Economic and clinical burden of managing transfusion-dependent ß-thalassemia in the United States.

AIMS: To describe clinical complications, treatment use, healthcare resource utilization (HCRU), and costs among patients with transfusion-dependent ß-thalassemia (TDT) in the United States. MATERIALS AND METHODS: Merative MarketScan Databases were used to identify patients with ß-thalassemia between 1 March 2010, and 1 March 2019. Patients were eligible for inclusion with ≥1 inpatient claim or ≥2 outpatient claims for ß-thalassemia and ≥8 red blood cell transfusions (RBCTs) during any 12-month period after and including the date of the first qualifying ß-thalassemia diagnosis code. Matched controls consisted of individuals without ß-thalassemia. Clinical and economic outcomes of patients were assessed during ≥12 months of follow-up, defined as the period from the index date (i.e. the first RBCT) to either the end of continuous enrollment in benefits, inpatient death, or 1 March 2020. RESULTS: Overall, 207 patients with TDT and 1035 matched controls were identified. Most patients received iron chelation therapy (ICT) (91.3%), with a mean of 12.1 (standard deviation [SD] = 10.3) ICT claims per-patient-per-year (PPPY). Many also received RBCTs, with a mean of 14.2 (SD = 4.7) RBCTs PPPY. TDT was associated with higher annual ($137,125) and lifetime ($7.1 million) healthcare costs vs. matched controls ($4183 and $235,000, respectively). Annual costs were driven by ICT (52.1%) and RBCT use (23.6%). Patients with TDT had 7-times more total outpatient visits/encounters, 3-times more prescriptions, and 33-times higher total annual costs than matched controls. LIMITATIONS: This analysis may underestimate the burden of TDT, as indirect healthcare costs (e.g. absenteeism, presenteeism, etc.) were not included. Results may not be generalizable to patients excluded from this analysis, including those with other types of insurance or without insurance. CONCLUSIONS: Patients with TDT have high HCRU and direct healthcare costs. Treatments that eliminate the need for RBCTs could reduce the clinical and economic burden of managing TDT.

Economic and Clinical Burden of Managing Sickle Cell Disease with Recurrent Vaso-Occlusive Crises in the United States.

INTRODUCTION: The aim of this study was to describe the clinical complications, treatment use, healthcare resource utilization (HCRU), and costs among patients with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) in the US. METHODS: Merative MarketScan Databases were used to identify patients with SCD with recurrent VOCs from March 1, 2010, to March 1, 2019. Inclusion criteria were ≥ 1 inpatient or ≥ 2 outpatient claims for SCD and ≥ 2 VOCs per year in any 2 consecutive years after the first qualifying SCD diagnosis. Individuals without SCD in these databases were used as matched controls. Patients were followed for ≥ 12 months, from their second VOC in the 2nd year (index date) to the earliest of inpatient death, end of continuous enrollment in medical/pharmacy benefits, or March 1, 2020. Outcomes were assessed during follow-up. RESULTS: In total, 3420 patients with SCD with recurrent VOCs and 16,722 matched controls were identified. Patients with SCD with recurrent VOCs had a mean of 5.0 VOCs (standard deviation [SD] = 6.0), 2.7 inpatient admissions (SD 2.9), and 5.0 emergency department visits (SD 8.0) per patient per year during follow-up. Compared to matched controls, patients with SCD with recurrent VOCs incurred higher annual ($67,282 vs. $4134) and lifetime ($3.8 million vs. $229,000 over 50 years) healthcare costs. CONCLUSION: Patients with SCD with recurrent VOCs experience substantial clinical and economic burden driven by inpatient costs and frequent VOCs. There is a major unmet need for treatments that alleviate or eliminate clinical complications, including VOCs, and reduce healthcare costs in this patient population.

Overuse of corticosteroids in patients with immune thrombocytopenia (ITP) between 2011 and 2017 in the United States.

Corticosteroids (CSs) are standard first-line therapy for immune thrombocytopenia (ITP). Prolonged exposure is associated with substantial toxicity; thus guidelines recommend avoidance of prolonged CS treatment and early use of second-line therapies. However, real-world evidence on ITP treatment patterns remains limited. We aimed to assess real-world treatment patterns in patients with newly-diagnosed ITP, using two large US healthcare databases (Explorys and MarketScan) between January 1, 2011 and July 31, 2017. Adults with ITP, ≥12 months of database registration prior to diagnosis, ≥1 ITP treatment, and ≥1 month enrollment following initiation of first ITP treatment were included (n = 4066 Explorys; n = 7837 MarketScan). Information on lines of treatment (LoTs) was collected. As expected, CSs were the most common first-line treatment (Explorys, 87.9%; MarketScan, 84.5%). However, CSs remained by far the most common treatment (Explorys ≥77%; MarketScan ≥85%) across all subsequent LoTs. Second-line treatments such as rituximab (12.0% Explorys; 24.5% MarketScan), thrombopoietin receptor agonists (11.3% Explorys; 15.6% MarketScan), and splenectomy (2.5% Explorys; 8.1% MarketScan) were used much less frequently. CS use is widespread in the US in patients with ITP across all LoTs. Quality improvement initiatives are needed to reduce CS exposure and bolster use of second-line treatments.

