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 opinion of the Economic 
and Social Committee, 
Having regard to the opinion of the 
Committee of the Regions, 
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; Having regard to the proposal from 
the Commission,1 
Having regard to the opinion of the 
Economic and Social Committee,2 
Having regard to the opinion of the 
Committee of the Regions,3 
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 nre 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 
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;. 
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 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 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; lc) 
2a) 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 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: 
5. Whereas rare diseases have been 
identified as a priority area for 
Community action within the 
framework for action in the field of 
public health1 
Whereas in its Resolution (A4-
0311/95) on the Medium-Term Social 
Action Programme 1995-19972 the 
Parliament asked the Commission to 
present, under the proper procedures, 
the action programme for rare 
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 5a) Whereas rare diseases have been 
identified as a priority area for 
Community action within the 
framework for action in ths field of 
public health1 
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 
Action Programme 1995-19972 the 
Parliament asked the Commission to 
present, under the proper procedures, 
the action programme for rare 
diseases foreseen in the Commission's 
framework communication on public 
health; 
C0M(93)559 final 
OJN°C 32 of 05.02.96 
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; 
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; 8a) 
10a) in particular health professionals 
and patients' and support group 
associations: 
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, 
Whereas the high level of 
technology currently available can 
contribute significantly to the 
acquisition of better knowledge and 
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 regard to their 
effectiveness and the achievement of 
the objectives set and, with a view 
where appropriate, to making the 
necessary understanding of. and the wider 
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 
emergency situation, so that the 
protection of the population can be 
ensured; 
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; 
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. 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: 
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 
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 patient support 
groups, and by festering the setting 
up of cluster response teams. 
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 
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. 3) 
Actions on identifying hanjBi^B 
responding rapidly to rare diseases 
clusters, 
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 
advisory committee, hereinafter 
referred to as "the CommitteeH, 
consisting of two representatives 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 and financing projects 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. In implementing this action plan, the 
Commission shall be assisted by an 
advisory committee, hereinafter 
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 
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 
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 
diseases especially for patients, 
health professionals and 
researchers. Objective : to provide knowledge about rare 
diseases especially for patients and 
their relatives, health professionals 
and researchers. 
11 
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 
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 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. The. 
information collected fa É&JŒÊiâ 
European database descril 
will be made as widely avi 
possible. 
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 importance for rare 
diseases. 
Supporting the monitoring (sentinel) 
of rare diseases, including birth 
defects, genetic disorders or diseases 
of different organ systems and the 
appropriate techniques for 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. 
Promoting the creation of rare 
diseases response teams and of 
specialised training courses for those 
investigating 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. 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 
Promoting J^^Û£ïÉQmt£®L„SÉ 
systems fpr J]^jm^SXà^s£jas& 
siiseises___ai ç^iiMusily_JsïsL 
including birth defects, genetic 
disorders or diseases of different 
organ systems -and the appropriate 
techniques for 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. 
rare diseases and of networking and 
training for the investigation and 
handling of clusters. 
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-2985 ? iHXC!H'"K-UriZ