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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
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ADVANCING THE
DISCIPLINE OF
REGULATORY SCIENCE

FOR MEDICAL PRODUCT
DEVELOPMENT

An Update on Progress and
a Forward-Looking Agenda

Workshop Summary

Morgan L. Boname, Amanda Wagner Gee, and Anne B. Claiborne,
Rapporteurs

Forum on Drug Discovery, Development, and Translation

Board on Health Sciences Policy

Health and Medicine Division

Images

THE NATIONAL ACADEMIES PRESS

Washington, DC

www.nap.edu

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

THE NATIONAL ACADEMIES PRESS 500 Fifth Street, NW Washington, DC 20001

This activity was supported by AbbVie, Inc.; the American Diabetes Association; American Society of Microbiology; Amgen Inc. (Contract No. GHCCOPS-CSARF-63987); Association of American Medical Colleges; AstraZeneca; Baxter BioScience; Burroughs Wellcome Fund (Contract No. 1015149); Critical Path Institute; Doris Duke Charitable Foundation (Contract No. 2015103); Eli Lilly & Co.; FasterCures; Friends of Cancer Research; GlaxoSmithKline (Contract No. 005319); Johnson & Johnson; Merck & Co., Inc. (Contract No. CMO-141224-000649); National Institutes of Health (NIH) (Contract No. HHSN263201200074I; Task Order HHSN26300023): National Cancer Institute (NIH), National Center for Advancing Translational Sciences (NIH), National Institute of Allergy and Infectious Diseases (NIH), National Institute of Mental Health (NIH), and National Institute of Neurological Disorders and Stroke (NIH); New England Journal of Medicine; Pfizer Inc.; Sanofi; Takeda Pharmaceuticals (Contract No. 53108); and U.S. Food and Drug Administration (Contract No. 1R13FD005496-01). Any opinions, findings, conclusions, or recommendations expressed in this publication do not necessarily reflect the views of any organization or agency that provided support for the project.

International Standard Book Number-13: 978-0-309-43884-1
International Standard Book Number-10: 0-309-43884-5
Digital Object Identifier: 10.17226/23438

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Copyright 2016 by the National Academy of Sciences. All rights reserved.

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Suggested citation: National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the discipline of regulatory science for medical product development: An update on progress and a forward-looking agenda: Workshop summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

Image

The National Academy of Sciences was established in 1863 by an Act of Congress, signed by President Lincoln, as a private, nongovernmental institution to advise the nation on issues related to science and technology. Members are elected by their peers for outstanding contributions to research. Dr. Ralph J. Cicerone is president.

The National Academy of Engineering was established in 1964 under the charter of the National Academy of Sciences to bring the practices of engineering to advising the nation. Members are elected by their peers for extraordinary contributions to engineering. Dr. C. D. Mote, Jr., is president.

The National Academy of Medicine (formerly the Institute of Medicine) was established in 1970 under the charter of the National Academy of Sciences to advise the nation on medical and health issues. Members are elected by their peers for distinguished contributions to medicine and health. Dr. Victor J. Dzau is president.

The three Academies work together as the National Academies of Sciences, Engineering, and Medicine to provide independent, objective analysis and advice to the nation and conduct other activities to solve complex problems and inform public policy decisions. The Academies also encourage education and research, recognize outstanding contributions to knowledge, and increase public understanding in matters of science, engineering, and medicine.

Learn more about the National Academies of Sciences, Engineering, and Medicine at www.national-academies.org.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

PLANNING COMMITTEE ON ADVANCING THE DISCIPLINE OF REGULATORY SCIENCE FOR MEDICAL PRODUCT DEVELOPMENT: AN UPDATE ON PROGRESS AND A FORWARD-LOOKING AGENDA1

MARTIN PHILBERT (Co-Chair), University of Michigan School of Public Health, Ann Arbor, MI

