癌症药物溢价——在美国、德国和瑞士，基于疗效和流行病学终点解释癌症药物与非癌症药物价格差异的横断面研究
The cancer premium - explaining differences in prices for cancer vs non-cancer drugs with efficacy and epidemiological endpoints in the US, Germany, and Switzerland: a cross sectional study.
作者信息
Serra-Burriel Miquel, Perényi Gellért, Laube Yannic, Mitchell Aaron P, Vokinger Kerstin N
机构信息
Institute of Law, University of Zurich, Switzerland.
Epidemiology, Biostatistics and Prevention Institute, University of Zurich, Zurich, Switzerland.
出版信息
EClinicalMedicine. 2023 Jul 13;61:102087. doi: 10.1016/j.eclinm.2023.102087. eCollection 2023 Jul.
BACKGROUND
High treatment prices of new cancer drugs are a global public health challenge to patients and healthcare systems. Policymakers in the US and Europe are debating reforms to drug pricing. The objective of this study was to assess whether drug efficacy or epidemiological characteristics (prevalence, incidence, mortality) explain the gap in treatment prices between cancer and non-cancer drugs in the US, Germany, and Switzerland.
METHODS
This cross-sectional study identified all new drugs approved in the US, Germany, and Switzerland between 2011 and 2020. Drug efficacy was extracted from pivotal trials, drug prices from public and commercial databases, and epidemiological characteristics from the Global Burden of Disease (GBD) 2019 study. We used regression models to explain drug prices with drug efficacy and epidemiological characteristics (prevalence, incidence, mortality).
FINDINGS
The cohort included 181 drugs, including 68 (37.5%) drugs approved for treatment of cancer. A significant negative correlation was found between incidence/prevalence and treatment prices, and a significant positive correlation was observed between mortality and treatment prices for both, cancer and non-cancer drugs. A significant association between relative drug efficacy and treatment prices of drugs was observed, however, less pronounced for cancer drugs. Our regression estimates indicated that after adjusting for efficacy and epidemiological characteristics, cancer drugs were on average approximately three times more expensive compared to non-cancer drugs in all three countries, indicating a cancer premium; i.e., treatment prices of cancer drugs were on average USD 74,412 (95% CI [62,810; 86,015]) more expensive in the US compared to non-cancer drugs, USD 37,770 (95% CI [26,175; 49,367]) more expensive in Germany, and USD 32,801 (95% CI [27,048; 38,555]) more expensive in Switzerland. Our model explained 72% of the variance in observed prices (R).
INTERPRETATION
Drug pricing reforms should target the cancer premium to improve access of patients to cancer drugs as well as to achieve equity across the different therapeutic areas and sustainability in the health care systems.
FUNDING
This study was funded by the Swiss National Science Foundation (SNSF, grant number PCEGP1_194607) and the Swiss Cancer Research Foundation (Krebsforschung Schweiz).
背景
新型抗癌药物高昂的治疗价格对患者和医疗保健系统构成了全球性公共卫生挑战。美国和欧洲的政策制定者正在讨论药品定价改革。本研究的目的是评估药物疗效或流行病学特征（患病率、发病率、死亡率）是否能解释美国、德国和瑞士癌症药物与非癌症药物治疗价格的差距。
方法
这项横断面研究确定了2011年至2020年期间在美国、德国和瑞士批准的所有新药。从关键试验中提取药物疗效，从公共和商业数据库中提取药物价格，从《2019年全球疾病负担》（GBD）研究中提取流行病学特征。我们使用回归模型，用药物疗效和流行病学特征（患病率、发病率、死亡率）来解释药物价格。
研究结果
该队列包括181种药物，其中68种（37.5%）药物被批准用于癌症治疗。在发病率/患病率与治疗价格之间发现了显著的负相关，在癌症和非癌症药物的死亡率与治疗价格之间均观察到显著的正相关。观察到药物的相对疗效与治疗价格之间存在显著关联，然而，癌症药物的这种关联不太明显。我们的回归估计表明，在调整疗效和流行病学特征后，在所有三个国家，癌症药物的平均价格比非癌症药物高出约三倍，这表明存在癌症溢价；也就是说，在美国，癌症药物的治疗价格平均比非癌症药物高出74412美元（95%置信区间[62810；86015]），在德国高出37770美元（95%置信区间[26175；49367]），在瑞士高出32801美元（95%置信区间[27048；38555]）。我们的模型解释了观察到的价格差异的72%（R）。
解读
药品定价改革应针对癌症溢价，以改善患者获得癌症药物的机会，并在不同治疗领域实现公平以及医疗保健系统的可持续性。
资金来源
本研究由瑞士国家科学基金会（SNSF，资助编号PCEGP1_194607）和瑞士癌症研究基金会（瑞士癌症研究）资助。
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