Healthcare Resource Utilization Burden Research Articles

Healthcare resource utilization burden one year post continuous positive airway pressure initiation among adults with excessive daytime sleepiness in obstructive sleep apnea in the United Kingdom

Healthcare resource utilization burden one year post continuous positive airway pressure initiation among adults with excessive daytime sleepiness in obstructive sleep apnea in the United Kingdom

RWD153 Investigating Trends of Behavioral Healthcare Resource Utilization Burden in Children and Adolescents with Suicidal Symptoms in the United States: An Analysis of Electronic Health Record Data

The increasing rates of mental health disorders and suicide among children and adolescents have highlighted the importance of understanding the burden of healthcare resource utilization for this population. This study aims to investigate trends in the utilization of behavioral health services by minors with suicidal or self-harm symptoms in the United States.

Health Care Resource Utilization by Patients with Alagille Syndrome.

To assess and characterize health care resource utilization (HRU) in children with the rare, genetic, multisystem disorder, Alagille syndrome. This retrospective analysis reviewed commercially insured and Medicaid-insured claims from October 1, 2015 to December 31, 2019 to assess HRU in patients with Alagille syndrome. As there is no specific International Classification ofDiseases-10 code for Alagille syndrome, patients were identified using the following algorithm: ≥1 claim with diagnosis code Q44.7 (other congenital malformations of the liver); <18years of age, with no history of biliary atresia (International Classification ofDiseases-10 code: Q44.2); and ≥6months of insurance eligibility prior to diagnosis. HRU was summarized per patient per year over all available claims postdiagnosis. A total of 171 commercially insured and 215 Medicaid-insured patients with Alagille syndrome were available for analysis. Annually, commercially insured and Medicaid-insured patients averaged 31 medical visits (range, 1.5-237) and 48 medical visits (range, 0.7-690), respectively. The most common visits were outpatient with the majority encompassing lab/imaging and primary care visits (commercially insured: 21 [range, 0.0-183]; Medicaid-insured: 26 [range, 0.0-609]). Inpatient visits were the highest driver of costs in both the commercial and Medicaid populations. Patients with Alagille syndrome have a substantial HRU burden driven largely by numerous outpatient visits and costly inpatient stays. Given the complexity and variability of Alagille syndrome presentation, patients may benefit from multidisciplinary and subspecialized care.

Economic and health care resource utilization burden of central nervous system metastases in patients with metastatic melanoma.

