1. Hemophilia 2009

2. Improving quality of life …until a cure…through L ower mortality I mproved outcomes F ewer hospitalizations E ducated independent patients 2000, Soucie, et al Mortality in hemophilia 1998, Nuss et al, Medical care in hemophilia www.hemoalliance.org

3. Established in 1975 – Congressional funding for treatment centers (Section 1131 of the Public Health Service Act) Model for Specialty Disease Management for fragile population with costly rare disease * Grant fundingintended to fully fund public health clinics Centers for Disease Control & Prevention Maternal & Child Health Bureau (Health & Human Services) Currently 143 funded centers in 48 states 2009 *1984 Article on Benefits of Comprehensive Care Center www.hemoalliance.org

4. Identify persons with hemophilia and thrombotic disorders Provide comprehensive diagnostics, treatment, education, and consultative services for physicians, patients and their families Provide educational programs for professional and paraprofessional individuals involved with bleeding disorder care Assess and provide treatment for the long-term complications of hemophilia including inhibitors, liver disease, AIDS, and psychosocial issues Advance knowledge through research in coagulation disorders 2009 www.hemoalliance.org

5. Physicians Nurses Physical Therapist Social Worker Pharmacists Reimbursement counselors Administrative staff Adjunct staff: Orthopedics, dental, genetics, coagulation lab, infectious disease, hepatology, radiology 2009 www.hemoalliance.org

6. State-of-the-art medical treatment for persons with hemophilia through the life span Education Research Outreach Model of comprehensive care for chronic disease 2009 www.hemoalliance.org

7. Clinical Research Pharmacy 2009 www.hemoalliance.org

8. 1890s - clotting link identified 1910s - mixed blood studies 1930s - normal plasma corrects defect 1940s - transfusion therapy 1950s - fresh frozen plasma 1960s - cryprecipitate developed 1970s - lyophilized product & home use 1980s/1990s - advances in product safety & purity 2009 www.hemoalliance.org

9. 2009 Clinical Services Diagnosis Treatment – in/ & out-patient Education and Disease Management Comprehensive multi-disciplinary clinic Specialized lab testing Genetics – education, prenatal testing Dental careevaluation, education Physical therapy Psychosocial support, crisis intervention, transitions, support groups, direct counseling Coordination of care at other institutions Integration of factor distribution with treatment www.hemoalliance.org

10. Recognition of bleeding episodes Early versus late treatment Replacement of clotting protein Appropriate intervention to prevent complications 2009 www.hemoalliance.org

11. Prophylaxis -- bleeding episodes Enhanced infusion protocol - effects of bleeding On demand therapy – treat each bleed episode 2009 www.hemoalliance.org

12. Lyophilized Factor VIII produced by recombinant technology 2009 www.hemoalliance.org

13. Circulating antibody to factor VIII or IX Affects 8-20% of severe fVIII patients Affects 1-3% of FIX Treatment increase factor bypass antibodies immune intolerance 2009 www.hemoalliance.org

14. HIV HEP B and C Parvo Virus CJD (Creutzfeldt-Jakob Disease) 2009 www.hemoalliance.org

15. Adequate factor VIII to overwhelm the inhibitor and maintain an adequate factor VIII level Porcine factor VIII Designer human-porcine hybrid FVIII molecules rFVIIa (NovoSeven) FEIBA Plasmapheresis 2009 www.hemoalliance.org

16. Provide education for disease management Promote healthy development consistent with disease Provide support for normalcy within community Provide supportive network for families 2009 www.hemoalliance.org

17. Clinical Research Pharmacy 2009 www.hemoalliance.org

18. Clinical research studies Improved products for treatment -- Not experimental New technology for delivery of care Viral safety improvements Outcomes research Joint outcome study Radiological evaluation Hemophilia Utilization Group Study Quality of Life Satisfaction surveys Radiosynoviorthesis 2009 www.hemoalliance.org

19. Better health care outcomes Delayed treatment leads to poor outcome Customized/individualized care Maximizes use of product and services Clinic visit vs. ED or hospitalization Better follow-up 2009 www.hemoalliance.org

20. To integrate factor distribution with clinical care for seamless coordination of care and better outcomes To be low cost provider To reflect and fulfill our non-profit, public sector mission in financial relationships with patients, insurers, and other providers. 2009 www.hemoalliance.org

21. LOCAL ACCESS Immediate treatmentreduces complications; requires less factor for treatment Reduces ED visits or hospitalization (only 72% of patients on home care) Facilitates admissions to local institutions- -factor procurement and staff education Provides 24/7 delivery throughout region 2009 www.hemoalliance.org

22. CUSTOMIZED TREATMENT Customized dosing based on assay availability and patient recovery data maximum use of resources Customized dispensingcontributes to treatment plan adherence and compliance Weekly case conference with team patient knowledge increases compliance Utilization reports and data 2009 www.hemoalliance.org

23. TO PATIENTS Uninsured care Compassionate collections policies TO INSURERS Contract performance analysis Opportunities to reduce costs through clinical studies participation 2009 www.hemoalliance.org

24. TOTAL BILLED CHARGES SUBTOTAL ACTUAL CHARGES IF BILLED AT AWP SUBTOTAL % COST SAVINGS OF BILLED CHARGES OFF AWP SUBTOTAL ADDITIONAL FREE RESEARCH DRUGS AT AWP VALUE GRAND TOTAL ACTUAL CHARGES + FREE RESEARCH DRUGS IF BILLED AT AWP GRAND TOTAL % COST SAVINGS OF BILLED CHARGES OFF AWP (including research) 2009 www.hemoalliance.org