ALBERT

Description: Introduction: The standard of care for patients with hemophilia A without inhibitors is factor VIII (FVIII) replacement therapy. The availability of non-factor therapy such as emicizumab (Hemlibra®; Genentech, Inc., South San Francisco, CA, USA) is changing the treatment landscape. A model was developed from the perspective of the US healthcare system to compare the cost-effectiveness of recombinant FVIII (rFVIII) products (standard half-life [SHL] and extended half-life [EHL]) versus non-factor therapy emicizumab in the treatment of patients with severe hemophilia A without inhibitors. Methods: The Markovian model used in this analysis included 5 mutually exclusive hemophilia A-related health states: with/without target joints (TJs), with/without arthropathy, and death. Health states changed or remained constant, depending on bleeds and recovery probability. Transition to the health state "death" was derived from mortality rates in the general US population according to age. Each health state was associated with costs and utilities that were summed over time. Estimated total costs and treatment effectiveness as measured by quality-adjusted life years (QALYs; a measure of health outcome that incorporates the impact on both quantity and quality of life) for each health state were then used to calculate incremental cost-effectiveness ratios for pairwise comparisons of treatments (prophylaxis or on-demand for a pooled analysis of 6 rFVIII products [SHL and EHL] vs emicizumab prophylaxis; Table 1) over a life-time horizon. Patient data used in the model were based on a systematic literature review and clinical trial results, and network meta-analysis. Model parameters included patient baseline characteristics, change in body weight by age, prior prophylaxis or on-demand treatment, annualized bleeding rate (ABR), probability of developing or resolving TJs or arthropathy, mortality, number infusions per bleed, medical check-ups, and hospitalization. Based on these data, patients in the model were assumed to be male and 33 years of age at baseline; 72.0% had TJs and 58.4% had arthropathy. Probability of developing TJs per joint bleed was estimated to be 0.9% (prophylaxis and on-demand treatment). Probability of developing arthropathy per joint bleed was estimated to be 2.2% for patients who received prior on-demand treatment and patients who received prior prophylaxis were assumed to have no risk of developing arthropathy. The model assumed life-long adherence to the same prophylactic hemophilia treatment. Drug cost was based on 2018 average sales price and dosing was on label. The model did not include treatment-specific adverse events or inhibitor development. Results: Prophylaxis with rFVIII (SHL and EHL) was estimated to be less costly and more effective (total $13,656,238; QALYs 17.61) versus non-factor prophylaxis with emicizumab (total $16,447,843; QALYs 17.58) over an estimated 70-year lifespan of a patient with severe hemophilia A, which suggests rFVIII prophylaxis is an economically preferable strategy. Total cost consists of costs directly related to prophylactic treatment (rFVIII $12,850,894 vs emicizumab $15,555,379) and costs associated with healthcare resources (rFVIII $805,344 vs emicizumab $892,464). rFVIII prophylaxis was also estimated to be less costly and more effective (total $13,656,238; QALYs 17.61) versus on-demand rFVIII treatment (total $13,823,123; QALYs 12.29). Conclusions: In this pooled analysis of select SHL and EHL rFVIII products, the results suggest that rFVIII prophylaxis is a cost-effective long-term intervention for patients with severe hemophilia A without inhibitors compared with non-factor prophylaxis with emicizumab and on-demand rFVIII treatment. Disclosures Sun: Shire US Inc., a Takeda company: Employment, Other: a Takeda stock owner. Wu:Shire US Inc., a Takeda company: Employment, Other: a Takeda stockowner. McDermott:BresMed Health Solutions Ltd.: Employment. van Keep:BresMed Health Solutions Ltd.: Employment.

