What are the "Orphan Drugs"

The "Orphan Drugs" are used for the diagnosis, prevention or cure of diseases, which endanger human life or are extremely severe, they are rare and their occurrence in the European Union is lower than five (5) cases per 100.000 persons. The pharmaceutical companies are not willing to produce such drugs bearing in mind the demand, since their production and marketing cost could not be covered by their expected sales without extra motives.

How the development of such drugs can be encouraged

The degree for providing motives to sponsors/ pharmaceutical industries for the development of orphan drugs has passed in the European Union in 2000. The products, which benefit by these motives outlined further below, are characterised as "Orphan Drugs".

What exactly is the EMEA (European Medicines Agency)

This organisation is responsible via its committee for the Orphan Drugs (COMP), for the evaluation of the characterisation petitions, submitted by individuals or companies which desire to produce drugs for rare diseases, i.e. "orphan" drugs. The Organisation gives also advice regarding the development of orphan drugs (contributing in the formulation of the protocol).

Which are the motives

• Exclusive rights.
  The Orphan Drugs are entitled exclusive marketing for a period of 10 years after the circulation license is granted. During this period, similar competitive drugs are not allowed to circulate in the market, under ordinary conditions.
  
  
• Contribution in the formulation of the protocol.
  The Organisation provides scientific advice for the .....development, as well as guidance for the completion of the file, so that the petitioner for the circulation license of an orphan drug can maximise the possibility to get it.
  
  
• Access to the central procedure.
  The orphan drugs have direct access to the central procedure of the Organisation with regard to the petition for acquiring the circulation license.
  
  
• Exemption from duties.
  A special sum of the European Committee, which is approved annually by the European Parliament, will be used by the Organisation for the exemption from various duties. The reduction of duties regarding all kinds of central procedures, including the petition duties for the acquisition of the circulation license and the fees for the formulation of the protocol, will also be considered.
  
  
• Research financed by the EU.
  The organisations, which develop orphan drugs, can be subsidized from programmes and initiations for research and development of the EU and the states-members, including the community programmes-frames.
  
  

What the characterisation of a drug as "orphan" entails

Characterising a drug as “orphan” entails that this drug is attached to the list of orphan drugs on the basis of the criteria set by the regulation (EC) No. 141/2000, a fact that allows access to the motives.

The classification of a drug as "orphan" does not mean that it can be used for the treatment of a specific disease. The drug must also fulfill the criteria, which, under a separate procedure, will justify granting the circulation license. The quality, safety and effectiveness of the drug to be used for a specific disease can only be evaluated after the petition for acquiring the circulation license, has been filed

Useful information sources

• Directives, press announcements of the COMP and public opinion summaries for each product
• The Community registrar for orphan drugs and the catalogue of the criteria set by the Community and the state-members for the orphan drugs.
• Fifth programme-frame
• Community programme of action for the rare diseases (1999-2003)

The procedure for the characterisation of orphan drugs at a glance

1. Notification to the EMEA of the intention of the sponsor to file a petition
2. Advice contribution by the EMEA before the filing of the petition
3. Filing the petition. Confirmation by the EMEA (1st day)
4. Valuation by the COMP/EMEA
5. Approval of the COMP opinion (till the 90th day)
6. Transmission of the opinion to the European Committee
7. Decision making by the Committee (within 30 days)
8. Publication in the community register and publication of the summary of the opinion

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