What is an orphan drug?
So-called “orphan” drugs are intended to treat diseases which are so rare that sponsors are reluctant to develop them under usual marketing conditions, as the small size of the market they represent would not allow sponsors to recoup the capital invested on the research and development of the product.
Patients with rare diseases cannot stay on the sidelines of the progress made by science and therapeutics, they have the same rights to treatment as any other patient. In order to stimulate research and development in the sector of orphan drugs, public authorities have implemented incentives for health and biotechnology industries. This started as early as 1983 in the United States with the adoption of the Orphan Drug Act, followed by Japan and in Australia in 1993 and 1997; Europe followed in 1999 by implementing a common policy on orphan drugs in its Member States.
The European Orphan Drug regulation
On 16 December 1999, the European Parliament and European Council adopted Regulation (EC) N° 141/2000 on orphan medicinal products.
In addition to this, the European Commission adopted Regulation (EC) N° 847/2000 on 27 April 2000, establishing the provisions for the application of criteria for orphan designation and defining the concepts of ’similar medicinal product’ and ’clinical superiority’.
According to European regulation n° 141/2000, only drugs for human use can be designated as “orphan drugs”. Therefore it does not concern veterinary medicines, medical devices, nutritional supplements and dietary products.
Drugs designated as orphan are entered in the Community register for Orphan Medicinal Products.
Availability of orphan drugs in Europe
The granting of marketing authorisation for a drug (list of marketed orphan drugs in Europe) does not mean the drug is available in all countries of the European Union. The marketing authorisation holder must decide beforehand on its commercialisation status within every country and the drug will then go through the necessary procedures in each country in order to establish reimbursement conditions, and usually its price.
Despite joint efforts, the heterogeneity of approaches between countries makes patients’ access to orphan drugs more complex.
Policies in favour of orphan drugs in Europe
A description of European policy in the field of rare diseases and orphan drugs can be found on the European Commission’s website:
A description of national initiatives throughout Europe and incentives put in place by the European Commission and by European Union, and surrounding, countries can be found on the site of the European Committee of Experts on Rare Diseases:
List of orphan drugs
Orphanet maintains a list of orphan drugs available in Europe which is updated monthly.
Orphanet also provides access to information on orphan drugs in development (orphan designation and clinical trials) or already on the market, by category of disease, type of product, name of substance, name of sponsor and country.