Description: Background: Von Willebrand disease (VWD) is the most common inherited bleeding disorder (clinically symptomatic prevalence rate ~1:10,000). Patients with VWD have impaired hemostasis due to a quantitative or qualitative deficit in von Willebrand Factor (VWF) that alters platelet adhesion and collagen binding and/or decreases FVIII concentrations. Most patients with VWD present with mild-to-moderate mucosal bleeding (epistaxis, menorrhagia), although life-threatening bleeding may also occur, especially in patients with VWD type 3 and some forms of VWD type 2. While bleeds associated with VWD have been well described in the literature, information on the burden associated with major bleeding events (MBE) is limited. Real-world data can be used to help understand the clinical and economic impact of these events. Aims: To estimate the prevalence, healthcare resource utilization (HCRU), and costs associated with MBE among patients with VWD. Methods: Patients with VWD with ≥1 year of continuous enrollment since the eligibility start date were identified from Truven databases (01/2008-12/2016). Patients with MBEs were identified using a medical claim associated with an ICD-9/10 CM diagnosis code for intracranial, GI, or eye bleed; or menorrhagia, epistaxis, and joint bleeds that required red blood cell transfusion in an inpatient (IP) setting or within 7 days of diagnosis in an outpatient (OP) setting. Prevalence was calculated as the proportion of eligible patients with ≥1 MBE during the observation period (from start to the end of continuous eligibility). To evaluate economic burden, patients with ≥1 MBE on or after the first diagnosis of VWD were compared with patients with no MBEs. HCRU and cost in the 12-month continuous enrollment period following the first MBE were compared with those from a similar 12-month period for patients without MBEs. Regression models were used, controlling for demographics, health plan, index year, Charlson Comorbidity Index (CCI), comorbidities, thrombotic events, and HCRU during the 12-month continuously enrolled baseline period. For patients with MBEs, the proportion of patients with comorbidities was compared between the 12-month baseline and study periods using McNemar test. Results: 19,785 VWD patients were identified (mean age 34 years, 75% female) During a median observation of 4 years, 15.1% of patients experienced ≥1 MBE (mean rate: 0.11 ± 0.64 MBE/year). GI bleeding was the most prevalent MBE, occurring in 13.4% of all patients. Although not common, the prevalence of intracranial bleeds (1.1%) was slightly higher in males than females (1.7% vs 0.9%). In the sample to evaluate economic burden, 773 patients with ≥1 MBE (age 44.5 ± 20.1 years) and 4285 patients without MBEs (age 34.2 ± 19.5 years) were selected. Patients with MBEs were significantly (p

Description: Background: von Willebrand disease (VWD), a rare, inherited bleeding disorder, is associated with impaired hemostasis resulting from a quantitative or qualitative deficit in von Willebrand factor. Limited information exists on the economic burden associated with major surgeries in this patient population, and real-world data may help determine the impact of these procedures. Aims: To estimate the incremental economic burden associated with major surgeries in patients with VWD compared with patients without VWD who had similar types of surgery. Methods: Accessing data from the Truven US health care database (January 2008 to December 2018), we analyzed data from patients with VWD (based on ≥2 diagnoses from different hospital admissions/physician visits, excluding laboratory and radiology orders) and patients without VWD who had undergone a major surgical procedure. The surgical procedure was defined as a medical claim associated with a major therapeutic operating room procedure (International Classification of Diseases, 9th/10th revision codes) or a major procedure (Current Procedural Terminology code). For patients with VWD, the surgical procedure had to have occurred on or after their first VWD diagnosis. Patients without VWD who had undergone major surgeries were selected from a 1% random sample of the Truven database. Patients from both groups (ie, VWD and non-VWD) were included in the study if they had continuous health care plan enrollment ≥12 months prior to (baseline period) and ≥12 months following (study period) their first major surgery, no diagnosis of acquired coagulation factor deficiency, and not undergone surgery used to reduce bleeding associated with VWD (ie, uterine ablation, nasal ablation, or hysterectomy). Patients with VWD were matched (1:1) with patients without VWD using propensity score matching. Health care resource utilization (HCRU: inpatient [IP] admission, emergency room [ER] visits, and outpatient [OP] visits) and associated costs (pharmacy or medical; adjusted to 2018 US dollars [USD] using the medical component of the Consumer Price Index) were measured over the 12-month study period. Adjusted analyses controlling for age, sex, region, health plan, index year, Charlson Comorbidity Index (CCI), comorbidity profile (anemia, anxiety, depression, fatigue, and obesity), and baseline HCRU were conducted using generalized linear regression models. Results: After propensity score matching, 2972 patients with VWD and 2972 patients without VWD who had ≥1 major surgery were selected for analysis (mean [SD] age, 40.53 [20.56] and 40.94 [20.33] years, respectively; female, 73.3% and 73.6%, respectively). Mean (SD) CCI was 0.66 (1.26) and 0.64 (1.30), respectively; and anemia, anxiety, depression, fatigue, and obesity were present in a similar proportion of each group (7−18% of patients with or without VWD). The most common major surgeries were musculoskeletal or digestive in patients with or without VWD (39.6% and 25.0% vs 37.1% and 23.4%, respectively). Patients with VWD were significantly (P