Expedited development

Definition:

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.