Excerpt
2023, Opinion: "Starting in 1992, the U.S. Food and Drug Administration (FDA) instituted the Accelerated Approval (AA) program allowing drugs that treat serious medical conditions and fill an unmet medical need to be approved based on a surrogate endpoint. The surrogate endpoint used for AA is a marker—a laboratory measurement or other measure that is thought to predict clinical benefit but is not itself a measure of clinical benefit. For instance, the first drug to treat Duchenne muscular dystrophy (DMD) received AA based on the surrogate endpoint of increased skeletal muscle dystrophin, which FDA concluded was reasonably likely to predict a clinical benefit in some DMD patients.[1] In 2012, the Food and Drug Administration Safety Innovations Act further amended the Federal Food, Drug, and Cosmetic Act (FDCA) to allow FDA to expand its authority to use an intermediate clinical endpoint, a measure of therapeutic effect that is considered reasonably likely to predict the clinical benefit, as a basis for AA. Using a surrogate or intermediate clinical endpoint allows FDA to approve these drugs earlier than possible under traditional approval. FDA may approve a drug based on an “adequate and well controlled” study that demonstrates the drug has a beneficial effect on a surrogate or intermediate clinical endpoint when the latter is considered reasonably likely to predict a real clinical benefit. FDA bases its decision on whether to accept the proposed surrogate or intermediate clinical endpoint on the scientific support for that endpoint." Read Via FDLI