Jommi Claudio, Bramanti Stefania, Pani Marcello, Ghirardini Alessandro, Santoro Armando
CERGAS (Centre for Research on Health and Social Care Management), SDA Bocconi School of Management), Bocconi University, Milan, Italy.
IRCCS Humanitas Research Hospital- Humanitas Cancer Center, Rozzano, Italy.
Front Pharmacol. 2022 Jun 23;13:915342. doi: 10.3389/fphar.2022.915342. eCollection 2022.
CAR T-cell therapy has emerged as a potentially transformative immunotherapy for certain B-cell malignancies including relapsed/refractory diffuse large B-cell lymphoma (DLBCL). Unhindered and appropriate access for eligible patients is essential to enable optimal outcomes and depends on effective interplay of stakeholders and processes along the patient's therapeutic journey. In Italy, CAR T-cell therapies have been awarded innovation status by the Italian Medicines Agency (AIFA) and were integrated into routine patient care under specific criteria. However, our analysis indicates that fewer than one in five DLBCL patients eligible under the EMA authorization, or around one in three DLBCL patients eligible under the AIFA criteria, received treatment with a licensed CAR T-cell therapy product in 2020. This publication describes key patient access barriers to CAR T-cell therapies in Italy and provides recommendations on potential solutions at the health system level.
嵌合抗原受体（CAR）T细胞疗法已成为治疗某些B细胞恶性肿瘤（包括复发/难治性弥漫性大B细胞淋巴瘤（DLBCL））的一种具有潜在变革性的免疫疗法。让符合条件的患者不受阻碍地获得适当治疗对于实现最佳治疗效果至关重要，这取决于患者治疗过程中利益相关者和流程的有效互动。在意大利，嵌合抗原受体T细胞疗法已被意大利药品管理局（AIFA）授予创新地位，并根据特定标准纳入常规患者护理。然而，我们的分析表明，在2020年，在欧洲药品管理局（EMA）授权标准下符合条件的DLBCL患者中，接受许可的嵌合抗原受体T细胞疗法产品治疗的不到五分之一，在意大利药品管理局标准下符合条件的DLBCL患者中，约三分之一接受了此类治疗。本出版物描述了意大利嵌合抗原受体T细胞疗法在患者获取方面的关键障碍，并就卫生系统层面的潜在解决方案提出了建议。
