Maple Health Group, LLC, New York, NY, USA.
EMD Serono Research & Development Institute, Inc, Billerica, MA, USA (an affiliate of Merck KGaA).
J Med Econ. 2022 Jan-Dec;25(1):1-6. doi: 10.1080/13696998.2021.2009711.
This study aimed to understand the impact of different efficacy endpoints on reimbursement decisions made by health technology assessment (HTA) bodies.
European Medicines Agency (EMA) oncology product marketing authorizations were screened to identify products that completed review by 3 HTA bodies during 2016-2019: United Kingdom's National Institute for Health and Care Excellence, Germany's Gemeinsamer Bundesausschuss, and France's Haute Autorité de Santé. Each decision's endpoint information, including overall survival (OS) and progression-free survival (PFS), was extracted. Each endpoint's influence on added benefits rating (the degree of added benefit as judged by the HTA agency) and full reimbursement (i.e. reimbursed population to label) decisions was tested using bivariate analyses.
An increasing trend was observed toward HTA submissions with immature OS data (36.8% and 71.4% in 2016 and 2019, respectively), which was a predictor of limited added benefit ( < .001). Regarding data availability, 63% of submissions provided OS, 2% provided PFS without OS; and 35% provided neither. OS availability significantly influenced added benefit ( < .001) but not full reimbursement ( > .05) decisions, whereas PFS without OS had no significant impact compared with either OS or PFS data for either outcome ( = .99).
The trend toward fewer products filing mature OS data over time suggests sponsors may be increasingly confident achieving reimbursement with surrogate endpoint data, although mature OS data provided the strongest correlation to positive reimbursement decisions. Notably, in some locally advanced settings, OS data maturity will take a long time to obtain. To expedite patient access to new medicines, payers should consider the acceptance of surrogate endpoints predictive of clinical benefit.
本研究旨在了解不同疗效终点对健康技术评估（HTA）机构做出的报销决策的影响。
筛选欧洲药品管理局（EMA）肿瘤产品的营销许可，以确定在 2016 年至 2019 年期间有 3 个 HTA 机构完成审查的产品：英国国家卫生与保健优化研究所、德国联邦联合委员会和法国卫生高级管理局。提取每个决策的终点信息，包括总生存期（OS）和无进展生存期（PFS）。使用双变量分析测试每个终点对附加效益评分（HTA 机构判断的附加效益程度）和全额报销（即标签涵盖的报销人群）决策的影响。
HTA 提交的 OS 数据不成熟的趋势呈上升趋势（2016 年和 2019 年分别为 36.8%和 71.4%），这是有限附加效益的预测因素（<0.001）。关于数据可用性，63%的提交提供 OS，2%的提交无 OS 的 PFS；35%的则两者都没有。OS 的可用性显著影响附加效益（<0.001），但不影响全额报销（>0.05）决策，而无 OS 的 PFS 与 OS 或 PFS 数据相比，对两种结果均无显著影响（=0.99）。
随着时间的推移，越来越多的产品提交不成熟的 OS 数据，这表明赞助商可能越来越有信心在使用替代终点数据实现报销，尽管成熟的 OS 数据与积极的报销决策相关性最强。值得注意的是，在一些局部晚期情况下，获得 OS 数据的成熟度需要很长时间。为了加快患者获得新药的速度，支付方应考虑接受预测临床获益的替代终点。
