Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series (2019)
Chapter: Appendix B: Workshop One Agenda
Appendix B
Workshop One Agenda
Workshop One: Incentives
September 19–20, 2017
National Academy of Sciences Building, Lecture Room
2101 Constitution Avenue, NW, Washington, DC 20418
The National Academies of Sciences, Engineering, and Medicine is convening a three-part workshop series 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.
This first workshop will include discussions and background materials that address:
- Aligning incentives and addressing barriers to support collection and use of real-world evidence in health product review, payment, and delivery.
Workshops two and three will foster discussions that will:
- Illuminate what types of data are appropriate for what specific purposes and suggest approaches for data collection that match the right data to the right questions. (Q1 2018)
- Examine and suggest approaches for operationalizing the collection and use of real-world evidence. (Q3 2018)
DAY 1: SEPTEMBER 19, 2017
| 8:00 a.m. | Breakfast Available Outside the Lecture Room |
| 8:20 a.m. | Welcome and Opening Remarks |
| GREGORY SIMON, Workshop Series Co-Chair Investigator Kaiser Permanente Washington Health Research Institute |
Keynote Address
| 8:30 a.m. | Vision and Goals of a Collaborative, Practical, and Sustainable Real-World Evidence Program |
|
SCOTT GOTTLIEB
Commissioner U.S. Food and Drug Administration |
|
| 8:50 a.m. | Discussion with Audience |
| Moderator: | Mark McClellan, Duke-Margolis Center for Health Policy |
Session I: Seeing Our Destination
Session Objectives:
- Explore what relevant facts the ultimate end users of evidence need to know in order to make informed decisions about using medical products.
- Discuss possible approaches to generating such fit-for-purpose evidence.
Moderator: Andy Bindman, University of California, San Francisco
| 9:00 a.m. | A Payer Perspective |
|
MICHAEL SHERMAN
Senior Vice President and Chief Medical Officer Harvard Pilgrim Health Care |
|
| 9:20 a.m. | Delivery System Perspective: Integrated Care Model at Kaiser Permanente |
|
MICHAEL HORBERG
Executive Director, Research, Community Benefit, and Medicaid Strategy Executive Director, Mid-Atlantic Permanente Research Institute Kaiser Permanente Mid-Atlantic Permanente Medical Group |
|
| 9:40 a.m. | Delivery System Perspective: Academic Health System |
|
DANIEL FORD
Director, Institute for Clinical and Translational Research Johns Hopkins University School of Medicine |
|
| 10:00 a.m. | A Patient-Focused Perspective |
|
SHARON TERRY
President and Chief Executive Officer Genetic Alliance |
|
| 10:20 a.m. | Discussion with Audience |
|
Additional Invited Discussants:
JOANNE WALDSTREICHER Chief Medical Officer Johnson & Johnson |
|
|
ELEANOR PERFETTO
Senior Vice President, Strategic Initiatives National Health Council |
|
| 11:10 a.m. | BREAK |
| 11:30 a.m. | Key Messages and Themes from the September 13 FDA/Duke-Margolis Workshop: Generating Fit-for-Purpose Evidence |
|
MARK MCCLELLAN, Workshop Series Co-Chair
Director Duke-Margolis Center for Health Policy |
|
| 11:50 a.m. | Discussion with Audience |
| 12:00 p.m. | BREAK (Lunch Available Outside the Lecture Room) |
Session II: Learning from Success
Session Objectives:
- Highlight successful completed and ongoing initiatives that could potentially be examined for real-world evidence collection and use.
- Explore the features that led to the success in the given examples and how they could apply to future applications:
- Conditions likely to make innovation successful; and
- Potential ways to recreate those conditions to make real-world evidence use more routine.
