Amended 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
COMMISSION OF THE EUROPEAN COMMUNITIES Brussels, 15. 04. 1998 COM(1998) 232 final 97/0146 (COD) Amended 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 (presented by the Commission pursuant to Article 189 a (2) of the EC-Treaty) EXPLANATORY MEMORANDUM (1) The initial proposal for a European Parliament and Council decision adopting a programma of Community action on rare diseases was adopted by the Commission on 26May 19971. (2) The Economic and Social Committee2 and the Committee of the Regions3 delivered favourable opinions on this initial Commission proposal. (3) In the light of the European Parliament's opinion after its first reading on 10 March 1998 the Commission, in accordance with article 189a (2) of the treaty, is now submitting an amended proposal for a decision. The text incorporates amendments that clarify and broaden the actions covered by the programme. (4) The European Parliament adopted 28 amendments of which 16 can be accepted by the Commission: 1 in toto, 2 with slight rewording and 13 partially (matched with a modified proposition). (5) The 12 amendments which are not acceptable by the Commission can be grouped as follows : - 6 concern the recitals in the preamble ; - 5 concern the articles of the decision ; - 1 concerns the annex ; The reasons for rejecting these 12 amendments are the following: - 4 amendments are not acceptable for legal reasons (6, 10, 20, 21) ; - 1 amendment falls outside the scops of the programme (18) - 3 amendments are unacceptably limiting (4, 15, 24) - 2 amendments are covered elsewhere (11,13) - 2 amendments are unacceptable due to budgetary limitations (14, 19). 1 OJN°C 0203 of 03. 07. 97 2 Opinion delivered on 29 October 1997, OJ N°C 19 21. 1. 98 3 Opinion delivered on 20 November 1997, OJ M°C 64 27. 2. 98 Amended proposal for a European Parliament and Council Decision adopting a programme of Community action 1999-20G3 on rare diseases in the context of the framework for action in the field of public health l<5c THE EUROPEAN PARLIAMENT AND THE COUNCIL OF THE EUROPEAN UNION Having regard to the Treaty establishing the European Community, and in particular Article 129 thereof; Having regard to the proposal from the Commission, Having regard to the proposal from the Commission,1 Having regard to the opinion of the Economic and Social Committee, Having regard to the opinion of the Economic and Social Committee,2 Having regard Committee of the Regions, to the opinion of the Having regard to the opinion of the Committee of the Regions,3 Acting in accordance with the procedure referred to in Article 189b of the Treaty, 1. Whereas, the very feet of the rareness of the low-prevalence diseases and conditions and the consequent lack of information about them can lead to people affected by these conditions not receiving the health resources and services they need; la) lb) Whereas Community actions should be directed towards the prevention of diseases and whereas action bv the Community can provide unique added yatoe to tackling problems, th? scale of which to individual countries is too small to allow the necessary analysis or proper action: Whereas the number of people affected by fodividval n re diseases may, by definition, be relatively small, but whereas the aggregation of all patients with rare diseases amounts to a substantial number in the population: ^OJÏPC 203 of 03. 07. 97 2'0JN°C 19 of 21. 01. 98 3OJN°C 64 of 27. 02. 98 lc) 2a) as life-threatening Whereas, for the purposes of this programme rare diseases will be defined or chronically debilitating diseases which low prevalence that are of such special combined efforts are needed to address them;. Whereas, in accordance with point (o) of Article 3 of the Treaty, Community action include contribution towards the attainment of a high level of health protection; shall rare diseases are Whereas considered to have little impact on society as a whole due to their low individually» but thsy prevalence pose serious difficult^ for sufferers and their families: Whereas understanding of rare diseases needs to be improved, since thev constitute warning signs from a public health perspective to the extent that problems shown to the affect a population could, as time passed, spread to the wmrnunity as a whole if the incidence of the causes were to become more frequent: section of small 129 expressly Whereas Article provides for Community competence in this field in so far as the Community contributes to it by encouraging co operation between the Member States and, if necessary, lending support to their action; promoting co-ordination of their policies and programmes, and fostering co-operation with third countries international organisations competent in the sphere of public health; whereas Community action should be directed towards the prevention of diseases, and the promotion of health education and information; and 5. Whereas rare diseases have been identified as a priority area for Community the framework for action in the field of public health1 action within Whereas rare diseases have been identified as a priority area for the Community framework for action in ths field of public health1 action within 5a) A programme strategy which of Community action on rare diseases must be undertaken as a part of a also coherent includes provisions for orphan medicinal products and research on rare diseases a should be Community databank compiled updated information on w$ diseases and control Whereas detection and systems should likewise be set up: and whereas store to in its Resolution Whereas (A4- 0311/95) on the Medium-Term Social Action Programme 1995-19972 the Parliament asked the Commission to present, under the proper procedures, for rare the action programme diseases foreseen in the Commission's framework communication on public health; in its Resolution Whereas (A4- 0311/95) on the Medium-Term Social Action Programme 1995-19972 the Parliament asked the Commission to present, under the proper procedures, for rare the action programme diseases foreseen in the Commission's framework communication on public health; Whereas, in accordance with the principle of subsidiarity, action on matters not under the exclusive competence of the Community, such as action on rare diseases, must be undertaken by the Community only if and in so far as, by reason of its scale or effects, it may be better achieved at Community level; l. OOM(93)559fiwJ OJN°C 32 of 03. 