As Prescribed


While many provisions of the FY 2023 Consolidated Appropriations Act (Omnibus) have received much attention, one has flown under the radar. In its explanatory statement on the Omnibus, the US Congress indicated that it is keeping an eye on how FDA approves orphan drug products. 

Congress states that it “encourages 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 requires 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 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. This is exemplified by how FDA’s authority to exercise regulatory flexibility when making approval decisions for rare disease products is inconsistently applied. If FDA does not adopt Congress’s recommendations, Congress could legislate the requirements, restricting FDA’s autonomy on advisory committee make-up.

For drug sponsors, congressional focus on the process of approving products for rare diseases will be considered a small step in the right direction and may provide rare disease product sponsors with added leverage to ensure that persons with the appropriate expertise are reviewing products. In the larger context of FDA approvals for rare disease treatments, this language may be a signal that significant changes in the orphan product review and approval process are on the way.

Dr. Peter Marks, director of the Center for Biologics Evaluation and Research, recently announced that FDA will pilot an Operation Warp Speed program for orphan products and is considering a process for the coordinated review of cell and gene therapies—which are frequently for rare or ultra-rare diseases—with other global regulators.

Rare disease product sponsors should keep a close eye on the new regulatory processes being put in place as they may present new opportunities to expedite development programs.