WFH Data on Rare Bleeding Disorders and Patient Registries

2006 WFH World Congress - Vancouver, May 21-25

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

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

WFH Data on Rare Bleeding Disorders and Patient Registries

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

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)

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.

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.

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.

WFH Data on Rare Bleeding Disorders and Patient Registries

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.

2004 WFH Global Survey Summary

Number of countries in this survey 96

Percentage of world population covered by survey 85

Number of people identified with hemophilia A and B 120,812

Number of people with hemophilia A 93,116

Number of people with hemophilia B 18,830

Number of people with von Willebrand disease 43,334

Number of people with other bleeding disorders 11,384

Total number of people with bleeding disorders 175,530

Number of countries using national registries 40

CyprusPanamaEl SalvadorPortugalLebanonGuatemalaSpainJapanBrazilEgyptBelizeBangladeshIndonesiaKenyaAzerbaijanNew ZealandPeruGeorgiaIcelandJamaicaBulgariaSerbiaSudanKorea

Norway PhilippinesSwitzerlandPalestineThailandSloveniaPolandFinlandTurkeySouth AfricaMalaysiaIrelandVenezuelaAustraliaSlovak RepublicHungary CanadaItalyUnited KingdomVietnamNepalSenegal

WFH Data on Rare Bleeding Disorders and Patient Registries

NicaraguaUzbekistánLithuaniaEcuadorRomaniaBelgiumCroatiaSingaporeIraqDenmarkGreeceColombiaIranUnited StatesEstoniaMalta

2004 all countries reporting some rare bleeding disorders

BelizeBangladeshIndonesiaKenyaAzerbaijanNew ZealandPeruGeorgiaIcelandJamaicaBulgariaSerbiaSudanKoreaNorway

WFH Data on Rare Bleeding Disorders and Patient Registries

PhilippinesSwitzerlandPalestineThailandSloveniaPolandFinlandTurkeySouth AfricaMalaysiaIrelandVenezuelaAustraliaSlovak RepublicHungary CanadaItalyUnited Kingdom

Countries with patient registries reporting data on Rare Bleeding Disorders in 2004

Source: WFH Global Survey 2004

WFH Data on Rare Bleeding Disorders and Patient Registries

Countries Reporting >100

Patients

REGIONAL DISTRIBUTION OF COUNTRIES REPORTING DATA ON RARE BLEEDING DISORDERS (2004)

Africa, 4

Americas, 13

Eastern Mediterranean, 6

Europe, 27

South East Asia, 3

Western Pacific, 9

WFH Data on Rare Bleeding Disorders and Patient Registries

New Global Survey questions for 2005Diagnosis Number of persons

B10. Hemophilia A      

B11. Hemophilia B      

B12. Hemophilia, type unknown      

B13. von Willebrand disease      

B14a. Other hereditary bleeding disorders: Factor I deficiency      

B14b. Other hereditary bleeding disorders: Factor II deficiency      

B14c. Other hereditary bleeding disorders: Factor V deficiency      

B14d. Other hereditary bleeding disorders: Factor V+VIII deficiency      

B14e. Other hereditary bleeding disorders: Factor VII deficiency      

B14f. Other hereditary bleeding disorders: Factor X deficiency      

B14g. Other hereditary bleeding disorders: Factor XI deficiency      

B14h. Other hereditary bleeding disorders: Factor XIII deficiency      

B14i. Other hereditary bleeding disorders: platelet disorders      

B14j. Other hereditary bleeding disorders: type unknown      

WFH Data on Rare Bleeding Disorders and Patient Registries

BangladeshBrazilColombiaCroatiaEgyptFinlandFranceGeorgiaHungaryIranIraqJapan

WFH Data on Rare Bleeding Disorders and Patient Registries

LithuaniaMalaysiaNetherlandsNew ZealandNigeriaPanamaPeruPortugalKoreaRomaniaSouth AfricaSpainSwitzerlandThailandTurkeyUKUSUzbekistanVenezuelaVietnam

Results so far:With 50% of member countries reporting, 70% are providing detailed RBD numbers.

WFH Data on Rare Bleeding Disorders and Patient Registries

Thank you