目标:癌症药物价格的上涨对患者和医疗保健系统构成了挑战。尽管价格通常分配给获得美国食品和药物管理局(FDA)批准的原始药物适应症,补充适应症批准的定价仍然不确定。这项研究确定并量化了与癌症药物价格相关的因素,明确分析原始和补充适应症。
方法:支持新适应症的临床试验证据和流行病学数据FDA批准(2003-2022)从药物@FDA数据库收集,ClinicalTrials.gov,和全球疾病负担研究。针对Medicare患者计算了特定指征的每月治疗费用。在回归分析中评估了对数价格和收集的变量之间的关联。
结果:我们在373种癌症适应症中确定了145种批准的药物。药物平均每月定价为24444美元(中位数=16013美元)。对于原始适应症,价格与总生存期(β=0.28,P=0.037)和无进展生存期(β=0.16,P=.001)的改善弱相关。原始适应症价格如下:(1)与疾病发生率(β=-0.21,P<.001)和患病率呈负相关;(2)与一流药物呈正相关(26%,P=.057),基因和细胞疗法(176%,P<.001),血液肿瘤(62%,P<.001),和有大量未满足需求的严重疾病(每残疾调整生命年6%,P<.001);(3)与随机对照3期试验的适应症呈负相关。价格与补充适应症的疗效相关性较差,临床证据,和流行病学。
结论:癌症药物价格是根据原始适应症的特征制定的,从而省略了补充适应症的价值。特定于指示的定价,覆盖范围,以及考虑每个适应症安全性的报销政策,功效,创新,和未满足的需求是必要的,以调整药物的价值和价格。
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.
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.
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.
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.