National Academies Press: OpenBook
« Previous: Appendix C: Workshop Two Agenda
Suggested Citation:"Appendix D: Workshop Three Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series. Washington, DC: The National Academies Press. doi: 10.17226/25352.
×
Page 161
Suggested Citation:"Appendix D: Workshop Three Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series. Washington, DC: The National Academies Press. doi: 10.17226/25352.
×
Page 162
Suggested Citation:"Appendix D: Workshop Three Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series. Washington, DC: The National Academies Press. doi: 10.17226/25352.
×
Page 163
Suggested Citation:"Appendix D: Workshop Three Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series. Washington, DC: The National Academies Press. doi: 10.17226/25352.
×
Page 164
Suggested Citation:"Appendix D: Workshop Three Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series. Washington, DC: The National Academies Press. doi: 10.17226/25352.
×
Page 165
Suggested Citation:"Appendix D: Workshop Three Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series. Washington, DC: The National Academies Press. doi: 10.17226/25352.
×
Page 166
Suggested Citation:"Appendix D: Workshop Three Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series. Washington, DC: The National Academies Press. doi: 10.17226/25352.
×
Page 167
Suggested Citation:"Appendix D: Workshop Three Agenda." National Academies of Sciences, Engineering, and Medicine. 2019. Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series. Washington, DC: The National Academies Press. doi: 10.17226/25352.
×
Page 168

Below is the uncorrected machine-read text of this chapter, intended to provide our own search engines and external engines with highly rich, chapter-representative searchable text of each book. Because it is UNCORRECTED material, please consider the following text as a useful but insufficient proxy for the authoritative book pages.

D Workshop Three Agenda Workshop Three: Application July 17–18, 2018 National Academy of Sciences Building, Lecture Room 2101 Constitution Ave. NW, Washington, DC 20418 The National Academies of Sciences, Engineering, and Medicine is convening a three-part workshop series, sponsored by the U.S. Food and Drug Administration, examining how real-world evidence development and uptake can enhance medical product development and evaluation. The workshops will advance discussions and common knowledge about complex issues relating to the generation and usage of real-world evidence, including fostering development and implementation of the science and technology of real-world evidence generation and usage. Workshop One (September 19–20, 2017) focused on how to align incentives to support collection and use of real-world evidence in health product review, payment, and delivery. Incentives need to address barriers impeding the uptake of real-world evidence, including barriers to transparency. Workshop Two (March 6–7, 2018) illuminated what types of data are appropriate for what specific purposes and suggested practical approaches for data collection and evidence use by developing and working through example use cases. Workshop Three (July 17–18, 2018) examined and suggested approaches for operationalizing the collection and use of real-world evidence through discussing “decision aids” about specific topics in study design. The decision aids are question lists developed to inform discussion around specific topics addressed at the third workshop. These discussions may help inform workshop attendees and other stakeholders about study design choices, including potential risks, costs, and reporting/transparency expectations. DAY 1: July 17, 2018 8:00 a.m. Breakfast Available Outside the Lecture Room 8:15 a.m. Welcome and Opening Remarks P REP UBLI CATI ON COP Y: UNCORR ECTED P ROOFS 161

162 EXAMINING THE IMPACT OF RWE ON MEDICAL PRODUCT DEVELOPMENT MARK MCCLELLAN, Workshop Series Co-Chair Director Duke-Margolis Center for Health Policy GREGORY SIMON, Workshop Series Co-Chair Investigator Kaiser Permanente Washington Health Research Institute SESSION I KEY CONSIDERATIONS FOR REAL-WORLD EVIDENCE APPLICATION Session Objectives: Examine how some organizations are currently considering traditional and real-world evidence. Discuss factors that may be influencing overall cost and time investment required by traditional evidence generation. Consider when non-traditional data sources may be beneficial to assess outcomes. 8:45 a.m. Update on the Innovative Medicines Initiative’s GetReal and View from the National Institute for Health and Care Excellence, United Kingdom PALL JONSSON Associate Director, Research and Development National Institute for Health and Care Excellence 9:05 a.m. Drivers of Expense and Delay ELLIOTT LEVY Senior Vice President, Global Development Amgen Inc. 9:25 a.m. Patient-Collected and -Owned Data KOMATHI STEM Chief Executive Officer and Founder monARC Bionetworks 9:45 a.m. BREAK SESSION II WHEN IS A REAL-WORLD DATA ELEMENT FIT FOR ASSESSMENT OF ELIGIBILITY, TREATMENT EXPOSURE, OR OUTCOMES? Session Objectives: Discuss potential bias-introducing steps in evidence generation from real-world data. Suggest key considerations in the data collection and evidence-generation processes that influence reliability of real-world data. Discuss how a decision aid laying out key questions and considerations might help inform current and future studies. P REP UBLI CATI ON COP Y: UNCORR ECTED P ROOFS

