Regulatory approval, reimbursement and postmarketing evidence of class 1 novel drugs in China, 2018-2023: an observational study.

Abstract

The global pharmaceutical industry is undergoing profound changes, with countries worldwide actively investing in innovation ecosystems. In China, innovation has been incentivised through policies aimed at regulatory modernisation, value-based reimbursement and health system integration. In particular, expedited regulatory pathways and inclusion of innovative drugs into the National Reimbursement Drug List (NRDL) through value-based pricing were key approaches adopted by Chinese health authorities. To describe developments under these policies, we map the regulatory approval, reimbursement and postmarketing evidence of class 1 novel drugs across three critical phases: regulatory pathways, value-based reimbursement price negotiation and real-world clinical evidence generation. A mixed-methods approach comprising a cross-sectional study and a literature review was used to aggregate the evidence. We first identified the innovative drugs classified as 'class 1 novel drugs' by the National Medical Products Administration from 2018 to 2023. Second, we collected and analysed evidence of class 1 novel drugs, including timelines of market access and NRDL inclusion, price reduction before and after reimbursement and clinical evidence updates from market access to postmarketing. 95 class 1 novel drugs were identified as being included in the NRDL, with the median regulatory-to-reimbursement interval shortened by more than 50%, from a median of 15 to 7 months. The median price reduction after the value-based pricing was 63%. Furthermore, compared with included drugs' real-world studies (RWS), clinical trials remained the backbone of evidentiary standards for regulatory approval and reimbursement decisions as they demonstrated superior methodological robustness compared with RWS, which often had limitations related to small sample sizes and inconsistent endpoint definitions. By linking regulatory approval, reimbursement and postmarketing evidence for China's class 1 novel drugs, this study provides an integrated evidence base to inform policy discussions on balancing innovation incentives, affordability and evidence generation.