaluating Impact of Universal Varicella Vaccination Strategies on Clinical Burden of Varicella and Herpes Zosterin England and Wales
4:30PM - 4:45PM
Pillsbury M1 , Sharomi O 2 , Xausa I3 , Nachbar R3 , Matthews I4 , Pawaskar M5 1 Merck & Co., Inc., Rahway, NJ, USA, 2 Merck & Co., Inc., West Point, PA, USA, 3 Wolfram Solutions, Champaign, IL, USA, 4 Merck Sharp & Dohme Ltd., Wokingham, WOK, UK, 5 Merck & Co., Inc., Kenilworth, NJ, USA
OBJECTIVES : England and Wales have not implemented universal varicella vaccination (UVV) primarily due to its hypothesized impact on herpes zoster (HZ) incidence. Our study evaluated long-term clinical impact of UVV and exogenous boosting on varicella and HZ in England and Wales. METHODS : An age-structured, deterministic, dynamic transmission model using a dynamic population was adapted to England and Wales to assess varicella cases, associated hospitalizations and HZ cases. Ten (one- and two-doses, with/ without catch-up) vaccination strategies at short (12m/18m) and medium (18m/40m) dose intervals with and without catchup for 2 doses at 14 and 15 years of age were compared to no vaccination over a 50-year time horizon. Four varicella vaccines were considered with monovalent and quadrivalent formulations [Varivax® , ProQuad® (V/MMRV-MSD) Varilrix® and Priorix-Tetra® (V/MMRV-GSK)]. Vaccination coverage was assumed to be 91% for first doses and 89% for 2nd doses and 87% for catch-up. The model accounted for the impact of exogenous boosting on HZ cases. RESULTS : All vaccine strategies substantially reduced the clinical burden of varicella over no vaccination: with 82-97% reduction in total varicella cases and 78%-86% reduction in the number of hospitalizations. One-dose strategies without catchup resulted in the smallest reduction in cases, hospitalizations, while the greatest reduction was seen with the 2-dose short-interval (12m and 18m) strategy. Incidence of HZ is estimated to be reduced by 7-11%. Strategies with V/MMRV-MSD vaccines were more effective in averting all four outcomes than with V/MMRV-GSK vaccines with similar intervals. CONCLUSIONS : Our model estimated that all one and two-dose UVV strategies significantly reducted the clinical burden of varicella including reduction in varicella related incidence, and hospitalization as well as reduction in HZ incidence compared to no vaccination in England and Wales. Policymakers should consider including UVV in their childhood immunization program to reduce disease burden.
Impact of the COVID-19 Pandemic: Investigations in Populations of Interest
Live
This session covers investigations on different populations of interest in regard to COVID-19 - pregnant and postpartum mothers, young adults and mental health, and infants.
Moderator
Filipa Sampaio, MSc, PhD
Uppsala University, Uppsala, Sweden
P26: The Practice of Face Masking Among Young Adults in South India: An Online Cross-Sectional Survey During Second Wave of COVID-19
4:15PM - 4:30PM
Kochuparambil J 1 , Issac A2 1 Mary Queen's Mission Hospital, Kanjirappally, India, 2 Mary Queen's Mission Hospital, Adoor, India
OBJECTIVES: COVID-19 pandemic urges the need for respiratory protective equipment like face masks as a public health measure to control the spread of infection. This study aimed to investigate the trends followed in the practice of mask-wearing by the South Indian population amid the second wave of COVID-19 outbreak in 2021. METHODS: A web-based, online cross-sectional survey was conducted among the young adult population in India in late April 2021. An eight-item questionnaire was designed to assess the social perceptions and attitudes regarding wearing a face mask as a part of universal safety precautions. The social perceptions towards wearing masks were categorized as excellent, good average and poor on a scale (Social Perception Scale -SPS) scored out of 8. The details collected using a predesigned google form are statistically analyzed using the Chi-square test with a p-value of < 0.05 is considered statistically significant. RESULTS: Among the 1283 participants who completed the questionnaire, 57% wore cloth masks followed by 26% wearing N95 masks and 12% wearing surgical masks. Even though the age of the study population varied from 19 – 76 years and with a male preponderance of 56.3% (n = 723), students and recent graduates participated largely in the study (71.8%, n = 922). A mean SPS score of 5.67±1.07 (out of 8) indicates that the social perception of the study population is good. A statistically significant association is observed between the SPS score and the age (p = 0.003), type of mask used (p < 0.001), and economic background of the study population (p <0.001). Breathing difficulty, communication problems, additional cost incurred and dermatologic issues were commonly reported barriers against mask-wearing. CONCLUSIONS: Adjunctive public health measures such as mask-wearing are crucial in curbing the COVID-19 transmission. By shaping an appropriate public attitude, policymakers can ensure compliance towards mask-wearing.
P27: Vaccination Coverage Trends for Hepatitis B in Infants from the Brazilian and Colombian Expanded Immunization Program: A Real-World Analysis of COVID-19 Pandemic Impact
4:30PM - 4:45PM
Lima P1 , Abreu A2 , Hernández F3 , Martins J2 , Kano B4 , Kashiura D2 , Julian G 1 1 IQVIA Real World Insights, São Paulo, SP, Brazil, 2 IQVIA Brasil, São Paulo, SP, Brazil, 3 IQVIA Colombia, Bogotá, Colombia, 4 IQVIA, São Paulo, SP, Brazil
OBJECTIVES : COVID-19 pandemic has posed major challenges for healthcare systems and societies worldwide. Mitigation measures and the fear of exposure to COVID-19 might have negatively impacted local health policies, such as pediatric immunization programs strategies. This observational study aims to analyze the vaccination coverage (VC) for hepatitis B in infants in Brazil and Colombia between 2015 to 2020. METHODS : This is a descriptive analysis using real-world data from the Expanded Immunization Program System from Brazil (SI-PNI) and the Epidemiological Surveillance System from Colombia (SIVIGILA). We calculated the annual variation of VC for hepatitis B in infants from 2015 to 2020 for both countries. RESULTS : Overall, Brazilian VC had an average annual decline of 3.6% in the pre-COVID-19 period (2015-2019), reaching the lowest coverage in 2019 (78.57%), while the Colombian VC had an increasing pattern for the same period (0.4% annually), reaching the highest coverage in 2017 (89.3%). In 2020, VC decreased by 19.8% in Brazil, compared with 2019. In Colombia, VC decrease was notably lower (1.0%). CONCLUSIONS : In Colombia, VC increase might be explained by the implementation of the national plan for hepatitis B elimination in infants during this period. In Brazil, VC coverages for several other infectious diseases have also faced a decrease during the last years, but no formal mitigation activity or plan was yet established. Although both countries showed a reduction of the VC coverage in 2020, the impact was considerably higher in Brazil. These trends could be explained by the distinct health strategies linked to the Expanded Immunization Programs for each country in preparation for the COVID-19 pandemic.
P28: Impact of COVID-19 on Mental Health in Young Adults in the United States
4:45PM - 5:00PM
Intermill T 1 , Gong CL2 , Gu NY3 1 University of San Francisco, Sacramento, CA, USA, 2 Children's Hospital Los Angeles, Los Angeles, CA, USA, 3 NYG Technologies, Santa Clarita, CA, USA
OBJECTIVES: To assess the impact of COVID-19 pandemic on mental health in young adults in the US. METHODS: Three waves of online surveys were designed to capture mental health status in the US (EuroQol grant: 84-2020RA): Wave1 (Apr 1st – May 6th , 2020 (n=2,734)), Wave2 (July 4th – Sept 4th , 2020 (n=2,454)), and Wave3 (Jan 10th - Mar 15th , 2021 (n=2,252)) using the EQ-5D-5L to evaluate respondent’s health-related quality-of-life (HRQoL) and the Patient Health Questionnaire (PHQ-4) to assess anxiety and depression. The EQ-5D-5L utility, VAS scores and 5 domains were stratified by age, gender, and race/ethnicity. Binary Logistic regressions were used to estimate the associations between anxiety/depression and various covariates. Chi-square tests were conducted for significant differences in mental health outcomes between age groups. RESULTS: Most participants were white (68.7%) non-Hispanic (89 %). On average, participants were 42 (±13) years old, 47% being female. In all 3 waves, self-reported anxiety and depression were significantly higher in young adults (18-34) compared with older adults (35+) (p<0.01). Anxiety scores were 42%, 53%, and 33% in waves 1-3 respectively for young adults, whereas 33%, 40%, and 22% were reported by adults 35-64 and 19%, 20%, and 12% were reported by adults 65+. Similar trends were observed for depression, with younger adults reporting 39%, 54% and 35%, compared with 27%, 38% and 22% for those aged 35-64 years and 14.5%, 16% and 14.85% for 65+. EQ-5D-5L utility in waves 1-3 were 0.82, 0.75, and 0.82 (P<0.01) and 74.7, 78.7, and 76.4 for EQ-VAS (P<0.01). Age and employment status were significant predictors for anxiety and depression outcomes. CONCLUSIONS: Mental health deterioration during COVID-19 was pronounced among young adults for all waves, especially in wave2. Findings suggest although people adapt over time, the US was ill-prepared for a mental health crisis, especially among young adults.
P25: Impact of COVID-19 on the Health-Related Quality-of-Life of Pregnant and Postpartum Persons
4:00PM - 4:15PM
Regan A1 , Aytha Swathi P2 , Nosek M1 , Gu NY 3 1 University of San Francisco, Sacramento, CA, USA, 2 University of San Francisco, Signal Hill, CA, USA, 3 NYG Technologies, Santa Clarita, CA, USA
OBJECTIVES: To assess the impact of COVID-19 on health-related quality-of-life (HRQoL) of those who were pregnant or recently pregnant during the pandemic. METHODS: Individuals who were pregnant any time since January 2020, the beginning of the pandemic, were invited to participate in an online, national US survey (EuroQol grant: 260-2020RA). Respondents were asked to self-report their experiences with COVID-19, to complete the EQ-5D-5L, and other measurements of HRQoL. To estimate the association between COVID-19 infection with the EQ-5D-5L outcomes, we used median regression for the EQ-5D utility and EQ-VAS scores, and ordinal logistic regressions for the EQ-5D-5L health items. Post-stratification weights were used to ensure representation by age, race and US census region. RESULTS: Among pregnant or postpartum persons, the median EQ-5D-5L utility score was 0.87 and EQ-VAS was 0.80. The median EQ-5D-5L utility score increased by 0.0058 (95% CI 0.0026, 0.009) for each additional year of age of the respondent. We observed no change in EQ-5D-VAS utility measures by maternal age ( ß= 0.00; 95% CI -0.09, 0.09). On average, comparing Black pregnant persons to White, EQ-5D-5L utility values were 0.44 points lower, and EQ-5D-VAS scores were 0.31 points lower. Although median EQ-5D-5L utility values were similar for those with and without a diagnosis of COVID-19 (0.87 and 0.88), utility values declined by 0.022 (95% CI -0.040, -0.010) for each unit increase in perceived COVID-19 severity. Similar results were observed for the EQ-5D-VAS scores. When we evaluated EQ-5D-5L items individually, respondents diagnosed with COVID-19 reported more problems related to anxiety/depression compared with those who did not (OR 2.43; 95% CI 1.35, 4.40). No other items were significantly associated with COVID-19. CONCLUSIONS: We observed lower HRQoL measures associated with severe COVID-19 infection during pregnancy. In particular, problems with anxiety and depression contributed most strongly to lowered HRQoL during pregnancy.
Methodological Developments in Survival Analytic Methods to Inform Cost-Effectiveness Models
Live
This session will explore advances in methods for developing appropriate parametric survival models to inform cost-effectiveness models. The session will include methods for fitting parametric models in the context of multistate models, methods for jointly analysing PFS and OS, estimating subgroup-specific effects and using external historical data to inform model development.
Moderator
Jeroen P Jansen, PhD
School of Pharmacy, University of California San Francisco, San Francisco, CA, USA, and PRECISIONheor, Oakland, CA, USA
P46: Effective Use of Reconstructed Survival and Comparative Effectiveness Data: A Case Study from Estimating Unreported Subgroup Survival in Advanced Stage Gastrointestinal Cancers
4:15PM - 4:30PM
Alagoz O 1 , Xiao H2 , Singh P2 , Gricar J2 , Dixon M2 , Kim I2 , Kurt M3 1 University of Wisconsin-Madison, Madison, WI, USA, 2 Bristol Myers Squibb, Lawrenceville, NJ, USA, 3 Bristol Myers Squibb, Princeton, NJ, USA
OBJECTIVES : This study devises a systematic approach that can utilize aggregate level survival and comparative effectiveness data published from randomized controlled trials (RCT) to assist subgroup-specific health economic and meta-analyses. METHODS : We developed a soft-constrained optimization model, which approximates the restricted mean survival time (RMST) for the overall population in each arm via weighted sum of the RMSTs of two subgroups of interest. Survivals of both subgroups in each arm were assumed to follow Weibull or log-logistic distribution. The constraint ensured that cumulative hazards between the arms were proportional for each subgroup at a sufficiently long pre-specified time point. Estimated subgroup-specific survival functions for the control arm were direct outputs of the model and were shifted by applying the reported hazard ratios from the forest plots to generate their counterparts for the intervention arm assuming proportional hazards between the arms. For validation, we tested our approach in a case study consisting of 10 distinct RCTs with reported subgroup-specific Kaplan-Meier (KM) curves from advanced stage gastrointestinal tumors. RESULTS : Across all 48 subgroups, on average, loglogistic model performed equally or better than Weibull model in performance criteria comparing overall survival (OS) rates, median OS and RMSTs. Predicted survival curves laid within the 95% confidence intervals (CIs) of reported KM-curves in 75% and 81% of the time for Weibull and loglogistic models, respectively. Predicted median survivals were within the 95% CIs of the reported medians in 34 and 40 subgroups for Weibull and loglogistic models, respectively. Average relative gap between the predicted and reported RMSTs was 10% in both models. Predicted RMSTs were within the 95% CI of reported RMSTs in 34 and 37 subgroups for Weibull and loglogistic models, respectively. CONCLUSIONS : Our elicitation approach is effective and demonstrably reliable in deriving unreported subgroup survival with flexible time-varying hazard functions.
P47: A Bayesian Hierarchical Mixture Cure Modelling (MCM) Framework for the Joint Utilization of Progression Free Survival (PFS) and Overall Survival (OS) in Estimating Long-Term Survivorship Rates in Previously Untreated Metastatic Melanoma: A ...
4:30PM - 4:45PM
Green N 1 , Paly V2 , Youn JH3 , Kurt M4 , Moshyk A5 , Baio G1 1 University College London, London, LON, UK, 2 ICON plc, New York, NY, USA, 3 ICON plc, Marlow, Bucks, UK, 4 Bristol Myers Squibb, Lawrenceville, NJ, USA, 5 Bristol Myers Squibb, Princeton, NJ, USA
OBJECTIVES: Differences in emergent survival plateaus between PFS and OS may imply clinically unintuitive dichotomy between the resulting proportions of long-term survivors (LTS) when they are analyzed separately via mixture cure models (MCM). We present a novel Bayesian hierarchical (BH) MCM framework assuming a dependency between PFS and OS to estimate LTS rates in CheckMate 067 and demonstrate its practical utility over frequentist MCMs in long-term QALY estimations. METHODS: Frequentist and BH MCMs were fitted to PFS and OS data from the trial with minimum 60-months of follow-up. In the frequentist MCMs, PFS and OS were modelled separately whereas in BH MCMs both endpoints were modelled jointly with a shared LTS rate. In both approaches, background mortality rates were taken from World Health Organisation’s age, gender and country-specific lifetables and time-to-event outcomes for the non-LTS were modeled using a range of standard parametric distributions. Estimated incremental QALYs gains for nivolumab containing therapies versus ipilimumab under both approaches were compared using local tariffs from US, Canada, UK, France, Sweden, Belgium, Netherlands, Portugal and Australia. RESULTS: Among all combinations of distributions considered for the BH MCM, the exponential-exponential fit adequately captured the observed survival trends for both endpoints with reasonable goodness-of-fit measures and shared LTS rates which were (95% credible intervals) 46.3% (32.8%, 62.6%) for nivolumab+ipilimumab, 37.8% (21.6%, 55.7%) for nivolumab, and 15.1% (6.8%, 26.0%) for ipilimumab. For each arm, shared LTS-rates were in between individually-estimated LTS rates from the OS and PFS data in the frequentist MCMs. Compared to frequentist MCMs, over 20-years BH MCMs produced higher incremental QALY gains for nivolumab+ipilimumab and nivolumab versus ipilimumab with differences ranging from 0.30-0.48 and 0.17-0.26, respectively. CONCLUSIONS: Our BH MCM framework can alleviate the disparity between individually estimated OS- and PFS-based LTS rates, and allow for more robust clinical inference and extrapolations.
P45: The Use of Historical Clinical Trial Data to Inform Survival Extrapolation
4:00PM - 4:15PM
Pham HA 1 , Smalbrugge D2 , Kroi F2 , Heeg B1 , Ouwens M3 1 Ingress-health, Rotterdam, ZH, Netherlands, 2 Ingress-health, Rotterdam, Netherlands, 3 AstraZeneca, Mölndal, O, Sweden
OBJECTIVES : Standard parametric distributions are commonly used for the extrapolation of survival data in cost-effectiveness analyses. However, survival data is often immature and uncertainty remains around the survival extrapolations. Mature historical data can be used to better predict survival beyond trial data. This study assessed two methods to incorporate historical data in the extrapolation of immature survival data. METHODS : Immature data of a breast cancer trial comparing pertuzumab+trastuzumab+docetaxel versus trastuzumab+docetaxel (follow-up time 38 months; data-cut 2015) was extrapolated and mature survival data (follow-up time 120 months; data-cut 2020) from the same trial was used to validate the extrapolations. The historical data was from a previous breast cancer trial including mature survival data of trastuzumab+docetaxel (follow-up time 50 months; data-cut 2005). Two methods to quantitatively inform the extrapolation of immature survival data with historical data were compared to standard parametric distributions: 1) historical shape parameter as informative prior for the shape of the immature data; 2) historical data as a third arm. Predictions were assessed with delta area under the curve (AUC) values based on the mature survival data. RESULTS : Without priors, the delta AUC was 7.59, 1.62, 13.15, 8.32, 25.15, and with the historical arm the delta AUCs were 9.65, 4.38, 6.79, 8.26, 21.81, for Weibull, loglogistic, lognormal, exponential, and Gompertz, respectively. With priors, the delta AUC were 8.43, 3.37, 9.11, 23.68, for Weibull, loglogistic, lognormal, and Gompertz, respectively (as for exponential there is no shape parameter). The loglogistic distribution without priors predicted the immature data the best. For three out of five distributions, the extrapolations with a historical arm resulted in better predictions compared to the extrapolations without prior. CONCLUSIONS : The impact of external data on clinically plausible survival extrapolations can further be improved by using historical data with longer follow-up with treatment patterns similar to the current standard of care.
Emerging Opportunities for the Use of Real World Data in Comparative Effectiveness Research
Live
This session illustrates applications around the use of real world data and electronic health records to conduct comparative effectiveness research studies or reduce uncertainty on long term outcomes in HTA evaluations.
Moderator
Isaac Corro Ramos, PhD
institute for Medical Technology Assessment, Eindhoven, NB, Netherlands
Isaac Corro Ramos, PhD obtained his Master’s degree in Mathematics (option Statistics and Operations Research) from the University of Sevilla in June 2001. Between June 2001 and July 2005 Isaac had several jobs in different working areas and countries. He worked as a high-school Mathematics teacher in Sevilla (Spain), as a software programmer in Madrid (Spain) and Vienna (Austria), and as a researcher in Rome (Italy). In July 2005 he started his Ph.D. at the Department of Mathematics and Computer Science of the Eindhoven University of Technology working on the STRESS (Statistical Testing and Reliability Estimation of Software Systems) project. He defended his thesis on December 15, 2009. Since August 2009 Isaac works as a scientific researcher at the Institute for Medical Technology Assessment (iMTA) in Rotterdam, The Netherlands. Since he enrolled iMTA he has worked in several research projects whose subjects include probabilistic modelling, cost-effectiveness analysis of health care technologies, value of information analysis and discrete event simulation.
P15: Transcatheter versus Surgical Aortic Valve Replacement: A Real-World Comparison of Clinical Outcomes Based on a German Claims Dataset
4:30PM - 4:45PM
Hardtstock F 1 , Wilke T2 , Maywald U3 , Spitzer S4 1 Cytel Inc, Wismar, Germany, 2 IPAM e.V., Wismar, Germany, 3 AOK PLUS, Dresden, Germany, 4 Praxisklinik Herz und Gefäße, Dresden, Germany
OBJECTIVES : This study aimed to describe clinical outcomes after transcatheter aortic valve implantation (TAVI) and surgical aortic valve implantation (SAVR). METHODS : This study consisted of a retrospective analysis of German health insurance claims data from 01/01/2013-30/06/2019. Continuously insured adults with either TAVI (OPS 5-35a.0) or SAVR (OPS 5-351.0) between 01/01/2014 and 30/06/2018, who had aortic valve stenosis (ICD-10 I35.0, I35.2) were included. Patients with previous TAVI or SAVR were excluded. Both cohorts were described with regards to their baseline characteristics (one-year baseline) and the incidence rate (IR) of events during the follow-up period for death, transient ischemic attack (TIA), stroke, major bleeding event, periprocedural complications, and myocardial infarction (MI). RESULTS : Overall, 2,932 TAVI and 826 SAVR patients were identified. Compared to SAVR patients, TAVI patients were on average older (81.75 years vs. 69.18 years), more often female (56.92% vs. 42.37%), more comorbid (CCI 5.86 vs. 3.82; CHA2DS2-VASc-Score 3.17 vs. 2.47), and they had a higher probability of previous TIAs (3.07% vs. 1.33%), strokes (8.29% vs. 4.00%), and MIs (10.57% vs. 3.87%). 3.07%/1.21% of TAVI/SAVR patients died during the index hospitalization. Outcomes were observed during a follow-up period of 2.43 years (TAVI) / 3.02 years (SAVR). The following IR have been observed for TAVI/SAVR: death (0.17 vs. 0.04; p<0.001), TIA (0.00 vs. 0.01, p=0.046), stroke (0.03 vs. 0.01, p<0.001), major bleeding event (0.08 vs. 0.04, p<0.001), periprocedural complications during index hospital stay (1.87 vs. 1.13, p<0.001), and MIs (0.02 vs. 0.00; p<0.01). CONCLUSIONS : TAVI has become the new standard of care in recent years and has replaced the classic aortic valve replacement, specifically in more fragile patients. The above results confirm that TAVI procedures are widely used in clinical practice, and that in line with current guidelines, physicians assess which patients should receive a TAVI or a SAVR procedure.
P14: Exploring the Potential for EHR-Derived Real-World Data to Reduce Uncertainty in HTA Decision-Making: A Case Study of Long-Term Survival Outcomes
4:15PM - 4:30PM
Pittell H 1 , Kent S2 , Groves B2 , Mpofu P3 , Baxi S3 , Copeland A3 , Bargo D4 , Adamson B3 , Jonsson P5 1 Flatiron Health, Great Neck, NY, USA, 2 National Institute for Health and Care Excellence (NICE), London, LON, UK, 3 Flatiron Health, New York, NY, USA, 4 Flatiron Health, Brooklyn, NY, USA, 5 National Institute for Health and Care Excellence (NICE), Manchester, UK
OBJECTIVES : Clinical trials are an important source of evidence for health technology appraisals (HTA). However, a key concern is uncertainty in survival due to immature data. This study investigates whether electronic health record (EHR)-derived data from the US may have the potential to reduce uncertainty in long-term outcomes, using NICE technology appraisal (TA) 531 as a case study. METHODS : We selected patients with previously untreated, Stage IV NSCLC, with positive or unknown PDL1 status, who initiated first-line pembrolizumab monotherapy between October 2016 and December 2020 from the nationwide de-identified EHR-derived Flatiron Health database. We applied additional lab and ECOG eligibility criteria. Outcomes were overall survival from treatment start and treatment duration. Sensitivity analyses assessed a sub-group with known PDL1 status and a time horizon ending at NICE TA publication. RESULTS : The study included 1109 patients (median age 72, 50% female). Real-world duration of therapy was median 5.0 months (95% Confidence Interval [CI]: 4.2-5.7). Median overall survival was 13.8 months (95% CI: 11.8-16.2) over the full study period. The sensitivity analysis excluding patients with missing PDL1 status found median overall survival of 14.9 months (95% CI: 12.5-17.6). The shorter time horizon (October 2016 to June 2018) estimated median overall survival of 13.1 months (95% CI: 10.8-NR). CONCLUSIONS : In this case, EHR-derived data offered longer follow-up time (max 49 months) than the trial follow-up (max 22 months) used for extrapolation. This cohort had a median overall survival of 13.8 months while the trial (n=154) estimated 30.0 months and a similar Medicare claims analysis (n=3079) estimated 11.4 months. Real-world median age was 7-9 years older than the trial. Our study demonstrates that EHRs can be a source of mature data on specific cohorts of interest with potential to contextualize trial evidence and inform HTA-decision making.
P13: Use of Real-World Big Data to Assess the Effectiveness on Overall Survival Among Chemotherapy or Immunotherapy in First Line Metastatic Non-Small Cell Lung Carcinoma Patients in an Italian Setting
4:00PM - 4:15PM
Degli Espositi L1 , Sangiorgi D2 , Ancona DD3 , Andretta M4 , Barbieri A5 , Bartolini F6 , Cavaliere A7 , Chinellato A8 , Ciaccia A9 , Cillo MR10 , Citraro R11 , Costantini A12 , De Francesco A11 , Dell'Orco S13 , Di Manno G13 , Ferrante F14 , Gentile S15 , Lavalle A15 , Moscogiuri R16 , Pastorello M17 , Procacci C3 , Re D18 , Santoleri F12 , Ubertazzo L19 , Vercellone A20 , Perrone V21 , Dovizio M 2 1 CliCon S.r.l. Health, Economics & Outcomes Research, Ravenna, Italy, 2 CliCon S.r.l. Health, Economics & Outcomes Research, Bologna, BO, Italy, 3 ASL BAT, Trani, Italy, 4 Azienda ULSS 8 Berica, Vicenza, Italy, 5 ASL Vercelli, Vercelli, Italy, 6 USL Umbria 2, Terni, Italy, 7 ASL Viterbo, Viterbo, Italy, 8 Azienda ULSS 3 Serenissima, Mestre (VE), Italy, 9 ASL Foggia, Foggia, Italy, 10 ASL Salerno, Salerno, Italy, 11 Azienda ospedaliero-universitaria Mater Domini, Catanzaro, Italy, 12 ASL Pescara, Pescara, Italy, 13 ASL Roma 6, Albano Laziale, Italy, 14 ASL Frosinone, Frosinone, Italy, 15 Direzione Generale per la Salute Regione Molise, Campobasso, Italy, 16 ASL Taranto, Taranto, Italy, 17 ASP Palermo, Palermo, Italy, 18 ASL Teramo, Teramo, Italy, 19 ASL Roma 4, Civitavecchia (RM), Italy, 20 ASL Napoli 3 SUD, Torre del Greco, Italy, 21 CliCon S.r.l. Health, Economics & Outcomes Research, Bologna, Italy
OBJECTIVES : The use of big data to assess the effectiveness of oncological treatments in clinical practice is gaining increasing interest. This analysis aimed to assess the overall survival of metastatic non-small cell lung (met-NSCLC) patients receiving chemotherapy (CT) or immunotherapy (I/O) as 1st line by using real-world data in a sample population in Italy. METHODS : A retrospective observational analysis based on administrative data from a sample of Italian Local Health Units was conducted. Met-NSCLC patients starting a 1st line therapy with CT or I/O between 2017-2018 were identified. Stopping inclusion period up to 2018 enabled at least a two-years follow-up period for each included patient. Kaplan Meier overall survival analysis considered time (months) from therapy initiation to death. Multivariable analysis was performed to adjust for cofounders such as age, gender, metastasis, BRAF test prescription and pharmacological treatments. RESULTS : A total of 3,126 (mean age±SD 68.6±9.8 years, 68.2% male) and 316 (mean age±SD 68.6±9.7 years, 74.4% male) patients initiated treatment with CT and I/O respectively. In both groups, the more frequent metastases detected were related to lymph nodes (42.1% CT, 24.1% I/O), bone (25.8% CT, 14.9% I/O) and brain (18.3% CT, 10.1% I/O). Median [95%CI] survival was 8.0 [7.4-8.6] and 14.6 [12.2-18.9] months for CT and I/O patients, respectively. Death was not reported in 31.2% of CT and in 44.3% of I/O cohorts. Multivariable analysis showed the risk of death to be significantly lower in patients treated with I/O compared to CT (HR [95%CI] 0.796 [0.681-0.930]). CONCLUSIONS : Results from our study showed among met-NSCL patients in 1st line a better overall survival of the I/O compared to CT patients and a reduced risk of death of I/O vs CT-treated patients. Our findings suggest real-world data could produce valuable insights into treatments and their outcomes in routine daily oncology practice, thus integrating the evidence from clinical trials.
P16: Recent Estimates of Survival in Patients with Advanced Non-Small Cell Lung Cancer (NSCLC) in the US (2010-2020)
4:45PM - 5:00PM
Kalilani L1 , Chao J2 , Hogea C3 , Stojadinovic A 4 , Giove TJ5 , Sun X1 , Aziez A6 , Velcheti V7 1 GlaxoSmithKline, Durham, NC, USA, 2 GlaxoSmithKline, Collegeville, PA, USA, 3 GlaxoSmithKline, Philadelphia, PA, USA, 4 GlaxoSmithKline, Upper Providence, PA, USA, 5 GlaxoSmithKline, Mississauga, ON, Canada, 6 GlaxoSmithKline, Zug, Switzerland, 7 New York University Langone, New York, NY, USA
OBJECTIVES : Despite availability of new treatments, the prognosis of lung cancer remains poor. This study aims to provide recent estimates of survival in patients with advanced non-small cell lung cancer (NSCLC) in the US. METHODS : The survival of patients with advanced NSCLC was estimated using two US databases together covering 2010–2020. The study included patients with stage III or IV NSCLC diagnosed between 2010–2016 in the Surveillance, Epidemiology, and End Results Program (SEER) database, and patients with stage IIIB, IIIC or IV NSCLC, diagnosed between 2017–2020, without known oncogenic driver mutations who had completed ≥4 cycles of 1L treatment (restricted to platinum-based combinations, immuno-oncology monotherapy, or ipilimumab/nivolumab) in the Flatiron Health database, a US Oncology Electronic Medical Record database. Overall survival (OS) was defined as time from diagnosis of stage III or IV NSCLC to death or to date of last confirmed activity. RESULTS : A total of 49,298 and 133,395 patients with stage III and IV diagnosis respectively were identified in SEER. The 1-, 3- and 5-year OS for patients with Stage III disease were 55.1%, 26.3% and 17.5%, and for stage IV disease were 25.8%, 7.4% and 4.0%, respectively. The Flatiron database had 1,045 patients with stage IIIB, 130 patients with stage IIIC and 3,210 patients with stage IV disease at diagnosis. The 1- and 3-year OS for stage IIIB/IIIC disease were 72.5% and 36.4%, and for patients with stage IV disease were 65.9% and 24.6%, respectively. CONCLUSIONS : Despite differences in study population characteristics between the two databases, the study shows that mortality in patients with advanced NSCLC remains high, underscoring the need for continued efforts to identify novel treatments and synergetic treatment combinations to improve patient outcomes.
Using Real World Evidence to Predict Risks of Adverse Outcomes and Disease Progression
Live
Understanding patients risks of adverse outcomes and their rate of disease progression, and how this varies according to patient characteristics is essential in understanding the benefits of intervention. In this session we will explore how large real world data sets are being used to quantify these outcomes using examples from oncology and rheumatology. We will also hear how novel approaches to accessing and analysing data may facilitate use of datasets that must be stored in different geographical locations.
Moderator
Praveen Thokala, MASc, PhD
Sheffield University, Sheffield, United Kingdom
Praveen Thokala joined the School of Health and Related Research (ScHARR) at the University of Sheffield after completing an MASc from the University of Toronto and a PhD from the University of Southampton.
During this time, he has completed several health economics projects including single technology appraisals (STA), multiple technology appraisals (MTA), and diagnostic assessment reports (DARs) for NICE. He has also recently led a modeling project for Institute for Clinical and Economic Review (ICER) in the US. His research interests include health economic modeling, multi-criteria decision analysis (MCDA), discrete event simulation modeling, and optimization. He co-supervised six PhD students to completion and currently co-supervises four PhD students.
In terms of MCDA, he has worked with the National Institute of Health and Care Excellence decision support unit (NICE DSU) in the UK on exploring the applicability of MCDA in HTA. He co-chaired the ISPOR Task Force on the use of MCDA in healthcare decision making and has been involved in several MCDA studies including supporting priority setting and benefit-risk analysis. He also co-edited a book titled, Multi-Criteria Decision Analysis to Support Healthcare Decisions.
P74: Treatment Patterns and Outcomes in Patients with Acute Myeloid Leukaemia (AML) in England: A Cancer Analysis System (CAS) Registry Retrospective Cohort Study
4:15PM - 4:30PM
Caseby S 1 , Cranmer H2 , Ohlmeyer V1 , Groucott J1 , Eaton J1 , Podkonjak T3 , Lambova A4 , Adamson E5 , Kearns I1 1 Takeda UK Ltd, London, UK, 2 Takeda Pharmaceuticals International Co., London, UK, 3 Takeda Pharmaceuticals International AG, London, UK, 4 IQVIA Ltd, Sofia, Bulgaria, 5 IQVIA Ltd, LONDON, UK
OBJECTIVES : Myelodysplastic syndromes (MDS) and AML are rare blood cancers existing on a continuum, with poor clinical outcomes. This retrospective cohort study obtained real-world data on rates of transformation from MDS to AML, treatment patterns in AML, and survival outcomes for patients with AML in England. METHODS : This study analysed retrospective data from the English national CAS registry and included all adult patients with MDS or AML treated with systemic anti-cancer therapy (SACT) from January 2013 to December 2018, with follow-up to December 2020. Three study cohorts were generated: non-transformed patients with MDS of any risk category (cohort A), patients with AML who transformed from MDS of any risk category (cohort B), and all other patients with de novo and secondary AML (cohort C). Patient characteristics, rates of transformation to AML and SACT treatments received after azacitidine were described; overall survival (OS) was estimated using Kaplan-Meier methods. RESULTS : Cohorts A, B and C included 6,549, 368 and 6,936 patients (total N=13,853), with median age at diagnosis 67, 71, 65 years, respectively. The majority of patients were male (58.54%) and of white ethnicity (87.58%). Transformation rates of patients with MDS of any risk category (total cohorts A and B) were 4.48-11.23% depending on year of diagnosis and available follow-up; median time to transformation was 13 months. Treatment sequences following azacitidine were similar in cohorts B (N=56) and C (N=1,572); the majority of patients did not receive further SACT (66.07%; N=37 and 68.58%; N=1,078, respectively). Median OS was 2.7 months from AML transformation for cohort B and 16 months from SACT initiation for cohort C. CONCLUSIONS : This nationwide study highlights the poor survival for patients with AML in England, particularly for patients who have transformed from MDS to AML. Treatment options are limited, with significant unmet need for new effective therapies.
P73: Risk of Hospitalization and Emergency Room Visits Among Community Oncology Patients
4:00PM - 4:15PM
Namasivayam G 1 , Rahman MM2 , Mohammad N3 , Chang B3 , Karhade M3 , Robert N3 , Wu N4 , Heller B5 , Hoang S6 , Alwardt S3 , Neubauer M3 , Staggs S4 , Moore L7 , Smith H3 1 Ontada, Livermore, USA, 2 Ontada, magnolia, USA, 3 Ontada, The Woodlands, TX, USA, 4 US Oncology Network, The Woodlands, TX, USA, 5 Southern Cancer Center, Mobile, AL, USA, 6 US Oncology, Austin, TX, USA, 7 Ontada, Shaker Heights, OH, USA
OBJECTIVES : Machine Learning (ML) solutions can be used to bring insights to providers at the point of care. This research aimed to establish an explainable ML model to predict patient risk of emergency room (ER) visits and hospitalizations within 30 days after an oncology practice visit. METHODS : A retrospective cohort of 98,686 patients within McKesson Specialty Health | The US Oncology Network’s iKnowMed electronic health records (EHR) system between 05/01/16 and 06/30/20 was identified for research purposes. Inclusion criteria included Oncology Care Model patients aged > 65 years and on cancer treatment. Approximately 300 clinical metrics from the EHR system were considered including diagnosis, staging, labs, vitals, treatments, comorbidities, drugs, and performance. Data were split into training (90%) and testing (10%). Model was selected using performance metrics (AUC, sensitivity, specificity, and accuracy). Model interpretations were reviewed by a team of oncologists. RESULTS : The extreme gradient boosting model (XGBoost) achieved the following testing results: AUC 72%, balanced accuracy 66%, sensitivity 65%, and specificity 67%. At threshold 0.5. The model correctly identified 65% of hospitalized and 67% of non-hospitalized patients. The top 5 attributes in order of SHapley Additive exPlanations (SHAP) impact values included albumin value (0.18), hemoglobin value (0.14), pain score (0.12), days since first chemotherapy (0.09) and lymphocytes (0.08). SHAP plots illustrate all associations between features and 30-day hospital admissions and ER risk. CONCLUSIONS : Real-world data were harnessed and applied in an ML approach to establish a high-performing patient ER visit and hospitalization prediction model. The next phase will include model deployment to several US Oncology Network practices to validate effectiveness in the real world. Results will inform providers when a patient is at risk for an ER visit or hospitalization with the aim of improving the overall quality of oncology care and reducing admissions and ER visits.
P76: Federated Analysis of Multi-Centric Real-World Data: A Feasibility Study
4:45PM - 5:00PM
Andreux M , Schwarz S, Blum M Owkin, Paris, France
OBJECTIVES: Real-World Data (RWD) brings evidence about potential benefits and risks of a medical product that complements evidence generated during clinical trials. An important aspect of RWD is to incorporate data from multiple cohorts so as to minimize bias. However, data sharing in a central repository is not always possible due to data regulations and operational hurdles, thereby impeding the adoption of Real-World Evidence. Federated analysis (FA) is a novel approach to execute analyses on non co-located datasets, where data analysis is executed locally to preserve privacy and ensure compliance. In this work, we investigate the potential of FA to perform statistical analysis over large, decentralized datasets. The main purpose of the study was to evaluate if common statistical operations can be performed in a federated fashion. METHODS: We performed a proof-of-concept federated analysis implementation of several common statistical analysis methods on the public cancer TCGA dataset, split in three centers to mimic a decentralized setting. We quantitatively tested the difference between results obtained with a Federated Analysis and with a standard pooled implementation. We also qualitatively assessed the difficulty to perform an FA analysis. RESULTS: Our findings indicate that it is possible to perform federated analysis for descriptive statistics (mean, variance), survival analysis (Kaplan-Meier curves and Cox Proportional Hazards Model), and hypothesis testing (log-rank test) that provides results equivalent to a pooled implementation. Furthermore, the implementation is qualitatively simple for experienced data scientists. CONCLUSIONS: FA holds the potential to perform RWD analysis in a private and compliant fashion over decentralized data. This is especially relevant for rare diseases where local data scarcity is commonplace. Future works will investigate the evolution of the gap between FA and pooled methods when additional privacy-preserving techniques, such as differential privacy, are used.
P75: Use of Electronic Health Records to Understand the Disease Progression in Lupus Nephritis (LN)
4:30PM - 4:45PM
Dhangar I , Mazumder D, Singh D Optum Global Solutions, Noida, UP, India
OBJECTIVES: LN, a common manifestations of Lupus, often progresses to Chronic Kidney Disease (CKD) or End Stage Renal disease (ESRD) within 5 years of LN diagnosis. Early identification and monitoring could possibly delay the progression to CKD/ESRD in LN patients. The objective is to evaluate disease progression among LN patients in the real-world.
METHODS: Patients with ≥2 records for lupus (ICD10) between 01/01/2016 and 31/12/2020 were identified using Optum de-identified electronic health records. The first record for lupus was set as index. Patients with continuous activity 6 months pre and 24 months post-index, with no lupus diagnosis during the pre-index were included. Patients were followed up to 24 months to assess evidence of LN (second index; based on ICD codes for proteinuria, nephritis, glomerular diseases, and renal failure). Patients were further followed for 12 months from LN diagnosis date to assess the progression to CKD/ESRD. We also evaluated the use of guideline recommended therapies and progression among the subset of patients with LN.
RESULTS: A total of 37,163 patients with lupus were identified after applying the study selection criterion. Nearly 8% of patients presented with LN at the time of lupus diagnosis. Approximately, 14% and 19% of patients developed LN in the first 12 months and 24 months of lupus diagnosis, respectively. Among patients who developed LN, nearly 41% progressed to CKD/ESRD within a year of diagnosis irrespective of their treatment status. Approximately, 60% patients progressed to CKD/ESRD during the 12 months follow-up period even after receiving the guideline recommended therapy.
CONCLUSIONS: Our analysis suggests that lupus patients may develop LN as early as 1 year of diagnosis, and subsequently to CKD/ESRD in a year. Well designed studies using EHR may provide greater insights into the factors associated with early progression, which will help design population health initiatives targeting early detection and intervention.
Methods for Decision Modelling and Economic Evaluation
Live
This session includes studies that address challenges in economic evaluation in an innovative way or offer possible examples of good practice that will be of interest to analysts working in diverse fields. Studies address estimation of cost parameters (for cardiovascular disease), discrete event simulation, impact of changing price over the lifecycle of the product, and fiscal health modelling.
Moderator
David Epstein, PhD
University of Granada, Granada, Spain
DAVID EPSTEIN is associate professor at the Department of Applied Economics, University of Granada, Spain. He has an MSc from University of York and a PhD from the University of Granada. His research interests include health technology appraisal, meta-analysis and cost-effectiveness analysis. David Epstein has participated in major clinical trials and other projects at national and international level. He is associate editor of Value in Health and Gaceta Sanitaria, co-coordinator of the Economic Evaluation group of the Spanish Health Economists’ Association, and member of the ISPOR, the Chartered Institute of Management Accountants and Royal Statistical Society.
P54: Cost-Effectiveness Analysis of Pharmacokinetic-Guided Prophylaxis versus Standard Prophylaxis in Adult Patients with Severe Hemophilia Α in China
4:15PM - 4:30PM
Gu C 1 , Jiao Z2 , Huang H1 , Han Y3 1 Takeda China, Beijing, China, 2 Shanghai Chest Hospital, Shanghai Jiao Tong University, Shanghai, China, 3 Sun Yat-sen University, Guangzhou, China
OBJECTIVES: The objective of the study was to assess the cost-effectiveness of pharmacokinetic (PK)-guided prophylaxis with recombinant human coagulation factor VIII (FVIII) against standard prophylaxis in Chinese adult patients with severe hemophilia A . METHODS: A Discrete Event Simulation (DES) model was developed to evaluate the cost-effectiveness of PK-guided individualized prophylaxis versus standard prophylaxis from the healthcare system perspective which simulated 10,000 patients over a 1-year time horizon. Standard prophylaxis FVIII dose was 30 IU/kg by intravenous injection. PK-guided prophylaxis dosage was adjusted for each patient to maintain the FVIII trough level at 1-5 IU/dL to reduce the risk of bleeding. Dosing interval for both approaches was set to be 48h. Population characteristics, clinical data and utilities were collected from published literature and expert survey. The health outcomes included Annual Joint Bleed Rate (AJBR) and Quality-Adjusted Life Years (QALYs). Model considered the prophylaxis drug costs and bleeding treatment costs in the evaluation. Incremental Cost Effectiveness Ratio (ICER) was estimated to support decision. Probabilistic sensitivity analysis was automatically incorporated throughout DES. RESULTS: A total of 94.3% of patients receiving PK-guided individualized prophylaxis achieved the goal of maintaining the trough concentration at a level of 1 to 5 IU/dL compared with 62.7% on standard prophylaxis. AJBR in PK-guided and standard prophylaxis were 1.527 vs 1.601 and QALYs gained were 0.8384 vs 0.8383 respectively. The average FVIII dose for prophylaxis in PK-guided prophylaxis was 28 IU/kg. Prophylaxis drug costs and bleeding treatment costs in PK-guided prophylaxis (RMB 999,034; RMB 30,557) were both lower than those in standard prophylaxis (RMB 1,072,828; RMB 31,948). A total average savings of RMB 75,185 was obtained by the PK-guided approach. CONCLUSIONS: PK-guided individualized prophylaxis is a dominant treatment compared with standard prophylaxis for adult patients with severe hemophilia A in China, with slightly higher QALYs but lower total costs.
