创新药物疗效风险分担协议的应用经验及启示

Application experience and inspiration from outcomes-based reimbursement of innovative drugs

目的研究创新药物疗效风险分担协议的应用经验,为我国创新药医保准入中引入疗效风险分担协议提供借鉴。方法选择英国、意大利、澳大利亚、西班牙以及中国台湾为研究对象,利用Excel软件建立114个协议的基本信息库和协议核心要素库,从协议的适应证分布情况、协议类型、实施维度、监测指标、数据来源与风险分担措施六个指标对样本进行分析。结果 114个协议样本涉及129个适应证,其中110个适应证属于抗肿瘤治疗领域,占样本85.3%。基于结果付款(Pb R)协议、基于证据发展的报销协议和附条件的连续治疗协议分别占比64.0%、32.5%和3.5%。在个体水平实施疗效风险分担协议占协议样本的65.8%。监测指标与疾病种类相关,79.2%的抗肿瘤药物使用总生存期作为监测指标。群体水平协议以临床研究为主要数据来源,个体水平协议以数据收集系统为主要来源。使用企业退款这一措施协议占样本的64.0%,调降支付标准和拒绝纳入目录这两类措施占样本的32.4%。结论签订抗肿瘤药物疗效风险分担协议、在个体水平使用Pb R协议、建立专门疗效数据收集系统、风险分担措施常用企业退款等经验对我国探索价值型医保改革具有借鉴意义。

AIM By analyzing the application experience of outcomes-based reimbursement(OBR) for innovative drugs to provide a reference for introducing OBR in medical insurance access of innovative drugs in China.METHODS Selecting the United Kingdom,Italy,Australia,Spain and Taiwan of China as research samples and using Excel to establish the basic information database and core element database of 114 OBR agreements,this paper analyzed from six indicators of the agreement samples:indication distributions,agreement types,implementation dimensions,monitoring indexes,data sources and risk sharing measures.RESULTS One hundred and twenty-nine indications were involved in 114 agreement samples,110 of which were antitumor drugs,accounting for 85.3% of the samples.Payment by results(PbR),coverage with evidence development and conditional treatment continuation accounted for 64.0%,32.5% and 3.5% respectively.The implementation of OBR at the individual level accounted for 65.8% of the agreement samples.The monitoring indexes were related to disease types in which 79.2% of antitumor drugs used the overall survival as the monitoring indexes.The main data source of population-level agreements was clinical study,and the main data source of individual-level agreements was data collection system.The use of enterprise refund as the risk sharing measures accounted for 64.0% of the samples,and decreasing payment standards and delisting from reimbursement catalogue were 32.4%.CONCLUSION The experience of signing OBR of anti-tumor drugs have signed more OBR,using PbR agreements at the individual level,establishing the special efficacy data collection system,the commonly using enterprise refunds for risk sharing measures can be used as a reference for exploring value-based medical insurance reform in China.