02. 96 C0M(93)559 final OJN°C 32 of 05. 02. 96 the co-ordination Whereas, the Community can provide added value to the actions of Member rare diseases, States concerning through of national measures, the dissemination of information and experiences, the joint establishment of priorities, the development of networking as appropriate, selection of European Community-wide projects and the motivation and mobilisation of all involved; 8a) 10a) 9. co-operation with Whereas the international organisations competent in the field of public health and with third countries should be fostered; 10. Whereas, by providing support for acquiring better knowledge and understanding and wider o£ dissemination of information about rare diseases and by developing actions complementary to existing Community programmes and actions, unnecessary while duplication, the programme will contribute to the achievement of the Community objectives set out in Article 129; avoiding in particular health professionals and patients' and support group associations: in Whereas, in accordance with the social and human rights model of disability the supported cornrnunication on Commission ecualitv of opportunity for people with disabilities (CQM(9ffKHPft groups and organisations of persons with rare diseases and their families should be associated with the implementation of the programme, the high level Whereas of technology currently available can contribute to the significantly acquisition of better knowledge and understanding of. and the wider dissemination of information about rare diseases, as stated above, and whereas this technology should be used to enhance the achievemant of the objectives and actions envisaged under the programme: 11. 12. 13. 14. 15. Whereas, in order to increase the value and impact of the programme, a continuous assessment of the actions undertaken should be carried out, with particular their effectiveness and the achievement of the objectives set and, with a view where appropriate, to making the necessary regard to Whereas this programme should be of five-year duration in order to allow sufficient time for actions to be implemented to achieve the objectives set; Whereas the introduction of specific Community arrangements will help to ensure that all Member States are swiftly informed in the event of an the emergency situation, so protection of the population can be ensured; that these Community Whereas arrangements for the rapid exchange of information do not affect the Member States' rights and obligations under Treaties or bilateral and multilateral conventions; between the Counci! and Whereas an agreement on a modus the European viverdi Parliament, the Commission concerning measures for the implementation of acts adopted under the procedure laid down in Article 139b of the Treaty was reached on 20 December 1994. 16. Whereas this decision lays down, a financial framework constituting the principal point of reference, within the meaning of point 1 of the Declaration the European Parliament, the of Council and the Commission of 6 March 1995, the budgetary for authority during the annual budgetary procedure; 17. Whereas the Community's financial perspective is valid up until 1999 and will have to be revised for the period beyond that date; 18. Whereas the financial framework for the last four years of the programme (2000-2003) shall be determined after the establishment of future financial perspectives; the HAVE DECIDED AS FOLLOWS: Article 1 Establishment of the programme 1. 2. A programme of Community action on rare diseases hereinafter referred to as "this programme", is hereby adopted for the period 1 January 1999 to 31 December 2003 in the context of the framework for action in the field of public health. The aim of this programme is to contribute towards ensuring a high level of health protection in relation to rare diseases by providing knowledge about these diseases by promoting and strengthening patient support groups and by fostering the setting up of cluster response teams. The actions to be implemented under this programme and their specific The aim of this programme is to contribute towards ensuring a high level of health protection in relation to rare diseases by providing knowledge about these diseases, especially for patients and health professionals, by promoting and strengthening support groups, and by festering the setting up of cluster response teams. patient objectives are set out in the Annex under the headings: Actions on Community information on rare diseases Actions in support of patient and family support groups Actions on handling rare diseases clusters 3) 1) 2) 3) Article 2 Implementation Actions on identifying hanjBi^B responding rapidly to rare diseases clusters, 1. The shall Commission ensure in close co-operation implementation, with the Member States, of the actions set out in the Annex. 2. The Commission shall co-operate with institutions and organisations active in the field of rare diseases. Article 3 Budget The financial framework for the implementation of the programme for the year 1999 shall be ECU 1,3 Million, in keeping with current financial perspectives. The financial framework for the final four years of the programme (2000-2003) shall be determined the establishment of the future financial perspectives. in detail after The annual appropriations shall be established Budgetary in accordance with the Authority financial perspectives. the by Arfic'e 4 Consistency and complementarity The Commission shall ensure that there is consistency and complementarity between the Community actions to be implemented under this programme and those implemented under other relevant Community programmes and actions. Article 5 Committee 1. 