Posted by Michael Wonder on 29 Jul 2019
FDA updates draft guidance on rare paediatric disease priority review voucher program
29 July 2019 - The U.S. FDA today issued a revised draft guidance, Rare Paediatric Disease Priority Review Vouchers – Guidance for Industry.
The revised draft guidance incorporates public comments received on the initial draft and provides FDA’s thinking regarding the provisions of the Advancing Hope Act of 2016, which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 paediatrics 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 paediatric disease priority review voucher eligibility requirements, the rare paediatric 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The agency is accepting public comment on the revised draft guidance.
