Implementation of the FDA Accelerated Approval pathway raises questions

Questions about the US Food and Drug Administration’s Accelerated Approval pathway are revealed today (August 15) in a study published in the Journal of the American Medical Association (JAMA).

Researchers from the London School of Economics and Political Science (LSE) and Brigham and Women’s Hospital/Harvard Medical School studied the clinical trials used by the FDA to authorise and confirm drugs via the Accelerated Approval between 2009 and 2013. They found that at least three years after being fast-tracked to market – and in some cases seven years and counting – half of the drugs had still not had the required post-approval studies completed. In most cases, the reasons behind these delays were not publicly available.

The Accelerated Approval pathway allows the FDA to expedite the approval of drugs treating serious illnesses or addressing unmet medical need on the basis of so-called surrogate measures – laboratory measurement, radiographic images, physical signs or other measures – that may not be strongly tied to actual patient clinical benefit. Once approved, these drugs must then undergo further clinical studies to determine whether the suggested benefits translate into real-life clinical improvements. If these studies fail to demonstrate clinical benefit, the FDA may withdraw the drug from market.