* Program subject to change
Nancy Devlin, PhD
University of Melbourne, Melbourne, VIC, Australia
Nancy S. Berg
ISPOR, Lawrenceville, NJ, USA
Live
Dana Goldman, PhD
University of Southern California, Los Angeles, CA, USA
Margaret Anderson, MA
Deloitte Consulting LLP, Washington, DC, USA
Sir Andrew Dillon
National Institute for Health and Care Excellence, London, United Kingdom
Tomas Philipson, PhD, MA
White House Council of Economic Advisers, Washington, DC, USA
ISSUE: With cost containment pressures and the need to optimize health and healthcare delivery, U.S. healthcare providers/hospitals must consider economic value, in addition to clinical evidence and feasibility of adoption, to inform medical device adoption decisions. As the majority of devices do not receive additional reimbursement by payers, providers often need to absorb the costs within operating budgets. Adoption decisions are frequently deliberated by hospital Value Analysis (VA) Committees which evaluate how a device may address a current problem, what evidence exists to demonstrate efficacy and safety, cost, and feasibility of integration to justify adoption. There are no guidelines to inform such evidence development needs, and requirements can vary considerably across institutions. Traditional HEOR methods may be important sources of data; however, hospital decision-makers may not be trained in such methods and may perceive bias in evidence provided by manufacturers. There is a need for clear guidance to consolidate the type of evidence required to support provider decision-making.
OVERVIEW: This panel will debate the evidence requirements for informing VA decisions by setting and device categories, with a focus on medical devices that do not achieve incremental reimbursement.
Nicole Ferko, MSc
EVERSANA, Burlington, ON, Canada
Paul Delatore, MBA
Alcon, Fort Worth, TX, USA
Gloria Graham, DNP, RN, CVAHP
Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA
Barbara Strain, MA, CVAHP
Association of Healthcare Value Analysis Professionals (AHVAP), Charlottesville, VA, USA
ISSUE
International Reference Pricing (IRP) is applied by a substantial number of countries: using the prices for the same product from other countries to inform initial pharmaceutical prices or their revision. The introduction of IRP in the U.S. has been proposed and is under serious consideration. The likely benefits and potential unintended consequences of IRP are the subject of controversy in the U.S., and globally among stakeholders: payers, policy makers, industry, and patients—with large savings expected by some and significant harm to innovation expected by others. Additionally, U.S. IRP implementation could negatively affect access to medicines in other countries. A debate about the potential impact of U.S. is timely and should address which countries and which stakeholders would benefit from it and which ones could lose in the short term or over the long run?
OVERVIEW
Andras Incze, PhD, MBA
Baden-Wuerttemberg Cooperative State University Germany & Akceso Advisors AG, Basel, BS, Switzerland
Jaime Espin, PhD
Andalusian School of Public Health, Granada, GR, Spain
Louis P. Garrison, PhD
University of Washington, The Comparative Health Outcomes, Policy, and Economics Institute, Seattle, WA, USA
Zoltan Kalo, PhD
1) Semmelweis University; 2) Syreon Research Institute, Budapest, PE, Hungary
ISSUE: The use of real-world evidence (RWE) in healthcare decision making has progressed rapidly in the last decade, moving from ideas and concepts to the development of methods and techniques to operationalise its use. Taking the next steps on this path requires global collaborative efforts from all stakeholders involved including payers, HTA organisations, regulators, patients, academics and developers of healthcare interventions. Expert forums and Think Tanks that bring these stakeholders together in a structured way have an important role in driving this collaboration forward and accelerating it to move from ideas to actions.
OVERVIEW: In 2020, the GetReal Initiative will progress from a partnership co-sponsored by the European Union and the pharmaceutical industry to an independent multi-national self-sustainable organisation. Its mission is to facilitate the adoption and implementation of RWE in drug development and healthcare decision making. As part of delivering this mission, the Initiative established a multi-stakeholder Think Tank of 23 European and US RWE experts from regulatory and HTA agencies, industry, the clinical community, and patient organizations to develop consensus and guidance on key policy and technical challenges facing RWE generation and use, and to support the development of solutions and tools for use by all in the RWE community.
This panel, representing members from the GetReal Initiative Think Tank, will debate whether potential conflicting interests of stakeholders is helping or hindering progress in this area. Panellists will discuss the role and mechanisms of formal stakeholder collaboration in progressing the use of RWE in healthcare decision making and share their experiences of formalising and operationalising these collaborative efforts. Audience participation will form an integral part of this session.
