A Middle Ground for Accelerated Drug Approval—Lessons From Aducanumab

This Viewpoint argues that the US Food and Drug Administration accelerated approval needs to be improved, with 4 potential changes suggested, based on consideration of the experience with aducanumab.

SPS commentary:

The following 4 changes are suggested:

 

  • 1) Surrogate or intermediate end points that are established as highly or moderately correlated with long-term clinical benefit should be used as primary outcome measures in the accelerated approval trials. Aducanumab was approved in the US for the treatment of Alzheimer disease on the basis of reduction in beta amyloid plaque, which has not been linked to cognitive improvement or delayed clinical decline in patients with dementia.
  • 2) Accelerated approval should not be granted until after there is agreement between the FDA and companies regarding the protocol for the confirmatory trial and recruitment has begun.
  • 3) There must be transparency regarding recruitment, the progress of the confirmatory trials, and a firm deadline for completion. Otherwise, the drug should be automatically withdrawn from the market
  • 4) Unless there is compelling scientific evidence, the indication for the drug should be that used for inclusion in the trials used for accelerated approval.

Journal of the American Medical Association