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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.},
      keywords     = {Humans / Amyotrophic Lateral Sclerosis: drug therapy /
                      Germany / Breast Neoplasms: drug therapy / Female / Research
                      Design / Clinical Trials as Topic: methods / Registries},
      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},
      pubmed       = {pmid:40237254},
      pmc          = {pmc:PMC12641071},
      doi          = {10.1002/cpt.3684},
      url          = {https://pub.dzne.de/record/282544},
}