ALASTAIR WOOD (Co-Chair), Symphony Capital, New York, NY

RUSS ALTMAN, Stanford University, CA

SHARON HESTERLEE, Myotonic Dystrophy Foundation, San Francisco, CA

RUSTY KELLEY, Burroughs Wellcome Fund, Research Triangle Park, NC

EMMA MEAGHER, University of Pennsylvania, Philadelphia, PA

ROBERT MEYER, University of Virginia School of Medicine, Charlottesville, VA

STEPHEN OSTROFF, U.S. Food and Drug Administration, Silver Spring, MD

PAUL SELIGMAN, Amgen Inc., Thousand Oaks, CA

BRIAN STROM, Rutgers, the State University of New Jersey, Newark, NJ

HMD Staff

ANNE B. CLAIBORNE, Forum Director

CHRISTOPHER J. DEFEO, Program Officer

EMILY R. BUSTA, Associate Program Officer

KATHRYN M. HOWELL, Senior Program Assistant

ANDREW M. POPE, Director, Board on Health Sciences Policy

___________________

1 The National Academies of Sciences, Engineering, and Medicine’s planning committees are solely responsible for organizing the workshop, identifying topics, and choosing speakers. The responsibility for the published workshop summary rests with the workshop rapporteurs and the institution.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

This page intentionally left blank.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

FORUM ON DRUG DISCOVERY, DEVELOPMENT, AND TRANSLATION1

RUSS ALTMAN (Co-Chair), Stanford University, CA

STEVEN GALSON (Co-Chair), Amgen Inc., Thousand Oaks, CA

MARGARET ANDERSON, FasterCures, Washington, DC

HUGH AUCHINCLOSS, National Institute of Allergy and Infectious Diseases, Bethesda, MD

CHRISTOPHER AUSTIN, National Center for Advancing Translational Sciences, Bethesda, MD

ANN BONHAM, Association of American Medical Colleges, Washington, DC

LINDA BRADY, National Institute of Mental Health, Bethesda, MD

GAIL CASSELL, Harvard Medical School Department of Social and Global Medicine (Visiting), Carmel, IN

ANDREW DAHLEM, Eli Lilly & Co., Indianapolis, IN

JAMES DOROSHOW, National Cancer Institute, Bethesda, MD

JEFFREY DRAZEN, New England Journal of Medicine, Boston, MA

JODIE GILLON, AstraZeneca, Summit, NJ

HARRY GREENBERG, Stanford University School of Medicine, CA (until December 31, 2015)

LYNN HUDSON, Critical Path Institute, Tucson, AZ

S. CLAIBORNE JOHNSTON, Dell Medical School, University of Texas, Austin

JACK KEENE, Duke University Medical Center, Durham, NC (until December 31, 2015)

RUSTY KELLEY, Burroughs Wellcome Fund, Research Triangle Park, NC

KATE KNOBIL, GlaxoSmithKline, Collegeville, PA

RONALD KRALL, University of Pittsburgh, Steamboat Springs, CO (until December 31, 2015)

FREDA LEWIS-HALL, Pfizer Inc., New York, NY

BERNARD MUNOS, InnoThink Center for Research in Biomedical Innovation, Indianapolis, IN

ELIZABETH (BETSY) MYERS, Doris Duke Charitable Foundation, New York, NY (until December 31, 2015)

JOHN ORLOFF, Baxalta, Deerfield, IL

ROBERT RATNER, American Diabetes Association, Alexandria, VA

MICHAEL ROSENBLATT, Merck & Co., Inc., Kenilworth, NJ

MICHAEL SEVERINO, AbbVie, Inc., North Chicago, IL

___________________

1 The National Academies of Sciences, Engineering, and Medicine’s forums and roundtables do not issue, review, or approve individual documents. The responsibility for the published workshop summary rests with the workshop rapporteurs and the institution.

Page viii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

ELLEN SIGAL, Friends of Cancer Research, Washington, DC

LANA SKIRBOLL, Sanofi, Washington, DC

BRIAN STROM, Rutgers, the State University of New Jersey, Newark

JANET TOBIAS, Ikana Media, New York, NY (until December 31, 2015)

JOHN WAGNER, Takeda Pharmaceuticals, Cambridge, MA

JOANNE WALDSTREICHER, Johnson & Johnson, New Brunswick, NJ

CARRIE WOLINETZ, National Institutes of Health, Bethesda, MD

JANET WOODCOCK, U.S. Food and Drug Administration, Silver Spring, MD

HMD Staff

ANNE B. CLAIBORNE, Forum Director

CHRISTOPHER J. DEFEO, Program Officer (until October 2015)

