Hemophilia Disease Management in Florida

Transcription

1 Hemophilia Disease Management in Florida Final report to Florida Agency for Health Care Administration Prepared and Submitted by Abraham G. Hartzema PharmD, MSPH, PhD, FISPE Richard Segal, PhD Jinghua He, MSc, PhD candidate Dawn Alayon, MPH, CPH Department of Pharmaceutical Outcomes and Policy College of Pharmacy, University of Florida And Heather Steingraber Florida Center for Medicaid and the Uninsured College of Public Health and Health Professions

2 Table of Contents Executive Summary... 4 Background... 5 Agency for Health Care Administration Disease Management Program... 5 Epidemiology of Hemophilia... 5 Hemophilia Overview... 5 Disease Progression and Outcomes... 6 Severity Levels of Hemophilia... 6 Treatment Options... 6 Prophylactic Treatment... 6 On-Demand Treatment... 8 Inhibitors... 8 Economic Factors in the Treatment of Hemophilia... 9 Costs and Economic Data... 9 Patient Adherence to Treatment Plans Interdisciplinary Provider Team Comprehensive Hemophilia Disease Management Program in Florida Methods Data Sources Analysis Descriptive Analysis Evaluation of the Overall DMP Evaluation of the DMP Vendor Statistical Analysis Plan Findings Part I. Descriptive Analysis Demographic Characteristics Health Care Resource Utilization Part II. Overall DMP Evaluation Demographics Characteristics Health Care Resource Utilization... 25

3 Health Care Expenditures Part III. Between-Vendor Comparison Demographic Characteristics Health Care Resource Utilization Health Care Expenditures Discussion Results Conclusion References Appendices... i Appendix A... i ICD-9-CM diagnosis codes used for outcomes and baseline characteristics identification.... i Appendix B... ii NDC Codes Used For Factor Product Identification... ii Appendix C... iv Coding Algorithms for Health Care Resource Utilization and Cost Identification... iv Appendix D... vi Appendix E... vii Cost per category without outlier exclusion (bucket code) ($)... vii Appendix F... viii

4 Executive Summary A contract between the Agency for Health Care Administration and two Hemophilia Vendors was executed on January 1, Due to contract implementation, fiscal agent activities, and recipient choice periods, the program became fully operational on June 1, Florida Medicaid started the two-year Comprehensive Hemophilia Disease Management Program (DMP) which is mandatory for all Florida Medicaid non-hmo recipients with at least one pharmacy record for factor products. Participants in the DMP chose either Caremark or CuraScript as their service provider for comprehensive care, including disease management services, patient education, and specialty pharmaceutical care for factor products. This study is an evaluation of the first year of the DMP implementation. The observation period was one year prior to DMP implementation (June 1, 2007 May 31, 2008) and the first year of the DMP (June 1, 2008 May 31, 2009). The objectives of the program evaluation are to (1) describe the DMP population; (2) evaluate the impact of the DMP on health care resource utilization and expenditures; and (3) compare and contrast the two DMP vendors. A total of 288 Florida Medicaid non-hmo recipients were identified as having hemophilia or von Willebrand disease (vwd). Of the 288 members, 281 recipients were receiving factor products through Medicaid during the pre-dmp period (2,880 member-months). During the DMP period, there were 241 participants (2,126 member-months). The population included mainly young men with varying racial/ethnic backgrounds, and few were identified with HIV/AIDS or Hepatitis infections. Most were diagnosed with Hemophilia A or B, followed by vwd and Factor X. This population was costly to Florida Medicaid: the average monthly total cost was $18,316 PMPM, ranging from $3 PMPM to $335,030 PMPM. Hemophilia-related costs accounted for 94.0% of the total cost, and the factor product cost accounted for 92.9% of the total cost. The DMP program was evaluated among 117 hemophilia participants with continuous Florida Medicaid enrollment over the two-year observation period. The number of hemophiliarelated emergency and hematologist visits increased during the DMP period, while the number of home health services and orthopedic surgeon visits decreased. These changes had little influence on the total health care expenditure. After excluding outliers, the amount of dispensed factor products increased approximately six percent during the DMP period compared to the pre-dmp period. This result may be due to the increased body weight of this young population as they mature and Medical and Scientific Advisory Council (MASAC) guidelines issued that promote increased use of prophylactic dosing; however, the associated expenditures remained nearly unchanged, presumably due to the discounted contract price. Mainly influenced by the factor product cost, the total health care expenditure of the DMP period remained nearly the same as compared to the pre-dmp period. The two vendors were also compared. The results showed no difference in utilization or expenditures. Overall, the comprehensive disease management program appears to have little impact on health care resource utilization except for reducing home health service. The expenditure of factor products is the major cost driver for the Florida Medicaid hemophilia recipients. The discounted factor product price appears to offset the cost associated with the increased factor product use. 4

