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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
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BIOMARKER TESTS
FOR MOLECULARLY
TARGETED THERAPIES

Key to Unlocking Precision Medicine

Committee on Policy Issues in the Clinical Development and Use of
Biomarkers for Molecularly Targeted Therapies

Laurene A. Graig, Jonathan K. Phillips, and Harold L. Moses, Editors

Board on Health Care Services

Institute of Medicine

images

THE NATIONAL ACADEMIES PRESS
Washington, DC
www.nap.edu

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
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THE NATIONAL ACADEMIES PRESS 500 Fifth Street, NW Washington, DC 20001

This study was supported by Contracts 200-2011-38807,TO#27 (Centers for Disease Control and Prevention), 185616 (Janssen Diagnostics), HHSN2632012000741 (National Cancer Institute), NGC19399 (Novartis), and OPP-3028.0 (Susan G. Komen). The study was also supported by the American Society for Radiation Oncology, American Society of Clinical Oncology, Breast Cancer Research Foundation, College of American Pathologists, Gilead Sciences, Inc., Pfizer Inc., and Quest Diagnostics. 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: 978-0-309-38134-5
International Standard Book Number-10: 0-309-38134-7
Digital Object Identifier: 10.17226/21860

Additional copies of this report are available for sale from the National Academies Press, 500 Fifth Street, NW, Keck 360, Washington, DC 20001; (800) 624-6242 or (202) 334-3313; http://www.nap.edu.

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

Printed in the United States of America

Cover credit: Original art reproduced by permission from Michael Guy.

Suggested citation: National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker tests for molecularly targeted therapies: Key to unlocking precision medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
×

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.

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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. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
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COMMITTEE ON POLICY ISSUES IN THE CLINICAL DEVELOPMENT AND USE OF BIOMARKERS FOR MOLECULARLY TARGETED THERAPIES

HAROLD L. MOSES (Chair), Ingram Professor of Cancer Research, Professor and Chair, Department of Cancer Biology, Professor of Medicine and Professor of Pathology, Microbiology, and Immunology, Director-Emeritus, Vanderbilt-Ingram Comprehensive Cancer Center, Vanderbilt University Medical Center

JOHN M. CARETHERS, John G. Searle Professor and Chair, Department of Internal Medicine, Professor of Human Genetics, University of Michigan Health System

MOLLY COOKE, Professor of Medicine and Director of Education, Global Health Sciences, University of California, San Francisco

GARRET A. FITZGERALD, Professor of Medicine and Pharmacology, McNeil Professor in Translational Medicine and Therapeutics, Chair, Department of Pharmacology, Director, Institute for Translational Medicine & Therapeutics, Perelman School of Medicine, University of Pennsylvania

FELIX W. FRUEH, Executive Partner, Opus Three LLC

DEBRA LEONARD, Professor and Chair of Pathology & Laboratory Medicine, University of Vermont Medical Center, University of Vermont College of Medicine

GARY H. LYMAN, Co-Director, Hutchinson Institute for Cancer Outcomes Research, Public Health Sciences and Clinical Research Divisions, Fred Hutchinson Cancer Research Center, Professor of Medicine, Public Health, and Pharmacy, University of Washington

ROBERT L. NUSSBAUM, Chief Medical Officer, Invitae; Professor Emeritus and Volunteer Clinical Professor, University of California San Francisco School of Medicine

REBECCA D. PENTZ, Professor of Research Ethics, Winship Cancer Institute, Emory University School of Medicine

EDITH A. PEREZ,1 Vice President and Head of the BioOncology Medical Unit, US Medical Affairs, Genentech; Deputy Director at Large, Mayo Clinic Cancer Center, Director, Breast Cancer Translational Genomics Program, Serene M. and Frances C. Durling Professor of Medicine, Division of Hematology/Oncology, Mayo Clinic

JANE PERLMUTTER, Independent Consultant, Gemini Group

___________________

1 Through August 2015.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
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VICTORIA M. PRATT, Director, Pharmacogenomics Laboratory, Department of Medical and Molecular Genetics, Indiana University School of Medicine

