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European regulatory strategy for supporting childhood cancer therapy developments

D. Karres, G. Lesa, F. Ligas, S. Benchetrit, S. Galluzzo, K. Van Malderen, J. Sterba, M. van Dartel, M. Renard, P. Sisovsky, S. Wang, K. Norga

. 2022 ; 177 (-) : 25-29. [pub] 20221006

Language English Country England, Great Britain

Document type Journal Article

Persistent link   https://www.medvik.cz/link/bmc22032346

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.

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$a Karres, Dominik $u Paediatric Medicines Office, Scientific Evidence Generation Department, Human Medicines Division, European Medicines Agency (EMA), Amsterdam, Netherlands. Electronic address: Dominik.Karres@ema.europa.eu
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$a 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.
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$a Lesa, Giovanni $u Paediatric Medicines Office, Scientific Evidence Generation Department, Human Medicines Division, European Medicines Agency (EMA), Amsterdam, Netherlands
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$a Ligas, Franca $u Paediatric Medicines Office, Scientific Evidence Generation Department, Human Medicines Division, European Medicines Agency (EMA), Amsterdam, Netherlands
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$a Benchetrit, Sylvie $u Agence Nationale de Sécurité Du Médicament et des Produits de Santé (ANSM), Paris, France; Paediatric Committee of the European Medicines Agency, Amsterdam, Netherlands
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$a Galluzzo, Sara $u Italian Medicines Agency (AIFA), Rome, Italy; Scientific Advice Working Party and Paediatric Committee of the European Medicines Agency, Amsterdam, Netherlands
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$a Van Malderen, Karen $u Federal Agency for Medicines and Health Products (FAMHP), Brussels, Belgium; Paediatric Committee of the European Medicines Agency, Amsterdam, Netherlands
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$a Sterba, Jaroslav $u Department of Pediatric Oncology, University Hospital Brno, And Faculty of Medicine, Masaryk University, International Clinical Research Center, St Anne's University Hospital, Brno, Czech Republic; Paediatric Committee of the European Medicines Agency, Amsterdam, Netherlands
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$a van Dartel, Maaike $u College Ter Beoordeling van Geneesmiddelen, Utrecht, Netherlands; Paediatric Committee of the European Medicines Agency, Amsterdam, Netherlands
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$a Renard, Marleen $u University Hospitals Leuven, Leuven, Belgium; Federal Agency for Medicines and Health Products (FAMHP), Brussels, Belgium; Paediatric Committee of the European Medicines Agency, Amsterdam, Netherlands
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$a Sisovsky, Peter $u State Institute for Drug Control, Bratislava, Slovakia; Paediatric Committee of the European Medicines Agency, Amsterdam, Netherlands
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$a Wang, Siri $u Norwegian Medicines Agency, Oslo, Norway; Paediatric Committee of the European Medicines Agency, Amsterdam, Netherlands
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$a Norga, Koen $u Antwerp University Hospital, Paediatric Committee of the European Medicines Agency, Federal Agency for Medicines and Health Products (FAMHP), Brussels, Belgium
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