Insight HEMOPHILIA. Drug Class. Injury Occurs. Background, new developments, key strategies INTRODUCTION. 20,000 patients in the US 1 MISSING LINK

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1 Drug Class Insight HEMOPHILIA Background, new developments, key strategies INTRODUCTION Hemophilia is a rare, inherited bleeding disorder in which the blood does not clot properly. 20,000 patients in the US 1 Occurs in 1 of 5,000 male births 1 Affects MALES almost exclusively 1 Extremely rare, but extremely expensive: Ranks 8th most costly for employers for all specialty diseases 2 Hemophilia is caused by a mutation that disrupts the body s ability to produce clotting factor proteins. There are two main forms of the disease, labeled A and B. 1 Low levels of clotting factor can lead to spontaneous bleeding in the joints or internal organs, or even inside the skull. Uncontrolled bleeding can cause joint disease, seizures and paralysis or death.1 Hemophilia can be mild, moderate, or severe. The lower the amount of the factor, the more likely it is that bleeding will occur. 1 MISSING LINK Lack of clotting factor reduces the body s ability to control bleeding. Injury Occurs Injury to blood vessel results in bleeding. Damaged area Vessel constricts and clotting factors are activated. Natural clotting factor Normal Natural clotting factor helps form a strong platelet plug. Hemophilia Lack of natural clotting factor means only a weak platelet plug can form. Less clotting factor A stable fibrin mesh forms a sealed clot over the platelet plug to stop the bleeding. Incomplete fibrin mesh allows bleeding to continue. Adapted from: Human Disease: Blood Clot Disease (Hemophilia). Jan 26, Accessed at: on Optum Page 1

2 All but the mildest forms of hemophilia are extremely expensive to treat. Unfortunately, the majority of people with hemophilia have the most severe form. Of all people with hemophilia 60% Severe: less than 1% of normal level of clotting factor; constant danger of bleeding episodes 25% Mild: 6-30% of normal clotting factor; may not be diagnosed until after serious injury 15% Moderate: 1-5% of normal clotting factor; some possible spontaneous bleeding, or after injury Nature Outlook: Haemophilia. A Primer on Haemophilia. Dec 16, 2014 Critical Breakthrough: Replacement Clotting Factor Compact, effective forms of replacement clotting factor helps many hemophilia patients live near-normal lives Factor VIII Concentrate 500 Factor IX Concentrate Hemophilia therapy consists of replacement therapy artificially raising a patient s level of clotting factor via infusion. 3 Fresh plasma contains only trace amounts of clotting protein and huge amounts were needed to control bleeding. Researchers learned to make concentrated forms of factor, but these still required thousands of units of donated plasma to prepare just one unit of concentrate. 3 Modern factor concentrates are made either with donated human blood plasma, or, beginning in the 1990 s, patients began using genetically engineered clotting factor. 3 Both forms of clotting factor are easy to store, mix, and use at home, so many patients self-administer their factor. It only takes about 15 minutes to receive the factor. 3 Immunity Some patients develop immunity to their infusions About 30% of type A and 5% of type B patients on preventive therapy develop immunity to the clotting factors called inhibitors. 4 Inhibitors make treatment of bleeding episodes much more complicated, which drives up factor costs up to or over $1 million per year is not uncommon. 4 $1 million Total annual cost Optum Page 2

3 UNDERSTANDING TREATMENT COSTS Hemophilia treatment costs can be extremely variable at the individual patient level. At the aggregate level, we can expect overall treatment spending to rise over time as hemophilia patients live longer and as new treatments come into play. Clotting factor costs One key cost variable is how much replacement clotting factor a patient needs. Mild and moderate cases require relatively little replacement factor, while severe cases need much more. Patients on preventive therapy use the most replacement factor but also show fewer bleeding episodes and ER visits. Total medical and indirect costs per year $350k $300k $250k $200k $150k $50k $0k 54% $59,101 Mild Nearly all spending (94%) for severe cases is due to factor costs. Compare to just over half (54%) for mild cases. $84,363 Moderate Severe (On-demand treatment) 94% $301,392 Severe (Preventive treatment) Journal of Medical Economics. Burden of illness: direct and indirect costs among persons with hemophilia A in the United States. Mar 9, [Epub ahead of print.] Accesses at: on Immunity (inhibitors) to factor infusions $201,471 The chart below illustrates the explosive effect inhibitors have on costs when inhibitors and comorbidities are added. Health Care Costs for Hemophilia in Commercially Insured Populations $9.0k $8.0k $7.0k $6.0k Total Annual Medical Cost Without inhibitors Including inhibitors $5.0k $4.0k $3.0k $696,279 $831,866 $577,640 $2.0k $1.0k $0k $144,306 $142,057 $188,056 All males with Type A Children with Type A Total Annual Medical Cost Adults with Type A plus HIV/HCV Haemophilia (2012), 18, DOI: /j x Page 3

