1. EDQM recommendations:
haemophilia care in Europe
Dr. Paul Giangrande
Oxford Haemophilia & Thrombosis Centre
and
Nuffield Department of Clinical Medicine
University of Oxford
paul.giangrande@ndm.ox.ac.uk

2. Background:
• Meeting held in Wildbad Kreuth (Germany):
April 26/27th 2013
• Meetings with similar format held there in
April 1999 and 2009
• Organising institutions:
– European Directorate for the Quality of Medicines
& Healthcare (Council of Europe)
– Ludwig-Maximilian-University, Munich (Germany)
– Paul-Ehrlich-Institut (PEI), Langen (Germany)

4. Biologicals 39: 189-193 (2011)

6. Background:
• Focus is on optimal use of blood products:
– Coagulation factor concentrates
– Immunoglobulins
– Albumin and blood (1999/2009)
• Closed meeting with invited clinicians and
regulators (companies attended part of meeting)
• Programme of lectures followed by working group
meetings
• Concluding meeting September 16th 2013

7. Council of Europe (1):
hub.coe.int
• Founded in 1949. Headquarters in Strasbourg.
• International organisation promoting co-operation
between all countries of Europe in the areas of
legal standards, human rights, democratic
development, rule of law and cultural cooperation.
• 47 member states, covering population of ≈ 820
million:
– Much larger bloc than 28 countries of EU
– Additional states have observer status: Canada, Israel,
Japan, Mexico, USA

9. Council of Europe (2):
hub.coe.int
• Cannot make binding legislation (unlike EU)
• Committee of Ministers includes Foreign Ministers
of all Member States
• Directorates include include:
– European Court of Human Justice (which enforces
European Convention on Human Rights)
– EDQM which maintains European Pharmacopeia and
develops guidance and standards in the areas of blood
transfusion, organ-, tissue- and cell transplantation,
pharmaceutical care and consumer health protection.

12. Our strengths in Europe:
• Several major pharmaceutical companies and
production facilities based in our continent
• Treatment usually provided free to patients (or at
least at highly subsidized prices)
• Strong professional and scientific interest in
haemophilia among health care professionals
– EAHAD held first conference in 2008
• Strong and influential patient organizations (EHC)
• Widespread availability of safe treatment has
improved life expectancy and quality of life

14. Haemophilia care in Europe:
• Survey of 35 countries conducted in 2012
– Sequel to similar survey of 19 countries in 2009
• Wide range in factor VIII consumption:
– 0.2 IU/capita (Armenia) → 8.56 (Sweden)
– 2 IU/capita was minimum recommended national
consumption at Wildbad Kreuth 2009 meeting
– Consumption still below this in 12/35 countries
– Consumption has risen in 15/19 surveyed in 2009

15. Principal recommendations (1):
1. In order to optimise the organisation of haemophilia
care nationally, it is recommended that a formal body
be established in each country to include the relevant
clinicians, national haemophilia patient
organisation, health ministry, paying authority and (if
appropriate) regulatory authority
–
–
Strong support for EUHANET project and certification of
haemophilia centres
Group felt that system of peer review should be established
in the longer term

16. Principal recommendations (2):
2. The minimum factor VIII consumption level in a
country should be 3 iu/capita
– Lower threshold not enough to gurantee access to
prophylaxis in children
3. Decisions on whether to adopt new product
should not be based solely on cost
– This is a potential problem with national tenders
– Concern that long-acting products may not be
affordable
– Biosimilars should not be overlooked
– Both plasma-derived and recombinant products will be
need for years to come

17. Principal recommendations (2):
4. Prophylaxis for children with severe haemophilia is
already recognised as the optimum therapy. Ongoing
prophylaxis for individual adults should also be
provided when required based on clinical decision
making by the clinician in consultation with the
patients.
5. Children with inhibitors who have failed immune
tolerance therapy, or who are not suitable for this
treatment, should be offered prophylaxis with
bypassing agents
–
Less enthusiasm for prophylaxis in adults with inhibitors, due
to likelihood of established joint damage and higher cost

18. Principal recommendations (3):
6. Single factor concentrates should be used as therapy
wherever possible in patients with rare bleeding
disorders.
– New plasma concentrates of fibrinogen as well as V and X are
being developed as well as recombinant VWF
7. Orphan drug designation for a factor concentrate should
not be used to hinder the development, licensing and
marketing of other products for the same condition
which have demonstrably different protein modification
or enhancement
– Concern expressed that companies can exploit legislation and
seek protected status for a product in order to secure market
exclusivity