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Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24685.
×

Appendix B

Workshop Agenda

REAL-WORLD EVIDENCE GENERATION AND EVALUATION OF THERAPEUTICS: A WORKSHOP

October 19, 2016
National Academy of Sciences Building, Room 120
2101 Constitution Avenue, NW, Washington, DC 20418

BACKGROUND AND WORKSHOP OBJECTIVES

The traditional process for evaluating new therapeutics does not produce the evidence that patients, clinicians, and payers need for real-world decisions. The volume and complexity of information about individual patients is greatly increasing with use of electronic records and personal devices. Possibilities for medical product development in the context of this wealth of real-world data are great, ranging from the ability to determine both large-scale and patient-specific effects of treatments to assessing how therapeutics affect patients’ lives through measurement of lifestyle changes. However, mechanisms to facilitate efficient use of real-world data to meet the decision-making needs of myriad stakeholders have not been established. An ad hoc committee will plan and conduct a 1-day public workshop that will examine opportunities and challenges for incorporating real-world evidence into evaluation of medical products.

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24685.
×

Subject-matter experts will be invited to participate in the workshop through presentations and discussions that will consider

  • Quality of data from real-world sources, including
    • Relevance and validity of different sources of real-world data (e.g., user collected, practice based) in the context of different clinical/scientific questions; and
    • Strengths and limitations of different data sources at different stages of treatment development and the licensing process.
  • Methodologies and best practices for high-quality, real-world evidence generation and application, including
    • Innovations in clinical trial design to maximize value of information for the full range of stakeholders;
    • Considerations of how evidence generation from existing studies could potentially inform the design of future clinical trials and amplify understanding of product efficacy;
    • Discussion of how shared goals of payers and regulators can better align evidence generation processes used for regulatory evaluation and decisions on use by payers; and
    • Re-evaluation of traditional distinctions between goals and methods of preapproval and postapproval research.
  • Other novel methodologies and approaches to improve development and evaluation of products using real-world evidence, including
    • Use of Web-based or digital technologies to enhance clinical trial evidence collection and participation; and
    • Techniques and case studies for effectively using electronic health record data.
8:30 a.m. SESSION I: BREAKING THE MOLD:
STAKEHOLDER PRIORITIES FOR IMPROVING EVIDENCE GENERATION

Session Objectives:

  • Examine different stakeholder evidence needs to support decision making and identify shared goals.
  • Discuss priorities for facilitating use of real-world evidence to address stakeholder needs.
  • Discuss aligning incentives to maximize generation and sharing of useful evidence.
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24685.
×
8:30 a.m. Opening Remarks and Introductions
STEVEN GALSON, Workshop Co-Chair

Senior Vice President for Global Regulatory Affairs and Safety

Amgen Inc.

GREG SIMON, Workshop Co-Chair

Investigator, Group Health Research Institute

Chair, Scientific Advisory Board, Depression and Bipolar Support Alliance

8:45 a.m. Keynote
ROBERT CALIFF
Commissioner

U.S. Food and Drug Administration

9:05 a.m. Stakeholder Perspectives
JOHN CARROLL
Professor of Medicine

Co-Medical Director, Cardiac and Vascular Center

University of Colorado Hospital

JOSEPH CHIN

Deputy Director, Coverage and Analysis Group

Centers for Medicare & Medicaid Services

NAFTALI ZVI FRANKEL
Patient and Consumer Advocate
RHONDA ROBINSON BEALE
Chief Medical Officer
Blue Cross of Idaho
RACHEL SHERMAN

Deputy Commissioner for Medical Products and Tobacco

U.S. Food and Drug Administration

PATRICK VALLANCE

President, Pharmaceuticals Research and Development

GlaxoSmithKline

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24685.
×
9:30 a.m. Moderated Discussion with Session I Speakers and Audience
ROBERT CALIFF, Moderator
Commissioner
U.S. Food and Drug Administration
10:45 a.m. Break
11:00 a.m. SESSION II: WHAT CAN WE LEARN FROM REAL-WORLD DATA?

Session Objectives:

  • Examine different sources of real-world data (e.g., user collected, practice based) and consider their reliability in the context of different clinical/scientific questions.
  • Discuss strengths and limitations of different data sources at different stages of treatment development and licensing process.
11:00 a.m. Background and Session Objectives
JOHN HERNANDEZ, Moderator
Head of Health Economics, Value and Access
Verily Life Sciences
NIGAM SHAH, Moderator

Associate Professor of Medicine, Biomedical Information Research

Stanford University

11:05 a.m. Sources for and Practical Use of Real-World Data
Data Sharing and Linking Records Across Electronic Health Record Vendors
JON WHITE

Deputy National Coordinator for Health Information Technology

Office of the National Coordinator for Health Information Technology

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24685.
×
PCORnet and Clinical Data Research Networks
RUSSELL ROTHMAN

