Use Accelerated Approval for Rare Diseases: Commentary

FDA should use accelerated approval and other regulatory techniques to speed the development and approval of treatments. That’s the thesis in a Salon commentary written by the executive director of the National Mucopolysaccharidoses Society. The commentary says that to date, agency regulators have not used accelerated approval, even for promising drug candidates.

“Regulators rarely approve promising treatments without time-consuming, logistically impossible, and ethically flawed clinical trials, even in cases when alternative regulatory pathways may exist,” the comment says. “This must change. Drug regulators must expand the use of accelerated approval for rare disease treatments. Lives hang in the balance…. It’s imperative that FDA review promising drugs appropriately via accelerated approval. Doing so will not only save patients, but also reignite interest in rare-disease research more broadly.”