How do we know whether a drug works? In the United States, the US Food and Drug Administration (FDA) has established guidance to inform drug development, including testing to establish safety and efficacy for a specific clinical indication, prior to market approval. The FDA generally requires that companies complete numerous clinical studies, including 1 or 2 pivotal trials, of which more than 80% are randomized; use double-blinded allocation; and include a placebo or active control comparator arm.1 Because these trials are often small, short, and use surrogate markers of disease for primary trial endpoints,1 some uncertainty remains as to drugs’ safety and efficacy at the time of approval. Nevertheless, once approved, clinicians may prescribe the drug for any clinical indication, not only the one that received FDA approval. This is known as off-label prescribing.
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Ross JS. Coordinating Clinical Trials to Confirm Drug Safety and Efficacy—Resolving Uncertainty. JAMA Intern Med. 2019;179(1):98. doi:10.1001/jamainternmed.2018.6037
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