JCO:神药虽好,是否划算?抗癌药的经济学分析
  • 免疫检查点抑制剂(I/O)未上市前,晚期恶性黑色素瘤的5年生存率为17%;I/O的出现大幅度提高了生存率,但是这类药物实在是太贵;
  • 本文纳入了多项三期临床试验的临床资料、医疗开支、生活质量等信息,对比了两种PD-1抗体单独使用、CTLA-4抗体单独使用、nivolumab和CTLA-4抗体联合使用的性价比;
  • 研究结果显示:一线单独使用PD-1抗体,二线单独使用CTLA-4抗体,是目前性价比最高、最值得推荐的治疗方案。
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Cost-Effectiveness of Immune Checkpoint Inhibition in BRAF Wild-Type Advanced Melanoma

免疫检查点抑制剂用于BRAF基因野生型晚期恶性黑色素瘤的成本效益分析

10.1200/JCO.2016.69.6336

2017-02-21, Report

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Purpose Patients who are diagnosed with stage IV metastatic melanoma have an estimated 5-year relative survival rate of only 17%. Randomized controlled trials of recent US Food and Drug Administration-approved immune checkpoint inhibitors-pembrolizumab (PEM), nivolumab (NIVO), and ipilumumab (IPI)-demonstrate improved patient outcomes, but the optimal treatment sequence in patients with BRAF wild-type metastatic melanoma remains unclear. To inform policy makers about the value of these treatments, we developed a Markov model to compare the cost-effectiveness of different strategies for sequencing novel agents for the treatment of advanced melanoma. Materials and Methods We developed Markov models by using a US-payer perspective and lifetime horizon to estimate costs (2016 US$) and quality-adjusted life years (QALYs) for treatment sequences with first-line NIVO, IPI, NIVO + IPI, PEM every 2 weeks, and PEM every 3 weeks. Health states were defined for initial treatment, first and second progression, and death. Rates for drug discontinuation, frequency of adverse events, disease progression, and death obtained from randomized phase III trials were used to determine the likelihood of transition between states. Deterministic and probabilistic sensitivity analyses were conducted to evaluate model uncertainty. Results PEM every 3 weeks followed by second-line IPI was both more effective and less costly than dacarbazine followed by IPI then NIVO, or IPI followed by NIVO. Compared with the first-line dacarbazine treatment strategy, NIVO followed by IPI produced an incremental cost effectiveness ratio of $90,871/QALY, and first-line NIVO + IPI followed by carboplatin plus paclitaxel chemotherapy produced an incremental cost effectiveness ratio of $198,867/QALY. Conclusion For patients with treatment-naive BRAF wild-type advanced melanoma, first-line PEM every 3 weeks followed by second-line IPI or first-line NIVO followed by second-line IPI are the most cost-effective, immune-based treatment strategies for metastatic melanoma.

First Authors:
Christine G Kohn

Correspondence Authors:
Christine G Kohn

All Authors:
Christine G Kohn,Simon B Zeichner,Qiushi Chen,Alberto J Montero,Daniel A Goldstein,Christopher R Flowers

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