Orphan Medicines Regulation

The growing number of rare diseases awaiting treatment are an important public health issue. Often the scarcity of incentives for drug manufacturers and the lack of documentation supporting the applications limit the number of new orphan drugs.

Orphan medcines legislation

Orphan medicines legislation aims at providing incentives for pharmaceutical companies to develop and market medicinal products to treat rare diseases.

A ground-breaking precedent was set by the United States in 1983 when the Orphan Drug Act came into effect. In the 1990s, first Japan, then Australia adopted orphan medicineslegislations.

The revolution reached Europe in 1999, with the EU Regulation on Orphan Medicinal Products (Regulation EC n° 141/2000), adopted by the European Parliament on 16 December, 1999 and published in January 2000. EURORDIS, representing the rare disease patient community in Europe, actively advocated for the adoption of this legislation.

Following the adoption of the regulation, an EU committee named the Committee for Orphan Medicinal Products (COMP) was created within the European Medicines Agency to review the applications for medicinal products seeking an "orphan" designation. 

History of Orphan medicines legislations

1983 – First Orphan Drug Act in the United States. 1990s – Orphan Drug Legislation adopted by Singapore (1991) Japan (1993) and Australia (1997). 1999 – Regulation on Orphan Medicinal Products adopted by the European Parliament . 2000 – Creation of the Committee for Orphan Medicinal Products (COMP) at the European Medicines Agency (EMA) in London.

Incentives provided by the EU Regulation

Market exclusivity in the EU

An orphan product receives a marketing authorisation from the EMA (European Medicines Agency). Competitive similar products cannot be placed on the market for 10 years after receiving marketing authorisation. In the case of paediatric drugs the monopoly is extended to 12 years.

Protocol assistance

The EMA provides protocol assistance (scientific advice for orphan products) in the form of scientific advice about the various tests and clinical trials necessary for drug development to pharmaceutical companies. This information is delivered at no cost or a reduced fee to optimise the development of orphan drugs and to ensure better compliance with the European regulatory requirements.

Fee reductions

During the approval process fee waivers for orphan designation and reduced fees are granted. These apply to marketing authorisation, inspections, variations and protocol assistance.

EU-funded research

Pharmaceutical companies developing orphan drugs may be eligible for specific grants from EU and Member State programmes as well as initiatives supporting research and development. This includes the Community framework programmes.

Orphan medicines centralised procedure at European level

The orphan medicines centralised procedure in Europe, from a request for orphan designation to marketing authorisation, is displayed in this document below.

Orphan drug Regulatory Process in EU


Page created: 19/08/2009
Page last updated: 07/11/2014
The voice of rare disease patients in EuropeEURORDIS The international voice of people living with rare diseases, Rare Diseases International is a EURORDIS initiativeRare Disease International Bringing together patients, families and experts to share experiences in a moderated multilanguage forum, RareConnect is a EURORDIS initiative RareConnect An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiativeRare Disease Day Join the largest gathering of rare disease stakeholders across Europe, at the biennial European Conference on Rare Diseases and Orphan Products. ECRD is a EURORDIS initiativeEuropean Conference on Rare Diseases