CELEX: 51998PC0232
Language: en
Date: 1998-04-15
Title: 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 thefieldof public health
        (presented by the Commission pursuant to Article 189 a (2)
                            of the EC-Treaty)
 ---pagebreak---  ---pagebreak---                               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
 ---pagebreak--- 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
 ---pagebreak---  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                Having regard to the proposal from
Commission,                                           the Commission,1
Having regard to the opinion of the Economic         Having regard to the opinion of the
and Social Committee,                                Economic and Social Committee,2
Having regard to the opinion of the                  Having regard to the opinion of the
Committee of the Regions,                            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) 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:
                                                lb) Whereas the number of people
                                                    affected by fodividval n r e 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
                                                    3
                                                      OJN°C 64 of 27.02.98
 ---pagebreak---                                            lc) Whereas       rare    diseases     are
                                               considered to have little impact on
                                                society as a whole due to their low
                                                prevalence individually» but thsy
                                                pose serious difficult^ for sufferers
                                                and their families:
 Whereas, for the purposes of this
 programme rare diseases will be
 defined     as    life-threatening    or
 chronically debilitating diseases which
 are of such low prevalence that
 special combined efforts are needed to
 address them;.
                                          2a)
                                               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
                                               affect a small section of the
                                               population could, as time passed,
                                               spread to the wmrnunity as a whole
                                               if the incidence of the causes were
                                               to become more frequent:
Whereas, in accordance with point (o)
of Article 3 of the Treaty, Community
action     shall include contribution
towards the attainment of a high level
of health protection;
Whereas Article 129 expressly
provides for Community competence
in thisfieldin 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        and        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;
 ---pagebreak---    5.      Whereas rare diseases have been
           identified as a priority area for           Whereas rare diseases have been
           Community action within the                 identified as a priority area for
           framework for action in the field of        Community action within the
                                                       framework for action in ths field of
           public health1
                                                       public health1
                                                  5a)
                                                                      A     programme    of
                                                       Community action on rare diseases
                                                       must be undertaken as a part of a
                                                       coherent strategy which also
                                                       includes provisions for orphan
                                                      medicinal products and research on
                                                      rare diseases and whereas a
                                                      Community databank should be
                                                      compiled         to    store  updated
                                                      information on w$ diseases and
                                                      Whereas detection and control
                                                      systems should likewise be set up:
          Whereas in its Resolution (A4-
          0311/95) on the Medium-Term Social          Whereas in its Resolution (A4-
          Action Programme 1995-19972 the             0311/95) on the Medium-Term Social
          Parliament asked the Commission to          Action Programme 1995-19972 the
          present, under the proper procedures,       Parliament asked the Commission to
          the action programme for rare               present, under the proper procedures,
          diseases foreseen in the Commission's       the action programme for rare
          framework communication on public           diseases foreseen in the Commission's
          health;                                     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.                                                      C0M(93)559 final
   OOM(93)559fiwJ
                                                        OJN°C 32 of05.02.96
   OJN°C 32 of03.02.96
 ---pagebreak---      Whereas, the Community can provide
     added value to the actions of Member
     States concerning rare diseases,
     through the co-ordination           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;
                                                   in particular health professionals
                                                   and patients' and support group
                                                   associations:
                                            8a)   Whereas, in accordance with the
                                                  social and human rights model of
                                                  disability supported in the
                                                  Commission cornrnunication on
                                                  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,
9.  Whereas co-operation with 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      o£    and     wider
    dissemination of information about
    rare diseases and by developing
    actions complementary to existing
    Community programmes and actions,
    while       avoiding       unnecessary
    duplication, the programme will
    contribute to the achievement of the
    Community objectives set out in
    Article 129;
                                            10a) Whereas the high level             of
                                                 technology currently available can
                                                 contribute     significantly to the
                                                 acquisition of better knowledge and
 ---pagebreak---                                             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.   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 regard to their
       effectiveness and the achievement of
      the objectives set and, with a view
      where appropriate, to making the
      necessary
12.   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;
13.   Whereas the introduction of specific
      Community arrangements will help to
      ensure that all Member States are
      swiftly informed in the event of an
     emergency situation, so that the
     protection of the population can be
     ensured;
14.  Whereas          these     Community
     arrangements for the rapid exchange
     of information do not affect the
     Member States' rights and obligations
     under Treaties or bilateral and
     multilateral conventions;
15.  Whereas an agreement on a modus
     viverdi       between the European
     Parliament, the Counci! and 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.
 ---pagebreak---  16.     Whereas this decision lays down, a
         financial framework constituting the
         principal point of reference, within the
         meaning of point 1 of the Declaration
         of the European Parliament, the
         Council and the Commission of 6
         March 1995, for the budgetary
         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 the future
        financial perspectives;
HAVE DECIDED AS FOLLOWS:
                   Article 1
     Establishment of the programme
1.      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.
2.     The aim of this programme is to            The aim of this programme is to
       contribute towards ensuring a high         contribute towards ensuring a high
       level of health protection in relation to  level of health protection in relation
       rare diseases by providing knowledge       to rare diseases by providing
       about these diseases by promoting          knowledge about these diseases,
       and strengthening patient support          especially for patients and health
       groups and by fostering the setting up     professionals, by promoting and
       of cluster response teams.                 strengthening     patient     support
                                                  groups, and by festering the setting
                                                  up of cluster response teams.
