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EMA Guidance: Procedural Advice for Orphan Medicinal Product Designation

On 11 November 2022, the European medicines agency released updated guidance for sponsors about the "Procedural Advice for Orphan Medicinal Product Designation".

Orphan medicine: A medicine for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition that is rare (affecting not more than five in 10,000 people in the European Union) or where the medicine is unlikely to generate sufficient profit to justify research and development costs.

This guidance details the requirements, in examining an application for orphan medicinal product designation, the Committee for Orphan Medicinal Products (COMP) will focus on determining whether the sponsor has established that the designation criteria are met.

A panel of experts will be established by the Committee to evaluate applications for orphan medicinal products in order to assist in developing a policy. 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.

All orphan applications should be created and submitted via the IRIS portal, which also contains the relevant procedural advice.

Click this LINK to know more detailed information about the Pre-submission meeting, Appointment of rapporteurs, Submission, Validation, Evaluation, Opinion, Appeal, Decision, and the General advice provided in this guidance.


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