FDA issues a revised Draft Guidance – Rare Pediatric Disease Priority

The U.S. Food and Drug Administration issued a revised draft guidance, Rare Pediatric Disease Priority Review Vouchers – Guidance for Industry. The revised draft guidance incorporates public comments received on the initial draft and provides FDA’s thinking regarding the provisions of the Advancing Hope Act of 2016, which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.

The agency is accepting public comment on the revised draft guidance.

 For more information, please visit: FDA In Brief.

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