YU SHYR, Harold L. Moses Chair in Cancer Research, Director, Vanderbilt Center for Quantitative Sciences, Director, Vanderbilt Technologies for Advanced Genomics, Analysis and Research Design, Professor, Department of Biostatistics, Biomedical Informatics, Cancer Biology, and Health Policy, Vanderbilt University

SEAN TUNIS, Founder, President and CEO, Center for Medical Technology Policy

TRACEY F. WEISBERG, President, New England Cancer Specialists

Study Staff

LAURENE GRAIG, Study Director

JONATHAN PHILLIPS, Research Associate

SARAH E. DE LEO, Christine Mirzayan Science and Technology Policy Graduate Fellow (from January to July 2015)

NOA NIR, Senior Program Assistant (from April 2015)

CELYNNE BALATBAT, Senior Program Assistant (until April 2015)

PATRICK BURKE, Financial Associate

SHARYL NASS, Director, National Cancer Policy Forum; Director, Board on Health Care Services

ADAM C. BERGER, Director, Roundtable on Translating Genomic-Based Research for Health (until August 2015)

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

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
×

Reviewers

This report 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 report as sound as possible and to ensure that the report 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 deliberative process. We wish to thank the following individuals for their review of this report:

JAMES BURNS, Sanofi

PAMELA B. DAVIS, Case Western Reserve University School of Medicine

NITA A. FARAHANY, Duke University

JOE GRAY, Oregon Health and Science University

ROBERT GREEN, Flatiron Health, Inc.

JAY L. HESS, Indiana University

LOUIS JACQUES, ADVI Consulting

LARRY KESSLER, University of Washington

FEDERICO A. MONZON, Castle Biosciences

TUDOR OPREA, University of New Mexico

JOHN PFEIFER, Washington University School of Medicine in St. Louis

ELDA RAILEY, Research Advocacy Network

SCOTT RAMSEY, Fred Hutchinson Cancer Research Center

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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
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CHARLES SAWYERS, Memorial Sloan Kettering Cancer Center

HAROLD VARMUS, Weill Cornell Medical College

Although the reviewers listed above have provided many constructive comments and suggestions, they were not asked to endorse the conclusions or recommendations nor did they see the final draft of the report before its release. The review of this report was overseen by LESLIE Z. BENET, University of California, San Francisco, and HUDA AKIL, University of Michigan. They were responsible for making certain that an independent examination of this report was carried out in accordance with institutional procedures and that all review comments were carefully considered. Responsibility for the final content of this report rests entirely with the authoring committee and the institution.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
×

Acknowledgments

The committee is grateful to many individuals who provided valuable input and information for the study, either through formal presentations or through informal communication with study staff and committee members:

Amy Abernethy, M.D., Ph.D., Flatiron Health

Samuel Aronson, M.A., Partners Healthcare

Robert Califf, M.D., Food and Drug Administration

Brian Carey, J.D., Foley Hoag, LLP

Joseph Chin, M.D., M.S., Centers for Medicare & Medicaid Services

Jeff Chodakewitz, M.D., Vertex Pharmaceuticals

Stephen Friend, M.D., Ph.D., Sage Bionetworks

Sarah Garcia, Ph.D., Personalis, Inc.

Levi Garraway, M.D., Ph.D., Harvard Medical School

Curtis Hanson, M.D., Mayo Clinic

Daniel F. Hayes, M.D., University of Michigan Comprehensive Cancer Center

Jonathan W. Heusel, M.D., Ph.D., Washington University School of Medicine

Clifford Hudis, M.D., Memorial Sloan Kettering Cancer Center

Louis Jacques, M.D., ADVI Consulting

Elaine Jeter, M.D., Palmetto GBA

Simon Mallal, M.D., Vanderbilt University Medical Center

Elizabeth Mansfield, Ph.D., Food and Drug Administration

Robert McDonough, M.D., Aetna

Laurence Meyer, M.D., Ph.D., University of Utah

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
×

Lincoln Nadauld, M.D., Intermountain Healthcare

Lee Newcomer, M.D., M.H.A., UnitedHealthcare

Marisa Papaluca, Ph.D., European Medicines Agency

Sharon Plon, M.D., Ph.D., Baylor College of Medicine

Paul Radensky, M.D., McDermott, Will & Emory

Scott Ramsey, M.D., Ph.D., Fred Hutchinson Cancer Research Center

Alan Rosenberg, M.D., Anthem

William Stead, M.D., Vanderbilt University School of Medicine

Shawn Sweeney, Ph.D., American Association for Cancer Research

Suzanne Topalian, M.D., Johns Hopkins Medicine

John Wagner, M.D., Ph.D., Takeda Pharmaceuticals

Sheila D. Walcoff, J.D., Goldbug Strategies, LLC

Catherine A. Wicklund, M.S., CGC, Northwestern University Feinberg School of Medicine