Comparing the burden of illness in patients with alopecia areata vs atopic dermatitis in the US population from a payer perspective.

BACKGROUND: Alopecia areata (AA) is an autoimmune disease characterized by nonscarring hair loss. AA frequently co-occurs with other inflammatory autoimmune conditions, presenting a significant clinical burden. OBJECTIVE: To compare the burden of illness, direct and indirect costs in adult patients with AA vs atopic dermatitis (AD). METHODS: This retrospective cohort study used US administrative claims data from the Merative MarketScan Commercial Claims and Encounters Database to compare commercially insured adults with AA to those with AD. Patients with an AA diagnosis between January 2017 and September 2019 were propensity score matched to patients with AD. Comorbidity burden, medication use, health care resource utilization, health care costs, and indirect costs during a 12-month follow-up period were compared between cohorts. RESULTS: Overall, 25,446 adult patients with AA were selected for the matched analysis with the AD cohort. Patients with AA generally had lower comorbidity burden than patients with AD; mean Deyo-Charlson Comorbidity Index scores were 0.36 (SD = 0.99) and 0.39 (SD = 0.92), for AA and AD, respectively (P = 0.007). Patients with AA had significantly lower proportions of allergic rhinitis, asthma, pruritus, skin infections, and urticaria, but higher proportions of thyroid disease, when compared with patients with AD (all P < 0.001). A smaller proportion of patients with AA had prescriptions for topical (45.3% vs 64.8%; P < 0.001) and oral (20.3% vs 29.6%; P < 0.001) corticosteroids and antianxiety and/or antidepressants (24.7% vs 29.7%; P < 0.001), but a significantly larger proportion for intralesional corticosteroids (triamcinolone) (49.6% vs 21.7%; P < 0.001), compared with patients with AD. Despite a lower comorbidity burden and generally less medication usage in patients with AA, total all-cause health care costs did not significantly differ between the AA and AD cohorts ($10,705 vs $10,816; P = 0.712), and outpatient costs were higher in patients with AA ($6,297 vs $5,859; P = 0.014). Female patients with AA had significantly greater costs for both outpatient and outpatient pharmacy when compared with female patients with AD. Patients with AA were more likely to have a claim for long-term disability (0.6% vs 0.3%; P = 0.001) and higher long-term disability-associated indirect costs ($73 [SD = $1,442] vs $25 [SD = $774]; P = 0.004) compared with patients with AD. CONCLUSIONS: We found similar total health care costs in patients with AA and AD, despite a lower proportion of comorbidities and prescription use in patients with AA. Outpatient costs were also significantly higher overall in patients with AA. Although often dismissed as a cosmetic condition, AA, an autoimmune disease, has a similar level of medical expenditure as AD. DISCLOSURES: This study was funded by Eli Lilly and Company. Mr Fenske and Drs Ding, Morrow, and Smith are employed by Eli Lilly and Company. Drs Manjelievskaia, Moynihan, and Silver are employed by Merative. Drs Manjelievskaia, Moynihan, and Silver were employed by IBM Watson Health at the time of study completion. IBM Watson Health received funding from Eli Lilly and Company to conduct this study.

Retrospective Cohort Study of the 12-Month Epidemiology, Treatment Patterns, Outcomes, and Health Care Costs Among Adult Patients With Complicated Urinary Tract Infections.

Background: Limited data are available in the United States on the 12-month epidemiology, outpatient (OP) antibiotic treatment patterns, outcomes, and costs associated with complicated urinary tract infections (cUTIs) in adult patients. Methods: A retrospective observational cohort study of adult patients with incident cUTIs in IBM MarketScan Databases between 2017 and 2019 was performed. Patients were categorized as OP or inpatient (IP) based on initial setting of care for index cUTI and were stratified by age (<65 years vs ≥65 years). OP antibiotic treatment patterns, outcomes, and costs associated with cUTIs among adult patients over a 12-month follow-up period were examined. Results: During the study period, 95 322 patients met inclusion criteria. Most patients were OPs (84%) and age <65 years (87%). Treatment failure (receipt of new unique OP antibiotic or cUTI-related ED visit/IP admission) occurred in 23% and 34% of OPs aged <65 years and ≥65 years, respectively. Treatment failure was observed in >38% of IPs, irrespective of age. Across both cohorts and age strata, >78% received ≥2 unique OP antibiotics, >34% received ≥4 unique OP antibiotics, >16% received repeat OP antibiotics, and >33% received ≥1 intravenous (IV) OP antibiotics. The mean 12-month cUTI-related total health care costs were $4697 for OPs age <65 years, $8924 for OPs age >65 years, $15 401 for IPs age <65 years, and $17 431 for IPs age ≥65 years. Conclusions: These findings highlight the substantial 12-month health care burden associated with cUTIs and underscore the need for new outpatient treatment approaches that reduce the persistent or recurrent nature of many cUTIs.

Health care resource utilization and costs associated with treatment among patients initiating calcitonin gene-related peptide inhibitors vs other preventive migraine treatments in the United States.

