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FDA Advisory Committee Preparation: Drugs (#2)


A pharmaceutical company was seeking approval for an improved formulation of a drug that reduced the number of treatments patients needed per year. During development, there was a potential signal for anaphylaxis that was concerning to the FDA.  The therapy was being used in other countries and post-marketing surveillance showed that the drug’s potential risk was similar to that seen with other available products.  The FDA questioned the benefit-risk from improved dosing since there was a potential for a life-threatening event, and other products were available. The FDA briefing document explicitly recommended non-approval.


3D collaborated with the client to credibly characterize and communicate the product’s safety parameters based on extensive reviews of all potential case reports. In order to corroborate the safety interpretation, we conducted an independent review of the safety data.  The learnings from these investigations helped define a risk evaluation and mitigation strategy (REMS) that was based on the potential risk and the patient population, and would augment safe use.  Finally, we collected data to confirm and quantify the benefits from the improved dosing regimen.  These combined efforts helped lessen uncertainty and clarified the benefit-risk profile, enabling an acceptable path to approval.


The FDA advisory committee vote was in favor of approval with augmentations to the proposed REMS program. The FDA gave clear guidance for approval, which the sponsor has agreed to implement, in order to gain approval in the forthcoming months.

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