Robert Wildin, M.D., National Institutes of Health

Marc Williams, M.D., Geisinger Health System

Janet Woodcock, M.D., Food and Drug Administration

In addition, we thank the individuals who spoke at the November 2014 National Cancer Policy Forum workshop Policy Issues in the Development and Adoption of Biomarkers for Molecularly Targeted Cancer Therapies. Workshop presentations and discussions informed committee deliberations. Speakers included:

Garnet Anderson, Ph.D., Fred Hutchinson Cancer Research Center

Dane Dickson, M.D., Palmetto GBA, Teton Cancer Institute

David Eberhard, M.D., Ph.D., University of North Carolina at Chapel Hill

Bill Gradishar, M.D., Northwestern University

Neil Hayes, M.D., M.S., M.P.H., University of North Carolina at Chapel Hill

Roy Herbst, M.D., Ph.D., Yale University

Matthias Holdhoff, M.D., Ph.D., Johns Hopkins University

Bruce Johnson, M.D., Dana-Farber Cancer Institute

Mia Levy, M.D., Ph.D., Vanderbilt University Medical Center

David Litwack, Ph.D., Food and Drug Administration

Patricia Lo Russo, D.O., Yale University

Karen Long, B.S., Abbott Molecular

Jennifer Malin, M.D., Ph.D., Wellpoint

Anne-Marie Martin, Ph.D., GlaxoSmithKline

Donna Messner, Ph.D., Center for Medical Technology Policy

Federico Monzon, M.D., Invitae

Kathryn Phillips, Ph.D., University of California, San Francisco

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
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Richard Schilsky, M.D., American Society of Clinical Oncology

Adrian Senderowicz, M.D., Ignyta, Inc.

Lilian Siu, M.D., Princess Margaret Hospital, Ontario Cancer Institute

David Solit, M.D., Memorial Sloan Kettering Cancer Center

Sean Tunis, M.D., M.Sc., Center for Medical Technology Policy

Mickey Williams, Ph.D., National Cancer Institute

Funding for this study was provided by the American Society for Radiation Oncology, American Society of Clinical Oncology, Breast Cancer Research Foundation, Centers for Disease Control and Prevention, College of American Pathologists, Gilead Sciences, Janssen Diagnostics, National Cancer Institute, Novartis, Pfizer Inc., Quest Diagnostics, and Susan G. Komen. The committee appreciates the support of these sponsors for the development of this study and report.

Many individuals within the National Academies of Sciences, Engineering, and Medicine were helpful to the study staff. We would like to thank Erin Balogh, Clyde Behney, Jill Eden, Chelsea Frakes, Greta Gorman, Ellen Kimmel, Tracy Lustig, Fariha Mahmud, Sharyl Nass, Bettina Ritter, Patti Simon, Mark Stewart, and Jennifer Walsh.

Finally, we would also like to thank Laura Penny for her copyediting and Ian Graig for his assistance with report graphics.

