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
Date: 1998-04-15
Institutions: European Commission
Subjects: action programme, exchange of information, health policy, illness, 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 

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Catalogue  number  :  CB-CO-98-245-EN-C 

ISBN  92-78-35074-5 

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