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@ARTICLE{Christopoulos:272845,
      author       = {P. Christopoulos and R. Schlenk$^*$ and D. Kazdal and M.
                      Blasi and J. Lennerz and R. Shah and J. Budczies and N.
                      Malek and S. Fröhling$^*$ and R. Rosenquist and P.
                      Schirmacher$^*$ and F. Bozorgmehr and J. Kuon and M. Reck
                      and M. Thomas and A. Stenzinger$^*$},
      title        = {{R}eal-world data for precision cancer medicine - a
                      {E}uropean perspective.},
      journal      = {Genes, chromosomes $\&$ cancer},
      volume       = {62},
      number       = {9},
      issn         = {1045-2257},
      address      = {New York, NY},
      publisher    = {Wiley-Liss},
      reportid     = {DKFZ-2023-00418},
      pages        = {557-563},
      year         = {2023},
      note         = {2023 Sep;62(9):557-563},
      abstract     = {Leveraging real-world data (RWD) for drug access is
                      necessary to overcome a key challenge of modern precision
                      oncology: tackling numerous low-prevalence oncogenic
                      mutations across cancers. Withholding a potentially active
                      medication in patients with rare mutations for the sake of
                      control chemotherapy or 'best' supportive care is neither
                      practicable nor ethically justifiable anymore, particularly
                      as RWD could meanwhile be used instead, according to
                      scientific principles outlined by the US Food and Drug
                      Administration, European Medicines Agency and other
                      stakeholders. However, practical implementation varies, with
                      occasionally opposite recommendations based on the same
                      evidence in different countries. In the face of growing need
                      for precision drugs, more transparency of evaluation, a
                      priori availability of guidance for the academia and
                      industry, as well as a harmonized framework for health
                      technology assessment across the European Union (EU) are
                      imperative. These could in turn trigger infrastructural
                      changes in national and pan-European registries, cancer
                      management guidelines (e.g. frequency of routine radiologic
                      restaging, inclusion of patient-reported outcomes), and the
                      health data space, to ensure conformity with declared
                      standards and facilitate extraction of RWD sets (including
                      patient-level data) suitable for approval and pricing with
                      minimal effort. For an EU-wide unification of precision
                      cancer medicine, collective negotiation of drug supply
                      contracts and funding solidarity would additionally be
                      required to handle the financial burden. According to
                      experience from pivotal European programs, off-label use
                      could potentially also be harmonized across EU-states to
                      accelerate availability of novel drugs, streamline
                      collection of valuable RWD, and mitigate related costs
                      through wider partnerships with pharmaceutical companies.},
      keywords     = {clinical trials (Other) / drug approval (Other) /
                      health-technology assessment (Other) / off-label (Other) /
                      precision medicine (Other) / real-world data (Other) /
                      real-world evidence (Other) / reimbursement (Other)},
      cin          = {HD01 / B340 / W010},
      ddc          = {610},
      cid          = {I:(DE-He78)HD01-20160331 / I:(DE-He78)B340-20160331 /
                      I:(DE-He78)W010-20160331},
      pnm          = {312 - Funktionelle und strukturelle Genomforschung
                      (POF4-312)},
      pid          = {G:(DE-HGF)POF4-312},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:36852573},
      doi          = {10.1002/gcc.23135},
      url          = {https://inrepo02.dkfz.de/record/272845},
}