单臂试验药物医保准入评估的方法学思考

Methodological Considerations on Medical Insurance Access Assessment of Drugs Approved Based on Single Arm Trial

近年来,越来越多创新药物基于单臂试验获批上市。但单臂试验药物的四个特征,即无参照药物、无直接比对数据、测量指标为替代终点以及证据不稳定,给医保准入评估带来较大的难度和不确定性。本文结合域外实践经验与我国药品目录调整情况,为优化单臂试验药物医保准入评估方法提供建议,推进医保准入管理精细化发展。

In recent years,more and more innovative drugs have been approved based on single-arm trials.However,the four characteristics of single arm trial drugs,i.e.lack of compared drugs,lack of direct comparison data,surrogate endpoints and unstable evidence,bring great difficulty and uncertainty to the assessment of medical insurance reimbursement decision.In this paper,based on overseas experience and the adjustment of China’s National Reimbursement Drug List,suggestions were provided for optimizing the evaluation method for single arm trial drugs,in order to promote the refined development of reimbursement access management.