P55: Excess Annual Hospital Costs Due to Cardiovascular Events in a Contemporary UK Population to Inform Health Technology Assessments
4:30PM - 4:45PM
Zhou J 1 , Wu R2 , Williams C1 , Mihaylova B1 1 University of Oxford, Oxford, UK, 2 Queen Mary University of London, LONDON, LON, UK
OBJECTIVES : Impacts of cardiovascular disease (CVD) events on hospital cost in contemporary populations are required to support health technology assessments. We developed models of annual hospital costs associated with a range of events of interest in CVD using the individual participant data from UK Biobank. METHODS : All hospital admissions of the 502,493 participants in UK Biobank were costed using the UK Healthcare Resource Groups reference costs (2019 UK£). Separate annual hospital costs models were indicated for participants with (n= 444,177) and without history of CVD (n= 57,556). We compared one- and two-part generalized linear regression models (GLMs) (part 1: logistic regression for probability of incurring cost, part 2- GLM with Gaussian, Poisson or Gamma distributions using identity, log or squared root links for costs, conditional on incurring any), adjusting for participants’ characteristics at entry (socio-demographic, clinical, prior diseases) and time-updated adverse events (myocardial infarction, stroke, coronary revascularization, cancer, diabetes, vascular death and non-vascular death) (p-value <0.01 in stepwise covariate selection). Standard errors were adjusted for clustering of annual periods by participant. Selected models were used to estimate the events’ impacts on annual hospital costs. RESULTS : For both participants with or without prior CVD, the two-part model with gamma distribution and identity link provided the best fit in modelling annual hospital costs. Overall, adverse events were associated with higher cost in participant with prior CVD than without prior CVD. In both populations, the highest excess costs were associated with years of incident cancer [mean and 95% confidence intervals for participants without and with prior CVD: 6095(5999-6191), 6232(5962, 6502)], coronary revascularization [6077(5920, 6234), 6693(6464, 6922)], stroke [5366(5129, 5603), 5856(5478,6234)], and non-vascular death [5317(5117, 5517), 6056(5652, 6460)]. CONCLUSIONS : These models could inform costs in health technology assessments in cardiovascular disease, such as cost-effectiveness analysis of interventions to reduce cardiovascular disease risks.
P53: The Early Lessons of COVID-19: The Need for a Broader Health-Economic Perspective
4:00PM - 4:15PM
Schöttler M1 , van der Schans S 2 , Postma M3 , Boersma C4 1 Health-Ecore B.V., Amersfoort, UT, Netherlands, 2 University Medical Center Groningen, University of Groningen, Groningen, GR, Netherlands, 3 University of Groningen, Groningen, Netherlands, 4 University Medical Center Groningen, University of Groningen, Groningen, the Netherlands; Department of Management Sciences, Open University, Heerlen, the Netherlands; Health-Ecore, Zeist, UT, Netherlands
OBJECTIVES: The current COVID-19 pandemic caused ~20,000 deaths and ~50,000 hospital admissions in the Netherlands. Efforts to manage this communicable disease and its impact on the health-care system without prior development of specific vaccines have put a strain on the fiscal budget. This study aims to indicatively quantify the impact of COVID-19 on the Dutch government’s fiscal position, simultaneously indicating the value of preventive vaccines from a payer perspective. METHODS: Dutch COVID-19 specific population data on laboratory-confirmed infections, hospital admissions and mortality, was collected from the domestic start of the COVID-19 pandemic on 27 February 2020 until the first administered vaccine on 6 January 2021. A fiscal health modelling approach was used to estimate the loss in tax revenues. Occurred productivity losses were added as an indicator for the future burden on the social security system. Tax revenue losses were caused by premature mortality, whereas the productivity losses occurred through mortality as well as morbidity. Outcomes were expressed in total monetary impact (€, 2020). RESULTS: The impact of the pandemic in the analysed time-period was estimated to amount to a total of €920.7 million. Tax loss due to premature mortality amounted to €58.8 million with 50% attributed to patients >60 years. Productivity loss due to morbidity summed up to €862 million with 46% due to patients 40-59 years. CONCLUSIONS: The fiscal impact of the current pandemic highlights the importance of a broader approach to health-economic analysis. A fiscal health framework, optimally linked to a disease simulation model, is a better instrument to inform decision-making in the context of communicable diseases. The reported fiscal estimates also highlight the benefit of investments in communicable disease prevention such as anticipative development of vaccines. In the decision-making process around pandemic preparedness measures, investment funding and real-options can consequently be informed by a fiscal health framework.
P56: Early Health Technology Assessment to Guide Introduction of Novel Technology into the Market: The Vostars System Case Study
4:45PM - 5:00PM
Lorenzoni V 1 , Trieste L2 , Turchetti G1 1 Institute of Management, Scuola Superiore Sant’Anna, Pisa, Italy, Pisa, Italy, 2 Scuola Superiore Sant'Anna, Pisa, Italy
OBJECTIVES : The aim of the study is to show potential approaches and usefulness of early Health Technology Assessment exemplifying the case study of the Video and Optical See-Through Augmented Reality Surgical System (VOSTARS), an advanced navigation tool for non-endoscopic surgeries, recently developed, also thanks to funding from H2020 programme. METHODS : Surgeons in potential fields of application were surveyed about current practice, potential application of the system, perceived usefulness, intention to use, organizational issues, etc.. Those data, data from in vitro and in vivo tests and inputs from literature were used to inform early evaluation of different HTA dimensions comprising also cost-effectiveness (CEA) and budget impact analysis (BIA). RESULTS : A total of 99 surgeons were surveyed. About the possibility to use the VOSTARS system, 39 surgeons (41%) declared they will absolutely or probably use it (45%). Positive feedbacks were also obtained for both the potential usefulness and the possibility of the system to improve performance of work (about 80% of surgeons). High degree of variability emerged about the opportunity to enhance job effectiveness and improve operating time, anyway surgeons offered insight into potential limits and benefits of the system, related to the possibility of personalized approach. Surgeons also informed the identification of specific surgical fields and interventions for which the system could allow satisfy unmet need (ie, neurosurgery and maxillofacial surgery). Although variability of results, CEA showed potential savings for the Italian National Health System (INHS) in both neurosurgery and maxillofacial surgery, when compared to freehand approach. The BIA highlighted 3-year savings of -1,269€ for a local HS performing about 50 procedures per year. CONCLUSIONS : Despite the evaluation underlined high degree of uncertainty about the economic implications, end users involvement helped highlighting technical issues to be solved, offered insight to better target the technology, assess fact related to acceptability and also design the potential market
Methodological Developments in Network Meta-Analysis and Comparative Effectiveness Research
Live
This session explores advances in evidence synthesis including a range of topical issues: managing randomised and non-randomised designs, single arm studies, unanchored indirect comparisons and appropriate methods for analysing survival endpoints.
Moderator
Matthijs Versteegh, PhD
Erasmus University Rotterdam, Rotterdam, Netherlands
Matthijs Versteegh, PhD, is director at the institute for Medical Technology Assessment (iMTA) of Erasmus University Rotterdam
P42: Comparison of Estimation Methods for Single-Arm Trials in Rare Diseases with Historical Control Groups
4:15PM - 4:30PM
Barlev A 1 , Brookhart MA2 , Xun P3 , Thirumalai D3 , Sadetsky N1 , Suissa S4 1 Atara Biotherapeutics, South San Francisco, CA, USA, 2 Duke University, Durham, NC, USA, 3 Atara Biotherapeutics, Thousand Oaks, CA, USA, 4 McGill University and Jewish General Hospital, Montreal, QC, Canada
OBJECTIVES : Randomized controlled trials are the gold standard for estimating treatment effects. However, in rare diseases with high unmet need, single-arm trials are used when randomization of patients to placebo or standard of care is infeasible or unethical. We evaluated various methods to control for confounding in estimating treatment effects in a small single-arm trial with a historical comparator group. METHODS : We used simulation to evaluate different techniques to estimate the “true” treatment effect on overall survival (OS) and objective response rate (ORR) in a specific target population using an external comparator design. We varied effect size, sample size, confounders, and correlation between confounders. We assessed two broad categories of methods: i) requiring specification for treatment allocation [propensity score (PS)-based inverse probability of treatment weighting (IPTW), standardized mortality/morbidity ratio (SMR), stabilized IPTW (SIPTW), overlap weighting (OW), stratification, and matching], and ii) adding outcome information (g-computation). Their precisions and accuracies were evaluated by a combination of 95% confidence interval (CI) coverage, power, bias, and mean square error (MSE). RESULTS : G-computation resulted in the most accurate and precise estimator of OS (95% CI coverage: 93.5%, power: 69.3%, bias: -0.001, MSE: 0.055) in a small sample size scenario of 30 treated subjects compared with 120 comparator subjects. Similar results were observed for ORR. In comparison, results for OS were: 95% CI coverage: 72.8%, 65.6%; power: 75.9%, 62.6%; bias: -0.026, 0.072; MSE: 0.114, 0.167 for SMR and IPTW, respectively. CONCLUSIONS : In our simulated example, the g-computation estimator performed best to control confounding in a small single-arm trial with an external comparator group. PS based methods (e.g., SMR & IPTW) may be suitable as an initial step in the creation of the comparator arm when researchers are blind to the outcome, while g-computation can be subsequently used to estimate the efficacy of treatment.
P41: Crosnma: A New R Package to Synthesize Cross-Design Evidence and Cross-Format Data
4:00PM - 4:15PM
Hamza T , Salanti G University of Bern, Bern, BE, Switzerland
OBJECTIVES: In network meta-analysis, we synthesize all relevant available evidence about health outcomes from competing treatments. That evidence might come from different study designs and in different formats: from non-randomized studies (NRS) or randomized controlled trials (RCT) as individual participant data (IPD) or as aggregate data (AD). To utilize all available evidence, we need a software that allows us to combine these different pieces of information accounting for their differences, e.g. RCTs have typically lower risk of bias than NRS. METHODS: We integrate the three-level hierarchical model that combine IPD and AD with the following four models that incorporate both RCT and NRS evidence by (a) ignoring their differences in risk of bias (b) using NRS to construct discounted treatment effect priors (c,d) adjusting for the risk of bias in each study and controlling the contribution of high risk of bias information in two different ways. RESULTS: We have implemented these models in a new R package, crosnma. This software allows us for conducting Bayesian network meta-analysis and meta-regression. Up to three study- or patient-level covariates can be also included, which may help explaining some of the heterogeneity and inconsistency across trials. The package runs a range of models with JAGS by generating the code automatically from user’s input. CONCLUSIONS: crosnma is a new R package to conduct Bayesian network meta-analysis and meta-regression to synthesise cross-design evidence and cross-format data. We believe that this package will encourage the investigators to not discard any relevant evidence on their analysis. Authors are supported by the HTx-project. The HTx project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement Nº 825162
P43: A Case Study and Simulation to Compare Different Indirect Treatment Comparison Methods Under Varying Access to Individual Patient Data
4:30PM - 4:45PM
Ayers D1 , Cope S1 , Phillippo DM2 , Jansen JP3 , Park J 1 , Yuan Y4 1 PRECISIONheor, Vancouver, BC, Canada, 2 University of Bristol, Bristol, BST, UK, 3 PRECISIONheor, San Anselmo, CA, USA, 4 Bristol-Myers Squibb, Princeton, NJ, USA
OBJECTIVES: To compare alternative methods for unanchored indirect comparisons of two interventions for overall survival when individual patient data (IPD) is available for both studies (IPD-IPD analyses), and when IPD is only available for one study and aggregate data (AD) for the other study (IPD-AD analyses). METHODS: A case study comparing nivolumab with standard of care (SoC) in 3L small cell lung cancer was performed using the CheckMate032 trial and the Flatiron Health database to compare unanchored comparisons without population adjustment, IPD-IPD adjustment analyses using inverse probability treatment weighting (IPTW), regression adjustment (RA), and doubly robust methods (DRMIPD-IPD ), and IPD-AD analyses using matching-adjusted indirect comparison (MAIC), simulated treatment comparison (STC), and a novel DRMIPD-AD . Relative treatment effects were expressed with Cox hazard ratios (HRs) and differences in restricted mean survival time (DRMST). A simulation study was performed evaluating the performance of the alternative methods in different scenarios. RESULTS: Conditional HRs from RA differed from the marginal HRs from IPTW due to non-collapsibility. Therefore, estimates of DRMST in months were used as the relative treatment effect measure for the case study, which provides a collapsible estimand for DR estimates and comparison between methods. DRMST with nivolumab versus SoC was higher for RA (3.22) than IPTW (2.72) and DRMIPD-IPD (2.71). MAIC, STC, and DRMIPD-AD estimates were similar (2.88; 2.92; 2.88) but with more uncertainty. The simulations found that when covariates differed substantially between studies, IPTW performed poorly in comparison to RA and DRM. CONCLUSIONS: Analysts should be aware of differences between marginal and conditional treatment effects when performing indirect comparisons of time-to-event outcomes; DRMST may be considered. Regression-based or DRM models may be preferable over other adjustment methods in cases with limited overlap in covariates between studies indirectly compared. Having IPD for both studies is preferable over having IPD for only one.
P44: Novel and Existing Flexible Survival Methods for Network Meta-Analyses
4:45PM - 5:00PM
Heeg B 1 , Garcia A2 , van Beekhuizen S3 , Verhoek A4 , Roychoudhury S5 , Cappelleri J6 , Postma M7 , Ouwens M8 1 Ingress-health, Rotterdam, Netherlands, 2 Ingress-Health, rotterdam, Netherlands, 3 Ingress-Health, Rotterdam, Netherlands, 4 Ingress-health, Rotterdam, ZH, Netherlands, 5 Pfizer, Inc., Gladstone, NY, USA, 6 Pfizer Inc, Newington, CT, USA, 7 University of Groningen, Groningen, Netherlands, 8 AstraZeneca, Gothenburg, Sweden
OBJECTIVES : The NICE Decision Support Unit has recently published technical support document 21, focusing on flexible survival methods and considering excess mortality. In this study, we research these flexible survival methods by assessing the effect of treatment on the different parameters of the distributions and the implementation of these flexible excess survival models in network meta-analyses (NMA). METHODS : Standard parametric, mixture- (MCM) and non-mixture-cure (nMCM), piecewise, and splines are used for comparison. For the base case model, selection treatment coefficients were assigned to all parameters. The lowest leave-one-out information criterion (LOOIC) defined the base case parametric distribution per flexible model. The best-fitting distribution per model was rerun with treatment coefficients only for those larger than their standard deviation, and the one with the lowest LOOIC was considered the base case. Models were compared based on incremental mean survival and uncertainty. We used a network of four trials in previously treated advanced non-small cell lung cancer, comparing nivolumab vs. docetaxel, pembrolizumab vs. docetaxel, and two trials comparing atezolizumab vs. docetaxel. RESULTS : The base case parametric distributions were log-logistic for standard parametric distribution, log-logistic for MCM, lognormal for nMCM, Weibull for mixture, log-logistic for piecewise, and log-logistic for spline models, with corresponding LOOICs 13418, 13417, 13420, 13417, 13418, and 13419. Regarding treatment effect parametrization, we applied no treatment effect on cure, for instance, but treatment effects on the scale for MCM and nMCMs. For piecewise, treatment effects were only applied on the second piece. After reducing the number of treatment effects, the mixture Weibull had the lowest LOOIC with 13407. The mean incremental survival with docetaxel as reference for mixture Weibull was 3.58[95%CI 1.2,5.26], 0.45[0.19,0.93] and 2.67[0.2,9.48] for nivolumab, pembrolizumab and atezolizumab, respectively. For standard log-logistic these were 1.67[0.94,2.55],1.01[0.55,1.61], and 0.43[0.17,0.77]. CONCLUSIONS : Treatment effect specification is important as outcomes and uncertainty differ over the tested models.
Using Real World Evidence in Simulation Models
Live
This session explores how Real World Evidence can be used in simulation models to inform policy decisions. The session includes case studies of using Real World Evidence in policy models in cardiovascular disease, oncology and to predict the emergence of multi-drug resistant bacterial infections. The session also explores how simulation models that directly rely on IPD can be used whilst preserving subjects data privacy.
Moderator
Beth Woods, MSc
University of York, York, United Kingdom
Beth is a Senior Research Fellow at the Centre for Health Economics (CHE), University of York. Prior to joining CHE Beth was a Director in the Health Economics team at Oxford Outcomes, a private consulting firm, where she specialised in applied economic evaluation from 2006-2013. Beth holds a BA in Economics from the University of Cambridge and an MSc in Economic Evaluation in Healthcare from City University. Beth’s research interests include drug pricing, the economic evaluation of interventions to combat AMR and the use of statistical and decision analysis to support reimbursement decision making.
P72: Prediction of the Prevalence of Resistance of Acinetobacter Baumannii to Colistin in the City of Valencia (Spain) with a New Agent-Based Model
4:45PM - 5:00PM
Aledo JA1 , Andreu-Vilarroig C2 , Cortes JC2 , Orengo J 3 , Villanueva RJ2 1 Universidad de Castilla-La Mancha, Albacete, Spain, 2 Universitat Politècnica de València, Valencia, Spain, 3 MSD (IA) LLC, Guaynabo, Puerto Rico, Coamo, PR, USA
OBJECTIVES: As a consequence of the increased prevalence of the carbapenem-resistant Acinetobacter baumannii , these are classified by the WHO and CDC as an urgent threat. The objectives of the study were: 1) to create an agent-based model; 2) to calibrate the model; 3) to predict the prevalence of resistance of Acinetobacter baumannii to colistin. METHODS: Using the demographic information of the City of Valencia between the years 2012-2020 we built a synthetic population, the population dynamics were obtained from the hospital flow of patients and from the epidemiological information of the periods 2016-2019 and 2015-2018 respectively. We included characteristics of the infection (community, hospital, origin of infection, hospital stay) and different states in which the patient could be found (susceptible, sensitive, resistant). We assumed that every person infected with Acinetobacter baumannii was hospitalized if they were sensitive or resistant to colistin. Among the parameters included in the model were the daily probability of change in the clinical status of new patients and that of hospitalized patients without infection or with nosocomial infection. Using the Particle Swarm Optimization (PSO) algorithm, we carry out the calibration process through simulation and optimization. RESULTS: With the built and calibrated model, we maintained the probability of being admitted daily (18.4%) and of nosocomial infection (2.6%) by Acinetobacter baumannii . We found that the increase in the number of cases resistant to Acinetobacter baumannii to colistin is related to age, the largest increase being in the ages> 60 years, followed by the age group 30 to 59 years and finally the group of <30 years old. CONCLUSIONS: The calibrated model adjusted the estimated prevalence to the real prevalence, which allowed us to make predictions of the increase in cases of Acinetobacter baumannii resistant to colistin by age groups, temporalizing the loss of effectiveness of colistin.
P69: Enabling Data Privacy in Health Economic Microsimulation Models by Using Subject-Level Synthetic Data
4:00PM - 4:15PM
Ivkovic M , Grand T Novo Nordisk A/S, Copenhagen, Denmark
Objectives: Health economic microsimulation models, that directly utilize subject-level data to inform reimbursement decisions, are increasingly replacing the more traditional cohort models that utilize summary data only. Sharing such microsimulation with, for example, HTA bodies requires careful attention to data privacy and security in order to protect personal information. This study aimed to explore the potential of an alternative solution which minimizes privacy risk: synthetic subject-level data, which mimics actual data, but holds no sensitive subject information. Methods: We synthesized key variables from a large phase III trial in people with obesity by fitting a classification and regression tree (CART) model to the observed data and using it to predict a synthetic dataset, sequentially generating the variables. Utilizing the publicly available software package synthpop in R, we attempted to identify the benefits and limitations of synthesizing data with the CART machine learning method. A special focus was on measures of similarity between observed and synthetic data. Results: We compared univariate and multivariate properties of data pre- and post-synthesis. Histograms and summary statistics demonstrated little to no difference in univariate distributions of synthetic vs actual variables, crucially preserving tail behaviour. The multivariate correlation structure of synthetic data greatly resembled that of the original data, to the point visual representations of them were nearly indistinguishable. Furthermore, CART’s properties ensure key subgroups are captured, which resulted in preservation of main subgroup interactions, as well. Conclusions : In this case study, a synthetic dataset with a very high degree of similarity to the actual subject-level data was constructed using machine learning techniques. This suggests that synthetic data may be a promising alternative in health economic microsimulation models to address data privacy and protection concerns. Future work will include validating the use of subject-level synthetic vs observed data in a health economic microsimulation modeling.
P70: Calibrating Cardiovascular Disease Policy Model Using Large Cohort Data
4:15PM - 4:30PM
Wu R 1 , Williams C2 , Schlackow I2 , Zhou J2 , Emberson J2 , Reith C2 , Keech A3 , Robson J1 , Wilkinson K4 , Armitage J2 , Collins R2 , Gray A2 , Simes J3 , Baigent C2 , Mihaylova B2 1 Queen Mary University of London, LONDON, LON, UK, 2 University of Oxford, Oxford, UK, 3 University of Sydney, Sydney, NSW, Australia, 4 Oxfordshire, Oxford, UK
OBJECTIVES : Declining cardiovascular disease (CVD) morbidity and mortality over the last decades suggest that policy models developed using historic data likely overestimate current risks. We illustrate an approach to calibrating a micro-simulation CVD model, developed using historic clinical trials data, using the 2006-2020 UK Biobank cohort. METHODS : A micro-simulation model, developed using individual participant data from the Cholesterol Treatment Trialists’ collaboration (CTT: 118,000 participants), was calibrated in the UK Biobank cohort (UKB: 502,000 participants). Original parametric survival models estimated risks of key endpoints (myocardial infarction, stroke, coronary revascularisation, incident cancer and vascular and nonvascular death) using CTT participants’ sociodemographic and clinical characteristics at entry and incidents of the key endpoints during follow-up (significant at p<0.01). Model calibration using UKB data involved re-fitting the intercept and linear predictors from these equations; excluding/de-novo estimating factors with substantially different effects; and adding factors and endpoint/s to enrich the model. Standard approaches to risk equation modelling and model validation were employed. Both original and calibrated models were used to predict endpoints in UKB. RESULTS : We demonstrate the feasibility of calibrating a detailed decision analytic model using individual participant data. However, it is a data intensive and cumbersome process likely to lead to modified strength of relationships between disease endpoints. The process required all equations to be calibrated with few factors de-novo estimated (e.g. smoking) but enabled new factors (e.g. cancer duration) and endpoint (incident diabetes) to be included. The incidence of endpoints predicted by the calibrated model, unlike in the original model, corresponded well to their observed incidence in the UKB, overall and in categories of participants. CONCLUSIONS : A new calibrated lifetime CVD model accurately predicts morbidity and mortality in contemporary UK populations. It will be made available to provide individualised projections of expected lifetime health outcomes and benefits of treatment.
P71: The Development of a Flexible and Easy to Tailor Disease Model to Estimate the Outcomes of Treatment Sequences in Advanced Melanoma by Combining Trial and Real-World Data
4:30PM - 4:45PM
de Groot S 1 , Blommestein HM2 , Leeneman B2 , Uyl-De Groot C2 , Haanen JBAG3 , Suijkerbuijk KPM4 , Aarts MJB5 , van den Berkmortel FWPJ6 , Blank CU3 , Boers-Sonderen MJ7 , van den Eertwegh AJM8 , de Groot JWB9 , Hospers GAP10 , Kapiteijn E11 , de Meza MM12 , Piersma D13 , van Rijn RS14 , Stevense-den Boer MAM15 , van der Veldt AAM16 , Vreugdenhil G17 , Wouters MWJM3 , Franken M18 , van Baal PHM2 1 Institute for Medical Technology Assessment, Erasmus University Rotterdam, Rotterdam, ZH, Netherlands, 2 Erasmus School of Health Policy and Management, Erasmus University Rotterdam, Rotterdam, Netherlands, 3 Netherlands Cancer Institute, Antoni van Leeuwenhoek, Amsterdam, Netherlands, 4 University Medical Center Utrecht Cancer Center, Utrecht, Netherlands, 5 Maastricht University Medical Center, Maastricht, Netherlands, 6 Zuyderland Medical Center, Sittard-Geleen, Netherlands, 7 Radboud University Medical Center, Nijmegen, Netherlands, 8 Cancer Center Amsterdam, Amsterdam UMC, Amsterdam, Netherlands, 9 Isala, Zwolle, Netherlands, 10 University Medical Center Groningen, Groningen, Netherlands, 11 Leiden University Medical Center, Leiden, Netherlands, 12 Dutch Institute for Clinical Auditing, Leiden, Netherlands, 13 Medisch Spectrum Twente, Enschede, Netherlands, 14 Medical Center Leeuwarden, Leeuwarden, Netherlands, 15 Amphia Hospital, Breda, Netherlands, 16 Erasmus MC Cancer Institute, Rotterdam, Netherlands, 17 Maxima Medical Center, Eindhoven, Netherlands, 18 Institute for Medical Technology Assessment, Erasmus University Rotterdam, Rotterdam, Netherlands
OBJECTIVES: Although in the last decade many novel treatments have been introduced for advanced melanoma (unresectable stage IIIc/V) patients, evidence on the outcomes of treatment sequences is lacking. Our aim was to develop a flexible disease model for advanced melanoma to estimate long-term effectiveness of treatment sequences, to support clinical guideline development and reimbursement decision making. METHODS: A semi-Markov model with a life-time horizon was developed. Transitions describing disease progression, next treatment and mortality were estimated from real-world data, as a function of time since start of treatment or disease progression, and patient characteristics. All transitions can be adjusted based on the relative effectiveness of treatments that were derived from a network meta-analysis. Additionally, the duration of treatment effect and time horizon can be changed to demonstrate outcomes under different assumptions. RESULTS: The model distinguishes three active treatment lines for BRAF mutant melanoma and two active treatment lines for BRAF wild-type melanoma. Depending on treatment, mean life expectancy of BRAF mutant melanoma patients with a poor, intermediate or favourable prognosis ranged from 2.2 to 3.4, 5.6 to 7.0 and 9.9 to 11.3, respectively. Mean life expectancy of BRAF wild-type melanoma patients with a poor, intermediate or favourable prognosis ranged from 4.3 to 5.8, 5.8 to 8.2 and 7.8 to 9.7, respectively. The scenario-analyses illustrate how estimates of life expectancy crucially depend on duration of treatment effect and time horizon. CONCLUSIONS: Our model is flexible and can be tailored to answer questions concerning (cost-)effectiveness. Treatments and sequences of treatments can be adjusted, as well as the duration of treatment effects and the transitions influenced by treatment effect. With this model, we show how to combine real-world data with data from clinical trials to benefit most from the advantages of both data sources, which can guide the development of future disease models.
Issues with Health Technology Assessment of Specialised Technologies
Live
This session reviews the experiences, outcomes, and consequences of HTA agencies' evaluations of specialised, innovative technologies such as gene and cell therapies and new cancer drugs. The authors discuss how HTA agencies have evaluated manufacturers' submissions, in particular their critical appraisal of assumptions about long term benefits and claims about whether therapies offer a cure. Authors reflect on how predictions of the uptake of innovative technologies compare with real world uptake, and report large discrepancies which may indicate inaccuracy or uncertainty in evaluation methods, and/or highlight barriers to adoption. The cost-effectiveness of screening strategies for inherited risk factors are also discussed.
Moderator
Thor-Henrik Brodtkorb, PhD
RTI Solutions, Ljungskile, O, Sweden
Thor-Henrik Brodtkorb, PhD, is senior director in Health Economics at RTI Health Solutions (RTI-HS). He holds a PhD in Health Technology Assessment from the University of Linköping and has been with RTI-HS for 7 years. He has been teaching courses in decision-analytic modeling at Linköping University as well as presented workshops and short courses on decision-analytic modeling techniques for organizations such as Pharma Industry Sweden, Swedish Agency for Health Technology Assessment and Assessment of Social Services (SBU), and the International Society for Pharmacoeconomics and Outcomes Research (ISPOR).
At RTI-HS, Dr. Brodtkorb leads development of cost-effectiveness, cost-utility, cost-consequence, and budget-impact models for pharmaceutical, device, and diagnostic technologies. These models have been used to support reimbursement decisions in more than 15 European countries including NICE in UK, SMC in Scotland, TLV in Sweden, and NOMA in Norway. He has developed models and analyses in the areas of oncology, alcohol dependence, major depressive disorder, Alzheimer’s disease, dermatology, multiple sclerosis, cardiology, orthopedics, and asthma. His research has been presented at professional conferences and published in peer-reviewed journals. He is also a coauthor of the newly published book Budget-Impact Analysis of Health Care Interventions: A Practical Guide.
P39: Better Outcomes and Value for Money with Cost-Effectiveness Modelling of Cascade Screening Strategies for Familial Hypercholesterolaemia
4:30PM - 4:45PM
Faria R 1 , Cox E1 , Saramago P2 , Haralambos K3 , Watson M4 , Humphries SE5 , Qureshi N6 , Woods B1 1 University of York, York, UK, 2 University of York, Heslington, York, UK, 3 University Hospital of Wales, Cardiff, UK, 4 , University Hospital Southampton NHS Foundation Trust, Southampton, UK, 5 University College London, London, UK, 6 University of Nottingham, Nottingham, UK
OBJECTIVES: Cascade screening for familial hypercholesterolaemia (FH) refers to the systematic testing of relatives of people known to have FH (termed ‘indexes’). Cascade screening is cost-effective compared to no screening, but alternative screening strategies have not been studied. Our objective was to identify the cost-effective strategy to select indexes to cascade, and to contacting and testing their relatives. METHODS: We developed a new cost-effectiveness model informed with routinely collected UK data from indexes and their relatives. Our decision tree model takes the UK National Health Service perspective and calculates, per index assessed to the cascade, the relatives diagnosed, cascade costs, quality-adjusted life years (QALYs), healthcare costs and incremental cost-effectiveness ratios (ICERs). RESULTS: We compared 1792 strategies. The cost-effectiveness frontier was mostly formed by strategies which contacted 1st and 2nd degree relatives of indexes with genetic FH simultaneously and directly. The cost-effective strategy diagnoses relatives according to whether they were on lipid lowering treatment, cholesterol, and age, with some having confirmatory genetic testing – it diagnoses 52% affected relatives, at a cascade cost of £536; ICER = £13,996/QALY. Sequential contact (i.e. contacting second degree relatives only when their first degree relative was diagnosed with FH), indirect contact via their index/relatives and genetically testing them diagnoses 36% of relatives, while direct and simultaneous contact with genetic testing diagnoses more relatives (56%); neither are in the cost-effectiveness frontier. If genetic testing is not available, cascade screening remains cost-effective, diagnosing 41% of relatives (ICER=£5,603 vs no cascade). Results are robust to alternative scenarios bar those affecting long-term benefits of diagnosis. CONCLUSIONS: Simultaneous and direct contact of relatives of indexes with genetic FH and a mixed approach to testing relatives is cost-effective and achieves better outcomes than sequential and indirect contact. Identifying this strategy required systematic comparison of multiple alternatives, which is only achievable with cost-effectiveness modelling.
P37: The Cure Myth: Experience from Recent Health Technology Assessment (HTA) Submissions in Relapsed/Refractory Diffuse Large B-Cell Lymphoma (R/R DLBCL)
4:00PM - 4:15PM
Mohseninejad L 1 , Hardy A2 , Westley T3 , Kongnakorn T1 1 Evidera, London, UK, 2 Evidera, Paris, France, 3 Evidera, Montreal, QC, Canada
OBJECTIVES: Recent clinical trials testing novel cancer therapies such as CAR-Ts are detecting a plateau in the survival curves, conveying a potential opportunity for cure. Long-term survival data are limited, and clinicians are often cautious to make conclusions on the basis of the trial outcomes. Nevertheless, cure assumptions have appeared in oncology HTA submissions such as those in R/R DLBCL. This study aims to investigate how cure assumptions are applied across different HTA submissions for therapies in R/R DLBCL. METHODS : Publicly available HTA dossiers in R/R DLBCL submitted to NICE, SMC, CADTH, ICER, PBAC and HAS in the past five years were reviewed. Information related to cure assumptions (methodology, cure time point, cure proportion) and the critiques received by the HTA bodies were reviewed and extracted. RESULTS : Of the twelve HTA documents identified, ten submissions applied cure assumption in overall survival extrapolation. These submissions focused on two CAR-T agents (axicabtagene and tisagenlecleucel) and polatuzumab vedotin. From the ten submissions that reported cure assumptions, six used cure-mixture models and four used simple cure assumptions. Eight models set mortality of cured patients to that of the general population, while two considered an adjustment factor. Cure was considered only for the intervention arm in seven models, while the rest considered it for all treatments. The timepoint and proportion of cure varied across the submissions. Critiques were mainly around the issues caused by immaturity of survival data, such as uncertainty in cure assumptions and the choice of cure timepoint. CONCLUSIONS : While cure modelling is frequently used in R/R DLBCL and in oncology, there seems to be no consensus on the implication of cure and its implementation in the economic analyses. Clear guidance must be provided by health authorities on how to approach the cure concept in economic modelling and how to validate the assumptions.
P40: HTA Agencies Perspective on Survival Modelling in Cell or Gene Therapy Appraisals
4:45PM - 5:00PM
Lissdaniels J 1 , Medin E2 1 Parexel International, Vällingby, Sweden, 2 Parexel International, Stockholm, Sweden
OBJECTIVES: The first cell- and gene therapies, so called advanced therapy medicinal products (ATMPs), have recently become available, making it possible to treat, and even potentially cure, very severe and sometimes previously untreatable conditions. These characteristics have led to a discussion among health economists about whether a specific methodological reference case is required for economic evaluation of gene therapies and the conclusion has been that a new methodological reference case is not required but that “the confluence of various characteristics can lead to specific methodological challenges...”. Traditional survival modeling may underestimate outcomes by assuming the same mortality rate for all patients, in situations where the treatment could lead to the cure of a patients. Mixture cure models have been suggested as a supplementary analysis, alongside standard parametric models. The aim of this study was to review reimbursement appraisal reports of the 12 EMA approved ATMPs, to identify the differences in methods and assumptions in survival modelling of long-term treatment effects across different HTA agencies. METHODS: Publicly available assessment reports were retrieved from each reimbursement agency’s website in the Nordics, the Netherlands, England and Wales, Canada and Australia for the relevant drugs. RESULTS: Across the HTA agencies different level of acceptance to non-standard survival modelling is seen. E.g. in appraisals of cell-therapies, mixture cure models have been accepted in the Nordic countries and in England and Wales but assumptions around the percentage of cured patients and the preferred source for the survival extrapolation post clinical trial follow-up differs. In appraisals of gene-therapies, the exploration of the impact of the main assumptions that drive model results have been recommended across the HTA agencies. CONCLUSIONS: There is yet not an established golden standard on how to apply survival modelling to ATPMs and the preferences on the methodology varies across HTA agencies.
P38: Sufficient Uptake of Highly Specialised Technologies?
4:15PM - 4:30PM
Dacosta RFD1 , Wang GD2 , Macaulay R 3 1 PrecisionADVISORS, London, UK, 2 PrecisionAdvisors, London, UK, 3 Precision Advisors, London, UK
The National Institute for Health and Care Excellence (NICE) introduced a Highly Specialised Technology (HST) pathway in 2015 to provide more consistency in appraising high cost therapies for rare and neglected diseases. This research evaluates the number of HSTs appraised to date and the actual number of patients treated under this pathway. All HST guidance was identified and disease prevalence/incidence, and recommendations were extracted (to 25-Jun-2021). Actual uptake data was provided by NHS England in April 2021. NICE published 14 recommendations through the Highly Specialised Technology (HST) pathway (after an average of 19.6 months after EC-approval) compared to 357 recommendations through the technology appraisal pathway (single or multiple) since 2015. Approximately 1254 patients have been treated with an HST approved therapy. 5 of the 14 HSTs have exceeded the number of patients treated than the NICE-estimated eligible patient population. Notably, 373 patients had been treated with eculizumab (HST1) since January 2015, compared with an estimated 20-30 patients diagnosed-per-year (estimated total uptake of 120-180 from recommendation). However, in some instances the number of patients treated with an HST were very low: zero patients for Strimvelis® (HST7), ≤10 for eliglustat (HST5), and ≤10 for metreleptin (HST14); much lower than any reported estimates in their respective HST appraisals (Strimvelis®: 3 diagnosed/year; eliglustat: 50-100 eligible patients; metreleptin: <200 eligible patients). Relatively few therapies have been recommended under the NICE HST pathway since 2015 compared to the standard NICE process with STA/MTA. Further, these recommendations have been over 19-months from EC-approval. Even following a positive NICE recommendation, several of these have not achieved meaningful patient access, potentially due to the lack of awareness for rare diseases, and likelihood of underdiagnoses.
Tue 30 Nov
11:00 - 12:15
Plenary Session
Joint HTA Dialogue for Specialized Therapies: Tower of Babel or Lingua Franca?
In many cases, clinical evidence for the efficacy of specialized therapies does not come in the form of randomized controlled trials (RCTs). It may not have clinical outcomes that are meaningful to patients or easily valued by payers. Regulatory authorities have established methods for evaluating such products and generally are able to find a pathway for their market authorizations.
Such therapies, however, often have less clear roads to pricing and reimbursement across countries because the health technology assessment (HTA) processes involved can require varying types of evidence when RCT data are not available or sufficient. While the data required for determining value are not always the same as needed for safety and efficacy, is it possible to achieve better agreement on the use of alternative sources of evidence for use in joint clinical assessment for HTA purposes? This session will discuss the usefulness and gaps of regulatory evidence pathways used for HTA purposes and the potential for a more unified approach to use of alternative evidence.
*Speakers to be added as confirmed!
Moderators
Guido Rasi, MD
Rome University Tor Vergata, Gemelli Polyclinic, ROMA, RM, Italy
Guido Rasi is now full professor of Microbiology at Rome University Tor Vergata and Chairman of Clinical Trial Center of Gemelli Polyclinic in Rome.
From March 2021 Advisor to Army General Francesco Paolo Figliuolo, Special Commissioner for the implementation and coordination of measures to contain and combat the COVID-19 epidemiological emergency and for the execution of the vaccination campaign in Italy.
He completed his second term as Executive Director of EMA in November 2020. He has been elected Chair of the International Coalition of Medicines Regulatory Authorities (ICMRA) from 1st October 2019 for a term of 3 years. He was Director General of the Italian Medicines Agency from 2008 to 2011 and member of the Management Board from 2004 and 2008. From 2005 to 2008 he was Director of Research at the Institute of Neurobiology and Molecular Medicine of the National Research Council (CNR) in Rome. From 1990 to 2005 Professor Rasi worked at the Institute for Experimental Medicine of the National Research Council, Italy He had a teaching and research experience at the University of California, Berkeley in 1999. Professor Rasi holds a degree in medicine and surgery, with specializations in internal medicine, allergology and clinical immunology, from the University of
Rome He is the author of numerous scientific publications He has received many awards and one honorary degree.
Speakers
Luciana Ballini, BSc. Soc.; MSc
Azienda USL di Modena, Regione Emilia Romagna, Bologna, Italy
Luciana Ballini is Head of the Regional Centre for Health Technology Assessment programme of Emilia-Romagna (Italy) and Director of the Clinical Governance and HTA Service of the Modena Public Health Trust.
Since 2006, she has been leading Emilia-Romagna’s participation in the EUnetHTA project and three Joint Actions, and she has served twice as Chair of the EUnetHTA Plenary Assembly. She has expertise in Evidence Based Medicine methodology, systematic review and meta-analysis, grading of evidence, identification and prioritization of research gaps and uptake of HTA results in decision making processes.
She is editor of the Cochrane Collaboration Review group named Effective Practice and Organization of Care (EPOC).
Jo De Cock, MS
INAMI - Institut National de l'Assurance Maladie-Invalidité, Brussels, Belgium
Hans-Georg Eichler, MD, MSc
Medical University of Vienna, Wien, Austria
Hans-Georg Eichler, MD, MS, is currently the Consulting Physician of the Association of Austrian Social Insurance Institutions. Before that, he was the Senior Medical Officer of the European Medicines Agency for more than 14 years, and professor and chair of clinical pharmacology at the Medical University of Vienna. He held a range of other full-time and honorary positions in academia, industry and government.
Rui Santos Ivo
INFARMED, Lisbon, Portugal
Rui Santos Ivo is currently President of INFARMED – National Authority of Medicines and Health Products, I.P., since June 2019. Member of the Management Board of the EMA, since March 2016. Member of the Executive Board of EUnetHTA - the European Network for Health Technology Assessment, since June 2016. Vice-chair of the Valletta Permanent Technical Committee/Valletta Declaration, since July 2017.
He is also an Invited Assistant Professor of Medicines Regulation, at the Faculty of Pharmacy of the University of Lisbon, where he is a member of the School Council.
He is an external elected member of the General Council of the University of Coimbra, since 2017.
Previously he was President (2014/2016) and Vice-President (2011/2014) of the Central Administration of the Health System (ACSS,IP) at the Ministry of Health, in Portugal. He was Coordinator of the Hospital Reform Project Team (2012/2015) and chaired the Governance College for the Public Health Subsystems (2015/2016).
R. Santos Ivo initiated his professional career as a hospital pharmacist at Hospital de Egas Moniz, in Lisbon. In 1993 he joined INFARMED, where he held various responsibilities, namely vice-president (1994-2000; 2016-2019) and President (2002-2005).
In 2000 and 2002 he worked as Administrator at the Directorate of the European Medicines Agency (EMA), in London, and in 2006 and 2008 he worked for the European Commission as Administrator at the Pharmaceuticals Unit, Directorate General for Enterprise and Industry, in Brussels.
He was a member of the Management Board of the EMA (2002/2005) and the first Chairman of the European Union Heads of Medicines Agencies Management Group (2004-2005).
In 2008-2011 he was Executive Director of the Portuguese Association of the Pharmaceutical Industry (APIFARMA).
R. Santos Ivo graduated in Pharmaceutical Sciences by the University of Lisbon in 1987. Specialist in Hospital Pharmacy (1992) and Pharmaceutical Regulation by the Portuguese Pharmaceutical Society (1997). Post graduate education on Health Law and Pharmaceutical Legislation (University of Lisbon Faculty of Law and National School of Public Health), Pharmaceutical Medicine (University of Basel), Regulation (London School of Economics and Political Science) and Health Management (Portuguese Catholic University (2000) and AESE (2015)).
Almofariz Prize, Personality of the Year in the Pharmaceutical Sector (2004), European Correspondent Member, Académie Nationale de Pharmacie, France (2014) and Medal (gold) for distinguished Services, Ministry of Health (2015).
11:00 - 17:00
Poster & Exhibit Viewing
Live
Stop in to learn about the latest technology, services, and devices and to connect one-on-one with exhibitors and sponsors during our Exhibit Viewing Hours. Be sure to reserve some time to view the latest research in HEOR through our virtual poster gallery. Browse, comment, and discuss our posters at any time while our virtual platform is active.
12:30 - 13:30
ISPOR Forums
When and How Can Health-Preference Measures Be Transferred Between Contexts?
Quantitative preference data are playing an increasing role in healthcare decision making, including patient preferences in quantitative benefit-risk assessments and public preferences in economic evaluation. However, the cost and time requirements of collecting preference data are barriers when resources are limited, regulatory deadlines are short, and/or the value of preference information is uncertain. Faced with similar problems in assessing environmental-quality values in the 1980s, economists developed principled strategies for adapting existing preference estimates for new applications. Until recently, the small number and varied designs of health preference studies have limited the ability to assess the transferability of preference estimates between contexts. However, as the evidence base has grown, it is now possible to utilize preference-calibration methods to transfer health-preference estimates. To ensure health economists optimize the use of their data, it will be important to develop guidance on when and how health-preference data can be transferred, how studies should be designed and reported with transferability in mind, and how the validity and reliability of transfers can be assessed. To facilitate discussion on this important topic, the forum will be organised into four parts. First, we will identify the state of the art of benefit-transfer methods in environmental economics, and discuss the lessons these hold for health economics, including consideration of the similarities and differences between environmental and health valuation. Second, we will consider the empirical evidence on the transferability of health preferences, including the unique insights offered by EuroQol’s 24 EQ-5D-5L country value sets on the transferability of health state values between geographies, and recent research on patterns in the way different patients make benefit-risk trade-offs. Third, we will present IMI PREFER’s recently developed framework on transferring preference data. Finally, we look forward to finding out what you think via polling and Q&A.
