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Accelerated Approval of Cancer Drugs—Righting the Ship of the US Food and Drug Administration

Accelerated Approval of Cancer Drugs—Righting the Ship of the US Food and Drug Administration Research Original Investigation Cancer Drugs Approved Based on the Surrogate End Point of Response Rate Invited Commentary HEALTH CARE POLICY AND LAW Accelerated Approval of Cancer Drugs— Righting the Ship of the US Food and Drug Administration Sarah S. P. DiMagno, BA; Aaron Glickman, BA; Ezekiel J. Emanuel, MD, PhD The US Food and Drug Administration (FDA) is caught be- Response rate is not itself a meaningful clinical outcome; tween Scylla and Charybdis, with competing pressures to speed the size of a tumor does not matter if patients’ lives are not ex- drugs to market and to ensure they are safe and effective. On tended or if their quality of life is not improved. Response rate is useful only if it reliably predicts these outcomes. Some- one side, patients, families, and advocates are clamoring for faster approval of new drugs what counterintuitively, even high response rates and shrink- that offer treatments for seri- ing tumors are not necessarily highly correlated with improve- Invited Commentary page 913 ous and life-threatening dis- ments in survival or symptoms. A systematic review of multiple eases. They believe that de- meta-analyses found that surrogate end points—ranging from Related articles pages 906 lay is potentially http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png JAMA Internal Medicine American Medical Association

Accelerated Approval of Cancer Drugs—Righting the Ship of the US Food and Drug Administration

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Publisher
American Medical Association
Copyright
Copyright 2019 American Medical Association. All Rights Reserved.
ISSN
2168-6106
eISSN
2168-6114
DOI
10.1001/jamainternmed.2019.0584
Publisher site
See Article on Publisher Site

Abstract

Research Original Investigation Cancer Drugs Approved Based on the Surrogate End Point of Response Rate Invited Commentary HEALTH CARE POLICY AND LAW Accelerated Approval of Cancer Drugs— Righting the Ship of the US Food and Drug Administration Sarah S. P. DiMagno, BA; Aaron Glickman, BA; Ezekiel J. Emanuel, MD, PhD The US Food and Drug Administration (FDA) is caught be- Response rate is not itself a meaningful clinical outcome; tween Scylla and Charybdis, with competing pressures to speed the size of a tumor does not matter if patients’ lives are not ex- drugs to market and to ensure they are safe and effective. On tended or if their quality of life is not improved. Response rate is useful only if it reliably predicts these outcomes. Some- one side, patients, families, and advocates are clamoring for faster approval of new drugs what counterintuitively, even high response rates and shrink- that offer treatments for seri- ing tumors are not necessarily highly correlated with improve- Invited Commentary page 913 ous and life-threatening dis- ments in survival or symptoms. A systematic review of multiple eases. They believe that de- meta-analyses found that surrogate end points—ranging from Related articles pages 906 lay is potentially

Journal

JAMA Internal MedicineAmerican Medical Association

Published: Jul 28, 2019

References