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@ARTICLE{Russek:282544,
      author       = {Russek, Martin and Peltner, Jonas and Haenisch, Britta},
      title        = {{S}upplementing {S}ingle‐{A}rm {T}rials with {E}xternal
                      {C}ontrol {A}rms—{E}valuation of {G}erman {R}eal‐{W}orld
                      {D}ata},
      journal      = {Clinical pharmacology $\&$ therapeutics},
      volume       = {118},
      number       = {6},
      issn         = {0009-9236},
      address      = {Hoboken, NJ},
      publisher    = {Wiley-Blackwell},
      reportid     = {DZNE-2025-01307},
      pages        = {1443 - 1450},
      year         = {2025},
      abstract     = {As single-arm trials (SATs) are increasingly used in
                      pharmaceutical research, the validity of such study designs
                      needs to be critically assessed. We characterize the
                      feasibility of supplementing SATs with real-world data
                      (RWD)-derived external control arms by determining the
                      proportion of SATs on breast cancer and amyotrophic lateral
                      sclerosis (ALS) for which an external control arm based on
                      RWD can be constructed. The main outcome measure is the
                      number and percentage of trials for which all important
                      eligibility criteria and at least one primary endpoint could
                      be identified in one of five German RWD sources. We surveyed
                      all SATs concerning breast cancer or ALS treatment
                      registered in the European Union’s clinical trial
                      registers between 2004 and 2023. Ten out of 379 breast
                      cancer SATs and 2 of 11 ALS SATs could feasibly be
                      supplemented with RWD-derived external control arms, if all
                      important eligibility criteria and a primary endpoint have
                      to be identifiable in the RWD source. Ninety-three breast
                      cancer trials had at least one outcome ascertainable in a
                      RWD source, and 35 trials had all important eligibility
                      criteria recorded in a RWD source. Nine ALS trials had at
                      least one primary endpoint ascertainable in RWD sources, and
                      2 had all important eligibility criteria recorded in a RWD
                      source. Our study shows that SATs with RWD-derived external
                      control arms will rarely be suitable to establish treatment
                      effects of medicines in the current setting for the
                      investigated phenotypes and that SATs should be designed
                      with limitations of the source of external controls in
                      mind.},
      cin          = {AG Hänisch},
      ddc          = {610},
      cid          = {I:(DE-2719)1013010},
      pnm          = {354 - Disease Prevention and Healthy Aging (POF4-354)},
      pid          = {G:(DE-HGF)POF4-354},
      typ          = {PUB:(DE-HGF)16},
      doi          = {10.1002/cpt.3684},
      url          = {https://pub.dzne.de/record/282544},
}