Pall Jonsson, BSc, PhD
National Institute for Health and Care Excellence, Manchester, United Kingdom
Bart Barefoot, JD
GlaxoSmithKline, Richmond, United Kingdom
Alison Bourke, BSc, MSc, FRPharmS FISPE
IQVIA, London, United Kingdom
Nirosha Mahendraratnam Lederer, PhD, MSPH
Duke-Margolis Center for Health Policy, Washington, DC, USA
PURPOSE: This workshop will help participants to understand: a) what heterogeneity in preferences means; b) how heterogeneity can be accounted for in stated preference studies; c) how understanding preference heterogeneity can contribute to decision making; and d) how preference heterogeneity is currently accounted for in preference studies.
DESCRIPTION: Background: Stated-preference methods have become widely used as researchers and decision makers increasingly elicit stakeholders’ healthcare preferences. Preferences are typically quantified as the average trade-offs that patients, caregivers, or physicians are willing to make between benefits, risks and other treatment aspects. However, preferences may be heterogeneous within a given population and approaches to account for such heterogeneity have received more attention in recent years. While a range of guidelines and frameworks have been published to establish quality standards in preference research, they offer little direct advice on how to account for preference heterogeneity. This lack of established guidance is matched with numerous methods and approaches that have been published over the years, such that practitioners face a challenging choice.
Marco Boeri, PhD
RTI Health Solutions, Belfast, United Kingdom
Sebastian Heidenreich, MSc, PhD
Evidera, London, United Kingdom
Deborah Marshall, PhD, BSc
Rheumatology Outcomes Research, Calgary, AB, Canada
PURPOSE: To train to consider linkages between cost-effectiveness thresholds and opportunity costs, and how value assessments can be used to minimize opportunity costs for high-need patients and public health purposes.
DESCRIPTION: The U.S needs to weigh how increasing healthcare costs draw from its ability to serve many individuals. Commercial payers and Medicaid face budget constraints, yet have to manage the health of multiple priority populations. These tradeoffs about how money is spent can lead to opportunity costs for certain beneficiaries, and opportunity costs create consequences. First, payers investing in technologies with rising costs for some patients limit healthcare budgets to deliver medically necessary services for other patients, or vice versa. Second, payers can overcome these budget constraints by pushing payer costs to the point of service or to all payers entailing cross-subsidization, which may reduce demand. Third, rising costs of health services and technologies create budget constraints on public health and primary care interventions that can reduce future dependence on high-cost alternatives. Fourth, healthcare costs could withdraw funds from other services that improve social welfare, such as housing, education and employment.
Rena Conti, PhD
Boston University, Chicago, IL, USA
William Padula, PhD, MS, MSc
Mark Sculpher, PhD
University of York, York, United Kingdom
David Vanness, PhD
The Pennsylvania State University, Hershey, PA, USA
11:45AM - 12:00PM
11:00AM - 11:15AM
11:15AM - 11:30AM
11:30AM - 11:45AM
Biogen
Charles Makin, BSPharm, MS, MBA, MM
Biogen, Cambridge, MA, USA
Dana P. Goldman, PhD
Peter Neumann, ScD
Merck & Co., Inc., Boston, USA
Sue Peschin, MHS
Alliance for Aging Research, Washington, DC, USA
PURPOSE
DESCRIPTION
Federico Augustovski, MSc, PhD, MD
Institute for Clinical Effectiveness and Health Policy, Buenos Aires, Argentina
John E. Brazier, PhD
University of Sheffield, Sheffield, United Kingdom
Clara Mukuria, MA MSc PhD
A. Simon Pickard, PhD
University of Illinois at Chicago, Chicago, IL, USA
2:30PM - 2:45PM
2:00PM - 2:15PM
2:45PM - 3:00PM
2:15PM - 2:30PM
To examine trends in hospital length of stay and total costs in hATTR patients with and without Hematopoietic Stem Cell Transplant (HSCT).
METHODS: The latest available 2016 National Inpatient Sample (NIS) data set from the Healthcare Cost and Utilization Project was utilized in order to determine the number of hospital admissions for patients with NHL. Propensity score matched analysis was conducted to compare hospital LOS and costs in hATTR patients with and without HSCT. Thirty comorbidities were assessed using Elixhauser scoring. Multivariate logistic regression was conducted to assess predictor variables for LOS and costs.
RESULTS:
In 2016, there were an estimated 23,335 hospitalizations with a diagnosis of hATTR, of which 260 also had a procedure code for HSCT. The mean age was 60.6 (SD 8.4) and 70.3 (SD 13.8) in hATTR patients with and without HSCT, respectively. 46.1% and 43.3% were female in hATTR with and without HSCT, respectively. Most common comorbidities (more than 20%) were congestive heart failure (46.1%), cardiac arrhythmias (40.4%), uncomplicated hypertension (38.2%), complicated hypertension and renal failure (44.7%). The propensity score matched hospital LOS was 19.1 and 6.4, with a statistically significant difference of 12.6 days (SE 1.61, P<0.05), in hATTR patients with and without HSCT. The propensity score matched hospital charges were $255,968 and $69,640, with a statistically significant difference of $186,327 (SE $30668 P<0.05), in hATTR patients with and without HSCT. Predictor variables for hospital LOS and costs were HSCT, cardiac arrhythmias and coagulopathy.