Moderators
Kevin Marsh, PhD
Evidera, London, United Kingdom
Kevin Marsh, PhD, is Vice President at Evidera in London, UK. He specializes in the use of preference data and decision analysis to inform health decisions, including pipeline optimisation, authorisation, reimbursement, and prescription decisions.
Dr Marsh’s research interests include stated and revealed preference methods, decision modelling, and MCDA. He has applied these and other research techniques for a range of organisations, including both regulatory and industry clients. He actively contributes to the methodological development of these techniques. He is currently co-Chairing ISPOR Health Preference SIG and is a member of the ISPOR Task Force on qBRA. He has previously chaired the ISPOR Task Force on the Use of MCDA in Health Care Decision-Making and an ISPOR working group on the use of preference data in Europe.
Speakers
G. Ardine de Wit, PhD
Julius Center for Health Sciences and Primary Care, Utrecht, Netherlands
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.
Nicolas Krucien, PhD
Evidera, London, United Kingdom
Nicolas Krucien, PhD, is lead data analyst for Evidera’s Patient Preferences team within Patient-Centered Research. Dr. Krucien joined Evidera by PPD in August 2019 after leaving his position as Research Fellow at the Health Economics Research Unit of the University of Aberdeen, which he has occupied for six years. He has 15 years of experience in health preference elicitation research. His research interests are mainly related to the modelling of choice-based preference data. Dr. Krucien has been published widely in international peer-reviewed journals, including Thorax, Critical Care Medicine, Pharmacoeconomics, Health Economics and Social Science & Medicine.
Bram Roudijk, PhD
EuroQoL Research Group, Rotterdam, Netherlands
Bram Roudijk works as a scientist at the EuroQol Research Foundation. He completed an MSc degree in Economics and a PhD degree in Health Economics and Outcomes Research at Radboud University Nijmegen, the Netherlands. Bram’s PhD thesis focused on research in the valuation of health related quality of life. Bram’s main research interests are valuing health related quality of life instruments, health preferences and health valuation and differences between health preferences between countries and cultures.
What Competencies are Necessary for HEOR Professionals? An Update on the ISPOR Competency Framework
ISPOR has established a set of competencies for HEOR professionals. The resulting 41 competencies are organized into 13 topic domains that collectively comprise the ISPOR Health Economics and Outcomes Research Competencies Framework. In this session, we will explain the collaborative process used by the ISPOR Institutional Council and Faculty Advisor Council to identify and validate the Framework. We will identify what competencies matter most in six common HEOR job types based on results from survey findings. Finally, we will explain ongoing efforts to systematically define the topical content within each competency, with 2021 efforts focusing on the statistical methods competency.
Moderators
Laura Pizzi, PharmD, MPH, RPh
ISPOR, Lawrenceville, NJ, USA
Dr. Laura Pizzi is a pharmacist and seasoned evaluation scientist who serves as Associate Chief Science Officer for the International Society of Pharmacoeconomics and Outcomes Research (ISPOR) as well as Professor in the Rutgers Ernest Mario School of Pharmacy and Rutgers School of Public Health. For the past 20 years, she has led interdisciplinary teams of outcomes methodologists, statisticians, and clinicians to develop and conduct outcome analyses on pharmacological therapies as well as a variety of non-pharmacological interventions. A particular interest is developing scientific evidence to inform the adoption of interventions to improve the quality of care for older adults. Her primary area of research expertise is in cost effectiveness analyses of public health interventions such as community-based health education and health promotion programs. Along with Dr. Jim Murray, she has co-led the ISPOR Competency Framework workgroup since 2017.
Speakers
Tsung-Ying Lee, BPharm, MClinPharm
University of Maryland School of Pharmacy, Baltimore, MD, USA
Tsung-Ying Lee is a pharmacist and health services researcher. He is currently a PhD candidate in the Department of Pharmaceutical Health Services Research at the University of Maryland School of Pharmacy.
Mr. Lee's research interests are in using real-world data and computational methods to generate high-quality evidence on comparative outcomes and value for health-related decision-makers. His past and current works examined the clinical and economic burden of a disease, the comparative effectiveness and cost-effectiveness of pharmaceutical interventions, as well as individual and contextual factors associated with outcome disparity and treatment effect heterogeneity.
James Murray, PhD
Eli Lilly and Company, Indianapolis, IN, USA
Jim Murray, Ph.D. is a Research Fellow within Lilly’s Global Patient Outcomes and Real World Evidence (GPORWE) Center of Expertise (COE). The GPORWE COE is a multi-disciplinary group that supports the other functions within GPORWE and Lilly in the development and commercialization of Lilly products. Jim provides expertise and scientific support for Real World Evidence capabilities and Observational Study Design. He is currently dedicated to external engagement efforts on the value of diagnostics and treatments for Alzheimer’s Disease.
Jim is married with two grown children. Jim’s hobbies include playing bass in the band Doghouse Grove and biking.
Jim has a Master’s degree in Information Systems and a Ph.D. in Decision Sciences and Operations Research both from the University of Wisconsin Madison.
Ebere Onukwugha, PhD
University of Maryland, 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.
Issue Panels and Workshops
Application of an Innovative Survival Extrapolation and Validation Method That Combines Clinical Trial Data, General Population Mortality and Expert Opinion
Live
PURPOSE: To describe an approach to generate and validate long-term survival estimates using clinical trial data, general population mortality and expert opinion.
DESCRIPTION: Long-term patient survival estimates are important for both clinical and regulatory decision making, but it can be challenging to extrapolate survival reliably over a lifetime time period when only short-term data from clinical trials are available.
Reimbursement agencies have highlighted the need for better alignment of survival extrapolations with clinical opinion and general population mortality. Additionally, regulatory agencies have expressed support for the use of external data sources in survival modelling provided that it is justified and that potential biases are mitigated. This workshop will describe an approach to the extrapolation and validation of long-term survival data that combines short-term trial data, general population mortality and elicited expert opinion. We will demonstrate the method using a new treatment for chronic kidney disease. The objectives of the workshop are:
To describe the methodology for the conduct of a robust expert elicitation survey, and the limitations of this approach. To show how incorporating formally elicited experts' judgments and general population data into survival models can improve the quality and validity of long-term extrapolations. To demonstrate, with case studies, the relevance of this approach for clinicians, regulators and reimbursement agencies. Audience participation will include two elements:
A real-time elicitation survey involving workshop participants. Participants will complete an elicitation survey requiring non-specialist knowledge before and after receiving training on judgemental biases and the elicitation process, to demonstrate the impact of judgmental biases on decision making and that accurate and reliable quantitative estimates can be elicited using a formalized survey protocol. Real-time polling to assess participant’s confidence in using elicited experts’ judgements in long-term survival extrapolation, at the start and end of the workshop.
Discussion Leaders
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.
Discussants
Mario Ouwens, PhD
University of Groningen, University Medical Center Groningen, Department of Health Sciences, Groningen, O, Netherlands
Mario Ouwens, PhD in biostatistics, is a senior statistical science director in AZ. He is group director of the medical and payer evidence statistics group and has many years worked in statistical innovation for health economics and real world evidence.
Carol Pollock, MBBS, PhD, FRACP, FAAHMS
Kolling Institute, Royal North Shore Hospital, University of Sydney, St Leonards, Australia
In 2021 Prof Carol Pollock was awarded an Officer in the Order of Australia (AO) for distinguished service to medical research, education and science, to nephrology, clinical practice and governance. She is an academic nephrologist with over 400 publications in basic research and clinical medicine and over 26,700 citations. She is Fellow of the Australian Academy of Health & Medical Sciences, was conferred a Vice Chancellors Award for Excellence in Research Supervision & recognised as a ‘Distinguished Professor’ by the University of Sydney. She is Chair of Kidney Health Australia, Chair of the NSW Bureau of Health Information & Deputy Chair of the Australian Organ, Tissue & Transplant Authority
Bart Willigers, PhD
AstraZeneca, Poslingford, United Kingdom
Decision making is an intrinsic human trait; a trait that few of us master.
Bart, as a Decision Scientist, provides clarity and insight to complex decision situations. This transparency enables decision makers to confidently plan a way forward. Decision Science combines psychology with rigorous analytics to enhance Decision Quality.
Currently, Bart assists AstraZeneca’s leaders to deal with their toughest decisions. Bart published many papers on the theory and application of Decision Sciences.
Qualitative Analysis of Video Assessments to Support Regulatory Submissions in Rare Diseases
Live
PURPOSE
: The workshop will provide an overview of the use of video assessments in rare disease to complement traditional endpoints during clinical trials. Furthermore, it will demonstrate potential analytical challenges pertaining to their use based on the speakers’ practical experience.
DESCRIPTION
:
Introduction (10 minutes). An overview of the regulatory space within the rare disease and description of the methodological challenges commonly faced in the rare disease clinical trials (e.g. small, heterogeneous patient populations and lack of validated, disease-specific outcome measures) will be presented.
Practical experience task (10 minutes). A description of the team’s recent practical experience of incorporating video evidence to support validation of a rare disease specific rating scale for clinicians and parents will be provided.
Interactive task (15 minutes). The audience will be engaged in an interactive task. Participants will be split into groups and instructed to assess videos of patients with rare disease (videos publically available on e.g. National Association for Rare Disease - NORD website). The participants of the workshop will be asked to rate videos with a scale/coding framework with an aim to explore potential challenges of analyzing this type of evidence.
Discussion (15 minutes). The team will guide the discussion pertaining to the video analysis results (interactive task), with a specific focus on lessons learned, while also highlighting the potential use of this data (e.g. confirming scale sensitivity to change, establishing, disease progression and scale inter-rater reliability) to support regulatory approvals.
Conclusion (5 minutes). Workshop with conclude with a short overview outlining the lessons learned from the session.
Discussion Leaders
Katja Rudell, PhD, MSc
Parexel International, Uxbridge, United Kingdom
Katja Rudell has cofounded the ISPOR Special Working Group in COA at ISPOR in 2019. She holds a PhD from University of London and has been working in Clinical Outcomes Research for 20 + years.
Discussants
Linda Abetz-Webb, MA
Patient-Centred Outcomes Assessments, Bollington, United Kingdom
Alessandra Girardi, PhD
Parexel International, London, LON, United Kingdom
Kavita Jarodia, PhD
Parexel International, Panchkula, HR, India
I am working as an Associate with over six years experience in academia and two year in consulting. As an associate within the Clinical Outcomes Assessment (COA) team, I am responsible for providing qualitative data analysis expertise using ATLAS.ti software. I am also actively involved in conducting literature searches and contribute to the technical reports. I have background in anthropology and worked extensively with the pediatric populations implementing various anthropometric techniques. I have also worked in nutritional studies as a part of my PhD project. I am also a member of ISPOR and have attended an array of courses pertaining to Patient-Reported Outcomes Measures (PROMs).
I have completed PhD in Biological Anthropology, from Panjab University, Chandigarh, India. This comprised of a 6 year long doctoral research fellowship at the University. I also have a record of scientific publications including peer-reviewed publications and conference presentations.
Can We ‘Borrow’ Real World Outcomes? What Can We Leverage from Chinese and US Data When Assessing Real World Outcomes for HTA Agreements for Rare Diseases in Europe?
Live
PURPOSE: To explore the conditions and methods under which real-world data on rare diseases may be robustly borrowed from US and Chinese sources, given the known limitations of European country-specific sources.
DESCRIPTION: Randomized controlled trials have short time-horizons, and large sample sizes or randomized comparators are not always feasible in rare disease settings. Thus, the evidence base increasingly being presented to payers for these conditions can be inadequate to robustly determine clinical and cost-effectiveness. This uncertainty can delay critical decisions on reimbursement, impacting patient access. While “value-based” arrangements can be made between payers and manufacturers, establishing real-world outcomes for rare conditions remains challenging. HTA submission in Europe that aims to leverage real world outcomes faces several key questions: Can outcomes based on small numbers of patients be trusted? How can this be used to reliably address uncertainties in evidence?
The moderator will describe the problem and illustrate how US data have been used to support post-authorization safety mandates in Europe (10 minutes). This will set up a discussion on conditions under which US data can serve as a benchmark for real world outcome evaluations in Europe (15 minutes). The audience will be polled to rate the acceptance of these from the research and stakeholder perspective. Then, a review of the available methodological approaches will be presented (propensity score matching, inverse probability of treatment weighting and quantitative bias analysis), with the audience voting on their familiarity with the methods and if they consider them robust (10 minutes). Finally, a case study of a rare condition (e.g., spinal muscle atrophy) leveraging Chinese RWD will be presented (15 minutes) – the results will be contextualized in the prior European findings and the audience will be engaged to assess face validity. The workshop will conclude with the summary of the results and a Q&A (10 minutes).
Discussion Leaders
Radek Wasiak, PhD
Cytel, London, United Kingdom
Discussants
Paul Arora, PhD
Cytel, Toronto, ON, Canada
Paul Arora is Vice President, Advanced Epidemiology at Cytel. He works in a leadership capacity in the real-world evidence space and contributes to the growth and development of multiple business lines. Paul was a co-founder of Lighthouse Outcomes, a specialty consulting firm focusing on advanced analytics for clinical epidemiology research (acquired by Cytel in 2019). He works on the application of cutting edge epidemiologic methods and machine learning to problems in the health outcomes space. Paul serves as an Assistant Professor in the Division of Epidemiology at the Dalla Lana School of Public Health, University of Toronto. He is trained as an Epidemiologist and has spent the past fifteen years leading evaluations in public health and clinical epidemiology. He has developed extensive experience in methods for evidence synthesis and has worked closely with public and private institutions internationally in senior consulting roles and has published more than 50 peer-reviewed scientific articles. In addition, he worked with the team at Lighthouse Outcomes and academic collaborators to develop software for Bayesian network meta-analysis of clinical trials (BUGSnet).
Sreeram Ramagopalan, PhD
F. Hoffmann-La Roche, Basel, Switzerland
Sreeram (Ram) Ramagopalan is the Global Head for Real World Evidence for Market Access at Roche. Dr Ramagopalan’s team strategically plans and executes real world research studies to obtain and maintain access for Roche medicines. Dr Ramagopalan holds a PhD in Epidemiology from the University of Oxford, as well as an MSc in Epidemiology from the London School of Hygiene and Tropical Medicine. Dr Ramagopalan has a strong interest in leveraging routinely collected data for market access as well as using more novel data sources to understand the patient impact of disease and benefits of treatment He is an international expert in real world evidence with over 275 peer reviewed publications, and is regularly invited to speak at meetings internationally.
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.
Estimating the Value of New Alzheimer’s Disease Therapies and Early Disease Screening
Live
ISSUE: Using simulation models to estimate the value of new disease-modifying therapies in Alzheimer’s disease and early screening, and the value of their comparison.
OVERVIEW: In June 2021, the FDA conditionally approved a new drug treatment for Alzheimer’s disease for persons in a pre-dementia state of the disease. Establishing its health-economic value comes with several challenges, among which is establishing the natural course by analyzing clinical and registry data and implementing assumptions on long-term treatment effectiveness. Various models have been developed, but comparisons between them have been challenging, particularly as some models are more parsimonious than others and differ in their primary objectives.
This panel will address key challenges associated with simulation modeling of Alzheimer’s disease-modifying treatments (DMTs), and provide insights about the importance of model inputs, model design choices, and the interpretation of results. This panel will begin with an introduction by the moderator (5 minutes), followed by brief presentations by each of the three panelists (3-5 minutes). The remaining time will be for open discussion led by the moderator. The panel will highlight key lessons learned from model cross-comparison organized by the International Pharmacoeconomic Collaboration on Alzheimer’s Disease (www.ipecad.org). Panelists will share their insights and takeaways from Alzheimer’s modeling in Europe and the United States
Moderators
Scott Johnson, PhD
Medicus Economics, LLC, Milton, MA, USA
Scott J. Johnson is a Principal at Medicus Economics. He has over 15 years of experience in health economics, specializing in econometric analyses of large databases, discrete choice analysis and design of prospective experiments, modeling, pricing, and policy analysis. He has published on health economics and policy in Health Affairs, Journal of Clinical Oncology, PharmacoEconomics, JAMA Ophthalmology, Gut, and Journal of Health Politics, Policy and Law. Scott works at a firm specializing in cost-effectiveness analysis, data analysis and market access research. He has a Ph.D. in Applied Economics from the Wharton School of the University of Pennsylvania.
Panelists
Inge de Kok, PhD
Erasmus University Medical Center, Rotterdam, Netherlands
Dr. Inge M.C.M. de Kok is an Assistant Professor in public health. The direct translation from research to public health decision making, in collaboration with decision makers and relevant stakeholders, is what fascinates her the most. As a trained quantitative epidemiologist, she is an expert in the development and use of microsimulation models to simulate the natural history of diseases, based on best-available diverse data. She has developed multiple microsimulation models (‘MISCAN’ framework) for cancer, and subsequently quantified the effects of possible interventions on population and individual health. Presently, she uses that expertise in other fields, e.g., in Mental Health disorders: In 2017 Dr. de Kok received a Veni grant by the NWO to develop a microsimulation model to predict the future burden of dementia. She shared her experience with international partners within numerous projects, amongst others as a workpackage leader in the EU funded project ‘EU-TOPIA’ and as a PI of the NCI-sponsored comparative modelling consortium 'CISNET'. Her models increased the understanding of disease processes, brought diverse data together and identified promising interventions.
Ron Handels, PhD
Maastricht University, maastricht, Netherlands
Ron Handels works as an assistant professor at the Alzheimer Centre Limburg at Maastricht University and is affiliated to the Karolinska Institutet; and is part of the International Pharmaco-Economic collaboration on Alzheimer's Disease (www.ipecad.org). He has a background in epidemiology and cost-effectiveness analysis in Alzheimer's disease (AD). He has published in the area of potential cost-effectiveness of diagnostics in AD and natural AD disease progression. His work has been funded by national and international funding schemes (among which H2020, JPND and IMI). On an occasional basis he has provided advisory for pharmaceutical organizations.
Jakub Hlavka, PhD
Sol Price School of Public Policy, University of Southern California, Los Angeles, CA, USA
Jakub Hlávka, PhD, is a Research Assistant Professor in the Health Policy and Management Department of the Price School of Public Policy and Schaeffer Center for Health Policy & Economics, University of Southern California. His NIH-funded research focuses on the modeling of dementia treatments and associated economic challenges, with a specific focus on Alzheimer’s disease and emerging disease-modifying therapies. He has also worked on models of COVID-19 pandemic interventions estimating their costs and benefits. His broader research interests include innovative payment models for pharmaceuticals, health system reform and the study of inequality.
He currently serves as a research consultant for the National Academies of Sciences, Engineering, and Medicine (Committee on Improving Representation of Women and Underrepresented Minorities in Clinical Trials and Research), and is a member of the International Pharmacoeconomics Collaboration on Alzheimer’s Disease (IPECAD). Prof. Hlávka has additional professional experience from Genentech where he assisted in R&D portfolio planning and as a consultant to the Tufts Medical Center's Center for the Evaluation of Value and Risk in Health where he studied oncology-specific outcome measures.
Dr. Hlávka holds a PhD from the Pardee RAND Graduate School, a master’s degree from Georgetown University, Edmund A. Walsh School of Foreign Service, and an undergraduate degree from the University of Economics in Prague.
Defining Established Clinical Management in the Era of Managed Access in HTA
Live
ISSUE: Comparator choice is highly influential on the value assessment of health technologies. Different HTA systems across Europe have different reference cases with respect to defining comparators. A notable point of difference relates to the extent to which treatments that do not represent "established clinical management” should be included, and how this term is defined. For example, treatments that made available only via a temporary/conditional managed access agreement (MAA), and are therefore used in clinical practice but might not be considered as “established” given the uncertainty surrounding their long-term reimbursement.
MAAs are being increasingly used as a route to market access, meaning that in some disease areas standard clinical practice is increasingly dominated by treatments made available through such arrangements. This context is expected to only become more prevalent as treatments become more expensive or associated with more uncertainty and MAAs see more usage. This raises the importance of the question of appropriately and consistently defining comparators.
OVERVIEW: Naomi van Hest will set the scene of how HTA reference cases for comparators differ across Europe and how these differences can impact decision-making (10 minutes), and will moderate the subsequent discussion. Jacoline Bouvy will present the argument for excluding treatments that are not formally part of established clinical management, due to conditional reimbursement through managed access agreements (10 minutes). Gérard De Pouvourville will present the counter argument to this, highlighting the reasons for including these treatments (10 minutes). Finally, Mark Sculpher will provide his thoughts on the strengths and limitations of the different approaches, and on considerations for HTA systems going forwards into an era of increasing managed access agreements (15 minutes). Audience participation will be encouraged via interactive polling and a Q&A discussion (10 minutes). It is expected that pharmaceutical companies, HTA bodies and consultancies will benefit from attending.
Moderators
Naomi van Hest, MSc, BSc
Costello Medical, London, United Kingdom
Naomi is a Consultant Health Economist at Costello Medical, leading the development of health economic models for reimbursement and market access, particularly in oncology. She has contributed novel research to ISPOR over the past 4 years, ranging from cost-effectiveness analyses, to survival extrapolation, reducing uncertainty and expert elicitation. Naomi has completed a Master's in health data analytics from University College London, and studied her undergraduate degrees in Australia.
Panelists
Jacoline Bouvy, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Jacoline is the Technical Director for NICE Scientific Advice. She signs off work across all services offered by NICE Scientific Advice and chairs national and parallel scientific advice meetings with medical device and pharmaceutical companies.
Previously, she worked in NICE's Science Policy and Research team where she led work on histology-independent cancer drugs, use of patient preference studies in HTA, and several other pan-European research projects.
Before joining NICE, Jacoline worked at the European Medicines Agency and held postdoctoral positions at Erasmus University Rotterdam and Utrecht University in the Netherlands.
She has a PhD from Utrecht University, the Netherlands and an MSc in health economics from Erasmus University Rotterdam, the Netherlands.
Gérard de Pouvourville, Professor
ESSEC Business School, Cergy Pontoise, France
Mark Sculpher, PhD
University of York, York, United Kingdom
Mark Sculpher is Professor of Health Economics and Director of the Centre for Health Economics, University of York. He is also Co-Director of the Policy Research Unit in Economic Evaluation of Health and Care Interventions, a programme of research for the UK Department of Health and Social Care funded by the National Institute for Health Research (NIHR).
Is Health Economics Ready for Paradigm Shift from Paternalistic to Patient Centric Healthcare System?
Live
ISSUE
: The digital transformation introduced connected health technologies such as sensors for health monitoring, new evidence to inform the decision making such as behavioral data, new technologies such as mobile applications to focus on prevention instead of treatment. The paradigm shift towards patient centric healthcare system can be defined by two major components. Firstly, it is about healthcare services sold directly to patients. Secondly, it is about the patient becoming more than the sole customer but also the data owner and the decision maker. How the healthcare governance should change to ensure that patient centricity becomes the reality? What kind of health economics advancements are needed in support of patient's decision making?
OVERVIEW
: Moderator will ask GPT3 to present AI vision of healthcare ecosystem in the digital era. With reference to that introduction, each panelist will be asked to define patient centric system from his/her own perspective. In particular, Andrzej Rys will elaborate on the future of the European and national governmental regulations to shape the patient ownership in the digital era from regulatory standpoint. Anne-Pierre Pickaert will discuss whether patients should be empowered to take the full responsibility for their health. George Valiotis will define patient centric system through the lenses of healthcare providers and discuss the shift from decision makers to health adviser. Finally, all panelists together with moderator and GPT3 will engage audience in the discussion about the role of health economics to ensure a smooth transition from paternalistic to patient-centric healthcare system. Should digital transformation allow patients to become the payers? The particular focus will be drawn to the data governance, responsibilities shifts across governmental bodies, and methodological advancements needed for new era of patient's decision making.
Moderators
Katarzyna Kolasa, PhD
PAREXEL and Kozminski University, Warsaw, MZ, Poland
Driven with the passion to health economics, I have more than 20 years of academic and industry experience in the field of healthcare. Holding various Regional and Global leader-ship positions, I have worked with the pricing & reimbursement challenges in the pharma and medtech industry.
Since 2018 I am a Professor at the Kozminski University leading Health Economics & Healthcare Management Division (HeM). My PhD was titled “The principle of equity and social justice with respect to financing of health service in Poland and Sweden”. My habilita-tion book „Optimal allocation of healthcare resources” was published in 2017.
My extensive knowledge in the field of health economics was acquired at the University of York, University of Lund, and University of Bergen as well as during the International Doc-toral Courses in the Health Economics and Policy organized by the Swiss School of Public Health.
At the Kozminski University, I am the leader of the International Master Program Health Economics & Big Data (HEBDA) financed by the EU research grant of the National Center for Research and Development which was listed as the best project in the POWER 2018 call. I collaborate closely with the health economics partners from the University of Lund, the University of Athens, University of Illinois, University of York and the University of Ap-plied Science in Berlin as well.
My first practical skills in the field of health economics were developed during six years employment contract at the Kalmar County Council in Sweden. After that, I worked in both global and regional Health Economics & Outcomes Research (HEOR) functions at different pharmaceutical companies for ten years in total. At AstraZeneca and BiogenIdec. I held Global and European positions respectively. At Bristol Myers Squibb and Lundbeck I lead Market Access teams in Central Eastern European (CEE) and Nordic Region. In the last four years, I gained experience with the pricing &reimbursement challenges in the field of medi-cal devices. Since 2017, I have supported Straub Medical as Global Market Access Lead. Before that, I was employed as a Senior Sales Director responsible for HEOR at GE Healthcare.
In October 2020, I was nominated to ISPOR’s Health Sciences Policy Council (HSPC). I am chair elect of ISPOR Special Interest Group Digital Health as well.
Guest Editor of Special Issue "Quest for Value Drivers of Digital Health Solutions in the Post COVID-19 Era" at International Journal of Environmental Research and Public Health (ISSN 1660-4601) (IF 3.2)
Currently I am leading the project of the optimal allocation of CT scanners for the Polish Ministry of Health. It is the adaption of evolutionary algorithms to analyze more than 120 mln of historical CT procedures. In addition, my research agenda relates to the value frame-works of medical devices and digital health interventions. I am leading the project of the development of integrated healthcare model for oncological care based on patients’ prefer-ence for the Polish HTA agency.
Panelists
Anne-Pierre Pickaert, MSc
Patvocates, PARIS, France
Anne-Pierre Pickaert has over 20 years of public health and access to medicines experience with a wide range of healthcare stakeholders (charity, patient organisation, governmental agency, consultancy and pharmaceutical industry). Upon her own diagnosis Anne-Pierre realised how much her health care background was of great help to navigate through the complexities of her leukaemia patient journey. In order to have a greater impact on patient access to care, Anne-Pierre has founded Care4Access, a consultancy dedicated to patient engagement and advocacy, and has also been collaborating with the think tank and social enterprise Patvocates.
Anne-Pierre is an engaged patient advocate with EGMOS and Association Laurette Fugain, two French patient organisations, to promote bone marrow donor recruitment and improve the management of graft versus host disease GvHD. She is a steering committee member of the Acute Leukemia Advocates Network (ALAN), as well as a board member of the Francophone Society for Bone Marrow Transplant and Cellular Therapies (SFGM-TC), and a member of the EBMT Patient Advocacy Committee. Anne-Pierre is also a campaign committee member of the Hematopoietic stem cell Transplantation Complications (HTC) project.
Andrzej Rys, MD
European Commission, Brussels, Belgium
George Valiotis, Executive Director
European Health Management Association, Brussels, Belgium
13:30 - 16:00
Poster Session And Exhibitor Meet Ups
Live
Interact with the authors of the latest research discoveries in HEOR through our virtual poster presentations and gallery. In between poster discussions, connect one-on-one with our exhibitors and sponsors. Engage with potential solution, service, and resource providers.
Virtual Poster Discussion Session 1
Live
14:00 - 15:30
Educational Symposia
AMR and Pull Incentives: Still a Vision or Becoming a Reality in Europe?
Antimicrobial resistance (AMR) is a global issue and poses a serious threat to the effective prevention and treatment of infections caused by bacteria, viruses, and fungi. Whilst appropriate initiatives such as stewardship are useful to restrict their use to instances where treatment options are limited, there are challenges in supporting the development of new antibiotics. The introduction of incentives aims to encourage innovation and the development of new antibiotics. Push incentives directly lower the R&D cost of developing a new antimicrobial, while pull incentives, such as value-based pricing and reimbursement initiatives, improve commercial prospects . The objective of this symposium is to provide information on the conceptual role and types of pull incentives in incentivising R&D, to discuss pull incentive initiatives implemented to date, and to increase understanding of how these can be most effectively developed and implemented at country level, based on experiences across Europe.
Sponsor
Shionogi
Moderators
Christine Årdal, MBA, PhD
Norwegian Institute of Public Health, Oslo, Norway
Christine Årdal MBA PhD has worked for over 20 years on access to medicines through different sectors. At the Norwegian Institute of Public Health, her research focuses on the policy aspects of antimicrobial access and innovation. Årdal was the co-lead of the research and innovation work package for the European Union’s Joint Action on Antimicrobial Resistance and Healthcare-Associated Infections (EU-JAMRAI), detailing European strategies to implement mechanisms to increase antibiotic access and innovation. She was also a co-lead in the DRIVE-AB research project, with a focus to stimulate innovation, the sustainable use, and the equitable availability of novel antibiotics to meet unmet public health needs.
Speakers
James Anderson, MSc MBA
The International Federation of Pharmaceutical Manufacturers and Associations, Maidenhead, United Kingdom
James joined the IFPMA as Executive Director of Global Health in January 2021, where he is responsible for leading the work on priorities in global health. This includes industry’s policy and advocacy on pandemic preparedness, antimicrobial resistance (AMR) and global access to medicines and vaccines. In his previous role as Head of Corporate Government Affairs at GSK, James led GSK’s strategic engagement with the UK Government and was a member of the Life Science Strategy Board and Vice-Chair of Business at OECD’s Healthcare Committee. He led GSK’s work on AMR and advised on AMR policy development at the WHO, UN, EU Commission and National Governments. He was also in the Founding team of the AMR Action Fund and led the development of the Davos Declaration and UNGA Industry AMR Roadmap.
Timo Minssen, LL.Lic., LL.M., M.I.C.L., Dipl. Jur.
University of Copenhagen, Lund, Sweden
Professor Timo Minssen is the Founder and Managing Director of the Center for Advanced Studies in Biomedical Innovation Law (CeBIL) at the University of Copenhagen. He is also a senior consultant at the Swedish Law firm X-officio Advokat AB. Holding law & life science-related LL.M. & LL.D./Jur. Dr. degrees, he specializes in IPRs, antitrust, and regulatory law in the health & life science sectors. He is the new chair of the International Network for AMR Social Science (INAMRSS). Moreover, he collaborates on several research projects scrutinizing regulatory challenges posed by new health technologies and health threats with inter alia Harvard Law School, Harvard Medical School, the University of Cambridge, and the Max Planck Institute for Innovation and Competition.
Lotte Steuten, Prof. PhD, MSc
OHE, City, University of London, London, 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 health economics and HTA field for >15 years in various academic roles and executive functions (non-profit and for-profit).
Lotte’s research speciality is decision analysis, focusing on the development and application of health economic analysis and health technology assessment (HTA), with the aim to accelerate patient access to high value health care services and treatments. She has co-authored >100 journal publications and reports.
To translate research into better decisions, she works effectively with pharmaceutical industry, technology assessors, payers and policy makers, (academic) researchers, clinical and patient representatives as well as capital investors. Her international career-path, including the UK, the US and the Netherlands, has provided her with deep insights in the fundamental differences and commonalities between the role of health economic research and HTA policies and practices in different healthcare systems.
In her current role at the Office of Health Economics she is responsible for the research-led consulting program, maintaining OHE's stellar reputation for objective, innovative and high-quality research and analysis globally, and meeting its charitable objectives.
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. Prior to that she co-founded Panaxea, a healthcare research consultancy, and served as its CEO and CSO from 2010 to 2018. As an active member of ISPOR for over 10 years, Lotte serves at the Board of Directors, is Co-Chair of the Virtual ISPOR Europe 2021 meeting, and contribute(d) to their various Taskforces, Shortcourses, Committees, and diversity initiatives.
14:15 - 15:15
Student Network Session
Student Network Roundtable Event
The ISPOR Student Roundtable Event offers opportunities for student attendees to hear from experts on a wide array of subject manner ranging from career advice to scientific topics. When the event was in person, the event format was to have 3 presenters each discussing a different topic with attendees rotating from one table to the next. In the virtual format, we would like to explore accomplishing "rotating" between tables by having Breakout rooms set up where attendees will be moved from one topic to the next after 15 minutes. At the completion of having attendees visit all 3 speakers there will be 15 minutes at the end of the event for open discussion for all speakers.
Topics
Branding Your Research Expertise Dalia Dawoud
Publishing Your Research Ibrahim Alabbadi
Fellowships and Internships Annesha White
Moderators
Ingrid Cox, MD, MSC
Menzies Institute for Medical Research, University of Tasmania, Hobart, TAS, Australia
Dr Ingrid Cox is a physician and postdoctoral fellow at the Menzies Institute for Medical Research, University of Tasmania in Australia. She has extensive experience in public health, working in various countries and capacities in the Caribbean, including health policy, planning, monitoring and evaluation, consulting, and project management of health focused international development projects. Her current research focuses mainly on lung disease, including the health economic and epidemiological aspects of interstitial lung disease and lung cancer in Australia.
Shrey Gohil, B. Pharm.
University of Houston, Houston, TX, USA
Enrique Saldarriaga, MS
EsSalud, Seattle, WA, USA
Enrique Saldarriaga is a PhD candidate in Health Economics at the University of Washington. He holds a MS in Epidemiology and a BS in Economics. His dissertation combines advanced statistical methods and infectious disease modeling to quantify the economic value of improving the estimates of HIV at the zip code level as a tool to improve resources allocation within a city and long-term health outcomes. Enrique's career goal is to leverage quantitative methods to inform decision making processes in healthcare technology adoption, interventions, public policy design and implementation. His research interests include decision science, econometrics, disease modeling, and public policy design and evaluation.
Prashant Srivastava, B.PHARM
Amity University, Gwalior, India
Daniela Yucuma, MD
Pontificia Universidad Javeriana, Bogota, CUN, Colombia
Speakers
Ibrahim Alabbadi, PhD, MBA, BPharm
University of Jordan, Amman, AM, Jordan
Prof Ibrahim Alabbadi is a qualified pharmacist by profession since graduation in1987 (King Saud University) experienced in pharmaceutical marketing with multinational as well as Jordanian companies in the Middle East. Dr Alabbadi got his MBA-Marketing (pharmaceutical pricing) in 2001 from the University of Jordan, completed his PhD in PharmacoEconomics from Queen's University – UK in 2006; Dr Alabbadi is a Professor in PharmacoEconomics and Pharmaceutical marketing; has more than 40 publications in peer reviewed journals, supervised 14 postgraduate students in applied research, worked in the University of Jordan as an Assistant Dean for training Affairs, Director of Scientific Research Documentation Office and Local Community Development & Networking Centre Director, and Deputy General Director for Administrative Affairs - Jordan University Hospital, Dean of Scientific Research, and; Dean, Faculty of Pharmacy (industrial and pharmaceutical business tracks) and head of Pharmacy Practice department, Yarmouk University – Jordan.
Prof Alabbadi was invited as a speaker and presenter to more than 150 national, regional and international scientific gatherings. In addition, he is an active member in many national and international health related committees e.g. JFDA, MoH, JPD, WHO-EMRO, ISPOR, NICE, MeTA, JCP-USAID, DHI, and HHC...etc. Furthermore, he is a reviewer for many international and local health related scientific journals, and an evaluator for outside academic institutions’ staff promotion. Dr Alabbadi is Jordan ISPOR chapter co-founder (since 2008) & current president, and ISPOR Arabic Network cofounder & ex-executive committee chair (2014-2016).
Ibrahim is the Editor-in-Chief of the Jordan Journal of Pharmaceutical Sciences (SCOPUS; Q3) Since Sept 2019 and The Deputy Editor of Value in Health Regional issues for Central Eastern Europe Western Asia and Africa (SCOPUS; Q1, Web of Science, Midline, PubMed) since Jan 2020
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.
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.
16:00 - 17:00
ISPOR Forums
Biosimilars Tendering in Europe: Key Learnings Towards Sustainable Practices
This forum aims to share insights on biosimilar tendering across Europe, with the objective of identifying learnings that can be leveraged to support sustainable tender practices and biosimilar competition. This forum emerges in the sequence of relevant topics highlighted in the ISPOR Science Strategy and the Biosimilars Special Interest Group, by aiming to adress healthcare system inefficiencies in terms of biosimilar procurement. While biological medicines have changed the course of many complex conditions, their high cost challenges healthcare budgets around the globe. Enhancing competition in the biological medicines market, by introducing biosimilars, offers a timely opportunity for healthcare systems to address inefficiencies, by lowering treatment costs, and possibly facilitating broader access to patients. Procurement mechanisms are of specific interest in the context of establishing sustainable biosimilar competition market dynamics, as these can have an important impact on biosimilar uptake, price dynamics and competitor concentration. A careful design of procurement mechanisms, and discussion on what this exactly entails, is needed. The tender landscape for biosimilars varies considerably across Europe and settings, and offers the opportunity to derive learnings and best practices. As a result, this forum will present an insightful multi-stakeholder exchange not only on learnings and opportunities from current practices, but also on key recommendations for an optimal tender design, to guarantee long term competition and access of biosimilars to patients. The Forum will follow the structure of a panel, with 10 minutes of the session reserved for Q&A with the Forum attendees. Catarina Lopes Pereira will moderate the session. Liese Barbier will share pan-European insights on biosimilar tender practices from the academic perspective. Jackie Vanderpuye-Orgle will provide an overview of industry considerations for fostering a sustainable biosimilars market. Ilan Akker will provide insights from the Dutch competition authority perspective, drawing from the findings of their recent sector enquiry.
Moderators
Catarina Lopes Pereira, MSc
Medac GmbH, Wedel, Germany
Catarina Lopes Pereira works as Global Marker Access Manager at Medac GmbH since February 2020. As part of the market access team, she is working to enable patient access to oncologic and autoimmune medicines, namely orphan medicines and biosimilars. Catarina is also the chair-elect of the ISPOR Special Interest Group on biosimilars since June 2021. Prior to joining Medac, Catarina worked as Senior Market Access Manager in Medicines for Europe and Health Economics & Outcomes Research Analyst in the Center for Evidence-Based Medicine in the Faculty of Medicine of the University of Lisbon.
MSc in Pharmaceutical Sciences by background, she obtained her master in the Faculty of Pharmacy of the University of Lisbon. Catarina also holds an Executive Master in Management from the Solvay Brussels School of Economics and Management in the Université Libre de Bruxelles.
Speakers
Ilan Akker, MSc
Dutch Authority for Consumers & Markets, The Netherlands, Netherlands
Liese Barbier, PhD, MSc, PharmD
KU Leuven, Leuven, VBR, Belgium
Liese Barbier is a post-doctoral researcher at the KU Leuven in Belgium, in the Regulatory Sciences & Pharmaco-Economics research unit. Liese’s research interests and activities focus on regulatory, clinical and policy aspects of medicinal products, including clinical drug development, regulatory evaluation, market access policies, and clinical product implementation. She has a particular interest in optimizing regulatory and decision-making frameworks for oncology medicines,and biologicals including biosimilars. Liese’s PhD research focused on regulatory, clinical and policy challenges related to biosimilar market entry in Europe, through which multi-stakeholder informed recommendations were generated to foster sustainable off-patent biological market dynamics. In 2019, Liese was seconded to the European Medicines Agency as National Expert. Here, Liese was part of the Oncology, Haematology and Diagnostics office of the Human Medicines Evaluation Division and focused as Product Lead on the evaluation of biosimilar candidates in oncology. Further, Liese provided scientific input and support to biosimilar related initiatives across the Agency and coordinated as Scientific Lead the Biosimilar Medicinal Products Working Party. Liese Barbier obtained her PharmD at KU Leuven in 2015, after which she gained clinical experience as a pharmacist in the in- and outpatient setting. Liese (co-)wrote 10 peer-reviewed publications in international journals and is regularly invited as speaker at international and national conferences. She has presented her work to academic, policy and healthcare professional audiences.
Jacqueline Vanderpuye-Orgle, PhD
Parexel International LLC, Massachusetts, MA, 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.
Issue Panels and Workshops
Key Considerations for Using Digital Endpoints in HTA Decision-Making: Case Studies, Challenges and Opportunities
Live
PURPOSE: To explore the development of novel digital endpoints for use in clinical trials and discuss the opportunities and challenges of their application in the health technology assessment (HTA) of new therapies.
DESCRIPTION: Endpoints derived from data captured with digital health technologies (DHTs) are increasingly being used in clinical trials to demonstrate drug efficacy. Despite their increasing use, there is a lack of guidance about the evidence requirements for and application fields of novel digital endpoints to be considered in the HTA of new therapies.
This workshop will share case studies from Mobilise-D and IDEA-FAST, two Innovative Medicines Initiative (IMI) project, which are part of the IMI Neuronet project portfolio, that are developing validated digital endpoints for use in clinical trials, and that will also be usable within a regulatory/HTA context. Mobilise-D is focused on mobility assessment across several diseases, including Parkinson’s disease and multiple sclerosis; and IDEA-FAST on the assessment of fatigue, sleep and daily living activities in neurodegenerative and immune-mediated inflammatory diseases. Firstly, Kai Langel will begin the session by defining digital endpoints and reflecting on the current status of their use within neurodegenerative diseases. David Nobbs and Martin Wohlrab will then present an overview of Mobilise-D and IDEA-FAST, reflecting on their experiences of developing validated digital endpoints for use within the context of clinical trials. Finally, Harriet Unsworth will present the issues and challenges facing the use of digital endpoints to support HTA submissions and the need for methodological guidance for their use in this context. We will engage the audience during an interactive Q&A session, moderated by Kai Langel allowing participants to share their experiences of developing digital outcomes and engagement with HTA processes. This workshop will be relevant for anyone interested in the use of DHTs to support evidence generation, including academics, HTA agencies and pharmaceutical companies.
Discussion Leaders
Kai Langel, BSc
Janssen, Alforja, Spain
Kai Langel, Senior Director, Strategy and Innovation, Global Regulatory Policy and Intelligence, Janssen R&D
Since 2000, Kai has been a pioneer in patient-facing solutions for clinical trials working with young innovative technology companies. In 2012, Kai co-founded eClinicalHealth, the developers of the Clinpal remote clinical trial platform. Through his involvement in technical, operational and scientific roles, he has gained deep understanding of broad aspects of the patient journey in clinical trials from recruitment and engagement through data capture.
Kai brings his innovation mindset and ways of working to support Janssen’s global regulatory policy work across a range of topics. Kai has a passion for broad pre-competitive collaboration and is motivated to modernize the ways our broader ecosystem works together to advance the adoption of novel tools and methods to support efficient and patient-centric pharmaceutical R&D.
Discussants
David Nobbs, PhD
F. Hoffmann-La Roche AG, Basel, BS, Switzerland
David Nobbs is currently Disease Area Lead for Neurodevelopmental Disorders in the Digital Biomarker Area of Roche. He is responsible for leading cross-functional teams toward the development and validation of digital endpoints for use in drug development. He co-leads the Regulatory, Dissemination, Impact, Sustainability and Exploitation work-package within the IMI IDEA-FAST Consortium and is a member of the EPFIA Digital Medicines Working Group. He has a special interest in the evidentiary requirements of regulators and health technology assessment (HTA) bodies for digital endpoints and has participated in scientific advice, qualification procedures and working groups with EMA, FDA and HTAs. He previously received a BA and MSc in Philosophy, before obtaining an MSc and PhD in Cognitive Neuroscience from University College London (UCL).
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.
Martin Wohlrab, MSc.
Dr. Margarete Fischer-Bosch Institute for clinical pharmacology, Stuttgart, Germany
I am Martin Wohlrab, working in the IMI Horizon project Mobilise-D as a scientist in the HTA and regulatory work package. I supported to write a publication on marketing authorizations by the EMA and organised the approach to HTA organisations.
