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` () (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 ().

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

1.3. To address the problem of rare diseases the Commission proposes a number of specific actions under three headings: i) actions to provide better 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).

1.4. 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 Development (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

2.1. The Committee welcomes the action programme 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 trends), it is important to ensure the programme's continuity - also beyond the year 2003.

2.3. Whilst the Committee agrees with the Commission 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.

3.1.1. The Commission should ensure that the definition 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 distinction between the 'networks` referred to in the second action under the first objective and the 'groups' collaboration and networking` mentioned in the second action of the second objective should be clarified.

3.3.2. The Committee feels that the Internet ('newsgroups` and 'chatgroups`) can also play a very useful role in fostering contacts between patients and professionals, 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).

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 complementarity between all the Community initiatives relating 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, development 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

() OJ C 203, 3. 7. 1997, p. 6.

() COM(93) 559 final.

() See its opinion of 6 July 1994 - OJ C 388, 31. 12. 1994.

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

() See its Resolution of 20 December 1995 - OJ C 350, 30. 12. 1995.