Haslam Alyson, Olivier Timothée, Prasad Vinay
Department of Epidemiology and Biostatistics, University of California San Francisco, San Francisco, California, USA.
Department of Oncology, Geneva University Hospital, Geneva, Switzerland.
Int J Cancer. 2025 Jun 15;156(12):2352-2359. doi: 10.1002/ijc.35347. Epub 2025 Jan 30.
In 2018, we estimated that eligibility for and response to immune checkpoint inhibitor (ICI) therapies were 44% and 12%, respectively. Since these estimates were published, there have been additional approvals. We sought to provide updated estimates of the percentage of patients with advanced and/or metastatic cancers in the US who are eligible for and respond to immune checkpoint inhibitors (ICIs). Using a cross-sectional analysis (2011-2023) of US Food and Drug Administration approvals (FDA) and deaths reported by the American Cancer Society, we estimated the percentage of patients in the US with advanced or metastatic cancers who are eligible and respond to ICI therapies and the long-term response of ICI drugs. Eleven ICI drugs have been approved for 20 tumor types in the metastatic setting. The estimated eligibility for ICIs increased from 1.54% in 2011 to 56.55% in 2023. The estimated response to ICIs increased from 0.14% in 2011 to 20.13% in 2023. The tumor types with the highest contribution to response and eligibility estimates in 2023 were non-small-cell lung cancer with PD-L1 expression ≤50% and PD-L1 expression >50%. Sixteen drug approvals had long-term progression-free survival (PFS) data available at 3 years follow-up, and 2 had PFS data at 5 years follow-up. Estimated eligibility and response have increased over time, but many people with advanced or metastatic cancers are currently ineligible for ICIs. Only about one-fifth of the patients will respond. Given the wide range of uses, the cost implications of ICIs globally are large.
2018年，我们估计免疫检查点抑制剂（ICI）疗法的适用率和有效率分别为44%和12%。自这些估计值公布以来，又有更多药物获批。我们试图提供美国晚期和/或转移性癌症患者中适合使用免疫检查点抑制剂（ICI）并对其有反应的患者比例的最新估计值。通过对美国食品药品监督管理局（FDA）的批准情况以及美国癌症协会报告的死亡数据进行横断面分析（2011 - 2023年），我们估计了美国晚期或转移性癌症患者中适合使用ICI疗法并对其有反应的患者比例以及ICI药物的长期疗效。在转移性治疗中，已有11种ICI药物被批准用于20种肿瘤类型。ICI的估计适用率从2011年的1.54%增至2023年的56.55%。ICI的估计有效率从2011年的0.14%增至2023年的20.13%。2023年，对有效率和适用率估计贡献最大的肿瘤类型是PD - L1表达≤50%和PD - L1表达>50%的非小细胞肺癌。16项药物获批有3年随访的长期无进展生存期（PFS）数据，2项有5年随访的PFS数据。随着时间推移，估计的适用率和有效率有所增加，但目前许多晚期或转移性癌症患者仍不适合使用ICI。只有约五分之一的患者会有反应。鉴于ICI的广泛应用，其在全球范围内的成本影响巨大。