CONCLUSIONS: hATTR patients with HSCT incur significantly longer hospital length of stay and nearly 4 times the costs compared to patients without HSCT. There is a need for better treatment management for patients with hATTR undergoing HSCT.
Sonal Singh, MD, MPH
University of Massachusetts School of Medicine, Worcester, MA, USA
Bart Heeg, PhD
Ingress-health Nederlands BV, Rotterdam, Netherlands
Miguel Hernan, MD, DrPhD
Harvard University, Boston, MA, USA
Kristian Thorlund, MSc, PhD
McMaster University, Hamilton, ON, Canada
ISSUE:
OVERVIEW:
Jason Shafrin, PhD
PRECISIONheor, Los Angeles, CA, USA
Michael Barr, MD, MBA, MACP
The National Committee on Quality Assurance, Washington, DC, USA
Anupam B. Jena, MD, PhD
Harvard Medical School, Boston, USA
Elizabeth Oyekan, PharmD. FCSHP, CPHQ
Precision for Value, Centennial, CO, USA
3:30PM - 3:45PM
3:45PM - 4:00PM
4:00PM - 4:15PM
4:15PM - 4:30PM
PURPOSE: To train audience members in the subtleties of economic modeling for healthcare service interventions, in contrast to more traditional approaches of health technology assessment for applications in pharmaceuticals and medical devices.
DESCRIPTION: Experts who focus their research and consulting efforts in applied methods such as value assessment (e.g. cost-effectiveness, budget impact) are mostly taught to focus on health technology assessment. This form of training has generated short-sightedness in the field of health economics and outcomes research whereby most experts have extensive experience modeling the value of technologies, but little experience modeling the value that health services provide by comparison. According to Shrank and colleagues (JAMA 2019) nearly $1 trillion in waste is now being produced, with more than 60% by the healthcare services sector. Therefore, it is more important than ever to retrain the ISPOR community to be cognizant, agile and prepared to conduct value assessments focused on differentiating high-value care alternatives in health services as well as technology. Using an approach developed by Padula et al. (J Patient Safety 2017), the discussants will conduct a workshop focusing on economic evaluation of health services through micro-costing, including labor-intensive services and procedures that enable cost-effectiveness and budget impact analyses, and ultimately promote the use of high-value care and reduced waste throughout healthcare.
Robert McQueen, PhD
University of Colorado, Aurora, CO, USA
Jonothan Tierce, CPhil
Monument Analytics, Colorado Springs, CO, USA
Juan Marcos Gonzalez, PhD
Duke Clinical Research Institute, Cary, NC, USA
Telba Irony, PhD
Janssen Pharmaceuticals Companies of Johnson and Johnson, Raritan, NJ, USA
Carol Mansfield, PhD
RTI Health Solutions, Research Triangle Park, NC, USA
Shelby Reed, PhD, RPh
Duke Clinical Research Institute, Durham, NC, USA
Leslie Wilson, Ph.D.
University of California, San Francisco, Hillsborough, CA, USA
5:15PM - 5:30PM
5:30PM - 5:45PM
5:00PM - 5:15PM
5:45PM - 6:00PM
OM1
Richard Gliklich, MD
OM1, Boston, MA, USA
Ying Bao, MD, ScD
Bristol-Myers Squibb, Lawrenceville, NJ, USA
Gary Curhan, MD, ScD
This symposium will introduce:
MED-CON-AVXS101-00051-GLO 03/2020
AveXis, Inc.
Omar Dabbous, MD, MPH
AveXis, Inc., Bannockburn, USA
Sean Sullivan, PhD, MSc, RPh
University of Washington, Seattle, WA, USA
ISSUE: There is growing concern that the pipeline of new antibiotics is insufficient to address the global public health challenge of antimicrobial resistance. One contributor to the dearth of new products may be that health payers’ assessments of novel antibiotics do not reflect the full value of those treatments, hence disincentivizing future drug development. Some have questioned whether quantifying health outcomes for a treated patient sufficiently captures the value of novel antibiotics, or whether additional elements of value associated with factors such as diversity, insurance, enablement and impact on infection transmission are relevant. If so, how can these be incorporated into the methods of economic evaluation based on the evidence available when products are licensed? Also, would a broader assessment of the value of new antibiotics be enough to bring more novel antibiotics to the market?