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
×
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
×

Acronyms

AABB

American Association of Blood Banks

AACR

American Association for Cancer Research

AAFP

American Academy of Family Physicians

ABL1

Abelson murine leukemia viral oncogene homolog 1

ABMS

American Board of Medical Specialties

ACC

American College of Cardiology

ACCE

analytic validity, clinical validity, clinical utility, and ethical/legal/social implications

ACGME

Accreditation Council for Graduate Medical Education

ACLA

American Clinical Laboratory Association

ACMG

American College of Medical Genetics and Genomics

ACP

American College of Physicians

ADLT

advanced diagnostic laboratory test

AHA

American Hospital Association; American Heart Association

AHLA

American Health Lawyers Association

AHRQ

Agency for Healthcare Research & Quality

ALK

anaplastic lymphoma kinase

AMA

American Medical Association

AMIA

American Medical Informatics Association

AMP

Association for Molecular Pathology

ASCO

American Society of Clinical Oncology

ASCP

American Society for Clinical Pathology

ASTRO

American Society for Radiation Oncology

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
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ATA

American Telemedicine Association

AV

analytic validity

BCBSA

Blue Cross and Blue Shield Association

BCR

breakpoint cluster region

BRAF

B-RAF proto-oncogene, serine/threonine kinase

BRCA

breast cancer susceptibility gene

CancerLinQ

Cancer Learning Intelligence Network for Quality

CAP

College of American Pathologists

CCHPCA

Center for Connected Health Policy

CDC

Centers for Disease Control and Prevention

CDD

coverage with data development

CDE

common data element

CDER

Center for Drug Evaluation and Research

CDRH

Center for Devices and Radiological Health

CDSS

clinical decision support system

CE

continuing education

CED

coverage with evidence development

CER

comparative effectiveness research

CEU

Continuing Education Unit

CF

cystic fibrosis

CFTR

cystic fibrosis transmembrane conductance regulator

CI

confidence interval

CLEP

Clinical Laboratory Evaluation Program

CLFS

Clinical Laboratory Fee Schedule

CLIA

Clinical Laboratory Improvement Amendments of 1988

CLIAC

Clinical Laboratory Improvement Advisory Committee

CME

Continuing Medical Education

CMS

Centers for Medicare & Medicaid Services

CMTP

Center for Medical Technology Policy

COI

conflict of interest

COLA

Commission on Office Laboratory Accreditation

COSMIC

Catalogue of Somatic Mutations in Cancer

CPG

clinical practice guideline

CPT®

Current Procedural Terminology

CU

clinical utility

CV

clinical validity

DNA

deoxyribonucleic acid

DoD

Department of Defense

DTWG

Diagnostic Test Working Group

Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
×
EGAPP

Evaluation of Genomic Applications in Practice and Prevention

EGFR

epidermal growth factor receptor

EHR

electronic health record

eMERGE

Electronic Medical Records and Genomics

ER

estrogen receptor

FACT

Foundation for the Accreditation of Cellular Therapy

FAERS

FDA’s Adverse Event Reporting System

FDA

Food and Drug Administration

FDCA

Federal Food, Drug, and Cosmetic Act

FISH

fluorescence in situ hybridization

FTC

Federal Trade Commission

GA4GH

Global Alliance for Genomics and Health

GAO

Government Accountability Office

GDG

guideline developing group

GENIE

Genomics Evidence Neoplasia Information Exchange

GINA

Genetic Information Nondiscrimination Act of 2008

GPC

Green Park Collaborative

GTR

Genetic Testing Registry

GWAS

genome-wide association study

HCPCS

Healthcare Common Procedure Coding System

HELP

Senate Committee on Health, Education, Labor & Pensions

HER2

human epidermal growth factor receptor 2

HGVS

Human Genome Variation Society

HHS

Department of Health and Human Services

HIV/AIDS

human immunodeficiency virus/acquired immune deficiency syndrome

HLA

human leukocyte antigen

HMO

health maintenance organization

HNSCC

head and neck squamous cell carcinoma

HPV

human papillomavirus

HRSA

Health Resources and Services Administration

ICD

International Classification of Diseases

IHC

immunohistochemistry

IOM

Institute of Medicine

ISPOR

International Society for Pharmacoeconomics and Outcomes Research

IT

information technology

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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
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IVCT

