The FDA is creating a new advisory committee related to potential treatments for genetic metabolic diseases. The Genetic Metabolic Diseases Advisory Committee will advise the agency on products used for the diagnosis, prevention or treatment of genetic metabolic diseases under the purview of the Division of Rare Diseases and Medical Genetics. It will also provide the FDA independent, knowledgeable advice and recommendations on technical, scientific and policy issues around medical products for genetic metabolic diseases. Committee members will evaluate evidence on key issues about the applications brought before the advisory committee and offer their recommendations for FDA consideration. The group will be comprised of experts in the areas of metabolic genetics, management of inborn errors of metabolism, small population trial design, translational science, pediatrics, epidemiology or statistics and related specialties.

“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,” said Patrizia Cavazzoni, M.D., director of the FDA’s Center for Drug Evaluation and Research (CDER). “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 committee will consist of nine voting members, including the committee chairperson. Individuals nominated as scientific members must be technically qualified experts in their relevant fields and have experience interpreting complex data. The committee will also include a consumer representative and an industry representative. Non-Federal members of this committee will serve either as special government employees or non-voting representatives. Members will be invited to serve for overlapping terms of up to four years.

“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, M.D., M.H.S., director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine (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.”

The FDA is encouraging qualified individuals interested in serving or nominating a representative to serve on the committee to submit nominations. Nominations can either be submitted electronically through the FDA Advisory Committee Membership Nomination Portal or by mail.