Ther Innov Regul Sci. 2025 Jul 16. doi: 10.1007/s43441-025-00842-7. Online ahead of print.
ABSTRACT
This study systematically evaluated the impact of China’s drug review system reform on pediatric drug approval efficiency using 310 pediatric drug registration records (2015-2024) from the CDE of China’s NMPA supplemented with the YaoZhi Database, with comparative analysis of adult drug review patterns. Post-2019 amendments to the DAL significantly reduced pediatric review timelines from a median of 450 days (2015-2019) to 377 days (2020-2024; P < 0.0001), representing a 16.4% reduction versus adult drugs (451 days). The standard review pathway showed even greater pediatric acceleration (385 days vs. adult 497.5 days; 22.6% reduction, P < 0.0001), demonstrating targeted regulatory resource allocation. While biologics exhibited significant review advantages (342 days vs. chemical drugs’ 403 days; P = 0.0085), structural imbalances persisted: high import dependency (73.9% imported vs. 26.1% domestic), inadequate child-appropriate formulations (< 10%), and critical therapeutic gaps (traditional Chinese medicines: 1.3%; rare disease drugs: < 5%). Efficiency gains were linked to expanded priority review adoption and optimized technical standards, yet unresolved deficits necessitate: establishing a dedicated pediatric review database with unified standards and conditional access; optimizing resource allocation for clinically urgent drugs; enhancing rare disease incentives; and accelerating age-appropriate formulation innovation-collectively enhancing regulatory science to address pediatric clinical needs.
PMID:40668501 | DOI:10.1007/s43441-025-00842-7