APPENDIX D 163 10:05 a.m. Introduction: A Proposed Framework for a Decision Aid PALL JONSSON, Session Moderator Associate Director, Research and Development National Institute for Health and Care Excellence 10:15 a.m. Looking Back: How Might a Decision Aid Inform a Real-World Example? JEFF ALLEN President and Chief Executive Officer Friends of Cancer Research 10:35 a.m. Looking Forward: How Decision Aid Might Apply to Future Studies Panel Discussion and Audience Q&A AYLIN ALTAN Senior Vice President of Research OptumLabs ROBERT BALL Deputy Director, Office of Surveillance and Epidemiology Center for Drug Evaluation and Research U.S. Food and Drug Administration LUCA FOSCHINI Co-Founder and Chief Data Scientist Evidation Health BRANDE YAIST Senior Director, Global Patient Outcomes and Real-World Evidence Eli Lilly and Company 12:00 p.m. BREAK (Lunch Available Outside the Lecture Room) SESSION III OBSCURING INTERVENTION ALLOCATION IN TRIALS TO GENERATE REAL- WORLD EVIDENCE: WHY, WHO, AND WHEN? Session Objectives: Discuss how variability in knowledge of treatment assignment group affects: o Provider and patient adherence and outcomes. o Study cost and reliability. Suggest key factors that could affect decisions to obscure intervention allocation. Discuss how a decision aid laying out key questions and considerations might help inform current and future studies. 1:00 p.m. Introduction: A Proposed Framework for a Decision Aid JONATHAN WATANABE, Session Moderator Associate Professor of Clinical Pharmacy National Academy of Medicine Anniversary Fellow in Pharmacy P REP UBLI CATI ON COP Y: UNCORR ECTED P ROOFS

164 EXAMINING THE IMPACT OF RWE ON MEDICAL PRODUCT DEVELOPMENT University of California, San Diego 1:10 p.m. Looking Back: How Might a Decision Aid Inform a Real-World Example? JOHN GRAHAM Head, Value Evidence and Outcomes GlaxoSmithKline ORLY VARDENY Minneapolis Department of Veterans Affairs Center for Chronic Disease Outcomes Research Associate Professor of Medicine University of Minnesota 1:30 p.m. Looking Forward: How Decision Aid Might Apply to Future Studies Panel Discussion and Audience Q&A CATHY CRITCHLOW Vice President, Center for Observational Research Amgen Inc. NANCY DREYER Chief Scientific Officer IQVIA ALEX JOHN LONDON Clara L. West Professor of Ethics and Philosophy Carnegie Mellon University JAMES P. SMITH Deputy Director, Division of Metabolism and Endocrinology Products Center for Drug Evaluation and Research U.S. Food and Drug Administration 2:50 p.m. BREAK SESSION IV HOW TIGHTLY SHOULD INVESTIGATORS ATTEMPT TO CONTROL OR RESTRICT TREATMENT QUALITY IN A PRAGMATIC OR REAL-WORLD TRIAL? Session Objectives: Discuss how variability in treatment delivery and adherence can affect results, including o Potential influence of variation in standard treatment practice, and o Considerations for balancing participant autonomy and safety. Suggest key factors that could help determine the base comparison and level of control suited to a particular trial. P REP UBLI CATI ON COP Y: UNCORR ECTED P ROOFS

APPENDIX D 165 Discuss how a decision aid laying out key questions and considerations might help inform current and future studies. 3:10 p.m. Introduction: A Proposed Framework for a Decision Aid JENNIFER GRAFF, Session Moderator Vice President of Comparative Effectiveness Research National Pharmaceutical Council 3:20 p.m. Looking Back: How Might a Decision Aid Inform a Real-World Example? LARRY ALPHS Deputy Chief Medical Officer Newron Pharmaceuticals 3:40 p.m. Looking Forward: How Decision Aid Might Apply to Future Studies Panel Discussion and Audience Q&A JUDITH CARRITHERS Director of Regulatory Services Advarra W. BENJAMIN NOWELL Director, Patient-Centered Research Global Healthy Living Foundation Co–Principal Investigator, ArthritisPower Patient Powered Research Network PETER STEIN Deputy Director, Office of New Drugs Center for Drug Evaluation and Research U.S. Food and Drug Administration 4:50 p.m. Day 1 Wrap-Up and Concluding Thoughts/Discussion with Audience 5:00 p.m. ADJOURN WORKSHOP DAY 1 DAY 2: July 18, 2018 8:00 a.m. Welcome MARK MCCLELLAN, Workshop Series Co-Chair Director Duke-Margolis Center for Health Policy GREGORY SIMON, Workshop Series Co-Chair Investigator Kaiser Permanente Washington Health Research Institute P REP UBLI CATI ON COP Y: UNCORR ECTED P ROOFS

