RE: A compelling case?22 Mar 2022 08:41
In the US, the Food and Drug Administration determines whether a new drug is sufficiently safe and effective to be made available to doctors for use by patients.1 To do this, it must find a balance between requiring sufficient high quality clinical evidence from premarket evaluation and allowing promising new drugs to enter the marketplace quickly with continued evaluation after approval. The FDA maintains a “usual requirement” of “more than one” well controlled clinical trial that independently proves a drug’s efficacy.2 However, it also describes several situations in which fewer trials or studies with non-clinical outcomes, such as surrogate markers of disease, might suffice for premarket evaluation.2 Thus, FDA approval is binary, but the clinical trial evidence that forms the basis of the FDA’s decision varies widely.
From 2005 to 2012 the FDA approved 188 new drugs for 206 indications, more than a third of which were approved on the basis of a single pivotal trial and 44% of which were approved on the basis of pivotal trials that used surrogate markers of disease instead of clinical outcomes for primary endpoints (41 approvals met both criteria).3
Regulation has historically occurred primarily in the period before a drug is approved and becomes widely available. Over the past decade, however, the FDA has adopted “lifecycle evaluation” (box 1).45 This approach was originally proposed by the Institute of Medicine in its 2007 report, The Future of Drug Safety, which was commissioned by the FDA after a series of well publicized postmarket drug safety concerns raised questions about drug safety in the US.46 In addition to monitoring drug safety, the lifecycle evaluation approach enables regulators to approve drugs based on clinical evidence that is less robust, with the understanding that drugs will continue to be evaluated after approval.
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