PDF(Pubmed)
Abstract：
BACKGROUND: 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.
RESULTS: 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.
CONCLUSIONS: 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.
RESULTS: 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.
CONCLUSIONS: 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.
摘要：
背景：罕见儿科疾病（RPD）优先审查凭证（PRV）计划于2012年颁布，以支持儿童新产品的开发。在申请凭证之前，申请人可以申请RPD指定，这证实了他们的产品治疗或预防一种罕见的疾病，其中严重的表现主要影响儿童。本研究描述了这些名称的趋势和特征。RPD名称的详细信息不可公开披露；这项研究是对该计划的RPD名称组成部分的首次分析。
结果：我们使用美国食品和药物管理局内部数据库分析了2013年至2022年之间的所有RPD名称。分析了多个特征，包括RPD指定的目标疾病，该产品是否针对新生儿疾病，产品类型（药物/生物制剂），以及支持指定的证据水平（临床前/临床）。在研究期间有569个RPD名称。最高的治疗领域是神经病学(26%，n=149),新陈代谢(23%，n=131),肿瘤学（18%，n=105）。RPD指定的主要目标疾病是杜兴氏肌营养不良症，神经母细胞瘤,镰状细胞病.新生儿科产品占6%(n=33)，超过一半用于药物产品，38%得到临床数据支持。
结论：创建RPDPRV计划是为了鼓励儿童开发新产品。这项研究的结果证实，从罕见的儿科癌症到罕见的遗传疾病，已经出现了广泛的疾病。需要继续支持为患有罕见疾病的儿童开发产品，以便为所有未满足需求的儿童寻找治疗方法。
结果：我们使用美国食品和药物管理局内部数据库分析了2013年至2022年之间的所有RPD名称。分析了多个特征，包括RPD指定的目标疾病，该产品是否针对新生儿疾病，产品类型（药物/生物制剂），以及支持指定的证据水平（临床前/临床）。在研究期间有569个RPD名称。最高的治疗领域是神经病学(26%，n=149),新陈代谢(23%，n=131),肿瘤学（18%，n=105）。RPD指定的主要目标疾病是杜兴氏肌营养不良症，神经母细胞瘤,镰状细胞病.新生儿科产品占6%(n=33)，超过一半用于药物产品，38%得到临床数据支持。
结论：创建RPDPRV计划是为了鼓励儿童开发新产品。这项研究的结果证实，从罕见的儿科癌症到罕见的遗传疾病，已经出现了广泛的疾病。需要继续支持为患有罕见疾病的儿童开发产品，以便为所有未满足需求的儿童寻找治疗方法。
