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@ARTICLE{Pearson:125219,
      author       = {A. D. J. Pearson and S. Pfister$^*$ and A. Baruchel and
                      J.-P. Bourquin and M. Casanova and L. Chesler and F. Doz and
                      A. Eggert$^*$ and B. Geoerger and D. Jones$^*$ and P. R.
                      Kearns and J. J. Molenaar and B. Morland and G.
                      Schleiermacher and J. Schulte$^*$ and J. Vormoor and L. V.
                      Marshall and C. M. Zwaan and G. Vassal},
      collaboration = {E. a. B. C. o. t. I. T. f. C. w. C. E. Consortium},
      title        = {{F}rom class waivers to precision medicine in paediatric
                      oncology.},
      journal      = {The lancet / Oncology},
      volume       = {18},
      number       = {7},
      issn         = {1470-2045},
      address      = {London},
      publisher    = {The Lancet Publ. Group},
      reportid     = {DKFZ-2017-01374},
      pages        = {e394 - e404},
      year         = {2017},
      abstract     = {New drugs are crucially needed for children with cancer.
                      The European Paediatric Regulation facilitates paediatric
                      class waivers for drugs developed for diseases only
                      occurring in adults. In this Review, we retrospectively
                      searched oncology drugs that were class waivered between
                      June, 2012, and June, 2015. 147 oncology class waivers were
                      confirmed for 89 drugs. Mechanisms of action were then
                      assessed as potential paediatric therapeutic targets by both
                      a literature search and an expert review. 48 $(54\%)$ of the
                      89 class-waivered drugs had a mechanisms of action
                      warranting paediatric development. Two $(2\%)$
                      class-waivered drugs were considered not relevant and 16
                      $(18\%)$ required further data. In light of these results,
                      we propose five initiatives: an aggregated database of
                      paediatric biological tumour drug targets; molecular
                      profiling of all paediatric tumours at diagnosis and
                      relapse; a joint academic-pharmaceutical industry
                      preclinical platform to help analyse the activity of new
                      drugs (Innovative Therapy for Children with Cancer
                      Paediatric Preclinical Proof-of-Concept Platform);
                      paediatric strategy forums; and the suppression of article
                      11b of the European Paediatric Regulation, which allows
                      product-specific waivers on the grounds that the associated
                      condition does not occur in children. These initiatives and
                      a mechanism of action-based approach to drug development
                      will accelerate the delivery of new therapeutic drugs for
                      front-line therapy for those children who have unmet medical
                      needs.},
      subtyp        = {Review Article},
      cin          = {B062 / L201 / L101},
      ddc          = {610},
      cid          = {I:(DE-He78)B062-20160331 / I:(DE-He78)L201-20160331 /
                      I:(DE-He78)L101-20160331},
      pnm          = {312 - Functional and structural genomics (POF3-312)},
      pid          = {G:(DE-HGF)POF3-312},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:28677575},
      doi          = {10.1016/S1470-2045(17)30442-4},
      url          = {https://inrepo02.dkfz.de/record/125219},
}