BACKGROUND: In patients with metastatic melanoma, central nervous system (CNS) involvement is associated with poor prognosis, increased costs, and higher health care resource utilization (HCRU); however, previous cost-estimate studies were conducted before widespread use of targeted therapies and immunotherapies. OBJECTIVE: To estimate costs and HCRU in patients with metastatic melanoma with and without CNS metastases in the current treatment era following introduction of targeted therapies and immunotherapies. METHODS: This real-world retrospective cohort study used data from the IQVIA PharMetrics Plus claims database to estimate and compare costs and HCRU in patients with metastatic melanoma by presence or absence of CNS metastases between January 2011 and June 2019. Patients with at least 2 melanoma claims, at least 2 metastatic claims, and continuous enrollment at least 6 months before and at least 1 month after first metastatic diagnosis were included. Mean per-patient-per-month (PPPM) costs are reported in 2019 US dollars. Analyses were also conducted by time period of first metastatic diagnosis: 2011-2014 (reflecting BRAF inhibitor monotherapy and anti-CTLA-4 therapy) and 2015-2019 (reflecting availability of BRAF and MEK inhibitor combinations and anti-PD-1/PD-L1 therapies). RESULTS: Of 4,078 patients, 1,253 (30.7%) had CNS metastases. Patients with CNS metastases were more likely to receive any treatment (89.1% vs 58.9%; P < 0.001), including systemic treatment (73.3% vs 55.4%; P < 0.001) and radiation (65.8% vs 11.8%; P < 0.001), and to have brain imaging any time after metastatic diagnosis (98.3% vs 67.2%; P < 0.001). In patients with CNS metastases, 40.0% had dexamethasone 4 mg within 30 days of CNS metastatic diagnosis. Patients with CNS metastases incurred higher total mean PPPM costs ($29,953 vs $14,996; P < 0.001). The largest contributors were total radiology ($2,351 vs $1,110), targeted therapies ($2,499 vs $638), and immunotherapies ($7,398 vs $5,036). HCRU and costs were higher in patients with vs without CNS metastases regardless of time period of first metastatic diagnosis. In patients with CNS metastases, use of any systemic treatment was increased in 2015-2019 vs 2011-2014 (81.2% vs 64.5%; P < 0.001), including chemotherapy (68.1% vs 50.0%; P < 0.001), immunotherapy (60.9% vs 30.1%; P < 0.001), and/or targeted therapies (32.7% vs 27.4%; P = 0.05). Mean total PPPM costs for patients with CNS metastases increased from $28,183 in 2011-2014 to $31,569 in 2015-2019 (P < 0.001); main drivers were immunotherapies and targeted therapies. CONCLUSIONS: CNS metastases occur frequently in patients with metastatic melanoma and are associated with significantly increased economic burden compared with patients without CNS metastases; the largest contributors to total costs in the current treatment era are radiology, targeted therapies, and immunotherapies. Brain imaging remains underused, and there is an opportunity to improve outcomes through early detection of CNS metastases, potentially reducing the high HCRU and costs associated with CNS metastases. DISCLOSURES: This study was funded by F. Hoffmann-La Roche Ltd. The sponsor was involved in the study design, data collection, data analysis, manuscript preparation, and publication decisions. Seetasith and Lee are employed by and report stock ownership in Genentech, Inc. Bartley and McKenna were employed by Genentech, Inc., at the time of this study and report stock ownership. Tawbi reports grants and personal fees from Genentech/Roche, Novartis, BMS, and Merck; grants from GSK and Celgene; and personal fees from Eisai, outside the submitted work. Kent, Burton, and Haydu have nothing to disclose. The results of this study were presented in part at the AMCP Nexus 2020 Virtual Meeting, October 19-23, 2020.

Does the 5\u20132-1 criteria identify patients with advanced Parkinson's disease? Real-world screening accuracy and burden of 5\u20132-1-positive patients in 7 countries

BackgroundThe burden of Parkinson’s disease (PD) worsens with disease progression. However, the lack of objective and uniform disease classification challenges our understanding of the incremental burden in patients with advanced Parkinson’s disease (APD) and suboptimal medication control. The 5–2-1 criteria was proposed by clinical consensus to identify patients with advancing PD. Our objective was to evaluate the screening accuracy and incremental clinical burden, healthcare resource utilization (HCRU), and humanistic burden in PD patients meeting the 5–2-1 screening criteria.MethodsData were drawn from the Adelphi Parkinson’s Disease Specific Program (DSP™), a multi-country point-in-time survey (2017–2020). People with PD who were naive to device-aided therapy and on oral PD therapy were included. Patients meeting the 5–2-1 screening criteria had one or more of the three clinical indicators of APD: (i) ≥5 doses of oral levodopa/day, OR (ii) “off” symptoms for ≥2 h of waking day, OR (iii) ≥1 h of troublesome dyskinesia. Clinician assessment of PD stage was used as the reference in this study. Clinical screening accuracy of the 5–2-1 criteria was assessed using area under the curve and multivariable logistic regression models. Incremental clinical, HCRU, and humanistic burden were assessed by known-group comparisons between 5 and 2-1-positive and negative patients.ResultsFrom the analytic sample (n = 4714), 33% of patients met the 5–2-1 screening criteria. Among physician-classified APD patients, 78.6% were 5–2-1 positive. Concordance between clinician judgment and 5–2-1 screening criteria was > 75%. 5–2-1-positive patients were nearly 7-times more likely to be classified as APD by physician judgment. Compared with the 5–2-1-negative group, 5–2-1-positive patients had significantly higher clinical, HCRU, and humanistic burden across all measures. In particular, 5–2-1-positive patients had 3.8-times more falls, 3.6-times higher annual hospitalization rate, and 3.4-times greater dissatisfaction with PD treatment. 5–2-1-positive patients also had significantly lower quality of life and worse caregiver burden.Conclusions5–2-1 criteria demonstrated potential as a screening tool for identifying people with APD with considerable clinical, humanistic, and HCRU burden. The 5–2-1 screening criteria is an objective and reliable tool that may aid the timely identification and treatment optimization of patients inadequately controlled on oral PD medications.