OVERVIEW: Many health systems and payers use economic evaluation methods to assess the value of new pharmaceuticals. Others use less formalised methods but, nonetheless, give focus to patients’ improved health outcomes as assessed in clinical trials. These methods may not fully capture the full value of novel antibiotics. Members of the panel will take different perspectives on this crucial issue. Nambiar will set the scene on the drug development and policy landscape for new antibiotics and the challenges associated with evidence development for regulatory approval. Sculpher will argue that a novel antibiotic’s value can be reflected in terms of health gains, but this needs to be at the population level and requires modelling and long-term evidence. Schneider will argue that a fuller value assessment necessitates consideration of the implications of antibiotic diversity and enablement, as well as resulting reductions infection transmission. Holland will argue that new value frameworks for novel antibiotics are needed to support greater investment in antibiotic R&D and improve patient access.
Sumathi Nambiar, MD, MPH
Food and Drug Administration, Silver Spring, MD, USA
Silas Holland, MA, MPH
MSD, Washington, DC, USA
Monika Schneider, PhD
ISSUE: While regulatory and health technology assessment (HTA) agencies are proactively evaluating the role of RWE in their decision-making, they are at different stages of guidance development and adoption. Their common goal is to promote high-quality RWE generation and articulate how RWE will be used in their decision-making. However, no one-size-fits-all approach to RWE generation exists; regulatory and HTA agencies have different evidence needs, different opinions on what elements are essential, and different perspectives on how RWE can inform decisions. These differences will drive the panel debate on what RWE study design and methodology is necessary for regulatory and HTA guidelines to promote high-quality and validated RWE. The panel will also discuss how manufacturers can meet disparate evidence requirements.
OVERVIEW: The moderator will present an overview of current RWE guidance from stakeholder groups, regulatory, and HTA agencies noting the similarities/differences in design and application (10 minutes). Panelists from regulatory, HTA, and industry will each deliberate on the merits and challenges of the guidances (10 minutes each). The moderator will then facilitate a debate on how the guidences impacts industry’s evidence development and what elements are most effective in promoting high-quality, transparent, and validated RWE. Stakeholders from industry, especially those generating RWE, and regulatory and HTA agencies should attend.
Nicolle Gatto, PhD
Aetion, New York, NY, USA
Hans-Georg Eichler, MD, MSc
European Medicines Agency, Wien, Austria
James Wu, MSc, MPH
Amgen, Thousand Oaks, CA, USA
Katherine Hamilton, MA (Cantab) PGCert (Health Economics)
Institute for Clinical and Economic Review, Cambridge, United Kingdom
Francois Houyez, Patient Advocate
European Organisation for Rare Diseases (EURORDIS), Paris, France
Michelle Mujoomdar, PhD
Canadian Agency for Drugs and Technologies in Health, Ottawa, ON, Canada
John Munro, BA
Wordwright Communications, London, LON, United Kingdom
10:30AM - 10:45AM
10:45AM - 11:00AM
Dina Jankovic, PhD, MSc
Office of Health Economics, London, United Kingdom
Lotte Steuten, PhD, MSc
Naomi van Hest, MSc, BSc
Costello Medical, Cambridge, CAM, United Kingdom
Jens Grueger, PhD
University of Washington, Boston Consulting Group, Freiburg, BW, Germany
Richard Willke, PhD
Jan Hansen, PhD
Genentech, South San Francisco, CA, USA
Marc Boutin, JD
National Health Council, Washington, DC, USA
Rachael Callcut, MD, MSPH
University of California, San Francisco (UCSF) Medical Center, Center for Digital Health Innovation, San Francisco, CA, USA
Nigam Shah, MBBS, PhD
Stanford University, Stanford, CA, USA
Michael Drummond, MCom, DPhil
Emily Tsaio, PharmD
Premera Blue Cross, Mountlake Terrace, USA
Kai Yeung, PharmD, PhD
Kaiser Permanente Washington Health Research Institute, Seattle, WA, USA
Raquel Aguiar-Ibáñez, MSc
Merck Canada Inc., Toronto, ON, Canada
Arielle G. Bensimon, PhD
Analysis Group, Inc., Boston, MA, USA
Jingshu Wang, PhD
Merck Sharp & Dohme Ltd, Kenilworth, USA
3:00PM - 3:15PM
3:15PM - 3:30PM
ISSUE: Real-world evidence (RWE) is crucial for supplementing the evidence from randomized clinical trials on the safety and effectiveness of medications and is rapidly becoming a key tool for healthcare decision makers. However, many published RWE studies suffer from major methodological flaws, which are known to result in considerable bias. Biased studies may lead to flawed decisions and to a lack of confidence in nonrandomized RWE. The wide variety and complexity of RWE designs and analyses makes recognizing the presence of such problems challenging for even well-trained decision-makers. Therefore, many decision-makers question the utility of RWE from nonrandomized designs.
