Procedural advice for orphan medicinal product designation

On 19 November 2019 the EMA updated this guidance for sponsors.

In examining an application for orphan medicinal product designation, the COMP will focus on determining whether the sponsor has established that the designation criteria are met, i.e.:

  • the life-threatening or debilitating nature of the condition;
  • the medical plausibility of the proposed orphan indication;
  • that the prevalence of the condition in the European Union is not more than five in 10,000 or that it is unlikely that marketing the medicinal product in the European Union, without incentives, would generate sufficient return to justify the necessary investment;
  • that no satisfactory method of diagnosis prevention or treatment exists, or if such a method exists, that the medicinal product will be of significant benefit to those affected by the condition.
Read the pdf
Copyright Widler & Schiemann AG 2015. All Rights Reserved. /