目的:为了说明实施不同的管理进入协议的财务后果((MEA)为荷兰的自体基因治疗Atidarsageneautotemcel(AA,Libmeldy®),同时还就如何构建多边环境协定提供了第一个系统的指导,以帮助未来的报销决策并为患者提供高成本的机会,一次性潜在治愈性疗法。
方法:比较了三种支付模式:(1)任意60%的价格折扣,(2)有折扣的基于结果的利差支付,以及(3)基于结果的利差支付与带有折扣的支付意愿模型相联系。对全面反应者的财务后果进行了估计(A),根据HTA报告(B)中提供的预测临床路径做出反应的患者,和不稳定的反应者(C)。在付款协议的时间段内,普通患者的相关成本,总预算影响,并计算以患者群体的质量调整生命年表示的相关获益.
结果:当患者根据HTA报告(方案B)中的预测临床路径做出反应时,与折扣相比,实施基于结果的报销模型(模型2和模型3)具有较低的相关预算影响,同时获得类似的收益(方案1,890万欧元至660万欧元,而920万欧元)。在不稳定响应者的情况下(场景C),在基于结果的方案中,付款人的成本较低(410万欧元和300万欧元,情景2.C和3C,分别)与实施折扣(920万欧元,情景1。C).
结论:基于结果的模型可以减轻偿还AA的财务风险。当临床表现类似于或差于预期时,与简单折扣相比,这可以是相当有益的。
OBJECTIVE: To illustrate the financial consequences of implementing different managed entry agreements (managed entry agreements for the Dutch healthcare system for autologous gene therapy atidarsagene autotemcel [Libmeldy]), while also providing a first systematic guidance on how to construct managed entry agreements to aid future reimbursement decision making and create patient access to high-cost, one-off potentially curative therapies.
METHODS: Three payment models were compared: (1) an arbitrary 60% price discount, (2) an outcome-based spread payment with discounts, and (3) an outcome-based spread payment linked to a willingness to pay model with discounts. Financial consequences were estimated for full responders (A), patients responding according to the predicted clinical pathway presented in health technology assessment
reports (B), and unstable responders (C). The associated costs for an average patient during the time frame of the payment agreement, the total budget impact, and associated benefits expressed in quality-adjusted life-years of the patient population were calculated.
RESULTS: When patients responded according to the predicted clinical pathway presented in health technology assessment
reports (scenario B), implementing outcome-based reimbursement models (models 2 and 3) had lower associated budget impacts while gaining similar benefits compared with the discount (scenario 1, €8.9 million to €6.6 million vs €9.2 million). In the
case of unstable responders (scenario C), costs for payers are lower in the outcome-based scenarios (€4.1 million and €3.0 million, scenario 2C and 3C, respectively) compared with implementing the discount (€9.2 million, scenario 1C).
CONCLUSIONS: Outcome-based models can mitigate the financial risk of reimbursing atidarsagene autotemcel. This can be considerably beneficial over simple discounts when clinical performance was similar to or worse than predicted.