| Moderator: | Gregory Simon, Kaiser Permanente Washington Health Research Institute |
| 1:00 p.m. | Generalizing and Scaling the Salford Lung Studies |
|
MARTIN GIBSON
Chief Executive Officer Northwest EHealth |
|
|
MARIE KANE
Chief Operating Officer Northwest EHealth |
|
| 1:30 p.m. | Using Sentinel to Evaluate Effectiveness or Efficacy |
|
RICHARD PLATT
Professor and Chair, Department of Population Medicine Harvard Medical School |
| 1:50 p.m. | Applying Lessons Learned from Device Registries to Other Treatment Types |
|
RACHAEL FLEURENCE
Executive Director National Evaluation System for Health Technology (NEST) Coordinating Center |
|
| 2:10 p.m. | Discussion with Audience |
|
Additional Invited Discussants:
JOHN GRAHAM Head, Value Evidence and Outcomes GlaxoSmithKline |
|
|
RACHEL SHERMAN
Principal Deputy Commissioner U.S. Food and Drug Administration |
|
| 3:00 p.m. | BREAK |
Session III: Getting Unstuck: Aligning Incentives
Session Objectives:
- In a series of presentations, discuss with treatment developers and evidence generators:
- Incentives maintaining the current data generation process; and
- Disincentives and potential barriers to incorporation of real-world evidence.
| Moderator: |
Petra Kaufmann, National Center for Advancing
Translational Sciences, National Institutes of Health |
| 3:20 p.m. | Contract Research Organization Perspective |
|
JOHN DOYLE
Senior Vice President and Managing Director QuintilesIMS |
| 3:40 p.m. | A Product Developer Perspective |
|
ELLIOTT LEVY
Senior Vice President, Global Development Amgen Inc. |
|
|
BRIAN D. BRADBURY
Executive Director, Center for Observational Research Amgen Inc. |
|
| 4:00 p.m. | An Academic Researcher Perspective |
|
DANIEL FORD
Director, Institute for Clinical and Translational Research Johns Hopkins University School of Medicine |
|
| 4:20 p.m. | Data Stewards: Organizations with Large Data Sources |
|
MARCUS WILSON
President HealthCore, Inc. |
|
| 4:40 p.m. | Discussion with Audience |
|
Additional Invited Discussants:
MICHAEL HORBERG Executive Director, Research, Community Benefit, and Medicaid Strategy Executive Director, Mid-Atlantic Permanente Research Institute Kaiser Permanente Mid-Atlantic Permanente Medical Group |
|
|
ANNA MCCOLLISTER-SLIPP
Chief Advocate for Participatory Research, Scripps Translational Science Institute Founder, VitalCrowd Co-Founder, Galileo Analytics |
|
| 5:30 p.m. | ADJOURN WORKSHOP DAY 1 |
DAY 2: SEPTEMBER 20, 2017
| 8:00 a.m. | Breakfast Available Outside the Lecture Room |
| 8:30 a.m. | Recap Day 1 and Discussion with Workshop Participants |
|
GREGORY SIMON, Workshop Series Co-Chair
Investigator Kaiser Permanente Washington Health Research Institute |
Keynote Address
| 9:00 a.m. | False Precision and Estimating the Reliability of Effects with the Traditional Evidence-Generating Process |
|
ROBERT CALIFF
Vice Chancellor, Health Data Science, Duke University Scientific Advisor, Verily Life Sciences |
Session IV: Getting Unstuck: Myth-Busting
Session Objective:
- Examine ideas—and misconceptions—about the necessity and acceptability of established evidence-generation practices.
| Moderator: | Robert Califf, Duke University and Verily Life Sciences |
| 9:30 a.m. | Moving from “One Study at a Time” to “All by All” Analyses |
|
PATRICK RYAN
Senior Director and Head, Epidemiology Analytics Janssen Research & Development |
|
| 9:50 a.m. | A Medical Product Developer Perspective |
|
JOHN GRAHAM
Head, Value Evidence and Outcomes GlaxoSmithKline |
|
| 10:10 a.m. | BREAK |
| 10:30 a.m. | Evolve or Die: The Urgent Need to Streamline Randomized Trials |
|
RORY COLLINS
Head of Nuffield Department of Population Health University of Oxford |
|
| 10:50 a.m. | A Regulatory Perspective |
|
JANET WOODCOCK
Director Center for Drug Evaluation and Research U.S. Food and Drug Administration |
|
| 11:10 a.m. | Discussion with Audience |
|
Additional Invited Discussant:
DEVEN MCGRAW Deputy Director, Health Information Privacy Office for Civil Rights U.S. Department of Health and Human Services |
|
| 12:30 p.m. | ADJOURN WORKSHOP DAY 2 |
