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Rare diseases, affect only or as much as 6 - 8% of humanity. They are very rare, but nonetheless exist
and people suffer because of them.
Orphan drugs - because this is the name of medicinal products used in rare diseases must have an orphan designation by submitting an application for Authorities.
The most important regulations
There is a common policy in all European Union Member States to obtain the status of an orphan medicinal product.
Regulation (EC) No 141/2000 adopted on 16 December 1999
lays down the EU procedure for designation of orphan medicines;
Regulation (EC) No 847/2000 adopted on 27 April 2000
sets out definitions essential for the application of the Orphan Regulation;
Regulation (EC) No 726/2004
provides the legal framework for the centralised authorisation and supervision of medicines
Regulation (EC) No 2049/2005
payment of fees to, and assistance from the EMA to micro, small and medium-sized enterprises (SMEs).
Regulation (EC) No 507/2006
provides the legal framework for the granting of a conditional marketing authorisation
Regulation (EC) No 1901/2006
market exclusivity for orphan medicines may be extended to twelve years if study results are submitted in compliance with an agreed paediatric investigation plan at the time of marketing authorisation.
EMA encourages Sponsors to consult before applying for orphan designation as the evaluation of the application takes min. 90 days and missing documents can not be added at this time. Consultations are free of charge and usually take the form of teleconferences.
At least 2 months before the planned submission of the application the request for consultation should be sent, and a minimum of one week before the scheduled date of the meeting, draft of an application should be delivered through the Eudralink platform.
Procedures for applying for orphan designation
1. Submission of an application to EMA
The application should be submitted before submitting the application for marketing authorization at any stage of the drug development through the Eudralink platform. Please note the application deadlines which EMA publishes on its website.
• cover letter
- Sponsor data
- active ingredients of the medicinal product;
- therapeutic indication
- justification that:
- There is currently no method to diagnose and treat people who are sick or the method is not
- The frequency of occurrence of the disease state in the Community
Disease is affecting up to 5 people per 10,000 people in the Community;
the medicinal product is to be used for the diagnosis, prevention or treatment of this condition.
- Potential investment profitability
Without adequate incentives, market introduction will not generate a sufficient return on investment in drug development; this product will be used when treating, diagnosing or preventing a serious, chronic, life-threatening condition.
• scientific part of the application
along with bibliographical references
• translation of the active substance / drug name (INN or current name) and
the proposed indication for all official European languages (and Icelandic and Norwegian).
All documents should be delivered in the appropriate format.
2. Validation of the application
EMA verifies the information contained in the application, prepares a summary for the COMP that may ask you to complete the data or documents.
3. Opinion COMP
(Committee for Orphan Medicinal Products, COMP) operating within the framework of the European Medicines Agency.
Within 90 days, COMP issues an opinion on whether to grant and orphan designation.
forwarded by EMA to the European Commission - adoption of the decision within 30 days,
the entry by the Commission into the Register of Orphan Medicinal Products of the Community;
publication of information on the granting orphan designation on the EMA website
The re-issue of the negative decision results in the Sponsor being unable to object.
4. Application for marketing authorization
Authorisation includes the therapeutic indication included in the application.
You can apply for marketing authorisation in other indications outside the scope of Regulation 141/2000;
The application is submitted to the EMA, considered by the CHMP (Committee for Medicinal Products for Human Use)
Authorisating a medicinal product for sale does not mean that it is available in all member countries of the European Union. Once approved for sale, the pre-market authorization holder must undergo individual procedures in each country to obtain reimbursement conditions and usually set the price of the medicine before placing the drug on the market.
Transfer of the sign
You can transfer the orphan medicinal product designation to another Sponsor by submitting an application to EMA.
Facilities for sponsors of research on orphan drugs:
Assistance in performing appropriate drug research to avoid objections when evaluating a marketing authorization application. The help consists in answering questions asked by the researcher / sponsor.
Scientific help in the preparation of the studies necessary to optimize the level of benefits and risks of the drug and to demonstrate the significant benefits of using the drug in a particular indication.
The procedure is available for medicines that have already obtained orphan status at a reduced price (pediatric medicines are exempt from payment).
EMA encourages you to submit a request for help in the scientific advice section of the FDA.
Centralized authorization procedure
Orphan drugs can be the subject of a marketing authorization application using a centralized procedure by submitting one application and receiving one valid decision in all EU countries.
For 10 years after obtaining the marketing authorization certificate, similar medicinal products will not be authorized for the same therapeutic indication (this period may be shortened to 6 years.
Fee reduction for marketing authorisation application
The possibility of applying for subsidies from EU and Member States programs as well as research and development initiatives. The European Commission offers grants for research on orphan drugs
Additional incentives for micro, small and medium-sized enterprises (SMEs)
Companies classified as SMEs benefit from further incentives in the development of medicines with an orphan indication. These include EMA administrative and procedural assistance and reduced fees
scientific advice specific to orphan drugs at a reduced price (e.g. application for admission to trading)
Derogation from the exclusive right on the market
A similar medicinal product may obtain a marketing authorization during the exclusivity period for an orphan medicinal product when:
The holder of the authorization of the original medicine will give his consent;
The authorization holder is unable to deliver a sufficient amount of medicinal product;
The new Applicant will determine that the second product is safer, more effective or exceeds in any clinical way the original product.
Removal of orphan medicinal products from the Register
An orphan drug can be removed from the Registry when it turns out that it no longer meets the criteria set out in Article 3 (frequency of occurrence / profitability of investments / existence of diagnostic, therapeutic and preventive methods).