BACKGROUND: Limited data are available on health care resource utilization (HCRU) and health care costs of calcitonin gene-related peptide monoclonal antibodies (CGRP mAbs) for preventive treatment of migraine. OBJECTIVE: To compare all-cause and migraine-related HCRU and direct health care costs in patients with migraine initiating CGRP mAbs, galcanezumab (GMB), vs standard-of-care (SOC) preventive treatments in the United States. METHODS: This retrospective observational study used insurance claims data collected from IBM MarketScan Research Databases. Adults (aged ≥ 18 years) with 1 or more claims for CGRP mAb (GMB, erenumab, or fremanezumab) or SOC preventive treatment between May 1, 2018, and June 30, 2019, were included. The date of earliest migraine treatment claim during this period was the index date. Annual all-cause and migraine-related HCRU included inpatient visits, emergency department visits, and acute and preventive migraine medication fills. After matching, HCRU and costs at 6- and 12-month follow-up in CGRP mAb, specifically GMB, vs SOC cohorts were analyzed using paired t-test and chi-square test. RESULTS: In the 12-month follow-up study, 4,528 patients using CGRP mAb (GMB, n = 426) and 10,897 patients using SOC were included. After matching, 3,082 pairs were identified in the CGRP mAb and SOC cohorts and 421 pairs in the GMB and SOC cohorts. After matching, all variables were well balanced across cohorts. At 12-month follow-up, the percentage decrease in acute and preventive migraine medication fills was significantly greater in the CGRP mAb (acute: -1.5% vs -0.2%, P < 0.001; preventive: -1.1% vs 3.8, P < 0.001) and GMB cohorts (acute: -1.5% vs -0.2%, P = 0.002; preventive: -1.8 vs 3.0, P < 0.001) compared with the SOC cohort. At follow-up, compared with the SOC cohort, the mean change of annual all-cause total costs was significantly higher in both the CGRP mAb ($6,043 vs $1,323, P < 0.001) and GMB cohorts ($8,398 vs $68, P < 0.001), and the mean change of annual migraine-related total costs was significantly higher in both the CGRP mAb ($3,416 vs $976, P < 0.001) and GMB cohorts ($4,334 vs $1,245, P < 0.001). Significant cost savings in mean acute and preventive migraine prescription costs occurred in both the CGRP mAb (acute: -$358 vs -$80, P < 0.001; preventive: -$298 vs $1,376, P < 0.001) and GMB cohorts (acute: -$280 vs -$36, P = 0.034; preventive: -$374 vs $1,537, P < 0.001) compared with the SOC cohort. CONCLUSIONS: Although treatment with CGRP mAbs and GMB increase total costs, they may lead to significantly greater cost savings in outpatient acute and preventive migraine medication costs vs SOC. Further studies assessing indirect health care costs are important to understand additional cost savings with CGRP mAbs. DISCLOSURES: Drs Varnado, Ye, and Schuh are employees and stockholders of Eli Lilly and Company. Dr Wenzel is a former employee of Eli Lilly and Company. Dr Manjelievskaia is an employee of IBM Watson Health. Ms Perry is a former employee of IBM Watson Health. This study was sponsored by Eli Lilly and Company. IBM Watson Health received funding for this study from Eli Lilly and Company. Independent analyses were conducted by IBM Watson Health. Eli Lilly and Company and IBM Watson Health collaborated on designing the study and interpreting results.

Treatment Patterns for Calcitonin Gene-Related Peptide Monoclonal Antibodies Including Galcanezumab versus Conventional Preventive Treatments for Migraine: A Retrospective US Claims Study.

Background: Most conventional, oral, preventive treatments for migraine are non-specific and ~50% of patients discontinue them within six months. In 2018, the Food and Drug Administration approved three preventive migraine treatments: monoclonal antibodies (mAb) targeting the calcitonin gene-related peptide (CGRP) pathway implicated in migraine; galcanezumab and fremanezumab which target CGRP ligand; and erenumab which targets CGRP receptor. Real-world treatment patterns for CGRP mAb are limited. Purpose: To compare real-world treatment patterns for CGRP mAb, specifically galcanezumab versus standard-of-care (SOC) migraine preventive treatments. Patients and methods: This retrospective, observational study included 12-month baseline and 6- and 12-month follow-up analyses using IBM® MarketScan® databases. Patients identified were aged ≥18 years with ≥1 claim (first claim=index) for CGRP mAb (erenumab, fremanezumab, or galcanezumab) or SOC preventives (eg, antiepileptics, beta-blockers, antidepressants, or onabotulinumtoxinA) as index drugs between May/01/2018 and June/30/2019. Propensity score matching was used to address confounding by observed covariates. Outcomes analyzed included proportion of days covered (PDC), persistence (≤60-day gap), and first non-index drug switch. Descriptive, chi-square (categorical), and t-test (continuous) analyses were conducted. Results: The study included 3082 (CGRP mAb versus SOC) and 421 (galcanezumab versus SOC) matched patient pairs with 12-month follow-up. Mean age across cohorts ranged 43.2-44.4 years (females: 85.7-88.6%). Compared with SOC, the CGRP mAb cohort had higher mean persistence (212.5 vs 131.9 days), adherence (PDC: 55.1% vs 35.2%), and more patients were adherent with PDC ≥80% (32.7% vs 18.7%) (all p <0.001). During 12-month follow-up, fewer patients discontinued CGRP mAb versus SOC (58.8% vs 77.6%, p <0.001). Galcanezumab versus SOC comparisons yielded similar results. In the CGRP mAb cohort, most switchers (28.3%) used galcanezumab as subsequent treatment. Largely similar results were observed for 6-month follow-up cohorts. Conclusion: Patients on CGRP mAb and specifically galcanezumab showed higher adherence and persistence than patients on SOC migraine preventive treatments.

