European Medicines Agency - Human Medicines - Orphan medicinal products - Introduction

Human medicines - Orphan medicinal products
Introduction
Medicines for rare diseases — so-called 'orphan' medicinal products — are intended for the diagnosis, prevention or treatment of life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union, or are medicines which, for economic reasons, would be unlikely to be developed without incentives.

The aim of the EU legislative framework1 for orphan medicines is to stimulate research and development of medicinal products for rare diseases by providing incentives to the pharmaceutical industry. This initiative helps to give patients suffering from rare diseases access to the same quality of treatment as other patients.

Applications for designation of orphan medicines are reviewed by the EMEA through the Committee for Orphan Medicinal Products (COMP).

The orphan medicinal products designated to date cover a wide variety of rare diseases, including genetic diseases and rare cancers, for most of which there are either no or only unsatisfactory treatment options. A large number of these diseases affect children and newborn babies.

This section of the EMEA website provides information about the incentives available for the development of orphan medicines in the EU, plus guidance and procedural information on applying for orphan designation of a medicinal product. It also provides information about the EMEA's scientific committee responsible for establishing opinions on orphan designation (the COMP), and lists summaries of the opinions adopted by this committee.

See also:

50th orphan medicine receives positive opinion - List of orphan-designated authorised medicines (as of 6 November 2008)
Orphan drugs and rare diseases at a glance
Orphan medicinal product designation (Quick-look leaflet in English)
1 The legislative framework (Regulation (EC) No 141/2000 adopted on 16 December 1999 (OJ No L 19, 22.1.2000) and Commission Regulation (EC) No 847/2000 of 27 April 2000 (OJ No L103/5, 28.4.2000)) introducing an EU public health policy on orphan medicinal products came into effect in the European Union in April 2000.


open here please:
European Medicines Agency - Human Medicines - Orphan medicinal products - Introduction