Pharmacy Department, Georges Pompidou European Hospital, AP-HP, 20 Rue Leblanc, 75015, Paris, France.
GRADES, Faculty of Pharmacy, Paris-Saclay University, 17 Av. des Sciences, 91400, Orsay, France.
BioDrugs. 2024 May;38(3):465-475. doi: 10.1007/s40259-024-00658-1. Epub 2024 Apr 20.
An ambitious reform of the early access (EA) process was set up in July 2021 in France, aiming to simplify procedures and accelerate access to innovative drugs.
This study analyzes the characteristics of oncology drug approvals through the EA process and its impact on real-life data for oncology patients.
The number and characteristics of EA demands concerning oncology drugs submitted to the National Health Authority (HAS, Haute Autorité de Santé) were reviewed until 31 December 2022. A longitudinal retrospective study on patients treated with an EA oncology drug between 1 January 2019 and 31 December 2022 was also performed using the French nationwide claims database (Systeme National des Données de Santé [SNDS]) to assess the impact of the reform on the number of indications and patients, and the costs.
Among 110 published decisions, the HAS granted 88 (80%) EA indications within 70 days of assessment on average, including 46 (52%) in oncology (67% in solid tumors and 33% in hematological malignancies). Approved indications were mostly supported by randomized phase III trials (67%), whereas refused EA relied more on non-randomized (57%) trials. Overall survival was the primary endpoint of 28% of EA approvals versus none of denied EAs. In the SNDS data, the annual number of patients with cancer treated with an EA drug increased from 3137 patients in 2019 to 18,341 in 2022 (+ 484%), whereas the number of indications rose from 12 to 62, mainly in oncohematology (n = 17), lung (n = 12), digestive (n = 9) and breast cancer (n = 9). Reimbursement costs for EA treatments surged from €42 to €526 million (+ 1159%).
The French EA reform contributed to enabling rapid access to innovations in a wide range of indications for oncology patients. However, the findings highlight ongoing challenges in financial sustainability, warranting continued evaluation and adjustments.
法国于 2021 年 7 月启动了一项雄心勃勃的早期准入（EA）流程改革，旨在简化程序并加速创新药物的准入。
本研究分析了通过 EA 流程批准的肿瘤药物的特点及其对肿瘤患者真实数据的影响。
回顾性审查了截至 2022 年 12 月 31 日提交给国家卫生管理局（HAS）的肿瘤药物 EA 请求的数量和特征。还使用法国全国索赔数据库（Systeme National des Données de Santé [SNDS]）对 2019 年 1 月 1 日至 2022 年 12 月 31 日期间接受 EA 肿瘤药物治疗的患者进行了一项纵向回顾性研究，以评估改革对适应证数量和患者数量以及成本的影响。
在 110 项已公布的决定中，HAS 平均在评估后 70 天内批准了 88 项（80%）EA 适应证，其中 46 项（52%）在肿瘤学领域（实体肿瘤占 67%，血液恶性肿瘤占 33%）。批准的适应证主要由随机 III 期试验支持（67%），而拒绝的 EA 更多地依赖于非随机试验（57%）。总体生存是 28%的 EA 批准的主要终点，而没有被拒绝的 EA 有此终点。在 SNDS 数据中，2019 年接受 EA 药物治疗的癌症患者人数每年增加 3137 人，到 2022 年增加到 18341 人（增加了 484%），而适应证数量从 12 个增加到 62 个，主要集中在肿瘤血液学（n=17）、肺癌（n=12）、消化系统（n=9）和乳腺癌（n=9）。EA 治疗的报销费用从 4200 万欧元飙升至 5.26 亿欧元（增加了 1159%）。
法国的 EA 改革有助于使肿瘤患者能够快速获得广泛适应证的创新药物。然而，研究结果强调了在财务可持续性方面仍存在挑战，需要持续评估和调整。
