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

被引：0

作者：

Mease, Catherine ^{[1
]}

Miller, Kathleen L. ^{[1
]}

Fermaglich, Lewis J. ^{[1
]}

Best, Jeanine ^{[2
]}

Liu, Gumei ^{[3
]}

Torjusen, Erika ^{[1
]}

机构：

[1] US FDA, Off Orphan Prod Dev, Off Commissioner, 10903 New Hampshire Ave, Silver Spring, MD 20993 USA

[2] US FDA, Off Pediat Therapeut, Off Commissioner, 10903 New Hampshire Ave, Silver Spring, MD 20993 USA

[3] US FDA, Off Therapeut Prod, Ctr Biol Evaluat & Res, 10903 New Hampshire Ave, Silver Spring, MD 20993 USA

来源：

ORPHANET JOURNAL OF RARE DISEASES | 2024年 / 19卷 / 01期

关键词：

Rare pediatric diseases; Food and Drug Administration; Children; Designations; Drug development; CLINICAL-TRIAL ENROLLMENT;

D O I：

10.1186/s13023-024-03097-x

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

BackgroundThe 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.ResultsWe 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.ConclusionsThe 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.

引用

页数：9

共 18 条

[1] Correction to: Analysis of the first ten years of FDA’s rare pediatric disease priority review voucher program: designations, diseases, and drug development
Catherine Mease
Kathleen L. Miller
Lewis J. Fermaglich
Jeanine Best
Gumei Liu
Erika Torjusen
[J]. Orphanet Journal of Rare Diseases, 19
[2] Impact Of The Priority Review Voucher Program On Drug Development For Rare Pediatric Diseases
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[J]. PLOS NEGLECTED TROPICAL DISEASES, 2018, 12 (08):
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[8] Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases
Miller, Kathleen L.
Fermaglich, Lewis J.
Maynard, Janet
[J]. ORPHANET JOURNAL OF RARE DISEASES, 2021, 16 (01)
[9] Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases
Kathleen L. Miller
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[J]. Orphanet Journal of Rare Diseases, 16
[10] Association of the Priority Review Voucher With Neglected Tropical Disease Drug and Vaccine Development
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