Greater Adherence and Persistence with Injectable Dulaglutide Compared with Injectable Semaglutide at 1-Year Follow-up: Data from US Clinical Practice.

PURPOSE: Greater medication adherence and persistence have been associated with improved glycemic control in patients with type 2 diabetes mellitus. This study compared adherence, persistence, and treatment patterns among patients naïve to glucagon-like peptide 1 receptor agonists initiating once-weekly injectable treatment with dulaglutide versus semaglutide over 6-month (6M) and 12-month (12M) follow-up periods. METHODS: This retrospective, observational cohort study used administrative claims data from three IBM MarketScan research databases. Data from adult patients with type 2 diabetes newly initiating treatment with dulaglutide or semaglutide between January 2018 and January 2020 (index date was defined as the earliest fill date), without evidence of glucagon-like peptide 1 receptor agonist use in the 6M baseline period, and with continuous enrollment in the 6M baseline and 6M or 12M follow-up period were included. Dulaglutide initiators were propensity score-matched, in a 1:1 ratio, to semaglutide initiators in each 6M and 12M follow-up cohort (26,284 and 13,837 pairs, respectively). FINDINGS: In the matched cohorts, baseline characteristics were balanced; the mean age was 53 years, and 50% of patients were women. Compared to semaglutide initiators, dulaglutide initiators were more adherent (6M, 63.4% vs 47.8%; 12M, 54.4% vs 43.3%; both, P < 0.0001), more persistent on therapy (6M, 72% vs 62%, 12M, 55.5% vs 45.3%, both, P < 0.001), and had more mean days of persistence (6M, 145 vs 132, 12M, 254.3 vs 220.7; both, P < 0.001). IMPLICATIONS: At both 6M and 12M follow-up, dulaglutide initiators had significantly greater adherence and greater persistence compared with matched semaglutide initiators.

The direct and indirect costs of adult atopic dermatitis.

BACKGROUND: Atopic dermatitis is considered a childhood illness, and the direct and indirect health care burden of atopic dermatitis in adults is not fully understood. OBJECTIVE: To measure the direct and indirect costs of atopic dermatitis among adults in 2018. METHODS: This retrospective cohort study compared commercial and Medicare-insured adults with atopic dermatitis in 2018 with directly matched (1:3) adults without atopic dermatitis. Atopic dermatitis prevalence was reported. Health care utilization, direct health care costs, and work loss data were compared between cohorts. This analysis was repeated for adults with atopic dermatitis in 2016 and 2017. RESULTS: 31,164 adults with atopic dermatitis in 2018 were identified and directly matched (1:3) to controls. Adults with atopic dermatitis had greater utilization of outpatient services, outpatient pharmacy services, and short-term disability benefits than controls. Unadjusted annual health care costs in 2018 were $4,979 higher for adults with atopic dermatitis ($14,603) than for the matched controls ($9,624), driven by outpatient services and pharmacy. Findings were supported by analyses of adults from 2016 and 2017 and multivariable analyses. One limitation of this study was that patients with mild cases of atopic dermatitis may not seek medical treatment and may be underrepresented in the study cohort. CONCLUSIONS: The direct health care and indirect (short-term disability) health care costs of atopic dermatitis present a significant health care burden among the adult population. DISCLOSURES: This study was funded by Eli Lilly and Company. Employees of Eli Lilly were involved in the planning, execution, and interpretation of the study. Pierce is employed by Eli Lilly and Company. Boytsov and Goldblum were employed by Eli Lilly and Company Health at the time this research was conducted. Manjelievskaia and Brouillette are employed by IBM Watson Health, which received funding from Eli Lilly and Company to conduct this study. Bonafede and Onyekwere were employed at IBM Watson Health at the time this research was conducted.

Economic burden of growth hormone deficiency in a US pediatric population.

