Analysis of the first ten years of FDA's rare pediatric disease priority review voucher program: designations, diseases, and drug development

• Published in: Orphanet journal of rare diseases 2024-02, Vol.19 (1), p.86-86, Article 86
• Main Authors: Mease, Catherine, Miller, Kathleen L, Fermaglich, Lewis J, Best, Jeanine, Liu, Gumei, Torjusen, Erika
• Format: Article
• Language: English
• Subjects: Case studies; Child; Children; Clinical trials; Datasets; Designations; Development and progression; Diseases; Drug Approval; Drug Development; Drug therapy; Duchenne's muscular dystrophy; FDA approval; Food and Drug Administration; Genetic disorders; Humans; Incentives; Infant, Newborn; Infants (Newborn); Laws, regulations and rules; Metabolism; Monetary incentives; Neonatal care; Neonates; Neonatology; Neoplasms - drug therapy; Neuroblastoma; Oncology; Orphan Drug Production; Orphan drugs; Pediatrics; Pharmaceutical research; Prevention; Product development; Rare diseases; Rare Diseases - drug therapy; Rare pediatric diseases; Sickle cell anemia; Sickle cell disease; Statistics; United States; United States Food and Drug Administration
• ISSN: 1750-1172; 1750-1172
• DOI: 10.1186/s13023-024-03097-x

The Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) Program was enacted in 2012 to support the development of new products for children. Prior to requesting a voucher, applicants can request RPD designation, which confirms their product treats or prevents a rare disease in which the serious manifestations primarily affect children. This study describes the trends and characteristics of these designations. Details of RPD designations are not publicly disclosable; this research represents the first analysis of the RPD designation component of the program. We used an internal US Food and Drug Administration database to analyze all RPD designations between 2013 and 2022. Multiple characteristics were analyzed, including the diseases targeted by RPD designation, whether the product targeted a neonatal disease, product type (drug/biologic), and the level of evidence (preclinical/clinical) to support designation. There were 569 RPD designations during the study period. The top therapeutic areas were neurology (26%, n = 149), metabolism (23%, n = 131), oncology (18%, n = 105). The top diseases targeted by RPD designation were Duchenne muscular dystrophy, neuroblastoma, and sickle cell disease. Neonatology products represented 6% (n = 33), over half were for drug products and 38% were supported by clinical data. The RPD PRV program was created to encourage development of new products for children. The results of this study establish that a wide range of diseases have seen development-from rare pediatric cancers to rare genetic disorders. Continued support of product development for children with rare diseases is needed to find treatments for all children with unmet needs.