Department of Economics, University of La Rioja, La Ciguena 60, 26006, Logroño, Spain.
Pharmacoeconomics. 2018 Jul;36(7):733-743. doi: 10.1007/s40273-018-0619-4.
In this article, we model the behavior of a pharmaceutical firm that has marketing authorization for a new therapy believed to be a candidate for personalized use in a subset of patients, but that lacks information as to why a response is seen only in some patients. We characterize the optimal outcome-based reimbursement policy a health authority should follow to encourage the pharmaceutical firm to undertake research and development activities to generate the information needed to effectively stratify patients. Consistent with the literature, we find that for a pharmaceutical firm that does not undertake research and development activities, when the treatment fails, the total price of the drug must be returned to the healthcare system (full penalization). By contrast, if the firm undertakes research and development activities that make the implementation of personalized medicine possible, treatment failure should not be fully penalized. Surprisingly, in some cases, particularly for high-efficacy drugs and small target populations, the optimal policy may not require any penalty for treatment failure. To illustrate the main results of the analysis, we provide a numerical simulation and a graphical analysis.
在本文中，我们对一家拥有新疗法营销授权的制药公司的行为进行建模，该疗法被认为是亚组患者个性化使用的候选药物，但缺乏为什么仅在某些患者中观察到反应的信息。我们描述了卫生当局应遵循的基于最佳结果的报销政策，以鼓励制药公司开展研究和开发活动，以生成有效分层患者所需的信息。与文献一致，我们发现，如果制药公司不开展研究和开发活动，导致治疗失败，那么药物的总价格必须返还给医疗保健系统（全额罚款）。相比之下，如果公司开展了使个性化医疗成为可能的研究和开发活动，则不应对治疗失败进行全面处罚。令人惊讶的是，在某些情况下，特别是对于高疗效药物和小目标人群，最佳政策可能不需要对治疗失败进行任何处罚。为了说明分析的主要结果，我们提供了数值模拟和图形分析。
