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Coordinating Clinical Trials to Confirm Drug Safety and Efficacy—Resolving Uncertainty

Coordinating Clinical Trials to Confirm Drug Safety and Efficacy—Resolving Uncertainty Clinical Review & Education Review Assessment of Pregabalin Postapproval Trials and the Suggestion for Efficacy in New Indications Editor's Note Coordinating Clinical Trials to Confirm Drug Safety and Efficacy—Resolving Uncertainty Joseph S. Ross, MD, MHS Howdoweknow whether a drug works? In the United States, trials,74%wereconsideredexploratoryand26%werepotentially the US Food and Drug Administration (FDA) has established confirmatory.Although5indications(accountingfor40%ofiden- guidance to inform drug development, including testing to es- tified confirmatory trials) ultimately secured FDA or European tablish safety and efficacy for a specific clinical indication, prior MedicinesAgencyapproval,clinicaltrialsfor22(67%)indications generated clinical agnosticism, a term the authors coined to de- to market approval. The FDA generally requires that com- scribeexploratory,underpoweredtrialsthatsuggestedanewclini- Related article page 90 panies complete numerous cal benefit. Furthermore, 63% of tested indications were not fol- clinical studies, including 1 or 2 pivotal trials, of which more lowed within 5 years by a confirmatory trial. than 80% are randomized; use double-blinded allocation; and High-quality, rigorous testing of drugs for a wide variety include a placebo or active control comparator arm. Because of clinical indications maximizes our understanding of these trials are often small, short, and use surrogate markers drugs’ use and benefit to patients. However, a medical lit- of disease for primary trial endpoints, some http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png JAMA Internal Medicine American Medical Association

Coordinating Clinical Trials to Confirm Drug Safety and Efficacy—Resolving Uncertainty

JAMA Internal Medicine , Volume 179 (1) – Jan 26, 2019

Coordinating Clinical Trials to Confirm Drug Safety and Efficacy—Resolving Uncertainty

Abstract

Clinical Review & Education Review Assessment of Pregabalin Postapproval Trials and the Suggestion for Efficacy in New Indications Editor's Note Coordinating Clinical Trials to Confirm Drug Safety and Efficacy—Resolving Uncertainty Joseph S. Ross, MD, MHS Howdoweknow whether a drug works? In the United States, trials,74%wereconsideredexploratoryand26%werepotentially the US Food and Drug Administration (FDA) has established...
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Publisher
American Medical Association
Copyright
Copyright 2018 American Medical Association. All Rights Reserved.
ISSN
2168-6106
eISSN
2168-6114
DOI
10.1001/jamainternmed.2018.6037
Publisher site
See Article on Publisher Site

Abstract

Clinical Review & Education Review Assessment of Pregabalin Postapproval Trials and the Suggestion for Efficacy in New Indications Editor's Note Coordinating Clinical Trials to Confirm Drug Safety and Efficacy—Resolving Uncertainty Joseph S. Ross, MD, MHS Howdoweknow whether a drug works? In the United States, trials,74%wereconsideredexploratoryand26%werepotentially the US Food and Drug Administration (FDA) has established confirmatory.Although5indications(accountingfor40%ofiden- guidance to inform drug development, including testing to es- tified confirmatory trials) ultimately secured FDA or European tablish safety and efficacy for a specific clinical indication, prior MedicinesAgencyapproval,clinicaltrialsfor22(67%)indications generated clinical agnosticism, a term the authors coined to de- to market approval. The FDA generally requires that com- scribeexploratory,underpoweredtrialsthatsuggestedanewclini- Related article page 90 panies complete numerous cal benefit. Furthermore, 63% of tested indications were not fol- clinical studies, including 1 or 2 pivotal trials, of which more lowed within 5 years by a confirmatory trial. than 80% are randomized; use double-blinded allocation; and High-quality, rigorous testing of drugs for a wide variety include a placebo or active control comparator arm. Because of clinical indications maximizes our understanding of these trials are often small, short, and use surrogate markers drugs’ use and benefit to patients. However, a medical lit- of disease for primary trial endpoints, some

Journal

JAMA Internal MedicineAmerican Medical Association

Published: Jan 26, 2019

References