BACKGROUND: Pediatric growth hormone deficiency (GHD) is a rare disorder of short stature that is currently treated with daily injections of somatropin. In addition to short stature, GHD is associated with other comorbidities such as impaired musculoskeletal development, cardiovascular disease, and decreased quality of life. OBJECTIVE: To analyze somatropin utilization, adherence, and health care costs among children with GHD who had either Medicaid or commercial health insurance. METHODS: Children (aged < 18 years) with a GHD diagnosis between January 1, 2008, and December 31, 2017, were identified in the IBM MarketScan Commercial and Medicaid databases. Patients with at least 12- and 6-month continuous enrollment pre- and postdiagnosis were eligible. Children with GHD were direct matched (1:3) to controls without GHD (or other short stature-related disorders) on age, gender, plan type, region, and race (Medicaid only). Index date was the date of the first GHD diagnosis during the selection window for GHD patients and using random assignment for controls. Patients were followed until the end of continuous database enrollment or December 31, 2018. Baseline comorbidities and medications were measured during the 12 months pre-index, whereas somatropin treatment patterns along with all-cause and GHD-related health care costs were measured during the variable follow-up period. Multivariable modeling was used to compare costs between GHD patients and controls and between somatropin-treated and -untreated GHD patients while adjusting for baseline characteristics. RESULTS: There were 6,820 Medicaid and 14,070 commercial patients with GHD who met the study inclusion criteria. Mean (SD) age at index was 9.5 (4.5) years for Medicaid patients and 11.1 (3.7) years for commercial patients. The majority of patients were male (> 65%), and mean follow-up time for all cases and controls was 3-4 years. Overall, 63.2% of Medicaid and 68.4% of commercial GHD patients were treated with somatropin at some point during follow-up. Among Medicaid GHD patients, the treatment rate was highest among White males and lowest among Black females. Adherence was low as the proportion of days covered was ≥ 80% for only 18.4% of Medicaid patients and 32.3% of commercial patients and 49.1% of treated Medicaid and 24.3% of treated commercial patients discontinued before turning age 13. After adjusting for baseline characteristics, all-cause non-somatropin annualized costs were 5.67 times higher (Δ$19,309) for Medicaid GHD patients and 5.46 times higher (Δ$12,305) for commercial GHD patients than matched non-GHD controls. Adjusted all-cause non-somatropin annualized costs were 0.59 times lower (Δ$14,416) for treated Medicaid patients and 0.69 times lower (Δ$7,650) for treated commercial patients than for untreated patients. CONCLUSIONS: Pediatric GHD presents a significant health care burden, and many patients remain untreated or undertreated. Untreated GHD was associated with higher non-somatropin health care costs than treated GHD. Strategies to optimize treatment and improve adherence may reduce the health care burden faced by these patients. DISCLOSURES: This study was funded by Ascendis Pharma, Inc. Smith and Pitukcheewanont are employed by Ascendis Pharma, Inc. Manjelievskaia, Lopez-Gonzalez, and Morrow are employed by IBM Watson Health, which received funding from Ascendis Pharma, Inc., to conduct this study. Kaplowitz is a paid consultant of Ascendis Pharma, Inc.

Adherence and persistence among patients with major depressive disorder enrolled in the vortioxetine tAccess Patient Support Program.

OBJECTIVE: The tAccess Patient Support Program (PSP) is a personalized support program for patients prescribed vortioxetine therapy. We assessed the impact of the tAccess PSP on adherence to and persistence with vortioxetine among adult patients with major depressive disorder (MDD). METHODS: A retrospective observational cohort study was conducted in patients with MDD receiving vortioxetine who were enrolled in the tAccess PSP. Eligible patients were 18 to 64 years of age and had ≥1 vortioxetine claim and ≥1 inpatient/outpatient medical MDD claim during the 12 months preceding or on the index date, defined as the date of the earliest vortioxetine claim. Study outcomes included medication adherence (proportion of days covered [PDC], adherent ≥80%) and persistence (the total number of days on therapy without a ≥30-day gap) at 90, 180, and 365 days. Additionally, persistence was reported for a subset of patients meeting the standardized Healthcare Effectiveness Data and Information Set (HEDIS) Antidepressant Medication Management (AMM) criteria (the percentage of adults aged 18-64 years who remained on an antidepressant for ≥84 days or ≥180 days), as defined by the National Committee for Quality Assurance. These data were reviewed alongside current HEDIS AMM data for commercially insured patients meeting the standardized metric. RESULTS: The study identified a total of 2635 patients with an MDD diagnosis and ≥90 days of follow-up time and a subset of 2238 patients meeting HEDIS AMM criteria. Mean PDC among all patients with MDD was 0.78, with 58.3% of patients achieving PDC ≥80%. During the 90-day follow-up, 62.1% of patients with MDD were persistent on vortioxetine. Among the subset of patients who met HEDIS AMM criteria, persistence was 83.4% and 69.9% at 84 and 180 days, respectively. In comparison, in 2017, HEDIS AMM criteria for antidepressant persistence were met by 67.8% of commercially insured patients at 84 days and 51.8% at 180 days. CONCLUSIONS: These initial results, reviewed alongside recent available HEDIS AMM data, suggest that the tAccess PSP may be beneficial in addressing treatment adherence and persistence in patients with MDD.

Validation of an International Classification of Disease, 10th revision coding adaptation for the Charlson Comorbidity Index in United States healthcare claims data.

PURPOSE: An International Classification of Disease (ICD-10) Charlson Comorbidity Index (CCI) adaptation had not been previously developed and validated for United States (US) healthcare claims data. Many researchers use the Canadian adaption by Quan et al (2005), not validated in US data. We sought to evaluate the predictive validity of a US ICD-10 CCI adaptation in US claims and compare it with the Canadian standard. METHODS: Diverse patient cohorts (rheumatoid arthritis, hip/knee replacement, lumbar spine surgery, acute myocardial infarction [AMI], stroke, pneumonia) in the IBM® MarketScan® Research Databases were linked with the IBM MarketScan Mortality file. Predictive performance was measured using c-statistics for binary outcomes (1-year and postoperative mortality, in-hospital complications) and root mean square prediction error (RMSE) for continuous outcomes (1-year all-cause medical costs, index hospitalization costs, length of stay [LOS]), after adjusting for age and sex. C-statistics were compared by the method of DeLong and colleagues (1988); RMSEs, by resampling. RESULTS: C-statistics were generally high (≥ ~ 0.8) for mortality but lower for in-hospital complications (~0.6-0.7). RMSEs for costs and hospitalization LOS were relatively large and comparable to standard deviations. Results were similar overall between the US and Canadian adaptations, with relative differences typically <1%. CONCLUSIONS: This US-based coding adaptation and a previously published Canadian adaptation resulted in similar predictive ability for all outcomes evaluated but may have different construct validity (not evaluated in our study). We recommend using adaptations specific to the country of data origin based on good research practice.