OVERVIEW: The panel and attendees will discuss the current state of nonrandomized RWE, as well as challenges and potential paths to improve validity of RWE and identification of high-quality RWE. Dr. Graff will moderate the panel, provide an overview of current challenges with nonrandomized RWE, and pose key questions for the panelists to debate (10 min). Dr. Bykov will present an assessment of the current state of nonrandomized RWE for medication safety and effectiveness from a structured literature review, and the accompanying structured approach to the review of RWE studies that incorporates specific major methodological issues that frequently bias observational studies (10 min). Dr. Bradt will present her view on the readiness of nonrandomized RWE for clinical decision-making and how ICER assesses the quality of nonrandomized RWE (10 min). Dr. Daw will present her views on the importance of using the best available information for coverage and reimbursement decisions and her experience evaluating evidence with diverse quality (10 min). We will reserve 15 minutes at the end of panelists presentations for audience participation and further response and interaction among panelists.
Jennifer Graff, PharmD
National Pharmaceutical Council, Washington, DC, USA
Pamela Bradt, MD, MPH
Institute for Clinical and Economic Review, Boston, MA, USA
Katsiaryna Bykov, PharmD, ScD
Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA
Jessica Daw, PharmD, MBA
UPMC Health Plan, Pittsburgh, USA
ISSUE: Rapid advances in medical innovation and accompanying increases in cost have driven US healthcare decision makers to focus great attention on “value.” To provide credible and relevant information on value specific to the complex decision contexts in healthcare, however, innovation in value assessment is needed: there is a general consensus that the existing methods are simply not up to the task. But there is more debate about what methodological advancements are needed. What are the highest priorities for advancing methods? And how do we do it?
OVERVIEW: Supported by a Eugene Washington PCORI Engagement Award, the Innovation and Value Initiative convened a summit on “Defining Needs and Progress Toward Improving Methods in Value Assessment.” Participants in the full-day working meeting represented diverse perspectives – including patients, payers, health systems, employers, regulators, and researchers – and collaboratively identified priorities for methods research and outlined a research agenda for advancing value assessment methods. This panel of meeting attendees will take up unresolved questions and key areas of persisting debate. Jennifer Bright will moderate and make the general case for advancing methods in value assessment. Lou Garrison will describe the current state of the science and provide the economics perspective. Eric Stanek will advocate for the areas in greatest need of improved methods and assessment capability from the payer perspective, and Leah Howard will provide perspective from the patient community. Panelists will provide brief remarks, reserving the majority of the session for panel discussion and audience questions.
Jennifer Bright, MPA
Innovation and Value Initiative, Alexandria, VA, USA
Lou Garrison, PhD
Leah Howard, JD
National Psoriasis Foundation, Alexandria, VA, USA
Eric Stanek, PharmD
HealthCore, Inc., Wilmington, DE, USA
ISSUE: Countries in all regions of the world are facing a crisis of affordability related to the reimbursement of prescription drugs. A multitude of disruptive treatments are promising significant benefit for patients - but they come with prices that are seemingly unsustainable. Furthermore, regulators are approving drugs with lower levels of evidence, creating substantial uncertainty for payers, clinicians, and patients. Orphan Drugs, gene and cell therapies, immunotherapies and other cancer drugs, and expensive drugs used to treat more common diseases are just some of the new blockbusters that are promising to improve health outcomes, but they are also contributing to the affordability crisis.
OVERVIEW: ISPOR has created HTA Round Tables in North America, Europe, Asia, Latin America, and the Middle East and Africa as a forum for HTA experts and payers to engage in dialogue on the challenges and opportunities associated with providing sustainable access to promising new drugs. Canadian participants at the 2019 North American Round Table shared information on new approaches to HTA, price negotiation, and price regulation that are designed to improve access, appropriate use, and affordability. During this issue panel, the benefits and challenges associated with these Canadian HTA and policy initiatives will be discussed and debated. Payers in Canada (and other jurisdictions) are strongly supportive of strategies that help bend the drug cost curve while still promoting early and equitable access to promising new treatments. Conversely, patients and representatives of the pharmaceutical industry in Canada are worried that these new initiatives will impede access and in fact stifle innovation; hence they are strongly opposed to many of these initiatives, particularly the new Patented Medicine Regulations that will come into force in Canada on July 1, 2020. Because many of these initiatives are controversial, the moderator will leave time for audience participation and debate.