I graduated from the University of Duisburg-Essen in 2018 with a Master's degree in health economics with further education in HTA at the University Berlin. After starting my job at a health insurance company, I soon decided to continue research at the Margarete Fischer Bosch Institute for Clinical Pharmacology in Stuttgart,
to make digital endpoints usable in everyday clinical practice and our telemedicine department.
What Should Go Where: The Thin Line Between Uncertainty Analysis and Model Validation
Live
PURPOSE: Highlight and discuss relationships between model validation and uncertainty analysis. Develop suggestions on whether, and how, to keep these aspects separated.
DESCRIPTION: Model validation and uncertainty analysis are closely related. Both are recommended in Guidelines for the Economic Evaluation of Health Technologies. ISPOR good practice recommendations and tools for implementation are available. However, the interrelationship between both aspects has not been fully explored.
Uncertainty analysis is performed assuming health-economic models and input data are valid. In contrast, model validation cannot be performed without paying attention to model uncertainty. This holds especially true for validating model outcomes against empirical data. An important question is how best to weigh these two aspects into policy decisions and assess how they impact decision (un)certainty. The workshop starts with an overview of the problem by the moderator, an expert in uncertainty and value-of-information analysis. (5 minutes-CR) An explanation of common validation elements, e.g. external and face validity, will follow. Various types of uncertainty (structural, methodological, and parameter uncertainty) will be discussed and connected to different elements of validation. This provides a common terminology for the rest of the workshop. (15 minutes-TF) We point out existing good practice guidelines, focusing on operationalising model validity in relation to parameter and decision uncertainty. This will be highlighted using examples of economic evaluation of anti-cancer medication. (15 minutes-SG) Finally, we discuss how aspects of model validity and uncertainty have influenced reimbursement decisions in the UK. (15 minutes-ICR) AUDIENCE INTERACTIVE ELEMENT Between-presentations questions are permitted (5 minutes). The audience participates in an exercise with the purpose of identifying how model validation and uncertainty can be operationalised and weighted in healthcare decision-making. Speakers present examples of different validation/uncertainty elements expected to influence policy recommendations. The audience will judge these examples before/after the presentations (by e-voting). Results will be discussed (10 minutes-CR moderates).
Discussion Leaders
Claire Rothery, PhD
University of York, York, NYK, United Kingdom
Discussants
Isaac Corro Ramos, PhD
institute for Medical Technology Assessment, Eindhoven, NB, Netherlands
Isaac Corro Ramos, PhD obtained his Master’s degree in Mathematics (option Statistics and Operations Research) from the University of Sevilla in June 2001. Between June 2001 and July 2005 Isaac had several jobs in different working areas and countries. He worked as a high-school Mathematics teacher in Sevilla (Spain), as a software programmer in Madrid (Spain) and Vienna (Austria), and as a researcher in Rome (Italy). In July 2005 he started his Ph.D. at the Department of Mathematics and Computer Science of the Eindhoven University of Technology working on the STRESS (Statistical Testing and Reliability Estimation of Software Systems) project. He defended his thesis on December 15, 2009. Since August 2009 Isaac works as a scientific researcher at the Institute for Medical Technology Assessment (iMTA) in Rotterdam, The Netherlands. Since he enrolled iMTA he has worked in several research projects whose subjects include probabilistic modelling, cost-effectiveness analysis of health care technologies, value of information analysis and discrete event simulation.
Talitha Feenstra, PhD
University Medical Center Groningen, Groningen, Netherlands
Talitha Feenstra is Associate Professor of PharmacoEconomics at the Groningen Research Institute of Pharmacy, Groningen University and is affiliated with the Dutch Institute for Public Health and the Environment (RIVM). She is a health economist with strong quantitative background, cum laude graduated in econometrics. Her research focuses on economic evaluations in health care, specifically for supporting personalized care by precision medicine. Ongoing projects concern analysis of routine data and simulation modeling in Diabetes Mellitus and mental health care. She developed the model validation reporting tool AdViSHE and worked for the Dutch Healthcare Institute on multi-use disease models.
Salah Ghabri, PhD
French National Authority for Health (HAS), Saint-Denis La Plaine, 75, France
Health economist researcher and senior scientific project manager at the French National Authority for Health (HAS) since 2012. My research interests revolve around economic evaluation of health technologies and health programs, analysis of uncertainty and health economic modelling and budget impact analysis. My expertise covers several areas of economic evaluation (e.g., chronic diseases, oncology), types of interventions (drugs, medical devices, and public programs) and Impact Evaluation Methods.
Optimising the Use of Managed Entry Agreements for Innovative Therapies: A Value-Based Negotiation Framework
Live
PURPOSE: To introduce a value-based negotiation framework for facilitating more structured negotiations of managed entry agreements (MEAs) for innovative therapies. Participants will have the opportunity to work with the framework to experience its practical applicability.
DESCRIPTION: Innovative therapies carry significant potential for patients. At the same time, delays in access frequently arise due to differences in manufacturer and payer perspectives regarding potential impact of a new product, and concerns around affordability or evidential uncertainties. There is a need to constructively manage these concerns, ultimately accelerating access to promising innovative therapies.
MEAs can ensure patient access to a treatment that would not be reimbursed because of affordability or evidential uncertainty concerns. There is, however, a push for greater MEA transparency and scrutiny. Moreover, lengthy negotiation processes further delay access and do not balance effectiveness with implementation burden. To support faster, more structured and transparent MEA negotiations, we propose a value-based negotiation framework. The framework, developed on the basis of current scientific literature and real-world expertise, aims to 1) systematically identify and prioritise manufacturer and payer concerns about a product, and 2) select a mutually acceptable combination of MEA terms that can best address priority concerns, with the lowest possible implementation burden. Discussion leaders will use 20 minutes to introduce the background and framework. Regulators, manufacturers, and payers will benefit from this workshop. INTERACTIVE ELEMENT: 5-10 minutes will be used to present a case example with three pre-populated key concerns to the audience, who will be split into two groups: ‘payers’ and ‘manufacturers’. Discussion leaders will use 20 minutes to guide each group in using the framework to identify a combination of agreement terms with the highest possible effectiveness to address the three provided concerns, and lowest possible implementation burden. In the last 10-15 minutes, the audience will discuss their experiences with the framework.
Discussion Leaders
Julien Patris, MA
Alnylam Pharmaceuticals, Bruxelles, Belgium
Julien Patris, Country Manager BeLux; Head of Policy Europe
Julien Patris is responsible for the general management of Alnylam’s operations in Belgium and Luxembourg. In his role, he oversees the P&L and the administration of the business in those two countries. In addition, Julien is the Head of Policy for the European region and lead government affairs activities at EU and national level. Julien has worked in several biotech and biopharmaceutical companies over the past two decades at international, EU and national level. Prior to his general management responsibilities, he had multiple leadership roles in Corporate Affairs, Government Affairs and Market Access. Julien has hands on experience in pricing and reimbursement and negotiations across multiple products and countries in Europe. He has authored and contributed to several peer-reviewed publications in the field of access, access policy and HTA, with a particular focus on alternative economic assessments in the field of rare diseases and orphan drugs. An economist by background, Julien has graduate from the College of Europe-Bruges (Economics), Sciences Po Strasbourg (Business, Finance) and Claude Bernard University Lyon (Pharmacy, Market Access). Julien is part of several informal think-tanks including the College of Europe Alumni in Health Group, the European Health Parliament Alumni Group. Julien lives with his wife and daughter in Brussels, Belgium.
Discussants
Gérard de Pouvourville, Professor
ESSEC Business School, Cergy Pontoise, France
Claudio Jommi, M.Sc.
SDA Bocconi School of Management, Bocconi University, Milano, Italy
Claudio Jommi is Professor of Practice of Health Policy and Director of the Master of International Health Care Management Economics and Policy of Health Policy at SDA Bocconi University. His research activity is focused on Market Access for Medicines, Pharmaceutical Economics, Policy and Management, Health Technology Assessment and Decision Making in Health Care. He published in international journals, including BMJ Open, Frontiers in Pharmacology, European Journal of Health Economics, Health Policy, Pharmacoeconomics, PLOS, Social Science and Medicine, and Value in Health.
Amanda Whittal, PhD
Dolon Ltd., Gottenheim, BW, Germany
Amanda Whittal is health psychologist by training and a Consultant at Dolon Ltd. She has been involved in a wide range of research and consulting projects with a focus on rare disease policy. Amanda worked on the orphan medicinal products workstream of the Horizon 2020 project ‘IMPACT-HTA: Improved methods and actionable tools for enhancing HTA.’
Using Federated Networks to Generate RWE: Why Not Now?
ISSUE: Evidence from observational studies has always been available to complement evidence from randomised clinical trials. However, recent advances in technology mean that such studies can be carried out on a large scale and cover a wide range of outcomes. This has resulted in lively debate on the place of RWE and its relevance to healthcare decision‑making. One approach to analysing real-world data is exemplified by the European Health Data and Evidence Network (EHDEN), a federated data network which includes data from across Europe which is standardised to a common data model. This issue panel explores whether such networks can provide meaningful RWE in regulatory, clinical and HTA contexts.
OVERVIEW: This issue panel will debate the value of using federated data networks for generating decision-grade RWE. Moderator Dalia Dawoud will open the session by briefly introducing the EHDEN network and how it is used to generate RWE. Dani Prieto-Alhambra will provide supporting evidence that shows how a federated data network has been used to respond in a timely way to regulatory COVID-19 epidemic evidence needs, focussing on research on the safety of hydroxychloroquine. Alex Asiimwe will describe how federated data networks can provide evidence to guide clinical practice, highlighting a use case that investigated the natural history and outcomes of prostate cancer patients managed by watchful waiting. Alan Lamb will provide the perspective of an HTA agency by describing how RWE is currently used in decision-making at NICE. He will highlight some of the challenges encountered in an HTA use case utilising the EHDEN network and whether and how these might be addressed.
The three 10-minute presentations will be followed by panel/audience discussion. Polling questions will be administered to seek the audience’s views on whether federated networks can generate meaningful evidence in each of the contexts examined.
Moderators
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.
Panelists
Alex Asiimwe, PhD
Bayer AG, Berlin, BE, Germany
Dr. Alex Asiimwe is a trained Clinical epidemiologist and is currently working with Bayer AG as Head of US Private Public partnerships. He is also an Associate Professor in clinical epidemiology. Alex has more than 15 years of experience in both industry and academic in the area of epidemiology and strategic partnerships.
In addition, Alex is also the project lead for TransCelerate and IMI PIONEER projects. Over the years, he has helped develop call text and build industry consortia for Bigdata@heart, PIONEER, HARMONY, DO-IT, GEETREAL, and EHDEN projects.
Alan Lamb, PhD
National Institute for Health and Care Excellence, Manchester, United Kingdom
Alan Lamb is a Scientific Adviser from the Science Policy & Research team at the National Institute for Health Care Excellence (NICE). NICE is responsible for producing evidence-based guidance and advice for health, public health and social care practitioners in the United Kingdom. He is involved with several European public-private research consortia, including the European Health Data & Evidence Network (EHDEN), which aims to address the current challenges in generating insights and evidence from real-world clinical data.
Daniel Prieto-Alhambra, MD MSc PhD
University of Oxford, Oxford, United Kingdom
Expanding the Notion of Value of New Health Technologies: Should Preventive Interventions Be Differently Considered Than Therapeutics?
Live
ISSUE: COVID-19 has unequivocally shown the broad health, economic and socioeconomic impacts that both vaccination and disease management can have on society, thus making a clear case for expanding health technology assessment (HTA) value frameworks. However, a reflection on how this should be done is needed. In particular, it is important to consider whether there are unique elements of the value of preventive interventions/vaccines which warrant the development of a separate value framework in comparison to therapeutics.
OVERVIEW: This panel will debate the pro’s and con’s for developing and implementing an expanded value framework specific to prevention interventions, using vaccines as a specific example. Josephine Mauskopf, the panel moderator will introduce the debate and will ask the audience for their initial thoughts on whether a unique framework is required or the same expanded elements of value be applied for vaccines and therapeutics(5 min).
The debate will be opened by Ekkehard Beck who will take the market access pathway perspective and provide insights into corresponding expansion challenges based on an analysis of current capture of value and role of HTA across countries (10 min). Following, Prof Maarten Postma will provide insights into the uniqueness of vaccines both from a public health and health economics perspective(10 min). Lastly, Prof Nancy Devlin will take a health economics perspective and will argue in favour of expanding the notion of value consistently across all health technologies, both preventive and therapeutic (10 min). Next, Josephine Mauskopf will present ideas to the audience about expanding the value framework and get their reactions via a live survey (5 min). After the discussion, the audience will again be asked to share their view on the question whether a unique framework is required for preventive interventions/vaccines.
Moderators
Josephine Mauskopf, PhD
RTI Health Solutions, Research Triangle Park, NC, USA
Josephine Mauskopf, PhD, MHA, is vice president of Health Economics at RTI HS. She has designed pharmaco¬economic research programs for drugs for bacterial infections, viral infections, psychiatric illness, and neurologic diseases. Dr. Mauskopf has completed an 8-year term as editor-in-chief of the journal Value in Health. She was co-chair of the two ISPOR budget impact analysis Task Forces and was co-chair of the ISPOR Task Force for the economic evaluation of vaccines for infectious diseases. She received the ISPOR Avedis Donabedian Lifetime Achievement Award in 2013 and the Marylin Dix-Smith Leadership Award in 2019.
Panelists
Ekkehard Beck, PhD
GSK, Munich, BY, Germany
Ekkehard Beck, PhD, Senior Director, Value Evidence and Outcomes Vaccines, GSK. Ekkehard Beck has given oral and poster presentations at ISPOR and other conferences such as ESPID, INFORMS. He has >10 years experience in health economics, epidemiology and outcomes research and published as lead author in journals such as ViH, Vaccine, etc.
Nancy Devlin, PhD
University of Melbourne, Melbourne, Australia
Nancy is Professor of Health Economics at the University of Melbourne and Senior Fellow at the Office of Health Economics, London. Her principal areas of research expertise are the measurement and valuation of patient reported health outcomes; the cost effectiveness thresholds used in making judgments about value for money in health care; and priority setting in health care. She is currently leading a large programme of research focused on improving measurement and valuation of quality of life in children (QUOKKA), funded by the Medical Research Future Fund (Australia). Nancy has published around 150 original peer reviewed journal articles and reports on a wide range of empirical and theoretical topics in health economics, and is co-author of Economic Analysis in Health Care, a leading textbook on health economics widely used in the UK and elsewhere and now in its second edition. Her work was highlighted in the UK’s NIHR 10-year anniversary report, which noted ‘The impact of her research is worldwide and highly significant in improving health and health care decision making’. Her research, submitted as a case study to the UK’s 2014 UK REF exercise, was judged by the sub-panel as ‘demonstrating very considerable impact in terms of reach and significance'. She is the elected Chair of Board of the EuroQol Research Foundation, the European-based international network of researchers that developed the EQ-5D, the world’s leading measure of patient reported outcomes. Nancy has also served as the President of ISPOR (2019-2020).
Maarten Postma, Professor
University of Groningen, Groningen, Netherlands
16:00 - 17:30
Spotlight Session
In “Deep” Thought: Advancing Machine Learning Methods in HEOR
Developments in the performance of machine learning (ML) methods have evolved significantly over the past decade with improvements in computational power (graphic processing units) combined with greatly expanded access to real world data. ML methods have traditionally focused on prediction and classification problems, but there is currently great interest in the potential of using ML for causal inference. Since regression methods have long been used for both prediction and causal inference, what are the relative merits of using ML versus regression for HEOR applications? The session will provide attendees with insight into the current state of the art in the performance of ML methods versus regression and attendees will gain valuable insight on how to apply ML methods to their own HEOR.
Moderators
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.
Speakers
Jenna Reps, Ph.D.
Janssen Pharmaceuticals, Inc., Raritan, NJ, USA
Jenna Reps is an associate director at Janssen Research and Development where she is focusing on developing novel solutions to personalize risk prediction. She is currently co-leading the patient level prediction OHDSI workgroup with the aim of developing open source and user-friendly software for developing risk models using data sets in the OMOP Common Data Model format.
Jenna received her BSc in Mathematics and MSc in Mathematical Biology at the University of Bath and her PhD in Computer Science at the University of Nottingham.
Vivek Rudrapatna, MD, PhD
University of California; School of Medicine, San Francisco, CA, USA
Panelists
Karin Groothuis-Oudshoorn, PhD
University of Twente, Enschede, Netherlands
I'm a biostatistician with an research interest in
the emerging field of statistical learning (or alternatively machine learning). I have a lot of experience in developing and analyzing predictive (regression) models and handling missing data with multiple imputation.
Next to that, my other research interest is in obtaining, in a methodologically sound way, patients’, physicians' and other stakeholders' preferences, values and needs to support decisions in health care. In that area my expertise are the design, analysis and interpretation of preference studies (conjoint analysis, best worst scaling) and multi-criteria decision analysis studies.
Wed 1 Dec
11:00 - 12:15
Plenary Session
Lessons Learned from EU Collaboration Efforts During the Pandemic - The End or the Beginning? From Vaccines to Orphans and Gene Therapies
COVID-19 pandemic has put strengths and weaknesses of healthcare systems including pharmaceuticals in a new light, both in Europe around the world and. Health systems responded effectively and cohesively to many challenges with new hospital beds created, countries allocating resources to regions in need, fostering collaboration in the pharmaceutical sector and among health care professionals aimed at bringing vaccines to European citizens. All these positive and encouraging efforts to respond adequately during the crisis create space for further development of European co-operation in post-COVID time.
In recent years we have seen how rare diseases, digital therapeutics, personalized medicine, cell and gene therapies, and other new therapeutic approaches have stretched our data and methodological capabilities. During this session panelists will consider what useful lessons we have learned from the SARS-CoV-2 pandemic so far, and what health systems would require from the HEOR community to respond to key challenges associated with these innovative solutions in emerging frontiers. A special focus will be put on an international partnership and dialogue aimed at special populations and technologies which have important implications for a range of European healthcare decisions, from regulatory pathways, pricing and reimbursement to managed entry agreements.
*Speakers to be added as confirmed!
Moderators
Marcin Czech, PhD, MD, MBA
The Institute of Mother and Child, Warsaw University of Technology, Warsaw, Poland
Marcin Czech is a full professor and head of the Department of Pharmacoeconomics at the Institute of Mother and Child in Warsaw, President of ISPOR, Poland Chapter, former Undersecretary of State/ Vice Minister at the Ministry of Health.
He is the author of over 250 articles and reports in the field of management, health economics, pharmacoeconomics and quality of life.
A medical doctor by education, specialist in epidemiology and specialist in public health, holding PhD degrees in medicine and management, MBA; completed postgraduate studies in Health Economics, Leadership Academy, university trainings at the University of York, University of St. Andrews, Mc. Master University.
Speakers
Lieven Annemans, PhD
Ghent University, Ghent, Belgium
Lieven Annemans is Senior Full Professor of Health Economics at the faculties of medicine of the ‘Vrije Universiteit Brussel’ and Ghent University. He is Past-President of ISPOR, was during 8 years chairman of the Flemish health council, and is currently chairman of the Flemish Comittee for Societal Revival. He is (co-)author of > 300 papers in peer-reviewed journals and published four books on health economics, among which “health economics for non-economists” (Pelckmans Pro, 2018).
Francis Arickx, Pharm.D
National Institute for Health and Disability Insurance RIZIV INAMI, Brussels, Belgium
Francis Arickx is the head of the directorate Reimbursement of Medicines and Pharmaceutical Policy within the Health Care Department at the National Institute for Health and Disability Insurance (NIHDI RIZIV/INAMI) in Belgium where he manages the departments responsible for administrative and scientific assessment and appraisal of reimbursement claims for medicines, orphan drugs, medical nutrition,…
Francis Arickx is the former secretary general for the Commission for Reimbursement of Medicines and acts as representative/expert for the Institute and Belgium on a number of national and European platforms (NM CAPR, IHSI, EUnetHTA,..). He is country coordinator for the BeNeLuxA Initiative (www.beneluxa.org), and today the overall coordinator of the Initiative.
Francis graduated in pharmaceutical sciences from the University of Ghent, Belgium and teaches ‘Health Policy’ in the Pharmaceutical Sciences Department at the University of Antwerp.
Andrzej Rys, MD
European Commission, Brussels, Belgium
Catherine Smallwood, DPhil, MSc
WHO Europe, 2100 København, United Kingdom
11:00 - 17:00
Poster & Exhibit Viewing
Live
Stop in to learn about the latest technology, services, and devices and to connect one-on-one with exhibitors and sponsors during our Exhibit Viewing Hours. Be sure to reserve some time to view the latest research in HEOR through our virtual poster gallery. Browse, comment, and discuss our posters at any time while our virtual platform is active.
12:30 - 13:30
ISPOR Forums
Linking HTA and Procurement: Forum Discussion on Assessing the Value of Medical Device - ISPOR SIG MD&D Key Project Presentation
The ISPOR Medical Device and Diagnostics Special Interest Group (MDD-SIG) is conducting a key project to characterize the usage of Health Technology Assessment (HTA) in procurement decisions. The goal of this forum is to discuss methods and initial results, and to engage the ISPOR community to inform and prioritize next steps. HTA is used to inform decision-making on the coverage and use of medical device and diagnostic technologies at the centralized, macroeconomic level and offers the potential of robust methods to help inform micro-level procurement decisions. Such use could reduce variation in access to these technologies; however, the nature of this use is not well characterized. Initial findings (predominantly from high-income countries) show that decision makers made their recommendations parallel to HTA by considering clinical data, cost-effectiveness, and safety; only a minority are based on HTA summaries and conclusions. There is little publication evidence of the generalizable link between HTA and procurement. Planned next steps involve a survey of relevant HTA and procurement stakeholders. This forum will elicit feedback on the most up-to-date version of that survey; attendees will have the opportunity to provide feedback and suggest prioritized next steps via ranking, polls, and open discussion. Discussion in this forum will include: mechanisms to increase the participation of the survey and finalize pertinent survey questions, the relative importance of different aspects of HTA in procurement decision-making, the costs and benefits of potentially duplicative assessments at the macro and micro levels, the role of the grey literature, and the role of ISPOR and its SIGs to better connect the HTA and procurement communities. The concluding period will allow for wrap up, questions, and review next steps. We welcome forum participation from MDD-SIG members, HTA professionals, payers, and anyone playing a role within or interested in medical technology procurement.
Moderators
Arthi Chandran, MS, MPH, DPH
Abbott, Santa Clara, CA, USA
Arthi Chandran is the former Vice President and Head of BD’s Health Economics and Outcomes Research (HEOR) function and has recently accepted the Divisional Vice President HEOR and Reimbursement leadership role at Abbott.
Arthi has been recognized for championing a cross-functional approach to innovation and market shaping with a focus on strategic evidence generation to enable broader adoption of health care technologies. She has successfully brought the practice of value integration into early stages of medical device product development, resulting in a portfolio of technologies with stronger value demonstration opportunities.
Arthi began her HEOR career at Pfizer Inc. where she was dedicated to supporting both the pain and women’s health therapeutic areas. She has been an HEOR leader in both US and Global roles and has focused on supporting growth and demonstrating value through strategic health economics and outcomes research programs to highlight differentiation in highly generic markets.
In addition to her professional leadership in the sciences, Arthi is passionate about the growth and professional development of young women. To that end she is an active board member of 1000 Dreams Fund. Arthi also serves on the board of the Healthcare Businesswomen’s Association’s Northern NJ Chapter.
Arthi holds a Masters in Public Health in Chronic Disease Epidemiology from Yale University, a Masters in Science in Quality Assurance and Regulatory Affairs from Temple University and a Doctorate in Health Policy and Management from the City University of New York.
Speakers
Michael Cangelosi, MA, MPH
Boston Scientific Corporation, Natick, MA, USA
Mr. Cangelosi is Associate Director of Health Economics and Market Access for Boston Scientific Endoscopy, Marlborough MA USA. He has been a practicing health economist for more than a decade and is active in the ISPOR Medical Device and Diagnostics Special Interest Group and the Boston Chapter of ISPOR. He trained in economics and public health prior to becoming a health economist and his research has focused on Gastroenterology, Pulmonary, and Nutrition. He has presented to various regulatory authorities and to large Integrated Delivery Network (IDN) Providers’ procurement bodies.
Akriti Chahar, MA, PhD Scholar
IQVIA, Gurgaon, India
Simon Eggington, MSc
Medtronic International Trading Sarl, Tolochenaz, VD, Switzerland
Simon Eggington is a health policy consultant at Medtronic and has a background in statistics and economic modelling. Prior to joining Medtronic in 2009, he worked for IMS Health and for the School of Health and Related Research at the University of Sheffield. Simon holds a BSc(Hons) in Applied Statistics and an MSc in Operational Research.
Issue Panels and Workshops
The PRO-CTCAE to Understand Symptomatic Toxicities with Cancer Medication: An American Dream or a Global Opportunity?
Live
ISSUE: The PRO-CTCAE is a set of patient-reported questions to evaluate the incidence, severity, frequency and interference of symptomatic toxicities associated with cancer treatment. The US FDA strongly advocates that sponsors systematically collect PRO-CTCAE data and has outlined a framework in which the data will inform the benefit-risk framework for new oncology treatments. The EMA has also expressed interest in PRO-CTCAE data but has not made clear statements on whether and how they would use PRO-CTCAE data in marketing authorization decisions. Beyond the regulators, the PRO-CTCAE has the potential to offer European (EU) HTA/payer agencies and healthcare professionals important information on patient-reported experiences with treatment. Only IQWiG have highlighted a potential use of PRO-CTCAE data, and no consensus statements from EU clinician groups exist.
The lack of guidance makes it difficult for sponsors to proactively generate and appropriately analyze and present PRO-CTCAE to EU stakeholders.
OVERVIEW: This panel will discuss how the use of the PRO-CTCAE is likely to evolve in EU markets and debate the opportunities and risks for using PRO-CTCAE to measure the patient experience. Three panelists will offer different perspectives: the EU regulator, the EU payer and the EU clinician.
Matt Reaney will provide an overview of what PRO-CTCAE is, and how it has been used to date (10 minutes). He will poll delegates on their perspective on collecting PRO-CTCAE data and pose 2 questions to the panel (25 minutes): (1) What are the opportunities for use of PRO-CTCAE data in decision-making in a European healthcare context in the coming years?; and (2) What are the risks to manufacturers and hurdles to a wide-spread adoption of PRO-CTCAE in oncology trials? Delegates will thereafter be invited to pose their own questions (15 minutes). At the end delegates will again be polled on their perspective on collecting PRO-CTCAE data.
Moderators
Matthew Reaney, CPsychol, CSci, MSc
IQVIA, Reading, United Kingdom
Matt Reaney is the Head of the Scientific and Analytic teams in the IQVIA Patient Centered Solutions organization. Alongside this he is a Practitioner Health Psychologist and retains academic/clinical links at 3 UK Universities
Panelists
Olivier Chassany, MD, PhD
Patient-Centered Outcomes Research, University Paris-Diderot, Paris, France
Professor of Therapeutics (Health Economics Clinical Trial Unit, AP-HP Paris hospitals, France), specialist in gastroenterology, with a long experience in developing Patient-Reported Outcomes (PRO) questionnaires. Involved for more than 20 years in the expertise of dossiers for EMA and French Drug Agency and for more than 30 years in Ethics Committees. Co-author of the EMA Reflection Paper on Health-Related Quality of Life. Deputy director of an academic research team on epidemiology and PRO (Université de Paris, Inserm). Current chair of the ISPOR SIG Clinical Outcomes Assessment (COA).
Tony Dhillon, MD
Royal Surrey Hospital NHS Foundation Trust, GUILDFORD, SRY, United Kingdom
Eva Dietrich, Pharmacist | Prof. | Dr. rer. nat. | MSc
University of Bonn Pharmaceutical Institute, Bonn, Germany
Professor Eva Susanne Dietrich is a pharmacist with more than 20 years professional experience in reimbursement, health politics, and drug evaluation.
She graduated at the University of Heidelberg, obtained her PhD in the field of pharmacoeconomic methodology from the University of Tuebingen and received a Master’s degree on Health Technology Assessment and Management from the University of Barcelona.
Professor Dietrich served as a deputy member of the Federal Joint Committee (G-BA) which specifies for almost 90 percent of the German population the services in medical care that are reimbursed. In parallel, she led the Department of Drugs, Remedies and Aids at the German National Association of Statutory Health Insurance Physicians (KBV) which concludes contracts with health insurance funds and other parties of the health care sector on the part of the 140,000 office-based physicians and psychotherapists in Germany. In the following years, she organized a scientific institute for Techniker Krankenkasse, one of the largest health insurance funds in Germany, and was responsible for the Management Division Health Sciences. Thereafter, she managed a scientific consulting firm in Basel for eight years and founded the Institute of Evidence-based Positioning in the Healthcare Sector in 2018.
An important emphasis of her professional and academic activities is the critical review of the evidence of new drugs, their classification in the existing care context and, more specifically, early benefit assessments and the German AMNOG process as well as the compilation of AMNOG dossiers.
Since 2000, Eva Susanne has been teaching at the department of clinical pharmacy at the University of Bonn, where she holds a honorary professorship and also leads a module in the drug regulatory affairs degree programme. Apart from this she taught pharmacoeconomics, evidence based medicine, and benefit legislation e.g. at the universities of Hamburg, Berlin and Marburg.
No Time to Wait: Can Early Access Schemes Work for Gene Therapies?
Live
ISSUE: Patients with high unmet needs often face rapid deterioration or even death while waiting for approval of new potentially transformative therapies. Hence most European countries have compassionate use mechanisms that enable pre-regulatory approval access to such medicines. Designed for repeat therapy, most require free provision of therapy for the duration of the scheme. Most gene therapies for rare diseases are single administration, giving the manufacturer no prospect of recouping any costs from those patients.
There is currently a stand-off between key stakeholders. Patients are no longer prepared to wait, demonstrated by a rise in medical crowdfunding campaigns. Payers, under pressure to allow access early, fear undermining HTA, pricing and reimbursement negotiations, particularly if high price benchmarks are set. Manufacturers worry about the sustainability of agreeing to provide one-time therapies for free. Several questions must be explored: How can early access mechanisms be designed to meet the needs of patients while protecting innovation? Where does the balance of responsibility between the manufacturer and the health care system lie? If manufacturers can be reimbursed during early access, how can we ensure the schemes are sustainable? How can stakeholders agree a price and a payment scheme? Is there a role for a European fund for early access? OVERVIEW: The moderator will briefly introduce the issue (4 mins) and the panel will then debate the concerns of paid early access schemes for one-time therapies like gene therapies and their perspective on the questions set out above. Panelists will each speak for 12 minutes, providing their perspectives on the issues. 20 minutes will be reserved for audience discussion.
The panel will be of interest to those working in health policy, the regulatory- HTA interface, industry and patient organisations, as well as payer and clinical representatives.
Moderators
Adrian Towse, MA, MPhil
Office of Health Economics, London, LON, 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
Oswald Bentinck, MSc
Novartis Gene Therapies, Wollerau, Switzerland
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.
Carole Longson, PhD
Carole Longson Consultant, Manchester, United Kingdom
Addressing Uncertainty Around Clinical and Population Health Data in Health Care Decision-Making: A COVID-19 Case Study
Live
PURPOSE: The COVID-19 pandemic showed preparing for future challenges is difficult, yet eminently necessary. Predicting uncertain future trends and planning for alternative scenarios is the new normal. This workshop will present modeling techniques and approaches that systematically deal with uncertainty around population health outcomes, treatment uptake, and health-care policy, and will discuss how these approaches can guide health care decision-making under uncertainty. Participants will be introduced to expert elicitation methods and modeling techniques used to generate actionable insights from expert assessments, and learn about the importance of high-quality input data and limitations of extrapolation methods. Participants will gain a broader understanding of the types of uncertainties to address in the context of COVID-19, such as estimated survival or vaccine uptake.
DESCRIPTION: This workshop will introduce both quantitative and qualitative techniques used to address different sources of uncertainties faced in health-care decision-making. Dr. Graf and Ms. Cope will provide an introduction and overview, along with an overview of uncertainties most commonly encountered (5 minutes). Prof. O’Hagan will provide an overview of expert elicitation methods like the Sheffield Expert Elicitation Framework (SHELF) and the Delphi method, and discuss how these methods can be used to inform key questions related to COVID-19 (20 minutes). Dr. Vardavas will discuss mathematical dynamic models, including complex population- and agent-based models as well as infectious disease transmission models, and will explore ways to address uncertainties around key modeling parameters for COVID-19 (20 minutes). Ms. Cope will review model applications for health economics (10 minutes), and will then, together with Dr. Graf, facilitate an interactive estimation exercise, where workshop participants will estimate key parameters such as COVID-19 survival rates, and also quantify their degree of uncertainty, using Zoom’s polling tool. Once submitted, audience estimates will be collected, visualized through an R-Shiny application, and discussed in detail (15 minutes).
Discussion Leaders
Marlon Graf, PhD
PRECISIONheor, Los Angeles, CA, USA
Marlon Graf is a 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.
Discussants
Shannon Cope, MSc
PRECISIONheor, Vancouver, BC, Canada
Shannon Cope, MSc, is a Vice President with PRECISIONheor. Shannon has over ten years of experience consulting for biopharmaceutical industry regarding evidence synthesis and economic evaluations to assess the value of new interventions.
Shannon leads and manages evidence synthesis projects involving systematic reviews, (network) meta-analyses, population-adjusted indirect comparisons (simulated treatment comparisons and matching-adjusted indirect comparisons), multi-level network meta-regressions and economic models. She has technical experience including synthesis methods related to aggregate and individual patient level data, time-to-event outcomes and repeated measures, and integration of evidence from expert elicitation. She has developed cost-effectiveness models incorporating flexible parametric models and been involved with HTA reimbursement dossiers in the UK, Scotland, Canada, Sweden, Netherlands, and Australia. She has provided methods-based trainings and workshops and published more than thirty articles in scientific peer-reviewed journals.
She has extensive experience in oncology, as well as several therapeutic areas including CAR-T therapy, cardiology, chronic obstructive pulmonary disease, asthma, and epilepsy. Prior to joining PRECISIONheor, Shannon was a Director at Mapi Group. She holds an MSc in Health Administration from the University of Toronto, Canada, and Bachelor of Health Sciences from McMaster University in Hamilton, Canada.
Anthony O'Hagan, PhD
University of Sheffield, Nottingham, NTT, United Kingdom
Tony O'Hagan is Professor Emeritus in Statistics at the University of Sheffield. His research is in the methodology and application of Bayesian statistics. In particular, he is active in the elicitation of expert knowledge in probabilistic form. He has facilitated many elicitation workshops and has trained others to be effective facilitators, notably in the field of pharmaceutical development. He is co-creator of the Sheffield Elicitation Framework (SHELF).
Raffaele Vardavas, PhD, MSci
RAND Corporation, los angeles, CA, USA
Raffaele Vardavas is a Mathematician at the RAND Corporation. His main area of research focuses on modeling the spread of infectious disease and behavioral contagion in social systems using both population-based and agent-based models. He has a Ph.D. in Physics from Imperial College London obtained in 2002. Before joining RAND in 2008, he was a post-doctoral researcher at UCLA in the applied mathematics and later in the biomathematics departments.
Policy and Statistical Issues in HTA Review of Histology-Independent Technologies (HIT) in Oncology
Live
PURPOSE
:
This workshop will provide an overview of issues identified by the recent Framework for the Health Technology Assessment of Histology‑independent Precision Oncology Therapies (Gaultney, 2021), and discuss statistical methods and sources of evidence to address these issues. DESCRIPTION
:
The National Institute for Health and Care Excellence (NICE) and other HTA Agencies have started to evaluate the use of HITs in precision oncology. Assessment of the clinical and economic benefits of such treatments can be complicated by the heterogeneity of patient populations covered by a histology-independent approval, as well as the types of evidence that are commonly generated for a technology approval (and thus are available for HTA review). Failure to adequately account for heterogeneity in cost-effectiveness across histologies may result in the reimbursement of a HIT for histologies in which it is not cost-effective, and likewise, failure to reimburse in histologies where it may be cost-effective. Inclusive Phase II trials for HIT indications can often fail to generate needed evidence for HTA review as they are unable to capture all possible histologies that are eligible for the technology, unable to gather sufficient numbers of patients within individual histologies, and are uncontrolled. As new agents are identified and evaluated, and post-approval data become available, analyses incorporating real-world evidence can supplement trials and aid in decision-making that can provide insight into subpopulation heterogeneity, particularly as it relates to quantifying the benefits of introducing a HIT into standard practice. AUDIENCE INTERACTIVE ELEMENTS Polls will assess prior participant HTA engagement, as well as experience with various HIT modelling approaches. Simulated histology-agnostic longitudinal datasets and related R code will assist in understanding how multilevel models can estimate prognostic and predictive value of HIT-relevant biomarkers, effectively quantify histology-specific uncertainty, and identify areas where additional information can have highest impact in an HTA submission.
Discussion Leaders
Jeremy Snider, PhD
Flatiron Health, New York, NY, USA
Jeremy Snider is a health services researcher with interests in evaluative sciences and statistics, health economics and outcomes research, pharmacoeconomics, oncology, and health systems. He currently is a Senior Quantitative Scientist at Flatiron Health, leading investigations and methods research relating to the Flatiron Health - Foundation Medicine Clinicogenomic Database. He completed his PhD in Health Services Research at the University of Washington - Seattle.
Discussants
Jacoline Bouvy, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Jacoline is the Technical Director for NICE Scientific Advice. She signs off work across all services offered by NICE Scientific Advice and chairs national and parallel scientific advice meetings with medical device and pharmaceutical companies.
Previously, she worked in NICE's Science Policy and Research team where she led work on histology-independent cancer drugs, use of patient preference studies in HTA, and several other pan-European research projects.
Before joining NICE, Jacoline worked at the European Medicines Agency and held postdoctoral positions at Erasmus University Rotterdam and Utrecht University in the Netherlands.
She has a PhD from Utrecht University, the Netherlands and an MSc in health economics from Erasmus University Rotterdam, the Netherlands.
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.
COVID-19: Challenges for HTA and the Need for a Best-Practice Framework During the Pandemic Recovery
Live
PURPOSE: This workshop will outline the challenges faced by health technology assessment (HTA) agencies when assessing treatments and diagnostics for COVID-19, and the role of HTA as healthcare systems begin to recover from the pandemic. Attendees should gain insight into the key difficulties and the potential utility of interim HTA methods.
DESCRIPTION: In the early phase of the pandemic, HTA took a back seat as policymakers prioritised speed in their response to the crisis. As transmission falls and more technologies for COVID-19 enter the market, HTA agencies are starting to be tasked with assessing their value. However, these assessments are proving to be challenging.
The Innovation of HTA Methods (IHTAM) Framework has been developed as part of the Horizon2020 funded project HTx (Next Generation Health Technology Assessment). We sought to follow IHTAM to develop best-practice guidance for assessing COVID-19 technologies during the pandemic recovery phase. First, we conducted a survey and subsequent roundtable workshop with representatives from global HTA agencies, to understand the challenges faced when assessing COVID-19 technologies and identify the case for interim guidance. In this workshop, Wim Goettsch will introduce the IHTAM Framework and its application in the context of COVID-19 (8 min.). Jamie Elvidge will present findings from the survey and roundtable workshop, outlining the challenges for HTA agencies when assessing COVID-19 technologies (10 min.). Roisin Adams will elaborate on key challenges from an HTA agency’s perspective (10 min.). Nicola Trevor will provide an industry view on challenges in demonstrating the value of COVID-19 technologies (10 min.). Jamie Elvidge will then consider the evolving role of HTA as the pandemic situation changes over time and where interim methods may be useful (5 min.). Finally, there will be audience Q&A including interactive polling (17 min.). This workshop will be of interest to all stakeholders involved in HTA.
Discussion Leaders
Wim Goettsch, PhD
National Health Care Institute (ZIN); Utrecht University, Division of Pharmacoepidemiology and Clinical Pharmacology, Diemen, Netherlands
Wim Goettsch, PhD is currently Special Advisor HTA at the Dutch National Health Care Institute He was the Director of the EUnetHTA JA3 (2016-2021) Directorate and Chair of the Executive Board of EUnetHTA between June 2016 and March 2018. Since 2019, he also has a position as an Associate Professor at Utrecht University (NL) where he is leading a new H2020 consortium with fifteen partners around Europe, called HTx on new methods for Health Technology Assessment for personalized medicine (2019-2024). He is currently Director in the Board of HTAi (2019-2022).
Discussants
Roisin Adams, MPharm, MSc, PhD
National Centre for Pharmacoeconomics, Dublin, Ireland
Dr. Roisin Adams is Head of HTA Strategy and External engagement for the NCPE. Dr. Adams led the HTA team for a number
of years before being seconded to the HSE to lead a new unit tasked with overseeing and managing high cost drugs in acute hospitals. Dr. Adams co-chairs the HTA domain of the Beneluxa initiative and oversees the EUNetHTA work of the NCPE. She also is a Director on the Board of the International Horizon Scanning Initiative. She has been awarded a number of grants from the Health Research Board and held advisory positions for Department of Health, the Health Information and Quality Authority and policy direction at EU level. In recent years she has led the COVID-19 specific assessments for the NCPE.
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).
Nicola Trevor, MSc
Janssen, High Wycombe, BKM, United Kingdom
Nicola Trevor has 12 years of experience in health economics covering academia, consultancy and the pharmaceutical industry. Nicola is currently Head of Health Economics, Market Access and Reimbursement at Janssen. Passionate about equality, diversity and inclusion, real world evidence and problem solving. Nicola also love pubs, cats and hiking.
The Potential of R Shiny User Interfaces to Support Health Economic Decision Making
Live
PURPOSE
:
To introduce participants to R Shiny user interfaces and demonstrate how they can be used to make health economic decision models more accessible to stakeholders. Sample demo:
https://darkpeakanalytics.shinyapps.io/sadm-mk2/ DESCRIPTION
:
As health economic decision models become more computationally burdensome, the shortcomings of Microsoft Excel are becoming increasingly apparent. As a result, high level programming languages such as R are becoming more popular. The script-based nature of these languages may be perceived as lacking an accessible interface, limiting the extent to which stakeholders, who are not modellers, can engage with economic models. The R package ‘shiny’ provides an attractive solution: it allows the creation of easy-to-use web interfaces, which allow decision makers to directly interact with the decision model. Users can easily input their own assumptions and explore completely new scenarios in real-time. The underlying R code can be submitted alongside the interface to be reviewed and transparently debugged. In the future, R Shiny user interfaces could complement HTA submissions and improve health policy decision making. The workshop will consist of 4 parts: Introduction to R and Shiny in HTA: current situation and future potential (Gianluca Baio) – 10 mins Interactive coding tutorial: building a simple Shiny app for a Markov model in R (Robert Smith) – 30 mins Tips for using R Shiny in practice for efficient scalability and multi-team projects (Rose Hart) – 10 mins Open questions and discussion – 10 mins Audience interactive element: Interactive R Shiny coding tutorial Source code will be provided to all participants Audience practice use of pre-deployed apps, demonstrating their utility.
Discussion Leaders
Paul Schneider, MD MSc
University of Sheffield, Sheffield, United Kingdom
Paul Schneider is a PhD student at the Wellcome Trust funded doctoral training centre for public health, economics, and decision science at University of Sheffield, UK.
https://bitowaqr.github.io/
His background is in clinical medicine: he is a physician by training and holds a doctoral degree from the University of Witten/Herdecke, Germany, as well as a research masters degree in health sciences from Maastricht University, the Netherlands.
Paul's research focuses on methodological and normative issues in the valuation of health, but he also has a keen interest in the use of modern software (R) and interactive user interfaces (shiny, JS) for health economic modelling.
More info: https://bitowaqr.github.io/
Discussants
Gianluca Baio, PhD
University College London, London, United Kingdom
Gianluca is Professor of Statistics and Health Economics in the Department of Statistical Science at University College London. He graduated in Statistics and Economics from the University of Florence (Italy) and completed a PhD programme in Applied Statistics again at the University of Florence, after a period at the Program on the Pharmaceutical Industry at the MIT Sloan School of Management, Cambridge (USA).
His main interests are in Bayesian statistical modelling for cost effectiveness analysis and decision-making problems in the health systems, hierarchical/multilevel models and causal inference using the decision-theoretic approach.
He leads the Statistics for Health Economic Evaluation research group within the department of Statistical Science and collaborates with the UK National Institute for Health and Care Excellence (NICE) as a Scientific Advisor on Health Technology Appraisal projects.
