Drug discovery in paediatric oncology: roadblocks to progress

Nat Rev Clin Oncol. 2014 Dec;11(12):732-9. doi: 10.1038/nrclinonc.2014.149. Epub 2014 Sep 16.


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.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Child
  • Child, Preschool
  • Drug Discovery / methods*
  • Humans
  • Infant
  • Neoplasms / drug therapy*