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@ARTICLE{Johann:169798,
      author       = {P. Johann$^*$ and D. Lenz and M. Ries},
      title        = {{T}he drug development pipeline for glioblastoma-{A} cross
                      sectional assessment of the {FDA} {O}rphan {D}rug {P}roduct
                      designation database.},
      journal      = {PLOS ONE},
      volume       = {16},
      number       = {7},
      issn         = {1932-6203},
      address      = {San Francisco, California, US},
      publisher    = {PLOS},
      reportid     = {DKFZ-2021-01552},
      pages        = {e0252924 -},
      year         = {2021},
      note         = {#EA:B062#},
      abstract     = {Glioblastoma (GBM) is the most common malignant brain
                      tumour among adult patients and represents an almost
                      universally fatal disease. Novel therapies for GBM are being
                      developed under the orphan drug legislation and the
                      knowledge on the molecular makeup of this disease has been
                      increasing rapidly. However, the clinical outcomes in GBM
                      patients with currently available therapies are still
                      dismal. An insight into the current drug development
                      pipeline for GBM is therefore of particular interest.To
                      provide a quantitative clinical-regulatory insight into the
                      status of FDA orphan drug designations for compounds
                      intended to treat GBM.Quantitative cross-sectional analysis
                      of the U.S. Food and Drug Administration Orphan Drug Product
                      database between 1983 and 2020. STROBE criteria were
                      respected.Four orphan drugs out of 161 $(2,4\%)$ orphan drug
                      designations were approved for the treatment for GBM by the
                      FDA between 1983 and 2020. Fourteen orphan drug designations
                      were subsequently withdrawn for unknown reasons. The number
                      of orphan drug designations per year shows a growing trend.
                      In the last decade, the therapeutic mechanism of action of
                      designated compounds intended to treat glioblastoma shifted
                      from cytotoxic drugs (median year of designation 2008) to
                      immunotherapeutic approaches and small molecules (median
                      year of designation 2014 and 2015 respectively) suggesting
                      an increased focus on precision in the therapeutic mechanism
                      of action for compounds the development pipeline.Despite the
                      fact that current pharmacological treatment options in GBM
                      are sparse, the drug development pipeline is steadily
                      growing. In particular, the surge of designated
                      immunotherapies detected in the last years raises the hope
                      that elaborate combination possibilities between classical
                      therapeutic backbones (radiotherapy and chemotherapy) and
                      novel, currently experimental therapeutics may help to
                      provide better therapies for this deadly disease in the
                      future.},
      cin          = {B062},
      ddc          = {610},
      cid          = {I:(DE-He78)B062-20160331},
      pnm          = {312 - Funktionelle und strukturelle Genomforschung
                      (POF4-312)},
      pid          = {G:(DE-HGF)POF4-312},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:34234357},
      doi          = {10.1371/journal.pone.0252924},
      url          = {https://inrepo02.dkfz.de/record/169798},
}