Procedural Advice for Orphan Medicinal Product Designation

On 27 September 2021 the EMA published 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.

The evaluation process has a maximum duration of 90 days without clock stops and cannot be lengthened to accommodate for the lack of data or other omissions in the application submitted by the sponsor. To assist in the development of a policy on orphan medicinal products, an expert network will be built up by the Committee, with expert(s) identified as appropriate to be involved in the evaluation of applications for orphan medicinal product designation.

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