European regulatory strategy for supporting childhood cancer therapy developments. (December 2022)

• Record Type: Journal Article
• Authors: Karres, Dominik; Lesa, Giovanni; Ligas, Franca; Benchetrit, Sylvie; Galluzzo, Sara; Van Malderen, Karen; Sterba, Jaroslav; van Dartel, Maaike; Renard, Marleen; Sisovsky, Peter; Wang, Siri; Norga, Koen
• Is Part Of: European journal of cancer. Volume 177(2022)
• Page Start: 25
• Page End: 29
• Publication Date: 2022-12
• Subjects: European Paediatric regulation -- Paediatric oncology drug development -- Cancer therapeutics; Cancer -- Periodicals; Neoplasms -- Periodicals; Cancer -- Périodiques; Cancer; Tumors; Electronic journals; Periodicals; Electronic journals; 616.994
• DOI: 10.1016/j.ejca.2022.09.025
• Languages: English
• ISSNs: 0959-8049

Abstract

Abstract: Introduction: 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. Objective: 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. Conclusion: 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. Highlights: Regulatory decision-making in the context of product development life cycle. PIP as a plan, allowing for evidence generation, able to be modified. Fostering collaborative development environment, not competitive. Need to identify priorities based on science. Collaboration among all stakeholders key, as exemplified by ACCELERATE.