Since the US Food and Drug Administration (FDA) established its accelerated drug approval pathway 28 years ago, more than 2 in 5 drugs granted fast-track approval have not been confirmed clinically effective as required, an in-depth investigation published online today in The BMJ has determined.
"Despite the pathway's good intentions to accelerate 'the availability of drugs that treat serious diseases,' experts are concerned that it is now being exploited — to the detriment of patients, who may be prescribed a drug that offers little benefit and possible harm, and to taxpayers," writes Elisabeth Mahase, clinical reporter at The BMJ, who carried out the analysis.
The FDA's accelerated approval pathway is intended to provide earlier access to drugs for serious diseases when there is lingering uncertainty at the time of approval regarding the drug's ultimate clinical benefit.
Required Studies Rarely Completed
As part of this fast-track pathway, drug manufacturers must conduct postapproval, phase 4 confirmatory trials to verify the anticipated clinical benefit. If these trials indicate no benefit, FDA approval can be withdrawn.
However, the analysis of FDA data shows once they are approved drugs are rarely taken off the market.
The BMJinvestigation that analyzed data up to the end of 2020 shows that 112 of the 253 (44%) medications granted accelerated approval have not been confirmed to be effective.
