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.

Agence Nationale de Sécurité Du Médicament et des Produits de Santé Paris France; Paediatric Committee of the European Medicines Agency Amsterdam Netherlands

Antwerp University Hospital Paediatric Committee of the European Medicines Agency Federal Agency for Medicines and Health Products Brussels Belgium

College Ter Beoordeling van Geneesmiddelen Utrecht Netherlands; Paediatric Committee of the European Medicines Agency Amsterdam Netherlands

Department of Pediatric Oncology University Hospital Brno And Faculty of Medicine Masaryk University International Clinical Research Center St Anne's University Hospital Brno Czech Republic; Paediatric Committee of the European Medicines Agency Amsterdam Netherlands

Federal Agency for Medicines and Health Products Brussels Belgium; Paediatric Committee of the European Medicines Agency Amsterdam Netherlands

Italian Medicines Agency Rome Italy; Scientific Advice Working Party and Paediatric Committee of the European Medicines Agency Amsterdam Netherlands

Norwegian Medicines Agency Oslo Norway; Paediatric Committee of the European Medicines Agency Amsterdam Netherlands

Paediatric Medicines Office Scientific Evidence Generation Department Human Medicines Division European Medicines Agency Amsterdam Netherlands

State Institute for Drug Control Bratislava Slovakia; Paediatric Committee of the European Medicines Agency Amsterdam Netherlands

University Hospitals Leuven Leuven Belgium; Federal Agency for Medicines and Health Products Brussels Belgium; Paediatric Committee of the European Medicines Agency Amsterdam Netherlands

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