The Food and Drug Administration (FDA) has recently turned down two applications for accelerated approval submitted by Regeneron Pharmaceuticals. The agency's decision stemmed from Regeneron's failure to initiate confirmatory studies designed to verify the drugs' clinical effectiveness. This move may indicate a more stringent approach by the FDA, leveraging the expanded powers granted by Congress in 2022 to ensure that accelerated approvals are followed by timely and conclusive evidence of patient benefit.
The FDA's accelerated approval pathway is intended to expedite the availability of treatments for serious conditions where there is an unmet medical need. It allows for approval based on surrogate endpoints reasonably likely to predict clinical benefit, contingent upon the completion of post-approval confirmatory trials. The recent rejections suggest a heightened emphasis on the timely commencement of these confirmatory studies.
This decision could be interpreted as a proactive measure to prevent situations where drugs remain on the market under accelerated approval without sufficient evidence of efficacy. However, it may also raise concerns about potentially delaying access to promising therapies for patients with life-threatening illnesses. The balance between ensuring drug efficacy and facilitating timely access remains a critical consideration for the FDA.
