Demonstrating significant benefit of orphan medicines: analysis of 15 years of experience in Europe

Laura Fregonese, Lesley Greene, Matthias Hofer, Armando Magrelli, Frauke Naumann-Winter, Kristina Larsson, Maria Sheean, Violeta Stoyanova-Beninska, Stelios Tsigkos, Kerstin Westermark, Bruno Sepodes

Research output: Contribution to journalReview articlepeer-review


In the European Union demonstration of 'significant benefit’ is mandatory if satisfactory methods exist for a disease targeted by a new orphan medicinal product. Significant benefit is required at the time of orphan designation, when it can be supported by preclinical studies, and at the time of marketing authorization, when clinical data are needed. For the first time, our work has identified, defined and organized the scientific grounds on which significant benefit is granted in the European Union, based on a review of the orphan medicinal products authorized in the years 2000–2015, and on the working experience of the Committee of Orphan Medicinal Products. The resulting conceptual framework is a tool for medicine developers to reflect on potential areas of advantage of their candidate products, and for a broad range of stakeholders to stimulate the discussion on the added value of orphan medicines across the whole development lifecycle. An analysis of the scientific grounds of the 'significant benefit’ as per the European Regulation, supporting the added value for patients of those orphan medicinal products that demonstrate to be of significant benefit.

Original languageEnglish
Pages (from-to)90-100
Number of pages11
JournalDrug Discovery Today
Issue number1
Publication statusPublished - Jan 1 2018
Externally publishedYes

ASJC Scopus subject areas

  • Pharmacology
  • Drug Discovery


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