Before approving a drug, the Food and Drug Administration (FDA) assesses a drug's effectiveness. This assessment may be based on evidence showing that a drug has a positive impact on a surrogate endpoint--a laboratory measure, such as blood pressure--instead of more direct clinical evidence, like preventing strokes. After approval, FDA often requires or requests a drug sponsor to further study the drug. Concerns have been raised about FDA's reliance on surrogate endpoints and its oversight of postmarketing studies. This report provides information on (1) all drug applications approved based on surrogate endpoints in FDA's accelerated approval process, (2) a subset of applications for potentially innovative drugs approved based on surrogate endpoints under FDA's traditional process, and (3) FDA's oversight of postmarketing studies. GAO identified drugs approved based on surrogate endpoints, obtained the status of related postmarketing studies, and reviewed FDA's oversight of a sample of 35 studies it required under its accelerated approval process, selected to include studies which were at varying levels of completion.
Recommendations for Executive Action
|Food and Drug Administration||1. To clarify FDA's enforcement authority under the accelerated approval process, the Commissioner of FDA should clarify the conditions under which the agency would utilize its authority to expedite the withdrawal of drugs approved based on surrogate endpoints under the accelerated approval process if sponsors either fail to complete required confirmatory studies with due diligence, or if studies are completed, but fail to demonstrate the clinical effectiveness of the drugs.|