1. The German Plan for Rare Diseases: A Development in Progress J.-Matthias Graf von der Schulenburg Krakow, 14 May 2010

2. Three questions What is the issue? What do we know? What has to be done? 2 © Prof. Dr. J.-M. Graf von der Schulenburg

3. Three questions 3 © Prof. Dr. J.-M. Graf von der Schulenburg What is the issue? What do we know? What has to be done?

4. 4 © Prof. Dr. J.-M. Graf von der Schulenburg Allocation Efficiency Stabilization Cost stability and continuity of the system Distribution Fair allocation FactsAppraisements Why do we need health service research? Dimensions of an economic evaluation…

5. Three questions 5 © Prof. Dr. J.-M. Graf von der Schulenburg “Are we actually moving in a circular course, Olaf?” What is the issue? What do we know? What has to be done?

6. 6 © Prof. Dr. J.-M. Graf von der Schulenburg Altogether only insufficient scientific knowledge (studies, publications etc.) about the life situation, the quality and the range of care for people with Rare Diseases in the German health care system

8. Discussions with experts and persons concerned

9. Discussions with focus groups

11. Coordinator: Prof. Bastida / Spain7 © Prof. Dr. J.-M. Graf von der Schulenburg

12. Which perspectives were taken into account? 8 © Prof. Dr. J.-M. Graf von der Schulenburg Patient organizations (people concerned) Evaluation of experiences Health care provider (physicians, industry) Evaluation of markets and science Public organizations/ benefactors Evaluation of structures

13. Rare Diseases are currently attracting insufficient attention in Germany 9 © Prof. Dr. J.-M. Graf von der Schulenburg Quelle: BMG-study 2009

14. Which individual care factors are important for patients? 10 © Prof. Dr. J.-M. Graf von der Schulenburg Close to residential care Europe-wide treatment options Fast access to new drugs … Psychosocial care Source: BMG-study 2009 Information about new treatment possibilities High competence of medical care

17. High ambulatory, inpatient and rehabilitative health care utilisation

18. Professionals in different sectors of care and interdisciplinary cooperation are necessary

19. Trans-sectoral concepts of care are essential11 © Prof. Dr. J.-M. Graf von der Schulenburg

20. …economically uninteresting: 12 © Prof. Dr. J.-M. Graf von der Schulenburg 600 Reimbursement 500 Other costs 400 Physiotherapy, dietguidance 300 Diagnosticservice 200 Laboratory checkup 100 Routine checkup 0 – 7 years 8 – 13 years 14 – 17 years 18 – 24 years 25 – 34 years 35 Jahre andolder Additional costs of the institute Source: Mucoviscidosis-study 2007 Costs of ambulatory treatment for Mucoviscidosis Rare Diseases from health care providers point of view:

21. Three questions 13 © Prof. Dr. J.-M. Graf von der Schulenburg Whatistheissue? What do weknow? Whathastobedone?

22. Recommendations of the study Implementation of National and European Centres of Reference or Expertise Development and implementation of Shared-Care Models Augment the evidence of drugs in Off-Label-Use Creating and using therapy guidelines / clinical pathways Intensified European co-operation Formation of a national coalition for Rare Diseases under clear objectives and with a firm time schedule Development of a German National Plan for Rare Diseases 14 © Prof. Dr. J.-M. Graf von der Schulenburg

23. Development of recommendations and realisation of them: A national action league for people with Rare Diseases (NAMSE), where the different perspectives of all relevant stakeholders are considered Aim/result: German National Plan for people with Rare Diseases Announcement: 8. March 2010 15 © Prof. Dr. J.-M. Graf von der Schulenburg

24. Aims of NAMSE Implement the recommendations of the Council of the EU Concentrate the current initiatives in the field of Rare Diseases Enable common, coordinated and targeted activities in the field of Rare Diseases Contributions for a German National Plan for people with Rare Diseases Assist the establishment of national Reference or Expertise Centres Encourage pilot projects and actions in the field of Rare Diseases 16 © Prof. Dr. J.-M. Graf von der Schulenburg

25. Organisation Steering committee (SC) Decisions and controlling board Members from different stakeholders of the health care system respectively point of view Decision-making about e.g. Basic goals Spheres of activity Creation and staffing of working groups Proving and evaluating results of the working groups Adopt recommendations and the implementation of them Meet bimonthly 17 © Prof. Dr. J.-M. Graf von der Schulenburg

26. Organisation Working groups Provide functional and continual work Will be attended by the responsible departments of the BMG, the BMBF and the self-help alliance for Rare Diseases in Germany ACHSE e.V. Staffing with experts and people concerned (according to the aim or question of the working group) Possibility to subdivide in the case of complex problems Sum up all results in written form Regularly reporting the steering committee Meeting frequency as required 18 © Prof. Dr. J.-M. Graf von der Schulenburg

27. Organisation External office of BMG Coordinating the activity of NAMSE (Communication centre) Scientific, administrative and organizational support of NAMSE Organisation of the conferences for the steering group as well as the working groups Public Relations in coordination with the steering group Preparation of (pilot-)projects 19 © Prof. Dr. J.-M. Graf von der Schulenburg

28. Spheres of activity Patient information/transfer of knowledge Diagnostics and therapy (including the development of guidelines) Health care as well as quality assurance National and European networks of Reference and Expertise Centres Research Other topics, created by the steering committee 20 © Prof. Dr. J.-M. Graf von der Schulenburg

29. Timetable Announcement of NAMSE March 2010 Constituent meeting Summer 2010 Finalisation German National Plan for Rare Diseases Year 2013 © Prof. Dr. J.-M. Graf von der Schulenburg 21

30. Thank you for your attention!