Willis Michael, Nilsson Andreas, Thet Lwin Zin Min, Brådvik Gunnar, Prelaj Arsela
The Swedish Institute for Health Economics, Lund, Sweden (Willis, Nilsson, Thet Lwin, Brådvik).
Medical Oncology Department 1, Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy (Prelaj).
J Manag Care Spec Pharm. 2025 Jan;31(1):69-81. doi: 10.18553/jmcp.2025.31.1.69.
Non-small cell lung cancer (NSCLC) presents a formidable global health challenge owing to significant morbidity, high mortality rates, and substantial economic burden. Recent advances in targeted therapies and immunotherapies have transformed NSCLC treatment, but efficacy varies across patients. Tailoring treatment to patients can improve outcomes and potentially improve cost-effectiveness (ie, value for money) as well. For NSCLC, cost-effectiveness must often be estimated using economic modeling, and estimates are only as good as the models. Existing cost-effectiveness models are not necessarily suitable for evaluating personalized medicines.
To identify and assess cost-effectiveness models of NSCLC.
We searched for studies indexed in PubMed and Embase from 2012 to October 2023 that described cost-effectiveness models of NSCLC. Study details were extracted, summarized, and evaluated for adherence to the Consolidated Health Economic Evaluation Reporting Standards.
We identified 237 unique models, 40% of which were published in 2022 or 2023. Despite cross-model heterogeneity, most models used the same 3 health states (progression-free survival, progressive disease, and death) combined with time-to-event equations that characterize risks. Thirty models included a diagnostic component, most of which considered guiding treatment selection using biomarkers. Adherence to the overall Consolidated Health Economic Evaluation Reporting Standards checklist was generally incomplete, and adherence to a subset of model-related questions even more so.
The large number of models that were found, almost half of which were published since 2022, underscores the importance of cost-effectiveness analysis in NSCLC. Variable adherence to best practices suggests opportunities for improvement, however, and making high-quality, open-source models available to researchers may be valuable.
非小细胞肺癌(NSCLC)由于发病率高、死亡率高和经济负担沉重,给全球健康带来了巨大挑战。靶向治疗和免疫治疗的最新进展改变了NSCLC的治疗方式,但疗效因患者而异。根据患者情况量身定制治疗方案可以改善治疗效果,并有可能提高成本效益(即性价比)。对于NSCLC,通常必须使用经济模型来估计成本效益,而估计结果取决于模型的质量。现有的成本效益模型不一定适用于评估个性化药物。
识别和评估NSCLC的成本效益模型。
我们检索了2012年至2023年10月在PubMed和Embase上索引的描述NSCLC成本效益模型的研究。提取、总结研究细节,并评估其是否符合《卫生经济评估报告标准合并版》。
我们识别出237个独特的模型,其中40%在2022年或2023年发表。尽管模型之间存在异质性,但大多数模型使用相同的3种健康状态(无进展生存期、疾病进展和死亡),并结合表征风险的事件发生时间方程。30个模型包含诊断部分,其中大多数考虑使用生物标志物指导治疗选择。对《卫生经济评估报告标准合并版》总体清单的遵守情况通常不完整,对与模型相关问题子集的遵守情况更是如此。
发现的大量模型(其中近一半自2022年以来发表)凸显了NSCLC成本效益分析的重要性。然而,对最佳实践的遵守情况参差不齐表明仍有改进空间,向研究人员提供高质量的开源模型可能很有价值。