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Suggested Citation:"Appendix B: Workshop One 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.
×

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)
Suggested Citation:"Appendix B: Workshop One 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.
×
  • 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

Suggested Citation:"Appendix B: Workshop One 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.
×
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
Suggested Citation:"Appendix B: Workshop One 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.
×
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

Suggested Citation:"Appendix B: Workshop One 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.
×
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

Suggested Citation:"Appendix B: Workshop One 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.
×
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
Suggested Citation:"Appendix B: Workshop One 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.
×

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
Suggested Citation:"Appendix B: Workshop One 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.
×
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
Suggested Citation:"Appendix B: Workshop One 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 183
Suggested Citation:"Appendix B: Workshop One 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 184
Suggested Citation:"Appendix B: Workshop One 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 185
Suggested Citation:"Appendix B: Workshop One 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 186
Suggested Citation:"Appendix B: Workshop One 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 187
Suggested Citation:"Appendix B: Workshop One 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 188
Suggested Citation:"Appendix B: Workshop One 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 189
Suggested Citation:"Appendix B: Workshop One 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 190
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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.

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