Assessment of Clinical Trials Supporting US Food and Drug Administration Approval of Novel Therapeutic Agents, 1995-2017

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Abstract
In the United States, the US Food and Drug Administration (FDA) issues approvals for new drugs and biologics that have demonstrated safety and efficacy in “adequate and well-controlled studies.”1 Pivotal trials are the most critical of these trials, often identified directly by FDA reviewers as the basis for approval and described in detail in FDA approval packages.1 Early guidance suggested that at least 2 such trials were required for approval,2 but the FDA has maintained a flexible interpretation, taking into consideration the ethical acceptability of conducting additional trials or the rarity of diseases when determining the sufficient threshold for safety and efficacy.3 As a result, the quantity and quality of evidence supporting recent drug approvals is variable, both in terms of the number of pivotal trials and their design features, such as randomization, blinding, choice of comparators and end points, number of treated patients, and trial duration.4-6
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