WRITTEN QUESTION P-2510/01 by Sebastiano Musumeci (UEN) to the Commission. Support for children suffering from a rare disease.
Date: 2001-09-07
Institutions: European Parliament, Provisional data
Subjects: child, market approval, medical research, medicinal product, pharmaceutical legislation, public health, research and development

C 115 E/102

Official Journal of the European Communities

EN

16.5.2002

(2002/C 115 E/107)

WRITTEN QUESTION P-2510/01

by Sebastiano Musumeci (UEN) to the Commission

Subject: Support for children suffering from a rare disease

(7 September 2001)

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).

16.5.2002

EN

Official Journal of the European Communities

C 115 E/103

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
(contact:
for Rare Disorders) which
guylaine.arduin@libertysurf.fr).

relations with

covers more

specifically

patients

(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.

(2002/C 115 E/108)

WRITTEN QUESTION P-2512/01

by Lousewies van der Laan (ELDR) to the Commission

(7 September 2001)

Subject: Foot-and-mouth disease: review of EU non-vaccination policy

In response to question E-1195/01 (1) in March of this year, the Commission states that it ‘fully supports a
review of the current approach towards vaccination’, but it points towards the lack of political support for
such a review in both the Council and the European Parliament. The Commission states that ‘this lack of
support reflects the well identified limitations of such a policy which include, but are not limited to, trade
implications.’

In April of this year, however, the European Parliament expressed its clear political support for a review in
resolution PE 303.051 (European Parliament resolution on the statement of the European Commission on
foot-and-mouth disease in the European Union), calling ‘on the Commission to review immediately the
basic non-vaccination policy of the European Union’.

At this time, the first week of September 2001, there is clear evidence that the FMD crisis is not yet fully
contained. This fact is especially worrying, since the disease is much harder to contain in winter than in
summer. In addition there is a risk that FMD is becoming endemic among wild animals in the UK.

Considering that the calculations on which the non-vaccination policy was based in 1991 took into
account only one outbreak of FMD every 10 years, does the Commission agree that the very real chance of
having another consecutive winter of FMD crises should lead to an urgent reconsideration of the non-
vaccination policy before the winter breaks?

The Commission correctly states that trade implications are at the root of the non-vaccination policy.
Would the Commission agree that the FMD crisis last winter taught us that the negative impact on many
sectors of the economy far outweighs the impact that generalised prophylactic vaccination would have on
EU external trade? Does the Commission agree that the economic impact of non-vaccination depends on
the frequency and intensity of outbreaks? If so, where is the turning point from positive to negative
economic impact?

What steps has the Commission taken so far to ensure that if a review of the non-vaccination policy
should lead to generalised prophylactic vaccination, sufficient vaccine would be available at short notice
should another FMD crisis break?

(1) OJ C 364 E, 20.12.2001, p. 58.