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Suggested Citation:"Appendix B: Statement of Task and Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
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Appendix B

Statement of Task and Workshop Agenda

Enabling Precision Medicine:
The Role of Genetics in Clinical Drug Development
A Workshop

March 8, 2017
Keck Center of the National Academies
500 Fifth Street, NW Room 100
Washington, DC 20001

Statement of Task

The current drug development process faces challenges of efficiency and overall sustainability due in part to high research costs, lengthy development timelines, and late-stage drug failures. Novel clinical trial designs that target genetically identified participants represent a potentially disruptive paradigm shift that could reduce overall health care costs associated with drug development, improve patient outcomes, and further realize the goals of precision medicine. An ad hoc committee will plan and conduct a 1-day public workshop that will examine and discuss successes, challenges, and best practices for effectively using genetic information in the design and implementation of clinical trials to support the development of precision medicines, including exploring the potential advantages and disadvantages of such trials across a variety of disease areas. Topics covered could include strategies for including genetically focused populations in clinical trial

Suggested Citation:"Appendix B: Statement of Task and Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×

design, logistical challenges in conducting genetics-based clinical trials, and mechanisms for engaging with and educating potential trial participants. Discussions will be held among a broad array of stakeholders, which may include patients, academic researchers, health care providers, and representatives from the biopharmaceutical sector.

Objectives

  • To explore how clinical trials with genetically identified participants can enable more efficient and effective drug development and advance precision medicine.
  • To highlight ongoing genetics-based clinical trials across a variety of diseases, examining best practices and lessons learned.
  • To learn about the logistical challenges and successes associated with genetics-based clinical trial design.
  • To examine possible mechanisms to engage participants and improve enrollment into clinical trials based on genetic characteristics.

The planning committee will develop the workshop agenda, will select and invite speakers and discussants, and may moderate the discussions. The proceedings of the workshop will be prepared by a designated rapporteur in accordance with institutional policy and procedures.

8:30 a.m. Opening Remarks

STEVEN GALSON, Co-Chair of the Forum on Drug Discovery, Development, and Translation

Senior Vice President, Global Regulatory Affairs and Safety Amgen Inc.

GEOFFREY GINSBURG, Co-Chair of the Roundtable on Genomics and Precision Health

Director, Duke Center for Applied Genomics and Precision Medicine; Professor of Medicine, Pathology, and Biomedical Engineering

Duke University Medical Center

8:35 a.m. Charge to the Speakers and Participants

LAURA NISENBAUM, Workshop Co-Chair

Advisor, Chorus Clinical Development

Eli Lilly and Company

Suggested Citation:"Appendix B: Statement of Task and Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×

SESSION I: OVERARCHING CONSIDERATIONS FOR IMPLEMENTING SUCCESSFUL GENETICS-BASED DRUG DEVELOPMENT

Session Objectives:

  • Discuss the perspective of patients with regard to the use of genetic data in clinical drug development.
  • Consider challenges and possible solutions associated with the development and use of biomarkers or genetic tests for molecularly targeted therapies.
  • Explore issues that pertain to the regulatory pathway for genetic tests and biomarkers used in clinical drug development.
Moderator: LAURA NISENBAUM, Advisor, Chorus Clinical Development, Eli Lilly and Company
8:45 a.m. Accelerating the Pace of Progress in Genetics-Based Drug Development: The Perspective of a Patient and Patient Advocate
JANE PERLMUTTER
President and Founder
Gemini Group
9:00 a.m. Clarifying Questions
9:10 a.m. Clinical Development and Use of Biomarkers for Molecularly Targeted Therapies: Recommendations from a National Academies Consensus Study
ROBERT NUSSBAUM
Chief Medical Officer
Invitae
9:30 a.m. Clarifying Questions
9:40 a.m. Navigating the Regulatory Pathway for Genetic Tests and Biomarkers for Clinical Drug Development
MICHAEL PACANOWSKI
Associate Director for Genomics and Targeted Therapy
Center for Drug Evaluation and Research
U.S. Food and Drug Administration
Suggested Citation:"Appendix B: Statement of Task and Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×
10:00 a.m. Clarifying Questions
10:10 a.m. Break