4 PREDICTING FUTURE TREATMENT SPENDING While factor concentrate is expensive, purchase prices have remained fairly flat and are expected to remain so in the short term. 5 Therefore, predicting costs over the short term means understanding the age profile of each patient. Over the longer term, overall treatment costs will go up due to demographic pressures. Long Term Costs will rise as growing numbers of patients live longer and develop additional complications (e.g., obesity, low bone density). Better treatments have increased Hemophiliac life expectancy Average life expectancy for hemophiliacs ~ 10 years less than 20 average s / Hemophiliacs who have consistently received proper treatment Today / Newborn with hemophilia living in a developed nation Normal U.S. male lifespan National Hemophilia Foundation. History of Bleeding Disorders. Accessed at: on National Haemophilia Council. What is the life expectancy of someone with haemophilia? Accessed at: ie/home/faqs/what_is_the_life_expectancy_of_someone_with_haemophilia/ on Hemophilia patients experience radical changes in the cost of their disease as they age. Payer costs change significantly by patient age Annual Health Insurance Expenditure Per Patient, $USD Short Term $600,000 $500,000 $400,000 $300,000 $200,000 $100,000 Hemophilia A Type B peak Hemophilia B Type A change $ Age Years American Society of Hematology: 56th Annual ASH Meeting & Exposition. The Changing Costs of Caring for Hemophilia Patients in the U.S.: Insurers and Patients Perspectives. Dec. 9, Paper 199. Optum Page 4

5 PROMISING TECHNOLOGY Some of the highest costs for hemophilia are due to patients who develop inhibitors (resistance) to the factor itself. 6 Unfortunately, treatments to overcome resistance are expensive (~$1 million) and not always effective. 6 This promising research aims to prevent the immune system from producing inhibitors. Preventing Inhibitor resistance Genetically engineered plant cells can be coaxed to produce type A and B factor proteins as part of their normal cell machinery. While plant proteins cannot be used to directly promote clotting, they can help desensitize the body s immune system and prevent rejection. 7 Note: Although human figures are used below, these studies are still in the animal phase. Human trials are expected to begin sometime in Lettuce leaf chloroplasts are genetically The resulting plant-based factor cannot engineered to produce human clotting be used directly to control bleeding. factor protein. Instead, the engineered lettuce and About 10,000 chloroplasts are clotting protein is freeze dried, powdered in each cell. and administered by adding to food. chloroplasts Engineered clotting protein cell wall 3The leaf cells protect clotting proteins from stomach acids. Cells release proteins once inside the intestinal tract. Another protein is added to help clotting factor bind to the intestinal wall. Clotting proteins are then processed by the immune system to produce tolerance. stomach large intestine 4Any existing inhibitors are reversed. The plant-based clotting protein defuses the immune system. Real clotting factor infusion can proceed with no allergic reaction. Leaf cell diagram adapted from: University of Cincinnati Clermont. College Biology Home page: Cells & Organelles. Sept 5, Process based on: Nature Outlook: Haemophilia. Lettuce Pills May Help Treat Haemophilia. Dec 16, Optum Page 5

6 OVERVIEW OF HEMOPHILIA MANAGEMENT PROGRAM Integrated management from BriovaRx specialty pharmacy ensures consistent management and holistic care coordination. BriovaRx Program Goals Clinical Management Provides individualized educational and clinical support for patients to help improve adherence to achieve optimal outcomes. Utilization Management Ensure clinically appropriate and cost-effective utilization by using the management strategies available to each client. Aggressive contracting Leading to better drug pricing. Available Networks Access to efficient, quality care and deep discounts for many specialty drugs. Assay Management We tightly manage how much factor medication is dispensed reduces waste and can save thousands of dollars per patient per year. Benefits MEMBERS Better health and support and guidance CLIENTS Total cost control and program management: clients can achieve up to $0.66 PMPM savings by exclusively using participating providers* PROVIDERS Better health support and guidance DEDICATED CALL CENTER We have a dedicated call center for hemophilia. The same specialty pharmacist within BriovaRx specialty pharmacy will regularly assess each patient s needs. Ongoing support to recognize and prevent bleeds Comprehensive assessments 2-4X per year Teach patients and caregivers how to self-infuse at home Ensure ready access to clotting factor Conduct routine compliance and adherence calls Supplies needed such as needles, tubing, syringes, etc. SPECIALTY PHARMACY HEMOPHILIA PROGRAM HTCS ARE LOCATED ACROSS THE COUNTRY The BriovaRx specialty pharmacy hemophilia program is designed to make patients as self-sufficient as possible. We also work closely with regional Hemophilia Treatment Centers (HTCs), that bring together specialty teams of doctors, nurses, & health professionals. The BriovaRx specialty pharmacy connects patients with the nearest local HTC which monitors each patient s treatment. Optum Page 6

