Hemophilia 2009

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

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

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

Physicians Nurses Physical Therapist Social Worker Pharmacists Reimbursement counselors Administrative staff Adjunct staff: Orthopedics, dental, genetics, coagulation lab, infectious disease, hepatology, radiology

State-of-the-art medical treatment for persons with hemophilia through the life span Education Research Outreach Model of comprehensive care for chronic disease

Clinical Research Pharmacy

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 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

Recognition of bleeding episodes Early versus late treatment Replacement of clotting protein Appropriate intervention to prevent complications

Prophylaxis -- bleeding episodes Enhanced infusion protocol - effects of bleeding On demand therapy – treat each bleed episode

Lyophilized Factor VIII produced by recombinant technology

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

HIV HEP B and C Parvo Virus CJD (Creutzfeldt-Jakob Disease)

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

Provide education for disease management Promote healthy development consistent with disease Provide support for normalcy within community Provide supportive network for families

Clinical Research Pharmacy

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

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

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

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

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

TO PATIENTS Uninsured care Compassionate collections policies TO INSURERS Contract performance analysis Opportunities to reduce costs through clinical studies participation

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)