Gustave Roussy, Service de Biostatistique et D'Epidémiologie, Villejuif, France; University Paris-Saclay, Univ. Paris-Sud, UVSQ, CESP, INSERM, Villejuif, France.
Dermatology and CIC, Assistance Publique des Hôpitaux de Paris, INSERM U976, University Paris Diderot-Saint-Louis Hospital, Paris, France.
Eur J Cancer. 2018 Dec;105:33-40. doi: 10.1016/j.ejca.2018.09.026. Epub 2018 Oct 29.
Since 2011, significant progress was observed in metastatic melanoma (MM), with the commercialisation of seven immunotherapies or targeted therapies, which showed significant improvement in survival. In France, in 2004, the cost of MM was estimated at €1634 per patient; this cost has not been re-estimated since. This study provided an update on survival and cost in real-life clinical practice.
Clinical and economic data (treatments, hospitalisations, radiotherapy sessions, visits, imaging and biological exams) were extracted from the prospective MelBase cohort, collecting individual data in 955 patients in 26 hospitals, from diagnosis of metastatic disease until death. Survival was estimated by the Kaplan-Meier method. Costs were calculated from the health insurance perspective using French tariffs. For live patients, survival and costs were extrapolated using a multistate model, describing the 5-year course of the disease according to patient prognostic factors and number of treatment lines.
Since the availability of new drugs, the mean survival time of MM patients has increased to 23.6 months (confidence interval [CI] :21.2;26.6), with 58% of patients receiving a second line of treatment. Mean management costs increased to €269,682 (CI:244,196;304,916) per patient. Drugs accounted for 80% of the total cost.
This study is the first that evaluated the impact of immunotherapies and targeted therapies both on survival and cost in real-life conditions. Alongside the introduction of breakthrough therapies in the first and subsequent lines, MM has been associated with a significant increase in survival but also in costs, raising the question of financial sustainability.
自 2011 年以来，转移性黑色素瘤（MM）取得了显著进展，七种免疫疗法或靶向疗法已商业化，这些疗法显著改善了患者的生存。在法国，2004 年估计每位 MM 患者的费用为 1634 欧元；此后尚未重新估计这一费用。本研究旨在实时临床实践中更新生存和成本情况。
从前瞻性 MelBase 队列中提取临床和经济数据（治疗、住院、放疗次数、就诊、影像学和生物学检查），该队列在 26 家医院共纳入了 955 名患者，从转移性疾病的诊断到死亡，收集每位患者的个体数据。使用 Kaplan-Meier 方法估计生存情况。从医疗保险的角度计算成本，使用法国关税。对于存活患者，使用多状态模型对生存和成本进行外推，根据患者预后因素和治疗线数描述疾病 5 年病程。
自新药物上市以来，MM 患者的平均生存时间延长至 23.6 个月（置信区间[CI]：21.2;26.6），58%的患者接受二线治疗。每位患者的管理费用增加至 269682 欧元（CI：244196;304916）。药物占总费用的 80%。
本研究首次评估了免疫疗法和靶向疗法在实时条件下对生存和成本的影响。随着一线和二线突破性疗法的引入，MM 不仅与生存时间显著延长相关，还与成本显著增加相关，这引发了关于财务可持续性的问题。
