WRITTEN QUESTION P-2510/01

by Sebastiano Musumeci (UEN) to the Commission

(7 September 2001)

Subject: Support for children suffering from a rare disease

Two children belonging to a family in Catania are suffering from an extremely rare disease (there are only 10 recorded cases in the whole world) aromatic amino acid decarboxylasis. The disease prevents the body from producing neurotransmitters, so that arterial pressure becomes uncontrollable and prevents movement.

The drug used to treat this disease is Dopaflex, which cannot be found in Italy. The children's parents therefore have to buy it in German pharmacies, which entails inconvenience and sacrifices, not least from the financial point of view, which are becoming less and less sustainable.

How can the Commission ensure, in desperate cases such as this, the right to obtain a drug free of charge and hence the right to life for two children in such a plight?

What steps has the Commission taken or does it intend to take in the field of scientific research to overcome rare diseases such as this and thus restore hope to families obliged to resign themselves to their fate?

Answer given by Mr Byrne on behalf of the Commission

(17 October 2001)

The conditions under which patients may obtain medicines at the present time fall under the responsibility of the Member States and therefore depend on the provisions governing their health care systems.

At the same time, the Community strategy in the domain of rare diseases has been developing in several policy areas:

- In the area of pharmaceutical policy, the Parliament and Council Regulation (EC) No 141/2000 of 16 December 1999 on orphan medicinal products(1) is now part of the acquis communautaire. This regulation aims to support the development of medicinal products to treat rare diseases by offering incentives for companies developing such treatments. One of these incentives is an automatic access to the centralised marketing authorisation procedure which ensures that a medicinal product, once authorised may immediately be placed on the market in all Member States. In this way, new medicinal products to treat rare diseases will be made available throughout the Community. Pricing and social security related activities, however, remain national competences and are outside the scope of the Orphan Medicinal Product Regulation. The availability and price of a particular medicinal product in a Member State is thus directly linked to the health care provisions of the Member State concerned and not to Community legislation.

- In the area of research on rare diseases conducted within the Community Framework Programmes for Research and Technological Development, the Commission is supporting research into rare diseases through its Framework Programmes for Research and Technological Development (RTD); in the Biomed 2 programme (1994-1998) 8,65 million have been spent for 23 projects, and the Fifth Framework Programme 31,6 million have been committed for 22 projects since its start in 1999. The Commission proposal for the next Framework Programme (2002-2006) includes research into rare diseases in one of its priority thematic programmes. Details on these research projects can be found on the following addresses: http://dbs.cordis.lu/fep/LIFE/LIFE_PROJl_search.html and http://www.cordis.lu/life/src/previous.htm.

- In the area of Public Health, the programme of Community action for 1999-2003 on rare diseases represents an important step in the consolidation of the Community policy for this area of public health. This programme was adopted by Decision of the Parliament and the Council of 29 April 1999(2).

Rare diseases are considered to have little impact on society as a whole owing to their low prevalence individually; low prevalence can be understood as meaning prevalence which is generally recognised as less than five per 10 000 in the Community. However, they pose serious difficulties for sufferers and their families. Understanding of rare diseases needs to be improved. The programme of Community action promotes the development of, and access to, a coherent and complementary European information network on rare diseases, using the databases and networking between groups of persons affected by the same rare conditions.

We suggest you consult the existing databases, Orphanet (European Database on Rare Diseases and Orphan Drugs (website: http://orphanet.infobiogen.fr/) and/or Eurordis (European Organisation for Rare Disorders) which covers more specifically relations with patients (contact: guylaine.arduin@libertysurf.fr).

(1) OJ L 18, 22.1.2000.

(2) Decision No 1295/1999/EC of the Parliament and of the Council of 29 April 1999 adopting a programme of Community action on rare diseases within the framework for action in the field of public health (1999 to 2003), OJ L 155, 22.6.1999.