Mitch Moneo, Bachelor of Journalism and Communication
Government of British Columbia, Victoria, BC, Canada
Brent Fraser, B.Sc. in Pharmacy; MBA
CADTH, Toronto, ON, Canada
Sang Mi Lee, B.Sc. in Pharmacy
pan-Canadian Pharmaceutical Alliance (pCPA), Toronto, ON, Canada
Tanya Potashnik, MA
Patented Medicine Prices Review Board, Ottawa, Canada
Elektra Papadopoulos, MD, MPH
U.S. Food and Drug Administration, Silver Spring, MD, USA
Bray Patrick-Lake, MFS
Evidation Health, San Mateo, CA, USA
Ernesto Ramirez, PhD
Kalahn Taylor-Clark, PhD, MPH
Sanofi, Washington, DC, USA
Winson Cheung, MD, MPH
Department of Oncology, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada
Alind Gupta, PhD
Lighthouse Outcomes, Toronto, Canada
Nicholas Mitsakakis, PhD
University of Toronto, Toronto, ON, Canada
Ryan Walton, MPH
AstraZeneca Canada, Mississauga, ON, Canada
Kimberly Westrich, MA
Alan Balch, PhD
Patient Advocate Foundation, Hampton, VA, USA
Karen Fields, MD
Moffitt Cancer Center, Tampa, FL, USA
Theresa Schmidt, MA
Discern Health, a W2O Company, Baltimore, MD, USA
6:00PM - 6:15PM
6:15PM - 6:30PM
Nancy Dreyer, MPH, PhD
IQVIA, Cambridge, MA, USA
Irena Guzina, MPH MSc
HAS Agency, La Plaine Cedex, France
Eoin McGrath, BA
European Bone Marrow Transplant Registry, Barcelona, Spain
Hannah Patrick, FFPHM
PURPOSE: This interactive workshop will examine the various indirect comparison methodologies that are available. Commonly used ITC methods such as network meta-analysis (NMA) and matching-adjusted indirect comparisons (MAIC) will be discussed using real-world case studies. Discussion leaders and international experts in ITC from Canada, US and Europe will describe methods to develop and implement global ITC strategies to support HTA and reimbursement submissions. The workshop will also provide guidance on selecting the appropriate ITC method to align with analytic objectives as well as data availability. It will also discuss which methodologies are commonly accepted by HTA agencies, and under what scenarios. The strengths and weaknesses of ITC approaches will also be highlighted by the panelists using real-world case studies.
DESCRIPTION: Indirect treatment comparisons are increasingly used to support healthcare decision making. Guidelines for conduct and transparent reporting of ITCs have been published but fail to discuss strategic considerations regarding selection of the most appropriate indirect comparison method to align with analytic objectives and in considering the types of data available to the research team. This workshop will describe how to develop and implement a global indirect treatment comparisons strategy to support health technology assessment and reimbursement submissions. We will present a matrix of relevant ITC methodologies geared toward guiding users to select the most appropriate ITC methodology for their technology assessment scenario based upon the number of treatments to be compared and the granularity of clinical data available (aggregate versus patient level). Commonly used indirect comparison methods such as NMA and MAIC are plotted within this schematic. Additional considerations of relevance including heterogeneity/inconsistency, feasibility of meta-regression analysis and limitations of clinical trial data will also be discussed. The strengths and weaknesses of using ITC approaches will also be highlighted by the panelists using real-world case studies, together with suggestions for future research.
Chris Cameron, MSc, PhD
EVERSANA, Sydney, Canada
Sandhya Nair, PhD
Janssen Pharmaceutica NV, Beerse, Belgium
Steven Peterson, MSc
Janssen Global Services, LLC, Horsham, PA, USA
Agata Schubert, MSc
Janssen-Cilag, Warsaw, MZ, Poland
ISSUE: Healthcare decisions are increasingly being supported by patient preference information (PPI). The current paradigm for considering PPI involves conducting “single-use” preference studies, tailored to a specific decision. While this model generates highly relevant behavioral insight, the cost and time required to conduct such studies inherently limits the decisions that can be informed by PPI. This is particularly the case with decisions early in product development, where the returns on collecting PPI are uncertain. To increase the use of PPI, there is a need to design multi-use preference studies. The panel will discuss the possibility of developing disease-level preference models, in which studies are designed to elicit preferences that can be applied to multiple decisions. The adoption of disease-level models will involve accepting a trade-off between lower resource requirements and shorter timelines but less customization. For instance, such models will require attributes that apply across a broader group of patients and attributes levels to cover broader ranges. The panel will address questions raised by such models: How diverse are decision problems, study designs and preferences within disease areas, and what is lost through adopting less customized models? What level of specificity is necessary to support decisions and does this vary between decisions and therapy areas? How broad could such a model be – indication, disease or therapy area? For which diseases would a multi-use model be most feasible? Which decisions would benefit from such a model?
