Paediatric Medicines Office, Scientific Evidence Generation Department, Human Medicines Division, European Medicines Agency (EMA), Amsterdam, Netherlands.
Paediatric Medicines Office, Scientific Evidence Generation Department, Human Medicines Division, European Medicines Agency (EMA), Amsterdam, Netherlands.
Eur J Cancer. 2022 Dec;177:25-29. doi: 10.1016/j.ejca.2022.09.025. Epub 2022 Oct 6.
Regulatory decisions on paediatric investigation plans (PIPs) aim at making effective and safe medicines timely available for children with high unmet medical need. At the same time, scientific knowledge progresses continuously leading frequently to the identification of new molecular targets in the therapeutic area of oncology. This, together with further efforts to optimise next generation medicines, results in novel innovative products in development pipelines. In the context of global regulatory development requirements for these growing pipelines of innovative products (e.g. US RACE for children Act), it is an increasing challenge to complete development efforts in paediatric oncology, a therapeutic area of rare and life-threatening diseases with high unmet needs.
Regulators recognise feasibility challenges of the regulatory obligations in this context. Here, we explain the EU regulatory decision making strategy applied to paediatric oncology, which aims fostering evidence generation to support developments based on needs and robust science. Because there is a plethora of products under development within given classes of or within cancer types, priorities need to be identified and updated as evidence evolves. This also includes identifying the need for third or fourth generation products to secure focused and accelerated drug development.
An agreed PIP, as a plan, is a living document which can be modified in light of new evidence. For this to be successful, input from the various relevant stakeholders, i.e. patients/parents, clinicians and investigators is required. To efficiently obtain this input, the EMA is co-organising with ACCELERATE oncology stakeholder engagement platform meetings.
儿科研究计划（PIP）的监管决策旨在为高未满足医疗需求的儿童及时提供有效和安全的药物。与此同时，科学知识不断进步，经常导致肿瘤治疗领域新的分子靶点的确定。这一点，再加上进一步努力优化下一代药物，导致开发管道中出现新的创新产品。在这些不断增长的创新产品（例如美国儿童 RACE 法案）的全球监管发展要求的背景下，完成儿科肿瘤学的开发工作是一个越来越大的挑战，儿科肿瘤学是一个罕见和危及生命的疾病治疗领域，有很高的未满足的需求。
监管机构认识到在这种情况下履行监管义务的可行性挑战。在这里，我们解释了欧盟应用于儿科肿瘤学的监管决策制定策略，该策略旨在促进证据生成，以支持基于需求和稳健科学的开发。由于在给定的药物类别或癌症类型内有大量产品正在开发中，因此需要确定和更新优先级，因为证据在不断发展。这还包括确定需要第三代或第四代产品，以确保有针对性和加速药物开发。
作为计划的商定 PIP 是一份活文件，可以根据新证据进行修改。为了使这一点成功，需要来自各个相关利益相关者的投入，即患者/父母、临床医生和研究人员。为了有效地获得这些投入，EMA 正在与 ACCELERATE 肿瘤学利益相关者参与平台会议共同组织。
