% IMPORTANT: The following is UTF-8 encoded.  This means that in the presence
% of non-ASCII characters, it will not work with BibTeX 0.99 or older.
% Instead, you should use an up-to-date BibTeX implementation like “bibtex8” or
% “biber”.

@ARTICLE{Depner:283030,
      author       = {Depner, Frank Lucas and Russek, Martin and Röthlein,
                      Christoph and Becker, Cornelia and Peltner, Jonas and
                      Pfeifer, Kerstin and Reviers, Evy and De Valck, Dirk and
                      Wicherski, Julia and Hartikainen, Sirpa and Tolppanen,
                      Anna-Maija and Haenisch, Britta},
      title        = {{K}ey {S}takeholders' {K}nowledge, {O}pinions, and
                      {I}nterests on {R}eal-{W}orld {E}vidence in the {R}egulatory
                      {P}rocess-{R}esults of an {EU}-{W}ide {S}urvey.},
      journal      = {Clinical and translational science},
      volume       = {18},
      number       = {12},
      issn         = {1752-8054},
      address      = {Oxford},
      publisher    = {Wiley-Blackwell},
      reportid     = {DZNE-2025-01442},
      pages        = {e70454},
      year         = {2025},
      abstract     = {Real-world data (RWD) and real-world evidence (RWE) are
                      increasingly gaining attention in supporting drug regulatory
                      decision making. This study assessed knowledge, opinions,
                      and usage patterns of key stakeholders regarding the status
                      of RWD/RWE and AI implementation in health technology
                      assessment (HTA) and drug regulation and aimed to identify
                      the primary obstacles hindering adoption of these
                      technologies. Four surveys tailored to different
                      stakeholders were created and disseminated online to the
                      respective target groups, including I) regulatory
                      authorities, HTA bodies, and the pharmaceutical industry,
                      II) academia, III) payers, and IV) patients and physicians.
                      The responses were analyzed using descriptive statistics or
                      qualitative content analysis with inductive coding. The
                      survey was active from May 6, 2024 to June 30, 2024.
                      Altogether, 221 respondents participated in the survey.
                      Among respondents from regulatory/HTA authorities or
                      industry, $75.4\%$ viewed RWD/RWE as important for future
                      decision making. Respondents from this group already using
                      RWD (n = 56) most frequently reported obstacles regarding
                      data quality $(89.3\%),$ data access $(62.5\%),$ and
                      data-coding standardization $(57.1\%).$ Patients and
                      physicians predominantly had positive expectations about the
                      use of RWD/RWE, and $94.3\%$ indicated willingness to share
                      healthcare data for research, but all respondents also
                      expressed concerns, with data privacy being most frequently
                      mentioned $(75.5\%).$ The results show that although
                      stakeholders are optimistic about RWD/RWE implementation
                      into regulatory practice, our survey suggests that
                      successful implementation may benefit from further
                      development in several areas, including guideline
                      harmonization, RWD infrastructure optimization and
                      accessibility, and professional education.},
      keywords     = {Humans / Technology Assessment, Biomedical: methods /
                      Technology Assessment, Biomedical: legislation $\&$
                      jurisprudence / Surveys and Questionnaires: statistics $\&$
                      numerical data / Stakeholder Participation / Health
                      Knowledge, Attitudes, Practice / Male / Female / Decision
                      Making / Adult / Middle Aged / European Union / RWD (Other)
                      / RWE (Other) / patients (Other) / stakeholders (Other) /
                      survey (Other)},
      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:41420643},
      pmc          = {pmc:PMC12717853},
      doi          = {10.1111/cts.70454},
      url          = {https://pub.dzne.de/record/283030},
}