       The actions to be implemented under
       this programme and their specific
 ---pagebreak---         objectives are set out in the Annex
       under the headings:
1)     Actions on Community information
       on rare diseases
2)     Actions in support of patient and
       family support groups
3)     Actions on handling rare diseases 3)
       clusters                                Actions on identifying hanjBi^B
                                               responding rapidly to rare diseases
                                               clusters,
                  Article 2
              Implementation
1. The       Commission        shall    ensure
   implementation, in close co-operation
   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 in detail after the
       establishment of the future financial
       perspectives.
       The annual appropriations shall be
       established     by    the     Budgetary
       Authority in accordance with the
       financial perspectives.
 ---pagebreak---                      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.      In implementing this action plan, the
         Commission shall be assisted by an      In implementing this action plan, the
         advisory      committee,    hereinafter Commission shall be assisted by an
         referred to as "the CommitteeH,         advisory committee,                   hereinafter
         consisting of two representatives from  referred to as "the Committee",
         each Member State, and chaired by       consisting of two* representatives
         the Commission representative.          from each Member State, and chaired
                                                 by the Commission representative.
2.       The representative of the Commission
         shall submit to the Committee a draft
         of the measures to be taken
         concerning, in particular:
(a)       the criteria, and procedures for
          selecting andfinancingprojects under
          this programme;
(b)       the evaluation procedure.
The Committee shall deliver its opinion on the
draft, within a time limit which the
chairperson may lay down according to the
urgency of the matter, if necessary by taking a
vote.
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.                                        Conectionofthe text due to a mistake in the English
                                                 version ofthe propoul COM(97) 223 final
 ---pagebreak--- 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
Commission proposals or Community
initiatives and the implementation of
programmes in other policy areas which are
relevant to the achievement of the objectives
of this programme.
                     Article 6
           International co-operation
         In the course of implementing this
         programme, co-operation with third
         countries and with international
         organisations competent in the field of
         public health shall be fostered.
         This programme shall be open to
         participation by the associated
         countries of Central Europe (CEC), in
         accordance with the conditions laid
         down in the Association Agreements
         or Additional Protocols related thereto
         concerning         participation     in
         Community        programmes.       This
         programme shall be open to
         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.
                     Article 7
          Monitoring and evaluation
!.        In the implementation of this
*
         Decision, the Commission shall take
         the necessary measures to ensure
                                              10
 ---pagebreak---          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.
         During the third year of 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 and the
         Council a final report on completion
         of this programme.
        The Commission shall incorporate
        into these two reports information
        on Community financing in the
        various fields of action and on
        complementarity with the other
        actions referred to in Article 4, as
        well as the results of the
        evaluations. It shall also send them
        to the Economic and Social
        Committee and the Committee of
        the Regions.
Done at Brussels,
For the European Parliament
        For the Council
The President
        The President.
                    ANNEX
     SPECIFIC OBJECTIVES AND
                  ACTPN8
L      ACTIONS ON COMMUNITY
INFORMATION ON RARE DISEASES
Objective: to provide knowledge about rare Objective : to provide knowledge about rare
             diseases especially for patients,         diseases especially for patients and
             health      professionals    and          their relatives, health professionals
             researchers.                              and researchers.
                                            11
 ---pagebreak---         Encouragement and support for the
        establishment of a European rare           Encouragement and support for the
        diseases database, with entries listing    establishment of a European rare
        the disease name, synonyms, a general      diseases database, with entries listing
        description of the disorder, symptoms,     the disease name, synonyms, a general
        causes, affected population, standard      description    of      the    disorder,
        treatments, investigational treatments     symptoms,        causes,       affected
        (when available) and a list of resources   population, standard treatments,
        that can be contacted for further          investigational treatments (when
        information about the condition.           available) and a list of resources that
                                                   can be contacted for further
                                                   information about the condition. The.
                                                   information collected fa É&JŒÊiâ
                                                   European database descril
                                                   will be made as widely avi
                                                   possible.
        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
        health professionals in order to
        improve      the      early detection,
        recognition,       intervention       and
        prevention of rare diseases.
H.       ACTIONS IN SUPPORT OF
PATIENT AND FAMILY SUPPORT
GROUPS
Objective:       to establish, foster and
strengthen voluntary organisations involved in
supporting people directly or indirectly
affected by rare diseases.
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
 ---pagebreak---       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 Objective : to have in place an effective system
            the problem of clusters, which is               of monitoring in order to ensure the
             of key importance for rare                     rapid identification, assessment and
             diseases.                                                 Qf rare disease? and
                                                            diseases clustery
       Supporting the monitoring (sentinel)              Promoting J^^Û£ïÉQmt£®L„SÉ
       of rare diseases, including birth                 systems fpr J]^jm^SXà^s£jas&
       defects, genetic disorders or diseases            siiseises___ai ç^iiMusily_JsïsL
       of different organ systems and the                including birth defects, genetic
       appropriate techniques for low-                   disorders or diseases of different
       prevalence diseases, in order to meet             organ systems -and the appropriate
       on the one hand the demands of                    techniques       for     low-prevalence
       detection, treatment and research and             diseases, in order to meet on the one
       on the other hand the demands of                  hand the demands of detection.,
       relevant statistical monitoring.                  treatment and research and on the
                                                         other hand the demands of relevant
                                                         statistical monitoring.
      Promoting the creation of rare
      diseases response teams and of
      specialised training courses for those
      investigating clusters.                           rare diseases and of networking and
                                                        training for the investigation and
                                                        handling of clusters.
      Supporting surveillance and early
      warning systems for clusters.
      Encouraging the exchange of expertise
      in the evaluation, assessment,
      communication and management of
      clusters of rare diseases that are
      associated with exogenic causes.
                                            13
 ---pagebreak---  ---pagebreak---                                                                    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
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