Department of Health Technology and Services Research, University of Twente, Enschede, the Netherlands.
The Netherlands Cancer Institute, Amsterdam, the Netherlands.
JAMA Oncol. 2020 Jun 1;6(6):909-916. doi: 10.1001/jamaoncol.2019.6846.
The financial consequences of high-expenditure innovative drugs and the association of these consequences with access to cancer treatment are substantial. With oncology being one of the major spending blocks of care and research, innovative policies are needed to secure the sustainability and accessibility of health care systems. Despite this strong interest, structured approaches are missing to date, and proposals are often based on opinion rather than fact.
To evaluate an inventory of policies to reduce drug prices at market launch and analyze the quantitative evidence on which these policies are based.
For this systematic review, a literature search of the Ovid MEDLINE, Embase, Business Source Premier, ABI/Inform, World Health Organization, and Organisation for Economic Co-operation and Development databases was conducted for articles published between January 1, 2001, and December 31, 2017. Publications that described proposed policies with a direct or obvious indirect association with pharmaceutical prices at market launch and with relevance to oncology and high-income countries were included. Evidence basis was assessed per article, and quantitative articles were categorized according to time and data use. Main price mechanisms and scored system disruptiveness per proposal were identified. Data were analyzed from January 1, 2018, to January 1, 2019.
Of the 4775 articles screened, 80 were selected, and an inventory of 23 policies to reduce medicine prices was created. Proposals were diverse but mainly applied the strengthening of competition as an underlying mechanism to reduce drug prices. Of the 80 studies, 23 used quantitative models, but existing evidence was insufficient to deduce price effects, especially considering system disruptiveness. The available evidence was used to identify promising proposals for which testing may be beneficial: transparency, delinkage, 2-part pricing, public research, orphan drug reform, and public clinical trials.
The findings suggest that despite the urgency of the search for proposals that lead to sustainable drug prices, careful and structured testing of proposals is needed because the implications for access to drug treatment can be substantial.
高支出创新药物的财务后果以及这些后果与癌症治疗可及性之间存在关联，这两者都非常重要。由于肿瘤学是医疗保健和研究的主要支出领域之一，因此需要创新政策来确保医疗保健系统的可持续性和可及性。尽管对此有着强烈的兴趣，但迄今为止仍缺乏结构化的方法，而且这些提案往往基于意见而非事实。
评估降低市场推出时药品价格的一揽子政策，并分析这些政策所依据的定量证据。
为了进行这项系统评价，我们对 Ovid MEDLINE、Embase、Business Source Premier、ABI/Inform、世界卫生组织和经济合作与发展组织数据库进行了文献检索，检索了 2001 年 1 月 1 日至 2017 年 12 月 31 日期间发表的文章。纳入的文献描述了与市场推出时药品价格直接或明显间接相关且与肿瘤学和高收入国家相关的拟议政策。按文章评估证据基础，并根据时间和数据使用情况对定量文章进行分类。确定了每种提案的主要价格机制和评分系统的破坏性。数据分析于 2018 年 1 月 1 日至 2019 年 1 月 1 日进行。
在筛选出的 4775 篇文章中，有 80 篇被选中，并创建了一个降低药品价格的 23 项政策清单。这些提案多种多样，但主要应用了竞争加强作为降低药品价格的基本机制。在 80 项研究中，有 23 项使用了定量模型，但现有证据不足以推断价格影响，特别是考虑到系统的破坏性。利用现有证据确定了一些有希望的提案，这些提案可能需要进行测试：透明度、脱钩、两部分定价、公共研究、孤儿药改革和公共临床试验。
研究结果表明，尽管迫切需要寻找可持续药品价格的提案，但需要仔细和结构化地测试提案，因为这对药物治疗的可及性可能会产生重大影响。
