Navigating challenges in clinical trials for bullous pemphigoid and pemphigus: learnings from the clinical trial graveyard and an international expert survey
This study analysed 32 failed drug development trials and surveyed 56 experts in the field of autoimmune bullous diseases to identify key challenges in bullous pemphigoid and pemphigus research. Clinical trials primarily failed due to unmet efficacy endpoints and recruitment difficulties, with experts highlighting the need to improve endpoint selection and patient enrolment strategies through international collaboration to develop more effective trial designs and advance new therapeutic options.
Authors
- Dedee Frances Murrell (ORCID: https://orcid.org/0000-0003-2971-0199)
- Victoria P. Werth (ORCID: https://orcid.org/0000-0003-3030-5369)
- Joost M. Meijer (ORCID: https://orcid.org/0000-0001-7654-3528)
- Patrick F. Dunn (ORCID: https://orcid.org/0000-0002-7459-1232)
- Barbara Horváth (ORCID: https://orcid.org/0000-0001-8559-3674)
- Maria C. Bolling (ORCID: https://orcid.org/0000-0003-2086-9363)
- Phyllis I. Spuls (ORCID: https://orcid.org/0000-0002-6035-2863)
- Eva W H Korte (ORCID: https://orcid.org/0000-0001-9166-5654)
- Marjolein A J Hiel (ORCID: https://orcid.org/0000-0002-7715-1697)
- Marc Yale (ORCID: https://orcid.org/0009-0006-6722-3569)
Institutions
- University Medical Center Groningen (NL)
- Amsterdam UMC Location University of Amsterdam (NL)
- UNSW Sydney (AU)
- St George Hospital (AU)
- Philadelphia VA Medical Center (US)
- St. George Hospital (IN)
- Institute of Infection and Immunity (CA)
- International Flame Research Foundation (IT)
- University of Pennsylvania (US)
- University of Amsterdam (NL)
Publication Details
- Journal
- British Journal of Dermatology
- Published
- 2025-09-12
- DOI
- https://doi.org/10.1093/bjd/ljaf366
- Primary Topic
- Autoimmune Bullous Skin Diseases
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Universität zu Lübeck
- European Academy of Dermatology and Venereology