5 Background Agency for Health Care Administration Disease Management Program The Florida Agency for Health Care Administration (AHCA) implements and manages the Florida Medicaid Program. Florida Medicaid is a state and federal partnership that provides health coverage for selected categories of people with low incomes and disabilities. Since 1970, its purpose is to improve the health of people who might otherwise go without medical care for themselves and their children. (AHCA, 2009) In 1998, Florida legislature passed the Medicaid Disease Management Initiative. This initiative offers systematic approaches for managing the health care needs of recipients who are at risk of or diagnosed with a specific disease, using various methods such as best practices, prevention strategies, and outcomes research to reduce overall costs and improve measurable outcomes. This study focuses on the Comprehensive Hemophilia Disease Management Program (DMP). In 1997, the Medicaid Reform Task Force found that approximately 10% of all Medicaid recipients accounted for more than 60% of program expenditures. They found that the average annual cost for a MediPass recipient with hemophilia exceeded $75,000. (Note: MediPass is the care case management program for Medicaid recipients.) (AHCA, 2009) Key components of the DMP are (AHCA, 2000): clinical practice guidelines; provider and recipient profiling; specialized (disease-specific) physician and other practitioner care; intensive care management; provider education; recipient education; claims analyses; quarterly and yearly outcome measurement and reporting. Medicaid identifies eligible recipients for disease management which include all Medicaid eligible recipients who have been prescribed factor replacement products and are enrolled in MediPass, a Minority Physician Network, a Provider Service Network, or Fee-For- Service. Eligible recipients are notified by Medicaid for participation in the program. Eligible recipients must choose one of the two contracted hemophilia disease management providers, but may change between the two providers at any time. Approximately 200 recipients receive services each month from the Hemophilia DMPs. (AHCA, 2009) Epidemiology of Hemophilia Hemophilia Overview Hemophilia is a rare X- linked hereditary bleeding disorder, primarily occurring in males. It has two main types: Hemophilia A (Factor VIII Deficiency) and Hemophilia B, also known as the Christmas Disease (Factor IX Deficiency), both of which can be classified as severe, 5

6 moderate and mild depending on the coagulation factor activity level. Hemophilia A is more prevalent than Hemophilia B. As a result, there are more studies on the treatment of the former. The incidence of hemophilia is about 1 in 5,000 live male births in the United States. All races and economic groups are affected equally. The national population of people living with hemophilia in the United States has been estimated to be 18,000, and 400 children are born each year with the disease (Blankenship, 2008). Disease Progression and Outcomes Hemophilia has a significant negative impact on patient health. Its complications include excessive bleeding, joint disease, severe pain due to bleeding into joints, and risk of infectious diseases transmitted through blood products. If left untreated, health consequences include nerve damage, loss of musculoskeletal function and death (Shapiro, 2007). Children may experience internal bleeding into the joints, muscles and soft tissues as well as nose and mouth bleeds (The Haemophilia Society, 2008). von Willebrand's Disease (vwd) is related to hemophilia (Miners et al, 2009) and mimics Hemophilia A. This is an autosomal inherited disorder in which there is deficiency or dysfunction of von Willebrand factor (vwf). (Treatment of Hemophilia, 2008). Historically, people with hemophilia did not survive past their third decade. Currently, advanced medical care, particularly the introduction of factor replacement therapy, has significantly prolonged life expectancy of people with hemophilia (Soucie et al, 1998). Severity Levels of Hemophilia The three categories for hemophilia severity are based on clotting levels: severe, moderate and mild. The severe form is defined as frequent spontaneous bleedings predominantly associated with the joints and muscles (World Federation, 2005). Specifically, for people with severe hemophilia there is a greater chance of internal bleeding into the joints, muscles and soft tissues (The Haemophilia Society, 2008), which is associated with high expenditures for managing these patients. The moderate form involves occasional spontaneous bleeding although severe bleeding may occur from surgery or trauma; while the mild form can also be associated with severe bleeding from surgery or trauma, but does not incur the other problems noted with severe disease due to better clotting factors. As a result, patients with moderate and mild disease may not be diagnosed until later in life (The Haemophilia Society, 2008). (Note: For the Hemophilia DMP, clotting levels are not used to set severity; severity is associated with frequent bleeding). Treatment Options Prophylactic Treatment In 1952, Professor Nillson in Sweden was one of the first to introduce prophylactic treatments to hemophilia patients (Fischer et al, 2002). Prophylactic treatments are preventive, while on-demand or episodic treatments are administered when bleeding occurs or bruises are 6