OVERVIEW: Kevin Marsh will introduce the panel, illustrating the ‘single-use‘ and ‘disease-level’ PPI models. Leo Russo will provide a sponsor perspective on the value of disease-level preference models. Axel Mühlbacher will list arguments in favor of the concept of a disease level model, identify methodological challenges and potential solutions. Heather Gelhorn will discuss the practical challenges of applying such models, including patient and regulatory perspectives.
Kevin Marsh, PhD
Evidera Ltd, Newport Pagell, Great Britain
Heather Gelhorn, PhD
Evidera, Bethesda, MD, USA
Axel Mühlbacher, PhD, MBA
Hochschule Neubrandenburg, Neubrandenburg, BW, Germany
Leo Russo, PhD
Pfizer, Inc, Collegeville, PA, USA
10:00AM - 10:15AM
10:15AM - 10:30AM
Deborah Freund, MA, MPH, PhD
Claremont Graduate University, Claremont, CA, USA
Anirban Basu, PhD
University of Washington, Seattle, USA
Susan Griffin, PhD
Darius Lakdawalla, PhD
Ruslan Horblyuk, PhD, MBA, MA
AESARA, Inc., Bristol, PA, USA
Cristina Masseria, MSc PhD
Pfizer, New York, USA
Sean Tunis, MD, MSc
Center for Medical Technology Policy, Baltimore, MD, USA
ISSUE: Methods currently used to obtain the utilities used in calculating QALYs rest on a combination of value judgements eg. about whose preferences are relevant; and assumptions about the properties utilities should have. But the theoretical rationale for these specific requirements is not strong. Given the non-trivial problems that exist with those methods, is it time for a radical re-think? What would a new paradigm look like, and what alternative, practical solutions could HEOR provide to support and strengthen future use of QALYs?
OVERVIEW: Current practice in assessing utility in QALYs rests on a number of strong value judgements – and is fraught with unresolved issues (eg what methods to use? how to elicit values < 0? etc). There is understandable reluctance to depart from the current paradigm, least it undermines the use of QALYs. In this issues panel, we consider three radical ideas: Dropping ‘dead’ as an anchor; dropping the reliance on the general public; and dropping utility as the theoretical basis for methods. In each case, the panellists attempt to show how practical alternative approaches exist, that could support a new basis for 'quality adjusting' QALYs. This session aims to engage the audience in discussion and debate.
Ning Yan Gu, PhD
NYG Technologies, Santa Clarita, CA, USA
Christopher Sampson, MSc
The Office of Health Economics, London, LON, United Kingdom
Matthijs Versteegh, PhD
institute for Medical Technology Assessment, Rotterdam, Netherlands
Weihsuan Lo-Ciganic, PhD, MSPharm, MS
University of Florida, Gainesville, USA
Agustin Lopez-Marquez, MSc
nference, Cambridge, MA, USA
Daniel Sheinson, PhD
Genentech Inc., San Francisco, CA, USA
Wei-Shi (Danny) Yeh, PhD
Genentech, Inc., San Francisco, CA, USA
Robyn Bent, MS
Robyn Carson, MPH
Allergan plc, Madison, NJ, USA
Arinesalingam Gnanasakthy, MSc, MBA
RTI Health Solutions, Succasunna, NJ, USA
Annie Kennedy, BS
Innovation and Value Initiative, Washington, DC, USA
PURPOSE: As key clinical outcome assessment (COA) endpoints and preference studies used to support regulatory decisions become more common, the challenges of incorporating COA-based concepts into preference attributes are becoming apparent. Using recently published research as a backdrop, we will frame the challenges and explore potential solutions within a workshop format. The goal of the workshop is to spur open discussion of the challenges and possible solutions for this topic.
DESCRIPTION: Understanding how to incorporate concepts from COA endpoints into attributes or attribute levels within a patient preference information (PPI) study is becoming more important as the patient voice is incorporated into regulatory frameworks. Essentially, the translatability between COA scores, concepts, and items vs. preference attributes and results are important to the future interpretation of link between the PPI results and the COA outcomes. The challenge lies in adequately representing COA concepts in a meaningful manner in PPI instruments, so that the PPI results can then be used to help interpret or choose a COA endpoint in a clinical study and better enable benefit-risk comparisons between COAs and other endpoints. Previously published work incorporating a COA concept into a PPI study will be presented to set the stage for discussion. Other presentations will build on this work to discuss other considerations or possible methods for incorporating COA concepts in PPI studies. Presentations will draw from published literature on stated preferences and psychometrics, as well as relevant FDA guidance documents. Each speaker will have 10 to 12 minutes for presentation and discussion. Attendees of the workshop will benefit from participating in a discussion of a challenging topic in an evolving field with industry, academia and regulatory experts.