Rose Hart, PhD
BresMed Health Solutions Ltd., Sheffield, United Kingdom
Rose Hart is a Senior Health Economist at BresMed, where she was a member of the Health Economics Analysis Team for 4 years before joining the Advanced Analytics Team. She is experienced in developing health economic models for early-phase cost effectiveness and for health technology assessment. As a specialist in R modelling, with and without Shiny interface functionality, she is responsible for internal Shiny training and led in developing BresMed’s intRface™ services and pilot model. Rose previously worked in the BioPharm team at GlaxoSmithKline, and researched for her PhD at the University of Sheffield.
Robert Smith, MSc
University of Sheffield, SHEFFIELD, United Kingdom
13:30 - 16:00
Poster Session And Exhibitor Meet Ups
Live
Interact with the authors of the latest research discoveries in HEOR through our virtual poster presentations and gallery. In between poster discussions, connect one-on-one with our exhibitors and sponsors. Engage with potential solution, service, and resource providers.
Virtual Poster Discussion Session 2
Live
14:00 - 15:30
Educational Symposia
Health Technology Assessment: The New Frontier for Gene Therapies in Europe
Live
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 the complexities involved in evaluating and valuing gene therapies, to enable patient access to these novel therapies in the European region. Panelists will discuss the affordability challenge posed by novel single-dose treatments, such as gene therapies, compared to chronic therapies historically reimbursed in Europe. They will explore the evolution of the reimbursement landscape for cell and gene therapies in Europe and reflect on the drug value evidence-based challenges for novel therapies such as gene therapy, including how to value benefits beyond quality of life and survival.
Sponsor
Novartis Gene Therapies
Moderators
Omar Dabbous, MD, MPH
Novartis Gene Therapies, Inc., Bannockburn, IL, USA
Speakers
Michael Drummond, MCom, DPhil
University of York, York, YOR, 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 650 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.
J.-Matthias Graf von der Schulenburg, PhD, MD
Leibniz University Hannover, Hannover, Germany
J.-Matthias Graf von der Schulenburg is editor in chief of the European Journal of Health Economics and the Health Economics Review. He teaches risk and insurance and health economics at the Leibniz University Hannover. For his research, he has received the Alexander von Humboldt Award 1990 and the American Risk and Insurance Award in 1992. He is member of the Academy of Sciences and Literature,Mainz and the European Academy for Sciences and Arts.
Mondher Toumi, MD, MSc, PhD
Aix Marseille University, Marseille, France
Professor Mondher Toumi is a MD by training and holds two MSc in Biostatistics, and in Biological Sciences (option pharmacology) and a PhD in Economic Sciences. He is professor of Public Health at Aix-Marseille University. After working for 12 years as a research manager in the Department of Pharmacology at the University of Marseille, he joined the Public Health Department in 1993. In 1995, he entered the pharmaceutical industry and worked there for 13 years.
Mondher Toumi was appointed global vice president at Lundbeck A/S in charge of health economics, outcome research, pricing, market access, epidemiology, risk management, governmental affairs, and competitive intelligence. In 2008, he founded Creativ-Ceutical, an international consulting firm dedicated to support health industries and authorities in strategic decision-making.
In February 2009 he was appointed professor at Lyon I University in the Department of Decision Sciences and Health Policies. The same year, he was appointed director of the chair of Public Health and Market Access. He launched the first European University Diploma of Market Access (EMAUD) an international course already followed by more than 350 students. Additionally, he recently created the Market Access Society to promote research and scientific activities around market access, public health and health economic assessment. Since 2009, he also chaired the Annual Market Access Day, a purely academic event. He is editor in chief of the Journal of Market Access and Health Policy (JMAHP), which is PubMed index.
Since September 2014, he joined the research unit EA3279 of the Public Health Department, at Aix-Marseille University (France) as full professor. Mondher Toumi is also visiting professor at Beijing University (Third Hospital).
He has been an active member of ISPOR from its creation. He served as co-chair of the Research Review Committee ISPOR Europe in 2012 and 2013. He contributed to Short Courses in ISPOR Asia and Europe. He is member of the Editorial Board of Value and Outcome Spotlight. From 2010 he contributed to ISPOR conferences with more than 196 posters and podium presentations and 11 issue panels and workshops. He co-Chaired two consecutive SIGs on orphan drugs. He also supported ISPOR African and Nigerian Chapter.
He is a recognized expert in health economics and an authority on market access and risk management. He has more than 200 scientific publications and oral communications and has contributed to several books. He just finished a book on Market Access soon to be available.
14:15 - 15:15
Women in HEOR Session
Relationships Matter: How to Leverage Mentoring to Advance Your Career
Mentorship provides advantages for professionals at any career stage. It supports career development, allows professionals to connect their motivations and values to their careers, enhances human connection, and builds relationships. A panel of HEOR leaders will discuss how to leverage mentoring for career growth and will share their mentorship experiences through multi-stakeholder perspectives. Panelists will examine issues such as: how to identify and gain career mentors, why mentorship does not have to take place in a formal mentoring program, the distinction between mentors and sponsors, and how mentoring benefits both the mentor and mentee. Panelists will encourage questions and interaction from the audience. This session is open to all Virtual ISPOR Europe 2021 registrants—women and men in HEOR.
Moderators
Julia F. Slejko, PhD
University of Maryland, 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.
Speakers
Jalpa Doshi, PhD
University of Pennsylvania, Philadelphia, PA, USA
Jalpa A. Doshi, PhD, is a Professor at the University of Pennsylvania. She is also Director of the Economic Evaluations Unit of the Center for Evidence-based Practice and Director of Value Based Insurance Design Initiatives at the Center for Health Incentives and Behavioral Economics. Her work applies health economics, outcomes research, and policy methods to address issues related to pharmaceutical access, outcomes, costs, and value. Her research has received widespread attention from the media including the NY Times and the Wall Street Journal and has informed policies of private insurers and government programs. She has served as co-editor of Value in Health and a member of the ISPOR Board of Directors.
Jan E. Hansen, PhD
Genentech, South San Francisco, CA, USA
Jan Hansen, PhD, is Vice President of the Evidence for Access Unit within Genentech’s US Medical Affairs. Her unit is staffed with researchers who are uniquely positioned and skilled to address both the value and quality drivers of access in the US. These researchers are tasked to consider both value and quality evidence when they develop access strategies and tactics. Jan is a seasoned executive with more than twenty-five years of experience in the health care and pharmaceutical industries. Jan’s pharmacoeconomics and health outcomes expertise were acquired throughout her vast career experiences in patient-based research, health care consulting, sales strategy and execution, and in both global and US focused outcomes research. She has held positions with increasing leadership responsibility at several well-regarded companies, including PCS-Pharmaceutical Data Services (now CVS-Caremark), NDC Health, Wolters Kluwer Health, Glaxo Wellcome (now GlaxoSmithkline), and Allergan. Her professional accomplishments have been recognized by several professional organizations and resulted in numerous appointments, including: the University of Iowa College of Pharmacy Advisory Council, past Chair and current member of the University of Washington College of Pharmacy’s Corporate Advisory Council, Board member (representing Genentech) of the National Pharmaceutical Council (NPC), member of the Drug Information Association (DIA) Advisory Council of North America, past NPC Corresponding Officer and Research Committee member (representing Allergan), current member of the Academy Health Institution Council, and Foundation of Managed Care Pharmacy (FMCP) Board of Trustee. Jan is a licensed pharmacist and holds a Ph.D. in Pharmacy Administration from the University of South Carolina at Columbia. She is also a speaker and guest lecturer on topics related to pharmacoeconomics and outcomes research. Jan’s ultimate passion is “building”; she takes great pride in building organizations and fostering young, innovative-thinking, yet applied researchers. For Jan, there is no greater joy than watching these researchers flourish and making a difference to patients’ lives.
Brian O'Rourke, PhD
Brian O'Rourke Health Care Consulting Inc., 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 the Chair of the Health Technology Assessment Steering Committee and a member of the Scientific Advisory Council for 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.
16:00 - 17:00
ISPOR Forums
Health Equity in HEOR: The State of Play
Concern for health equity has been brought to the front and centre of policy agendas across the world by the Covid-19 pandemic and renewed racial justice movements. Unfair social differences in health associated with social disadvantage are known variously as “health disparities” (e.g., in the US), “health inequalities” (e.g., in the UK), and “health inequities” (e.g., in the World Health Organisation). Over the past decade, some progress has been made in developing research methods for analysing health equity impacts and trade-offs in Health Economics and Outcomes Research (HEOR) alongside standard methods for analysing effectiveness and cost-effectiveness. In practice, however, these methods are still rarely used to inform decision making, and there are large gaps in collection of the data needed to support equity-informative HEOR. An important early step for advancing health equity in HEOR is to understand current practices – specifically, what health equity data and methods are currently used across the globe and what important knowledge gaps exist? The goal of this forum, hosted by the newly formed ISPOR Health Equity Research Special Interest Group, is to provide participants with a global perspective on the current state of play of equity-informative HEOR and to discuss future directions. Short presentations will aim to deliver an understanding of experience to date from a number of global regions (i.e., North America, South America, Europe, Southeast Asia). Following this, participants will be encouraged to contribute their own thoughts and experience on equity-informative HEOR, as well as global and regional priorities and challenges, through a moderated discussion.
Moderators
Stacey Kowal, BS, MS, MSc
Genentech, Alameda, CA, USA
Stacey Kowal currently serves as a principal researcher in Genentech’s Health Policy and Systems Research team where her work focuses on identifying and testing new methods to assess the impact of new healthcare technologies. Her background includes both US and global work to inform health technology assessment and reimbursement as well as design of public health interventions, drawing on disciplines of applied mathematics, economics, and international health policy. Stacey’s current research aims to increase focus on equity effects in health economics and outcomes research (HEOR), including integration of distributional elements into healthcare decision-making. Stacey is a Udall scholar, a Truman scholar and a Marshall scholar. She holds a BS in Mathematics from Alma College, an MSc in public health from the London School of Hygiene and Tropical Medicine and an MSc in International Health Policy and Health Economics from the London School of Economics.
Speakers
Diana Beatriz Bayani, BA, MSc
Saw Swee Hock School of Public Health, University of Singapore, Singapore, Singapore
Dana Bayani is currently a PhD student at the Saw Swee Hock School of Public Health, National University of Singapore. Her research involves exploring the use of real-world data for HTA and value-based payments, and modelling for oncology drug evaluations. Before moving to Singapore, she led the HTA Study Group, the predecessor of the HTA Unit in the Philippine Department of Health (DOH), which produces evidence to inform coverage decisions of drugs, national health insurance benefit packages and programmes. She has experience in HTA policy research, economic evaluations, cost-of-illness studies, and health financing policy research and has served as consultant for private, public, and non-profit sectors locally and overseas. Dana is trained in epidemiology, health economics, and policy with a masters degree from the London School of Economics and Political Science and the London School of Hygiene and Tropical Medicine.
Richard Cookson, DPhil
University of York, York, YOR, United Kingdom
Richard Cookson is a professor at the Centre for Health Economics, University of York. He has helped pioneer “equity-informative” methods of policy analysis, including distributional cost-effectiveness analysis; health equity indicators for healthcare quality assurance; and methods for investigating public concern for reducing health inequality. He has co-chaired international working groups on equity, worked in the UK Prime Minister’s Delivery Unit and served on various NHS advisory committees.
Manuel Antonio Espinoza, MD MSc PhD
Pontificia Universidad Catolica de Chile, Santiago, Chile
Manuel Espinoza is Associate Professor in the Department of Public Health and Chief of the Health Technology Assessment Unit, both at Pontificia Universidad Católica de Chile. He is also Visiting Honorary Fellow in the Centre for Health Economics at University of York and Consultant for the Interamerican Bank of Development. He has served as advisor and consultant for several public entities in Latin America as well as institutions such as the World Bank and World Health Organization. Manuel holds a medical doctor degree and Master in Epidemiology both from Pontificia Universidad Católica de Chile; a Master in Biostatistics from Universidad de Chile, and Master and PhD in Health Economics, both from University of York in the UK. Manuel´s work has been focused on the development of methods and processes for prioritization in health care as well as applied research in health economic evaluation.
Issue Panels and Workshops
Assessing the Value of Orphan Drugs Using Conventional Cost-Effectiveness Analysis: Is It Fit for Purpose?
Live
ISSUE: Conventional cost-effectiveness analysis (CEA; i.e., assessing pharmaceuticals through a cost per quality-adjusted life year [QALY] framework) originated from a societal commitment to maximize population health despite limited resources: a utilitarian approach that has produced pricing and reimbursement systems that generally favor health technologies for common diseases. This framework has been slow to evolve with our understanding of the impact of rare diseases, which in turn has complicated the assessment of orphan medicinal products (OMPs) meant to treat rare diseases.
OVERVIEW: Elisabeth Fenwick will moderate and will begin the panel discussion with a brief overview of conventional CEA and the context of rare diseases / OMPs. The primary limitations of conventional CEA with respect to OMPs are (1) the rigidity of the cost per QALY threshold, (2) the difficulty of fitting the patient experience of a rare disease into the QALY construct, and (3) the restricted scope of how costs and benefits are measured. Proposed approaches for addressing these shortcomings can be broadly grouped into three non-mutually exclusive categories: (1) augmenting the valuation context, (2) adjusting the valuation context, and (3) increasing the willingness-to-pay threshold for OMPs. The panel will discuss and debate the shortcomings of conventional CEA when applied to the evaluation of orphan drugs in a rare disease setting. Additionally, the panel will discuss and debate the merits and limitations of augmented and alternative approaches to conventional CEA within the aforementioned context. Lastly, the panelists will debate the future of conventional CEA in a context where pharmaceuticals are becoming more specialized and targeting increasingly less prevalent diseases and smaller patient subgroups.
Moderators
Elisabeth Fenwick, PhD
Pharmerit International, Oxford, United Kingdom
Elisabeth Fenwick is a senior director in the Modeling and Meta-Analysis team at Open Health, based in Oxford in the UK.
Liz provides scientific and strategic support to HE projects globally. She has extensive experience in economic evaluation and health economic modeling having worked in the field for over 20 years. She has worked on a variety of projects in a wide range of disease areas including oncology, respiratory, infectious diseases, cardiology, ophthalmology, and orphan diseases.
Liz has also contributed to methods in the field, in particular relating to decision analytic modeling and simulation methods, probabilistic decision analytic modeling and value of information analysis. Liz was a member of the ISPOR joint task force on good research practices in modeling and a co-author on the joint taskforce paper on uncertainty and co-chaired/co-authored the recent ISPOR task force assessing emerging good practice in value of information analysis for research decisions.
Liz has a PhD and MSc in Health Economics as well as an MSc in Operations Research and joined Open Health from ICON plc where she led the modeling team for the global HE group. Prior to her consultancy career, Liz spent over 15 years as an academic working at University of York, McMaster University, and most recently University of Glasgow.
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 150 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.
Declan Noone, MSc
European Haemophilia Consortium, Brussels, Belgium
Maarten Postma, Professor
University of Groningen, Groningen, Netherlands
Is Regulatory Acceptance of Surrogate Endpoints for CAR-Ts a Sufficient Basis for Acceptance by HTA Bodies?
Live
ISSUE: Regulators such as the FDA and EMA have approved many transformative therapies, including CAR-Ts, before efficacy on patient relevant endpoints has been demonstrated. The primary endpoint is often a surrogate – an interim endpoint anticipated to predict patient relevant outcomes.
It is difficult to draw robust conclusions about the relative benefit an intervention offers patients from surrogate endpoint data. Compounding the difficulty - the pivotal study for many CAR-Ts are single arm trials. The challenge becomes most acute during health technology assessment (HTA). Regulators need only be confident that improvements in the surrogate will translate to the
existence of patient relevant benefit, HTA bodies must establish the
magnitude of benefit to determine value.
OVERVIEW: This panel will debate whether HTA bodies should align with the regulators when assessing surrogacy data for CAR-Ts. Daniel Gladwell will moderate - briefly summarising the EMA and FDA’s considerations regarding the surrogate dependent evidence base for previously assessed CAR-Ts before posing key questions to the panel: 1) Is regulatory acceptance of surrogate endpoints for a CAR-T sufficient basis for acceptance by HTA bodies? 2) Are better methods available than those employed by regulators? 3) Are those methods feasible with the typical CAR-T evidence base? Drawing on a recent H2020 research project, Oriana Ciani will present the latest development in surrogate validation frameworks for HTA, including why they are an improvement on the regulator’s approach. As an Industry HTA expert, Adam Parnaby will outline why surrogate validation methods and criteria used by HTA bodies are challenging for CAR-Ts and how they need to shift towards the regulators’ to avoid delayed patient access. Drawing on his NICE committee experience Stephen Palmer will outline a way forward encouraging greater alignment between the methods of surrogate validation and the requirements of HTA decisions and economic models.
Moderators
Daniel Gladwell, PhD
BresMed, Sheffield, United Kingdom
Dan joined BresMed in 2011 and became a Board member in 2019. Before joining BresMed, Dan worked in R&D for Takeda Europe, leading the pre-, peri- and post-launch health economics and outcomes research (HEOR) planning for diabetes and obesity compounds. At BresMed, he has led the development of cost-effectiveness models and health technology assessment (HTA) submissions – providing strategic guidance on HEOR and market access.
Panelists
Oriana Ciani, PhD
SDA Bocconi School of Management, Milan, MI, Italy
Oriana Ciani is Associate Professor of Practice at SDA Bocconi in Milan. She holds a MSc in Biomedical Engineering from Politecnico di Milano and postgraduate degree in Healthcare Management from Bocconi University. She received her PhD from the University of Exeter with a thesis focusing on the evaluation of surrogate end points. She has been 2020 Fulbright Research Scholar at Yale School of Medicine and Yale School of Public Health. Oriana's research interests are centred on the use of evidence synthesis techniques to inform policy decisions, health technology assessment (HTA) and healthcare policies evaluation.
Stephen Palmer, PhD
University of York, York, Great Britain
Stephen Palmer is a Professor at the Centre for Health Economics (CHE), University of York. CHE was one of the world’s first research institutes dedicated to the study of the economics of health and health care and has established a leading international reputation. Stephen has over 25 years’ experience of health economic evaluation, regulatory and reimbursement processes. He has published over 175 peer-reviewed publications. His principal areas of expertise relate to the methodology and application of decision-analytic modelling and Bayesian approaches to Health Technology Assessment. He has worked closely with policy makers throughout his career and led a programme of work at CHE supporting the National Institute for Health and Clinical Excellence (NICE) between 2005 and 2019. He was also a member of the NICE Technology Appraisal Committee for 10 years and is currently a member of the NICE Decision Support Unit.
Adam Parnaby, MSc
BMS, Braine-l’Alleud, Belgium
Adam Parnaby is Senior Director, Health Policy, Strategy & Economics at Bristol Myers Squibb, specialising in health policies in Europe relating to pricing, access and HTA. He is the Vice Chair of the EFPIA Health Technology Assessment Working Group where he is the topic lead on HTA methodology. He received a B.A in Economics (1995) from Durham university and a M.Sc. in Health Economics (1997) from York. He started his career at the Health Economics Research Group at Brunel University (1998) before joining GlaxoSmithKline UK (2000). He then held Regional and Global health economics, access, pricing and policy roles at Pfizer (2007), Sanofi (2009) and Celgene (2012). His policy expertise includes HTA methods, innovative payment models, healthcare efficiency and the value of innovation.
Addressing Payment Challenges for Alzheimer’s Disease-Modifying Therapies: US and European Perspectives
Live
ISSUE: The expected value of emerging Alzheimer's Disease (AD) disease-modifying therapies may require innovative approaches. Given that the benefit may accrue long after a therapy is delivered, it will create disincentives for US-based commercial plans, and given its social sector spillovers, it may require new approaches even in European single-payer systems.
OVERVIEW: The approval of aducanumab to treat Alzheimer’s disease in the U.S. will require payers to address the inherent challenges associated with reimbursement - what is the expected value of therapy, what elements of value should be considered given the large social sector/caregiver spillovers, and how can access to these therapies be made sustainable.
In the US, commercial payers may have limited incentive to provide access to therapies of uncertain benefit to patients who may soon age into Medicare, preventing private plans from capturing much of the therapy’s benefit. In Europe, and the UK in particular, single-payer systems may face different challenges stemming from the therapy’s cost exceeding willingness-to-pay thresholds and healthcare budgets, with many of the benefits accruing to caregivers and other stakeholders. Important elements of value, such as innovation and hope, are only informally included in decision making. This panel will discuss the challenges associated in both contexts with providing timely access, assigning proper value to the therapy, and ensuring that reimbursement policies are sustainable both at the payer and societal levels.
Moderators
Adrian Towse, MA, MPhil
Office of Health Economics, London, LON, 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
Jakub Hlavka, PhD
Sol Price School of Public Policy, University of Southern California, Los Angeles, CA, USA
Jakub Hlávka, PhD, is a Research Assistant Professor in the Health Policy and Management Department of the Price School of Public Policy and Schaeffer Center for Health Policy & Economics, University of Southern California. His NIH-funded research focuses on the modeling of dementia treatments and associated economic challenges, with a specific focus on Alzheimer’s disease and emerging disease-modifying therapies. He has also worked on models of COVID-19 pandemic interventions estimating their costs and benefits. His broader research interests include innovative payment models for pharmaceuticals, health system reform and the study of inequality.
He currently serves as a research consultant for the National Academies of Sciences, Engineering, and Medicine (Committee on Improving Representation of Women and Underrepresented Minorities in Clinical Trials and Research), and is a member of the International Pharmacoeconomics Collaboration on Alzheimer’s Disease (IPECAD). Prof. Hlávka has additional professional experience from Genentech where he assisted in R&D portfolio planning and as a consultant to the Tufts Medical Center's Center for the Evaluation of Value and Risk in Health where he studied oncology-specific outcome measures.
Dr. Hlávka holds a PhD from the Pardee RAND Graduate School, a master’s degree from Georgetown University, Edmund A. Walsh School of Foreign Service, and an undergraduate degree from the University of Economics in Prague.
Jason Shafrin, PhD
FTI Consulting, Los Angeles, CA, USA
Jason Shafrin, Ph.D. is a Senior Managing Director at FTI Consulting's Center for Healthcare Economics and Policy. Dr. Shafrin has over 15 years of health economics research experience serving as trusted advisor and expert to a wide variety of healthcare life sciences companies, governments and non-governmental organizations (NGOs). Dr. Shafrin is the former Director of Research at the Innovation and Value Initiative and is the Founder and Editor of the Healthcare Economist blog.
Howard Thom, BA, MSc, PhD
University of Bristol, Bristol, GLS, United Kingdom
Howard Thom is a Senior Lecturer in Health Economics at the University of Bristol and Senior Director at the consultancy Clifton Insight. His research interests are value of information, uncertainty in economic models, network meta-analysis, and R for health economics.
Rapid and Adaptive Modelling During the COVID-19 Pandemic: What We Can Learn from Population Biology
Live
ISSUE: The COVID-19 pandemic has highlighted many challenges for HEOR modelers, but these challenges are not necessarily new. The need for modeling to be responsive to fast-paced changes in technology, treatments and care delivery made accurate and timely modeling of critical outcomes difficult. Borrowing modeling theories and methods from population biology may offer a new direction for rigorous and timely value assessment.
OVERVIEW: This panel will discuss the challenges associated with existing modeling, slow adaptiveness in data collection and forecasting techniques, which are being used to support decision making during the COVID-19 global pandemic and why health decision analysis tools may have become less effective in responding to COVID-19 problems. We will consider well-established modeling solutions from population biology to determine their applicability to address future challenges of healthcare systems at multiple scales from micro level treatment implications within patients to long term impact on quality of life and societal economics. Such multi-scale modeling would represent a fundamental shift in how health and medical research are approached. Dr. Mubayi will moderate and provide an overview of the health modeling as well as a framework for thinking through the data and modeling challenges being learned during current COVID-19 pandemic. Dr. Meltzer will describe answers from ecology of infectious diseases to those challenges that may be useful to use them as an input to healthcare modeling methods. Dr. Townsend will review advanced modeling techniques from population biology that have been or could be implemented in supporting COVID-19 decision making. Finally, Dr. Hyman will describe efforts relating to assessing population dynamics tools for value assessment that can adapt in real time using continuously changing empirical information for evaluating health technologies. After 5 minutes of introduction from the moderator, each panelist will present for approx 8 minutes, reserving 30 minutes for Q/A discussion.
Moderators
Anuj Mubayi, PhD
PRECISIONheor, Los Angeles, CA, USA
Panelists
James Mac Hyman, PhD
Tulane University, New Orleans, LA, USA
Martin Meltzer, PhD
Center for Disease Control and Prevention, Atlanta, GA, USA
Lead, Health Economics and Modeling Unit (HEMU), Division of Preparedness and Emerging Infections, CDC. Led modeling teams supporting CDC’s responses to: 2009 H1N1 influenza pandemic; 2012 contaminated steroid injectable product; Ebola outbreaks in West Africa (2014-16) and the Democratic Republic of the Congo (2018-19); Zika epidemic (2016-17); Current COVID Response. Research includes: Responses to bioterrorist weapons; Economics of controlling diseases such as rabies, RSV, Japanese encephalitis, dengue, hepatitis A, meningitis, Lyme, and malaria. Published ~ 300 publications, including > 150 peer-reviewed papers + > 60 tools. Associate editor for Emerging Infectious Diseases.
Jeffrey Townsend, PhD
Yale University, New Haven, CT, USA
Professor Townsend received his Ph.D. in 2002 in organismic and evolutionary biology from Harvard University, under the advisement of Daniel Hartl. His Ph.D. was entitled "Population genetic variation in genome-wide gene expression: modeling, measurement, and analysis", and constituted the first population genetic analysis of genome-wide gene expression variation. After making use of the model budding yeast S. cerevisiae for his Ph.D. research, Dr. Townsend accepted an appointment as a Miller Fellow at the University of California-Berkeley in the Department of Plant and Microbial Biology, where he worked to develop molecular tools, techniques, and analysis methodologies for functional genomics studies with the filamentous fungal model species Neurospora crassa, co-advised by Berkeley fungal evolutionary biologist John Taylor and molecular mycologist Louise Glass. In 2004, he accepted his first appointment as an Assistant Professor in the Department of Molecular and Cell Biology at the University of Connecticut. In 2006 he was appointed as an Assistant Professor the Department of Ecology and Evolutionary Biology at Yale University. In 2013 he began to work on statistical approaches to fit mathematical models of disease spread and emergence, and to work on the somatic evolution of cancer, and was appointed as an Associate Professor of Biostatistics and Ecology & Evolutionary Biology, in 2017 he was named Elihu Associate Professor of Biostatistics and Ecology & Evolutionary Biology, and in 2018 he was appointed Elihu Professor of Biostatistics and Ecology & Evolutionary Biology.
23 Recommendations in Modelling Precision Medicine: Learning from Case Studies in Tumour Agnostic Treatments, DPYD Genotyping and Maturity Onset Diabetes of the Young
Live
PURPOSE: 1) Demonstrate solutions to challenges in health economic modelling of precision medicine (PM) using three cases: tumour-agnostic treatments for NTRK fusion-positive (NTRK+) cancers, DPYD genotyping prior to fluorpyrimidine-based chemotherapy (ToxNav) and maturity onset diabetes of the young (MODY).
2) Discuss the appropriateness of these solutions based on published recommendations for modelling PM of the HEcoPerMed consortium and handling data limitations upon EMA approval.
DESCRIPTION: PM aims to better stratify patients, enabling more targeted healthcare. While holding the promise of generating more value for patients, the assessment of PM is complicated by data limitations and complex test-treatment pathways. To address these challenges, 23 modelling recommendations were published in 2021 by the EU-funded HEcoPerMed consortium, addressing the modelling of test-treatment combinations, non-randomized controlled data, additional elements of value, premature survival data and other issues.
This workshop demonstrates the practical application of the recommendations in three case studies. The health economic model on NTRK+ cancers shows how comparative effectiveness can be estimated for single-arm basket trials and how risk of death while waiting for gene test results can be incorporated. The MODY case demonstrates the importance of patient stratification, test positioning and predictive accuracy, and the impact of detecting of inheritable mutations on relatives. The ToxNav case evaluates routine DPYD testing for personalised dosing of fluorpyrimidine chemotherapy and toxicity management. Physicians’ compliance, and clinical decisions in real-world settings are incorporated. This workshop invites responses to the modelling solutions applied in the case studies and seeks input to improve the guidance. The workshop benefits: health economists working in PM, manufacturers of PM, payers, regulatory authorities, and organisations like ICPerMed, EUnetHTA, EMA. INTERACTIVE ELEMENT We apply real-time polling to invite comments on the appropriateness of the recommendations for modelling PM, and conclude with an audience discussion on the discrepancy between EMA and payer data needs.
Discussion Leaders
Matthijs Versteegh, PhD
Erasmus University Rotterdam, Rotterdam, Netherlands
Matthijs Versteegh, PhD, is director at the institute for Medical Technology Assessment (iMTA) of Erasmus University Rotterdam
Discussants
Balázs Nagy, PhD
Syreon Research Institute, Budapest, Hungary
Graduated in Economics and Business Administration at University of Debrecen (MSc in 2000; PhD in 2009). Between 2000-2004 he worked at the Hungarian National Health Insurance Fund and the Hungarian Ministry of Health, specialized in the field of managed care and health care finance. He spent the 2003-2004 school year with a doctoral fellowship at the University of Sheffield, ScHARR, followed by a 4-year joint research fellowship at the Corvinus University of Budapest and University of Sheffield. He joined Eötvös Loránd University (ELTE) in 2009 where he was a leading lecturer of MSc in ‘Health Policy, Finance and Analysis’ program over 10 years. He completed his habilitation at ELTE in 2019. Now he is an associate professor at the Semmelweis University, Center for Health Technology Assessment. He is a honorary research fellow of the University of Sheffield. Balazs has been the member of Syreon Research Institute since 2011; he is an owner and as the head of the Modelling Division, taking the lead in economic modelling related activities. His further research interests are health technology assessment, health insurance, health policy, healthcare financing and risk adjusted capitation payment.
Maureen Rutten-van Mölken, PhD
Erasmus University Rotterdam, Rotterdam, Netherlands
Maureen Rutten-van Mölken is professor of Economic Evaluation of Innovations for Health at the Erasmus School of Health Policy & Management, Erasmus University Rotterdam. She is also scientific director of the Institute for Medical Technology Assessment (IMTA).
Her expertise includes economic evaluation studies, either alongside clinical trials or using probabilistic decision analytic modelling, and outcomes research. She has a special interest in the cost-effectiveness and the financing and payment of complex multi-faceted interventions that involve system-level changes like integrated care programs, disease management programs, hospital at home programs, outpatient rehabilitation programs, remote monitoring etc. She also has an interest in the health economics of personalized medicine.
She is the coordinator of SELFIE, a Horizon2020 project on integrated care in multi-morbidity, and contributes to Horizon2020 project HEcoPerMed that develops guidance on health economic modelling, and financing and payment strategies for personalised medicine.
Sarah Wordsworth, PhD
University of Oxford, Oxford, United Kingdom
Spotlight Session
Methods to Estimate the Value of New Antibiotics in the Context of Antimicrobial Resistance
Live
Antimicrobial resistance (AMR) is a major challenge to public health internationally. There is also widespread concern regarding the limited number of new antibiotics coming to market to provide genuine therapeutic options in the face of multi-drug resistant infections. A range of policies ha s been developed to incentivize the development of new antibiotics including various forms of public funding and payments for new products which are delinked from the volumes used. There has also been concern that the methods conventionally used in health technology assessment to estimate the value of new pharmaceuticals is too narrow for new antibiotics and this has contributed to low levels of expected revenue for manufacturers. This session will focus on the appropriate methods of value assessment for new antibiotics, the sources of evidence needed to implement them, and the challenges associated with decision making.
Moderators
Mark Sculpher, PhD
University of York, York, United Kingdom
Mark Sculpher is Professor of Health Economics and Director of the Centre for Health Economics, University of York. He is also Co-Director of the Policy Research Unit in Economic Evaluation of Health and Care Interventions, a programme of research for the UK Department of Health and Social Care funded by the National Institute for Health Research (NIHR).
Panelists
Alec Morton, PhD
University of Strathclyde, Glasgow, United Kingdom
Alec Morton is Professor and Head of Department of Management Science at Strathclyde Business School, University of Strathclyde in Glasgow, Scotland. He has degrees from the University of Manchester and the University of Strathclyde. He has worked for Singapore Airlines, the National University of Singapore, and the London School of Economics, has held visiting positions at Carnegie Mellon University in Pittsburgh, Aalto University in Helsinki, the University of Science and Technology of China (USTC) in Hefei, and the National Audit Office and is a member of the International Decision Support Initiative. His main interests are in decision analysis and health economics. His research is funded by the European Commission, the Department of Health, the Medical Research Council and Engineering and Physical Sciences Research Council, and the Chief Scientist's Office of the Scottish NHS.
Alec has been active in the INFORMS Decision Analysis Society, EURO and ISPOR. He is on the Editorial Board of Decision Analysis and is an Associate Editor for the EURO Journal on Decision Processes, the Transactions of the Institute of Industrial Engineers, and OR Spectrum. Past consulting clients include the National Audit Office, the Department of Health, the Environment Agency, the Nuclear Decommissioning Authority and the Global Fund to Fight AIDS, Tuberculosis & Malaria. His papers have won awards from the International Society for Pharmacoeconomics and Outcomes Research and the Society for Risk Analysis. His book Portfolio Decision Analysis with Jeff Keisler and Ahti Salo won the INFORMS Decision Analysis Society publication award in 2013 and his paper "CUT: A Multicriteria Approach for Concavifiable Preferences" (with Nikos Argyris and Jose Figueira) was a finalist for the same prize in 2016 .
John Rex, MD
F2G Ltd., Advent Life Sciences, Wellesley Hills, MA, USA
John H. Rex, MD, FACP
Dr. Rex is a physician and drug developer with more than 30 years of development and policy experience focused on antimicrobial agents. He is currently CMO for F2G, Ltd. (an antifungal biotech), is an operating partner with a venture capital group (Advent Life Sciences), is Chair of the Scientific Advisory Board of the $1b AMR Action Fund, and was (2015-2019) a voting member on the US Presidential Advisory Council on Combating Antibiotic Resistant Bacteria (PACCARB). He also blogs regularly at http://amr.solutions/blog.html.
His experience includes moving compounds from preclinical development through all development phases via academic positions (NIH, Bethesda, MD; McGovern Medical School-Houston) and VP-level roles at a multinational pharmaceutical firm (AstraZeneca). Other past activities include advancing novel regulatory paradigms for antibacterials, publications on novel reimbursement models for antibiotics, co-founding of a public-private partnership (CARB-X), co-founding the New Drugs for Bad Bugs (ND4BB) program of Europe’s Innovative Medicines Initiative (IMI), and a 4-year term as Industry Representative on the FDA Anti-Infective Drugs Advisory Committee (AIDAC, 2007–2011).
Beth Woods, MSc
University of York, York, United Kingdom
Beth is a Senior Research Fellow at the Centre for Health Economics (CHE), University of York. Prior to joining CHE Beth was a Director in the Health Economics team at Oxford Outcomes, a private consulting firm, where she specialised in applied economic evaluation from 2006-2013. Beth holds a BA in Economics from the University of Cambridge and an MSc in Economic Evaluation in Healthcare from City University. Beth’s research interests include drug pricing, the economic evaluation of interventions to combat AMR and the use of statistical and decision analysis to support reimbursement decision making.
17:00 - 18:00
ISPOR Women in HEOR Social Hour
Live
Meet and continue the conversation with today’s Women in HEOR panelists at this Social Hour, and network with your fellow peers and those who support the advancement of women in the field. This social hour is hosted by the ISPOR Women in HEOR Initiative and is intended to be lively, interactive, and open to both men and women.
Thu 2 Dec
11:00 - 12:00
ISPOR Forums
Current Controversies in Rare Diseases and Evidence-Based Advocacy
The purpose of this forum is two-fold: firstly, describe the process used to identify current controversies in rare diseases conducted to inform the future strategy of the ISPOR Rare Disease Special Interest Group (SIG); secondly, to present the outline of the Key Project scheduled by the Rare Disease SIG, on the development of guidelines for ‘Evidence-Based Advocacy’ (EBA). The Rare Disease SIG will present the results of a consultation process informing the future strategy of the SIG and priority research. Various stakeholders in the area will be consulted, through semi-structured interviews, with four main areas of interest: Regulatory, Health Economic Evaluation & Equity, Data Collection, and EBA. The interviews will elicit the views of the stakeholders on challenges in Rare Diseases and key areas of research. The results will be presented at the forum with an aim to have a dialogue between the panelists and the audience, to identify the medium-term research direction and strategic planning of the SIG, as well as Member Engagement and Key Projects for the SIG. The Rare Disease SIG has already scheduled a Key Project, on EBA. There is no consensus or consistency in terms of how Patient Advocacy Groups (PAGs) can best illustrate unmet need/burden of their respective beneficiary populations. Given the increasingly important role of PAGs (particularly in the Rare Disease space) the purpose of this project is to generate a set of guidelines to empower PAGs to better represent their beneficiaries. The focus will be on evidence generation to limit the welfare losses associated with incomplete information, delivery of healthcare in a more equitable way and use of evidence-based research to advocate for health policy shaping. A multi-disciplinary Working Group of experts will develop a set of guidelines to be published and made freely available to PAGs.
Moderators
Jamie O'Hara, MSc
HCD Economics, The Innovation Centre, Chesire, United Kingdom
Jamie is an industry economist with expertise in the application of economic/econometric methods and in identifying optimal research methodologies. He has worked with a range of organisations across the public, private and third sectors, as well as with charities. Jamie has provided specialist HEOR advice in the development of multi-billion-dollar market access initiatives for various pharmaceutical companies. This has primarily included the development of evidence to identify and quantify patient unmet need/burden. Jamie is currently CEO of HCD economics and holds a senior position in the University of Chester and leads a team of 50 researchers.
Speakers
Mohit Jain, PhD, MBA
BioMarin Europe Ltd, London, SRY, United Kingdom
Mohit is responsible for Market Access in BioMarin, leading the EMEA region, coordinating between the global regional teams and heading BioMarin’s Centres of Excellence for HEOR and Patient Outcomes. BioMarin is a company focused on developing life transforming therapies for rare disease patients with a number of gene therapies in development. Mohit has been with BioMarin for ten years and prior to this worked in other companies including large pharma and consulting. By background a scientist and then worked in corporate finance before finding a calling in Market Access. Mohit is married with two lovely girls aged 11 and 13 years, occasionally trying to fit in a run.
Persefoni Kritikou, PhD
HCD Economics, Daresbury, United Kingdom
Persefoni is a health economist with working experience of more than 10 years in both pharmaceutical and consultancy companies, in the UK, Sweden, and Greece. She has worked across various activitites in pharmacoeconomics (from cost-effectiveness analysis to health technology assessment) and across the whole spectrum of real-world evidence (from study conceptualization to study development and analysis). She has a great interest in Rare Disease research and is currently a Co-Chair to the ISPOR Rare Disease Special Interest Group.
Brian O'Mahony, FIBMS, FACSLM, FIOSH, DMLM, Dip SHWW
Irish Haemophilia Society, Dublin, D, Ireland
Sheela Upadhyaya, MSc
NICE - National Institute for Health and Care Excellence, London, LON, United Kingdom
Issue Panels and Workshops
Open Source Health Economics and Outcomes Research: Why and How to Do It? How to Connect with Users?
Live
PURPOSE: The objectives of this workshop are to introduce the concepts of open source models (OSMs) to the audience, to discuss the motivations and challenges in developing OSMs, and to demonstrate how to share them with key stakeholders.
DESCRIPTION: Health-economic models are routinely developed to inform health policy decisions. However, they are often considered to be black boxes because the reporting of the models’ structure, assumptions, input data, and source code lacks transparency. Open source models (OSMs), health-economic models freely available for anyone to use, review, and modify, have been proposed as a solution to increase health-economic models’ transparency.
This workshop will start with an introduction of the concepts of OSMs. The importance of certain aspects of surrounding the development, maintenance, and use of OSMs will also be illustrated based on results of a recent survey on OSMs amongst ISPOR members (10 minutes, Pouwels). An example of a developed OSM in R, comparing anticoagulants for stroke prevention in atrial fibrillation, will be showcased. Challenges associated with making health-economic models open source, such as greater scrutiny and possible misuse by vested interests, will be discussed (13 minutes, Thom). The workshop will continue by elaborating on the “why and when” to develop OSMs based on factors such as the intended audience, complexity of the model, and considering commercial sensitivity(13 minutes, Hart). Afterwards, ways to make HE models accessible for a broader audience will be presented based on the experiences of the Peer Models Network (13 minutes, Harvard). Dr. Harvard will provide a tour of the Peer Models Network website, Twitter, and YouTube pages, and present the results of the Peer Models Network evaluation survey. The session will be concluded by a discussion between audience and panel members (11 minutes). This session may benefit modellers and policymakers intending to develop, commission, or use OSMs.
Discussion Leaders
Xavier Pouwels, PhD
Department of Health Technology & Services Research, University of Twente., Apeldoorn, GE, Netherlands
Xavier is currently working at the Department of Health Technology & Services Research. His research interests are the use of simulation models to evaluate the value of new health care technologies and how to improve the transparency of health economic models. Currently, Xavier is involved in research projects focusing on the early detection of (risk factors for) cardiovascular diseases.
Discussants
Rose Hart, PhD
BresMed Health Solutions Ltd., Sheffield, United Kingdom
Rose Hart is a Senior Health Economist at BresMed, where she was a member of the Health Economics Analysis Team for 4 years before joining the Advanced Analytics Team. She is experienced in developing health economic models for early-phase cost effectiveness and for health technology assessment. As a specialist in R modelling, with and without Shiny interface functionality, she is responsible for internal Shiny training and led in developing BresMed’s intRface™ services and pilot model. Rose previously worked in the BioPharm team at GlaxoSmithKline, and researched for her PhD at the University of Sheffield.
Howard Thom, BA, MSc, PhD
University of Bristol, Bristol, GLS, United Kingdom
Howard Thom is a Senior Lecturer in Health Economics at the University of Bristol and Senior Director at the consultancy Clifton Insight. His research interests are value of information, uncertainty in economic models, network meta-analysis, and R for health economics.
Precision Healthcare: How Big Data and AI Technology Can Help
Live
PURPOSE: This workshop will discuss how real world data and predictive analysis can improve precision healthcare from different perspectives in different countries.
DESCRIPTION: Background: A 10-minute introduction by Dr. Yang will have an overview of data analytics and AI/machine learning -based methods that are increasingly adopted in the medical field to predict population health outcomes and improve personalized health care delivery. The proliferation of these techniques has enabled researchers to analyze patients’ information collected from electronic medical records or other data sources and develop predictive models to forecast patients’ disease progression and outcomes. New technologies can also turn the model into tools to facilitate personalized decision making in real time.
Three case studies each for 10 minutes will be presented as follows: a) Predictive models in the Tulane health system to manage diabetic patients through identifying the high risk patients and implementing the ADA’s Diabetes Inside program; b) A predictive model to identify candidiasis timely among systemic inflammatory response syndrome (SIRS) patients in intensive care unit (ICU) in China; c) A predictive model on mortality of COVID-19 patients using machine learning methods and real world data in China and then being deployed in Brunei to help their government manage the pandemic more efficiently. The workshop will benefit HEOR researchers, health care providers, stakeholders (health systems and payers) for population health management and personalized medicine. Audience Interactive Element:
Two 20-minute breakout rooms will be used for presenting demos for case studies to encourage audience participation. Audience will also be encouraged to brainstorm on other potential applications of predicative models on improving precision healthcare using machine learning and real world evidence.
Discussion Leaders
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.
Discussants
Yun Long, M.D. Ph.D.
Peking Union Medical College Hospital, Beijing, China
Dr. Yun Long is the Director of the Department of Critical Care Medicine at Peking Union Medical College Hospital. He will discuss a predictive model used to identify Candidiasis infection in patients with systemic inflammatory response syndrome (SIRS) to improve outcomes for patients in the ICU in China.
He received his Ph.D. in emergency medicine from Peking Union Medical College in 2005. He has worked as a resident and physician at Peking Union Medical College Hospital for over 20 years. His most recent positions include professor physician and vice director of the Department of Critical Care Medicine. He is currently the director of the department.
