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2008年至2022年美国癌症新药上市价格、临床疗效及证据确定性分析。

An analysis of US net cancer drug launch prices and clinical efficacy and certainty of evidence from 2008 to 2022.

作者信息

Abuloha Sumaya, Harvey Benjamin P, Niu Shu, Alshehri Alaa, Miri Melissa, Clifford Katherine, Chambers James, Svensson Mikael

机构信息

Department of Pharmaceutical Outcomes and Policy, College of Pharmacy, University of Florida, Gainesville, Fl 32611, United States.

Department of Clinical Pharmacy and Pharmacy Practice, College of Pharmacy, Yarmouk University, Irbid, 21163, Jordan.

出版信息

Health Aff Sch. 2025 Mar 13;3(4):qxaf051. doi: 10.1093/haschl/qxaf051. eCollection 2025 Apr.

Abstract

Over the last 15 years, cancer drug prices have increased substantially in the United States (US), with question marks on whether this can be justified based on improved patient outcomes. This study aimed to analyze the relationship between US cancer drug net launch prices and clinical efficacy and certainty of evidence. FDA-approved cancer drug indications from 2008 to 2022 were extracted and matched with net launch price data, 5 distinct measures of clinical efficacy, and measures on the certainty of evidence around the clinical efficacy. Descriptive statistics and linear regression models were used to assess if higher net launch prices were associated with better patient outcomes. Cancer drug launch prices (net per 1 year/course of treatment) have increased from around $100 000 in 2008 to $200 000 in 2022. The results did not support the idea that drugs with higher net launch prices had more impressive clinical efficacy or more robust evidence around the clinical efficacy. US cancer launch prices do not seem to be value-based, which may imply distorted incentives for the allocation of research and development investments.

摘要

在过去15年里,美国的抗癌药物价格大幅上涨,而基于改善患者治疗效果这一点,这些价格上涨是否合理存疑。本研究旨在分析美国抗癌药物的净上市价格与临床疗效以及证据确定性之间的关系。提取了2008年至2022年美国食品药品监督管理局(FDA)批准的抗癌药物适应症,并将其与净上市价格数据、5种不同的临床疗效衡量指标以及围绕临床疗效的证据确定性衡量指标进行匹配。使用描述性统计和线性回归模型来评估更高的净上市价格是否与更好的患者治疗效果相关。抗癌药物的上市价格(每1年/疗程的净额)已从2008年的约10万美元增至2022年的20万美元。研究结果并不支持净上市价格较高的药物具有更显著的临床疗效或围绕临床疗效有更确凿证据这一观点。美国抗癌药物的上市价格似乎并非基于价值,这可能意味着研发投资分配的激励机制存在扭曲。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/80f4/11970244/f4da8a6508a4/qxaf051f1.jpg

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