ArticleTherapeutic innovation & regulatory science2026
Strategic Drivers Behind Early Withdrawal of Orphan Designations in the EU: A Retrospective Analysis (2000-2024).
Article in Therapeutic innovation & regulatory science, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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Who cites it
1 citing paper in PubMed.
- Rebalancing innovation, affordability, and access for orphan drugs in the European Union.The Lancet regional health. Europe · 2026Review
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Authors and funding
3 authors.
Funding
Abstract
purposeThe orphan legislation came into force in the European Union (EU) in 2000, providing incentives for the development of orphan medicines. To be eligible for incentives, the applicant needs to apply for an orphan designation (OD). However, at any time, the marketing authorisation holder (MAH) can request the removal of the OD. The possible motives underpinning premature removal of OD have been the subject of speculation. Our aim is to study every early OD removal for the orphan medicinal products (OMPs) approved in the EU between 2000 and 2024 and determine the main reasons behind this phenomenon.
methodsWe identified all the orphan medicines approved between 2000 and 2024. We considered approval date by the European Commission (EC), orphan designation (OD) status, company name, active substance name, trade name, ATC code and Therapeutic Area, and the date of the removal of the OD. Information on the OD withdrawal was cross-checked with the documents on the EMA website, and the legal status of the patent and supplementary protection certificates (SPC) was checked at the European Patent Register.
resultsDuring the period 2000-2024, 285 OMPs were approved by the EC. Overall, 41 (11.8%) orphan designations were prematurely removed, corresponding to 23 different OMPs.
conclusionsThree 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.
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