Dr. Long has a long-term commitment to severe respiratory, severe Hemodynamics, severe patients with mechanical circulatory support, severe infections and other fields of clinical and scientific research. He is the first to bring the concept of “Circulation protective mechanical ventilation”in China.
Dr. Long is responsible for and involved in 11 national-level and provincial-level research projects and published dozens of papers including 6 SCI papers.
In the past years, Dr. Long has participated in the rescue and treatment of public health emergencies throughout the country and won the title of “Outstanding Individual in the fight against Ebola”awarded by the National Health and Family Planning Commission in the fight against Ebola virus in Guinea.
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.
Justin Wong, Ph.D.
Ministry of Health, Bandar Seri Begawan, Brunei
Dr Justin became the Medical Superintendent (MS) Public Health in 2016. He headed the Disease Control Division, Ministry of Health before being MS and prior to that he had many other placements inside the Public Health arena in the UK such as at the Public Health England, Health Protection Agency and Suffolk County Council.
Justin is a member of the Fellowship of the Faculty of Public Health of the Royal College of Physicians (2014) and has a GMC Certificate of Completion of Training (CCT) in Public Health Medicine. He also has other appointments under both local and international committees relating to his field. These include appoints at WHO Guidelines Development Group for the Programmatic Management of Drug-Susceptible (2016), WHO Task Force on Addressing Ethical Issues in TB Care and Control (2015) and Honorary Teaching Fellow at Norwich Medical School, University of East Anglia (2012-2014).
He obtained both his Bachelor of Science in Immunology and Pathology (B. Sc.) (2005) and his Bachelor of Medicine and Bachelor of Surgery (MBBS) (2007) from Imperial College London. He continued to pursue a Master of Philosophy in Public Health (M. Phil.) from the University of Cambridge in 2010. He has also have made a number of publications which has been published in various journals, some notable ones are Egan M, Wong J, Lock K. Use of retail data in the assessment of natural experiments: the case of Reducing the Strength, an intervention to reduce alcohol availability. The Lancet 2014, 384; S30 and Wong J, Robin L, Bagade A. How can we measure and monitor system performance in hospitals? The case for an evaluation toolkit. The Network Casebook 2012, 1; 45-48.
Can We Get More Value From Scientific Knowledge Spillovers? The Under-Appreciated Value of “Failed” R&D Efforts
Live
PURPOSE
: The purpose of this workshop is to illustrate the value of scientific knowledge spillovers (SKS) using failed R&D efforts as specific examples, recognizing that they may be undersupplied and to discuss the potential policy solutions to encourage more generation and use of SKS using the example of information-sharing from failures.
DESCRIPTION
: Recent experience with COVID-19 has reminded us of the importance of scientific progress in enabling pharmaceutical innovation. Developing novel therapies is a highly risky but rewarding process: it not only produces innovative drugs, but also valuable scientific knowledge that benefits the community of innovators. This workshop explores whether the existing reward system for pharmaceutical R&D leads to socially optimal level of knowledge generation and sharing, with a particular focus on the value of “failed” R&D efforts. Prof. Garrison will first discuss the concept of SKS and its importance in health technology assessment (10 minutes). Dr. Xie will describe, with examples, channels and influence of SKS throughout the new drug development process (10 minutes). Prof. Magazzini will focus on the value of “failed” R&D efforts, and how the community of innovators can learn from the “failure” of each other (15 minutes). Lastly, Prof. Towse will discuss policy approaches and implications to incentivize more information sharing from “failed” R&D efforts and innovation efforts (15 minutes). Insights from the workshop are applicable across all stakeholders: researchers will gain insights into available methods and data to evaluate SKS and future research opportunities; innovators and patients can learn more about the importance of information-sharing and collaboration in R&D process; and payers and HTA bodies can learn about promising approaches and suggested next steps for integrating SKS into decision-making.
Discussion Leaders
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 150 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.
Discussants
Laura Magazzini, PhD
Sant’Anna School of Advanced Studies, Pisa, Italy
Laura Magazzini is Associate Professor in Econometrics at the Institute of Economics of the Sant’Anna School of Advanced Studies and EMbeDS (Economics and Management in the Era of Data Science).
Her research interests are in the fields of industrial organization, economics of innovation, microeconometrics, competition policy and econometric methods. She has long-established experience in the analysis of microeconomic data, with a focus on the dynamics of innovation and competition that characterize the Life Science industry. With regards to econometric methods, her interests focus on estimation of linear (and non linear) panel data models both in a static and dynamic framework, limited dependent variable models, and on simulation-based estimation methods.
Laura has published in international peer-reviewed journals such as: Nature Reviews Drug Discovery, The Journal of Health Economics, The Economic Journal, Management Science, Research Policy, Economics Letters, Econometric Reviews, Journal of Evolutionary Economics, The World Economy, The European Journal of Health Economics, Empirical Economics.
Adrian Towse, MA, MPhil
Office of Health Economics, London, LON, 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.
Richard Xie, PhD
The Innovation and Value Initiative, Newton, MA, USA
Richard Xie, is a health economist and serves as Health Economics and Outcomes Research (HEOR) Manager at the Innovation and Value Initiative. He has a track record of conducting HEOR studies to inform health policy design and valuation of pharmaceutical therapies. Formerly he was a consultant at the Analysis Group, managing a portfolio of studies to demonstrate the value of novel therapies for pharmaceutical clients. Xie was also a health policy researcher at the Leonard D. Schaeffer Center for Health Policy and Economics at the University of Southern California, examining policy questions in medication non-adherence, health disparities, and healthcare innovation evaluation. His work has been published in journals including Journals of the American College of Cardiology, Journal of General Internal Medicine, PharmacoEconomics, and PLOS One. Xie earned his doctorate in economics from the University of Southern California.
Do Single-Arm Trials Bring Peril to Network Meta-Analyses (NMAs)?
ISSUE: The growing number of highly specialized products, including cell-based and gene therapies, is an accelerating, likely permanent trend in drug development. These products often achieve regulatory approval based on single-arm trials due to ethical considerations or difficulty of recruiting patients. HTA agencies must then assess highly specialized products based on single-arm pivotal trials alongside historical study data or prospective local real-world evidence (RWE) on standard care. Several manufacturers have already supported regulatory and HTA filings using single-arm trials alongside historical trial data and/or prospective RWE.
This development challenges the established role of NMAs in HTA processes. NMAs are a core tool to establish comparative value of healthcare interventions, both from clinical and societal perspectives, given that it is not feasible to conduct head-to-head studies for every treatment. HTA bodies rely on NMA techniques to derive necessary estimates of comparative effectiveness for use in economic models. The question therefore arises regarding the outlook for use of NMAs in determining the comparative effectiveness for highly specialized products given the growing use of single-arm trials.
OVERVIEW: Dr Naci will introducing the topic and present current and evolving approaches to comparative effectiveness research, including those relevant to single-arm pivotal trials (10 min). Next, the panelists will present their perspectives (10 min each). Dr. Dawoud will argue for the continuing place of NMAs and the evolution of innovative methodological approaches, including those relating to using single-arm trials. Dr. Heeg will argue that single-arm trials can be acceptable for HTAs under certain conditions, including that the RWE control arm must be considered in the single-arm trial protocol. Dr. Cappelleri will argue for the necessity of approving highly specialized products based on single-arm trials to expedite access. Next, panelists or the moderator will pose challenges to begin the debate section (10 min). General Q&A will follow (10 min).
Moderators
Huseyin Naci, PhD
London School of Economics and Political Science, London, LON, United Kingdom
Huseyin Naci (M.H.S., Ph.D.) is Associate Professor of Health Policy at the London School of Economics. He conducts research and teaches on health care policy and practice in Europe and the US.
Huseyin’s research to date has evaluated the quality and quantity of the evidence base underpinning the approval, adoption, reimbursement, and use of new drugs and devices in Europe and the US. His research has appeared in leading medical and health policy journals, including JAMA, The Lancet, The BMJ, Health Affairs, and The Milbank Quarterly.
Huseyin has a Ph.D. from the Department of Social Policy at the LSE and a Master’s in Health Sciences from the Johns Hopkins Bloomberg School of Public Health. He was previously a Fellow in Pharmaceutical Policy Research (2012-13) and a Pyle Fellow in Population Medicine (2013-14) at the Harvard Medical School and Harvard Pilgrim Health Care Institute. In 2018-19, he was the recipient of the UK Harkness Fellowship in Health Care Policy and Practice, based at the Harvard Kennedy School of Government.
Panelists
Joseph Cappelleri, MS, MPH, PhD
Pfizer Inc, Newington, CT, USA
Joseph C. Cappelleri is executive director of biostatistics at Pfizer Inc. The most published author in the history of Pfizer, and among the most prolific in the pharmaceutical industry, he has co-authored approximately 1,200 external presentations and 600 publications on clinical and methodological topics, including on regression-discontinuity designs, meta- analysis, and health measurement scales. He earned his MS in statistics from the City University of New York (Baruch College), PhD in psychometrics from Cornell University, and MPH in epidemiology from Harvard University. Dr. Cappelleri is the 2021 recipient of the ISPOR Avedis Donabedian Outcomes Research Lifetime Achievement Award.
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.
Bart Heeg, PhD
Ingress-health, Rotterdam, Netherlands
1. Muresan B, Mamolo C, Cappelleri JC, Leip E, Viqueira A, Heeg B. An indirect comparison between bosutinib, nilotinib and dasatinib in first-line chronic phase chronic myeloid leukemia. Curr Med Res Opin. 2021 May;37(5):801-809.
2. Munshi NC, Avet-Loiseau H, Anderson KC, Neri P, Paiva B, Samur M, Dimopoulos M, Kulakova M, Lam A, Hashim M, He J, Heeg B, Ukropec J, Vermeulen J, Cote S, Bahlis N. A large meta-analysis establishes the role of MRD negativity in long-term survival outcomes in patients with multiple myeloma. Blood Adv. 2020 Dec 8;4(23):5988-5999.
3. Hungria V, Martínez-Baños DM, Mateos MV, Dimopoulos MA, Cavo M, Heeg B, Garcia A, Lam A, Machnicki G, He J, Fernandez M. Daratumumab Plus Bortezomib, Melphalan, and Prednisone Versus Standard of Care in Latin America for Transplant-Ineligible Newly Diagnosed Multiple Myeloma: Propensity Score Matching Analysis. Adv Ther. 2020 Dec;37(12):4996-5009
4. Sbarigia U, Vincken T, Wigfield P, Hashim M, Heeg B, Postma M. A comparative network meta-analysis of standard of care treatments in treatment-naïve chronic hepatitis B patients. J Comp Eff Res. 2020 Oct;9(15):1051-1065
5. Mateos MV, San-Miguel J, Goldschmidt H, Sonneveld P, Dimopoulos MA, Heeg B, Hashim M, Deraedt W, Hu P, Lam A, He J. The effects of different schedules of bortezomib, melphalan, and prednisone for patients with newly diagnosed multiple myeloma who are transplant ineligible: a matching-adjusted indirect comparison. Leuk Lymphoma. 2020 Mar;61(3):680-690.
6. Dimopoulos MA, Cavo M, Mateos MV, Facon T, Heeg B, van Beekhuizen S, Gebregergish SB, Nair S, Pisini M, Lam A, Slavcev M. A matching-adjusted indirect treatment comparison (MAIC) of daratumumab-bortezomib-melphalan- prednisone (D-VMP) versus lenalidomide-dexamethasone continuous (Rd continuous), lenalidomide-dexamethasone 18 months (Rd 18), and melphalan-prednisone-thalidomide (MPT). Leuk Lymphoma. 2020 Mar;61(3):714-720.
7. Cortes JE, Muresan B, Mamolo C, Cappelleri JC, Crescenzo RJ, Su Y, Gambacorti-Passerini C, Heeg B, Douglas Smith B. Matching-adjusted indirect comparison of bosutinib, dasatinib and nilotinib effect on survival and major cytogenetic response in treatment of second-line chronic phase chronic myeloid leukemia. Curr Med Res Opin. 2019 Sep;35(9):1615-1622.
How and When Should Evidence From Patient Preference Studies Be Integrated Into HTA: Aligning Methodological, Agency and Industry Perspectives
Live
ISSUE: Guided by a recently developed conceptual framework, the panel will discuss how and when quantitative measures of patient-preferences (PP) should be integrated into health technology assessment (HTA). Specifically, the panel will discuss the following uses of PP in HTA: endpoint selection, estimating minimum required clinical benefit, predicting uptake, supporting economic evaluation, and providing weights for a multi criteria decision analysis (MCDA).
OVERVIEW: ISPOR’s Science Strategy contains a commitment to improve the usefulness of PP in HTA. This has been the focus of recent ISPOR panels. However, these debates have been challenged by the multiple and overlapping insights generated by applications of PP. A recently published framework can facilitate this debate by identifying distinct HTA PP use cases: selection of endpoints, estimating minimum required benefit, predicting uptake, estimating utility, estimating willingness to pay, and addressing distributional considerations.
Using this framework to structure the discussion, the panel will assess how and when PP should be used in HTA and whether methodological, HTA agency and industry perspectives align on this topic. Before the panel, the moderator will collect the panelists’ perspectives, identify areas of agreement and disagreement. During the panel, the moderator will introduce the framework and the starting positions of the panelists (10 mins). The audience will be invited to vote on uses of PP (5 mins). Focusing on the areas of disagreement, the panelists will then discuss the use cases, representing different perspectives – methodological (10 mins), HTA agency (10 mins) and industry (10 mins). The discussion will include: recent experiences of industry-HTA agency interaction on PP studies; how PP should be weighed against or integrated with other evidence, obstacles to using PP and how these might be overcome. Finally, the audience will be invited to vote again and contribute to the discussion (15 mins).
Moderators
Kevin Marsh, PhD
Evidera, London, United Kingdom
Kevin Marsh, PhD, is Vice President at Evidera in London, UK. He specializes in the use of preference data and decision analysis to inform health decisions, including pipeline optimisation, authorisation, reimbursement, and prescription decisions.
Dr Marsh’s research interests include stated and revealed preference methods, decision modelling, and MCDA. He has applied these and other research techniques for a range of organisations, including both regulatory and industry clients. He actively contributes to the methodological development of these techniques. He is currently co-Chairing ISPOR Health Preference SIG and is a member of the ISPOR Task Force on qBRA. He has previously chaired the ISPOR Task Force on the Use of MCDA in Health Care Decision-Making and an ISPOR working group on the use of preference data in Europe.
Panelists
Irina Cleemput, PhD, MSc
Belgian Health Care Knowledge Centre (KCE), Brussels, Belgium
Irina Cleemput is Scientific Programme director at the Belgian Health Care Knowledge Centre (KCE). She graduated as PhD in Health Economics at the KULeuven in 2003. On behalf of KCE, Irina co-leads the work package on recommendations and chairs the stakeholder advisory group for HTA bodies and payers in PREFER (international study about incorporating patient preferences in the medical product development life cycle). Her main areas of research are currently patient needs assessment and patient involvement in HTA.
Andrii Danyliv, PhD
Novartis Pharma AG, Basel, 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.
Esther de Bekker-Grob, PhD
Erasmus University Rotterdam, Rotterdam, ZH, Netherlands
Esther de Bekker-Grob, PhD, is Professor of Health Economics and Health Preferences in the Erasmus School of Health Policy & Management at Erasmus University Rotterdam. Additionally, she is co-director of the Erasmus Choice Modelling Centre and co-director of the Health Policy Evaluation lab. She is internationally known for her stated-preference research, both methodological and applied contributions for a broad range of medical topics in primary healthcare, clinical care and public health. She currently serves as a board member of the International Academy of Health Preference Research (IAHPR). She holds several prestigious personal grants.
Challenges in Quantifying the Value of Digital Therapeutics: New Challenges to Bridge the Efficacy-Effectiveness Divide
Live
ISSUE: COVID-19 has highlighted the value of new digital technologies, which—in the case of digital therapeutics—have the potential to replace pharmaceuticals as a treatment for certain conditions. Digital therapeutics offer much promise: they allow better tracking real-world treatment use, permit for direct communication on treatment progress with clinicians, and the user experience can be improved over time. At the same time, digital therapeutics introduce new challenges: clinicians may not be able to distinguish treatments with high vs. low quality evidence, provider time reviewing digital therapeutics output may not be reimbursed, and there are data privacy concerns. What regulatory, payer and institutional policies need to be enacted to ensure that clinical trial results translate into the real-world?
OVERVIEW: This panel aims to identify barriers to and opportunities for maximizing the effectiveness of digital therapeutics in the real world. Dr. Shafrin will moderate the panel, providing a brief overview of some recent digital therapeutics and some of the key challenges and opportunities. Dr. Schueller will provide the academic and policy perspective, describing the unmet need for digital solutions in mental health, the need for regulatory clarity, reimbursement needs, and his digital mental health initiative known as the Day One Project. Dr. Cañadas will represent the perspective of the digital therapeutics’ manufacturer, describing opportunities where digital therapeutics can improve on pill-based treatment, as well as exploring challenges her company has faced. Finally, Dr. Burton will provide the employer and payer perspective, describing the type of evidence that would help support the case that a given digital therapeutic will be effective in the real world.
Moderators
Jason Shafrin, PhD
FTI Consulting, Los Angeles, CA, USA
Jason Shafrin, Ph.D. is a Senior Managing Director at FTI Consulting's Center for Healthcare Economics and Policy. Dr. Shafrin has over 15 years of health economics research experience serving as trusted advisor and expert to a wide variety of healthcare life sciences companies, governments and non-governmental organizations (NGOs). Dr. Shafrin is the former Director of Research at the Innovation and Value Initiative and is the Founder and Editor of the Healthcare Economist blog.
Panelists
Wayne Burton, MD
American Express, Hinsdale, IL, USA
Wayne N. Burton, M.D. FACP FACOEM
Dr. Wayne Burton is a strategic advisor and healthcare consultant. Previously he was the Corporate Medical Director for American Express from 2009 to 2017 and the Corporate Medical Director for JPMorgan Chase and its legacy Banks from 1982 to 2009.
He has been the recipient of several awards including the Adolph G. Kammer Merit in Authorship Award from the American College of Occupational and Environmental Medicine (ACOEM), the Jonas Salk Health Leadership Award from the March of Dimes and the Mark Dundon Research Award from the Health Enhancement Research Organization (HERO).
Dr. Burton is Board Certified in Internal Medicine and is Associate Professor of Clinical Medicine, Feinberg School of Medicine, Northwestern University and Adjunct Professor of Environmental & Occupational Sciences at the University of Illinois at Chicago.
He is co-editor of the International Journal of Health and Productivity. He has co-authored over 100 peer reviewed medical journal articles and book chapters on employee health and productivity and disease management,
Megan Coder, PharmD, MBA
Digital Therapeutics Alliance, Arlington, VA, USA
Megan Coder, PharmD, MBA, is Vice President of Global Policy with the Digital Therapeutics Alliance (DTA), an international non-profit trade association of industry leaders and stakeholders dedicated to improving clinical and health economic outcomes through the use of high quality, evidence-based digital therapeutics. DTA’s membership spans 15 countries across four major healthcare industries.
Trained as a pharmacist, Megan graduated from the University of Wisconsin—Madison and completed an Executive Residency in Association Management & Leadership with the American Pharmacists Association Foundation. Prior to joining DTA, Megan worked with Voluntis, Iodine, the Pharmaceutical Care Management Association, and the Pharmacy Technician Certification Board.
Stephen Schueller, PhD
University of California, Irvine, Irvine, CA, USA
Stephen Schueller, PhD, is an Associate Professor of Psychological Science and Informatics at the University of California, Irvine. As a clinical psychologist and mental health service researcher his work broadly looks creating more scalable mental health resources that make treatment more available and accessible, especially with technology. This includes the development, evaluation, and implementation of web- and mobile-based interventions. He also serves as the Executive Director of One Mind PsyberGuide, a project the aims to empower consumers to make informed choices around digital mental health products.
11:00 - 17:00
Poster & Exhibit Viewing
Live
Stop in to learn about the latest technology, services, and devices and to connect one-on-one with exhibitors and sponsors during our Exhibit Viewing Hours. Be sure to reserve some time to view the latest research in HEOR through our virtual poster gallery. Browse, comment, and discuss our posters at any time while our virtual platform is active.
12:30 - 13:30
ISPOR Forums
ISPOR Machine Learning Methods in HEOR Emerging Good Practices Task Force Forum: Is ML Ready for Prime Time?
The ISPOR Machine Learning Methods Emerging Good Practices Task Force has developed guidance for HEOR and decision-makers in the use of machine learning (ML) methods. The report considers six applications of ML methods that are important to HEOR: (1) machine learning-assisted cohort selection; (2) feature selection; (3) predictive analytics; (4) causal inference; (5) health economic evaluation; and reflection on (6) ethics and transparency. The task force’s goal is to introduce ML methods and their value in conducting research on health economics, as well as patient- and system-level outcomes research to the ISPOR audience and to describe problems for which ML methods are appropriate with particular attention to four major content areas: the prediction of risk of various health care events; the causal estimation of treatment effects; developing models for economic evaluation; and model/data transparency. Dr. Crown will moderate the session with Dr. Kreif discussing the difference in using ML for prediction / classification versus causal inference. Her examples will illustrate the use of ML for special populations (personalized medicine and the identification of patient clusters) and its value for identifying potentially beneficial treatments for COVID-19, rather than waiting for the results of randomized trials. Dr. Jonsson will discuss the payer perspective on the use of evidence generated by ML with particular focus on issues of transparency--both with respect to the black box nature of some ML algorithms, and clarity in how the analyses were conducted. He will also introduce the PALISADE Checklist. ML is a very powerful tool for exploring data but when does the exploration end and the analysis begin? Dr. Felizzi will discuss a case study of improving the prediction of disease progression with health systems implications and optimal delivery of care. Polling will be used during the presentations and for feedback on the task force’s recommendations.
Moderators
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.
Speakers
Federico Felizzi, PhD
Novartis, Basel, BS, Switzerland
Federico Felizzi is a Global HEOR Director at Novartis. Previously, e served as HTA Statistician, Health Economic Modeler and HTA Evidence Lead at Roche. In this role he contributed to the submission of new interventions to HTA assessors in key markets. Through thought partnerships he helped co-build evidence packages with a strong focus on outcomes certainty. Prior to joining Roche, he worked as Statistician in Technical R&D at Novartis. In this role, he co-worked with formulation scientists and analytical chemists to assess and quantify the technical risks of transforming a drug substance into a drug product.
He holds a Degrees in Mathematics a PhD in Systems Biology from ETH Zurich and an MBA at IE Business School.
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.
Noemi Kreif, PhD
Centre for Health Economics, University of York, York, United Kingdom
Noemi Kreif is a research fellow at the Centre for Health Economics, at the University of York. She holds a PhD in Health Economics from the London School of Hygiene and Tropical Medicine and she has recently held a UK Medical Research Council Early Career Fellowship in the economics of health, on statistical methods to address confounding in economic evaluation. Her methodological work includes translating advanced causal inference methods to health economic evaluation and decision modelling, in the complex settings of time-varying confounding, and more recently, for the economic evaluation of health system level interventions.
Issue Panels and Workshops
Novel Approaches to Identify and Quantify Patient Input for Health Technology Assessment (HTA)
Live
PURPOSE
: The purpose of this workshop is to demonstrate novel methods to systemically identify and quantify patient input to inform HTA.
DESCRIPTION
: Existing methods (e.g., economic models) used to inform HTA usually fail to capture a comprehensive set of clinical and economic outcomes of importance to patients or consider value elements that influence patient decision-making. There are gaps in existing methods to systematically identify and quantify patient input for use in HTA. Various promising approaches have been proposed and are being tested. In this workshop, Dr. Chapman will first briefly discuss the importance and challenges of incorporating patient input in HTA (10 minutes). Drs. Susan dosReis and Julia Slejko will discuss their approach to incorporating patient input for a patient-driven HTA. Using the PAVE Center’s patient-informed value elements, they will illustrate with an application in major depressive disorder how to elicit and quantify patient input on value elements and how to translate this into meaningful patient-driven HTA (20 minutes). Dr. Frank will discuss a proof-of-concept study using goal attainment scaling and work with patient communities to “crowd source” patient input in rheumatoid arthritis, which can be used subsequently to inform multi-criteria decision analysis (MCDA). (10 minutes) Insights from the workshop are applicable across all stakeholders: researchers will gain insight into available methods to incorporate patient input into methods to inform HTA; innovators and patient groups can learn about how to ensure comprehensive patient input is incorporated into the data collection process; and payers and HTA bodies can learn about specific approaches useful for integrating patient input into decision-making.
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
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.
Lori Frank, PhD
RAND Corporation, Kensington, MD, USA
Lori Frank, PhD is a Senior Scientist with the RAND Corporation. Her work is focused on incorporating patient and consumer perspectives in health outcomes research and valuation, and methods to improve treatment decision-making. She is Past President of the International Society for Quality of Life Research and serves on the Board of the Personalized Medicine Coalition. She has held positions with the National Institute on Aging, Georgetown University, Medimmune LLC/AstraZeneca, and the Veteran’s Administration. She served as founding Director of the Evaluation & Analysis program at PCORI. Her PhD is from the Pennsylvania State University and her MA is from the Johns Hopkins University.
Julia F. Slejko, PhD
University of Maryland, 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.
Cost-Effectiveness Analysis of Diagnostic and Screening Strategies: When and How to Compare a Googolplex of Strategies?
Live
PURPOSE: Cost-effectiveness modelling of diagnostic and screening strategies enables the comparison of multiple test- and treat strategies well beyond the number of comparators in a clinical study.
The flipside is that often hundreds, thousands, or a googolplex of strategies can be formulated. Comparing them all may be infeasible, inefficient, and the results difficult to interpret and communicate. As cost-effectiveness modelling is increasingly used to evaluate diagnostic/screening tests, inform guidelines and national policy, addressing this challenge is essential. We will present approaches to the cost-effectiveness analysis of many diagnostic strategies and discuss with the audience how best to handle their complexity.
DESCRIPTION: Martin Henriksson will introduce the challenges and policy implications, followed by case studies providing food for thought:
Cascade screening for familial hypercholesterolemia, where strategies were selected according to their probability of being cost-effective (Rita Faria). From two billion to seven strategies in screening for atrial fibrillation, modelling outcomes for all potential strategies, systematically narrowing down to a few potentially cost-effective ones (Mattias Aronsson). Metamodeling using an existing simulation model, to exhaustively evaluate all potential screening strategies for colorectal cancer (Erik Koffijberg). We will discuss:
How to present the results of 100s of strategies? Can we determine beforehand or during evaluation which (further) strategies might be interesting to evaluate (and ignore the rest)? How can metamodeling support evaluation of many strategies? We will demonstrate the challenges in selecting and comparing multiple testing strategies with live polls (e.g. guessing the number candidate strategies for evaluation, the strategies which are unlikely to be cost-effective); and encourage the audience to take part in the discussion by asking for their views and questions throughout. This interactive workshop will be relevant for analysts performing and decision makers considering cost-effectiveness analyses of diagnostics, as well as anyone interested in cost-effectiveness modelling of many complex pathways.
Discussion Leaders
Martin Henriksson, PhD
Linköping University, Linköping, Sweden
Discussants
Mattias Aronsson, PhD
AstraZeneca, Södertälje, Sweden
My research have focused on the development and application of novel modelling methods but also includes studies on utility instruments and quality-adjusted life-year weights.
Rita Faria, MSc
University of York, York, United Kingdom
Rita Faria is a health economist at the University of York, UK, since 2010. Rita conducts economic evaluation of health and care interventions, from drugs to devices, diagnostic tests, and long-term care. Her research has influenced UK government’s policy and informed national clinical guidelines. She is a NICE committee member and a pharmacoeconomics expert for Portugal’s HTA agency.
Erik Koffijberg, PhD, MSc
University of Twente, Enschede, OV, Netherlands
Dr H. (Erik) Koffijberg has an MSc in Technical Computer Science and a PhD in Decision-Analytic Modelling in Neurology. He has published >125 peer-reviewed scientific papers and contributed extensively methods development for 1) (early) HTA of new tests and clinical prediction rules; 2) optimizing screening strategies; 3) identifying patient heterogeneity to tailor interventions to subgroups; 4) prioritizing research, based on systematic reviews and meta-analyses. His work focuses on the use of decision analytic modelling for impact assessment of diagnostic technologies on healthcare and healthcare delivery.
Assessing the Value of Digital Health Technologies: The Experience of Germany and England
Live
PURPOSE: 1) To present the differences in health economics when applied to digital health technologies (DHTs) and pharmaceuticals
2) To compare the English and German processes for bringing DHTs to market, specifically the NICE Medical Technologies Evaluation Programme and Evidence standards for Digital Health Technologies
DESCRIPTION: COVID-19 has accelerated the use ofDigital Health Technologies (DHTs), allowing them to deliver on their promise to transform healthcare delivery and health production. England and Germany are among the frontrunners in introducing these new technologies to their system.
Approaches to assessing value for money of DHTs differ from traditional approaches to evaluating pharmaceuticals. This is because of fundamental differences in the properties of DHTS and pharmaceuticals, that affect many aspects of value assessment. For example, DHTs can have multiple effects beyond patients’ health gains, such as on organisational effectiveness and efficiency. This workshop will be of interest to individuals that want to understand how economic evaluations and health economics principles can be applied to DHTs. Simon Brassel will provide an overview of how traditional health economics principles are challenged when evaluating DHTs. Eleanor Bell will provide insights from the NICE Medical Technologies Evaluation Programme and Evidence Standards for Digital Health Technologies in England, explaining the type of economic evaluations that are required. Pamela Aidelsburger will present the experience from the Digital Healthcare Act in Germany, where initial attempts have been made to formalise value assessment approaches for DHTs. AUDIENCE INTERACTIVE ELEMENT Each presenter will use interactive polling to engage the audience and understand its view on key issues such as the most urgent challenges when evaluating DHTs and the countries’ solutions to those challenges. The workshop will be concluded by a Q&A session, where speakers will answer questions from the audience and discuss the differences between the English and German approach to evaluating DHTs.
Discussion Leaders
Martina Garau, MSc
Office of Health Economics, London, United Kingdom
Discussants
Pamela Aidelsburger, Dr. med. M.P.H postgrad. M.Sc
CAREM GmbH, Königsdorf, BY, Germany
Dr. Pamela Aidelsburger, MD, M.P.H. postgrad, M.Sc. is CEO of the CAREM GmbH since 2004.
She has now over 20 years of experience in benefit assessment, economic evaluation, and consultancy for public organisations as well as for pharmaceutical industry and medical device manufactures. With the introduction of the DiGA (Digitale Gesundheitsanweisungen) directory in Germany she supported DiGA manufacturers on their way through the assessment procedure.
Eleanor Bell, MSc Health Economics and Decision Science
Office of Health Economics, London, United Kingdom
Simon Brassel, MSc, Dipl.Ing
Office of Health Economics, London, United Kingdom
Simon is a Health Economist and Electrical Engineer and works as a Principal Economist at the Office of Health Economics in London. He has over eight years of professional experience in international healthcare markets covering the whole spectrum of health technologies, from pharmaceuticals to digital health technologies.
Before joining the OHE, Simon worked at the EMEA headquarters of Fresenius Medical Care, the world’s largest provider of products and services for patients suffering from chronic kidney disease. There, he held various roles within the field of therapy marketing and product management and in the field of Health Economics, Market Access and Policy Affairs.
Before Fresenius, Simon worked as a technology consultant at the VDI Technologiezentrum in Berlin. As part of a small team, he worked on the National Strategy Process on Innovations in Medical Technology, which three German Federal Ministries initiated. He also served as a grants officer and helped fund a range of collaborative research projects in the health-tech field with support from the German Federal Ministry of Research and Education.
Simon also holds an MSc in Health Policy (Health Economics) from the London School of Economics and Political Sciences (LSE), a Dipl.-Ing. in Electrical Engineering from the Technische Universität München (TUM), and a joint Diploma in General Management from the University of Rochester and the University of Bern.
Independent Scientific Opinion for CE Mark or Early Dialogue for HTA: A Unique Expert Panel for Both?
Live
ISSUE: The European Union (EU) Medical Devices Regulation 2017/745 (MDR) became effective in May 2021 with a key aim to improve clinical evidence generation. The legislative proposal for regulation of health technology assessment (HTA) in the EU appears to be moving forward with the hope of supporting market access processes for innovative technologies by centralizing clinical assessments. These regulations foresee a key role for expert panels to inform Notified Bodies (NBs) during conformity assessment for CE mark and in early dialogue for HTA for coverage decisions, but how and whether they should be combined remains an open question.
OVERVIEW: The panel will debate the benefits and intended/unintended consequences of instituting a unique expert panel for CE mark and HTA. They will address how such a proposal might affect the roles of NBs and various stakeholders, including European and national regulatory agencies, HTA bodies, payers, policymakers, manufacturers, patients, and if and how a unique panel might negatively/positively affect timely, equal access to safe, effective innovation. Paul Piscoi (10’) will present the EU policy viewpoint regarding cooperation on HTA, proposed EU HTA Regulation and MDR. Sabina Hoekstra (10’) will discuss NB interaction with EU and HTA bodies, stakeholders and manufacturers and if and how a unique CE Mark/HTA expert panel might fit in the current scenario. Andrea Rappagliosi (10’) will discuss industry’s perspective on pathways to CE Mark and reimbursement under MDR and proposed EU HTA Regulation, discussing interactions with NBs and stakeholders in preparing clinical documentation for CE Mark/HTA. 10’ will be allocated to the introduction of the panel issue and 20’ will be allotted to discussion. The entire Medical Device and HTA ecosystem would benefit from attending.
Moderators
Rosanna Tarricone, MSc, PhD
SDA Bocconi School of Management, ROMA, RM, Italy
Rosanna Tarricone is Associate Dean at SDA Bocconi School of Management, Milan. She graduated in Business Administration at Bocconi University and holds an MSc in Health Services Management and PhD in Public Health and Policy, both from the London School of Hygiene and Tropical Medicine, University of London, UK. She has over 100 publications in the areas of health policy, healthcare management, economic analysis of health care services, and health technology assessment (HTA).
In the last 10 years, Rosanna has been awarded over 10 multi-annual competitive research grants from international institutions such as the European Union, the Swiss Bridge Award, and InHealth (USA). She has been the leader of MedtecHTA, a large, three-year EU-funded research project that has made recommendations on how to improve methods for assessing the effectiveness and cost-effectiveness of medical devices and she currently serves as scientific supervisor of COMED, a three-year EU-funded research project on improving assessment methods of costs and outcomes analysis of medical technologies, including mhealth.
Rosanna serves as an expert for the DG Health of the European Commission. She facilitated the continuous exchange of experiences and harmonisation of methods and procedures between the HTA Agencies of the EU Member States within EUnetHTA. Since 2007 Rosanna is an advisor for the Ministry of Health (MoH) of Italy and has contributed to the design and development of the National Programme of HTA for Medical Devices.
Rosanna is an active member of ISPOR where she served on the ISPOR Board of Directors from 2016-2018.
Panelists
Sabina Hoekstra-van den Bosch, PharmD FRAPS
TÜV SÜD Medical Health Services, Veenendaal, GE, Netherlands
Sabina Hoekstra-van den Bosch, PharmD FRAPS is Regulatory Strategy Principal at Notified Body TÜV SÜD and acts as representative of TÜV SÜD and Notified Bodies at European level. She is Chair of NBCG-Med, the EU Commission’s working group of Notified Bodies, and Vice-President of Team-NB, the EU Notified Bodies’ association.
Sabina has 20 years of experience as pharmaceutical and medical device regulator, serving subsequently in the Medicines Evaluation Board, the Ministry of Health and the governmental organization for clinical investigations in the Netherlands.
She has worked as Lead for European Regulation in Philips, where she acted as leader and expert in European Medical Device and Pharmaceutical Regulations on corporate level and was involved in EU MDR/IVDR implementation.
Sabina has a long track record as volunteer in global professional organizations (DIA and RAPS). She is a regular speaker and faculty member in educational conferences on regulatory topics.
She was co-founder and Chair of the RAPS Netherlands Chapter. She is Fellow of RAPS since 2015 and serves in RAPS Global Board of Directors since 2020.
Sabina holds a PharmD from Leiden University.
Paul Piscoi, MD
European Commission, Bruxelles, Belgium
Andrea Rappagliosi, MSc
Edwards Lifesciences, Nyon, VD, Switzerland
Andrea Rappagliosi
Vice-President, Public Affairs EMEA, Canada and LATAM
Edwards Lifesciences
Nyon, Switzerland
Andrea is currently Vice-President Public Affairs EMEA, Canada and LATAM at Edwards Lifesciences and member of the regional Executive Leadership Team. He is leading the Market Access, Government Affairs, Communication and Patients Advocacy engagement for the company.
Born in Rome, Andrea received a law degree from the University of Rome, La Sapienza. Andrea began his professional career in the Italian Senate. Recently he worked in the Sanofi group as VP Public Affairs Europe and before as VP, Market Access, Health Policy and Medical Affairs at Sanofi Pasteur MSD. At SPMSD Joint Venture he was member of its Executive Committee from 2012 to 2016. Before, he worked at Baxter Healthcare, Serono International and GSK in different European and International positions in the public affairs and market access policy area.
In the last ten years, Andrea represented the healthcare Industry in several European EU Commission and Member States initiatives such as the HTANetwork, EUnetHTA Joint-Action 3, and the EU Active & Healthy Aging Innovation Partnership and the EU Joint Action on Vaccination. He was President of EuropaBio the European Association of the biotech Industry (2009-2011), and of Vaccines Europe, the European Vaccines manufacturers association (2013-2017). Andrea is a founding member of the Global Policy Forum at HTAi – the scientific and professional society for all those who produce or use health technology assessment (HTA)
T: + 41 79 5968808
E: andrea_rappagliosi@edwards.com
Who and What Are We Missing in Real-World Data? How Do We Improve the Capture of Social Determinants of Health?
Live
ISSUE: The proliferation of real-world data (RWD) have led biopharmaceutical manufacturers, regulators, payers, and providers to focus on utilizing RWD to support evidence-based decision-making in healthcare. The inclusion of high-quality sociodemographic information (e.g. race, gender, ethnicity, geography), especially for people historically underrepresented in medical research, is critical to inform research which enables the delivery of more equitable care with respect to access, quality, and outcomes. Across Europe, comparative data on health outcomes, comprehensive social determinants, and socio-economic structures are variable, making stratified analyses among these underrepresented populations difficult. In both the US and EU, race, ethnicity, and gender are recorded inconsistently across different RWD sources, with many lacking reliable information on these variables and data collection methods. The absence of this data and its analysis can lead to gaps in evidence-generation, further contributing to inequities in healthcare delivery.
OVERVIEW: During this session, the moderator will provide an overview of data capture for key socio-demographic information in the US and EU and its impact on research analysis and insights. Then, panelists will discuss how a lack of sociodemographic information in RWD inhibits efforts to deliver equitable healthcare in different healthcare systems, and how the collection and analysis of high-quality RWD can be utilized to advance health research and care delivery for all populations. The discussion will be followed by an audience Q&A.
Moderators
Nirosha Lederer, PhD
Aetion Inc, Boston, MA, USA
Nirosha Mahendraratnam Lederer, PhD, is director of real-world evidence strategy at Aetion. In this role, she advises clients on real-world evidence trends and cultivates partnership opportunities to advance real-world evidence use in decision making. Dr. Lederer has more than 15 years of research experience using real-world data to support high-value decision making in the US health care 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 real-world data and real-world evidence for Food and Drug Administration (FDA) decision making. She previously worked as a subject matter expert in patient-focused drug development at the FDA Oncology Center of Excellence. She also worked at Avalere Health as manager of health economics and outcomes research/evidence-based medicine policy. She received her PhD in health outcomes and policy from the UNC Eshelman School of Pharmacy, during which she was a Worldwide Health Economics and Outcomes Research Fellow with Bristol-Myers Squibb. She also received her MSPH in health policy and management from the Johns Hopkins Bloomberg School of Public Health and the BA in public health from the Johns Hopkins University.
Panelists
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.
Alice Turnbull, PhD, MA, BSc
Health Data Research UK, London, United Kingdom
As Programme Director at Health Data Research UK (HDR UK), Dr Alice Turnbull supports the Better Care programme in its vision to improve people’s lives by equipping clinicians and patients in the UK with the best possible data-based information to make decisions about their care. A key focus of this programme, and the work of HDR UK more broadly, is to champion diversity and inclusion through data; capturing data which represents all of us and can be used to fully understand and inform the health and care of everyone.
Alice joined HDR UK from the Public Health England (PHE) where she led the collection of the national SACT dataset through the National Cancer Registration and Analysis Service leading. Before this Alice ran the Mainstreaming Cancer Genetics programme, a collaboration between the Institute of Cancer Research and the Royal Marsden Hospital to increase access to genetic testing for individuals with cancer, and has held roles in life sciences consultancy and academic research.
When not working on all things health data-related Alice enjoys yoga, hiking and can often be found baking a cake.
James Wu, MSc, MPH
Kite Pharma/Gilead, Santa Monica, CA, USA
Jim has recently joined Kite Pharma/Gilead, as the Global Health Economics & Outcomes Research lead for one of Kite’s CAR T cell therapies. He joins Kite from Amgen, where he was Principal Health Economist in Global Health Economics for seven years. In his work at Amgen, Jim acquired extensive experience in oncology, health technology assessment, and partnership with patient advocacy groups including serving as a Resident Scholar to the Friends of Cancer Research (Washington, DC) for three years. In addition, Jim has been a long-standing contributor to the Patient Centered Outcomes Research Institute (PCORI), as a grants reviewer for several programs (including the Improving Methods Program), as an inaugural panel member on the Rare Diseases Advisory Panel (2013-2018), and as an industry stakeholder for the PCORI National Patient-Centered Clinical Research Network (PCORnet) (2015-2018). Prior to Amgen, Jim worked in various biotech roles with increasing responsibilities including Genentech, Elan, Abbott, and Hyperion Therapeutics, developing expertise in health economics, real-world evidence, market access, reimbursement, contracting, pricing, and commercial operations. Jim holds a BS in Cellular Biology, Molecular Biology, and Biochemistry from the University of Michigan (Ann Arbor, MI), an MS in Immunology from Brown University (Providence, RI) and an MPH from New York Medical College.
Spotlight Session
At First I Was Afraid, I Was Petrified... Issues and Possible Solutions to the Problems of Extrapolating Survival Curves from Limited Trial Data
Live
PURPOSE
To explore the almost ubiquitous problem of performing survival extrapolation with heavily censored data from clinical trials, discuss the implications of current practice and introduce possible novel methods to alleviate the problem.
DESCRIPTION
Economic evaluations as part of health technology assessments (HTA) typically require estimates of lifetime survival benefit for new oncologic therapies. Interim analyses of trials with limited follow up are increasingly used to inform FDA and EMA regulatory approval, but the high degrees of administrative censoring in these trials create significant challenges when it comes time to extrapolate survival outcomes over a lifetime time horizon.
Current approaches of extrapolation often assume that the treatment effect observed in the trial can continue indefinitely, while in reality it is likely to vary over time, particularly in the long term. In this workshop, we will firstly present a brief review of current methods to inform long term survival benefit in the presence of heavily censored data and their main limitations and implications for the wider economic analysis. Secondly, we will present a brief introduction to the advantages of Bayesian modelling, specifically in survival analysis in HTA. Finally, we will present an innovative methodology based on “blending” survival curves as a possible solution. The basic idea is to mix a flexible model (e.g. Cox semi-parametric) to fit as well as possible the observed data and a parametric model encoding assumptions on the expected behaviour of underlying long-term survival. The two are “blended” into a single survival curve that is identical with the flexible model over the range of observed times and increasingly similar to the parametric model over the extrapolation period. This approach allows a wide range of plausible scenarios to be considered as well as the inclusion of genuine information, based e.g. on hard data or expert opinion.
Discussion Leaders
Zhaojing Che, MSc
University College London, London, United Kingdom
Zhaojing Che, is a PhD student in the Department of Statistical Science at University College London. Her supervisors are Prof Gianluca Baio and Dr Nathan Green. She has a MSc degree in Statistics at Imperial College London. Zhaojing's main interest is Bayesian statistical modelling in health economic evaluation. Her current focus is on Bayesian Survival analysis techniques especially for issues of extrapolation in the presence of non proportional hazards.
