hemophilia 2008. improving quality of life …until a cure…through l ower mortality i mproved...

Hemophilia

2008

Improving quality of life…until a cure…through

Lower mortality

Improved outcomes

Fewer hospitalizations

Educated independent patients2000, Soucie, et al Mortality in hemophilia1998, Nuss et al, Medical care in hemophilia

2008www.hemoalliance.org

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*1984 Article on Benefits of Comprehensive Care Center


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


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


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


HTC Services

• Clinical

• Research

• Pharmacy


Hemophilia Treatment Milestones

1890s - clotting link identified1910s - mixed blood studies1930s - normal plasma “corrects” defect1940s - transfusion therapy1950s - fresh frozen plasma1960s - cryprecipitate developed1970s - lyophilized product & home use1980s/1990s - advances in product safety & purity


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


Goals of Treatment

• Recognition of bleeding episodes

• Early versus late treatment

• Replacement of clotting protein

• Appropriate intervention to prevent complications


Treatment Methods

• Prophylaxis -- ⇩ bleeding episodes

• Enhanced infusion protocol - ⇩ effects of bleeding

• “On demand” therapy – treat each bleed episode


Lyophilized Factor VIII produced by recombinant technology


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


Viral Exposure

• HIV

• HEP B and C

• Parvo Virus

• CJD (Creutzfeldt-Jakob Disease)


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


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


HTC Services

• Clinical

• Research

• Pharmacy


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


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


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.


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


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


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


FINANCIAL RELATIONSHIPS

TO PATIENTS• Uninsured care• Compassionate collections policiesTO INSURERS• Contract performance analysis• Opportunities to reduce costs through

clinical studies participation


Overview of ECONOMIC VALUE brought by340B 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)


hemophilia 2008. improving quality of life …until a cure…through l ower mortality i mproved...

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