7 observed (Berntorp, 2009). Prophylactic treatments appear helpful in preventing joint bleeding and hemophiliac arthropathy. These treatments are mostly administered to those with severe cases of hemophilia to maintain factor levels in the blood and to decrease the incidence of spontaneous bleeding. Mild and moderate hemophilia patient s factor levels are not as critical as severe cases and usually only require on-demand treatments. Prophylactic treatments assist in maintaining factor levels in the blood (Kern et al, 2004), and they have been shown to reduce bleeds and prevent severe joint damage. As a result, timing of prophylactic treatment is critical; however, no age has been specified as the gold standard of care for initiating therapy. Berntorp (2009) followed-up with a group of boys with severe hemophilia and found that those who started prophylactic treatment between the ages of one and two developed normal joints and led normal lives. Other studies agree that treatment initiation should be at a young age based on diagnosis (Fischer et al (2002a), Berntrop (2009), Kern et al (2004)). Prophylactic dosages are based on weight. Berntorp et al (2009) recommends for Hemophilia A, IU of factor VIII per kg body weight and administered three times weekly, and for Hemophilia B, IU of factor IX two times weekly. Prophylactic treatment is strongly encouraged for severe hemophilia patients by the U.S. National Hemophilia Foundation Medical and Scientific Advisory Committee and the World Health Organization (Fischer et al, 2002b). The World Federation of Hemophilia (WFH) created a comprehensive guide based on published consensus guidelines which encourages the prevention of bleeding and the use of prophylactic treatment. Moderate cases for hemophilia may require prophylactic treatment based on the level of factor present in the blood (WFH, 2005). Mild and moderate cases of hemophilia do not necessarily need prophylactic treatment according to these organizations. For those cases, desmopression (DDVAP) is administered when a bleeding event occurs (on-demand therapy) (NHLBI); this is a hormone that releases the stored factor already present in the blood stream. DDAVP is provided to those who have five percent or greater FVIII and are responsive to pre-tests (WFH, 2005). Further, general consensus among studies indicates that prophylactic treatments are effective in ameliorating the effects of hemophilia. Manco-Johnson conducted a multi-center, randomized study, observing the effects of prophylactic treatment versus on-demand care. The study followed boys with Hemophilia A from the ages of less than 30 months to six years of age, comparing prophylactic and on-demand treatment. The study found boys treated with prophylactic treated experienced less joint and total hemorrhages as compared to the on-demand group. Fischer et al (2002) analyzed data from two retrospective studies involving patients with severe disease: on-demand treatment in France and prophylactic treatment in the Netherlands. The findings demonstrated that those with prophylaxis had better health outcomes such as fewer incidents of joint bleeds per year, arthropathy and history of orthopedic surgery. The on-demand group has a greater number of incidents of bodily pain and limited physical functioning and social functioning. Despite the advances in prophylactic treatments, the majority of severe hemophilia patients do not take advantage of this therapy. In 1995, only 33% of all children with hemophilia in the United States received prophylactic treatments (Manco-Johnson, 2007). In 2004, 51.5% children under six years old with severe hemophilia received prophylaxis (Manco-Johnson, 2007). Prophylaxis has been shown to decrease the likelihood of the development of inhibitors in patients compared to patients treated with on-demand treatment (Petrini, 2007); regardless of 7

8 whether plasma-derived or recombinant derived factors are used (Goew et al, 2007). An inhibitor is a type of antibody which attempts to destroy substances, such as following treatment to replenish factor XIII or IX, they do not recognize. The antibody attaches to the factor VIII or IX and inhibits its ability to stop bleeding (WHF, 2009). Now, that so many children with hemophilia have become adults, one argument is to whether or not to continue prophylactic treatment into adulthood in terms of effectiveness. For adults, the prophylactic interventions are secondary rather than primary treatments; the results are better with children from ages 9 to 12 (Tagliaferri et al, 2008). Finally, regardless of whether a patient is treated with prophylactic treatment, occasionally on-demand treatments are necessary for emergent bleeds. On-Demand Treatment Currently, there is more literature on the efficacy of prophylactic treatments than ondemand treatments alone for severe hemophilia patients. This form of treatment is commonly given when the clotting factor concentrate is administered before surgical procedures, after an injury, or once a bleed has started (Haemophilia Society, 2008), or when a bruise is observed, and for mild or moderate cases (NHLBI). On-demand is an acute form of therapy, managing, rather than preventing Hemophilia associated health problems. Among its disadvantages is that one waits until the patient experiences a bleeding event before administering the treatment. Thus patients may have repeated bleeding, which could lead to orthopedic problems such as muscular atrophy and hemophilic arthropathy. (Miners et al, 2009). In the Manco-Johnson study, the ondemand, episodic care group developed life-threatening hemorrhages, including intracranial and gastrointestinal; the prophylactic treated patients did not experience those adverse events. Other, common health problems associated with on-demand treatment are central nervous system (CNS) hemorrhages (Hoots WK, 2007). Inhibitors A major consequence of prophylactic treatment is the development of inhibitors, which is common among Hemophilia A patients (20-30% of severe cases), and occurs in Hemophilia B at five percent or less (Tjønnfjord et al, 2007; The Hemophilia Society, 2008). Antibodies are created against the factor treatment, and may render this form of treatment ineffective. Inhibitors manifest early, approximately within 10 to 20 days of exposure. Causes for inhibitors development include gene mutations, ethnicity, and family history (Abshire, 2007). Upon developing higher inhibitor levels, therapy changes may be made to either provide higher doses of a given product or use of a more expensive activated clotting factor. When high inhibitor levels occur, alternative therapies are available, including NovoSeven, FEIBA, and immune tolerance induction (ITI). NovoSeven, recombinant factor VIIa (rfviia), is a genetically engineered clotting factor that was introduced in the early 1990s (The Haemophilia Society, 2008; Tjønnfjord et al, 2007). FEIBA is a factor eight inhibitor bypassing agent, plasma-derived treatment. Currently, bloodborne pathogens have not been reported with FEIBA (Tjønnfjord et al, 2007). Both of these treatments have been shown to be effective in preventing bleeds but not as effective as non-inhibitor treatments (Tjønnfjord et al, 2007). Immune tolerance induction (ITI), or immune tolerance therapy (ITT) (The Haemophilia Society, 2008), is an alternative treatment option to rid inhibitors. For those with FIX inhibitors, ITI is difficult to implement 8

