Advisory Committee Changes May Be on the Horizon for Orphan Drug Approval

May 5, 2023

The US Food and Drug Administration (FDA) relies on recommendations from its several advisory committees when weighing whether to approve a drug. However, Congress is placing extra scrutiny on the composition of these committees when debating orphan drugs, as laid out in an explanatory statement in the 2023 FY Consolidated Appropriations Act. Currently, they are expected to be populated with experts in the general field, but there is no provision to make sure rare disease experts are on the panel.

According to Jacqueline R. Berman and Angela Silva, “Despite the idea of a rare disease expert pool failing to move forward, the issue of advisory committee composition has not retreated. Rather, there is renewed congressional attention in the space. In the explanatory statement to the omnibus, Congress “encourage[d] the FDA to work to include no less than two expert members on each advisory committee when that committee is reviewing a drug that has been designated as an orphan drug” and required that FDA “report the percentage of recommendations made by advisory committees with respect to orphan drugs.” Including a provision that encourages FDA to give experts a place on orphan drug advisory committees indicates that Congress shares industry and patient concerns that the FDA advisory committee members and reviewers may not have the necessary experience with certain diseases to make difficult decisions on the risk-benefit analysis for treatments.”

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