Akinsoji Toluwase, Dragojlovic Nick, Darviot Cécile, Meunier Michel, Harrison Mark, Lynd Larry D
Faculty of Pharmaceutical Sciences, University of British Columbia, 2405 Wesbrook Mall, Vancouver, BC, V6 T 1Z3, Canada.
Department of Engineering Physics, Polytechnique Montréal, Montreal, QC, Canada.
Pharmacoeconomics. 2025 Jun 20. doi: 10.1007/s40273-025-01512-0.
Companion diagnostics (CDx) are critical to precision medicine. Developing and commercializing new CDx faces regulatory and economic challenges. This study aims to illustrate the utility of an early health technology assessment in quantifying the unmet clinical need and commercial opportunity created by the limited accuracy of existing programmed cell death ligand 1 CDx.
The study uses an early health technology assessment and market sizing to assess the potential value of a novel programmed cell death ligand 1 CDx for non-small cell lung cancer (NSCLC). Decision tree-based cost-effectiveness models were used to evaluate clinical and economic outcomes for improved programmed cell death ligand 1 testing in atezolizumab-treated patients with stage II-IIIA and metastatic NSCLC from a US payer perspective in 2023 US Dollars. Three strategies were examined: standard care, new CDx for cytology specimens only, and new CDx for all patients. Commercial opportunities from the perspectives of diagnostics and pharmaceutical manufacturers were assessed using headroom and threshold analyses.
Headroom analyses indicated that a new CDx is not cost effective for metastatic NSCLC but holds significant value for stage II-IIIA NSCLC. Assuming perfect sensitivity and specificity, the incremental cost-effectiveness ratio for the new CDx in stage II-IIIA NSCLC was $57,650/quality-adjusted life-year and $54,950/quality-adjusted life-year for cytology specimens only and all patients, respectively. A threshold analysis showed that at a $500 price point, the new CDx is cost effective at sensitivity levels of 0.9 for all patients and 0.8 for cytology only. The total addressable US market for the CDx manufacturer was estimated at $2.6 million per year with a $500/test kit price.
A novel, highly accurate CDx for stage II-IIIA NSCLC could provide significant value to patients, payers, and manufacturers.
伴随诊断（CDx）对精准医疗至关重要。开发新型CDx并将其商业化面临监管和经济挑战。本研究旨在阐明早期卫生技术评估在量化现有程序性细胞死亡配体1（PD-L1）CDx准确性有限所造成的未满足临床需求和商业机会方面的作用。
本研究采用早期卫生技术评估和市场规模评估，以评估一种新型非小细胞肺癌（NSCLC）程序性细胞死亡配体1 CDx的潜在价值。基于决策树的成本效益模型用于从美国医保支付方的角度，以2023年美元评估在阿替利珠单抗治疗的II-IIIA期和转移性NSCLC患者中改进程序性细胞死亡配体1检测的临床和经济结果。研究了三种策略：标准治疗、仅用于细胞学标本的新型CDx以及用于所有患者的新型CDx。使用空间分析和阈值分析从诊断和制药制造商的角度评估商业机会。
空间分析表明，新型CDx对转移性NSCLC不具有成本效益，但对II-IIIA期NSCLC具有显著价值。假设具有完美的敏感性和特异性，II-IIIA期NSCLC中新型CDx的增量成本效益比分别为仅用于细胞学标本的患者和所有患者为57,650美元/质量调整生命年和54,950美元/质量调整生命年。阈值分析表明，在500美元的价格点，新型CDx在所有患者的敏感性水平为0.9且仅细胞学标本的敏感性水平为0.8时具有成本效益。估计CDx制造商在美国的总潜在市场为每年260万美元，检测试剂盒价格为500美元/次。
一种用于II-IIIA期NSCLC的新型、高度准确的CDx可为患者、医保支付方和制造商提供显著价值。