9 (Mannucci, 2008). This therapy would last for long periods of time, ranging from 9 to 24 months with an 80% success rate (Hemophilia Society, 2008). Generations of recombinant factor therapies have evolved. One of the newer treatment options is recombinant plasma/albubin-free FVIII concentrate (rahf-pfm, Advate). Advate was developed from Chinese hamster ovaries (CHO), thus there may be a risk of inducing antigenicity from non-human (trans-species) sourced products develop inhibitors (Shapiro, 2007). Economic Factors in the Treatment of Hemophilia 1 Hemophilia has become a costly chronic disease because of the life-time dependence on expensive factor products and extra demands for health care resources. It has been reported that the total annual cost of hemophilia care in 1998 was $30,820 for those patients receiving ondemand treatment and $87,865 for patients receiving prophylactic treatment (Globe et al, 2004). The factor prices have not decreased over time. In 2004, the reported median cost for factor prophylactic inhibitor treatments was $55,853/year and non-inhibitor treatments $2,760 per year less (Bohn et al, 2004). Please note that there is no comparison price for prophylactic treatment reported. This study consisted of twelve patients with inhibitors to FVII to FIX (cases) identified in the hemophilia surveillance system (HSS) at two centers were matched on age, severity of hemophilia descriptive matched analysis was conducted to examine the annual differences in cost of product used and hospitalizations. Thus, factor product utilization is the major cost driver, accounting for approximately 45 to 93% of the total health care costs in the treatment of people with hemophilia. Further, the total health care cost of people with hemophilia is influenced by disease severity, inhibitor history, HIV and Hepatitis viral infection, and route of factor product administration (intravenous infusion vs. through a port) (Globe et al, 2003). Costs and Economic Data All hemophilia treatments are costly and represent a significant financial burden for the payers. Different countries have conducted cost analyses to explore the relative total costs of treating patients with prophylactic versus on-demand therapy. Though prophylactic treatment is more costly in terms of drug costs, the overall health of the people with hemophilia appears to be better compared to patients given on-demand treatment, and thus may reduce long-term health care costs. In the United Kingdom, Miners et al. (2009) conducted a cost effectiveness study for prophylactic treatment compared to on-demand treatment for severe Hemophilia A. The analysis showed a mean expected life-time cost of 644,000 ($1,037, USD) for on-demand treatment and 858,000 ($1,382,495.39) for prophylactic therapy, respectively. Quality of Adjusted Life Years (QALY) was reviewed as well, which showed that on-demand therapy was QALY, whereas for prophylactic therapy was 19.58, thus a 5.63 QALY increase. In Canada, a cost study was conducted involving Canadian boys with severe Hemophilia A for a year prior to, and a year after target joint bleeding (Kerns et al, 2004). Target joint bleeding may lead to chronic deformities, leading to severe pain along the joint. Specifically, the 1 Unable to find studies about the Medicaid hemophilia population. 9

10 study focused on the pre- and post-target joint (TJ) to review costs incurred by the patient once TJ occurs when administered prophylactic treatment. The study defined TJ as three bleeds into any single joint within a consecutive three-month period. The enrolled population had an average age of 54 months (range, months), with ankles being most often affected, followed by elbows and knees (46% vs. 28% and 23%, respectively). Each of the boys in the study developed TJ bleeding, as defined by the study protocol, which then increased cost of care. The time period was one year before and after treatment. The pre-tj mean overall cost, included the infusion costs, clinic visits, diagnostic tests/blood work, per boy was $20,091 Canadian Dollar vs. $42,612 (CND) vs. the post-tj, the mean cost was $43,891. Factor VIII use accounted for 87% of the total cost the year before development of a target joint and 93% the year after the target joint was affected. The factor cost was $18,381 pre-treatment vs. $38,538 post-treatment. In the United States, a cost utilization study was performed for 336 Hemophilia A patients, recruited from five comprehensive Hemophilia treatment centers during Nearly half of the study sample was covered by Medicaid, while the others had commercial insurance. (Globe et al, 2004). The study took into account the different cost components of care (e.g. physician visits, factor VIII concentrate, emergency room, hospitalization), and found the total annual cost of care was $139,102, with a mean cost of factor VIII of $130,438 for the entire study sample, in The same study also found annual on-demand costs to be $69,656 for severe and $18,890 per patient for mild and moderate hemophilia. In another U.S. study, Manco-Johnson performed a randomized, clinical, multi-center trial to discover an effective way to prevent arthropathy in people with severe hemophilia in the United States (Manco-Johnson et al, 2007). During the course of this trial, Manco-Johnson found that at a price of $1 per unit of recombinant factor VIII, the cost of prophylaxis for a child weighing 50 kg (110 pounds) could reach $300,000 per year. Patient Adherence to Treatment Plans The patient s ability to follow prescribed plans is a contributing factor to the effectiveness of the treatment. Bentrop et al conducted a study for the hemophilia population in Sweden, based on data from randomized, controlled trials comparing prophylaxis with ondemand treatment. For those who use prophylactic treatment, 90% of patients, ages of 12 and under follow the treatment plans and the percentage drops considerably to 36% for ages 19 to 29 (Berntrop, 2009). Physicians decisions to prescribe the treatments are based on patient s ability to adhere. An option is to ensure patient adherence by tailoring the treatment to individual needs. Additional suggestions include psychosocial training for people with hemophilia to become more independent, and to engage in physical activity. 10

