By setting mandatory requirements, paediatric regulations implemented in the EU and the US have allowed the development of – and improved access to – high-quality drugs for the paediatric population. It is acknowledged that paediatric development remains a challenge in many circumstances and, ten years after the implementation of the EU Paediatric Regulation, associated requirements are still seen as constraints by industry. Early planning and interactions with regulators on paediatric development have been proven to facilitate, to a certain extent, agreement on a plan and its integration into a global development plan. This article addresses some of the challenges faced by sponsors to integrate the paediatric population in the overall drug development plan, which often aims at being “global”, ie, global from a population point of view (including both adults and children) and also from a geographical perspective (eg, the EU and the US).
Paediatric population; Paediatric Regulation; Paediatric investigation plan (PIP); Paediatric study plan (PSP); Best Pharmaceuticals for Children Act; Paediatric Research Equity Act; Global drug development; Extrapolation; Modelling; Simulation.
Ségolène Saintpierre, Senior Regulatory Scientist, Drugs & Biologics
Ségolène is involved in different regulatory projects, conducting research and scientific writing in the framework of the design and implementation of Regulatory Strategies for the development of drugs and biologics. She also participates in lifecycle management and Marketing Authorization Application activities, including the setting of regulatory strategy, preparation of variation application dossiers, and review of product information.
Delphine Kazancigil, Director, Regulatory Science, Drugs & Biologics
Delphine is an experienced regulatory science professional who has an in-depth knowledge of the European (EU) legislation and regulatory requirements. Delphine assists clients with her scientific, technical and regulatory expertise in the design of products development plans and associated regulatory and registration strategies. In particular, she has significant experience in writing pediatric investigational plans (PIP).
Marie Deneux, Senior Director, Regulatory Science, Drugs & Biologics
Marie assists international clients in the design and implementation of global product development plans and associated regulatory strategy, in particular in the area of rare diseases. She is involved in the development of biotech products, new chemical entities, cell and gene therapy medicinal products and repurposed drugs. Marie has extensive experience in interactions with the European Medicines Agency (EMA), national European Health Authorities and the Food and Drug Administration (FDA), in particular in coordinating SME meetings, Scientific Advice Request/Protocol Assistance/Parallel Scientific Advice with FDA and FDA Type B meetings and associated background documentation, while taking into consideration global development constraints.