Pearson Andrew D J, Rossig Claudia, Mackall Crystal L, Shah Nirali N, Baruchel André, Daems Sam, Anderson John, Biondi Andrea, Bird Nicholas, Bodmer Nicole, Brivio Erica, Buechner Jochen, Calkoen Friso G, Cooper Todd, de Rojas Teresa, Fox Elizabeth, Gardner Rebecca, Ghorashian Sara, Heenen Delphine, Ifversen Marianne, Jacoby Elad, Juan Manel, Knox Leona, Komanduri Krishna, Larghero Jerome, Locatelli Franco, Ludwinski Donna, Majzner Robbie G, McDonough Joe, Minard-Colin Veronique, Nysom Karsten, Pappo Alberto, Park Julie R, Qasim Waseem, Quintarelli Concetta, Rives Susana, Rouce Rayne H, Scobie Nicole, Seitz Christian, Tasian Sarah K, Weigel Brenda, Weiner Susan, Zwaan C Michel, Vassal Gilles
ACCELERATE, Brussels, Belgium.
University Children's Hospital Muenster, Muenster, Germany.
Lancet Oncol. 2025 Apr;26(4):e214-e224. doi: 10.1016/S1470-2045(24)00736-8.
Realising the potentially substantial benefits of chimeric antigen receptor (CAR) T-cell therapy for children with cancer is hindered by non-scientific barriers that are also relevant for other rare diseases. A solely commercial development model will not deliver optimally due to insufficient return on investment for pharmaceutical companies. Access to therapies is restricted for patients who might benefit and advancing innovation in the academic research setting is difficult. Challenges relating to CAR T-cell therapies in paediatric malignancies and how they might be addressed were discussed in a meeting convened by ACCELERATE-an international multistakeholder organisation aiming to advance the timely investigation of new anticancer drugs. New academic and biopharma hybrid development models could benefit rare populations and coordination of early development can promote synergy and avoid duplicative efforts. Following promising first-in-child trials, new models are needed to support pivotal trials, decentralised manufacturing, registration, and reduced costs. The European Medicines Agency and the US Food and Drug Administration encourage academic development and early discussions. A biotech company funded via a pooled investment vehicle could provide access to safe and effective products for children and adolescents with cancer through registration and reimbursement.
嵌合抗原受体（CAR）T细胞疗法对患癌儿童具有潜在的巨大益处，但实现这一疗法却受到一些非科学障碍的阻碍，这些障碍对其他罕见病也同样存在。由于对制药公司而言投资回报率不足，单纯的商业开发模式无法实现最优效果。可能受益的患者获得治疗的机会受到限制，在学术研究环境中推进创新也很困难。在由ACCELERATE召集的一次会议上，讨论了儿科恶性肿瘤中CAR T细胞疗法相关的挑战以及应对这些挑战的方法。ACCELERATE是一个国际多方利益相关者组织，旨在推动对抗癌新药的及时研究。新的学术与生物制药混合开发模式可能会使罕见病群体受益，早期开发的协调能够促进协同效应并避免重复工作。在开展了前景良好的首例儿童试验之后，需要新的模式来支持关键试验、分散式生产、注册以及降低成本。欧洲药品管理局和美国食品药品监督管理局鼓励学术开发和早期讨论。通过集合投资工具资助的一家生物技术公司可以通过注册和报销，为患有癌症的儿童和青少年提供安全有效的产品。
