The German Plan for Rare Diseases: A Development in Progress

J.-Matthias Graf von der Schulenburg

Krakow, 14 May 2010

Three questions

What is the issue?

What do we know?

What has to be done?

2© Prof. Dr. J.-M. Graf von der Schulenburg

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© Prof. Dr. J.-M. Graf von der Schulenburg

AllocationEfficiency

StabilizationCost stability and continuity of the system

DistributionFair allocation

Facts Appraisements

Why do we need health service research? Dimensions of an economic

evaluation…

Three questions

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“Are we actually moving in a circular course, Olaf?”

What is the issue?

What do we know?

What has to be done?

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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

How do we proceed?

BMG-study• Literature research• Discussions with experts and persons concerned• Discussions with focus groups• Wide survey with a questionnaire

Specific evaluation depending on an indication (CF-study)

EU-study• Social Economic Burden and Health-Related Quality of Life in Patients

with Rare Diseases in Europe (BURQOL-RD)• Coordinator: Prof. Bastida / Spain

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Which perspectives were taken into account?

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Patient organizations (people concerned) Evaluation of experiences

Health care provider(physicians, industry)

Evaluation of markets and science

Public organizations/benefactors

Evaluation of structures

Rare Diseases are currently attracting insufficient attention in Germany

9© Prof. Dr. J.-M. Graf von der Schulenburg

Quelle: BMG-study 2009

Which individual care factors are important for patients?

10© Prof. Dr. J.-M. Graf von der Schulenburg

High competence of medical care

Information about new treatment possibilities

Psychosocial care

…

Fast access to new drugs

Europe-wide treatment options

Close to residential

care

Source: BMG-study 2009

Patients with Rare Diseases need a comprehensive and specialised care

Qualified health care providers (medical and non-medical) are necessary Only a few specialists University hospitals

Patients have a high and comprehensive demand for care The German health care system is separated into different sectors High ambulatory, inpatient and rehabilitative health care utilisation Professionals in different sectors of care and interdisciplinary

cooperation are necessary Trans-sectoral concepts of care are essential

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…economically uninteresting:

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0 – 7years

8 – 13years

14 – 17years

18 – 24years

25 – 34years

35 Jahreand older

300

400

500

600

100

200

Costs of ambulatory treatment for Mucoviscidosis

Source: Mucoviscidosis-study 2007

Rare Diseases from health care providers point of view:

Reimbursement

Other costs

Physiotherapy, diet guidance

Diagnostic service

Laboratory checkup

Routine checkup

Additional costs of the institute

Three questions

13© Prof. Dr. J.-M. Graf von der Schulenburg

What is the issue?

What do we know?

What has to be done?

Recommendations of the study

1. Implementation of National and European Centres of Reference or Expertise

2. Development and implementation of Shared-Care Models

3. Augment the evidence of drugs in Off-Label-Use

4. Creating and using therapy guidelines / clinical pathways

5. Intensified European co-operation

6. Formation of a national coalition for Rare Diseases under clear objectives and with a firm time schedule

7. Development of a German National Plan for Rare Diseases

14© Prof. Dr. J.-M. Graf von der Schulenburg

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

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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

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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

Organisation

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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

Organisation

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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

Organisation

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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

Spheres of activity

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Timetable

Announcement of NAMSE► March 2010

Constituent meeting► Summer 2010

Finalisation German National Plan for Rare Diseases ► Year 2013

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Thank you for your attention!