AGG Food & Drug team leader Alan Minsk was quoted in a Pink Sheet article titled, “What ‘Confirmatory Evidence’ Does US FDA Need When Sponsors Want to Rely on a Single Study,” published on October 10, 2022.
FDA’s recent approval of Amylyx Pharmaceuticals Inc.’s amyotrophic lateral sclerosis drug Relyvrio highlights when the agency may accept confirmatory evidence to support approval based on a single adequate and well-controlled trial. Regarding its approval, the agency said data on an overall survival benefit can serve as confirmatory evidence to support use of the Phase II CENTAUR trial to approve the ALS drug, even though the survival data were collected in the same study. Although not unprecedented, the decision was unusual, and shows the agency’s flexibility to stretch its 2019 guidance, “Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products,” for a disease with high unmet need.
Alan predicted the single trial approach is “going to be the exception, not the rule,” and elaborated that it will be applied in cases where there is an unmet need.
“If you’re going to go for one, you better nail the one,” he said, emphasizing the risks of planning to rely on a single study.
As more concrete examples of when and what types of confirmatory evidence are acceptable may be coming soon, Alan stated it would be helpful if the new guidance contains a discussion of therapeutic areas where the single trial approach has been used, common mistakes that sponsors make, and what the agency has learned since the 2019 guidance.
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