REBECCA A. ENGLISH, Program Officer

AMANDA WAGNER GEE, Program Officer (as of November 2015)

EMILY R. BUSTA, Associate Program Officer (until December 2015)

MORGAN L. BONAME, Associate Program Officer (as of December 2015)

OLIVIA C. YOST, Senior Program Assistant (as of December 2015)

KATHRYN M. HOWELL, Senior Program Assistant (until November 2015)

ANDREW M. POPE, Director, Board on Health Sciences Policy

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

Reviewers

This workshop summary has been reviewed in draft form by individuals chosen for their diverse perspectives and technical expertise. The purpose of this independent review is to provide candid and critical comments that will assist the institution in making its published workshop summary as sound as possible and to ensure that the workshop summary meets institutional standards for objectivity, evidence, and responsiveness to the study charge. The review comments and draft manuscript remain confidential to protect the integrity of the process. We wish to thank the following individuals for their review of this workshop summary:

Brian Alexander, Harvard Medical School

Jesse Goodman, Georgetown University

Peter Honig, Pfizer Inc.

Although the reviewers listed above have provided many constructive comments and suggestions, they did not see the final draft of the workshop summary before its release. The review of this workshop summary was overseen by Barry S. Coller, The Rockefeller University. He was responsible for making certain that an independent examination of this workshop summary was carried out in accordance with institutional procedures and that all review comments were carefully considered. Responsibility for the final content of this workshop summary rests entirely with the rapporteurs and the institution.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
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This page intentionally left blank.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

This page intentionally left blank.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×

Acronyms and Abbreviations

AD Alzheimer’s disease
ADPKD autosomal dominant polycystic kidney disease
API application program interface

BLAERS

Behavioral Log-Based Adverse Event Reporting System

BQRT Biomarker Qualification Review Team

CDER

Center for Drug Evaluation and Research

CDISC Clinical Data Interchange Standards Consortium
CDRH Center for Devices and Radiological Health
CERSI Center of Excellence in Regulatory Science and Innovation
COU context of use
C-Path Critical Path Institute
CPIM Critical Path Innovation Meeting
cTAP collaborative Trajectory Analysis Program
CTSA Clinical and Translational Science Award

DMD

Duchenne muscular dystrophy

EHR

electronic health record

EMA European Medicines Agency

FAERS

FDA Adverse Event Reporting System

FDA U.S. Food and Drug Administration
FDAAA FDA Amendments Act of 2007
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
×
FHIR Fast Healthcare Interoperability Resources
fMRI functional magnetic resonance imaging
FNIH Foundation for the National Institutes of Health

HL7

Health Level Seven International

IND

Investigational New Drug

IOM Institute of Medicine
IRSA Innovations in Regulatory Science Awards

MDEpiNet

Medical Device Epidemiology Network

NDA

New Drug Application

NIH National Institutes of Health
NIST National Institute of Standards and Technology

PhRMA

Pharmaceutical Research and Manufacturers of America

PK/PD pharmacokinetic/pharmacodynamic
PMDA Pharmaceuticals and Medical Devices Agency
PSTC Predictive Safety Testing Consortium

RCT

randomized controlled trial

UCSF

University of California, San Francisco

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
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Page viii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
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Page xiii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/23438.
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Advancing the Discipline of Regulatory Science for Medical Product Development: An Update on Progress and a Forward-Looking Agenda: Workshop Summary Get This Book
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The field of endeavors known as "regulatory science" has grown out of the need to link and integrate knowledge within and among basic science research, clinical research, clinical medicine, and other specific scientific disciplines whose focus, aggregation, and ultimate implementation could inform biomedical product development and regulatory decision making. Substantial efforts have been devoted to defining regulatory science and communicating its value and role across the scientific and regulatory ecosystems. Investments are also being made in technology infrastructure, regulatory systems, and workforce development to support and advance this burgeoning discipline.

In October 2015, the National Academies of Sciences, Engineering, and Medicine held a public workshop to facilitate dialogue among stakeholders about the current state and scope of regulatory science, opportunities to address barriers to the discipline's success, and avenues for fostering collaboration across sectors. Participants explored key needs for strengthening the discipline of regulatory science, including considering what are the core components of regulatory science infrastructure to foster innovation in medical product development. This report summarizes the presentations and discussions from the workshop.

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