Director, Center for Health Services Research

Chief, Internal Medicine and Pediatrics

Vanderbilt University

Potential for Data Analytics
DAVID DORE

Vice President, Epidemiology and Principal Epidemiologist

Optum Life Sciences

Using Data from Activities on Mobile Devices
LUCA FOSCHINI

Co-Founder and Chief Data Scientist

Evidation Health

11:45 a.m. Moderated Discussion with Session II Speakers and Audience
12:30 p.m. Lunch
1:15 p.m. SESSION III: THE PROMISE OF REAL-WORLD
EVIDENCE: STRATEGIES FOR BUILDING FROM SUCCESSFUL USE CASES

Session Objectives:

  • Discuss examples of successful approaches to generating and incorporating real-world evidence into development and evaluation of medical products.
  • Identify opportunities and challenges to scaling up successful practices and adapting them to new purposes.
1:15 p.m. Background and Session Objectives
JESSE BERLIN, Moderator

Vice President and Global Head of Epidemiology

Johnson & Johnson

CATHY CRITCHLOW, Moderator

Vice President and Head, Center for Observational Research

Amgen Inc.

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24685.
×
1:20 p.m. Successful Use Cases of Real-World Evidence
Case Study #1: Salford Lung Study
ANDREW RODDAM

Vice President and Head of Real-World Evidence

GlaxoSmithKline

Case Study #2: Transcatheter Valve Therapy (TVT) Registry
MICHAEL MACK

Chair, Cardiovascular Service Line

Baylor Scott & White Health

Case Study #3: Sentinel Initiative
LESLEY CURTIS
Professor of Medicine

Director for Pragmatic Health Services Research

Duke Clinical Research Institute

Case Study #4: Observational Health Data Sciences and Informatics (OHDSI)
NIGAM SHAH

Associate Professor of Medicine, Biomedical Information Research

Stanford University

2:20 p.m. Moderated Discussion with Stakeholder Reaction Panel
MARC BERGER

Vice President, Real-World Data and Analytics

Pfizer Inc.

LAURA DEMBER

Professor of Medicine

Renal, Electrolyte, and Hypertension Division

University of Pennsylvania

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24685.
×
LOUIS FIORE

Executive Director

Massachusetts Veterans Epidemiology Research and Information Center

RHONDA ROBINSON BEALE
Chief Medical Officer
Blue Cross of Idaho
SEAN TUNIS

Founder, President, and Chief Executive Officer

Center for Medical Technology Policy

3:30 p.m. Break
3:45 p.m. SESSION IV: REAL-WORLD EVIDENCE OF THE FUTURE: POTENTIAL STRATEGIES FOR A WAY FORWARD

Session Objectives:

  • Outline an ideal future state for incorporating real-world evidence into evaluation of medical products.
  • Identify short- and long-term next steps at any stage of clinical research to achieve seamless use of real-world evidence.
  • Discuss incentives that should be explored.
3:45 p.m. Reflecting on Tactics and Strategies for a Way Forward: Discussion with Workshop Co-Chairs, Session Moderators, Panelists, and Audience
MARK MCCLELLAN, Moderator
Director
Margolis Center for Health Policy
Duke University
STEVEN GALSON, Workshop Co-Chair

Senior Vice President for Global Regulatory Affairs and Safety

Amgen Inc.

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24685.
×
GREG SIMON, Workshop Co-Chair

Investigator, Group Health Research Institute

Chair, Scientific Advisory Board, Depression and Bipolar Support Alliance

JESSE BERLIN

Vice President and Global Head of Epidemiology

Johnson & Johnson

NAFTALI ZVI FRANKEL

Patient and Consumer Advocate

JOHN HERNANDEZ

Head of Health Economics, Value and Access

Verily Life Sciences

FREDA LEWIS-HALL

Chief Medical Officer and Executive Vice President

Pfizer Inc.

NIGAM SHAH

Associate Professor of Medicine, Biomedical Information Research

Stanford University

RACHEL SHERMAN

Deputy Commissioner for Medical Products and Tobacco

U.S. Food and Drug Administration

4:20 p.m. Moderated Discussion with Session IV Panel and Audience
5:00 p.m. Adjourn
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24685.
×
Page 53
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24685.
×
Page 54
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24685.
×
Page 55
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24685.
×
Page 56
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24685.
×
Page 57
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24685.
×
Page 58
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24685.
×
Page 59
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24685.
×
Page 60
Next: Appendix C: Participant Biographies »
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The volume and complexity of information about individual patients is greatly increasing with use of electronic records and personal devices. Potential effects on medical product development in the context of this wealth of real-world data could be numerous and varied, ranging from the ability to determine both large-scale and patient-specific effects of treatments to the ability to assess how therapeutics affect patients’ lives through measurement of lifestyle changes.

In October 2016, the National Academies of Sciences, Engineering, and Medicine held a workshop to facilitate dialogue among stakeholders about the opportunities and challenges for incorporating real-world evidence into all stages in the process for the generation and evaluation of therapeutics. Participants explored unmet stakeholder needs and opportunities to generate new kinds of evidence that meet those needs. This publication summarizes the presentations and discussions from the workshop.

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