Real-World Effectiveness of Mepolizumab in Patients with Severe Asthma: An Examination of Exacerbations and Costs.

RATIONALE: Results from clinical trials in patients with severe eosinophilic asthma have demonstrated that mepolizumab is well tolerated and is associated with improved asthma control as evidenced by reductions in both exacerbations and maintenance oral corticosteroid use, and improvements in lung function, asthma control, and quality of life. However, real-world data are lacking on the impact of mepolizumab treatment. OBJECTIVE: To assess the effect of mepolizumab treatment on the rate of asthma exacerbations and asthma exacerbation-related costs in a real-world setting. METHODS: This retrospective cohort study (GSK ID: 209017; HO-18-19168) analyzed data from patients with severe asthma ≥12 years of age at mepolizumab treatment initiation (index date) with ≥12 months pre- (baseline) and post-index (follow-up) data from a commercial claims database (patients were identified from November 1, 2015 to March 31, 2017). Asthma exacerbations (primary objective) and asthma exacerbation-related costs (secondary objective) in the baseline and follow-up periods were compared. Other analyses included the number of mepolizumab administrations and the use of concomitant asthma medications. RESULTS: Data were analyzed from 346 patients. Mepolizumab significantly reduced the proportion of patients with any exacerbation and exacerbations requiring hospitalization, compared with baseline. Significant reductions in the rate of all exacerbations of 38.4% (from 2.68 to 1.65 events/patient/year; P<0.001) and of exacerbations requiring hospitalization of 72.7% (from 0.11 to 0.03 events/patient/year; P=0.004) were observed, compared with baseline. Mean total asthma exacerbation-related costs (excluding mepolizumab acquisition and administrative costs) per person were significantly lower during follow-up compared with baseline (P<0.05) and the use of asthma medications, including oral and inhaled corticosteroids, was also lower. CONCLUSION: This study confirms the clinical benefit observed in previous mepolizumab clinical trials and demonstrates that mepolizumab is effective in a real-world setting.

Assessing the Association of Formulary Copayment Changes with Real-World Treatment Patterns in Patients with Rheumatoid Arthritis on Etanercept.

BACKGROUND: Rheumatoid arthritis (RA) is a chronic disease that requires long-term treatment to improve or maintain stable disease activity. Tumor necrosis factor inhibitors (TNFi), a class of biologic disease-modifying antirheumatic drugs (bDMARD), are effective at treating symptoms and inhibiting joint progression. Although treatment changes are not recommended in patients with stable disease, health plans have recently enacted formulary changes with higher copayments that could disrupt patient access to TNFis. OBJECTIVE: To assess the association of formulary copayment changes with real-world treatment patterns, treatment effectiveness, and health care costs among bDMARD-naive patients with RA receiving the TNFi etanercept. METHODS: This retrospective observational cohort analysis used the IBM Watson Health MarketScan Commercial Claims and Encounters Database. Adult patients with RA with 6 months of stable etanercept use (no refill gap ≥ 45 days) from January 1, 2013, through December 31, 2015, were selected and the index date was set to the first fill date after the stable-use period. Average etanercept copayment was calculated at the drug-plan level. Copayment change was defined as a monthly increase of at least $40 to account for copayment changes attributable to etanercept wholesale acquisition costs between 2014 and 2015. This amount also corresponded to the 90th percentile of average plan-level changes in etanercept copayments in the database, representing an average change in copayment by a payer. Patients were followed ≥ 12 months before and after the index date to track etanercept treatment changes and ≥ 12 months after a treatment change to track costs after etanercept copayment changes. Etanercept persistence, bDMARD switching, refill gaps, and treatment effectiveness (using a validated effectiveness algorithm) were described for patients with or without copayment change during the 12 months post-index or postchange. We also assessed the mean total of all-cause and RA-related expenditure during the 12-month post-index (or postchange) period. RESULTS: 1,970 stable patients met study inclusion criteria (mean [standard deviation] age: 50.3 [9.5] years; 77.8% female) and were evaluated. Of these, 133 (6.8%) patients had a copayment change ≥$40 during follow-up. Overall, most patients (60.3%) persisted on etanercept for the 12-month follow-up period, while 13.0% switched from etanercept, and 8.1% discontinued (refill gap of ≥ 45 days). Nearly half (48.0%) of all patients were considered effectively treated according to a validated algorithm. Compared with patients without a copayment change, those with a copayment change were more likely to switch biologics (19.5% vs. 12.6%; P = 0.021). Although statistical significance was not reached, patients with a copayment change were less likely to be persistent (54.1% vs. 60.7%; P = 0.135), and less likely to be effectively treated (42.1% vs. 48.4%; P = 0.161) than patients without a copayment change. All-cause and RA-related expenditures at baseline and post-copayment change were similar between patients with and without a copayment change. CONCLUSIONS: Changing formulary copayment of etanercept was associated with higher switching without difference in costs or health care utilization between copayment and no copayment change groups. DISCLOSURES: This study was sponsored by Amgen. Bonafede, Manjelievskaia, and Lopez-Gonzalez are employees of IBM Watson Health, which received funding from Amgen to conduct this study. Oko-osi, Collier, and Stolshek are employees and shareholders of Amgen. Gharaibeh was an employee of Amgen at the time of study execution and manuscript drafting. The authors have no other relationships that present a potential conflict of interest. Data pertaining to this study were presented in a poster at the 2018 ACR/ARHP Annual Meeting; October 19-24, 2018; Chicago, IL.

