New Guidelines On The Application Of Certain Aspects Of The Orphan Medicines Regulation Published, European Commission

Main Category: Regulatory Affairs / Drug Approvals
Article Date: 03 Oct 2008 - 4:00 PST

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The European Commission has published two guidelines concerning the application of Articles 8(1), (2) and (3) of Regulation (EC) No 141/2000 on orphan medicinal products.

These relate to:

-- the possibility for Member States to inform the European Medicines Agency that the criteria on which market exclusivity was granted may no longer be met (potentially resulting in the market exclusivity period for the medicine concerned being shortened); and

-- assessment of the similarity of medicinal products, which is a requirement for accepting marketing authorisations when orphan medicinal products have been authorised for similar indications.

The guidelines are available on the European Commission website as follows:

-- Review of the period of market exclusivity of orphan medicinal products - C(2008) 4051 final
-- Assessing similarity of medicinal products versus authorised medicinal products - C(2008) 4077 final

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

About the European Medicines Agency (EMEA)

The European Medicines Agency (EMEA) is a decentralised body of the European Union with headquarters in London. Its main responsibility is the protection and promotion of public and animal health, through the evaluation and supervision of medicines for human and veterinary use.

-- The EMEA is responsible for the scientific evaluation of applications for European marketing authorisation for medicinal products (centralised procedure). Under the centralised procedure, companies submit a single marketing authorisation application to the EMEA. Once granted by the European Commission, a centralised (or 'Community') marketing authorisation is valid in all European Union (EU) and EEAEFTA states (Iceland, Liechtenstein and Norway).

-- All medicinal products for human and animal use derived from biotechnology and other hightechnology processes must be approved via the centralised procedure. The same applies to all human medicines intended for the treatment of HIV/AIDS, cancer, diabetes, neurodegenerative diseases, autoimmune and other immune dysfunctions, and viral diseases, as well as to all designated orphan medicines intended for the treatment of rare diseases. Similarly, all veterinary medicines intended for use as performance enhancers in order to promote the growth of treated animals or to increase yields from treated animals have to go through the centralised procedure.

-- For medicinal products that do not fall under any of the abovementioned categories, companies can submit an application for a centralised marketing authorisation to the EMEA, provided the medicinal product constitutes a significant therapeutic, scientific or technical innovation, or the product is in any other respect in the interest of patient or animal health.

-- The safety of medicines is monitored constantly by the Agency through a pharmacovigilance network. The EMEA takes appropriate actions if adverse drug reaction reports suggest changes to the benefitrisk balance of a medicinal product. For veterinary medicinal products, the Agency has the responsibility to establish safe limits for medicinal residues in food of animal origin.

-- The Agency also plays a role in stimulating innovation and research in the pharmaceutical sector. The EMEA gives scientific advice and protocol assistance to companies for the development of new medicinal products. It publishes guidelines on quality, safety and efficacy testing requirements. A dedicated office established in 2005 provides special assistance to small and mediumsized enterprises (SMEs).

-- Five scientific committees, composed of members of all EU and EEAEFTA states, conduct the main scientific work of the Agency: the Committee for Medicinal Products for Human Use (CHMP), the Committee for Medicinal Products for Veterinary Use (CVMP), the Committee for Orphan Medicinal Products (COMP), the Committee on Herbal Medicinal Products (HMPC) and the Paediatric Committee (PDCO). A sixth scientific committee - the Committee for Advanced Therapies (CAT) - will be established at the end of 2008.

-- The Agency brings together the scientific resources of some 40 national competent authorities in 30 EU and EEAEFTA countries in a network of over 4,000 European experts. It contributes to the European Union's international activities through its work with the European Pharmacopoeia, the World Health Organization, and the ICH and VICH trilateral (EU, Japan and US) conferences on harmonisation, among other international organisations and initiatives.

-- The EMEA is headed by the Executive Director and has a secretariat of approximately 470 staff members in 2008. The Management Board is the supervisory body of the EMEA, responsible, in particular, for budgetary matters.

-- The Agency is also involved in referral procedures relating to medicinal products that are approved or under consideration by Member States in noncentralised authorisation procedures.