Washington, DC - The U.S. Food and Drug Administration Monday issued a revised draft guidance, Rare Pediatric Disease Priority Review Vouchers – Guidance for Industry. The revised draft guidance incorporates public comments received on the initial draft and provides FDA’s thinking regarding the provisions of the Advancing Hope Act of 2016, which updated the definition of a rare pediatrics disease as one that is a rare disease and one where the disease is serious or life-threatening with the serious or life-threatening manifestations primarily affecting individuals from age zero to 18.

Among the key changes from the initial draft are an explanation of the rare pediatric disease priority review voucher eligibility requirements, the rare pediatric disease designation process and examples to illustrate the agency’s current thinking on these review determinations.

“Despite continued progress, too many rare diseases still have no approved therapies. One of the FDA’s top priorities is to provide incentives and clear advice to medical product developers so that they can advance innovations that can help address the significant unmet medical needs for patients with rare diseases. This holds especially true in rare diseases that impact children,” said Janet Maynard, M.D., director of the FDA’s Office of Orphan Products Development, which along with the Office of Pediatric Therapeutics, oversees the Rare Pediatric Disease Designation program. “One program in place intended to advance the development of drugs and biologics for certain serious and life-threatening rare pediatric diseases is the Rare Pediatric Disease Priority Review Voucher Program. Under this program, a drug company that receives an approval for a drug or biologic for a rare pediatric disease may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product. To provide clarity and advice to drug developers, today we are issuing an updated draft guidance about how this program works. This updated draft guidance reflects input from rare disease and pediatric experts from across the agency, highlighting our commitment to addressing the needs of patients with rare diseases. When finalized, this draft guidance will provide the agency’s current thinking regarding the updated definition of rare pediatric disease and explain the statutory requirements to earn a rare pediatric disease priority review voucher.”