Impact of cytomegalovirus complications on resource utilization and costs following hematopoietic stem cell transplant.

Objective: The impact of cytomegalovirus (CMV) infection on healthcare resource utilization (HCRU) and costs post-allogeneic hematopoietic stem cell transplant (allo-HSCT) has not been well studied in the US. This retrospective, observational cohort study examined such outcomes in the first year following allo-HSCT.Methods: The IBM MarketScan administrative claims database was used to identify adults who underwent a first allo-HSCT between 1 January 2010 and 30 April 2015. Patients were required to have continuous medical and pharmacy enrollment for ≥12 months before and after the allo-HSCT. HCRU and medical costs (2016 US$) were compared by the presence or absence of CMV infection over 1-year follow-up.Results: A total of 1825 adults met the inclusion criteria (57.5% male; mean age 50.8 years). During the follow-up period, 410 (22.5%) patients had a CMV-related claim. Patients with CMV infection were significantly more likely to have a 60-day-(31.2 vs. 19.4%), 100-day-(50.0 vs. 30.5%) or 365-day readmission (78.0 vs. 57.8%) compared to those without a CMV-related event (all p < .001). During follow-up, patients with CMV infection had significantly greater mean total costs, reflecting higher inpatient costs ($677,240 vs. $462,562), outpatient costs ($141,366 vs. $94,312) and prescription drug costs ($27,391 vs. $22,082) (all p < .001). Valganciclovir (59.8%) and ganciclovir (33.7%) were the most commonly utilized anti-viral agents in patients with CMV.Conclusions: CMV infection was associated with significantly higher healthcare resource utilization and costs during the first year post-allo-HSCT. Additional research is warranted to further evaluate the consequences of post-HSCT CMV infection, as well as cost-effective measures to minimize its occurrence.

A comparison of treatment patterns, healthcare resource utilization, and costs among young adult Medicaid beneficiaries with schizophrenia treated with paliperidone palmitate or oral atypical antipsychotics in the US.

BACKGROUND: Much of the burden associated with schizophrenia is attributed to its early onset and chronic nature. Treatment with once monthly paliperidone palmitate (PP1M) is associated with lower healthcare utilization and better adherence as compared to oral atypical antipsychotics (OAAs). This study aimed to evaluate real-world effectiveness of PP1M and OAA therapies among US-based adult Medicaid patients with schizophrenia, overall and among young adults aged 18-35 years. METHODS: Adult patients with a diagnosis of schizophrenia and at least two claims for PP1M or OAA between January 1, 2010 and December 31, 2014 were selected from the IBM Watson Health MarketScan Medicaid Database. Treatment patterns and healthcare resource utilization and costs were compared between PP1M and OAA treatment groups following inverse probability of treatment (IPT) weighting to adjust for potential differences. Utilization and cost outcomes were estimated using OLS and weighted Poisson regression models. RESULTS: After IPT weighting, the young adult PP1M and OAA cohorts were comprised of 3,095 and 3,155 patients, respectively. PP1M patients had a higher duration of continuous treatment exposure (168.2 vs 132.5 days, p = .004) and better adherence on the index medication (proportion of days covered ≥80%: 19.0% vs 17.1%, p < .049). Young adults treated with PP1M were 37% less likely to have an all-cause inpatient admission (odds ratio [OR] = 0.63, 95% confidence interval [CI] = 0.53-0.74) and 33% less likely to have an ER visit (OR = 0.67, 95% CI = 0.55-0.81) compared to OAA young adult patients, but 27% more likely to have an all-cause outpatient office visit (OR = 1.27, 95% CI = 1.02-1.56). PP1M patients incurred significantly lower medical costs as compared to OAA patients. CONCLUSIONS: Medicaid patients with schizophrenia treated with PP1M have higher medication adherence and have fewer hospitalizations as compared to patients treated with OAAs. PP1M may lead to reduced healthcare utilization and improved clinical outcomes.

Costs for Breast Cancer Care in the Military Health System: An Analysis by Benefit Type and Care Source.

