为了加速新药的获取，法国实施了一项称为临时使用授权 (ATU) 的早期获取计划，直至 2021 年。我们分析了根据 ATU 提交的免疫检查点抑制剂 (ICIs) 临床报告，并评估了其与批准 ICI 的临床相关性。我们纳入了由法国药物安全机构 Agence nationale de sécurité du médicament et des produits de santé（ANSM；法国国家药品和保健品安全局）授予的 ATU 的所有 ICI，适用于 1 月 1 日期间的癌症患者2010 年和 2020 年 12 月 31 日。我们从制药公司提交的 ATU 报告中收集了患者的临床和药物警戒数据，并将这些数据与相应的关键临床试验 (CT) 的数据进行比较。ATU 为 11 种癌症中 7 种 ICI 的 5807 名患者提供了早期治疗机会迹象，其中 1 个没有相应的 ATU 报告。在 10 份可用的 ATU 报告中，只有 1 份包含所有必需的数据。 40.5% 的患者可以获得临床随访表。数据报告的差异使我们无法比较 CT 和 ATU 报告之间的严重不良事件。临床医生和制药公司对于 ICI 是否导致 163 例永久性治疗中断常常存在分歧，Cohen 偏倚和患病率调整后 κ = 0.52，95% CI 0.33-0.68。尽管 93 例非进展性肿瘤死亡的一致性几乎是完美的（κ = 0.88，95% CI 0.66-0.97），但 29% 的 ATU 患者死亡仍然无法解释，并且据报道与制药公司的治疗无关。法国 ATU 促进了早期获得新药ICI 适用于许多癌症患者。然而，数据消耗阻碍了有效的现实世界监控。版权所有 © 2024 作者。由爱思唯尔有限公司出版。保留所有权利。

To accelerate access to new drugs, France operated an early access program known as Temporary Authorizations for Use (ATUs) until 2021. We analyzed clinical reports submitted under ATUs for immune checkpoint inhibitors (ICIs) and assessed their clinical relevance regarding the approval of ICIs in oncology.We included all ICIs granted an ATU by the French drug safety agency, Agence nationale de sécurité du médicament et des produits de santé (ANSM; French National Agency for the Safety of Medicines and Health Products), for patients with cancer between 1 January 2010 and 31 December 2020. We collected patients' clinical and pharmacovigilance data from ATU reports submitted by pharmaceutical companies and compared these data with those from corresponding pivotal clinical trials (CTs).The ATUs provided early access to 5807 patients with seven ICIs across 11 cancer indications, 1 of which had no corresponding ATU report. Of the 10 available ATU reports, only 1 included all required data. Clinical follow-up forms were available for 40.5% of patients. Differences in data reporting prevented us from comparing serious adverse events between the CTs and ATU reports. Clinicians and pharmaceutical companies often disagreed on whether ICIs caused 163 permanent treatment discontinuations, with Cohen's bias- and prevalence-adjusted κ = 0.52, 95% CI 0.33-0.68. Although agreement was almost perfect for 93 nonprogressive tumor deaths (κ = 0.88, 95% CI 0.66-0.97), 29% of ATU patient deaths remained unexplained and were reported as unrelated to treatment by the pharmaceutical companies.French ATUs facilitated early access to new ICIs for many patients with cancer. However, data attrition hindered effective real-world monitoring.Copyright © 2024 The Author(s). Published by Elsevier Ltd.. All rights reserved.