Impact of China's Drug Review and Approval System Reform on Pediatric Drugs: an Analysis Based on Registration Data from 2015 to 2024.

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.