Simons Martijn J H G, Uyl-de Groot Carin A, Retèl Valesca P, Mankor Joanne M, Ramaekers Bram L T, Joore Manuela A, van Harten Wim H
Department of Clinical Epidemiology and Medical Technology Assessment, Maastricht University Medical Centre, Maastricht, The Netherlands; Care And Public Health Research Institute, Maastricht University, Maastricht, The Netherlands.
Erasmus School of Health Policy and Management/Institute for Medical Technology Assessment, Erasmus University Rotterdam, Rotterdam, The Netherlands.
Value Health. 2023 Jan;26(1):71-80. doi: 10.1016/j.jval.2022.07.006. Epub 2022 Aug 13.
This study aimed to investigate the cost-effectiveness, budget impact (BI), and impact of uncertainty of future developments concerning whole-genome sequencing (WGS) as a clinical diagnostic test compared with standard of care (SoC) in patients with locally advanced and metastatic non-small cell lung cancer.
A total of 3 likely scenarios to take place within 5 years (according to experts) were simulated using a previously developed, peer reviewed, and published decision model. The scenarios concerned "WGS results used for treatment selection" (scenario 1), "WGS-based biomarker for immunotherapy" (scenario 2), and "off-label drug approval for WGS results" (scenario 3). Two diagnostic strategies of the original model, "SoC" and "WGS as a diagnostic test" (base model), were used to compare our scenarios with. Outcomes were reported for the base model, all scenarios separately, combined (combined unweighted), and weighted by likelihood (combined weighted). Cost-effectiveness, BI, and value of information analyses were performed for WGS compared with SoC.
Total costs and quality-adjusted life-years for SoC in metastatic non-small cell lung cancer were €149 698 and 1.235. Incremental outcomes of WGS were €1529/0.002(base model), -€222/0.020(scenario 1), -€2576/0.023(scenario 2), €388/0.024(scenario 3), -€5041/0.060(combined unweighted), and -€1715/0.029(combined weighted). The annual BI for adopting WGS for this population in The Netherlands ranged between €682 million (combined unweighted) and €714 million (base model). The consequences of uncertainty amounted to €3.4 million for all scenarios (combined weighted) and to €699 000 for the diagnostic yield of WGS alone (combined weighted).
Our findings suggest that it is likely for WGS to become cost-effective within the near future if it identifies more patients with actionable targets and show the impact of uncertainty regarding its diagnostic yield. Modeling future scenarios can be useful to consider early adoption of WGS while timely anticipating on unforeseen developments before final conclusions are reached.
本研究旨在调查全基因组测序（WGS）作为局部晚期和转移性非小细胞肺癌患者临床诊断检测方法与标准治疗（SoC）相比的成本效益、预算影响（BI）以及未来发展不确定性的影响。
使用先前开发、同行评审并发表的决策模型模拟了5年内可能发生的3种情况（根据专家意见）。这些情况涉及“WGS结果用于治疗选择”（情况1）、“基于WGS的免疫治疗生物标志物”（情况2）以及“WGS结果的超说明书用药批准”（情况3）。使用原始模型的两种诊断策略“标准治疗”和“WGS作为诊断检测”（基础模型）进行比较。分别报告基础模型、所有情况、合并情况（合并未加权）以及按可能性加权（合并加权）的结果。对WGS与标准治疗进行成本效益、预算影响和信息价值分析。
转移性非小细胞肺癌标准治疗的总成本和质量调整生命年分别为149698欧元和1.235。WGS的增量结果为1529欧元/0.002（基础模型）、-222欧元/0.020（情况1）、-2576欧元/0.023（情况2）、388欧元/0.024（情况3）、-5041欧元/0.060（合并未加权）以及-1715欧元/0.029（合并加权）。在荷兰，采用WGS对该人群的年度预算影响在6.82亿欧元（合并未加权）至7.14亿欧元（基础模型）之间。所有情况的不确定性后果为340万欧元（合并加权），仅WGS诊断率的不确定性后果为69.9万欧元（合并加权）。
我们的研究结果表明，如果WGS能识别出更多具有可操作靶点的患者，并显示其诊断率的不确定性影响，那么在不久的将来它可能具有成本效益。对未来情况进行建模有助于考虑尽早采用WGS，同时在得出最终结论之前及时预测意外发展。
