Impact of Early Access Reform on Oncology Innovation in France: Approvals, Patients, and Costs

IF 5.4 2区医学 Q1 IMMUNOLOGY

BioDrugs Pub Date : 2024-04-20 DOI:10.1007/s40259-024-00658-1

Tess Martin, Catherine Rioufol, Bertrand Favier, Nicolas Martelli, Isabelle Madelaine, Christos Chouaid, Isabelle Borget

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引用次数: 0

Abstract

Background

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.

Objective

This study analyzes the characteristics of oncology drug approvals through the EA process and its impact on real-life data for oncology patients.

Methods

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.

Results

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%).

Conclusion

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.

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早期获取改革对法国肿瘤学创新的影响：批准、患者和成本

背景法国于2021年7月对早期用药（EA）程序进行了雄心勃勃的改革，旨在简化程序并加快创新药物的使用。方法回顾了截至2022年12月31日提交给国家卫生管理局（HAS，Haute Autorité de Santé）的有关肿瘤药物的EA需求的数量和特征。此外，还利用法国全国报销数据库（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，以评估改革对适应症和患者数量以及费用的影响。结果在已公布的110项决定中，HAS平均在70天内批准了88项（80%）EA适应症，其中46项（52%）涉及肿瘤（67%涉及实体瘤，33%涉及血液恶性肿瘤）。获批的适应症大多得到了随机 III 期试验（67%）的支持，而被拒绝的 EA 则更多地依赖于非随机试验（57%）。在获批的 EA 中，28% 的主要终点是总生存期，而在被拒绝的 EA 中则没有。在SNDS数据中，每年接受EA药物治疗的癌症患者人数从2019年的3137人增加到2022年的18341人（+ 484%），而适应症数量从12个增加到62个，主要集中在肿瘤（17个）、肺癌（12个）、消化道癌（9个）和乳腺癌（9个）。EA 治疗的报销费用从 4,200 万欧元激增至 5.26 亿欧元（+ 1159%）。然而，研究结果凸显了财政可持续性方面的持续挑战，需要继续评估和调整。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

BioDrugs 医学-免疫学

CiteScore

12.60

自引率

2.90%

发文量

审稿时长

>12 weeks

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