11 Interdisciplinary Provider Team According to the World Federation of Hemophilia, successful treatment of patients with hemophilia involves a team of health care professionals, not just physician contacts. It is recommended by the World Federation of Hemophilia (WFH) that a comprehensive team would include professionals well-versed in bleeding disorders such as hematologists, nurse coordinators, physiotherapists and social workers. In the United States alone, there are 142 federally funded hemophilia treatment centers (HTC) (Linney, 2010). According to Linney, there is a 40% higher mortality rate among those who do not seek treatment from HTCs for the study period as observed in records on 2,950 males reviewed in the study. Treatment plans improve the life expectancy of Hemophilia patients and reduce costs. Comprehensive Hemophilia Disease Management Program in Florida A contract between the Agency for Health Care Administration and two Hemophilia Vendors was executed on January 1, Due to contract implementation, fiscal agent activities, and recipient choice periods, the program became fully operational on June 1, Florida Medicaid started the two-year Comprehensive Hemophilia Disease Management Program (DMP) which was mandatory for all Florida Medicaid non-hmo recipients with at least one pharmacy record for a factor product within the 18 month period prior to identification of eligibility for the program. Florida non-hmo Medicaid recipients with vwd were also eligible for enrollment in the DMP. Florida Medicaid HMO recipients were excluded from the program, and dual eligible recipients (Medicaid and Medicare) were enrolled voluntarily. Participants in the DMP chose either Caremark or CuraScript as their service provider for comprehensive care, including disease management services, care management, 24/7 toll-free access to a nurse/pharmacist helpline, physician and recipient education, and dispensing/home delivery of factor products. Florida Medicaid reimburses both providers at Average Wholesale Price minus 39% (AWP-39%) for the factor products they dispense to DMP participants. The care management component of the program is provided by the vendors at no cost to the state or recipients. Methods Data Sources Two data sources were used in the data analysis: (1) participant records provided by the DMP vendors; and (2) Florida Medicaid claims database. Vendor-provided participant records were available for the DMP period which were used to identify disease type and disease severity. The Florida Medicaid claims database provides comprehensive data including health care resource utilization and associated costs during both the pre-dmp and DMP periods. The pre- DMP period was defined as the one-year period immediately prior to the DMP program implementation (June 1, 2007 to May 31, 2008). The DMP period was defined as the one-year period that participants actually received DMP interventions following the completion of their enrollment (June 1, 2008 to May 31, 2009). The demographic information was summarized for 11

12 age, gender, race, disease type and co-infections of HIV/AIDS or Hepatitis. Based on the information available in the claims data, the following utilization and cost categories were examined: Total health care resource utilization and cost for the following categories: hospital inpatient (hospital admission and length of hospitalization) emergency room visit outpatient visit physician office service home health service pharmacy (number of prescription fillings regardless of type of medications) other health care service (health care service that did not belong to any categories above, such as dental, laboratory, physical therapy, etc). Hemophilia-related health care resource utilization and cost for the following categories: hospital inpatient (hospital admission and length of hospitalization) emergency room visit outpatient visit physician office visit home health service factor product amount dispensed See Appendix A for the ICD-9-CM codes for hemophilia claims and co-infections. See Appendix B for NDC codes used for factor product identification (MOE or MOF classes). See Appendix C for the detailed coding algorithms for each outcome of interest. Analysis Descriptive analyses were conducted to characterize the Florida Medicaid hemophilia population with regard to the patterns and frequencies of health care events and expenditures. Next, the overall DMP program was evaluated by using a pre-post study design. Finally, the, two DMP vendors were compared against each other. Descriptive Analysis The primary descriptive analyses consisted of all recipients with hemophilia enrolled in Florida Medicaid non-hmo program for at least one month between June 1, 2007 and May 31, The recipients with hemophilia were identified by AHCA according to pharmacy claims of factor products (MOE or MOF classes). Findings from the claims data are presented for the twoyear study period, as well as the one-year pre-dmp period (June 1, 2007 to May 31, 2008) and the one-year DMP period (June 1, 2008 to May 31, 2009). Since Florida Medicaid recipients could drop in and out of the Medicaid program at any time during the observation period, the findings for both utilization and costs were summarized on the basis of the annual total and per member per month (PMPM), (i.e. 20,000 factor product units PMPM and $2,000 PMPM for factor product costs). All cost values were adjusted to the Florida Medicaid fiscal year , based on the Florida Medicaid specific inflation factors provided by AHCA. 12

13 Evaluation of the Overall DMP A pre-post design was implemented to evaluate the DMP program. Each patient served as his/her own control (self-controlled, case series design), which helped to adjust for factors that did not change over time, such as disease severity and chronic co-morbid conditions. Another advantage of this design is its capability to answer questions about changes over time for the outcomes of interest. Florida Medicaid non-hmo Hemophilia enrollees were included in this analysis only if they had Continuous Florida Medicaid non-hmo enrollment for at least 12 months immediately prior to the DMP period (June 1, 2007 May 31, 2008), and Continuous Florida Medicaid non-hmo DMP enrollment for 12 months during the DMP period (June 1, 2008 May 31, 2009). Evaluation of the DMP Vendor A concurrent, non-equivalent comparison group design was used to compare the two DMP vendors, Caremark and CuraScript. Florida Medicaid non-hmo Hemophilia participants were included if they had Continuous Florida Medicaid DMP enrollment for 12 months during the DMP period (June 1, 2008 May 31, 2009). No vendor switching during the DMP period (June 1, 2008 May 31, 2009). Continuous Florida Medicaid non-hmo enrollment for one year immediately prior to the DMP period (June 1, 2007 May 31, 2008). The purpose of this criterion is to collect baseline demographic and clinical characteristics. Statistical Analysis Plan See Appendix D for descriptions of the statistical testing procedures used for the DMP evaluation. Findings Part I. Descriptive Analysis This section describes all identified recipients with hemophilia who enrolled in the Florida Medicaid non-hmo program for at least one month between June 1, 2007 and May 31, 2009 in terms of their demographic characteristics, health care resource utilization, and cost. Demographic Characteristics The demographic characteristics of the study population are summarized in Table 1a. A total of 288 Florida Medicaid recipients were included in the descriptive analysis. These individuals contributed a total of 5,006 member-months of observation during the two-year study period. Analyses were also conducted for the pre-dmp and DMP periods, separately. Of the 288 recipients, 281 of these recipients received care during the pre-dmp period between June 1, 2007 and May 31, 2008, (2,880 member-months). These recipients would have participated in 13