Economic Burden of Hospitalizations for Patients with Newly Diagnosed Acute Myeloid Leukemia in Remission in the United States: Retrospective Analysis of an Administrative Claims Database

INTRODUCTION: Managing patients with acute myeloid leukemia (AML) requires extensive healthcare resource utilization and costs; hospitalization is the largest component of medical costs for AML. In the phase 3 QUAZAR AML-001 trial, oral azacitidine (Oral-AZA [CC-486]) was associated with significant improvements in overall survival and relapse-free survival (RFS) vs. placebo (Wei, 2020). Prolonged RFS with Oral-AZA may translate into substantial economic benefits, with lower hospitalization-related costs due to reduced rates of hospitalization and days in hospital (Oliva, ASH 2020). Real-world economic benefits of prolonged remission remain insufficiently studied.OBJECTIVE: This study aimed to examine the economic burden of hospitalizations among patients with newly diagnosed AML in remission from a retrospective analysis of real-world data from a US claims database.METHODS: Using a retrospective cohort design, adult patients were selected from the IBM MarketScan TM Commercial and Medicare Supplemental Databases, with ≥2 outpatient claims or 1 inpatient claim with a primary International Classification of Disease 9th/10th Revision (ICD-9/ICD-10) code for AML between July 1, 2012, and September 30, 2019. Patients were required to have 1) at least 6 months of continuous enrollment, with pharmacy benefits, prior to diagnosis, 2) received systemic induction therapy as first-line (1L) therapy for AML on or after the index diagnosis date, and 3) attained remission from 1L systemic therapy.Patients were followed from remission after systemic induction therapy, with or without consolidation, until the end of the follow-up period. Eligible patients were organized into cohorts based on their duration of remission (DOR): Cohort A included patients with < median DOR and Cohort B had patients with ≥ median DOR. Hospitalization incidence and duration during the follow-up period were calculated using a per-patient per-year (PPPY) metric. Hospitalization-related costs were compared using a generalized linear model (GLM) with gamma distribution and log-link function. Generalized estimating equations (GEE) clustered on the patient were used to compare incidence and duration of hospitalizations. All costs were adjusted for inflation and reported in 2019 US dollars (USD).RESULTS: In all, 693 patients met all selection criteria and were assessed for DOR. The median DOR for all patients was 167 days; cohorts A and B included 346 and 347 patients, respectively, with median times from remission to end of follow-up of 106.5 and 460 days. Baseline characteristics were comparable between cohorts; mean [SD] age was 55.1 [14.4] years, 49.4% of patients were male, and mean [SD] Charlson comorbidity index [CCI] was 0.8 [1.2]. The PPPY number of hospitalizations was higher in Cohort A than in Cohort B (4.56 vs 2.09, respectively), as was the PPPY total length of hospital stay (51.1 vs 19.7 days). The PPPY hospitalization-related costs were $345,728 in Cohort A and $125,018 in Cohort B; the cumulative hospitalization cost per patient was $148,430 higher in Cohort A at 12 months and $177,500 higher at 18 months (Figure). Multivariate GEE and GLM models with adjustment for patient characteristic covariates (eg, age, sex, CCI) identified prolonged remission was significantly associated with reduced number, duration, and cost of hospitalization (P < 0.001, all comparisons).CONCLUSIONS: In a real-world setting, prolonged AML remission was associated with significantly lower rates and durations of hospitalization, which were estimated to result in substantial cumulative cost savings. These data underscore the importance of active maintenance therapies that delay relapse for patients with AML. While the costs of long-term therapy face increasing scrutiny, these costs should be weighed relative to the potential economic benefit of prolonged remission and reduced burden of healthcare resource utilization. [Display omitted] DisclosuresChen: Bristol Myers Squibb: Current Employment, Current equity holder in publicly-traded company. Papademetriou: SmartAnalyst Inc.: Current Employment. Potluri: Bristol Myers Squibb: Consultancy.

Burden of Migraine in Patients With Preventive Treatment Failure Attending European Headache Specialist Centers: Real-World Evidence From the BECOME Study.