Discussants
Gianluca Baio, PhD
University College London, London, United Kingdom
Gianluca is Professor of Statistics and Health Economics in the Department of Statistical Science at University College London. He graduated in Statistics and Economics from the University of Florence (Italy) and completed a PhD programme in Applied Statistics again at the University of Florence, after a period at the Program on the Pharmaceutical Industry at the MIT Sloan School of Management, Cambridge (USA).
His main interests are in Bayesian statistical modelling for cost effectiveness analysis and decision-making problems in the health systems, hierarchical/multilevel models and causal inference using the decision-theoretic approach.
He leads the Statistics for Health Economic Evaluation research group within the department of Statistical Science and collaborates with the UK National Institute for Health and Care Excellence (NICE) as a Scientific Advisor on Health Technology Appraisal projects.
Victoria Paly, MHS
ICON plc, New York, NY, USA
Victoria is a Senior Principal in ICON's Global Health Economics, Outcomes Research & Epidemiology (GHEORE) group. She has a Masters degree in Health Economics from the Johns Hopkins University Bloomberg School of Public Health. Victoria specializes survival analysis in support of economic evaluations for health technology assessment.
13:30 - 16:00
Poster Session And Exhibitor Meet Ups
Live
Interact with the authors of the latest research discoveries in HEOR through our virtual poster presentations and gallery. In between poster discussions, connect one-on-one with our exhibitors and sponsors. Engage with potential solution, service, and resource providers.
Virtual Poster Discussion Session 3
Live
14:00 - 15:30
Educational Symposia
Early-Stage Treatment in Oncology: How Can We Break the Access Logjam?
Live
Early-stage treatments in neoadjuvant and adjuvant settings carries hope for many cancer patients: improved patient-relevant outcomes, curative potential, and prolonged survival effects have motivated attempts to try and treat patients earlier. As earlier treatment options are being considered, the use of endpoints other than overall survival has increased in recent years. For instance, between 2014 and 2016, nearly 75% of the European Medicines Agency’s approvals for oncology medicines were based on randomized controlled trials with disease-relevant endpoints. As the value and conditions around the acceptability of these endpoints remain contested, it is imperative to understand if, and how, access to early-stage cancer treatment is affected by the use of non-OS, disease-relevant endpoints. How do different countries in Europe take these endpoints into account, and what are best practices to ensure safe and timely access of neoadjuvant and adjuvant cancer treatments for patients?
Sponsor
MSD
Moderators
Duane Schulthess, BA, MBA
Vital Transformation, Wezembeek Oppem, VBR, Belgium
Duane Schulthess is the Managing Director of Vital Transformation, a consultancy focused on quantifying and defining innovation and value in the health-care sector. His 2019 BMJ publication titled “Are CAR-T therapies living up to their hype?” is considered a ground-breaking analysis in the field of real-world evidence, and has been cited in presentations by the European Medicines Agency and EUnetHTA.
Previously, Duane was the EMEA Head of Corporate Development of The Wall Street Journal, and the Commercial Director of Science|Business. He is a senior associate of the UK’s Royal Society of Medicine.
Speakers
Oriana Ciani, PhD
SDA Bocconi School of Management, Milan, MI, Italy
Oriana Ciani is Associate Professor of Practice at SDA Bocconi in Milan. She holds a MSc in Biomedical Engineering from Politecnico di Milano and postgraduate degree in Healthcare Management from Bocconi University. She received her PhD from the University of Exeter with a thesis focusing on the evaluation of surrogate end points. She has been 2020 Fulbright Research Scholar at Yale School of Medicine and Yale School of Public Health. Oriana's research interests are centred on the use of evidence synthesis techniques to inform policy decisions, health technology assessment (HTA) and healthcare policies evaluation.
Marcin Czech, PhD
Institute of Mother and Child at Warsaw University of Technology, Warsaw, MZ, Poland
Marcin Czech is a full professor and head of the Department of Pharmacoeconomics at the Institute of Mother and Child in Warsaw, President of ISPOR, Poland Chapter, former Undersecretary of State/ Vice Minister at the Ministry of Health. He is the author of over 250 articles and reports in the field of management, health economics, pharmacoeconomics and quality of life. A medical doctor by education, specialist in epidemiology and specialist in public health, holding PhD degrees in medicine and management, MBA; completed postgraduate studies in Health Economics, Leadership Academy and university trainings.
Miguel Korte, Ph.D.
MSD, Kenilworth, NJ, USA
My brief education summary:
- German Pediatric Hematology-Oncology Pediatrician: (Schwerpunkt Kinder-
Hämatologie und-Onkologie). Certified by Bezirksärztekammer Rheinhessen, Mainz,
Germany - MAY 2009
- PhD approved with “magna cum laude”. Certified by the Johannes Gutenberg-University,
Mainz, Germany. Titel: „ Possibilities and therapeutic implications of cytologic findings in
Pediatrics (with special reference to pediatric hematology and oncology) “. DEC 2008
- German Pediatrician: (Facharzt für Kinderheilkunde und Jugendmedizin). Certified by
„Bezirksärztekammer Rheinhessen“. Mainz, Germany - 21.MAR.2007
- German MD Certification (Approbation als Arzt). Certified by „Landesamt für Soziales,
Jugend und Versorgung Rheinland-Pfalz“. Koblenz, Germany - 20.MAR.2006
- Chief of Resident: Completed Pediatrics Residency Chief Program at the José de San
Martín University Hospital of the University of Buenos Aires (UBA) School of Medicine
(JUN 2000 – MAY 2001).
- Pediatrician: Certificate as Pediatrician by the University of Buenos Aires, School of
Medicine (NOV 2001).
- Pediatrician: Certificate as Pediatrician by the Argentine Pediatric Society (DEC 2000)
- Residency: Completed Pediatrics Residency Program at the José de San Martín University
Hospital of the University of Bs. As. (UBA) School of Medicine (JUN 1997 – MAY 2000)
University: MD - University of Salvador (USAL), Buenos Aires November 1996
I am a dynamic and driven leader, entrepreneurial mindset and passion for innovation with demonstrated success collaborating across functions and geographies. Decisive, intelligent risk taker and resourceful problem solver who inspires change through influence. Strong sense of ethic and integrity and the desire for impact. Adaptable, organized and data-driven with a strategic vision of the evolving oncology field.
Michele Pistollato, PhD
Charles River Associates, London, LON, United Kingdom
Michele Pistollato is a Principal at Charles River Associates. He specialises on policy topics in Life Sciences, ranging from value assessment and pricing dynamics for pharmaceuticals to public policy issues regarding specific therapy areas, such as oncology, cardiovascular diseases and rare diseases. Recent work includes research analyses on the consequences of price transparency for pharmaceuticals, the ideal environment for a well-functioning European HTA process and the challenges for oncology combinations.
14:30 - 15:30
New Professional Networking Session
Live
Hosted by the ISPOR New Professional Steering Committee, meet and chat with your fellow peers in HEOR and ISPOR leaders during our nightly social hours. During this hour, we will be focusing on your career story. Hear from the hosts on their journey to become the professional that they are today, as well as their favorite experiences with ISPOR. We invite you to join this Social Hour to share your story and network with others!
16:00 - 17:00
ISPOR Forums
ISPOR Task Force on Emerging Good Practice in Quantitative Benefit-Risk Assessment: A Roadmap
Benefit-risk assessment (BRA) is used in various phases along the medical product lifecycles (e.g., marketing authorization and surveillance, health technology assessment and clinical decisions) to understand how favourable the benefit-risk profile of a product is compared to others. A BRA begins with defining its decision context, selecting key benefit-risk attributes of a products and comparator(s), measuring their performances on these attributes, and analysing presented evidence and its uncertainties. If the product’s benefit-risk balance remains unclear after these steps, the decision can warrant a quantitative BRA (qBRA). Such analyses integrate trade-off preference data with evidence on product performances using an evaluation framework (e.g., multi-criteria decision analysis (MCDA)), to perform an overall assessment of individual benefit-risk profiles. Despite enthusiasm of regulators toward qBRA, little guidance exists on how to conduct and report a scientifically rigor qBRA. The task force will describe their preliminary recommendations on five steps of qBRA: formulating the research question, developing a model, eliciting preferences, conducting analysis, and communicating results (Ho). We will discuss how these recommendations fit regulatory requirements and illustrate the use of these recommendations in a case study on a novel agent for treating plaque psoriasis (Pignatti). Through polling, we will gather the audience’s views on the most important qBRA steps requiring recommendations and compare them to our Delphi panel results (Veldwijk). These results will inform the TF’s qBRA reporting checklist. Finally, we will discuss the audience’s experience of implementing qBRAs (Tervonen).
Moderators
Tommi Tervonen, PhD
Evidera, London, LON, United Kingdom
Tommi Tervonen is Senior Director of Patient-Centered Research 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 P Ho, MSc
Google Health, 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.
Jorien Veldwijk, PhD
Erasmus University Rotterdam, Rotterdam, Netherlands
Issue Panels and Workshops
Predicting Real-World Outcomes in Mental Health From Structured and Unstructured Electronic Health Records Data
Live
PURPOSE:
Mental health is a particular challenge for measuring and predicting outcomes because of a lack of quantitative data – (biomarkers, vital signs) – and the preponderance of unstructured data in electronic health records (EHR). This workshop will show how quantitative data can be collected in EHR and used to develop predictive models incorporating medication data, demographics, unstructured or semi-structured data like clinician notes, mental status examination (MSE), social stressors, family history and medication side effects.
DESCRIPTION:
Clinical Global Impression-Severity (CGI-S) scores are an example scale that can be used as part of measurement-based care (MBC) and can also provide a tool for training predictive machine-learning (ML) models. We will also show how Natural Language Processing (NLP) can be used to extract structured information from semi-structured input such as MSE and used to make predictions of care outcomes. For example, ML can be used to predict which patients with Major Depressive Disorder (MDD) will recover, and which will go on to be diagnosed with Treatment Resistant Depression (TRD). We will contend that given the scale of the unmet clinical need in mental health care, and the limited resources available for care, the use of MBC supported by ML could improve the quality and efficiency of care. Quantitative measures are not widely collected, and we will encourage the audience to challenge our contention that this would be beneficial and could be done without imposing a significant burden on clinicians. We anticipate a lively discussion. We will make 4 brief presentations followed by a Q&A/discussion session. The presentations will be:
Measurement based care: Prof. Bischof [10 mins] Predicting treatment resistant depression: Dr. Sarkar [10 mins] Generating structured outcome data from semi-structured clinical notes: Dr. Patel [10 mins] Transferring research insights into clinical practice: Prof. Correll [10 mins] Followed by a Q&A/discussion. [20 mins]
Discussion Leaders
Christoph Correll, MD
Charité Universitätsmedizin, Glen Oaks, NY, USA
Christoph U. Correll is Professor of Psychiatry at The Zucker School of Medicine at Hofstra/Northwell, NY, USA, plus Professor and Chair of the Department of Child and Adolescent Psychiatry, Charité University Medicine, Berlin, Germany. His expertise covers the identification, and treatment of severe mental illness, psychopharmacology, epidemiology, clinical trials, and physical health in mental health. He has authored >700 articles, served on several expert consensus panels, and received >40 research awards for his work. Since 2014, the year of inception of this metric, he has been listed every year by Clarivate/Web of Science as one of the “most influential scientific minds” and “top 1% cited scientists in the area of psychiatry”.
Discussants
Evelyne Bischof, MD, MPH
Shanghai University of Medicine & Health Sciences, Shanghai, 31, China
As an internal medicine specialist with focus on oncology and longevity, I’m passionate to bring precision medicine by AI- and machine learning data translation towards individualized prevention and treatment.
EDUCATION
Intern MD Massachusetts General Hospital, Beth Israel Medical Deaconess and Dana Farber (2009-2010), Columbia University (2010-2011)
MD (residency, fellowship, attending physician since 2016) University Hospital Basel, Switzerland
Associate professor Shanghai University of Medicine and Health Sciences, Shanghai (2016-now)
Lecturer, Jiaotong University School of Medicine, Shanghai (2016-now)
Associate professor Shanghai University of Medicine and Health Sciences, Shanghai (2016-now)
President of Swiss Young Internists, 2013-2020
ACCREDITATIONS
Internal medicine board certification (Switzerland)
Rashmi Patel, MD PhD
King's College London, London, United Kingdom
Dr Rashmi Patel is an NIHR Advanced Fellow at the Institute of Psychiatry, Psychology & Neuroscience, King’s College London, and Vice President for Medical and Scientific Affairs at Holmusk. He is a board-certified psychiatrist with expertise in mental healthcare data analytics using natural language processing to extract clinical information from electronic health record data to provide insights into treatment outcomes.
Joydeep Sarkar, PhD
Holmusk, Singapore, Singapore
Practitioners' Guide to Using the Generalized Risk-Adjusted Cost-Effectiveness (GRACE) Model for Health Technology Assessment
Live
PURPOSE: To provide “guidebook” assistance for practitioners conducting health technology assessments (HTAs) using the Generalized Risk-Adjusted Cost-Effectiveness (GRACE) method for valuing medical interventions.
DESCRIPTION:
GRACE generalizes traditional Cost-Effectiveness Analysis (CEA) by introducing decreasing returns to health-related quality of life (QoL). Three important changes emerge for the proper conduct of HTAs: (1) In GRACE (compared with current CEA practice), WTP is lower for low-severity illnesses and rises exponentially with illness severity. In stark contrast, conventional CEA assumes that WTP does not vary with disease severity. Similarly, GRACE shows why WTP for QoL improvements is greater for disabled- than for otherwise-similar non-disabled persons, an issue creating major concern regarding current CEA methods, which impute lower value to QoL improvement for disabled persons. (2) Mean improvements in QoL must be adjusted to account for uncertainty in health outcomes; lower uncertainty in QoL outcomes increases value, independent of mean improvements. (3) Willingness to trade life expectancy (LE) for QoL improvements varies with untreated illness severity. We will review GRACE-related methods to conduct all of these analyses, combining information on population risk preferences and information regarding treatment-specific distributions of health outcomes. GRACE requires new “done-once” estimates of population preferences regarding how health creates utility and uncertainty in health outcomes. We will discuss how to estimate relevant parameters using “economics of happiness” methods, which provide
all necessary parameters for GRACE analyses. GRACE requires more-detailed evidence on distributions of health outcomes than does traditional CEA, all collected at illness/treatment levels, including variance and skewness of outcome distributions in both untreated and treated states. We will show how to obtain these data from current HTA methods (Randomized Controlled Trials, Comparative Effectiveness studies, or Monte-Carlo simulations). We will conclude by discussing how to combine the population-level risk-preference data with illness/treatment specific data to complete GRACE evaluations.
Discussion Leaders
Mendwas Dzingina, PhD
Pfizer, LONDON, LON, United Kingdom
Name: Dr Mendy Dzingina (MBBS, MSc, PhD)
Job title: Senior Director, HEOR Early Development, Vaccines & Oncology
Patient and Health Impact
Employer: Pfizer
Affiliations: Visiting Lecturer, King's College London, UK.
Discussants
Darius Lakdawalla, PhD
School of Pharmacy and Sol Price School of Public Policy, University of Southern California, 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. His work has appeared in leading peer-reviewed journals of economics, health policy, and medicine, including the American Economic Review, Quarterly Journal of Economics, Health Affairs, the Journal of Health Economics, and the New England Journal of Medicine. In addition, his work has been featured by prominent popular press outlets, such as the Wall Street Journal, National Public Radio, Forbes, and the New York Times.
Charles Phelps, PhD
University of Rochester, Pittsford, 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.
Economic Evaluation of Public Health Response to COVID-19: Is Health Economics up to the Job?
Live
PURPOSE: In response to covid-19, governments have applied stringent public health measures but these were mostly not subjected to economic evaluation. Although lack of time was a factor, the key reason was that health economics had not yet developed the requisite methodologies. Many new approaches to the economic evaluation of covid-19 public health responses have now emerged. Our leader and discussants, who have contributed to these developments, will review their strengths and weaknesses, and present the latest breakthroughs in this fast moving and vital field.
DESCRIPTION: Dr Thom will briefly review public health measures introduced across Europe, and their health and economic impacts. He will highlight the general absence of input from health economists. Dr Zala will present emerging approaches to the economic evaluation of responses to covid-19; other discussants will interact with Dr Zala to critique these methods. Approaches will include cost-utility analyses that assign utilities to cases, hospitalisations, and deaths and then use infectious disease models and/or observed data to evaluate the impact of public health measures; macroeconomic optimal control methods; and infectious disease models linked to impacts on gross domestic product. Dr Keogh-Brown will discuss the LSHTM’s pioneering macroeconomic Computable General Equilibrium modelling approach to pandemic-related policy analysis. Other discussants will raise objections. Dr Keogh-Brown will respond with possibilities for synthesising methodologies, pointing to potential future research to integrate health economics of covid-19 and pandemic modelling. Professor Vassall will discuss the different methods from a health and public policy perspective. Dr Thom will close with a conclusion that health economics is up to the job, but that much work needs to be done. We will use live polling to assess the acceptability of each approach and identify key concerns about their use.
Dr Thom will talk for 5-10 minutes and each discussant for 10-15 minutes.
Discussion Leaders
Howard Thom, BA, MSc, PhD
University of Bristol, Bristol, GLS, United Kingdom
Howard Thom is a Senior Lecturer in Health Economics at the University of Bristol and Senior Director at the consultancy Clifton Insight. His research interests are value of information, uncertainty in economic models, network meta-analysis, and R for health economics.
Discussants
Marcus Keogh-Brown, BSc, MSc, PhD
London School of Hygiene and Tropical Medicine, London, United Kingdom
Marcus Keogh-Brown is an Associate Professor at the London School of Hygiene and Tropical Medicine. His work focusses on macroeconomic modelling of health including applications to infectious disease, non-communicable disease and full model integration.
Henning Tarp Jensen, MSc, PhD
London School of Hygiene and Tropical Medicine, London, United Kingdom
Anna Vassall, PhD
London School of Hygiene and Tropical Medicine, London, United Kingdom
Darshan Zala, BSc, MSc
ZALA PRMA Limited, SUTTON, United Kingdom
Darshan works with industry on health technology assessments and HEOR evidence generation. He also has experience in academia, including a NICE evidence review group. He has publications in economic evaluation, econometrics, trial analyses and real world data evaluations.
Are Health Technology Assessments (HTA) Replicable? Implications of Uncertainty for “Best Practice” and Policy Decision Making
Live
ISSUE: HTAs draw on evidence that is often incomplete and immature. HTA organizations do their best to describe uncertainty’s impact on projected benefits, costs, and cost-effectiveness, but they sometimes come up short because they do not consider all plausible alternative assumptions. HTAs can miss plausible assumptions because they typically convene one group of experts and may settle on a single best option. If there are plausible alternatives, would they produce substantially different estimates? A recent study examined these questions by comparing the results of a completed HTA (Institute For Clinical and Economic Review, PARP inhibitors in ovarian cancer) with a repeat assessment performed by two independent sets of clinical and health economic experts ( https://cevr.tuftsmedicalcenter.org/news/2021/unknown-unknowns ). The three sets of assessments substantially differed with corresponding differences in cost effectiveness.
OVERVIEW: Moderator will frame the issue of the precision associated with assessments and its importance in determining an intervention’s cost effectiveness and associated patient access. Josh Cohen, as the principal investigator on the above study, will share what the study entailed and its findings. Nick Crabb will provide a perspective on how those results relate to NICE. Steve Pearson will provide a US perspective on both methods improvement and policy decision-making. Abigail Colson will discuss how elicitation might be improved. Moderator will engage the audience on the impact for HTAs.
Moderators
Bobby Dubois, MD, PhD
National Pharmaceutical Council, Washington, DC, USA
Robert W. Dubois, MD, PhD, is the Chief Science Officer of the National Pharmaceutical Council (NPC), which sponsors and participates in research on the appropriate use of pharmaceuticals and the clinical and economic value of pharmaceutical innovation. NPC’s research contributes to the scientific foundation for informed discussions about health care access, coverage, appropriate use and value.
As NPC’s chief science officer, a position he has held since 2010, he oversees NPC’s research on policy issues related to the appropriate role of real-world evidence in decision-making, how best to determine value of health care services, the relationship between access and health outcomes, and approaches to maintain an environment supportive of innovation.
Dr. Dubois, who is board certified in internal medicine, brings more than 25 years of experience in health care research, with a particular focus on understanding and ensuring that patients receive high value health care. He has co-founded and led various health care research organizations in developing quality research with practical application.
Dr. Dubois was previously the Chief Medical Officer of Cerner Life Sciences, and he co-founded Protocare Sciences and was its executive vice president, chief medical officer, and later its CEO.
Throughout his career, Dr. Dubois’ primary interest has centered on defining “what works” in health care and finding ways for that evidence to inform health care decision-making. He is a recognized expert in the areas of defining best practice, disease management and appropriateness of care. He has authored more than 175 peer-reviewed articles on comparative effectiveness, evidence-based medicine, the development of practice guidelines and determining the optimal use of high-cost medical services.
Dr. Dubois received his AB from Harvard College, his MD from the Johns Hopkins School of Medicine and his PhD in health policy from the RAND Graduate School. He is the associate editor of the Journal of Comparative Effectiveness Research and is on the editorial board for Health Affairs and The American Journal of Managed Care.
Panelists
Josh Cohen, PhD
Tufts Medical Center, Boston, MA, USA
Joshua Cohen, PhD, is the Deputy Director and Chief Science Officer of the Center for the Evaluation of Value and Risk in Health at the Tufts Medical Center Institute for Clinical Research and Health Policy Studies, and a Research Associate Professor of Medicine at Tufts School of Medicine. Dr. Cohen’s work focuses on the advent of “drug value frameworks”, with a special focus on the Institute for Clinical and Economic Review (ICER) framework, promotion of "open source" practices for health economic simulation models, and the potential benefits of risk-targeted disease screening.
Abigail Colson, MPP
University of Strathclyde, Glasgow, United Kingdom
Abigail Colson is a Lecturer in the Department of Management Science at Strathclyde Business School. Her work focuses on decision and uncertainty analysis, including the structuring, elicitation, and validation of expert judgements. Much of her work is in the field of public health, including program evaluation, cost-effectiveness and benefit-cost analysis, and prioritisation. She is particularly interested in health evaluation in low- and mid-income countries and supporting economic analysis and health technology assessment of new antibiotics.
Nick Crabb, PhD
NICE, Manchester, United Kingdom
Nick Crabb is Programme Director, Scientific Affairs at the National Institute for Health and Care Excellence (NICE). Prior to joining NICE in 2010 as the associate director responsible for establishing and managing the Diagnostics Assessment programme, Nick had a 20-year career in analytical science, process technology and general management in the chemical, pharmaceutical and contract laboratory industries. In 2014 he was appointed to his current role where he oversees the Science Policy and Research programme. Nick has broad scientific and policy interests relating to the evaluation of technologies and interventions to support the development of clinical, public health and social care guidance. His experience includes consideration of HTA issues arising from the availability of novel new products such as cell and gene therapies and work on methods issues relating to the evaluation of antimicrobials.
Steven Pearson, MD
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.
Previously, he has served as a Visiting Scientist in the Department of Bioethics at the NIH, a Special Advisor on Technology and Coverage Policy at the Center for Medicare and Medicaid Services, and the Vice Chair of the Medicare Evidence Development and Coverage Advisory Committee (MedCAC). His publications include over 150 peer-reviewed articles and commentaries on the role of evidence in the health care system, and the book No Margin, No Mission: Health Care Organizations and the Quest for Ethical Excellence, published by Oxford University Press.
Modelling the Disease Not the Technology: Do Multi-Use Disease Models Represent a Win-Win for HTA Agencies, Patients and Model Developers?
Live
ISSUE: Many health economic models are single use and developed to address a specific decision problem. In contrast, multi-use disease models represent a whole disease (e.g. breast cancer or diabetes) and are suitable for different decision problems providing consistency between assessments. These models offer economies of scale in validation and uncertainty analysis. They can also serve other purposes in addition to reimbursement.
However, many challenges exist concerning the development, stakeholder involvement, maintenance, and access to multi-use disease models. The question hence arises whether multi-use disease models are indeed a good idea or rather express the hybris of over-enthusiastic model builders? What about the “horses for courses” argument and reduction of cross model validation? How to select the conditions for which to develop multi-use models? Who is to pay for their development? Who should construct and maintain them? Depending on choices made, stakeholders (consultancy companies, HTA agencies, patients) will face different responsibilities and financial consequences. This issue panel brings together different stakeholders to consider the acceptability, implications and viability of the multi-use disease based modelling approach.
OVERVIEW: The moderator presents a summary of an expert panel survey, highlighting terminology, criteria and challenges (10 minutes). This introduces the first discussion: Should HTA agencies advocate the use of multi-use disease models? Each of the three panelists (HTA agency, open source modelling-academic and consultancy company) will comment on the desirability of multi-use models.
The second discussion will focus on how to best develop and maintain multi-use disease models, avoiding black boxes and outdated models. The moderator will present five possible business cases (5 minutes) and each of the panelists will comment. The audience will be actively engaged in voting rounds regarding statements on the pros and cons of multi-use disease models and the business cases, and will get ample opportunity to react to the panelists.
Moderators
Talitha Feenstra, PhD
University Medical Center Groningen, Groningen, Netherlands
Talitha Feenstra is Associate Professor of PharmacoEconomics at the Groningen Research Institute of Pharmacy, Groningen University and is affiliated with the Dutch Institute for Public Health and the Environment (RIVM). She is a health economist with strong quantitative background, cum laude graduated in econometrics. Her research focuses on economic evaluations in health care, specifically for supporting personalized care by precision medicine. Ongoing projects concern analysis of routine data and simulation modeling in Diabetes Mellitus and mental health care. She developed the model validation reporting tool AdViSHE and worked for the Dutch Healthcare Institute on multi-use disease models.
Panelists
Elisabeth Fenwick, PhD
Pharmerit International, Oxford, United Kingdom
Elisabeth Fenwick is a senior director in the Modeling and Meta-Analysis team at Open Health, based in Oxford in the UK.
Liz provides scientific and strategic support to HE projects globally. She has extensive experience in economic evaluation and health economic modeling having worked in the field for over 20 years. She has worked on a variety of projects in a wide range of disease areas including oncology, respiratory, infectious diseases, cardiology, ophthalmology, and orphan diseases.
Liz has also contributed to methods in the field, in particular relating to decision analytic modeling and simulation methods, probabilistic decision analytic modeling and value of information analysis. Liz was a member of the ISPOR joint task force on good research practices in modeling and a co-author on the joint taskforce paper on uncertainty and co-chaired/co-authored the recent ISPOR task force assessing emerging good practice in value of information analysis for research decisions.
Liz has a PhD and MSc in Health Economics as well as an MSc in Operations Research and joined Open Health from ICON plc where she led the modeling team for the global HE group. Prior to her consultancy career, Liz spent over 15 years as an academic working at University of York, McMaster University, and most recently University of Glasgow.
Saskia Knies, PhD
Health Care Institute Netherlands, Diemen, Netherlands
Petros Pechlivanoglou, PhD
The Hospital for Sick Children, Toronto, ON, Canada
Priority Setting and Assessment of AI Supported Health Technologies: Current Practice and How to Move Forward
Live
ISSUE: AI-supported technologies are rapidly developing and have the potential to improve healthcare quality at reduced cost. However, few examples exist of successfully deployed AI-technologies in clinical practice that are adequately assessed.
New challenges for health technology assessment (HTA) are emerging with the introduction of AI, including the availability and complementarity of large, high quality data sets and algorithms that continuously evolve. Furthermore, limitations or inability of most AI supported technologies to ‘explain’ their decision-making process highlights the importance of new assessment aspects such as trustworthiness, transparency, interpretability and explainability. AI supported technologies challenge the applicability of traditional HTA methods. It calls for early dialogue between stakeholders to set priorities and to identify the evidence required to inform decision making.
OVERVIEW: This panel will debate emerging issues of assessing AI supported health technologies, including priority setting for HTA of AI.
Americo Cicchetti will moderate the panel and provide an overview of the current landscape of HTA of digital health solutions and pose key questions for the panellist to discuss: Does HTA of AI-supported technologies differ from other digital health technologies? What is required of data-collection methods? How should we set priorities for HTA so they are driven by needs rather than data availability? Signe Daugbjerg will discuss why traditional HTA methods needs to be updated to assist decision-makers in assessing the potential value associated with AI-technology adoption. Leandro Pecchia will report on difficulties emerging while assessing AI-based medical technologies during three European large scale projects, ACTIVAGE, GATEKEEPER and ODIN, focusing on the use of AI for improving health and wellbeing in later life. Wija Oortwijn will discuss a sustainable priority strategy for HTA of AI solutions that focuses on the needs of patients and the healthcare system.
Moderators
Americo Cicchetti, Professor
Università Cattolica del Sacro Cuore, Rome, Italy
Americo Cicchetti is Professor of Healthcare Management at Università Cattolica del Sacro Cuore, Faculty of Economics, Rome. He is Director of the Advanced School of Health Economics and Management (ALTEMS).
Chief of Research, Health Technology Assessment Unit and Biomedical Engineering “A. Gemelli” University Hospital – Irccs Rome, Italy.
Visiting Researcher at the Center of Medical Education and Healthcare of Thomas Jefferson University, Philadelphia (USA)
Member of the Scientific Advisory Board of the European Health Management Association (EHMA).
Member of the Price & Reimbursement Committee of the Italian National Drug Agency (AIFA) from 2009-2015. Member of the Technical Advisory Group at the National Program for HTA of Medical Devices of the Italian Ministry of Health (2017-).
He served as Director of the Health Technology Assessment international (2005-2008) and then member of the Executive Committee (from 2010 to present) as Treasurer and Secretary.
Founder and Past President of the Italian Society of Health Technology Assessment. Chairman of the Health Policy forum of SIHTA.
Member of EUNetHTA Joint Action 3, Leader of the Associate Partner Università Cattolica del Sacro Cuore.
Panelists
Signe Daugbjerg, PhD
Università Cattolica del Sacro Cuore, Rome, RM, Italy
Signe Daugbjerg, is the Director of the AI Unit at the Advanced School of Health Economics and Management (ALTEMS) at the Catholic University in Rome, Italy. She is a Health Technology Assessment & Artificial Intelligence expert and works with the implementation of AI supported health technologies, with a specific focus on methods to holistically assess potential effect and value associated with AI adaptation. Signe is an expert evaluator and reviewer for the European Commissions since 2015. She holds a PhD degree in Health and Medical Sciences from the University of Copenhagen with a background in clinical epidemiology and social inequality in health.
Wija Oortwijn, PhD
Radboud University Medical Centre, Nijmegen, Netherlands
Dr. Wija Oortwijn is Senior Researcher in the field of global health technology assessment (HTA) at the Radboud University Medical Centre (Radboudumc), Department of Health Evidence as well as Associate Professor at the Leiden University Medical Centre (Public Health and General Practice Department) in the Netherlands. She studied health sciences, holds a PhD in Medicine and has almost 30 years of relevant professional experience in HTA and health policy analysis around the globe. Her key expertise includes priority setting, policy and programme evaluation (ex-ante, ex-post, impact assessment) and health services evaluation.
She has extensive experience in providing support to governments regarding developing and implementing HTA, health system strengthening and health policy. This includes numerous projects, seminars and consultations in the European Union as well as in Kazakhstan, Moldova, South Africa, Ukraine, and several others, working with Ministries of Health, the European Commission, World Bank, and World Health Organization. Currently, she is providing guidance for institutionalizing HTA mechanisms for coverage decision making in Moldova. Furthermore, she is coordinating a master course on HTA at the Radboud university, and is a faculty member of VALIDATE (values in doing HTA), an e-learning course for integrated HTA funded by Erasmus+ programme: www.validatehta.eu.
She has co-authored 10 book chapters concerning different aspects of HTA, edited several journal issues, and has written more than 60 other scholarly published papers. Furthermore, she has been a keynote speaker at several key HTA events. For more information: https://www.researchgate.net/profile/Wija-Oortwijn
She is a founding Member of the Dutch Society for HTA (NVTAG) and the international Society for HTA (HTAi). She is currently President of HTAi and co-chairing the HTAi-ISPOR task force on deliberative processes for HTA. She is also associate editor of the International Journal of Technology Assessment in Health Care (IJTAHC).
Leandro Pecchia, PhD
University of Warwick, Coventry, United Kingdom
Fri 3 Dec
11:00 - 12:00
ISPOR Forums
Managed Entry Agreements, Coverage with Evidence Development and MCDA: Nice 'Buzzwords' vs Reality in CEE
Managed Entry Agreements, Coverage with Evidence Development and Multi-Criteria Decision Analysis are tools for assessing mainly the highly innovative health technologies from more holistic and objective perspectives, including social impact, ethical and organizational dimensions and other issues, critical for proper evaluation of the innovation and its value for patients, caregivers, healthcare professionals, payers and society. Limited examples of utilizing those tools and methods are already available in CEE region and further research is needed in order to apply them correctly within the context of CEE region healthcare settings, taking into considerations usually limited capacities and capabilities of stakeholders engaged in a process of assessment and appraisal.
Moderators
Oresta Piniazhko, PhD
State Expert Centre of the Ministry of Health of Ukraine, Kyiv, Ukraine, Lviv, 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.
Speakers
Bertalan Németh, PhD
ISPOR CEE Consortium Executive Committee and Syreon Research Institute, Budapest, PE, 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.
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 Centre of the Ministry of Health of Ukraine, Kyiv, Ukraine, Lviv, 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.
Towards Universal Health Coverage in Africa: Case Studies on Access to Essential Medicines and Health Technology Assessment
According to the World Health Organization (WHO), Universal health coverage (UHC) refers to all individuals having access to required health services, of sufficient quality, without suffering financial hardship. While many health systems in Africa are striving to achieve UHC in the near future, most are still facing the perils of lack of access to essential life saving medicines, malnutrition, high levels of child and maternal mortality and the growing double burden of infectious and chronic diseases. These challenges call for a close examination of the progress toward UHC and formulation of lessons to learn from success stories. This forum will focus on presenting case studies from different parts of Africa in the areas of accessibility of essential medicines and the situation of health technology assessment as a basis for resource allocations for UHC.
Moderators
Eskinder Eshetu Ali, .
ISPOR Africa Network Research Committee and Addis Ababa University, Addis Ababa, 1, Ethiopia
Dr Eskinder Eshetu Ali is a pharmacist by training and works as an Assistant Professor of Social and Administrative Pharmacy at the Department of Pharmaceutics and Social Pharmacy, Addis Ababa University (AAU), Ethiopia. He is also the chair-elect of the ISPOR Africa chapter and the principal investigator of ISPOR Africa Research network.
Speakers
Peter Agyei-Baffour, PhD
ISPOR Ghana Chapter and Kwame Nkrumah University of Sciences and Technology, Kumasi, Ghana
Eskinder Eshetu Ali, .
ISPOR Africa Network Research Committee and Addis Ababa University, Addis Ababa, 1, Ethiopia
Dr Eskinder Eshetu Ali is a pharmacist by training and works as an Assistant Professor of Social and Administrative Pharmacy at the Department of Pharmaceutics and Social Pharmacy, Addis Ababa University (AAU), Ethiopia. He is also the chair-elect of the ISPOR Africa chapter and the principal investigator of ISPOR Africa Research network.
Daniel Erku, BPharm, Dip Ed, RPh
ISPOR Ethiopia Chapter and University of Gondar, Gondar, Ethiopia
Dr. Daniel Erku is an applied health economist and health policy analyst with a professional background in pharmacy. His research focuses on employing HTA as an effective tool to support priority setting in LMICs. Dr. Erku is president of ISPOR-Ethiopia Chapter, and Chairs Centre for Research and Engagement in Assessment of Health Technology (CREATE), a not-for-profit, collaborative, multi-stakeholder network of researchers established to spread and emphasize the importance of HTA for UHC in LMICs.
Issue Panels and Workshops
Realising the Potential of Real World Data and Evidence: How Can Pharmaceutical Companies Optimise through Capabilities, Structure and Governance?
Live
ISSUE: In the pharmaceutical industry, real world data and evidence (RWD/E) is increasingly being used to define, differentiate, and deliver value across the drug life cycle. However, as an emerging and underdeveloped capability within organisations, there are considerable challenges to its effective use. The challenge of finding the appropriate organisational structure to reduce the gap between a company’s RWD/E ambition and its ability to deliver on it, is a topic of lively debate within industry. While some advocate for a more centralised approach, with the creation of companywide Centre of Excellence (CoE), others are pushing for more distributed groups of RWD/E experts and tools across the organisation. In between these two extremes, there is an option of adopting a hybrid approach.
OVERVIEW: Panellists will debate the merits and limitations of three distinct organisational models of RWD/E generation (i.e., centralised, federated, and hybrid), exploring how each relate to defined guiding principles and governance practices. Callum Caldwell will moderate the panel and provide an overview of the challenges facing industry, with respect to RWE generation, and capabilities (i.e., skills, processes, technologies, human resource, and expertise) required. Each panellist will take 5-10 minutes to describe a selected organisational model and advocate for its use. Maurille Feudjo Tepie will argue for the pooling of resources within a single group, function or CoE – a centralised model. Simon Teal will argue for a federated model (RWE practitioners spread throughout an organisation), where governance may be mandated through guidelines and Standard Operating Procedures (SOPs). Paul Petraro will advocate for a hybrid approach, where RWE use case will drive the organizational practice. The panel will conclude asking for audience perspectives and questions. The panel will be a rare chance for cross-industry leaders and RWE practitioners to come together, share learnings and discuss topics usually deemed commercially sensitive.
Moderators
Simon Teal, BSc
Bayer AG, Berlin, Germany
Simon Teal, Head of Integrated Evidence Generation (IEG) Data Science, Research & Analytics at Bayer Pharmaceuticals
Simon brings over 14 years of experience working in Real World Evidence (RWE), Health Economic & Outcomes Research (HEOR) & Market Access, and Medical Affairs. Simon joined Bayer in 2014 as a Senior Global Project Leader HEOR in the Global Market Access department, and in 2016 moved on to be a cross-functional RWE Strategy Lead. In 2018, Simon was made Head of RWE Strategy & Outcomes Data Generation in Medical Affairs, and since October 2020, Head of IEG Data Science, Research & Analytics. Before joining Bayer, Simon worked at Pfizer in the Global Outcomes Research team as well as EU Regional Medical Affairs, with a focus on rare and orphan diseases. Simon’s previous experience includes Journal Editor for Medical and Scientific review journals. Simon received his Bachelor of Science degree in Molecular Genetics and Biotechnology from University of Sussex, UK.
Panelists
John Cai, MD
Merck, North Wales, PA, USA
John Cai, MD, PhD, FAMIA, is Executive Director, Real-world Data Analytics and Innovation, in the Merck Center for Observational and Real-World Evidence. He is leading a team of data scientists and outcomes researchers to generate real-world evidence and insights through innovative and advanced analytics. John has more than 20 years of experience in biomedical and clinical research across academic, biotech, and pharmaceutical settings. John received his medical training from China Medical University and his Medical Informatics training from Harvard Medical School. Pursuing a passion for both medicine and computing, John has co-authored peer-reviewed publications in the areas of medical informatics, machine learning, clinical trials, and cancer genomics. John is a Fellow of the American Medical Informatics Association (AMIA), and also serves in the AMIA Industry Advisory Council.
Maurille Feudjo Tepie, Msc. PhD
Amgen Ltd, London, United Kingdom
Dr. Maurille Feudjo Tepie is an Observational Research Director at Amgen and leads the Amgen ELMAC (Europe, Latin-America, Middle East, Africa and Canada) team of the Centre for Observational Research. Prior to joining Amgen in 2009, he spent six years at GSK. Maurille obtained his PhD in Medical Statistics from the London School of Hygiene and Tropical Medicine and then completed a one year post-doctoral fellowship. He is a keen contributor to scientific discussions pertaining to the challenges and/or opportunities offered to regulators, payers, academics, prescribers and to the bio-pharmaceutical industry by advances in computing science and the increased availability of real-world data.
Paul Petraro, ScD, MPH
Boehringer Ingelheim Pharmaceuticals Inc., Ridgefield, CT, USA
Paul is an epidemiological professional who drives RWE and advances his organization to the level of excellence required to support data-driven decision making in bringing value to patients. Paul is a trained Epidemiologist and currently Heads the Global Real World Evidence Analytic Center of Excellence. With a strong background in epidemiologic methods and real-world evidence (RWE), including observational and experimental study designs and analysis, value evidence and outcomes, health economics and outcomes research, post authorization safety/effectiveness studies and risk management as well as in-depth knowledge of public and private epidemiologic resources and databases.
A Manifesto for the Standardisation of Health Technology Assessment Data and Code: Is It Even Possible?
Live
ISSUE: HTA models are often implemented poorly in terms of readability, reproducibility, portability and reliability. Methods and tools for improving this situation already exist in other fields such as software engineering. Is now the time to adopt these and in what form? Is this too much of a leap forward or should we be defining where we eventually want to end up? Is this sort of approach even possible for the ubiquitous Excel model?
OVERVIEW: The panel will debate the pros and cons of HTA data and code standardisation. Felicity Lamrock will moderate the panel and provide an overview of the current situation. The building of models has become standardised using the CHEERS checklist. In a similar way, is it a realistic aim to standardise the implementation of these models? Often, on receipt of some new model code, the steps taken to interrogate it are something like i) what does it look like? ii) does it run? iii) is it correct? Nathan Green will argue that in order to facilitate this process we are essentially looking for a sensible consistency in three areas: i) consistent code, ii) data and metadata, iii) testing. Standardised formats and templates could help achieve this. Dr Trela-Larsen will represent the practicalities of such an approach and argue from the perspective of an HTA agency and raise potential issues.
Moderators
Felicity Lamrock, PhD
Queens University Belfast, Belfast, ANT, United Kingdom
Dr. Felicity Lamrock is a Lecturer in Data Analytics at Queen’s University Belfast. Felicity was previously a statistician at the National Centre for Pharmacoeconomics (NCPE) working with a team of pharmacists and clinicians on Health Technology Assessments to advise the Health Service Executive on the recommendation of new drug therapies in Ireland. She still remains a statistical advisor for the NCPE and is exploring how Northern Ireland could benefit from more decision modelling/pharmacoecononomic assessment.
Panelists
Nathan Green, PhD
University College London, London, LON, United Kingdom
Nathan studied mathematics and statistics at the University of Newcastle-Upon-Tyne and obtained a PhD in applied probability from the University of Liverpool. After working for the Ministry of Defence for several years, applying novel Bayesian Inference ideas, he moved back into academia and the field of public health in 2010.
Research Interests include Bayesian statistical modelling for cost effectiveness analysis and decision-making problems in the health systems, hierarchical/multilevel models and causal inference using the decision-theoretic approach.
Lea Trela-Larsen, PhD
National Centre for Pharmacoeconomics, Dublin, D, Ireland
Lea Trela-Larsen is a senior statistician at the National Centre for Pharmacoeconomics (NCPE) in Ireland. She completed a Mathematics BSc Hons and a PhD in Epidemiology and Statistics both at University of Bristol. She has previously worked as an analyst with the UK Government Statistical Service, as a senior research associate at University of Bristol working with National Joint Registry, and as a post-doctoral researcher at University of Limerick working with the NCPE on a project examining stakeholder requirements for real-world drug utilisation evidence.
When Decision Meets Precision: Is It Time to Evolve Current Approaches to Health Technology Assessments for the Evaluation of Precision Oncology Therapeutics?
Live
ISSUE
: With small trial sizes, lack of active comparators and limited knowledge of the prognostic value of biomarkers complicating the generation of evidence at the assessment stage, precision oncology therapeutics are experiencing difficulties in meeting the evidence requirements of reimbursement agencies. As HTA agencies and payers prepare to review the expanding pipeline of precision oncology therapeutics, how can we introduce new approaches and greater flexibility into the assessment of these products across their lifecycle to help address uncertainties and share risk across stakeholders?
OVERVIEW
: Scientific advancements over the past two decades have ushered in a new paradigm of precision medicine, which is changing drug development and care delivery. Precision oncology, in particular, is redefining how we view cancer, from an organ-specific to a molecular disease, with therapeutics targeting increasingly smaller, better-defined populations based on their molecular profile. With cancer treatment moving toward stratification based on the molecular makeup of a tumour, randomized control trials have shifted to smaller, single arm and basket trials involving a subset of individuals (sometimes using surrogate endpoints or no comparators). Given the evidence limitations of these trials, including the lack of similar therapies with which to make comparisons, HTA agencies are faced with significant challenges in assessing these products, ultimately hindering their adoption and the ability of patients to benefit from these innovations. The session will aim to:
Present an overview of some of the complexities associated with generating and assessing evidence for precision oncology therapeutics [5 minutes]; Discuss and debate potential approaches to addressing these challenges while balancing the potentially conflicting priorities of HTA agencies and payers (represented by Dr. Brian O’Rourke), patients (represented by Anne-Pierre Pickaert) and clinicians (represented by Dr. Jean-Yves Blay) [40 minutes]; and Use interactive online tools (e.g. polls) to gather questions and feedback from the audience [15 minutes].
