FDA Launches New Advisory Committee for Genetic Metabolic Diseases

Adding to the lengthy list of advisory committees under its belt, the United States Food and Drug Administration (FDA) announced its plans to create a new advisory committee to specialize in genetic metabolic disease treatments. The announcement, published on December 12, 2023, clarified that the advisory committee would fall under the Division of Rare Diseases and Medical Genetics, a subset of the Center for Drug Evaluation and Research (CDER) Office of Rare Diseases, Pediatrics, Urologic, and Reproductive Medicine (ORPURM).

FDA advisory committees play a critical role in evaluating and approving medical products. Comprised of multiple members with varying backgrounds, the committees evaluate products on safety, efficacy, and appropriate use, using these conclusions to advise the FDA on regulatory actions, such as drug approval.

“Advisory committees allow the FDA to receive invaluable input from clinicians, industry experts, academia, patients, caregivers, and other external stakeholders when evaluating the potential benefits and risks of a new therapy. They are an essential part of the FDA’s work,” Patrizia Cavazzoni, MD, director of the FDA’s CDER, said in the press announcement. “These expert panels support the agency by helping ensure the FDA’s decisions on the safety and efficacy of drug products are informed and in line with our priorities and goals.”

The nine members, including the committee chairperson, are recruited based on their expertise and may include researchers, engineers, medical personnel, chemists, biologists, and more. For this panel, the administration is looking to recruit experts on metabolic genetics, management of inborn errors of metabolism, small population trial design, translational science, pediatrics, epidemiology, or statistics.

Typically, the average term is 1–4 years, and other individuals that represent consumers, industry, and patients supplement the panel. The primary advisory board is chosen based on nominations, with nomination packages requiring a complete curriculum vitae, acknowledgment and consent forms, and at least one letter of recommendation. Consumer and industry representatives can also be nominated; however, industry members do not vote to maintain an unbiased decision process. Patient representatives can be recruited through the FDA Patient Representative Program.

“Genetic metabolic diseases include very rare diseases that individually affect a limited number of patients. Drug development for these conditions has unique and complex challenges; therefore, few treatments are available to patients,” said Janet Maynard, MD, MHS, director of ORPURM. “This new advisory committee will provide a forum for discussion of complicated issues with specialized and diverse technical and scientific experts in the field of metabolic genetics.”