Applying for orphan drug designation (ODD)

23/02/2023 -

An orphan medicinal product is 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. 

About 30 million people living in the European Union (EU) suffer from a rare disease.

 

Orphan designation can only be granted in the EU, by the European Commission, once an application for designation has been reviewed by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (Regulation (EC) No 141/2000 of 16 December 1999). 

A sponsor applying for designation of a medicine as an orphan medicinal product can apply for designation at any stage of the development of the medicine before the application for Marketing Authorization is made.

 

Sponsors can apply for orphan designation to EMA: 

Sponsors are encourage to send the application a few days before any of the published submission deadlines available on the EMA website.

Deadlines for submission of applications have been fixed by EMA in order to synchronize evaluation of applications for orphan designation with the meetings of the COMP.

  • By previously requesting a pre-submission meeting/teleconference with the EMA, free of charge, at least two months prior to submitting the orphan designation application via the IRIS portal.

Sponsors should create an initial draft application before creating the application for the pre-submission discussion. The draft submission should be populated with the relevant data and documents, but should not be submitted, at least one week before the pre-submission meeting date. 

The pre-submission meeting/teleconference is strongly recommended as the evaluation process by the COMP has a maximum duration of 90 days without clock stops.

 

The application for Orphan Drug Designation should include: 

  • General administrative scientific information (name of the active substance(s), proposed orphan condition, invented name, strength, pharmaceutical form and route of administration, Sponsor/contact person).

The sponsor must provide proof of establishment in a country of the EEA.

  • Scientific document :

Section A-Description of the condition 

Section B-Prevalence of the condition

Section C-Potential for return on investment

Section D-Other methods for diagnosis, prevention or treatment of the condition

Section E-Description of the stage of development 

  • Section F-Bibliography

All published references referred to should be submitted together with the application.

  • Section G-Transfer of the Orphan designation to another sponsor and change in the name of the Sponsor and/or the address of the Sponsor

If applicable. Both the applicant and the sponsor need to be established in the Union. 

  • Section H-Amendment of an existing designation

In exceptional cases, change of the designated condition is possible.

 

The full content of the application for Orphan Drug Designation should be submitted in English. If the bibliographical references submitted are not in English, a summary in English should be included where possible.

 

The EMA Secretariat will review the submission of the application within a month from the submission deadline. Once the validation process is successfully completed, the timetable to start the procedure for the evaluation will be confirmed.


The Committee for Orphan Medicinal Products (COMP) evaluation will focus on determining whether the sponsor has established that the designation criteria are met:

  • The medicine is intended for the treatment, prevention or diagnosis of a life-threatening or chronically debilitating condition;
  • The prevalence of the condition in the European Union is not more than five in 10,000 or it is unlikely that marketing the medicinal product in the European Union, without incentives, would generate sufficient return to justify the necessary investment;
  • No satisfactory method of diagnosis prevention or treatment exists, or if such a method exists, the medicinal product will be of significant benefit to those affected by the condition.

 

Before day 90, the COMP adopts its opinion. EMA forwards then the COMP opinion to the European Commission (EC) for the Decision process and to the sponsor via the online portal. The decision is adopted by the Commission, within 30 days of its receipt of the COMP opinion and forwarded to the sponsor.


Following the EC decision on the designation, a public summary of opinion on orphan designation will be published on the EMA website.

Upon a favorable decision by the Commission, the designated medicinal product shall be entered in the Community Register of Orphan Medicinal Products. 

 

What can AxeRegel do for your company?

AxeRegel can assist you with the preparation of your orphan drug designation application dossier in Europe and provide regulatory support during the procedure.

 

Sources / Links:

Tags : #Exploitant pharmaceutical site