2. In implementing this action plan, the Commission shall be assisted by an hereinafter committee, advisory to as "the CommitteeH, referred consisting of two representatives from each Member State, and chaired by the Commission representative. The representative of the Commission shall submit to the Committee a draft of taken the measures concerning, in particular: to be (a) (b) the criteria, and procedures for selecting and financing projects under this programme; the evaluation procedure. The Committee shall deliver its opinion on the draft, within a the time chairperson may lay down according to the urgency of the matter, if necessary by taking a vote. limit which The opinion shall be recorded in the minutes; in addition, each Member State shall have the right to ask to have its position recorded in the minutes. In implementing this action plan, the Commission shall be assisted by an advisory hereinafter committee, referred to as "the Committee", consisting of two* representatives from each Member State, and chaired by the Commission representative. Conectionofthe text due to a mistake in the English version of the propoul COM(97) 223 final The Commission shall take the utmost account of the opinion delivered by the Committee. It shall inform the Committee on the manner in which its opinion has been taken into account. The representative of the Commission shall keep the Committee regularly informed about or Community Commission proposals initiatives of programmes in other policy areas which are relevant to the achievement of the objectives of this programme. implementation and the Article 6 International co-operation In the course of implementing this programme, co-operation with third international countries and with organisations competent in the field of public health shall be fostered. by the This programme shall be open to participation associated countries of Central Europe (CEC), in accordance with the conditions laid down in the Association Agreements or Additional Protocols related thereto in concerning This Community programme to shall be participation by Cyprus and Malta on the basis of additional appropriations in accordance with the same rules as those applied to the EFTA countries, in accordance with procedures to be agreed with those countries. participation programmes. open Article 7 Monitoring and evaluation !. * the implementation of In this Decision, the Commission shall take the necessary measures to ensure 10 the monitoring and continuous evaluation of the programme taking account of the general and specific objectives referred to in Article 1 and in the Annex. the third year of During this programme, the Commission shall present to the European Parliament and to the Council an evaluation report The Commission shall submit to the European Parliament the Council a final report on completion of this programme. and The Commission shall incorporate into these two reports information on Community financing in the various fields of action and on the other complementarity with actions referred to in Article 4, as well the evaluations. It shall also send them the Economic and Social to Committee and the Committee of the Regions. results of the as Done at Brussels, For the European Parliament For the Council The President The President. ANNEX SPECIFIC OBJECTIVES AND ACTPN8 ACTIONS ON COMMUNITY L INFORMATION ON RARE DISEASES Objective: to provide knowledge about rare diseases especially for patients, and professionals health researchers. Objective : to provide knowledge about rare diseases especially for patients and their relatives, health professionals and researchers. 11 of the causes, standard Encouragement and support for the establishment of a European rare diseases database, with entries listing the disease name, synonyms, a general disorder, description affected symptoms, treatments, population, investigational (when available) and a list of resources that further can be information about the condition. The. information collected fa É&JŒÊiâ European database descril will be made as widely avi possible. treatments contacted for Encouragement and support for the establishment of a European rare diseases database, with entries listing the disease name, synonyms, a general description of the disorder, symptoms, causes, affected population, standard treatments, investigational treatments (when available) and a list of resources that can be contacted for further information about the condition. Promoting access to information and co-ordinating existing information systems and services by supporting the setting up and strengthening of networks at local, regional, national and community level. Organising consensus meetings among to health professionals detection, improve recognition, and prevention of rare diseases. early intervention in order the ACTIONS H. PATIENT AND FAMILY SUPPORT GROUPS IN SUPPORT OF to establish, Objective: and strengthen voluntary organisations involved in indirectly supporting people directly or affected by rare diseases. foster 4. Promoting the establishment of groups of persons with the same rare conditions or those professionally involved in order to disseminate their experience, to facilitate training and to 12 co-ordinate their activities at national and Community level. Promoting the groups' collaboration and networking and the setting up and fostering of umbrella bodies, focusing particularly on efforts to encourage the continuity of work and cross- national co-operation. BOL ACTIONS ON HANDLING RARE DISEASES CLUSTERS Objective, to ensure an efficient handling of the problem of clusters, which is of key rare importance diseases. for Objective : to have in place an effective system of monitoring in order to ensure the rapid identification, assessment and Qf rare disease? and diseases clustery Supporting the monitoring (sentinel) of rare diseases, including birth defects, genetic disorders or diseases of different organ systems and the appropriate low- techniques prevalence diseases, in order to meet on the one hand the demands of detection, treatment and research and on the other hand the demands of relevant statistical monitoring. for the creation of rare Promoting diseases teams and of specialised training courses for those investigating clusters. response Supporting surveillance and early warning systems for clusters. Promoting J^^Û£ïÉQmt£®L„SÉ J]^jm^SXà^s£jas& systems fpr ç^iiMusily_JsïsL siiseises___ai including birth defects, genetic disorders or diseases of different organ systems -and the appropriate techniques low-prevalence diseases, in order to meet on the one hand the demands of detection. , treatment and research and on the other hand the demands of relevant statistical monitoring. for rare diseases and of networking and training for the investigation and handling of clusters. the evaluation, Encouraging the exchange of expertise in assessment, communication and management of clusters of rare diseases that are associated with exogenic causes. 13 ISSN 0254-1475 COM(98) 232 final DOCUMENTS EN 05 15 16 01 Catalogue number : CB-CO-98-245-EN-C ISBN 92-78-35074-5 Office for Officia! Publications of the European Communities L - 2 9 85 ? iHXC!H'"K-UriZ