EDQM Recommendations 2013


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EDQM Recommendations 2013

  1. 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. 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)
  3. 3. Biologicals 39: 189-193 (2011)
  4. 4. 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
  5. 5. 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
  6. 6. 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.
  7. 7. 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
  8. 8. 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
  9. 9. 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
  10. 10. 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
  11. 11. 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
  12. 12. 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