Affiliations: Committee for Orphan Medicinal Products, European
Agency for the Evaluation of Medicinal Products, London, UK | Clinical Pharmacology and Therapeutics Department,
Sant Pau Hospital, Autonomous University of Barcelona, Spain
Note: [] a
Abstract: The European Parliament and the Council have recently adopted a
legal framework which aims to foster research and promote the development of
new therapies for the treatment of rare diseases. It has been well recognised
that the US Orphan Drug Act has been a major breakthrough in promoting product
development for patients suffering from rare disorders and now after 17 years
of experience it is regarded as an essential tool to promote and preserve
public health. Several sources indicate that almost 8000 different severe and
incapacitating conditions may exist affecting as many as 25--30 million
European citizens. Of these disorders about 4000 have a genetic origin
affecting primarily neonates and children. The European Agency for the
Evaluation of Medicinal Products (EMEA) is responsible for putting in place a
community procedure for the evaluation of the applications orphan medial
product designation through the creation of a new scientific body, the
Committee for Orphan Medicinal Products (COMP). This paper reviews the role and
the objectives of the COMP and the EMEA in the development of medicinal
products for rare disorders and the key elements for submitting an application
for the designation which if successful leads to several incentives.