SESSION II: CASE STUDIES IN PRECISION DRUG DEVELOPMENT

Session Objectives:

  • Explore recent examples of precision drug development in order to identify lessons that can be applied across disease fields.
  • Examine clinical development plans for precision medicines, including
    • using our understanding of the underlying biology to inform trial design and product development;
    • effects on research costs and timeline; and
    • patient recruitment and stratification.
Moderator: RICHARD SCHILSKY, Chief Medical Officer, American Society of Clinical Oncology
10:25 a.m. Lung Master Protocol (Lung-MAP): A Biomarker-Driven Protocol for Accelerating Therapeutic Development for Squamous Cell Lung Cancer
ROY HERBST

Professor of Medical Oncology; Associate Director for Translational Research

Yale Cancer Center

10:40 a.m. STARTRK-2 Clinical Trial: A Basket Study of Entrectinib for the Treatment of Solid Tumors with Specific Gene Rearrangements
ZACHARY HORNBY
Chief Operating Officer
Ignyta
10:55 a.m. Genetic Testing and Clinical Drug Development for Spinal Muscular Atrophy
JOHN STAROPOLI
Associate Medical Director
Biogen
Suggested Citation:"Appendix B: Statement of Task and Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×
11:10 a.m. Using Genetics in Clinical Trials for Mitochondrial Diseases
MARNI FALK

Associate Professor of Pediatrics, University of Pennsylvania

Director, Mitochondrial Disease Clinical Center, Children’s Hospital of Philadelphia

11:25 a.m. Panel Discussion with Audience and Speakers
12:10 p.m. Working Lunch

SESSION III: INTEGRATING GENETICS INTO THE DRUG DEVELOPMENT PATHWAY FOR COMPLEX DISEASES

Session Objectives:

  • Illuminate novel ways to apply genetics/genomics discoveries into clinical development plans for complex diseases as a way to increase efficiency and improve outcomes.
  • Survey innovative ways to engage participants in precision clinical trials, including communication, outreach, and education-based initiatives, with additional attention paid to increasing the diversity of such trials.
  • Discuss additional logistical challenges associated with the execution of a successful genetics-based clinical trial for a complex disease, including
    • techniques for patient stratification, including considerations for employing genetic risk scores; and
    • timelines for developing molecularly targeted therapies, including trial execution.
Moderator RUSS ALTMAN, Professor, Stanford University
1:00 p.m. Patient Perspective on Genetics-Enabled Drug Development
THERESA V. STRONG
Director of Research Programs
Foundation for Prader-Willi Research
1:15 p.m. Clarifying Questions
Suggested Citation:"Appendix B: Statement of Task and Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×
1:20 p.m. Potential Advantages and Pitfalls to Using Genetics in Drug Development for Complex Diseases
MATT NELSON
Head of Genetics
GlaxoSmithKline
1:35 p.m. Clarifying Questions
1:40 p.m. Challenges in Leveraging GWAS Findings During Drug Development: A Case Study
REBECCA BLANCHARD

Head of Clinical Pharmacogenomics and Operations

Merck & Co., Inc.
1:55 p.m. Clarifying Questions
2:00 p.m. Updates and Strategies from the TAILOR-PCI Trial
MICHAEL FARKOUH
Professor and Vice Chair of Research, Department of Medicine University of Toronto
2:15 p.m. Clarifying Questions
2:20 p.m. Panel Discussion with Audience and Speakers
G. SCOTT CHANDLER

Vice President and Global Head of Licensing and Early Development Safety

Genentech/Roche
JESSICA LANGBAUM
Principal Scientist
Banner Alzheimer’s Institute
MARK TRUSHEIM
Visiting Scientist