Introduction: Breast cancer care imposes a significant financial burden to U.S. healthcare systems. Health services factors, such as insurance benefit type and care source, may impact costs to the health system. Beneficiaries in the U.S. Military Health System (MHS) have universal healthcare coverage and access to a network of military facilities (direct care) and private practices (purchased care). This study aims to quantify and compare breast cancer care costs to the MHS by insurance benefit type and care source. Materials and Methods: We conducted a retrospective analysis of data linked between the MHS data repository administrative claims and central cancer registry databases. The institutional review boards of the Walter Reed National Military Medical Center, the Defense Health Agency, and the National Institutes of Health Office of Human Subjects Research reviewed and approved the data linkage. We used the linked data to identify records for women aged 40-64 yr who were diagnosed with breast cancer between 2003 and 2007 and to extract information on insurance benefit type, care source, and cost to the MHS for breast cancer treatment. We estimated per capita costs for breast cancer care by benefit type and care source in 2008 USD using generalized linear models, adjusted for demographic, pathologic, and treatment characteristics. Results: The average per capita (n = 2,666) total cost for breast cancer care was $66,300 [standard error (SE) $9,200] over 3.31 (1.48) years of follow-up. Total costs were similar between benefit types, but varied by care source. The average per capita cost was $34,500 ($3,000) for direct care (n = 924), $96,800 ($4,800) for purchased care (n = 622), and $60,700 ($3,900) for both care sources (n = 1,120), respectively. Care source differences remained by tumor stage and for chemotherapy, radiation, and hormone therapy treatment types. Conclusions: Per capita costs to the MHS for breast cancer care were similar by benefit type and lower for direct care compared with purchased care. Further research is needed in breast and other tumor sites to determine patterns and determinants of cancer care costs between benefit types and care sources within the MHS.

The continuum of breast cancer care and outcomes in the U.S. Military Health System: an analysis by benefit type and care source.

PURPOSE: This study investigates transition rates between breast cancer diagnosis, recurrence, and death by insurance benefit type and care source in U.S. Military Health System (MHS). METHODS: The MHS data repository and central cancer registry linked data were used to identify women aged 40-64 with histologically confirmed breast cancer between 2003 and 2007. Three-state continuous time Markov models were used to estimate transition rates and transition rate ratios (TRRs) by TRICARE benefit type (Prime or non-Prime) and care source (direct, purchased, or both), adjusted for demographic, tumor, and treatment variables. RESULTS: Analyses included 2668 women with transitions from diagnosis to recurrence (n = 832), recurrence to death (n = 79), and diagnosis to death without recurrence (n = 91). Compared to women with Prime within each care source, women with non-Prime using both care sources had higher transition rates (TRR 1.47, 95% CI 1.03, 2.10). Compared to those using direct care within each benefit type, women utilizing both care sources with non-Prime had higher transition rates (TRR 1.86, 95% CI 1.11, 3.13), while women with Prime utilizing purchased care had lower transition rates (TRR 0.82, 95% CI 0.68, 0.98). CONCLUSIONS: In the MHS, women with non-Prime benefit plans compared to Prime had higher transition rates along the breast cancer continuum among both care source users. Purchased care users had lower transition rates than direct care users among Prime beneficiaries. IMPLICATIONS FOR CANCER SURVIVORS: Benefit plan and care source may be associated with breast cancer progression. Further research is needed to demonstrate differences in survivorship.

Breast Cancer Treatment and Survival Among Department of Defense Beneficiaries: An Analysis by Benefit Type and Care Source.

Background: Use of treatment for breast cancer is dependent on the patient's cancer characteristics and willingness to undergo treatment and provider treatment recommendations. Receipt of breast cancer treatment varies by insurance status and type. It is not clear whether different benefit types and care sources differ in breast cancer treatment and outcomes among Department of Defense beneficiaries. Methods: The objectives of this study are to assess whether receipt of breast cancer treatment varied by benefit type (TRICARE Prime vs non-Prime) or care source (direct care, purchased care, and both) and to examine whether survival and recurrence differed by benefit type and/or care source among female Department of Defense beneficiaries with the disease. Study subjects were women aged 40-64 yr, diagnosed with malignant breast cancer between 2003 and 2007. Multivariable logistic regression analyses were conducted to assess the likelihood of receiving treatment by benefit type or care source. Multivariable Cox proportional hazard models were used to investigate differences in survival and recurrence by benefit type or care source. Findings: A total of 2,668 women were included in this study. Those with Prime were more likely to have chemotherapy, radiation, hormone therapy, breast-conserving surgery, surveillance mammography, and recurrence than women with non-Prime. Survival was high, with 94.86% of those with Prime and 92.58% with non-Prime alive at the end of the study period. Women aged 50-59 yr with non-Prime benefit type had better survival than women with Prime of the same age. No survival differences were seen by care source. In regard to recurrence, women aged 60-64 yr with TRICARE Prime were more likely to have recurrent breast cancer than women with non-Prime. Additionally, women aged 50-59 yr who used purchased care were less likely to have a recurrence than women who used direct care only. Discussion/Impact/Recommendations: To our knowledge, this is the first study to examine breast cancer treatment and survival by care source and benefit type in the Military Health System. In this equal access health care system, no differences in treatment, except mastectomy, by benefit type, were observed. There were no overall differences in survival, although patients with non-Prime tended to have better survival in the age group of 50-59 yr. In regard to care source, women who utilized mostly purchased care or utilized both direct and purchased care were more likely to receive certain types of treatment, such as chemotherapy and radiation, as compared with women who used direct care only. However, survival did not differ between different care sources. Future research is warranted to further investigate variations in breast cancer treatment and its survival gains by benefit type and care source among Department of Defense beneficiaries.