Opinion of the Economic and Social Committee on the 'Proposal for a European Parliament and Council Decision adopting a programme of Community action 1999-2003 on rare diseases in the context of the framework for action in the field of public health'
Date: 1997-10-29
Institutions: European Economic and Social Committee
Subjects: EU programme, action programme, health policy, illness, public health

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mission on both the criteria and procedures for selecting
and financing projects under the programme, and the
evaluation procedure.

3.4.
Article 7: Monitoring and evaluation. The Com-
mittee is pleased to note that it will receive copies of the
evaluation and implementation reports.

Brussels, 29 October 1997.

The President

of the Economic and Social Committee

Tom JENKINS

Opinion of the Economic and Social Committee on the ‘Proposal for a European Parliament
and Council Decision adopting a programme of Community action 1999-2003 on rare diseases
in the context of the framework for action in the field of public health’ (1)

(98/C 19/02)

On 27 June 1997 the Council decided to consult the Economic and Social Committee, under
Article 129 of the Treaty establishing the European Community, on the above-mentioned
proposal.

The Section for Protection of the Environment, Public Health and Consumer Affairs, which
was responsible for preparing the Committee’s work on the subject, adopted its opinion on
7 October 1997. The rapporteur was Mr Fuchs; co-rapporteurs were Mr Lemmetty and
Mr Linssen.

At its 349th plenary session (meeting of 29 October 1997), the Economic and Social Committee
adopted the following opinion by 101 votes to three, with one abstention.

1.

Introduction

1.1.
The proposed five-year action programme (1999-
2003), which is based on Article 129 of the Treaty and
which is accompanied by a explanatory communication,
seeks to address the problem of rare diseases in the
Community by complementing ongoing work on this
subject in the Member States. The problem of rare
diseases, which, in contrast to the United States, has not
been dealt with systematically in most of the Member
States to date, was identified as one of the eight priority
areas for Community action in the 1993 Commission
communication on the framework for action in the field
of public health (2).

1.2.
In response to this communication both the
Economic and Social Committee (3) and the European

Parliament (4) supported the idea of an action programme
on rare diseases. Furthermore, the Council invited the
Commission to take stock of existing knowledge and
experience regarding rare diseases and to examine the
situation of orphan drugs in the Community (5).

1.3.
To address the problem of rare diseases the
Commission proposes a number of specific actions
i) actions to provide better
under three headings:
information on the different aspects of rare diseases (e.g.
the establishment of a European rare diseases database);
ii) actions in support of patient and family support
groups (e.g. the establishment of such groups and their
collaboration); and iii) actions on handling rare diseases
clusters (e.g. the monitoring of rare diseases and the
creation of rare diseases cluster response teams).

(1) OJ C 203, 3. 7. 1997, p. 6.
(2) COM(93) 559 final.
(3) See its opinion of 6 July 1994 — OJ C 388, 31. 12. 1994.

(4) See its Resolution A4-0311/95 on the Medium-Term Social
Action Programme 1995-1997 — OJ C 32, 5. 2. 1996.
(5) See its Resolution of 20 December 1995 — OJ C 350,

30. 12. 1995.

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1.4.
Two methods will be employed to implement
these actions: i) support for projects carried out in
Member States and at Community level and ii) support
for additional specific activities to achieve the objectives
of the programme. In these tasks the Commission will
be assisted by an Advisory Committee consisting of
representatives from the Member States.

1.5.
As for the two other public health related action
programmes recently proposed by the Commission
(‘Injury prevention’ and ‘Pollution-related diseases’) the
Community contribution foreseen for (co-)financing the
actions during the first year (1999) is ECU 1,3 million
whereas the financial framework for the final four years
as well as the annual allocations will be determined after
the establishment of the future financial perspectives.

1.6.
Parallel to the proposed action programme,
research activities on the development of orphan drugs
and on rare diseases are supported under the Fourth
Framework Programme on Research & Technical Devel-
opment (1994-1998). In addition, the Commission is
currently preparing a regulation with the aim of laying
down a procedure for the designation of medicinal
products as orphan medicinal products and to provide
incentives for the research, development and placing on
the market of such products.

2. General comments

The Committee welcomes the action programme
2.1.
as a step towards a more systematic approach to the
problem of rare diseases in the Community. It has
however serious doubts about whether the objectives of
the programme can be achieved with the envisaged
budgetary provisions.

2.2.
To optimize the effectiveness of many of the
proposed actions (e.g. the monitoring of long-term
it is important to ensure the programme’s
trends),
continuity — also beyond the year 2003.

2.3. Whilst the Committee agrees with the Com-
mission that Community initiatives on rare diseases can
bring important benefits, it would at the same time
stress the need for additional action and coordination
at Member State level.