Massachusetts Institute of Technology Sloan School of Management

Suggested Citation:"Appendix B: Statement of Task and Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×
L. KEOKI WILLIAMS

Associate Director, Center for Health Policy and Health Services Research

Henry Ford Health System
3:20 p.m. Break

SESSION IV: FINDING INNOVATIVE WAYS TO INTEGRATE GENETICS RESEARCH INTO THE DRUG DEVELOPMENT PROCESS

Session Objective:

  • Explore innovative, “outside-the-box” ideas for genetics-based clinical trials that could make the therapeutic development pathway more efficient.
Moderator: BRAY PATRICK-LAKE, Director of Stakeholder Engagement, Duke University, Clinical and Translational Science Institute
3:35 p.m. Return on Investment from Patient Input on Development
DAVID LEVENTHAL
Director, Clinical Innovation
Pfizer Worldwide Research and Development
3:55 p.m. SaME Therapeutics: Grouping Rare Disease Patients by Shared Molecular Etiology to Accelerate Clinical Trials
P. J. BROOKS
Program Director, Division of Clinical Innovation
National Center for Advancing Translational Sciences
4:15 p.m. New Targets, New Modalities, New Challenges—The Inconvenient Path of Human Genetics in Drug Discovery
ROBERT PLENGE
Vice President and Head of Translational Medicine
Merck & Co., Inc.
4:35 p.m. Panel Discussion with Audience and Speakers
Suggested Citation:"Appendix B: Statement of Task and Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×

SESSION V: LOOKING FORWARD—RECAP AND CONCLUSIONS

Moderator: ESTEBAN BURCHARD, Professor, University of California, San Francisco
5:05 p.m. Summary of Key Points—Session I
LAURA NISENBAUM, Workshop Co-Chair
Advisor, Chorus Clinical Development
Eli Lilly and Company
5:10 p.m. Summary of Key Points—Session II
JOHN CARULLI
Director, Precision Medicine
Biogen
5:15 p.m. Summary of Key Points—Session III

RUSS ALTMAN, Co-Chair of the Forum on Drug Discovery, Development, and Translation

Professor of Bioengineering, Genetics, Medicine, and (by courtesy) Computer Science

Stanford University
5:20 p.m. Summary of Key Points—Session IV

BRAY PATRICK-LAKE

Director, Stakeholder Engagement

Duke University, Clinical and Translational Science Institute

5:25 p.m. Final Remarks

ESTEBAN BURCHARD, Workshop Co-Chair

Professor and Vice Chair, Department of Bioengineering and Therapeutic Sciences and Medicine

University of California, San Francisco

5:30 p.m. ADJOURN
Suggested Citation:"Appendix B: Statement of Task and Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×
Page 99
Suggested Citation:"Appendix B: Statement of Task and Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×
Page 100
Suggested Citation:"Appendix B: Statement of Task and Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×
Page 101
Suggested Citation:"Appendix B: Statement of Task and Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×
Page 102
Suggested Citation:"Appendix B: Statement of Task and Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×
Page 103
Suggested Citation:"Appendix B: Statement of Task and Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×
Page 104
Suggested Citation:"Appendix B: Statement of Task and Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×
Page 105
Suggested Citation:"Appendix B: Statement of Task and Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×
Page 106
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Those involved in the drug development process face challenges of efficiency and overall sustainability due in part to high research costs, lengthy development timelines, and late-stage drug failures. Novel clinical trial designs that enroll participants based on their genetics represent a potentially disruptive change that could improve patient outcomes, reduce costs associated with drug development, and further realize the goals of precision medicine.

On March 8, 2017, the Forum on Drug Discovery, Development, and Translation and the Roundtable on Genomics and Precision Health of the National Academies of Sciences, Engineering, and Medicine hosted the workshop Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development. Participants examined successes, challenges, and possible best practices for effectively using genetic information in the design and implementation of clinical trials to support the development of precision medicines, including exploring the potential advantages and disadvantages of such trials across a variety of disease areas. This publication summarizes the presentations and discussions from the workshop.

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