US FDA breakthrough therapy designation and consumer drug advertising: a recipe for confusion

In 2012, the US Congress gave the Food and Drug Administration authority to grant “breakthrough therapy” designation to expedite development and regulatory review of new drugs. The designation is used for potential treatments for serious conditions when preliminary evidence suggests they may perform substantially better than available alternatives on “clinically significant endpoints.” Preliminary evidence can include surrogate marker endpoints reasonably likely to predict clinical benefit, such as imaging changes and biomarkers. The programme has been successful in at least one way: an analysis of FDA approved drugs from 2015 to 2022 found that breakthrough designation was associated with a two year reduction in clinical development time.1 However, these therapies are approved on the basis of less rigorous evidence, often a single pivotal clinical trial instead of two, and trials that are smaller and weaker in design.2Manufacturers had requested around 1400 breakthrough designations for candidate drugs as of October 2023, 537…
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