美国的癌症药物价格：疗效、创新、临床试验证据和流行病学。
Cancer Drug Prices in the United States: Efficacy, Innovation, Clinical Trial Evidence, and Epidemiology.
机构信息
Department of Medical Oncology, National Center for Tumor Diseases, Heidelberg University Hospital, Heidelberg, Baden-Wuerttemberg, Germany; Schumpeter School of Business and Economics, University of Wuppertal, Wuppertal, North Rhine-Westphalia, Germany.
Schumpeter School of Business and Economics, University of Wuppertal, Wuppertal, North Rhine-Westphalia, Germany; Department of Personalized Oncology, University Hospital Mannheim, Heidelberg University, Mannheim, Baden-Wuerttemberg, Germany; Division of Personalized Medical Oncology, German Cancer Research Center (DKFZ), Heidelberg University, Heidelberg, Baden-Wuerttemberg, Germany; DKFZ-Hector Cancer Institute, University Medical Center Mannheim, Mannheim, Baden-Wuerttemberg, Germany.
出版信息
Value Health. 2023 Nov;26(11):1590-1600. doi: 10.1016/j.jval.2023.06.020. Epub 2023 Jul 28.
OBJECTIVES
Rising cancer drug prices challenge patients and healthcare systems. Although prices are routinely assigned to original drug indications receiving US Food and Drug Administration (FDA) approval, the pricing of supplemental indication approvals remains uncertain. This study identifies and quantifies factors associated with cancer drug prices, distinctly analyzing original and supplemental indications.
METHODS
Clinical trial evidence and epidemiologic data supporting new indications' FDA approval (2003-2022) were collected from the Drugs@FDA database, ClinicalTrials.gov, and Global Burden of Disease study. Indication-specific monthly treatment costs were calculated for Medicare patients. The association between log-prices and collected variables were assessed in regression analyses.
RESULTS
We identified 145 drugs approved across 373 cancer indications. Drugs were priced at $24 444 per month on average (median = $16 013). For original indications, prices weakly correlated to improvements in overall survival (β = 0.28, P = .037) and progression-free survival (β = 0.16, P = .001). Original indications' prices were as follows: (1) negatively associated with disease incidence (β = -0.21, P < .001) and prevalence; (2) positively correlated with first-in-class drugs (26%, P = .057), gene and cell therapies (176%, P < .001), hematologic cancers (62%, P < .001), and severe diseases with substantial unmet needs (6% per disability-adjusted life-year, P < .001); and (3) negatively correlated to indications with randomized-controlled phase 3 trials. Prices were poorly associated with supplemental indications' efficacy, clinical evidence, and epidemiology.
CONCLUSIONS
Cancer drug prices are set based on the original indication's characteristics, thereby omitting the value of supplemental indications. Indication-specific pricing, coverage, and reimbursement policies considering each indication's safety, efficacy, innovativeness, and unmet needs are necessary to align a drug's value and price.
目的
癌症药物价格的上涨给患者和医疗体系带来了挑战。尽管药物的价格通常根据获得美国食品和药物管理局（FDA）批准的原始药物适应证来确定，但补充适应证批准的定价仍不确定。本研究旨在明确并量化与癌症药物价格相关的因素，分别对原始适应证和补充适应证进行分析。
方法
从 Drugs@FDA 数据库、ClinicalTrials.gov 和全球疾病负担研究中收集了支持新适应证 FDA 批准的临床试验证据和流行病学数据（2003-2022 年）。为医疗保险患者计算了适应证特异性的每月治疗费用。采用回归分析评估了对数价格与收集变量之间的关系。
结果
我们共确定了 373 种癌症适应证中 145 种获批的药物。这些药物的平均月价格为 24444 美元（中位数为 16013 美元）。对于原始适应证，价格与总生存期（β=0.28，P=0.037）和无进展生存期（β=0.16，P=0.001）的改善呈弱相关。原始适应证的价格具有以下特征：（1）与疾病发病率（β=-0.21，P<0.001）和患病率呈负相关；（2）与首创药物（26%，P=0.057）、基因和细胞疗法（176%，P<0.001）、血液系统癌症（62%，P<0.001）和严重疾病且存在大量未满足需求（每残疾调整生命年增加 6%，P<0.001）呈正相关；（3）与具有随机对照 3 期试验的适应证呈负相关。价格与补充适应证的疗效、临床证据和流行病学之间的相关性较差。
结论
癌症药物的定价基于原始适应证的特征，从而忽略了补充适应证的价值。为了使药物的价值与其价格相匹配，有必要制定针对每个适应证的安全性、疗效、创新性和未满足需求的适应证特异性定价、覆盖范围和报销政策。
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