7 MEET JOHN John was diagnosed with severe hemophilia when he was an infant. Without effective preventive therapy he would risk spontaneous bleeding, disability or death. John was transitioned into the BriovaRx hemophilia specialty pharmacy program shortly after his employer switched to OptumRx. What does it mean to manage the total cost of John's condition? Synchronize member touch points and data into ONE system. Opportunities Traditional PBM OPTIMIZED* 1 Prior Authorization YES 2 Assay Management NOT OPTIMIZED 3 Adherence Programs NOT OPTIMIZED Clinical Management Program Connect members with quality physicians and Hemophilia Treatment Centers Manage co-morbid depression with synchronized behavioral referrals NOT OPTIMIZED MISSED MISSED OptumRx Connected Care A traditional PBM looking only at pharmacy claims or pharmacy solutions can miss opportunities. Our ONE team approach manages multiple consumer touch points to promote real behavior change, like lower cost alternatives, medication adherence or engagement in clinical management programs. Our Connected Care model leverages all member touch points to capitalize on every opportunity to guide each member to their best next health action. 7 Weight monitoring and healthy lifestyle programs to encourage condition-appropriate exercises and diet MISSED * The synchronized care management model described here depends on a minimum specific set of OptumHealth care management services, plus OptumRx pharmacy services. Please speak to your OptumRx or UnitedHealthcare representative for more information about how synchronization can work for you. Connected Engagement: Every interaction is an opportunity to reduce health risk and lower costs Pharmacy Customer Service Online Health Care Advisor Synchronized Touch Points Self Service + Outreach + Mobile Inbound Nurse Advisor Optum Page 7

8 THE OPTUMRx DIFFERENCE: HEALTH CARE CONNECTED Even ordinary PBMs can do an adequate job at the ordinary things PBMs do negotiate discounts, pay claims, manage formularies and so on. But OptumRx is more than an ordinary PBM: We are a complete pharmacy care services company. We focus on managing total condition spending by connecting pharmacy s impact across all benefits. Connected Data and Expertise Incremental 3-6% savings * compared to total pharmacy spend Connected Value Care Management, Wellness, Behavioral Program Connections Advanced Nurse Advisor Pharmacist Consults 360 Alert System Connected Medical, Pharmacy, Specialty Connected Engagement Core PBM Value Like other PBMs, OptumRx delivers savings through core PBM programs Connected Care PDL Management Clinical Programs Utilization Management Exclusions Specialty Pharmacy Home Delivery Adherence programs Drug cost management Claims processing Network * Estimated additional savings over traditional core PBM services based on total pharmacy spend. Illustrative only; results may vary. Optum Page 8

9 References 1. Centers for Disease Control and Prevention. Hemophilia Facts. Aug 26, Accessed at: ncbddd/hemophilia/facts.html on UnitedHealth Center for Health Reform & Modernization, Issue Brief. The Growth of Specialty Pharmacy. April, Accessed at: UHG/PDF/2014/UNH-The-Growth-Of-Specialty-Pharmacy. ashx on National Hemophilia Foundation. History of Bleeding Disorders. Accessed at: Bleeding-Disorders/History-of-Bleeding-Disorders on Specialty Pharmacy Continuum. Hemophilia: Overview and New and Emerging Treatments. May, Volume 3, Spring Issue. Accessed at: id=500&i=may+2014&i_id=1063&a_id=27523 on Morningstar Healthcare Observer. The Hemophilia Market. January CDMI Report. Understanding Hemophilia: A Managed Care Review. Fall Accessed at: _FINAL2.pdf on Nature Outlook: Haemophilia. Lettuce Pills May Help Treat Haemophilia. Dec 16, Accessed at: scientificamerican.com/article/lettuce-pills-you-heard-thatright-may-help-treat-haemophilia/ on For more information about how you can manage the cost of Hemophilia, please contact your OptumRx representative Optum Circle, Eden Prairie, MN Optum and the Optum logo are registered trademarks of Optum. All other brand or product names are trademarks or registered marks of their respective owner. Because we are continuously improving our products and services, Optum reserves the right to change specifications without prior notice. Optum is an equal opportunity employer. M33018-N 4/ Optum. All Rights Reserved. Page 9