Hemophilia U & I Inc., USA Payor Education Presentation Last Updated; January, 2012

Hemophilia Chronic genetic disorder characterized by a protein deficiency that results in delayed blood clotting. Individuals with hemophilia typically have internal bleeds affecting their joints and muscles. While hemophilia is the result of a mutation on the X chromosome, approximately 33% of cases are random mutations and not inherited. Since the mutation is located on the X chromosome, males (having only one X) tend to be more symptomatic than females. Hemophilia A is the deficiency of the Factor VIII protein. Hemophilia B is the deficiency of the Factor IX protein. Facts

Hemophilia There are roughly 20,000 hemophiliacs in the US, nationwide. Hemophilia A accounts for about 80% of the cases while Hemophilia B makes up the other 20%. Severe hemophilia accounts for roughly 50% of all cases in the United States. Moderate and mild hemophilia each make up approximately 25% of the cases. The average medical cost for a person with hemophilia (using all severities and both Hemophilia A & B) is approximately $150,000. Clotting factor replacement products account for about 90% of hemophiliac’s annual medical costs. The average cost for one dose of factor replacement is $1,500 - $2000 and near 30% of bleeds require more than one dose. By the Numbers

Hemophilia Treatments for hemophilia include intravenous clotting factor replacement, icing the injury site, as well as compression and elevation of the site. In order to avoid costly delays that cause a bleeding episode to get worse and therefore require more factor and time in order to recover, most individuals are trained to infuse by themselves at home. Factor replacement therapy can take three forms; (which form should be utilized is determined by the patient and their doctor) –Scheduled prophylaxis treatment is used to maintain a safe level of clotting protein through intravenous infusions on a regular basis. –Some people infuse prior to a more physical activity such as jogging or biking to prevent bleeds at that time, however they do not infuse on a regular schedule. –Others wait until they have a bleed occur before undergoing therapy. Treatment

Hemophilia Many Complication can cause the costs of treating a person with hemophilia to increase. here are several complications that are frequently seen with hemophilia population. Co-morbidities such as HIV infection and Hepatitis can mean additional medications and doctors visits and/or hospitalizations that drive up cost of care. (Luckily HIV and Hepatitis have not been known to be transmitted through factor use for many years, meaning that these really only impact the older generations now.) Inhibitors result from the individual’s immune system rejecting the clotting factor used in the replacement therapy. When this occurs, the cost of treatment and factor usage can skyrocket. –Roughly 33% of the people with severe hemophilia develop inhibitors. –Immune tolerance therapy (also called Inhibitor Override Therapy) and bypass therapy are the primary treatments for inhibitor patients. –Once a person begins Override Therapy, compliance to the treatment regimen is critical to success which means long term quality of life improvements as well as large cost reductions. Joint damage is a large cause of long term problems and recurring bleeding episodes. Treating a joint bleed correctly when they do occur can prevent or limit the long term damage, preventing many target joints from developing and greatly reducing the time and factor needed to recover when they do occur. To this end, clinical management and outcomes monitoring is an important part of cost reduction. Complications

Hemophilia at U & I, HEMOPHILIA IS OUR ONLY BUSINESS U & I’S UTILIZATION MANAGEMENT PROGRAM SIGNIFICANTLY LOWERS HEMOPHILIA COSTS AND IMPROVES QUALITY OF LIFE U & I UTILIZATION COST FOCUS: HEMOPHILIA SELF CARE EDUCATION, BLEEDING PREVENTION, REDUCING INFUSION, AND ASSAY MANAGEMENT U & I UTILIZATION MANAGEMENT PROCESS – ASSESSMENT/MONITORING OF RISK WITH MEASUREMENT OF TREATMENT/COMPLIANCE – PRODUCTS/SERVICES/EDUCATION –PERSONAL DELIVERY INTERVENTION –CONTINUITY OF CARE WITH HTC/PAYOR OUTCOMES REPORTING why u&i ?

Hemophilia U & I Inc., USA 5236 West Seneca St. Vernon, NY Payor Education Presentation