1. www.eurordis.org
NATIONAL POLICIES FOR RARE DISEASES
IN BALKAN AREA :
THE FRAMEWORK
Christel Nourissier
General Secretary, EURORDIS
Balkan Congress Rare Diseases_26-27 June 2009

2. www.eurordis.org
A new EU Legislative and Policy Framework
For Rare Diseases

3. A legal basis
• The Commission Communication on Rare Diseases (adopted 11
December 2008)
• The Council Recommendation on Rare Diseases (9 June 2009)
Chapter 1 To Member States : Establish and implement plans or
strategies for rare diseases …or explore appropriate measures for
rare diseases in other public health strategies,
• in order to aim to ensure that patients with rare diseases have access
to high quality care, including diagnostics, treatments, habilitation for
those living with the disease and, if possible, effective orphan drugs
• take note of the development of guidelines and recommendations for
the elaboration of national action for rare diseases… in the framework
of the ongoing European Project for Rare Diseases National Plans
Development (EUROPLAN)
3 Cluj-Napoca,26-27 June 2009

4. The Recommendation on Rare Diseases
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5. What is EUROPLAN ?
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6. Collection and exchange of information
A 5 years plan
already evaluated in France
National plans in Bulgaria and Romania
Many national initiatives that we shall discuss today…
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7. WP 8 (co-ordinated by EURORDIS)
Objectives
Organising National Conferences
to present and discuss the transferability at the local level
of the EC Recommendation and EUROPLAN Recommendation
Commission Communication on RD EUROPLAN recommendations on
& RD National Plan
Council Recommendation on RD
Common policy guidelines shared in all EU countries
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8. Activities of WP8
• Creation of an advisory Group EURORDIS - National Alliances-EuroPlan: 6
advisors
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• Identification of 15 National Conferences on RD after call for proposal 
• Support to the organisation of 15 National Conferences on RD
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9. Participation to Call for Conferences
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10. Lay out of national conferences
The perfect blend
The main ingredient:
The NATIONAL SITUATION
…and an essential requirement:
To let emerge COMMON ELEMENTS which are anchored to the
COUNCIL RECOMMENDATION
and the
EUROPLAN RECOMMENDATIONS
The final result:
The NATIONAL PLAN for RD
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11. Themes for Working Groups
The Working Groups’ discussion themes will be related to the areas of the
COUNCIL RECOMMENDATION
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12. Today’s focus
• Prenatal / Neonatal screening;
Is screening covering the entire country? Which diseases are screened?
• Diagnostic genetic services
Are the genetic tests cost covered by the state?
• Treatment of patients with RD:
 Orphan drugs
 Rehabilitation
 Social care
• Availability of relevant information on RD
• Training for professionals
• Support to the activities of patient organizations
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• Established services for integration of patients in daily life (ex. center for
integration through occupational therapy)? Who is funding these centers? Do you
think that might be established a supportive environment to fund the socio
medical services for RD patients which prove their efficiency?
• Cross border cooperation/ access to medical services in RD
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