14 the DMP had the program been available. Further, 241 recipients (2,126 member-months) of the 288 recipients were provided care during the DMP period. They were the actual DMP participants. Gender: Most enrollees were male in the entire population. The proportion was similar for both the pre-dmp (89.0%) and DMP period (90.5%). Age: The average age in the entire population was 13.0 years old (SD=11.4). The median age was 10.4 years old (IQR=10.4). Approximately 80% recipients were 18 years old or younger. The age distribution was similar for both the pre-dmp and DMP period. Race: In the entire population, Whites accounted for 29.9%, Blacks accounted for 14.2%, Hispanics counted for 26.0%, and other races accounted for 29.9%. The race distributions were similar for both the pre-dmp and DMP periods. Disease Type: Hemophilia A was the most prevalent disease type, accounting for 68.8% in the two year period for the entire population. The prevalence rates of Hemophilia B and vwd were 13.9% and 13.5%, respectively. Only 3.8% recipients had other clotting disorders, such as factor X deficiency. The disease type distributions were similar for both the pre-dmp and DMP period. Co-infections: A small proportion of the recipients had infection history of HIV/AIDS (2.1%), Hepatitis 4.9%), or both (2.4%). The distributions were similar for both the pre-dmp and DMP period. 14

15 Table 1a. Demographics and Co-infections of Florida Hemophilia Population (Overall) 2-Year Period Pre-DMP Period DMP Period Medicaid Hemophilia Population (06/01/ /31/2009) (06/01/ /31/2008) (06/01/ /31/2009) Total Recipients % % % Member-Month (Average per member) Gender (%) Male % % % Female % % % Average (Year) SD Median (Year) IQR Years (%) % % % Age 5-10 Years (%) % % % Years (%) % % % Years (%) % % % (%) % % % 60 (%) 2 0.7% 2 0.7% 2 0.8% White (%) % % % Race Black (%) % % % Hispanic (%) % % % Other (%) % % % Hemophilia A % % % Disease Type Hemophilia B % % % vwd % % % Other Clotting Disorder % % % HIV/AIDS only 6 2.1% 7 2.5% 5 2.1% Co-infection Hepatitis only % % 8 3.3% Both 7 2.4% 6 2.1% 3 1.2% 1 Other includes Asians, Native Americans and any other unspecified races Table 1b compares the demographic characteristics between two vendor groups, Caremark and CuraScript, during the DMP period. There were 117 participants served by Caremark for at least one month. They contributed a total of 926 member-months. There were 133 participants served by CuraScript for at least one month. They contributed a total of 1,200 member-months. Nine participants switched between these two vendors during the DMP period and contributed to both groups. 15

16 Table 1b. Demographics and Co-infections of Florida Hemophilia Population by Vendor Medicaid Hemophilia Population Caremark CuraScript Total Participants member-month membermonth Gender Male % % (%) Female % 6 4.5% Average (Year) SD 13.8 (12.0) 13.3 (11.1) Median(Year) IQR 12.0 ( ) 10.7 ( ) 0-4 Years (%) % % Age 5-10 Years (%) % % Years (%) % % Years (%) % % (%) % % 60 (%) 2 1.7% 0 0.0% White (%) % % Race Black (%) % % Hispanic (%) % % Other (%) % % Mild % 9 6.8% Moderate 4 3.4% % Hemophilia A Severe % % Unknown 6 5.1% 3 2.3% Total % % Disease Mild 6 5.1% 5 3.8% Type Moderate 8 6.8% 2 1.5% Hemophilia B Severe 9 7.7% 5 3.8% Unknown 0 0.0% 2 1.5% Total % % vwd % % Other Clotting Disorder 3 2.6% 7 5.3% 1 Nine patients switched vendors during the one-year period and are included in the findings of each group. Gender: Most participants were male for both vendor groups. Caremark had more female participants than CuraScript (14.5% vs. 4.5%). Age: The Caremark group was slightly older than the CuraScript group. The average age was 13.8 years old vs years old. The median age was 12.0 years old vs years old. Caremark participants were more likely to be above 18 years old (24.8% vs. 18.8%), and less likely to be below 4 years old (17.9% vs. 26.3%) than CuraScript participants. Race: Caremark had a larger proportion of Hispanics (34.2% vs. 16.5%) and a smaller proportion of Whites (22.2% vs. 34.6%) than CuraScript. The proportion of blacks and other races were nearly the same between the two vendors. 16

