Expedited development

Accelerated review of new treatments by the US Food and Drug Administration (FDA).

The FDA has established four mechanisms to speed up the development and approval of new drugs:

• Priority review is for products intended for treatment, prevention or diagnosis of a serious condition. To qualify for priority review status, a product must show significantly improved safety or efficacy. The main advantage of priority review is a commitment to more rapid FDA assessment (a decision within 6 months rather than 10 months).
• Accelerated approval applies to products for a serious or life-threatening condition, and allows developers to infer efficacy based on the use of a surrogate endpoint that is reasonably likely to predict a clinical benefit. As a result, clinical trials can be shorter. Post-licensing studies are generally required to confirm that the anticipated longer-term clinical benefits are actually achieved.
• Fast-track designation is for drugs intended to be used alone or in combination to treat serious or life-threatening disease and to address unmet needs, such as greater efficacy, enhanced safety or use in a special population. The FDA undertakes more rapid review, potentially including approval following preliminary evaluation of clinical data. Developers are also entitled to regular meetings with the FDA review team.
• Breakthrough designation is applied to drugs intended to be used alone or in combination to treat a serious or life-threatening disease. Preliminary clinical evidence should indicate that the drug is a substantial improvement over current therapies. This evidence can include data from phase 1 or 2 trials and be based on surrogate endpoints. As well as this advantage, developers benefit from close and collaborative dialogue with the FDA to accelerate clinical development.

Fact Sheet: Breakthrough Therapies (FDA, 2018)

Guidance for industry expedited programs for serious conditions –Drugs and biologics (FDA, 2013)

Overview of FDA expedited programs with a focus on breakthrough therapy (FDA, 2015) 

Expedited Programs for Serious Conditions––Drugs and Biologics (FDA, 2014)