2004 年至 2018 年美国和德国新药的供应情况。
Availability of New Medicines in the US and Germany From 2004 to 2018.
机构信息
Faculty of Economics and Business Administration, University of Duisburg-Essen, Essen, Germany.
London School of Economics, London, United Kingdom.
出版信息
JAMA Netw Open. 2022 Aug 1;5(8):e2229231. doi: 10.1001/jamanetworkopen.2022.29231.
IMPORTANCE
Germany's unique approach to coverage determination and pricing has ensured that effective medicines remain on the market, often at prices reduced through negotiation. However, less is known about trade-offs of this approach with regard to initial availability of medicines.
OBJECTIVE
To examine differences in the timing and scope of new medicines available in Germany and the US.
DESIGN, SETTING, AND PARTICIPANTS: This retrospective cohort study analyzed initial availability of new medicines approved by regulatory agencies in Germany and the US between January 1, 2004, and December 31, 2018, and followed up through December 31, 2019. Data analysis was conducted from January 1, 2020, to July 1, 2022. A total of 599 novel approvals were reviewed. Generic, biosimilar, vaccine, and combination medicines were excluded.
EXPOSURES
US Food and Drug Administration approvals were reviewed for therapies categorized as new molecular entities or new active ingredients. German approvals were reviewed from secondary administrative data of authorized medicines that determine availability in Germany, including data presented by the European Medicines Agency.
MAIN OUTCOMES AND MEASURES
Approvals were analyzed to determine the percentage of medicines approved and available in the US, Germany, or both countries and compare the times to reach the market.
RESULTS
Analysis of 599 new medicines demonstrated that fewer were available in Germany compared with the US (80% vs 92% of all potential therapies) and that the median difference in time to market was 4 months (95% CI, -44.40 to 44.76 months). Forty-nine medicines were approved in Germany but not in the US, 75% of which were rejected by the US Food and Drug Administration, were withdrawn, or had US equivalent agents.
CONCLUSIONS AND RELEVANCE
In this cohort study, fewer new medicines were available in Germany compared with the US between 2004 and 2018. In addition, drugs entered the German market later than in the US.
重要性
德国独特的覆盖范围确定和定价方法确保了有效的药物仍在市场上销售，且价格往往通过谈判降低。然而，对于这种方法在药物初始供应方面的权衡，人们了解得较少。
目的
研究德国和美国新上市药物的时间和范围差异。
设计、设置和参与者：本回顾性队列研究分析了 2004 年 1 月 1 日至 2018 年 12 月 31 日期间，监管机构在德国和美国批准的新药物的初始供应情况，并随访至 2019 年 12 月 31 日。数据分析于 2020 年 1 月 1 日至 2022 年 7 月 1 日进行。共审查了 599 种新批准的药物。排除了通用、生物类似物、疫苗和组合药物。
暴露情况
审查了美国食品和药物管理局批准的新分子实体或新活性成分的治疗药物。德国的批准是从授权药物的二级行政数据中审查的，这些数据包括欧洲药品管理局提供的数据，以确定德国的供应情况。
主要结果和措施
分析批准情况以确定在美国、德国或两国都批准和供应的药物的百分比，并比较达到市场的时间。
结果
对 599 种新药物的分析表明，德国的供应情况比美国少（所有潜在疗法中，德国有 80%的药物，而美国有 92%），市场准入时间中位数差异为 4 个月（95%CI，-44.40 至 44.76 个月）。有 49 种药物在德国获得批准但未在美国获得批准，其中 75%的药物被美国食品和药物管理局拒绝、撤回或有美国等效药物。
结论和相关性
在这项队列研究中，2004 年至 2018 年期间，德国新上市药物的数量比美国少。此外，药物进入德国市场的时间晚于美国。
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