Neuroblastoma, the commonest paediatric extra-cranial tumour, remains a leading cause of death from cancer in children. There is an urgent need to develop new drugs to improve cure rates and reduce long-term toxicity and to incorporate molecularly targeted therapies into treatment. Many potential drugs are becoming available, but have to be prioritised for clinical trials due to the relatively small numbers of patients. Areas covered: The current drug development model has been slow, associated with significant attrition, and few new drugs have been developed for neuroblastoma. The Neuroblastoma New Drug Development Strategy (NDDS) has: 1) established a group with expertise in drug development; 2) prioritised targets and drugs according to tumour biology (target expression, dependency, pre-clinical data; potential combinations; biomarkers), identifying as priority targets ALK, MEK, CDK4/6, MDM2, MYCN (druggable by BET bromodomain, aurora kinase, mTORC1/2) BIRC5 and checkpoint kinase 1; 3) promoted clinical trials with target-prioritised drugs. Drugs showing activity can be rapidly transitioned via parallel randomised trials into front-line studies. Expert opinion: The Neuroblastoma NDDS is based on the premise that optimal drug development is reliant on knowledge of tumour biology and prioritisation. This approach will accelerate neuroblastoma drug development and other poor prognosis childhood malignancies.

b Instituto de Investigación Sanitaria La Princesa Madrid Spain

c Paediatric Drug Development Children and Young People's Unit Royal Marsden Hospital London UK

e Hoffman La Roche Basel Switzerland

Emma Children's Hospital Amsterdam Netherlands

f Department of Paediatric and Adolescent Oncology Institut Gustave Roussy Villejuif France

g Department of Pediatric Oncology and Hematology Charite University Hospital Berlin Germany

h Department of Paediatric Adolescents and Young Adults Oncology and INSERM U830 Institut Curie Paris France

i Department Paediatric Oncology Great Ormond Street Hospital London UK

j Division of Clinical Studies Institute of Cancer Research London UK

k Centre for Medical Genetic Ghent University Ghent Belgium

l Princess Maxima Center for Pediatric Oncology University of Amsterdam Amsterdam Netherlands

m Department of Pediatric Oncology University of Essen Essen Germany

o Department of Women's and Children's Health Karolinska Institute Stockholm Sweden

p School of Biosciences University of Kent Canterbury UK

Paediatric Phase 1 2 Clinical Trials Unit Paediatric Haematology and Oncology Hospital Niño Jesus Madrid Spain

q Wolfson Childhood Cancer Research Centre Newcastle University Newcastle UK

r Department of Pediatric Oncology and Hematology University of Cologne Cologne Germany

s European Medicines Agency London UK

t Masaryk University University Hospital Brno Czech Republic

Theodor Boveri Institute and Comprehensive Cancer Center Mainfranken Biocenter University of Wurzburg Wurzburg Germany

u Department of Pediatric Oncology International Clinical Research Center St Anne's University Hospital Brno Czech Republic

v RECAMO Masaryk Memorial Cancer Centre Brno Czech Republic

w Pediatric Hematology Oncology Unit Antwerp University Hospital Antwerp Belgium

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

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