Pearson Andrew D J, Weiner Susan L, Adamson Peter C, Karres Dominik, Reaman Gregory, Rousseau Raphaël, Blanc Patricia, Norga Koen, Skolnik Jeffrey, Kearns Pam, Scobie Nicole, Barry Elly, Marshall Lynley V, Knox Leona, Caron Hubert, Wariabharaj Darshan, Pappo Alberto, DuBois Steven G, Gore Lia, Kieran Mark, Weigel Brenda, Fox Elizabeth, Nysom Karsten, de Rojas Teresa, Vassal Gilles
ACCELERATE, Europe.
Children's Cancer Cause, USA.
Eur J Cancer. 2022 May;166:145-164. doi: 10.1016/j.ejca.2022.01.033. Epub 2022 Mar 12.
Rapid evaluation and subsequent regulatory approval of new drugs are critical to improving survival and reducing long-term side-effects for children and adolescents with cancer. The international multi-stakeholder organisation ACCELERATE was created to advance the timely investigation of new anti-cancer drugs. ACCELERATE has enhanced communication and understanding between academia, industry, patient advocates and regulators. It has promoted a mechanism-of-action driven drug development approach and developed Paediatric Strategy Forums. These initiatives have facilitated prioritisation of medicinal products and a focused and sequential strategy for drug development where there are multiple potential agents. ACCELERATE has championed the early assessment of promising drugs in adolescents through their inclusion in adult early phase trials. ACCELERATE has strongly supported alignment between the European Medicines Agency and the US Food and Drug Administration and identification of unmet medical needs through multi-stakeholder collaboration. Early engagement between all stakeholders in the development of new drugs is critical. Innovative clinical trial designs are required, necessitating early discussion with sponsors and regulators. Amplifying the patient advocate voice through inclusion across the drug development continuum will lead to better, patient-centric trials. By these means, children and adolescents with cancer can maximally and rapidly benefit from innovative products to improve outcomes and reduce burdensome sequelae.
快速评估新药并随后获得监管批准对于提高癌症儿童和青少年的生存率以及减少长期副作用至关重要。国际多方利益相关者组织ACCELERATE的成立是为了推动对新型抗癌药物的及时研究。ACCELERATE加强了学术界、产业界、患者权益倡导者和监管机构之间的沟通与理解。它推动了一种基于作用机制的药物开发方法，并建立了儿科战略论坛。这些举措促进了药品的优先排序以及针对多种潜在药物的集中且有序的药物开发策略。ACCELERATE通过将有前景的药物纳入成人早期试验，倡导对青少年进行早期评估。ACCELERATE大力支持欧洲药品管理局和美国食品药品监督管理局之间的协调一致，并通过多方利益相关者合作确定未满足的医疗需求。新药开发过程中所有利益相关者的早期参与至关重要。需要创新的临床试验设计，这就需要尽早与申办者和监管机构进行讨论。通过在药物开发的整个过程中纳入患者权益倡导者的声音来扩大其影响力，将带来更好的、以患者为中心的试验。通过这些方式，癌症儿童和青少年能够最大程度地迅速受益于创新产品，以改善治疗效果并减轻繁重的后遗症。
