目的:管理进入协议(MEAs),尤其是基于财务的协议在欧洲国家通常用于创新癌症药物。这些协议促进了获得创新治疗的机会,同时减轻了付款人的财务风险。这项研究的重点是荷兰政府为偿还pembrolizumab而达成的机密价格协议,扩大适应症对成本效益的影响,以及该协议的可行性或可取性。
方法:我们选择了五个适应症,其中派姆单抗被认为是有效的,并为每个适应症开发了部分生存模型。利用已发表试验的生存和无进展生存数据来重建个体患者数据,我们使用参数模型推断30年的时间范围。生活质量和成本的投入来自现有文献,并被索引。
结果:根据适应症,每个质量调整生命年(QALY)的增量成本效益比(ICER)在35,313欧元至322,349欧元之间。只有一个适应症低于80,000欧元(或100,000欧元)的成本效益阈值。在应用荷兰内部药品的平均报告折扣时,ICER在每QALY收益20,881欧元至252,934欧元之间,并且在五个指标中的三个指标中达到了80,000欧元(或100,000欧元)的门槛。
结论:我们的结果表明,派姆单抗在某些适应症中可能具有成本效益,取决于建立的保密价格协议。然而,当价格固定在一个适应症上时,报销不具成本效益的护理的可能性仍然是可能的。基于适应症的定价(IBP)可以帮助调整受适应症扩大的创新药品的价值和价格。
OBJECTIVE: Managed entry agreements (MEAs) and especially financial based agreements are commonly used in European countries for innovative cancer pharmaceuticals. These agreements facilitate access to innovative treatments while mitigating financial risks for payers. This study focuses on the confidential price agreement made by the Dutch government for the reimbursement of
pembrolizumab, the implications of broadening indications on cost-effectiveness, and the viability or desirability of said agreement.
METHODS: We selected five indications where
pembrolizumab was deemed effective and developed portioned survival models for each indication. Survival and progression-free survival data from the published trials were utilized to recreate individual patient data and we extrapolated --using parametric models-- to a time horizon of 30 years. Inputs for both quality of life and costs were derived from available literature and were indexed.
RESULTS: The incremental cost-effectiveness ratios (ICERs) ranged between €35,313 and €322, 349 per quality-adjusted life-year (QALY) depending on the indication. Only one indication fell under the €80,000 (or €100,000) cost-effectiveness threshold. When applying the average reported discount on intramural pharmaceuticals in the Netherlands, ICERs ranged between €20,881 and €252,934 per QALY gained, and the €80,000 (or €100,000) threshold was met in three indications out of five.
CONCLUSIONS: Our results show that
pembrolizumab could be cost-effective in some indications, depending on the confidential price agreement established. However, the possibility of reimbursing not cost-effective care when the price is anchored in one indication remains possible. Indication-based pricing (IBP) could help align value and price for innovative pharmaceuticals that are subject to indication broadening.