Premium
Inclusion of Infants and Neonates in Pediatric Orphan Product Approvals
Author(s) -
Park Kyunghun,
Virparia Riddhi,
Green Dionna J.,
Epps Carla,
Wharton Gerold T.,
McCune Susan K.,
Burckart Gilbert J.
Publication year - 2021
Publication title -
clinical pharmacology and therapeutics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.941
H-Index - 188
eISSN - 1532-6535
pISSN - 0009-9236
DOI - 10.1002/cpt.2306
Subject(s) - orphan drug , medicine , pediatrics , food and drug administration , population , clinical trial , off label use , newborn screening , pharmacology , environmental health , bioinformatics , biology
The Orphan Drug Act (ODA) of 1983 was enacted to provide financial incentives to drug sponsors to develop therapies for rare diseases. Although this act increased the number of orphan products approved, there are still a limited number of products available for the pediatric population because orphan drug products are exempt from the Pediatric Research Equity Act. The objectives of this study were (i) to evaluate the pediatric orphan drug studies submitted to the US Food and Drug Administration (FDA) in the period of 2007–2018 and (ii) to examine whether orphan drug products were fully labeled with a pediatric indication in infants and neonates. Out of the 468 indications evaluated, 171 (37%) were FDA‐labeled for use in the pediatric population. Labeling for the 12 to < 18 years age group was most common (98%). Fifty‐two percent of FDA‐labeled pediatric indications included the newborn to < 2 years of age group. In this newborn to < 2 years age group, the indication was labeled without pivotal clinical trials in 43% of the programs. Of the 60 new indications not labeled down to birth, 50% were found to have an age of onset and diagnosis that occurs earlier than the age approved for use of the product for that indication. In summary, although the ODA has been successful in improving pediatric access to medications for rare diseases, our analysis identified the incomplete labeling for pediatric patients under 2 years of age. Strategies to include the birth to < 2 years old group of pediatric patients in orphan drug development programs should be explored.