Many drugs that treat serious or life-threatening conditions pursue long-term clinical benefits, such as improved survival or decreased morbidity, that would take years to track in clinical trials. Accelerated Approval allows the FDA to approve drugs based on more easily-obtained data that predicts these long-term benefits. Sponsors are then required to prove efficacy in post-market trials. The Accelerated Approval regulations were first instituted by the FDA in 1992 in order to speed the availability of new drugs to treat HIV/AIDS. The designation has also been frequently applied to cancer drugs and legislation passed in 2012 included a provision to expand its use to other conditions.
- To be considered for Accelerated Approval, a drug must address a serious or life-threatening condition and
- The drug must demonstrate effect on an intermediate clinical endpoint (or surrogate endpoint)—a result that is reasonably likely to predict clinical long-term benefit and can be measured earlier than that benefit (e.g. tumor shrinkage can be used as an endpoint to predict survival benefit in some instances of cancer).
- FDA judgment takes into account the severity, rarity, or prevalence of the condition and the availability or lack of current treatments.
When to Submit a Request:
- Accelerated Approval impacts clinical trial design and post-market planning, so drug sponsors are generally advised to discuss it with FDA during development.
- A drug granted Accelerated Approval is approved based on evidence of impact on a surrogate endpoint rather than evidence of impact on the actual clinical benefit the drug is intended to provide.
- This approval is generally conditional on a sponsor’s agreement to demonstrate the drug’s long-term safety and efficacy in post-approval trials. If a sponsor fails to do this, either by refusing to conduct a trial or by conducting a trial that finds the drug to be unsafe or ineffective, FDA may withdraw approval.
Fast Track vs. Breakthrough Therapy:
- Priority Review, Fast Track, and Breakthrough drugs can also be eligible for Accelerated Approval generally advised to discuss it with FDA during development.
The Perfect Candidate: Accelerated Approval most benefits drugs pursuing long-term clinical outcomes with predictive intermediate endpoints where patients cannot afford to wait through lengthy pre-approval trials, e.g. a drug designed to improve survival in patients with HIV that demonstrates the ability to reduce the amount of virus in the blood stream.