3. Specific comments

3.1.
The Committee attaches great importance to the
definition of ‘rare diseases’. It recognizes the difficulties
of finding an appropriate definition and agrees that both
quantitative and qualitative aspects should be taken into
account. The Committee endorses the disease incidence

proposed by the Commission (‘diseases with a generally-
accepted prevalence in the total Community population
of less than five per 10 000’), even though it is aware
that in the United States a less restrictive disease
prevalence criterion is used.

The Commission should ensure that the defi-
3.1.1.
nition used in the action programme is consistent with
the one used in the planned regulation on orphan
medicinal products.

3.2.
The Committee has serious misgivings about the
budgetary provisions contained in the programme.
Firstly, the Community contribution proposed for the
implementation of the programme during the first year
is so small that only some of the proposed actions can
be supported, and even then at very modest levels.
Secondly, the fact that funding for the final four years,
and thus the continuity of the programme, is in no way
ensured should also be seen as a major weakness.

3.2.1.
Thus, for the programme to be successful
the ESC considers it essential that the Community
contribution for 1999 is substantially increased and that
the budgetary authorities then ensure its continuity at
that increased funding level after 1999.

3.3.
The Committee is broadly in agreement with the
choice of the three specific objectives of the programme
and the concrete actions proposed under each of them.

3.3.1. With respect to the actions on Community
information the Committee would suggest that it should
be possible to consult the proposed European rare
diseases database via the Internet. Moreover, the distinc-
tion between the ‘networks’ referred to in the second
action under the first objective and the ‘groups’ collabor-
ation and networking’ mentioned in the second action
of the second objective should be clarified.

3.3.2.
The Committee feels that the Internet (‘news-
groups’ and ‘chatgroups’) can also play a very useful
role in fostering contacts between patients and pro-
fessionals, as in most cases people suffering from rare
diseases live far apart from one another.

3.3.3. With regard to the handling of rare disease
clusters, the Committee thinks that before task forces
are set up an effort should first be made to reach general
agreement on how to respond when such clusters occur.
The Committee would point out that practical guidelines
have been drawn up by, for example, the Centre for
Disease Control. Such guidelines must also be established
separately for different diseases as the way they need to
be handled is clearly very different (e.g. search for
infection carriers, causes of cancer, causes for frequent
occurrence of genetic diseases).

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3.3.3.1.
In this connection the Committee would
further stress the importance of monitoring systems for
rare diseases in order to identify trends over time and
regional clusters. As such trends do not generally emerge
until after 10 years, the Committee thinks that the
continuity of the proposed action programme should be
ensured, even after 2003.

3.4.
It goes without saying that the Commission
should ensure that there is consistency and comp-
lementarity between all the Community initiatives relat-

ing to rare diseases. This particularly applies to the
proposed action programme, current and future research
activities on rare disease and the envisaged regulation
on orphan medicinal products (where research, develop-
ment and marketing of such medications must be closely
coordinated).

3.5.
The Committee is pleased to note that, as part
of the programme, the Commission envisages fostering
cooperation with relevant international organizations
and third countries; these should include Japan and the
United States.

Brussels, 29 October 1997.

The President

of the Economic and Social Committee

Tom JENKINS

Opinion of the Economic and Social Committee on the ‘Proposal for a European Parliament
and Council decision adopting a programme of Community action 1999-2003 on pollution-
related diseases in the context of the framework for action in the field of public health’ (1)

(98/C 19/03)

On 27 June 1997 the Council decided to consult the Economic and Social Committee, under
Article 129 of the Treaty establishing the European Community, on the above-mentioned
proposal.

The Section for Protection of the Environment, Public Health and Consumer Affairs, which
was responsible for preparing the Committee’s work on the subject, adopted its opinion on
7 October 1997. The rapporteur was Mr Lemmetty; co-rapporteurs: Mr Fuchs and Mr Linssen.

At its 349th plenary session (meeting of 29 October 1997), the Economic and Social Committee
adopted the following opinion by 106 votes in favour and 3 abstentions.

1.

Introduction

The Commission has presented a communication
1.1.
concerning a programme of Community action on
pollution-related diseases in the context of the frame-
work for action in the field of public health. The
communication is accompanied by a proposal for a
European Parliament and Council Decision adopting a
programme of Community action on pollution-related
diseases to be carried out within this framework in the
period 1999-2003.

1.2.
The action programme is based on Articles 3(o)
and 129 of the Treaty establishing the European Com-
munity which set health protection as a priority objec-

tive. In its communication of 24 November 1993 on the
framework for action in the field of public health (2), the
Commission defined a framework for future action at
Community level in order to attain these objectives.

1.2.1.
Following on the Communication, the Com-
mission has also recently adopted action programmes
on injury prevention and rare diseases.

1.3.
The Economic and Social Committee has empha-
sized the link between environmental pollution and
health on a number of occasions in the past. In its

(1) OJ C 214, 16. 7. 1997, p. 7.

(2) COM(93) 559 final, 24. 11. 1993.