IMPORTANT ISSUES FOR THE HEMOPHILIA AND BLEEDING DISORDERS COMMUNITY OF WISCONSIN

BACKGROUND:  HEMOPHILIA is a bleeding disorder in which a clotting factor (usually factor VIII or Factor IX) is missing or does not function normally.  Most persons with hemophilia are male.  There are three different severities of hemophilia- severe, moderate and mild.  Many persons with hemophilia bleed internally into muscles and joints.  Bleeds in the brain or other organs can also occur and can be life-threatening. Approximately one in 5,000 males has hemophilia.  All races and economic groups are affected equally. 

VON WILLEBRAND DISEASE is a bleeding disorder in which von Willebrand factor, a protein that works with factor VIII, is missing or does not function normally.  von Willebrand disease affects both males and females and impacts 1%- 3% of the population.

WOMEN WITH BLEEDING DISORDERS, typically with a factor deficiency or von Willebrand disease, are prone to heavy or prolonged menstrual cycles and excessive bleeding after child birth (in addition to other bleeding symptomotology).

WISCONSIN TREATMENT CENTERS  In Wisconsin, four federally-funded hemophilia treatment centers (HTCs) provide medical care to 1630 patients with hemophilia, von Willebrand disease and other factor deficiencies.

ISSUES

Many persons with hemophilia and a number of persons with von Willebrand disease require the use of clotting factor concentrates to either treat or try to prevent bleeding.  Clotting factor concentrates are incredibly expensive biotech drugs that are often included in the category of specialty pharmacy. 

• ACCESS TO A FULL FORMULARY OF CLOTTING FACTOR CONCENTRATES   There are a number of different clotting factor concentrates in different classifications of drugs.  Per the National Hemophilia Foundation Medical and Scientific Advisory council:  “Clotting factor therapies are neither pharmacologically or therapeutically equivalent and vary based on purity, half-life, recovery, method of manufacture, viral removal and inactivation processes, potential immunogenicity, and other attributes.”  All clotting factor concentrates are brand name.  None are generic.  It is important that there be no formulary restriction on clotting factor concentrates in any Medicaid formulary.

• ACCESS TO FEDERALLY-FUNDED HEMOPHILIA TREATMENT CENTERS (HTCs) THAT PROVIDE CLOTTING FACTOR CONCENTRATES  There are  presently three HTCs in Wisconsin (Green Bay- Hemophilia Outreach Center, Madison- University of Wisconsin and Milwaukee-  Blood Center of Wisconsin) that serve patients and provide clotting factor concentrates to Wisconsin Medicaid patients.  It is important that these three HTC providers continue to be Medicaid approved so they can continue to earn revenue to support essential, non-reimbursed HTC patient services.

• ADEQUATE FUNDING FOR THE WISCONSIN CHRONIC DISEASE PROGRAM (WCDP)- also known as the Disease Aids Program   The WCDP helps to pay for the uncovered cost of  the very expensive clotting factor concentrates based on patient need, after all insurance has paid, for uninsured or underinsured hemophilia patients.  (The WCDP also pays benefits for chronic renal disease and cystic fibrosis.)   Adequate funding for the WCDP must be maintained, taking into account increased numbers of patients utilizing the program as well as health services and pharmacy inflation.  

• STANDARD OF SERVICE FOR ANY PROVIDER THAT SUPPLIES CLOTTING FACTOR CONCENTRATES TO PATIENTS WITH BLEEDING DISORDERS FOR ADMINISTRATION IN THE HOME   Any Medicaid or WCDP approved provider of clotting factor concentrates should meet a high standard of service to assure that all patients receive their potentially life-saving medications in an efficient and efficacious manner.