17 Disease Type and Severity: Hemophilia A was the most prevalent disease type for both vendor groups. The prevalent rate of Hemophilia A was relatively lower in the Caremark group than the CuraScript group (59.8% vs %). Caremark had relatively higher prevalent rates of Hemophilia B (19.6% vs. 10.6%) and vwd (17.1% vs. 9.0%) than CuraScript. Taking both hemophilia types together, 49.6% Caremark and 60.2% CuraScript participants had diseases at severe level. Health Care Resource Utilization The total and hemophilia-related health care resource utilizations were examined for all recipients with hemophilia who were enrolled in Florida Medicaid non-hmo program for at least one month between June 1, 2007 and May 31, The analyses were performed for the two year study period as a whole, as well as the pre-dmp period (June 1, 2007 May 31, 2008) and the DMP period (June 1, 2008 May 31, 2009) separately. Total health care resource utilization The descriptive analysis findings on total health care resource utilization for the 288 Florida Medicaid non-hmo enrollees are summarized in Tables 2a. Health Care Resources Utilization and Expenditure in Florida Non-HMO Hemophilia Population Table 2a. Total Health Care Resource Utilization 2-year Period 1 Pre-DMP Period 2 DMP Period 3 Medicaid Hemophilia Population (06/01/ /31/2009) (06/01/ /31/2008) (06/01/ /31/2009) Total PMPM Total PMPM Total PMPM Inpatient Hospital Admission Hospitalization Day 1, Emergency Visit Outpatient Visit 1, Physician Office Visit 2, , , Home Health Service 3, , Pharmacy Claims 10, , , Other Service 9, , , Based on 288 patients and 5,006 member-months 2 Based on 281 patients and 2,880 member-months 3 Based on 241 patients and 2,126 member-months Hospital Inpatient: o There were a total of 311 hospital admissions (0.06 PMPM) and 1,509 hospitalization days (0.3 days PMPM) during the two-year study period. o The average monthly rates were similar between the pre-dmp and DMP periods. The hospital admission rate was 0.06 PMPM vs PMPM. The hospitalization day rate 17

18 was slightly lower in the pre-dmp period than the DMP period, which was 0.27 PMPM vs PMPM. Emergency Room Visits: o There were 580 emergency room visits during the two-year study period. The overall average monthly rate was 0.12 PMPM. o The average rate monthly was similar for the pre-dmp and DMP periods, which was 0.11 PMPM vs PMPM. Outpatient Visits: o There were 1,845 Outpatient visits during the two-year study period. The overall average monthly rate was 0.37 PMPM. o The average monthly rate was slightly lower for the pre-dmp period than the DMP period (0.34 PMPM vs PMPM). Physician Office Visit: o There were 2,828 physician office visits during the two-year study period. The overall average monthly rate was 0.56 PMPM. o The average monthly rate was similar between the pre-dmp and DMP periods, 0.59 PMPM vs PMPM. Home Health Services: o There were a total of 3,266 home health services provided to the study population during the two-year period. The overall average monthly rate was 0.65 PMPM. o The total home health service utilization reduced significantly, from 2,399 events in the pre-dmp period to 867 events in the DMP period. The average monthly rate dropped approximately 50% from 0.83 PMPM to 0.41 PMPM. Pharmacy: o There were 10,789 prescription claims during the two-year study period. The overall average monthly rate was 2.16 PMPM. o The average monthly rate was similar between the pre-dmp and DMP period, which was 2.08 PMPM vs PMPM. Other Services: o There were 9,635 other health care services provided during the two-year study period, which did not belong to any categories above. The overall average monthly rate was 1.92 PMPM. o The average monthly rates were 1.85 PMPM in the pre-dmp period and 2.02 PMPM in the DMP period. 18

19 Hemophilia-related health care resource utilization The descriptive analysis findings on hemophilia-related health care resource utilization were summarized in Table 2b. Medical claims were included only if the primary diagnosis codes were related to hemophilia, vwd or bleeding disorders. Pharmacy claims were included for analysis only if the prescribed medications were for factor products. Medicaid Hemophilia Population Health Care Resources Utilization and Expenditure in Florida Non-HMO Hemophilia Population Table 2b. Hemophilia-Related Health Care Resource Utilization 2-year Period 1 Pre-DMP Period 2 DMP Period 3 (06/01/ /31/2009) (06/01/ /31/2008) (06/01/ /31/2009) Total % % % within within PMPM Total PMPM Total within Category Category Category PMPM Inpatient Hospital Admission % % % 0.01 Hospitalization Day % % % 0.05 Emergency Visit % % % 0.02 Outpatient Visit 1, % % % 0.25 Physician Office Visit 1, % % % 0.20 Specialist Hematologist Orthopedist Home Health Service 2, % , % % 0.30 Factor Product Units 79,074, , ,757, , ,317, , Based on 288 patients and 5,006 member-months 2 Based on 281 patients and 2,880 member-months 3 Based on 241 patients and 2,126 member-months Hemophilia-Related Hospital Inpatient: o There were a total of 66 hemophilia-related hospital admissions (0.01 PMPM) during the two-year study period, which accounted for 21.2% total hospital admissions. o There were 253 hemophilia related hospitalization days (0.05 days PMPM) during the two-year study period, which accounted for 16.8% total hospitalization days. o The average monthly rates were similar between the pre-dmp and DMP periods. The hospital admission rate was 0.01 PMPM for both periods. The hospitalization day rate was 0.05 PMPM for both periods. Hemophilia-Related Emergency Room Visits: o There were 82 hemophilia-related emergency room visits during the two-year study period with an overall average monthly rate of 0.02 PMPM, which accounted for 14.1% total emergency room visits. o The monthly rate in the pre-dmp period appeared slightly lower than that in the DMP period, which was 0.01 PMPM vs PMPM. Hemophilia-Related Outpatient Visits: o There were 1,007 outpatient visits during the two-year study period with an overall monthly average of 0.20 PMPM, which accounted for 54.6% total outpatient visits. o The average rate was slightly lower in the pre-dmp than the DMP period, which was 0.17 PMPM vs PMPM. 19