Moderators
Charles Khoury, MSc, MBA
Shift Health, Toronto, ON, Canada
As the Head of Consulting of Shift Health, Charles Khoury has led hundreds of life sciences strategy projects including engagements focused on advancing the research strategies of leading universities; planning national health innovation infrastructure in the Middle East; working with top-5 biopharmaceutical firms in strengthening market position; and helping health research organizations in Latin America and Africa secure partners and funding. Charles earned his MSc in Medical Genetics and Microbiology from the University of Toronto—conducting basic research in the field of virology—and earned an MBA from the Schulich School of Business at York University—majoring in Strategy and Marketing.
Panelists
Jean-Yves BLAY, MD, PhD
Centre Léon Bérard - Unicancer France, LYON, France
Professor Jean-Yves Blay, MD is a medical oncologist, General Director of the Centre Leon Berard, the Comprehensive Cancer Centre of Lyon France, researcher and a Professor at the University Claude Berard, France. Since 2019, he is the President of the French Federation of Cancer Centers Unicancer.
His work focuses on sarcoma, genomics and targeted treatment of cancer, immune-oncology, and the relationship between tumour immunologic microenvironment and malignant cells, with the goal of clinical applications in the field of diagnosis, prognosis and treatment.
He has been distinguished with the Hamilton-Fairly award from ESMO (2012) and the Henry and Mary-Jane Mitjaville prize from the National Academy of Medicine (2013). He was the President of the ESMO 2019 congress (30000 participants.
Professor Blay is the Director of the LYRICAN SIRIC (previously LYRIC) since 2018. He is also the President of the French Sarcoma Group, the Network director of NETSARC+ network of sarcoma reference center for the INCA since 2019. He serves as secretary for the World Sarcoma Network (WSN), a think tank of all worldwide sarcoma research groups. He serves as the Network Director of ERN-EURACAN, designated by the EU Commission in 2016 to improve the quality of care for patients with rare cancers in the European Union.
Previously, he served as the President of EORTC between 2009 and 2012. He was the former Principal Investigator of Conticanet, an EU Commission network of excellence of FP6, and he PI of the FP7 project EUROSARC.
Professor Blay has co-authored over 1000 peer-reviewed articles and book chapters, and was distinguished as Highly Cited Researcher in 2019. He advises various national and international institutions and research organisations, and is an active member of several oncology scientific organisations and societies, such as ESMO, CTOS, ASCO and AACR.
Brian O'Rourke, PhD
Brian O'Rourke Health Care Consulting Inc., 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 the Chair of the Health Technology Assessment Steering Committee and a member of the Scientific Advisory Council for 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.
Anne-Pierre Pickaert, MSc
Patvocates, PARIS, France
Anne-Pierre Pickaert has over 20 years of public health and access to medicines experience with a wide range of healthcare stakeholders (charity, patient organisation, governmental agency, consultancy and pharmaceutical industry). Upon her own diagnosis Anne-Pierre realised how much her health care background was of great help to navigate through the complexities of her leukaemia patient journey. In order to have a greater impact on patient access to care, Anne-Pierre has founded Care4Access, a consultancy dedicated to patient engagement and advocacy, and has also been collaborating with the think tank and social enterprise Patvocates.
Anne-Pierre is an engaged patient advocate with EGMOS and Association Laurette Fugain, two French patient organisations, to promote bone marrow donor recruitment and improve the management of graft versus host disease GvHD. She is a steering committee member of the Acute Leukemia Advocates Network (ALAN), as well as a board member of the Francophone Society for Bone Marrow Transplant and Cellular Therapies (SFGM-TC), and a member of the EBMT Patient Advocacy Committee. Anne-Pierre is also a campaign committee member of the Hematopoietic stem cell Transplantation Complications (HTC) project.
See the Future: How Important Will Structured Expert Elicitation Become for HTA in the Next 5 Years?
Live
PURPOSE: To discuss best practices, challenges, and the increasing use of Structured Expert Elicitation (SEE) in the context of healthcare decision-making and demonstrate its applicability through an interactive, live experiment.
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.
In November 2020, NICE released a report on the case for change of its methods for health technology assessments (HTAs). One topic discussed here is the use of expert elicitation in a structured, quantitative manner. It is expected that this form of elicitation – particularly the elicitation of uncertain quantities – will be increasingly used to inform submissions to NICE. In light of this anticipated change, we will provide an interactive demonstration of how SEE may be used in practice to inform HTA, and discuss best practices in SEE, challenges in its implementation in HCDM based on our recent experiences, and its expected use in the future. James Horscroft will summarize the different approaches to SEE and set up a live SEE experiment, using the audience as the experts. Jeremy Oakley will present an overview of the Sheffield Elicitation Framework method, how it should be conducted, its advantages and disadvantages, and its applicability to HCDM. Laura Bojke will discuss her recent work on the use of SEE in an HTA context, the dos and don’ts, and what may constitute a set of minimum standards for SEE applied to HCDM. Finally, the panel will review and discuss the results of the experiment and provide their thoughts on the future of SEE in HTA.
Discussion Leaders
James Horscroft, PhD
BresMed Health Solutions, 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.
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.
Jeremy Oakley, PhD
University of Sheffield, Sheffield, United Kingdom
Addressing Challenges in Retrospective Studies of Rare and Ultra-Rare Diseases
Live
PURPOSE: To provide guidelines for retrospective studies of with rare- and ultra-rare diseases that use administrative data. The workshop will draw on real-world HEOR study examples.
DESCRIPTION: Innovations in medicine increasingly target rare and ultra-rare diseases, many of which do not have specific diagnosis codes. Researchers charged with quantifying the "real-world" burden of disease may be stymied by the inability to easily distinguish patients with the disease. Challenges range from diseases grouped with similar diseases under the same ICD code to diseases that are not assigned any ICD diagnosis code. Additional challenges arise when rare diseases that do not lend themselves to a standard pre-/post-index study. This workshop will provide guidelines for developing algorithms to identify rare and ultra-rare disease patients, methods for defining study timeframes, measures and outcomes, and examples of applying these guidelines in real-world HEOR studies. Examples will be drawn from HEOR analyses, including ultra-rare genetic disorders amenable to gene and other therapies.
Topics to be addressed include: 1) methods for patient identification when disease specific diagnosis codes are not available or insufficient; 2) study timeframes for diseases where patients cannot be followed for a standard pre-/post-index period; 3) assessing outcomes in the absence of clinical data; 4) calibrating study design and results to reflect clinical input. At the start of the presentation, the speaker will ask the audience for examples of challenges they face in HEOR research in rare and ultra-rare disease. After the main presentation, the speakers will provide two example diseases and discuss and hold an interactive discussion on how session learnings could be applied to a retrospective study of these diseases.
Discussion Leaders
Naomi Sacks, PhD
PRECISIONheor, Boston, MA, USA
Discussants
Philip L Cyr, MPH
Precision Value & Health and PrecisionADVANCE, Boston, MA, USA
Bridget Healey, MPH
PRECISIONheor, Boston, MA, USA
Bridget Healey is a researcher and data scientist with experience in the design and analysis of longitudinal real-world evidence studies in multiple therapeutic areas, including rare and ultra-rare diseases. She has expertise using data sources in retrospective studies, including survey, EHR, and clinical trial data. Most recently, she is a co-author of a JCP article (under review) "Self-Reported Healthcare Resource Utilization in Clinical Trials." Ms. Healey has a Master of Public Health degree with concentrations in biostatistics and epidemiology from Boston University
Josh Noone, PhD
Novo Nordisk, Mooresville, NC, USA
Josh serves as the director of GLP-1 Diabetes research strategy at Novo Nordisk Incorporated covering the US market. Prior to NNI Josh worked in HEOR at Grifols Pharmaceuticals working on the immunoglobulin rare disease portfolio as well as several years in consulting working on both rare and chronic diseases.
Ajay Sheshadri, M.S, M.D
University of Texas MD Anderson Cancer Center, Houston, TX, USA
Dr. Ajay Sheshadri is an Associate Professor of Pulmonary Medicine at the University of Texas MD Anderson Cancer Center and specializes in the diagnosis and treatment of lung graft-versus-host disease after hematopoietic transplantation, also known as bronchiolitis obliterans syndrome.
Student Network Session
Student Research Spotlight
Live
5 Student Presenters were chosen based on receiving the highest scored abstracts for Virtual ISPOR Europe 2021. Students will present a 5-minute elevator pitch about their research. After each presenter finishes there will be 5-minute Q&A with the session moderators.
Presentations Healthcare Resource Use of People Who Use Illicit Opioids in England: A Retrospective Cohort Study Using Clinical Practice Research Datalink (CPRD) and Hospital Episode Statistics (HES) Speaker: Naomi van Hest
Cost Effectiveness Analysis of Pembrolizumab Monotherapy in Advanced PD-L1 ≥50% Non-Small Cell Lung Cancer (NSCLC) in the Irish Setting Speaker: Ryan Wong Chu
Measuring Patient Preferences: Comparison of Five Discrete Choice Modeling Methods Speaker: Mengqian Zhang
Impact of Shared Decision Making on Outcomes Among Patients with Pain: A Systematic Review & Meta-Analysis Speaker: Srujitha Marupuru
Cost-Utility Analysis of Empagliflozin in Chronic Heart Failure with Reduced Ejection Fraction: A UK Healthcare System Perspective Speaker: Yan Zhi Tan
Moderators
Khalid Kamal, MPharm, PhD
West Virginia University, Morgantown, WV, USA
Khalid M. Kamal, M. Pharm., Ph.D. is a Professor and Chair of the Department of Pharmaceutical Systems and Policy at West Virginia University School of Pharmacy, Morgantown, WV. Dr. Kamal's primary research and teaching interests have been pharmacoeconomics, patient-reported outcomes research, research methods, and improving quality of care using real-world data sources such as electronic medical records and specialty pharmacy data. He has been a Visiting Professor teaching pharmacoeconomics and decision modeling courses at institutions such as Kobe Gakuin University in Kobe, Japan and King Saud University in Riyadh, Saudi Arabia. Dr. Kamal serves as the Chair (2019-2023) of the Faculty Advisory Council within ISPOR.
Zeba M Khan, RPh, MS, PhD
Rutgers University, Piscataway, NJ, USA
Speakers
Ryan Chu, ,
University College Cork, Cork, CO, Ireland
Srujitha Marupuru, PharmD, MS, PhD student
University of Arizona, TUCSON, AZ, USA
Yan Zhi Tan, MSc (Health Economics and Management), BSc (Pharm) (Hons)
Erasmus University Rotterdam, Brussels, Belgium
Naomi van Hest, MSc, BSc
Costello Medical, London, United Kingdom
Naomi is a Consultant Health Economist at Costello Medical, leading the development of health economic models for reimbursement and market access, particularly in oncology. She has contributed novel research to ISPOR over the past 4 years, ranging from cost-effectiveness analyses, to survival extrapolation, reducing uncertainty and expert elicitation. Naomi has completed a Master's in health data analytics from University College London, and studied her undergraduate degrees in Australia.
Mengqian Zhang, MSc
Tianjin University, Tianjin, 12, China
11:00 - 15:00
Poster & Exhibit Viewing
Live
Stop in to learn about the latest technology, services, and devices and to connect one-on-one with exhibitors and sponsors during our Exhibit Viewing Hours. Be sure to reserve some time to view the latest research in HEOR through our virtual poster gallery. Browse, comment, and discuss our posters at any time while our virtual platform is active.
12:30 - 13:30
ISPOR Forums
Do ICERs Support the Digital Revolution? Forum Discussion - ISPOR Digital Health Special Interest Group
In the era of digital transformation, multiple trends are helping to shape our healthcare systems. Behavioral data enable patients to be interconnected with different health technologies. Patients are taking more active role in decision-making processes both as data providers and sometimes even as sole payers. The sociodemographic predictors of diseases reveal themselves in the process of data collection this allowing us to better understand that the treatment outcomes are sometimes dependent on multiple factors occurring outside of the healthcare system. Beyond machine learning and big data analysis, algorithms drive integrated disease management and personalized treatment pathways. Therefore, we can no longer simplify cost effectiveness evaluation to merely comparing health technology A versus health technology B. Instead, we also need to consider drawing comparisons to treatment pathway A versus treatment pathway B. In the digital era of integrated healthcare models, the question emerges as to whether we should consider moving into new methodological approaches accommodating multi-stakeholder interests? Do clinical outcomes reflect fully the value of digital health interventions? Do definitions of digital health interventions reflect their value? Do incremental cost-effectiveness ratios (ICERS) as we calculate them today support the digital revolution? Building on the conclusions of a recent systematic review, leaders of the ISPOR’s Digital Health Special Interest Group will discuss topics pertinent to the technology assessment of digital health innovations and engage the audience in an interactive debate. Participants will be encouraged to share their experiences, perspectives on to whether we should consider moving into new methodological approaches accommodating multi-stakeholder interests and provide insights for additional methodologies and considerations. 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 economic evaluation of digital health solutions.
Moderators
Carl V. Asche, PhD, MBA, MSc
University of Illinois College of Medicine, Peoria, IL, USA
Research Professor at the University of Illinois. Academic work has comprised of authoring over 100 papers appearing in the medical and economic literature. Currently serves on numerous health economics-focused boards and committees, including Pharmacoeconomics (PEC) journal editorial board, Agency for Healthcare Research and Quality (AHRQ) special emphasis panel on health information technology, and a variety of pharmaceutical industry advisory boards. Current research is funded by a variety of state/federal agencies including the National Institute on Health (NIH) and the Department of Defense (DoD).
Speakers
Katarzyna Kolasa, PhD
PAREXEL and Kozminski University, Warsaw, MZ, Poland
Driven with the passion to health economics, I have more than 20 years of academic and industry experience in the field of healthcare. Holding various Regional and Global leader-ship positions, I have worked with the pricing & reimbursement challenges in the pharma and medtech industry.
Since 2018 I am a Professor at the Kozminski University leading Health Economics & Healthcare Management Division (HeM). My PhD was titled “The principle of equity and social justice with respect to financing of health service in Poland and Sweden”. My habilita-tion book „Optimal allocation of healthcare resources” was published in 2017.
My extensive knowledge in the field of health economics was acquired at the University of York, University of Lund, and University of Bergen as well as during the International Doc-toral Courses in the Health Economics and Policy organized by the Swiss School of Public Health.
At the Kozminski University, I am the leader of the International Master Program Health Economics & Big Data (HEBDA) financed by the EU research grant of the National Center for Research and Development which was listed as the best project in the POWER 2018 call. I collaborate closely with the health economics partners from the University of Lund, the University of Athens, University of Illinois, University of York and the University of Ap-plied Science in Berlin as well.
My first practical skills in the field of health economics were developed during six years employment contract at the Kalmar County Council in Sweden. After that, I worked in both global and regional Health Economics & Outcomes Research (HEOR) functions at different pharmaceutical companies for ten years in total. At AstraZeneca and BiogenIdec. I held Global and European positions respectively. At Bristol Myers Squibb and Lundbeck I lead Market Access teams in Central Eastern European (CEE) and Nordic Region. In the last four years, I gained experience with the pricing &reimbursement challenges in the field of medi-cal devices. Since 2017, I have supported Straub Medical as Global Market Access Lead. Before that, I was employed as a Senior Sales Director responsible for HEOR at GE Healthcare.
In October 2020, I was nominated to ISPOR’s Health Sciences Policy Council (HSPC). I am chair elect of ISPOR Special Interest Group Digital Health as well.
Guest Editor of Special Issue "Quest for Value Drivers of Digital Health Solutions in the Post COVID-19 Era" at International Journal of Environmental Research and Public Health (ISSN 1660-4601) (IF 3.2)
Currently I am leading the project of the optimal allocation of CT scanners for the Polish Ministry of Health. It is the adaption of evolutionary algorithms to analyze more than 120 mln of historical CT procedures. In addition, my research agenda relates to the value frame-works of medical devices and digital health interventions. I am leading the project of the development of integrated healthcare model for oncological care based on patients’ prefer-ence for the Polish HTA agency.
Laura Vinuesa, D.V.M., MSc
Clarivate, London, LON, United Kingdom
Laura Vinuesa, D.V.M., M.Sc., is a senior business insights analyst on the Oncology Market Assessment team at Clarivate. She has authored reports in various oncology indications, covering both solid tumors and hematological malignancies. Previously, she was an EU Market Access Analyst in the Global Market Access Insights Team at DRG. She has expertise in market access, pricing and reimbursement, health technology assessment, and health policy. Prior to joining DRG, she was a Market Access Analyst at Pfizer Spain. Dr. Vinuesa obtained her veterinary degree from Complutense University in Madrid and her master’s degree from EPHOS Business School in Madrid.
Zsombor Zrubka, MD, MBA, PhD
Óbuda University, Budapest, PE, Hungary
Zsombor is an associate professor and the head of the Health Economics Research Center at the University Research and Innovation Center at Óbuda University, Budapest, Hungary and a researcher at the Corvinus Institute for Advanced Studies, Corvinus University of Budapest, Budapest, Hungary
Is “15% of the Scale Range” Universally Applicable to Define a Small, But Relevant Individual Change for Patient-Reported Treatment Benefits?
In 2020, the Institute for Quality and Efficiency in Health Care (IQWiG) updated its general methods on benefit assessment. An important recommendation, within this update, was the inclusion of considering the score scale range of an instrument when defining a threshold to designate important individual-level changes. Specifically, the threshold should be at least 15% of the scale range. The ISPOR Clinical Outcome Assessment Special Interest Group has developed a paper discussing the possible implications of this recommendation, such as not taking into account clinical practice standards, contradicting with regulatory recommendations, raising the bar for patients to get access to preferred medicines and reducing the scientific value of established minimal important difference (MID) research for 20 years. This forum will begin with Dr. Wieseler presenting the rationale and the advantages of this unique definition of small but relevant individual-level changes applied to all PROs. The forum will then discuss pros and cons from different perspectives: with Dr. Bjorner discussing an instrument developer’s perspective and Dr. Bennett, the industry perspective.
Moderators
Olivier Chassany, MD, PhD
Patient-Centered Outcomes Research, University Paris-Diderot, Paris, France
Professor of Therapeutics (Health Economics Clinical Trial Unit, AP-HP Paris hospitals, France), specialist in gastroenterology, with a long experience in developing Patient-Reported Outcomes (PRO) questionnaires. Involved for more than 20 years in the expertise of dossiers for EMA and French Drug Agency and for more than 30 years in Ethics Committees. Co-author of the EMA Reflection Paper on Health-Related Quality of Life. Deputy director of an academic research team on epidemiology and PRO (Université de Paris, Inserm). Current chair of the ISPOR SIG Clinical Outcomes Assessment (COA).
Speakers
Bryan Bennett, PhD, Cpsychol
Bristol-Myers Squibb, Uxbridge, United Kingdom
Dr Bryan Bennett is currently employed by Bristol Myers Squibb as Asset/Indication Lead in the Patient-reported Outcomes Assessment team. He has been involved in Clinical Outcomes Assessments for more than 20 years in clinical practice, academia, consulting and industry.
Jakob Bjorner, MD, PhD
QualityMetric Incorporated, LLC, Virum, Denmark
Jakob Bue Bjørner specializes in applying modern psychometric theory to the measurement of health outcomes. Jakob has worked with the SF-36® and other patient reported outcomes questionnaires for more than 25 years. Jakob has been Chief Science Officer at Qualitymetric since 2004. Jakob co-developed Qualitymetric’s Dynamic Health Assessment (DYNHA®) system, as well as numerous computerized adaptive health assessments of generic and disease-specific health outcomes. Jakob earned his MD and PhD from the University of Copenhagen. He is a professor of epidemiology at the Danish National Institute of Occupational Health and honorary professor at the University of Copenhagen.
Beate Wieseler, PhD
Institute for Quality and Efficiency in Health Care (IQWiG), Cologne, Germany
Beate Wieseler is Head of the Department of Drug Assessment at the German Institute for Quality and Efficiency in Health Care (Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, IQWiG). At IQWiG she is responsible for the scientific assessment of pharmaceuticals, the development of assessment methods as well as the Institute’s collaboration with German and international external parties. Prior to joining IQWiG in 2005, Beate Wieseler worked in clinical research and regulatory affairs for about 10 years.
Issue Panels and Workshops
Public Procurement of Innovation: Improving Outcomes, Quality and Value to Patients, Healthcare Systems and Society Illustrating Mitmeva Case
Live
ISSUE: Public authorities in EU spend 14% of GDP purchasing services and products. If spent strategically on innovative solutions, public procurement can contribute to higher quality and sustainable public health and lead to economic and social benefits. Public Procurement of Innovation provides a framework for procuring to deliver better outcomes for patients and society while increasing efficiency of public spending. However nowadays strategic procurement possibilities are not sufficiently used as still 55% of procurement uses lowest price as only award criterion. Public procurement directives leave buyers entirely free to opt for purchases based on quality-based criteria which may include social, environmental, innovative, accessibility or other qualitative criteria. There is a need to further debate and deploy PPI to benefit patients, healthcare systems and society.
OVERVIEW: Panel will debate on opportunities PPI brings for improving quality of care. Moderator will provide an overview of current state of the art experiences and pose key questions for panelists to debate: What are the benefits of PPI for the administration? What barriers exists within the public administration and private sector? What are best practices for ensuring appropriate usage of PPI that could overcome these barriers? How stakeholders should engage to lead a change on the way innovation is procure (from price to value)?. Ramón Maspons will provide insight on how PPI should be prioritized to innovate on healthcare delivery from the public administration, from the perspective of a region with large experience on PPI. Laura Sampietro will represent HTA by presenting a PPI case to improve the quality and efficiency of clinical management of patients with Aortic Stenosis - MITMEVA. Andrea Rappagliosi will provide insight from the MedTech developing Industry perspective to inform how PPI can support invest smart decisions to delivering excellence in healthcare, describing the experience of the industry with the PPI case MITMEVA.
Moderators
Carmen Laplaza Santos, MD
European Commission, Brussels, Belgium
Panelists
Ramon Maspons, MSc
Agency for Health Quality and Assessment (AquAS), Departament de Salut, Generalitat de Catalunya, Ministry of Health, Government of Catalonia, Barcelona, Spain
Andrea Rappagliosi, MSc
Edwards Lifesciences, Nyon, VD, Switzerland
Andrea Rappagliosi
Vice-President, Public Affairs EMEA, Canada and LATAM
Edwards Lifesciences
Nyon, Switzerland
Andrea is currently Vice-President Public Affairs EMEA, Canada and LATAM at Edwards Lifesciences and member of the regional Executive Leadership Team. He is leading the Market Access, Government Affairs, Communication and Patients Advocacy engagement for the company.
Born in Rome, Andrea received a law degree from the University of Rome, La Sapienza. Andrea began his professional career in the Italian Senate. Recently he worked in the Sanofi group as VP Public Affairs Europe and before as VP, Market Access, Health Policy and Medical Affairs at Sanofi Pasteur MSD. At SPMSD Joint Venture he was member of its Executive Committee from 2012 to 2016. Before, he worked at Baxter Healthcare, Serono International and GSK in different European and International positions in the public affairs and market access policy area.
In the last ten years, Andrea represented the healthcare Industry in several European EU Commission and Member States initiatives such as the HTANetwork, EUnetHTA Joint-Action 3, and the EU Active & Healthy Aging Innovation Partnership and the EU Joint Action on Vaccination. He was President of EuropaBio the European Association of the biotech Industry (2009-2011), and of Vaccines Europe, the European Vaccines manufacturers association (2013-2017). Andrea is a founding member of the Global Policy Forum at HTAi – the scientific and professional society for all those who produce or use health technology assessment (HTA)
T: + 41 79 5968808
E: andrea_rappagliosi@edwards.com
Laura Sampietro-Colom, MD, PhD
Hospital Clinic Barcelona, Barcelona, Spain
Dr Laura Sampietro-Colom is the Deputy Director of Innovation and Head of the Unit for the Assessment of Innovations and New Technologies at the Hospital Clinic of Barcelona (Spain). In her everyday work she is supporting clinicians to prove the value for their clinical practice of both health care technologies they are developing and for those new technologies that want to be introduced in the Hospital. She is also involved in Innovative Public Procurement and Value Based Purchasing with doctors and procurers at hospital
Rethinking Our Pricing Perspective: Do We Realize How Price Components Impact the Affordability of Drugs?
Live
ISSUE: The WHO guideline on country pharmaceutical pricing policies 2020 covers ten pricing policies applied to set, manage, or influence pharmaceutical products' prices. We found that two neglected policies in research, policy discussion and public forum discussion like ISPOR, are: (a) Mark-up regulation across the pharmaceutical supply and distribution chain; and (b) Tax exemptions or tax reductions for prescription pharmaceutical products.
A more comprehensive view of prices is required to address the affordability challenge of pharmaceuticals. A clear understanding of individual price component rules and regulations and a cross-country comparison could help us understand healthcare systems' real impact. How do different countries compare? How are the differences impacting the actual cost of medication to patients and health insurers in different countries?
OVERVIEW: This panel will debate the challenges and opportunities posed by distribution and taxation price component regulations across various countries that regulate how pharmaceutical distribution mark-ups and taxes (e.g., through VAT) are implemented. Using price component simulations based on country-specific rules and price logic, the panel will explore the consequences of the different approaches to distribution and taxation. Giovanny Leon will provide an overview of the current landscape of price components regulation across markets worldwide and moderate the panel discussion by posing some critical questions for the panelists to debate: (a) What best practices exist to ensure the more affordable cost to health systems or patients? (b) Which distribution and/or taxation regulations are unnecessary, impacting the total cost of medicines for healthcare systems and patients, and why? (c) Are there meaningful differences across diverse price ranges? And (d) Should high-budget impact drugs be handled differently? Panos Kanavos will reflect from the perspective of higher-income countries. Zoltán Kaló will adopt a lower-income country perspective. Christophe Carbonel will represent the perspective of the pharmaceutical industry.
Moderators
Giovanny Leon, MsC, MBA
Novartis Ag, BAsel, BS, Switzerland
Giovanny Leon
Pricing and Access Director, Latin America & Canada
Novartis AG, Basel. Switzerland
Giovanny is a senior pharmaceutical executive with intensive experience in various management roles in LatAm and the Novartis Global HQ for almost 30 years. He is passionate about rethinking pricing, access, and affordability. With the ambition to help reimagine healthcare—a lifelong learner on leadership and innovation.
His current areas of research and practice are:
1) Rethink price transparency and affordability, reshaping policy debate of price impact on access.
2) Drive early understanding and readiness to respond to the health care system's needs, including developing the organizational capabilities to ensure an environment proactively favoring patient access to innovation.
He has an MBA and a master's in marketing and commercial management from Universidad Complutense, Madrid. He has completed several Management & Leadership programs from the University of Cambridge, London Business School, Columbia University, IESA, Universidad de Los Andes, ITAM, ESPM, IAE.
Panelists
Christophe Carbonel, MBA
Novartis AG, Basel, Switzerland
Christophe is the Head of Global Pricing, Negotiations and Governance at Novartis Pharma. He has more than 20 years of experience in Access: in global access strategy as Head of Access and HEOR of a franchise, as Head of access in Japan and now implementing access strategies worldwide. Christophe is also committed to providing better access in lower income markets in line with the Novartis Access principles.
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.
Panos Kanavos, PhD
The 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.
Can Single-Arm Trials of CAR-T Therapies Meet the Evidence Requirements of Global Health Technology Assessment Agencies? Are There Innovative Ways to Address the Comparative Evidence Needs?
Live
ISSUE: Many CAR-T therapies are being developed for late-stage rare hemato-oncology diseases, where there is a significant unmet need. Under these conditions, RCTs may not be possible due to lack of clinical equipoise leading to ethical concerns due to emerging data on the effectiveness of these types of therapies; significant unmet need where alternative treatment options are not able to deliver the efficacy to prolong disease progression, improve quality of life and symptoms of disease, and extend survival. Many HTA agencies still have a strong preference for RCT data for comparative effectiveness. In the absence of evidence from RCTs, indirect treatment comparison (ITC) methods can be used to generate comparative data, however, these methods are not always accepted by HTA agencies. There is an urgent need to explore this issue from a multi-stakeholder perspective
OVERVIEW: This panel will debate whether single-arm studies of CAR-T therapies can meet the evidence requirements of global HTA bodies. Dr. Satish Valluri will moderate the panel and provide an overview of the current landscape of HTA requirements on comparative evidence for CAR-T therapies, as well as pose key questions for the panelists to debate, including:
What are the challenges with conducting RCTs for CAR-T therapies? What are alternative approaches to RCTs that can generate robust comparative evidence? What role can RWE play in these situations? Under what clinical and methodological conditions is it sufficient to generate comparative data using RWE to justify the value of CAR-T therapies? Which methods are most acceptable from an HTA perspective, and what are the challenges? International experts from the US, Canada, and Europe will each describe their unique perspective from industry, HTA and academia on ITC methods to develop comparative evidence in support of HTA submissions involving CAR-T therapies. Target stakeholders include HTA agencies, patient advocacy organizations, and pharma/biotech manufacturers.
Moderators
Satish Valluri, PhD
Janssen Pharmaceutical Companies, Raritan, NJ, USA
Satish Valluri is currently the head CAR-T global market access at Janssen Inc. He is accountable for developing and executing an integrated market access strategy for cell therapies including generating real-world and comparative effectiveness evidence, value proposition development, and reimbursement. He has 15 years of progressive experience across different geographies in strategy development, patient access, health economics, outcomes research, and pricing & reimbursement for both in-line and pipeline medicines. Satish has a PhD degree in health services research from the University of Maryland at Baltimore.
Panelists
Chris Cameron, MSc, PhD
CRG-EVERSANA, Sydney, NS, Canada
Clare Hague, PhD
Cilag GmbH International, Zug, Switzerland
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.
Zack Pemberton-Whiteley, BPTC LLM
Leukaemia Care, Worcester, United Kingdom
A Best Practice Guideline for Backlog Modelling to Inform Policy Decisions During a Pandemic and Beyond
Live
PURPOSE: COVID-19 pandemic has impacted healthcare services worldwide. As a strategy to increase capacity during the pandemic, hospitals cancelled elective and non-urgent surgery. This resulted in many patients waiting for operations and an ever-increasing backlog of elective surgery. Delays in delivering healthcare services is likely to increase patient morbidity, mortality and associated health care costs. Evidence-based policy decisions regarding backlog management require forecasting and optimization models. These models should consider hospital and national perspectives. Several optimization outcomes can be considered, including backlog size, mortality, quality of life (QOL) and costs. For evidence-based policies, these capacity optimization models should be informed by data such as backlog size, capacity, as well as the impact of interventions on capacity and outcomes in terms of life expectancy, QOL and costs.
This workshop aims to propose a best practice guideline for modelling backlogs considering relevant optimization perspectives, optimization outcomes and relevant inputs needed for evidence-based backlog/capacity models to inform hospital and national policy interventions. It should inform local and national policymakers on minimal future data requirements and models needed to optimize population health from a hospital and health care perspective during and post-COVID-19.
DESCRIPTION: First 10 minutes: Project’s background and methodology. Discussion on existing policy frameworks at hospital and national levels relating to backlog management
Second 10 minutes: Evaluate the evidence on backlog size (caused by COVID-19) and its impact in terms of life years lost or quality-adjusted life years. Next 10 minutes: Presentation of existing models that optimize backlog reduction and policy strategies, according to different parameters of optimization. Next 20 minutes: Propose a best practice guideline for backlog modelling that focuses on how to best manage the backlog and the data needed to achieve population health outcomes, and discuss the relevance guideline across health systems. Final 10 minutes: Q&A
Discussion Leaders
Karin Cerri, PhD
Johnson & Johnson, Tervuren, Belgium
Karin Cerri (PhD)
Head Health Economics and Market Access EMEA Johnson & Johnson MedTech
From the beginning, Karin’s career and academic interests have focused on enabling patient access to innovative medicines and technologies. This has been shaped by the desire to learn more about how funding and access for patients is created, and how value to the healthcare system is provided, through healthcare innovation. Karin has over 20 years’ pharmaceutical and medical device industry experience.
Karin holds a Bachelor of Arts in Human Sciences from the University of Oxford, and a Master of Science in International Health Policy, Health Economics and Health Promotion and a PhD in Health Technology assessment both from the London School of Economics and Political Science. Karin has a strong background in health economic analyses and evidence generation strategies and has led and published a wide range of Health Economic and Outcomes Research studies including economic models, mixed treatment comparisons, observational studies and HTA methodologies. She is a visiting researcher at the LSE.
Karin has lived in Italy, Japan, the United Kingdom and is currently living in Belgium with her family.
Discussants
Alexander Carter, BSc(Hons), MSc
London School of Economics & Political Science, London, United Kingdom
Alexander Carter, PhD, is a senior lecturer in practice at the Department of Health Policy, London School of Economics and Political Science.
Jonathan Clarke, MA(Cantab), MB, BChir, MRCS(Eng), MPH, PhD, FRSA
Imperial College London, London, United Kingdom
Jonathan is a Sir Henry Wellcome Postdoctoral Fellow in the EPSRC Centre for Mathematics of Precision Healthcare at Imperial College London. A clinician by training, he aims to explore three pressing questions facing health systems across the world: How should clinical information be shared across the healthcare system? How will changes to clinical services affect patients and clinicians? How can we organise primary and secondary care services to make patient care safer, fairer, and more efficient? He applies network analysis to explore healthcare in the National Health Service and internationally as a complex system, and in doing so empower patients, clinicians and policy makers to make informed decisions to improve how healthcare is delivered.
James Kinross, BSc(Hons), MBBS, PhD, FRCS(Gen)
Imperial College London, London, United Kingdom
Use of Whole Disease Models and Pathway Models in Health Economic Research: Benefits and Risks
Live
PURPOSE
: To provide an overview of existing whole disease models (WDMs) and pathway models worldwide and discuss the benefits and risks of using those models from the perspective of academia, regulator and industry.
DESCRIPTION
: WDMs and pathway models are large-scale, system-level models which can evaluate multiple decision questions across an entire care pathway. Whilst these models can offer several advantages as a platform for undertaking economic analyses, the development of a WDM requires a significant initial investment of time and resources and presents additional challenges for model verification and validation. This workshop will bring together key experts from different sectors to discuss the benefits of risks of using WDMs and pathway models for academia, regulators and industry.
Workshop structure Presentation One (12 minutes): PT will give an introduction to WDM and pathway models and discuss the pilot work of applying those modelling approach to NICE clinical guidelines. Presentation Two (12 minutes): HJ will provide an overview of existing WDMs and pathway models worldwide and discuss the reusability of those models. Presentation Three (12 minutes): JC will discuss the benefits and risks of using WDMs and pathway models in the industry, using three models developed by Evidera as case studies. Discussion (12 minutes): PC will lead the discussion, drawing on his experiences of developing, updating and adapting the UKPDS model to different countries. Stakeholders likely to benefit from attending the workshop Health economic modellers, or commissioners of health economics models, who need to evaluate multiple decision questions across the entire care pathway of a given disease, or stakeholders who are interested in reusing/adapting existing models. Health policy makers and commissioners who would like to adopt a whole system approach by moving resources from treatment to prevention and early intervention and moving resources from hospital to community.
Discussion Leaders
Philip M Clarke, PhD
University of Oxford, Oxford, United Kingdom
Discussants
J. Jaime Caro, MDCM, FACP, FRCPC
Evidera, McGill University, London School of Economics, 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.
Lily Jin, MBBS, MSc, PhD
King’s College London, London, LON, United Kingdom
Dr Huajie Jin (Lily) is a Senior Lecturer in Health Economics at King’s Health Economics, King's College London. Her role at King’s involves designing and conducting economic evaluations for a range of policymakers and research organisations, including the NHS England, the National Institute for Health and Clinical Excellence (NICE), the National Institute for Health Research (NIHR), Medical Research Council (MRC), as well as voluntary sector and commercial organisations. Before joining King’s, Lily used to work on the NICE clinical guidelines for three years. Between the Year 2011-2013, Lily was an honorary Research Fellow of Cardiff University and an Honorary Research Associate of Swansea University.
Lily’s research focuses on health economic modelling, trial-based economic evaluations and systematic reviews. She has conducted economic evaluations for a range of healthcare interventions, including screening tests, diagnostic tests, drugs, radiotherapies, haemodialysis and service-level interventions, such as training for GPs, and liaison modes between primary care and secondary care services. Her work has been published in journals such as “The Lancet Psychiatry”, “JAMA Network Open”, “PharmacoEconomics” and “Value in Health”. She was the principal health economist of three NICE clinical guidelines and three NICE Medtech innovation briefings (MIBs).
Paul Tappenden, MSc, PhD
ScHARR - University of Sheffield, Sheffield, United Kingdom
An Introduction to Patient Experience Mapping for the HEOR Community
Live
PURPOSE:
This workshop will provide an introduction to patient experience mapping for researchers. The speakers will introduce the Patient Experience Mapping Toolbox (PEMT), which includes data collection (interview guide, visual aid), participant enrollment (plain language consent form, sample screening guide), and project management (coordinator and interviewer guides) tools available free for public use. The speakers will describe 1) how the tools can be adapted and used by researchers to understand chronic disease patient experiences before diagnosis, while receiving a diagnosis, and after diagnosis; and 2) how outputs can be applied to enhance patient centricity of clinical research and care delivery.
DESCRIPTION:
A thorough understanding of patient experiences from pre-diagnosis to living with the condition while actively undergoing treatment or symptom management is key to developing patient-centered health research, policy, medical product development, and care delivery. This includes but is not limited to understanding the impact of a condition on the patient and their family, treatment options/preferences and desired outcomes. Dr. Bell will moderate and describe why a comprehensive understanding of patient experiences is key to advancing the patient centricity of health economics and outcomes research (10-minutes). Dr. Oehrlein will introduce the PEMT, including an overview of how to the tools were developed and how they can be adapted for different types of research (15-minutes). Dr. McElwee will describe why patient experience mapping can benefit from more standardization and describe opportunities to incorporate results from patient experience mapping exercises into drug development (10-minutes). The speakers will then have an audience Q/A (25-minutes). The audience will learn how to customize the tools in the PEMT for their research needs. The audience will be engaged via polling questions throughout the workshop (example question below):
If you have experience with patient experience mapping, were your maps developed by interviewing patients and/or the peer-reviewed literature?
Discussion Leaders
Stacie Bell, PhD
National Psoriasis Foundation, Portland, OR, USA
Discussants
Newell McElwee, PharmD, MSPH
Boehringer-Ingelheim, Ridgefield, CT, USA
Elisabeth Oehrlein, PhD, MS
National Health Council, 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.
13:45 - 15:00
Plenary Session
Broader Value Assessments for Special Populations and Technologies: Are We Closing a Gap or Widening a Divide?
Live
Special technologies such as cell and gene therapies, bring emerging opportunities for meeting the needs of patients facing exceptional challenges including rare and seriously debilitating or life-threatening diseases. Broader value elements previously recognized by health economists – such as productivity, education, health equity, and risk preferences – are shown to be relatively important for rare diseases and special technologies. Why does this matter? What broader value elements are prioritized by patients and the public? Which are currently (not) considered in HTA and reimbursement decisions, and why? What at are the real-world implications of consider broader value elements, and what are the myths of doing so? This session will consider emerging needs and new frontiers in broader value assessment. World leading experts will discuss what is needed from HEOR experts, industry, HTA and payers to generate the willingness, ability, and evidence for broader value assessment of special technologies in special populations.
*Speakers to be added as confirmed!
Moderators
Lotte Steuten, Prof. PhD, MSc
OHE, City, University of London, London, 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 health economics and HTA field for >15 years in various academic roles and executive functions (non-profit and for-profit).
Lotte’s research speciality is decision analysis, focusing on the development and application of health economic analysis and health technology assessment (HTA), with the aim to accelerate patient access to high value health care services and treatments. She has co-authored >100 journal publications and reports.
To translate research into better decisions, she works effectively with pharmaceutical industry, technology assessors, payers and policy makers, (academic) researchers, clinical and patient representatives as well as capital investors. Her international career-path, including the UK, the US and the Netherlands, has provided her with deep insights in the fundamental differences and commonalities between the role of health economic research and HTA policies and practices in different healthcare systems.
In her current role at the Office of Health Economics she is responsible for the research-led consulting program, maintaining OHE's stellar reputation for objective, innovative and high-quality research and analysis globally, and meeting its charitable objectives.
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. Prior to that she co-founded Panaxea, a healthcare research consultancy, and served as its CEO and CSO from 2010 to 2018. As an active member of ISPOR for over 10 years, Lotte serves at the Board of Directors, is Co-Chair of the Virtual ISPOR Europe 2021 meeting, and contribute(d) to their various Taskforces, Shortcourses, Committees, and diversity initiatives.
Speakers
Avril Daly, Business and International relations
Retina International, Zürich, Switzerland
Annie Hubert
ESAH Biopharma Consulting, Brussels, MA, Belgium
Annie works as a consultant in European government affairs, market access and regulatory affairs for medicinal products, with specific expertise on advanced therapies. In May 2021, she joined FIPRA International as a Special Advisor for Healthcare. In the last 10 years, Annie has led different initiatives in the field of advanced therapies. Annie’s advocacy efforts contributed to regulatory improvements for ATMP companies in Belgium and she organised the first European ATMP Investor Day meeting in Brussels. Between 2014 and 2020, Annie served as Senior Director, European Public Policy at the Alliance for Regenerative Medicine. As part of her work at the Alliance for Regenerative Medicine, she has been involved in numerous activities and contributions to improving the policy environment for ATMP developers in Europe.
Prior to consulting, Annie gathered nearly 30 years of experience across various positions working for several biopharmaceutical companies, including Amgen, Pfizer and GSK. She has been an active member in various industry associations and has led industry initiatives in the areas of biotechnology and Health Technology Assessment.
Annie holds a Master in Pharmacy (University of Louvain, Belgium) and has been qualified as an industrial pharmacist since 1983. She has lectured in different educational courses for healthcare professionals and students in Belgium. Annie is a Belgian national and along with her native French speaks English and Dutch.
Gillian Leng
NICE, London, United Kingdom
Professor Gillian Leng CBE
Gillian Leng is the Chief Executive of NICE. As a junior doctor she was struck by variations in clinical practice and this developed into a passion for using evidence to improve care. Her career has spanned research, evidence synthesis, management and healthcare.
Her aim is to transform the organisation with new methods and processes to put NICE at the forefront of evaluating new medicines, devices and diagnostics, and deliver dynamic, living guidelines.
Gillian trained in medicine at Leeds, worked on clinical trials and epidemiological research in Edinburgh, and was a public health consultant in London. She was an editor of the Cochrane Collaboration, and is now chair of the Guidelines International Network.
Peter 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.
Ben Osborn
Pfizer, Tadworth, MA, United Kingdom
After graduating from Leeds University with a BSc in Physiology and Sports Science, Ben joined Pfizer in 1998. He started out as a representative and from there his career took him through a variety of leadership roles in both Sales and Marketing. In 2007, Ben took an opportunity to move into Oncology, driven by his desire to work in a field which gave him the opportunity to combine his scientific, customer and commercial skills. He then joined the Regional EU Commercial team in Pfizer Oncology. In 2012, Ben became UK Head of Pfizer Oncology where was heavily involved in shaping UK cancer policy through his work with government, PAGs and as a co-chair of the industry body’s cancer group. During his tenure he successfully led the organisation to gain access for patients to innovative cancer medicines through both NICE and the Cancer Drugs Fund. He also sat on the ‘NHS Molecular Diagnostics in Cancer working group’ and the South London Academic Health Science Network as the industry representative. In April 2015, Ben was promoted to Chief Marketing Officer, Pfizer Innovative Health where he led the marketing organisation across Europe, Japan, Korea and ANZ. He is passionate about developing a strong organisational culture and mind set of unlocking access to ensure more patients benefit from our innovation and science. Most recently he has been appointed UK Country Manager and Managing Director for Pfizer UK. Ben is married to Nicola and they have three children. He’s also a keen runner competing as often as time allows.