Division of Medical Oncology and Hematology, Princess Margaret Hospital, 610 University Ave, Toronto, Ontario M5G 2M9, Canada.
J Clin Oncol. 2011 Jun 20;29(18):2543-9. doi: 10.1200/JCO.2011.35.2393. Epub 2011 May 23.
The relationship between market pricing of new anticancer drugs and the magnitude of clinical benefit caused by them has not been reported.
Randomized clinical trials (RCTs) that evaluated approved new agents for solid tumors by the U.S. Food and Drug administration since the year 2000 were assessed. Hazard ratios (HRs) and 95% CIs were extracted for time-to-event end points described for each RCT. HRs were pooled for three groups: agents directed against a specific molecular target, for which the target population is selected by a biomarker (group A); less specific biologic targeted agents (group B); and chemotherapeutic agents (group C). Monthly market prices of these different drugs were compared.
For overall survival (OS), the pooled HR was 0.69 (95% CI, 0.59 to 0.81) for group A (six drugs, six trials); it was 0.78 (95% CI, 0.74 to 0.83) for group B (seven drugs, 14 trials); and it was 0.84 (95% CI, 0.79 to 0.90) for group C (eight drugs, 12 trials). For progression-free survival (PFS), the pooled HR was 0.42 (95% CI, 0.36 to 0.49) for group A (six drugs, seven trials); it was 0.57 (95% CI, 0.51 to 0.64) for group B (seven drugs, 14 trials); and it was 0.75 (95% CI, 0.66 to 0.85) for group C (six drugs, 10 trials). Tests for heterogeneity between subgroups were highly significant for PFS (P < .001) and OS (P = .02). The median monthly prices for standard doses of drugs were $5375 for group A, $5644 for group B, and $6584 for group C (P = .87).
New agents with specific molecular targets are clinically the most beneficial, but their monthly market prices are not significantly different from those of other anticancer agents.
新抗癌药物的市场定价与其临床获益程度之间的关系尚未有报道。
评估了 2000 年以来美国食品和药物管理局批准的用于实体瘤的新药物的随机临床试验（RCT）。为每个 RCT 描述的时间事件终点提取了风险比（HR）和 95%置信区间（CI）。将 HR 汇总为三组：针对特定分子靶标的药物，其靶人群通过生物标志物选择（A 组）；靶向治疗药物（B 组）；和化疗药物（C 组）。比较了这些不同药物的每月市场价格。
总生存期（OS）方面，A 组（6 种药物，6 项试验）的 HR 为 0.69（95%CI，0.59 至 0.81）；B 组（7 种药物，14 项试验）为 0.78（95%CI，0.74 至 0.83）；C 组（8 种药物，12 项试验）为 0.84（95%CI，0.79 至 0.90）。无进展生存期（PFS）方面，A 组（6 种药物，7 项试验）的 HR 为 0.42（95%CI，0.36 至 0.49）；B 组（7 种药物，14 项试验）为 0.57（95%CI，0.51 至 0.64）；C 组（6 种药物，10 项试验）为 0.75（95%CI，0.66 至 0.85）。亚组间异质性检验对 PFS（P<.001）和 OS（P=.02）均有显著意义。标准剂量药物的中位月价格为 A 组 5375 美元，B 组 5644 美元，C 组 6584 美元（P=.87）。
具有特定分子靶点的新型药物在临床上最有益，但它们的每月市场价格与其他抗癌药物并无显著差异。