20 Hemophilia-Related Physician Office Visits: o There were 1,070 physician office visits during the two-year study period with an overall average monthly rate of 0.21 PMPM, which accounted for 37.8% total physician office visits. o The average rate was similar between the pre-dmp and the DMP period, which was 0.22 PMPM vs PMPM. Specialist Visits: o There were 172 hematologist visits during the two-year study period with an overall average monthly rate of 0.03 PMPM in the pre-dmp and 0.04 PMPM in the DMP periods. o There were 69 orthopedic surgeon visits during the two-year observation period. These visits all occurred during the pre-dmp period. No such visits occurred during the DMP period. Hemophilia-Related Home Health Services: o There were 2,481 hemophilia related home health services during the two-year study period with an overall average monthly rate of 0.5 PMPM, which accounted for 76.0% of total home health services. o The average rate decreased approximately 50% from 0.64 PMPM in the pre-dmp to 0.30 PMPM in the DMP period. Factor Product: o More than 79 million factor product units were dispensed during the two year study period with an overall average rate of 15,796 units PMPM. o The average rates were lower in the pre-dmp than that in the DMP period, which was 14,151 units PMPM vs. 18,023 units PMPM. Health Care Expenditures The total and hemophilia-related health care expenditures were examined. All expenditures were adjusted to fiscal year values based the Florida Medicaid inflation factors that AHCA provided. 20

21 Total health care expenditure 2c. The descriptive analysis findings on total health care expenditures are summarized in Table Medicaid Hemophilia Population Health Care Resources Utilization and Expenditure in Florida Non-HMO Hemophilia Population Table 2c. Total Health Care Expenditure ($) 1 2-year Period 2 Pre-DMP Period 3 DMP Period 4 (06/01/ /31/2009) (06/01/ /31/2008) (06/01/ /31/2009) Total % % Total PMPM % Total PMPM Total Total Total Cost Cost Cost Cost Cost PMPM Total 91,688, % 18,316 49,985, % 17,356 41,702, % 19,616 Inpatient 2,880, % 575 1,467, % 510 1,413, % 665 Emergency Visit 233, % , % 48 94, % 45 Outpatient Visit 527, % , % , % 112 Physician Office Visit 173, % , % 37 66, % 31 Home Health Service 181, % , % 45 52, % 24 Pharmacy 86,124, % 17,204 46,945, % 16,300 39,179, % 18,429 Other Service 1,273, % , % , % 257 Cost Per Bucket Category 1 All expenditures were adjusted to fiscal year values 2 Based on 288 patients and 5,006 member-months 3 Based on 281 patients and 2,880 member-months 4 Based on 241 patients and 2,126 member-months See Appendix E Total Health Care Cost: o Approximately, $91.7 million were spent for the 288 recipients during the two-year study period with an overall average monthly cost of $18,316 PMPM, ranging from $3 PMPM to $335,030 PMPM. o The average monthly cost was approximately $2,000 lower during the pre-dmp than the DMP period, which was $17,356 PMPM vs. $19,616 PMPM Pharmacy Cost: o Pharmacy cost was the largest cost category in this hemophilia population. A total of $86.6 million were spent during the two-year study period, accounting for about 94% of the total health care cost. o The average monthly total cost was also lower in the pre-dmp than the DMP period, which was $16,300 PMPM vs. $18,429 PMPM. Hospital Inpatient Cost: 21

22 o Hospital inpatient cost was the second largest cost category, accounting for 3.1% of the total health care cost. Approximately, $2.9 million were spent for hospital inpatient services in this population during the two-year study period. o The average monthly cost was $510 PMPM for the pre-dmp and $665 PMPM for the DMP period. Other Cost Categories: o The other cost categories collectively accounted for only about 4% of the total health care cost, including emergency visits, outpatient visits, physician office visits, home health services, and other services. Hemophilia-related health care expenditure The descriptive analysis findings on hemophilia-related health care expenditures are summarized in Tables 2d. Total Hemophilia-Related Health Care Cost: o More than 94% of the total health care costs were hemophilia-related. o The average monthly cost was $16,380 during the pre-dmp and $18,534 during the DMP period. Factor Product Cost: o Factor product cost was the main cost driver for this hemophilia population. A total of $86.6 million were spent during the two-year study period, accounting for about 92.9% of the total health care cost, 98.5% of the total hemophilia-related health care costs, and 99.0% of the total pharmacy cost. o The average monthly cost was $16,131 PMPM in the pre-dmp and $18,233 PMPM in the DMP period. Other Hemophilia-Related Cost Categories: The other hemophilia related cost categories collectively accounted for only approximately 1.5% of the total hemophilia-related health care cost, including hemophilia related hospital inpatient, emergency visit, outpatient visit, physician office visits, home health service, hematologist visits, orthopedic surgeon visits, and others. 22