www.eurordis.org

Eurordis - Rare Diseases Europewww.eurordis.org - eurordis@eurordis.org

Sources of informationEurordis

www.eurordis.org – Monthly electronic newsletterFull list of designated orphan drugs available at:

http://ec.europa.eu/enterprise/pharmaceuticals/register/alforphreg.htm

European Medicines Agency (EMEA)http://emea.europa.eu

www.eurordis.org

Rare Diseases Europe

WHAT IS AN ORPHAN DRUG?

April2007

• 450 orphan drugs designated since 2000• 37 orphan drugs with marketing authorisation since 2000

• 2 million EU citizens potentially benefiting from these drugs

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Pharmaceutical companies are unwilling to develop suchdrugs under normal market conditions, as the cost ofbringing them to the market would not be recovered bythe expected sales of drugs without providing incentives.

WHAT IS AN ORPHAN DRUG?

INCENTIVES PROVIDED BY THE EU REGULATION

• Market exclusivity in the EUSimilar competitive products cannot be placed on the marketfor 10 years after the granting of marketing authorisationand 12 years if paediatric studies are performed.

• Protocol assistanceScientific advice is provided to pharmaceutical companies bythe EMEA (European Medicines Agency, based in London) tooptimise drug clinical development meeting Europeanregulatory requirements.

• Access to the centralised procedureOrphan drugs have direct access to the EMEA centralisedprocedure for the application for marketing authorisation.

• Fee reductionsFee waiver for orphan designation and reduced fees formarketing authorisation, inspections, variations and protocolassistance.

• EU-funded researchPharmaceutical companies developing orphan drugs may beeligible for grants from EU and Member State programmesand initiatives supporting research and development,including the Community framework programmes.

ORPHAN DESIGNATION

Orphan designation may be granted at any stage of drugdevelopment, provided proper scientific justification of theintended use is submitted. Orphan designation is given by theCommittee for Orphan Medicinal Products (COMP) at the EMEA(European Medicines Agency).

Orphan designation is not an endorsement for the use of thedrug for the designated condition. An application for marketingauthorisation must first be submitted by the pharmaceuticalcompany. Efficacy, safety and quality criteria need to be satisfiedfor the granting of a marketing authorisation.

ORPHAN DRUG LEGISLATION DEVELOPMENT

1983 – First Orphan Drug Act in the United States1990s – Orphan Drug Legislation adopted by Singapore

(1991) Japan (1993) and Australia (1997)1999 – Adoption by the European Parliament of the

Regulation on Orphan Medicinal Products2000 – Creation of the Committee for Orphan Medicinal

Products (COMP) at the EMEA (European Medicines Agency)

Orphan drugs are medicinal products intended for the diagnosis, prevention or treatment of rare diseases, which are diseases affecting less than 1 in 2,000 persons or a maximum of 250,000 citizens in the European Union.

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