OBJECTIVES:

Accelerated approval time for innovative drugs improved patient accessibility in China, while adding difficulties to the assessment of effectiveness and safety for reimbursement decisions. The national payer in China is embracing real world evidence (RWE) to bridge the gap in annual national reimbursement drug list (NRDL) negotiation. The study aims to describe the current landscape of RWE in the reimbursement decision-making process, to identify challenges and opportunities, and to provide insights for future development.

METHODS:

Literature review was performed by searching HTA agency websites from NICE, HAS, CADTH, PBAC, and IQWiG. Publicly available materials for all negotiated drugs in 2017-2021 NRDL were reviewed and analyzed. In-depth interviews with local key opinion leaders (KOLs) in health economics were also conducted to understand the use of RWE in China reimbursement decision-making.

RESULTS:

Global HTA submissions supported with RWE increased from 6% in 2011 to 39% in 2021 and increased acceptance of RWE was observed. In contrast, among 143 drugs summited for 2022 NRDL, only 5 cases (3%) included RWE based on local data and they mainly focused on cost related measures. Their impact on reimbursement decision-making is also relatively difficult to be assessed. Major factors limiting the application of RWE in NRDL include short time window between NDA approval and NRDL submission, limited availability of secondary data source, and no explicit rules on how RWE contributes to reimbursement decision. To drive more widely use of RWE, its application in supporting NRDL renewal and potential conditional reimbursement agreement for high-value drugs can be explored, while establishing clear policy framework to guide future advancement.

CONCLUSIONS:

There is growing recognition in the value of RWE for reimbursement decision-making in China. Further advancement requires joint efforts from broad stakeholders to establish guidelines that prioritize the applicable areas, ensure transparency in process, and enhance data availability.