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预测细胞和基因疗法在法国的潜在影响:预测产品上市情况和接受治疗的患者人数。

Forecasting the potential impact of cell and gene therapies in France: projecting product launches and patients treated.

作者信息

Lee Ming Kei, Seyedmousavi Sama, Auvity Sylvain, Pourroy Bertrand, Elleboode Vincent, Kachaner Isabelle, Jansen Christel, Lilliu Herve

机构信息

Inbeeo, London, United Kingdom.

Université Paris Cité, INSERM, UMRS-1144, Optimisation Thérapeutique en Neuropsychopharmacologie, Paris, France.

出版信息

Front Med (Lausanne). 2024 Feb 19;11:1324602. doi: 10.3389/fmed.2024.1324602. eCollection 2024.

Abstract

OBJECTIVE

To evaluate the potential impact of cell and gene therapies (CGTs) in France by forecasting the number of patients that will be treated with CGTs over the period 2023-2030 by therapeutic area and region.

METHODS

A review of CGTs in clinical development and related disease epidemiology was conducted to forecast the number of CGT launches and patient population between 2023 and 2030. The number of expected launches was identified by filtering the clinical development pipeline with estimated time to launch and probability of success values from Project ALPHA. Disease prevalence and incidence in France were combined with projected adoption rates derived from historical data to forecast the patient population to be treated.

RESULTS

Up to 44 new CGTs are forecasted to launch in France in the period 2023-2030, which translates into more than 69,400 newly treated patients in 2030. Leading indications in terms of newly treated patients per year include cardiovascular disease, hematological cancers and solid tumors with 27,300, 15,200 and 13,000 newly treated patients in 2030, respectively.

DISCUSSION

The forecast suggests that the future landscape of CGTs will undergo a shift, moving from CGTs targeting (ultra) rare diseases to more prevalent diseases. In France, this will likely pose organizational challenges hindering patient access to these transformative therapies. Further research and planning around network organization and patient distribution are needed to assess and improve the readiness of the French healthcare system for ensuring access for this growing number of patients to be treated with CGTs.

摘要

目的

通过按治疗领域和地区预测2023年至2030年期间接受细胞和基因疗法(CGT)治疗的患者数量,评估CGT在法国的潜在影响。

方法

对处于临床开发阶段的CGT和相关疾病流行病学进行综述,以预测2023年至2030年期间CGT的上市数量和患者群体。通过用预计上市时间和来自阿尔法项目的成功概率值筛选临床开发管道,确定预期上市数量。将法国的疾病患病率和发病率与从历史数据得出的预计采用率相结合,以预测接受治疗的患者群体。

结果

预计2023年至2030年期间法国将有多达44种新的CGT上市,这意味着2030年将有超过69400名新接受治疗的患者。就每年新接受治疗的患者数量而言,主要适应症包括心血管疾病、血液系统癌症和实体瘤,2030年分别有27300名、15200名和13000名新接受治疗的患者。

讨论

该预测表明,CGT的未来格局将发生转变,从针对(超)罕见疾病的CGT转向更常见的疾病。在法国,这可能会带来组织方面的挑战,阻碍患者获得这些变革性疗法。需要围绕网络组织和患者分布进行进一步研究和规划,以评估和提高法国医疗保健系统的准备程度,确保越来越多接受CGT治疗的患者能够获得治疗。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2238/10910012/4aabe17256e0/fmed-11-1324602-g0001.jpg

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