Procedural advice for orphan medicinal product designation: Guidance for sponsors

Frequently asked questions ................................................................... 10

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https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/procedural-advice-orphan-medicinal-product-designation-guidance-sponsors_en.pdf

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Wednesday, April 3, 2024

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Geotags:

EUROPEAN UNION, EC, IRELAND, JAPAN, EU, US, UNITED STATES

Summary

Key Points:

Frequently asked questions ................................................................... 10
Procedural advice for orphan medicinal product designation
EMA/420706/2018

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1.
This Regulation lays down the procedure for
the designation of medicinal products as orphan medicinal products and provides incentives for the
development and placing on the market of designated orphan medicinal products.
The Regulation also
establishes the Committee for Orphan Medicinal Products (COMP) within the European Medicines
Agency (EMA), which is responsible for examining applications for orphan medicinal product
designation.
As of 28 April 2000, the date this Regulation entered into force, sponsors have been able to
submit applications for orphan medicinal product designation to the EMA.
Procedural advice for orphan medicinal product designation
EMA/420706/2018
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2.
Objectives
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.
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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The complete application should include:

Procedural advice for orphan medicinal product designation
EMA/420706/2018

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Document

Format

General administrative and scientific information completed online via the portal.
Important:
In preparing an application for orphan medicinal product designation, sponsors are requested to follow
the Commission guideline (2022/C 440/02) on the format and content of applications for designation
as orphan medicinal products.
Please refer to the Procedural advice on
appeal procedure for Orphan Medicinal Product Designation (EMEA/2677/01 Rev.3).
The grounds for the
Procedural advice for orphan medicinal product designation
EMA/420706/2018

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appeal should be based only on the original information provided in the application for orphan
designation.
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The European Commission will enter the designated medicinal product in the Community Register
of Orphan Medicinal Products.
General advice
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Full information on the procedure for orphan medicinal products designation is available on the
EMA orphan designation website.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the sponsor may submit an
application for orphan medicinal product designation to the Agency at any stage of development of the
Procedural advice for orphan medicinal product designation
EMA/420706/2018

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medicinal product as long as the criteria for designation may be justified.
A request for orphan medicinal product designation may be made for
a new orphan indication for an already authorised medicinal product.
Procedural advice for orphan medicinal product designation
EMA/420706/2018
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Which are the sponsor?s options in case of negative outcome for orphan designation?