166 EXAMINING THE IMPACT OF RWE ON MEDICAL PRODUCT DEVELOPMENT SESSION V HOW CAN BIAS IN OBSERVATIONAL COMPARISONS BE ASSESSED AND MINIMIZED? Session Objectives: Discuss methods to assess presence of and optimally reduce bias from unmeasured confounding. Suggest key considerations for assessing—and communicating—uncertainty in observational studies. Discuss how a decision aid laying out key questions and considerations might help inform current and future studies. 8:10 a.m. Introduction: A Proposed Framework for a Decision Aid DAVID MARTIN Associate Director for Real-World Evidence Analytics U.S. Food and Drug Administration 8:20 a.m. Looking Back: How Might a Decision Aid Inform a Real-World Example? HECTOR IZURIETA Epidemiologist, Office of Biostatistics and Epidemiology Center for Biologics Evaluation and Research U.S. Food and Drug Administration 8:40 a.m. Looking Forward: How Decision Aid Might Apply to Future Studies Panel Discussion and Audience Q&A GREGORY DANIEL, Session Moderator Deputy Director Duke-Margolis Center for Health Policy JESSICA FRANKLIN Assistant Professor of Medicine Harvard Medical School NICOLE GORMLEY Team Lead, Division of Hematologic Products Office of Hematology and Oncology Products Center for Drug Evaluation and Research U.S. Food and Drug Administration JAVIER JIMENEZ Vice President and Global Head for Real-World Evidence and Clinical Outcomes Sanofi HENG LI Mathematical Statistician Center for Devices and Radiological Health U.S. Food and Drug Administration P REP UBLI CATI ON COP Y: UNCORR ECTED P ROOFS

APPENDIX D 167 MARK VAN DER LAAN Professor, Biostatistics and Statistics University of California, Berkeley 10:00 a.m. BREAK SESSION VI FDA PANEL Session Objectives: Hear updates and perspective of current thinking about real-world evidence in Europe. Discuss challenges, opportunities, and remaining gaps for moving forward with real- world evidence application. 10:15 a.m. A European Perspective ALASDAIR BRECKENRIDGE, Session Moderator Emeritus Professor of Clinical Pharmacology University of Liverpool 10:30 a.m. Reflections from FDA STEVEN ANDERSON Director, Office of Biostatistics and Epidemiology, Center for Biologics Evaluation and Research U.S. Food and Drug Administration JACQUELINE CORRIGAN-CURAY Director, Office of Medical Policy, Center for Drug Evaluation and Research U.S. Food and Drug Administration JEFF SHUREN Director, Center for Devices and Radiological Health U.S. Food and Drug Administration 11:15 a.m. Panel Discussion with Audience 11:50 a.m. Synthesis of Workshop Discussions MARK MCCLELLAN, Workshop Series Co-Chair Director Duke-Margolis Center for Health Policy GREGORY SIMON, Workshop Series Co-Chair Investigator Kaiser Permanente Washington Health Research Institute 12:00 p.m. ADJOURN WORKSHOP DAY 2 P REP UBLI CATI ON COP Y: UNCORR ECTED P ROOFS

Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series Get This Book
×
Buy Paperback | $60.00
MyNAP members save 10% online.
Login or Register to save!
Download Free PDF

Randomized controlled trials (RCTs) have traditionally served as the gold standard for generating evidence about medical interventions. However, RCTs have inherent limitations and may not reflect the use of medical products in the real world. Additionally, RCTs are expensive, time consuming, and cannot answer all questions about a product or intervention. Evidence generated from real-world use, such as real-world evidence (RWE) may provide valuable information, alongside RCTs, to inform medical product decision making.

To explore the potential for using RWE in medical product decision making, the National Academies of Sciences, Engineering, and Medicine planned a three-part workshop series. The series was designed to examine the current system of evidence generation and its limitations, to identify when and why RWE may be an appropriate type of evidence on which to base decisions, to learn from successful initiatives that have incorporated RWE, and to describe barriers that prevent RWE from being used to its full potential. This publication summarizes the discussions from the entire workshop series.

  1. ×

    Welcome to OpenBook!

    You're looking at OpenBook, NAP.edu's online reading room since 1999. Based on feedback from you, our users, we've made some improvements that make it easier than ever to read thousands of publications on our website.

    Do you want to take a quick tour of the OpenBook's features?

    No Thanks Take a Tour »
  2. ×

    Show this book's table of contents, where you can jump to any chapter by name.

    « Back Next »
  3. ×

    ...or use these buttons to go back to the previous chapter or skip to the next one.

    « Back Next »
  4. ×

    Jump up to the previous page or down to the next one. Also, you can type in a page number and press Enter to go directly to that page in the book.

    « Back Next »
  5. ×

    To search the entire text of this book, type in your search term here and press Enter.

    « Back Next »
  6. ×

    Share a link to this book page on your preferred social network or via email.

    « Back Next »
  7. ×

    View our suggested citation for this chapter.

    « Back Next »
  8. ×

    Ready to take your reading offline? Click here to buy this book in print or download it as a free PDF, if available.

    « Back Next »
Stay Connected!