WFH Data on Rare Bleeding Disorders and Patient Registries

1. WFH Data on Rare Bleeding Disorders and Patient Registries 2006 WFH World Congress - Vancouver, May 21-25

2. WFH Data on Rare Bleeding Disorders and Patient Registries Rare disorders are part of the new WFH Strategic Plan – Enhancing access to treatment for von Willebrand disease, rare factor deficiencies, and inherited platelet disorders: “Surveys of our stakeholders identified an unmet need for people with other inherited bleeding disorders, such as von Willebrand disease, rare factor deficiencies, and platelet disorders, which often are undiagnosed or misdiagnosed. We will expand the scope of our activities to improve treatment for people with these disorders.” WFH Strategic Plan, 2006 - 2010

3. WFH Data on Rare Bleeding Disorders and Patient Registries • “Enhance Access to Treatment for von Willebrand Disease, Rare Factor Deficiencies, and Inherited Platelet Disorders… • we will: • Improve diagnosis and enhance data collection on their prevalence by country; • Collaborate with regulators and industry to enhance access to safe treatment; • Expand training, educational materials, and web-based resources; • Seek out and establish links with other healthcare professionals; • Since many of these disorders disproportionately impact women, actively integrate women with bleeding disorders within our volunteer structure/committees; • Create visibility for WFH initiatives on other inherited bleeding disorders.” • WFH Strategic Plan, 2006 - 2010

4. 2006 WFH RBD Activities Improve diagnosis and data collection 2005 WFH survey expanded to learn prevalence & geography (Factors I, II, V, V+VIII, VII, X, XI, XIII) Seek to expand global markets Limited therapeutic options globally Encourage multinational studies Need an appropriate regulatory pathway Participate in and support the work of the RBDD Establish a WFH committee to guide future activities WFH Data on Rare Bleeding Disorders and Patient Registries

5. WFH Data on Rare Bleeding Disorders and Patient Registries • Global surveys begun 1998 • First report 1999 • Now annual • Seeks information on • Basic demographics • Resources for care and treatment • Prevalence of infectious complications (HCV, HIV)

6. WFH Data on Rare Bleeding Disorders and Patient Registries WFH Global Survey Data Each year questionnaires are sent to national hemophilia associations linked with the WFH with the request that they in turn work with physicians or health officials, as necessary, to complete the survey. The WFH reviews the completed questionnaires for inconsistencies, which are clarified where possible by communicating directly with the participating organization.

7. WFH Data on Rare Bleeding Disorders and Patient Registries WFH Global Survey Data The 2004 survey is the sixth completed WFH survey. Data were collected in 2004 and supplemented with data from previous surveys. The survey includes data on more than 170,000 people with hemophilia, von Willebrand disease and other bleeding disorders in 96 countries. Collection of data for 2005 is taking place now.

8. WFH Data on Rare Bleeding Disorders and Patient Registries WFH Global Survey Data Source: Participation in the survey is voluntary and self-reported. Although these data are self-reported, fairly consistent information on hemophilia care has been obtained from countries with similar economic capacities, validating its use for program planning.

9. WFH Data on Rare Bleeding Disorders and Patient Registries The WFH encourages our member organizations to build patient registries in collaboration with health care professionals, treatment centres and governments.

10. 2004 WFH Global Survey Summary

11. WFH Data on Rare Bleeding Disorders and Patient Registries 2004 all countries reporting some rare bleeding disorders Norway Philippines Switzerland Palestine Thailand Slovenia Poland Finland Turkey South Africa Malaysia Ireland Venezuela Australia Slovak Republic Hungary Canada Italy United Kingdom Vietnam Nepal Senegal Cyprus Panama El Salvador Portugal Lebanon Guatemala Spain Japan Brazil Egypt Belize Bangladesh Indonesia Kenya Azerbaijan New Zealand Peru Georgia Iceland Jamaica Bulgaria Serbia Sudan Korea Nicaragua Uzbekistán Lithuania Ecuador Romania Belgium Croatia Singapore Iraq Denmark Greece Colombia Iran United States Estonia Malta

12. WFH Data on Rare Bleeding Disorders and Patient Registries Countries with patient registries reporting data on Rare Bleeding Disorders in 2004 Philippines Switzerland Palestine Thailand Slovenia Poland Finland Turkey South Africa Malaysia Ireland Venezuela Australia Slovak Republic Hungary Canada Italy United Kingdom Belize Bangladesh Indonesia Kenya Azerbaijan New Zealand Peru Georgia Iceland Jamaica Bulgaria Serbia Sudan Korea Norway

13. WFH Data on Rare Bleeding Disorders and Patient Registries Countries Reporting >100 Patients Source: WFH Global Survey 2004

14. WFH Data on Rare Bleeding Disorders and Patient Registries

15. WFH Data on Rare Bleeding Disorders and Patient Registries New Global Survey questions for 2005

16. WFH Data on Rare Bleeding Disorders and Patient Registries Results so far: With 50% of member countries reporting, 70% are providing detailed RBD numbers. Lithuania Malaysia Netherlands New Zealand Nigeria Panama Peru Portugal Korea Romania South Africa Spain Switzerland Thailand Turkey UK US Uzbekistan Venezuela Vietnam Bangladesh Brazil Colombia Croatia Egypt Finland France Georgia Hungary Iran Iraq Japan

17. WFH Data on Rare Bleeding Disorders and Patient Registries Thank you