Health insurance reimbursement management of off-label drug use in the United States and its implications for China
- VernacularTitle:美国超说明书用药医保支付管理研究及对我国的启示
- Author:
Keyao CAO
1
;
Wei LI
1
Author Information
1. School International of Pharmaceutical Business，China Pharmaceutical University，Nanjing 211198，China
- Publication Type:Journal Article
- Keywords:
off-label drug use;
health insurance fund;
prior authorization;
health insurance reimbursement;
evidence-based decision-making
- From:
China Pharmacy
2026;37(12):1535-1540
- CountryChina
- Language:Chinese
-
Abstract:
OBJECTIVE To provide reference for improving relevant policies of health insurance reimbursement management for off-label drug use in China. METHODS The institutional framework and core mechanisms of public health insurance reimbursement for off-label drug use in the United States were systematically reviewed. Experiences were summarized and optimization suggestions for health insurance reimbursement of off-label drug use in China were proposed. RESULTS & CONCLUSIONS The United States has established a clearly defined institutional framework with division of responsibilities between drug regulation and health insurance reimbursement： FDA is responsible for marketing approval and promotional regulation， while the Centers for Medicare & Medicaid Services （CMS） is responsible for formulating reimbursement policies， distinguishing between “whether a drug can be used” and “whether a drug can be reimbursed”. Reimbursement management is built on an evidence-based decision-making foundation， primarily relying on dynamically updated authoritative drug compendia， supplemented by published medical literature. CMS dynamically updates the list of authoritative drug compendia through an annual public application and evaluation process. Additionally， prior authorization mechanisms are employed to conduct prospective reviews of high-cost， high-risk off-label drug uses， thereby controlling the risk to health insurance funds. It is recommended that China pilot evidence-based reimbursement for off-label drug use in disease areas with prominent clinical needs， relying on real-world research evidence and selecting drugs with certain evidence-based support to explore reimbursement pathways for off-label drug use. Meanwhile， prior authorization mechanisms should be introduced， combined with health insurance information platforms to achieve standardized reviews， so as to enhance the controllability of health insurance fund expenditures while ensuring rational drug use and safeguarding patients’ health rights and interests.