in vitro clinical test

IVD

in vitro diagnostic

KRAS

kirsten rat sarcoma viral oncogene homolog

LCD

local coverage determination

LDP

laboratory-developed procedure

LDT

laboratory-developed test

LDTS

laboratory-developed testing service

LIS

laboratory information system

MAC

Medicare Administrative Contractor

MED-C

Molecular Evidence Development Consortium

MedPAC

Medicare Payment Advisory Commissions

MMWR

Morbidity and Mortality Weekly Report

MOC

Maintenance of Certification

MoPath

Molecular Pathology

MVP

Million Veterans Program

NASEM

The National Academies of Sciences, Engineering, and Medicine

NCCN

National Comprehensive Cancer Network

NCD

national coverage determination

NCI

National Cancer Institute

NCPF

National Cancer Policy Forum

NGS

next-generation sequencing

NGTS

next-generation tumor sequencing

NHGRI

National Human Genome Research Institute

NIH

National Institutes of Health

NLM

National Library of Medicine

NLP

natural language processing

NQF

National Quality Forum

NRC

National Research Council

NSCLC

non-small-cell lung carcinoma

NYSDOH

New York State Department of Health

OIG

Office of the Inspector General

ONC

Office of the National Coordinator for Health Information Technology

OSTP

Office of Science and Technology Policy

PAMA

Protecting Access to Medicare Act of 2014

PBRSA

performance-based risk-sharing arrangement

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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
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PCAST

President’s Council of Advisors on Science and Technology

PCORI

Patient-Centered Outcomes Research Institute

PCORNet

National Patient-Centered Clinical Research Network

PCTF

Payer Communication Task Force

PD-1

programmed cell death protein 1

PET

positron emission tomography

PFS

physician fee schedule

PGR

progesterone receptor

PGRN

Pharmacogenomics Research Network

PheWAS

phenome-wide association study

PIK3CA

phosphatidylinositol-4,5-bisphosphate 3-kinase, catalytic subunit alpha

PMA

premarket approval

PMC

Personalized Medicine Coalition

PMI

Precision Medicine Initiative

PROMIS

Patient Reported Outcomes Measurement Information System

PT

proficiency testing

PTEN

phosphatase and tensin homolog

RCT

randomized controlled trial

RNA

ribonucleic acid

RUSP

Recommended Uniform Screening Panel

SACGHS

Secretary’s Advisory Committee on Genetics, Health, and Society

SACHDNC

Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children

SNOMED CT

Systematized Nomenclature of Medicine’s Clinical Terms

SNP

single-nucleotide polymorphism

TAPUR

Targeted Agents and Profiling Utilization Registry

TCGA

The Cancer Genome Atlas

VA

Department of Veterans Affairs

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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
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Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
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Page xxii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
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Page xxiii Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
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Page xxiv Cite
Suggested Citation:"Front Matter." National Academies of Sciences, Engineering, and Medicine. 2016. Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine. Washington, DC: The National Academies Press. doi: 10.17226/21860.
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Biomarker Tests for Molecularly Targeted Therapies: Key to Unlocking Precision Medicine Get This Book
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Every patient is unique, and the evolving field of precision medicine aims to ensure the delivery of the right treatment to the right patient at the right time. In an era of rapid advances in biomedicine and enhanced understanding of the genetic basis of disease, health care providers increasingly have access to advanced technologies that may identify molecular variations specific to an individual patient, which subsequently can be targeted for treatment. Known as biomarker tests for molecularly targeted therapies, these complex tests have the potential to enable the selection of the most beneficial treatment (and also to identify treatments that may be harmful or ineffective) for the molecular underpinnings of an individual patient’s disease. Such tests are key to unlocking the promise of precision medicine.

Biomarker tests for molecularly targeted therapies represent a crucial area of focus for developing methods that could later be applicable to other areas of precision medicine. The appropriate regulatory oversight of these tests is required to ensure that they are accurate, reliable, properly validated, and appropriately implemented in clinical practice. Moreover, common evidentiary standards for assessing the beneficial impact of biomarker-guided therapy selection on patient outcomes, as well as the effective collection and sharing of information related to those outcomes, are urgently needed to better inform clinical decision making.

Biomarker Tests of Molecularly Targeted Therapies examines opportunities for and challenges to the use of biomarker tests to select optimal therapy and offers recommendations to accelerate progress in this field. This report explores regulatory issues, reimbursement issues, and clinical practice issues related to the clinical development and use of biomarker tests for targeting therapies to patients. Properly validated, appropriately implemented biomarker tests hold the potential to enhance patient care and improve outcomes, and therefore addressing the challenges facing such tests is critical.

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