An urgent need remains for new paediatric oncology drugs to cure children who die from cancer and to reduce drug-related sequelae in survivors. In 2007, the European Paediatric Regulation came into law requiring industry to create paediatric drug (all types of medicinal products) development programmes alongside those for adults. Unfortunately, paediatric drug development is still largely centred on adult conditions and not a mechanism of action (MoA)-based model, even though this would be more logical for childhood tumours as these have much fewer non-synonymous coding mutations than adult malignancies. Recent large-scale sequencing by International Genome Consortium and Paediatric Cancer Genome Project has further shown that the genetic and epigenetic repertoire of driver mutations in specific childhood malignancies differs from more common adult-type malignancies. To bring about much needed change, a Paediatric Platform, ACCELERATE, was proposed in 2013 by the Cancer Drug Development Forum, Innovative Therapies for Children with Cancer, the European Network for Cancer Research in Children and Adolescents and the European Society for Paediatric Oncology. The Platform, comprising multiple stakeholders in paediatric oncology, has three working groups, one with responsibility for promoting and developing high-quality MoA-informed paediatric drug development programmes, including specific measures for adolescents. Key is the establishment of a freely accessible aggregated database of paediatric biological tumour drug targets to be aligned with an aggregated pipeline of drugs. This will enable prioritisation and conduct of early phase clinical paediatric trials to evaluate these drugs against promising therapeutic targets and to generate clinical paediatric efficacy and safety data in an accelerated time frame. Through this work, the Platform seeks to ensure that potentially effective drugs, where the MoA is known and thought to be relevant to paediatric malignancies, are evaluated in early phase clinical trials, and that this approach to generate pre-clinical and clinical data is systematically pursued by academia, sponsors, industry, and regulatory bodies to bring new paediatric oncology drugs to front-line therapy more rapidly.

Belgian Federal Agency for Medicines and Health Products Brussels Belgium

Boehringer Ingelheim Germany

Bristol Myers Squibb 5 Research Parkway Wallingford CT 06492 USA

Cancer Research UK Clinical Trials Unit Institute of Cancer and Genomic Sciences University of Birmingham Birmingham UK

Centre for English Language Teaching University of York UK

Clinical Cooperation Unit Pediatric Oncology Heidelberg Germany

Create for Chloe and UK representative for aPODD UK

Department of Clinical Research Institut Gustave Roussy Paris Sud University Paris France

Department of Paediatric Oncology Faculty of Medicine University Hospital Brno and Masaryk University Brno Czech Republic; Regional Centre for Applied Molecular Oncology Masaryk Memorial Cancer Institute ICRC Brno Brno Czech Republic

Department of Pediatric and Adolescent Oncology Gustave Roussy France

Division of Clinical Studies The Institute of Cancer Research London UK

Genentech Inc 1 DNA Way South San Francisco CA 94080 USA

German Cancer Research Center and Heidelberg University Hospital Heidelberg 69120 Germany

Hoffman La Roche Basel Switzerland; Department of Pediatric Oncology Emma Children's Hospital Academic Medical Center Amsterdam The Netherlands

Novartis Pharma AG Basel CH 4002 Switzerland

Paediatric Haematology Oncology Unit Antwerp University Hospital Antwerp University Belgium

PODD Foundation London UK

Product Development Scientific Support Department European Medicines Agency Canary Wharf London UK

TetraLogic Pharmaceuticals Malvern PA 19355 USA

The Institute of Cancer Research The Royal Marsden NHS Foundation Trust Sutton London UK

The Institute of Cancer Research The Royal Marsden NHS Foundation Trust Sutton London UK; Children and Young People's Unit The Royal Marsden NHS Foundation Trust Sutton Surrey UK

U830 INSERM Recherche Translationelle en Oncologie Pédiatrique and Department of Pediatric Oncology Institut Curie Paris France

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Joint adolescent-adult early phase clinical trials to improve access to new drugs for adolescents with cancer: proposals from the multi-stakeholder platform-ACCELERATE