Fraser Bocell, PhD
U.S. Food and Drug Administration, Silver Springs, MD, USA
David Gebben, PhD
FDA, Silver Spring, MD, USA
Bennett Levitan, MD, PhD
Janssen Research & Development, LLC, Titusville, NJ, USA
Kevin Weinfurt, PhD
Duke University, Durham, NC, USA
PURPOSE:
DESCRIPTION:
Fabrizio Gianfrate, PhD, MSc
University of Ferrara, Ferrara, Italy
Judith Glennie, BScPhm, MSc, PharmD
J.L. Glennie Consulting Inc., Aurora, ON, Canada
Maximilian Hunt, BSc
CBPartners, New York, USA
Melanie Whittington, PhD
University of Colorado Anschutz Medical Campus, Liberty, MO, USA
PURPOSE: Friends of Cancer Research (Friends) convened 12 data partners in a collaborative effort, titled Real World Evidence (RWE) Pilot 2.0, to evaluate outcomes for advanced non-small cell lung cancer (NSCLC) population receiving stand of care systemic frontline therapies. This collaboration evaluated real-world oncology data including claims-based datasets, electronic health records, and data captured by prior authorization systems and cancer registries. The workshop will share learnings specific to identification of variability between data sources, the methodological considerations to address differences and align populations for comparison of outcomes, and focus on the potential path forward to harmonize data across various sources, developing a common language across real-world data sources to improve utilization of these data.
DESCRIPTION: The workshop will begin with an introduction to the Friends RWE Pilot 2.0, including an overview of data source types used, followed by:
Henry (Joe) Henk, PhD
OptumLabs, Eden Prairie, MN, USA
Pallavi Mishra-Kalyani, PhD
Yanina Natanzon, PhD
Syapse, San Francisco, CA, USA
Nicholas Robert, MD
Ontada, Irving, TX, USA
PURPOSE: Patient-generated health data (PGD), including contributions to social media sites and patient platforms, can provide a wealth of information regarding the patient experience living with a medical condition and its treatments. It may also provide clues to possible safety signals, benefits and risks, product quality and access. FDA sought to examine the extent to which PGD obtained through social listening could provide information about chronic pain patients’ (CPPs) perspectives regarding the treatment and management of their condition. A recent exploratory project undertaken in collaboration with the Inspire™ patient platform will be presented and implications of results will be discussed. This workshop will include presentations from FDA (CDRH, OMHEE) and Inspire™.
DESCRIPTION: Presentations will highlight the types of information gleaned from social listening and its potential to provide meaningful, actionable insights that can serve as evidence to inform decision making. A brief background and methodology will be discussed, as will data resulting from the following key lines of inquiry:
- Chronic pain patients’ (CPPs) descriptions of pain, its treatment and management
- Common challenges and barriers faced by CPPs, e.g., stigma, perception, health literacy, and treatment access
- CPPs treatment goals and measures of success
- Types of information shared by CPPs and the nature of these resources
- The effect of demographics on these themes
- CPPs experience with mitigating and managing dependence/addiction
Discussion of these topics will lead to greater clarity regarding the benefits and challenges of using social listening to elucidate patient perspective and experience, and its potential for use as scientific evidence in regulatory decision making.
Anne Hammer, MA
Claire Harter, MA
Inspire, Wichita, KS, USA
Christine Merenda, MPH, BSN, RN
Marina Ness, MPH
Inspire, Arlington, VA, USA
PURPOSE: Using an illustrative case example, this workshop will demonstrate how MCDA models are built and interpreted, and identify specific opportunities for researchers to incorporate patient views on value elements.
DESCRIPTION: Researchers, VA bodies, and the patient community agree that current approaches to VA inadequately capture the many dimensions of value. In particular, patients are concerned that cost-effectiveness analyses are the primary driver of VA, focusing on one data point rather than the many dimensions of value. The ISPOR VA Framework Special Task Force suggested MCDA as a promising approach to aggregate the different dimensions of value. MCDA is “a methodology for appraising alternatives on individual, often conflicting criteria, and combining them into one overall appraisal.” MCDA may improve transparency and reduce stakeholder concerns that the multi-dimensionality of patient-defined “value” has not been incorporated. Indeed, MCDA models can accommodate different perspectives by varying the weights assigned to different value dimensions. Despite the promise of MCDA, its application within health care and VA is nascent. This introduction to MCDA leverages a case example and lessons learned from a Roundtable on Patient-Centered MCDA held in February 2020. It is relevant for VA bodies, researchers, patients, students, and policymakers.
Jon Campbell, PhD
University of Colorado Anschutz Medical Campus, Hingham, USA
Elisabeth Oehrlein, PhD, MS