Strategic Drivers Behind Early Withdrawal of Orphan Designations in the EU: A Retrospective Analysis (2000–2024)
Three main motives for the early removal of the OD were identified: lack of clinical evidence supporting the significant benefit for the new indication proposed, the companies' preference towards SPC extensions for the paediatric indication (instead of the two additional years of marketing exclusivity), or the new therapeutic indication added is not rare. There is no evidence of commercial "pay to enter" agreements between pharmaceutical companies.
Authors
- Georges Siotis (ORCID: https://orcid.org/0000-0002-4731-5398)
- L. Bouwman (ORCID: https://orcid.org/0000-0003-3098-6774)
- Micaël Castanheira (ORCID: https://orcid.org/0000-0002-3511-9947)
Institutions
- Center for Economic and Policy Research (US)
- Université Libre de Bruxelles (BE)
- American Pharmacists Association (US)
- Centre Pour La Recherche Economique et ses Applications (FR)
- National Research Fund Luxembourg (LU)
- Centre for Economic Policy Research (GB)
- Universidad Carlos III de Madrid (ES)
Publication Details
- Journal
- Therapeutic Innovation & Regulatory Science
- Published
- 2025-10-23
- DOI
- https://doi.org/10.1007/s43441-025-00890-z
- Primary Topic
- Pharmaceutical Economics and Policy
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Fonds De La Recherche Scientifique - FNRS