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Drug discovery in paediatric oncology: roadblocks to progress

Nature Reviews Clinical Oncology volume 11, pages 732–739 (2014)Cite this article

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Abstract

Approval of new cancer drugs for paediatric patients generally occurs after their development and approval for treating adult cancers. As most drug development occurs in the industry setting, the relatively small market of paediatric oncology does not provide the financial incentives for companies to actively pursue paediatric oncology solutions. Indeed, between 1948 and January 2003 the FDA approved 120 new cancer drugs, of which only 30 have been used in children. This slow rate of development must be addressed in a meaningful way if we are to make progress in the most pressing settings in childhood cancer. In this Viewpoint article, the key opinion leaders in the field weigh in and offer practical advice on how to address this issue.

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Acknowledgements

P.J.H. is supported by grants CA77776, CA165995 and NO1-CM-42216 from the National Cancer Institute. G.P. thanks Francois Doz and Gabriele Calaminus for useful discussions. K.P.-J. is supported by the UK National Institute for Health Research Great Ormond Street Hospital University College London Biomedical Research Centre award and the European Union's Seventh Framework Programme (FP7/2007–13) under the European Network for Cancer Research in Children and Adolescents project (grant number 261474).

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Authors and Affiliations

  1. The Children's Hospital of Philadelphia, 3501 Civic Center Boulevard, CTRB 10060, Philadelphia, 19104, PA, USA

    Peter C. Adamson

  2. The Research Institute, Nationwide Children's Hospital, 700 Children's Drive, Columbus, 43205, OH, USA

    Peter J. Houghton

  3. University Hospital of Padua, Via Giustiniani 3, 35128, Padua, Italy

    Giorgio Perilongo

  4. Institute of Child Health, Cancer Section, University College London, 30 Guilford Street, WC1N 1EH, London, UK

    Kathy Pritchard-Jones

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  1. Peter C. Adamson
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  2. Peter J. Houghton
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  4. Kathy Pritchard-Jones
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Corresponding authors

Correspondence to Peter C. Adamson, Peter J. Houghton, Giorgio Perilongo or Kathy Pritchard-Jones.

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Adamson, P., Houghton, P., Perilongo, G. et al. Drug discovery in paediatric oncology: roadblocks to progress. Nat Rev Clin Oncol 11, 732–739 (2014). https://doi.org/10.1038/nrclinonc.2014.149

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