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@ARTICLE{Reaman:294420,
author = {G. Reaman and L. Stancato and G. Vassal and J. M. Maris},
title = {{C}rossing {O}ceans: {P}reclinical {C}ollaboration to
{I}mprove {P}ediatric {D}rug {D}evelopment.},
journal = {Educational book / American Society of Clinical Oncology},
volume = {40},
number = {40},
issn = {1548-8748},
address = {Alexandria, VA},
publisher = {American Society of Clinical Oncology},
reportid = {DKFZ-2024-02249},
pages = {409 - 416},
year = {2020},
abstract = {Changes in the regulatory environment affecting pediatric
cancer drug development in the United States and the
European Union provide unprecedented opportunity to advance
the concept of precision medicine to children with cancer.
Increasing evidence suggests that new drugs and biologic
products directed at molecular targets presumed to be
etiologically associated with many adult cancers may well
provide therapeutic options for selected subsets of children
with cancer despite their histologic and biologic
differences. Regulatory requirements for early evaluation of
appropriate new drugs for children based on their molecular
mechanism of action, rather than the specific clinical
indications for which they are developed and/or approved,
will shorten the unacceptable time lag between
first-in-human and first-in-children studies. The relative
scarcity of pediatric patients eligible for
biomarker-directed studies and the ever-expanding compendium
of new targeted agents mandate rational, science-based
decision-making in selecting and prioritizing appropriate
drugs to study early in development. A critical component of
the evidence base in such decision-making includes
preclinical testing of relevant drugs in pediatric
tumor-specific in vitro and in vivo models. Established
preclinical testing programs with academic
investigator-industry collaborations are actively engaged in
such activities. International collaboration is required to
address the resource constraints and increasing number of
potential products to be tested in a timely, efficient,
nonduplicative, and cost-effective manner.},
keywords = {Child / Drug Development / Humans},
ddc = {610},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:32207673},
doi = {10.1200/EDBK_278893},
url = {https://inrepo02.dkfz.de/record/294420},
}