1. Hemophilia 2008

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 2008 www.hemoalliance.org

3. Comprehensive Hemophilia Care in U. S. 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 funding—intended 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 *1984 Article on Benefits of Comprehensive Care Center 2008 www.hemoalliance.org

4. Mission of the HTC 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 2008 www.hemoalliance.org

5. Comprehensive Care Team Physicians Nurses Physical Therapist Social Worker Pharmacists Reimbursement counselors Administrative staff Adjunct staff: Orthopedics, dental, genetics, coagulation lab, infectious disease, hepatology, radiology 2008 www.hemoalliance.org

6. Role of Hemophilia Treatment Centers State-of-the-art medical treatment for persons with hemophilia through the life span Education Research Outreach Model of comprehensive care for chronic disease 2008 www.hemoalliance.org

7. HTC Services Clinical Research Pharmacy 2008 www.hemoalliance.org

8. Hemophilia Treatment Milestones 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 2008 www.hemoalliance.org

9. Clinical Services Diagnosis Treatment – in/ & out-patient Education and Disease Management Comprehensive multi-disciplinary clinic Specialized lab testing Genetics – education, prenatal testing Dental care—evaluation, education Physical therapy Psychosocial support, crisis intervention, transitions, support groups, direct counseling Coordination of care at other institutions Integration of factor distribution with treatment 2008 www.hemoalliance.org

10. Goals of Treatment Recognition of bleeding episodes Early versus late treatment Replacement of clotting protein Appropriate intervention to prevent complications 2008 www.hemoalliance.org

11. Treatment Methods Prophylaxis -- ⇩ bleeding episodes Enhanced infusion protocol - ⇩ effects of bleeding “On demand” therapy – treat each bleed episode 2008 www.hemoalliance.org

12. Lyophilized Factor VIII produced by recombinant technology 2008 www.hemoalliance.org

13. Inhibitor 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 2008 www.hemoalliance.org

14. Viral Exposure HIV HEP B and C Parvo Virus CJD (Creutzfeldt-Jakob Disease) 2008 www.hemoalliance.org

15. Therapies for Hemophilia patients with Inhibitors 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 2008 www.hemoalliance.org

16. GOALS of Hemophilia Centers Provide education for disease management Promote healthy development consistent with disease Provide support for normalcy within community Provide supportive network for families 2008 www.hemoalliance.org

17. HTC Services Clinical Research Pharmacy 2008 www.hemoalliance.org

18. Research Services 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 2008 www.hemoalliance.org

19. Benefits of Service Integration 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 2008 www.hemoalliance.org

20. HTC PHARMACY MISSION 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. 2008 www.hemoalliance.org

21. INTEGRATION LOCAL ACCESS Immediate treatment—reduces 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 2008 www.hemoalliance.org

22. INTEGRATION CUSTOMIZED TREATMENT Customized dosing based on assay availability and patient recovery data—maximum use of resources Customized dispensing—contributes to treatment plan adherence and compliance Weekly case conference with team—patient knowledge increases compliance Utilization reports and data 2008 www.hemoalliance.org

23. LOW COST PROVIDER DRUG PRICING METHODOLOGY Cost based vs. % mark-up basis Allows for product use based on therapeutic benefits, not profit As pharmacy volume increases, overhead decreases, price per unit decreases “threshold pricing”—highest utilization patients caps profits for HTC 2008 www.hemoalliance.org

24. FINANCIAL RELATIONSHIPS TO PATIENTS Uninsured care Compassionate collections policies TO INSURERS Contract performance analysis Opportunities to reduce costs through clinical studies participation 2008 www.hemoalliance.org

25. Overview of ECONOMIC VALUE brought by 340B Outpatient Hemophilia Program 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) 2008 www.hemoalliance.org