IntroductionMigraine is consistently ranked as one of the most disabling neurological conditions in the world, often causing a substantial impairment of daily activities and quality of life. It also carries a high economic burden of direct and indirect healthcare costs. Patients with difficult-to-treat migraine often cycle through different preventive therapies, but real-world prospective evidence describing the burden of migraine in patients with prior preventive treatment failure (PPTF) in Europe is limited. In BECOME, we aimed to characterize and assess the prevalence and burden of migraine in patients with PPTF attending specialist headache centers in Europe and Israel. Furthermore, we assessed this burden in pre-specified subgroups based on the frequency of monthly migraine days (MMD) and number of PPTFs.MethodsBECOME was a prospective, non-interventional study conducted in two concurrent parts across 17 countries in Europe and Israel. In part 1, patients visiting the centers over a 3-month period were screened for frequency of PPTF, MMD, and other characteristics. In part 2, patients from part 1 with ≥ 1 PPTF and ≥ 4 MMD were enrolled, and impact of migraine on patient-reported outcomes, and healthcare resource utilization (HRU) were examined.ResultsIn part 1 (n = 20,837), 62.2% of patients reported ≥ 1 PPTF. In part 2 (n = 2419), 15.3% of patients reported ≥ 4 PPTF. In part 2, the migraine burden measured by the EuroQoL 5 dimensions 5 level (EQ-5D-5L) questionnaire indicated an impact of at least moderate severity in performing usual activities in 26.5% of patients, pain/discomfort in 51.2%, and 26.1% reported being at least moderately anxious/depressed. Most patients reported a severe impact on daily activities and disability due to migraine. Abnormal Hospital Anxiety and Depression subscale scores of ≥ 11 were observed in 29% (anxiety) and 19.8% (depression) of the population. In part 2, analysis of HRU showed 21.2% patients visited an emergency department and 8.4% were hospitalized for headache/migraine in the past year.ConclusionsThis study provides real-world evidence of the high personal, social, and HRU burden of migraine in Europe and Israel.Supplementary InformationThe online version contains supplementary material available at 10.1007/s40122-021-00331-3.

PCN237 Patterns of Healthcare Resource Utilization (HRU) in Unfit Patients with Acute Myeloid Leukemia (AML) Receiving First-line Systemic Treatment or Best Supportive Care (BSC): A Multicenter International Study (CURRENT)

Until recently, treatment options for patients with AML unfit for intensive chemotherapy included hypomethylating agents (HMA), low-dose cytarabine (LDAC), and BSC. Primary analysis of CURRENT demonstrated clinical outcomes remain poor for patients (Kondo. ASH 2020). This analysis evaluates HRU burden with first-line treatment in CURRENT among unfit patients with AML. This retrospective chart review included unfit patients with primary/secondary AML who received HMA, LDAC, Other systemic therapies, or BSC. Unconfirmed AML, acute promyelocytic leukemia, or clinical trial patients were excluded. HRU endpoints, including hospitalizations, outpatient consultations, transfusions, and medications, were analyzed descriptively. The final data cutoff (31Mar2020) included 1762 patients (57% male; median age 75 years; 35% JAPAC, 27% WEC, 25% EEMEA, 13% LATAM): 809 HMA, 199 LDAC, 302 Other, and 452 BSC. Median (min−max) treatment durations were 118 (0−1450), 35 (1−1132), 33 (1−1124), and 57 (0−1680) days, respectively. Of those who received HMA/LDAC/Other, 18% received second-line systemic therapy. Hospitalizations were frequent (HMA 82%, LDAC 93%, Other 83%, BSC 83%): Median (min–max) number of hospitalizations was 6 (1−50), 5 (1−26), 4 (1−15), and 2 (1−21), respectively, with a median (min−max) of 8 (1−546), 16 (1−696), 18 (0−933), and 8 (1−157) days hospitalized, most commonly for treatment administration (HMA/Other 61%, LDAC 72%) and infection (BSC 49%). HMA had the most outpatient consultations (HMA 79%, LDAC 53%, Other 63%, BSC 66%; median 13, 6, 11, 6 visits, respectively), and transfusions (HMA 80%, LDAC 57%, Other 57%, BSC 71%); median RBC and platelet events ranged from 7−13 and 2−11, respectively. Antibiotics/antivirals were used more often than antifungals and growth factors (72–92%, 34–63%, 7–28%, respectively). Antibiotics/antivirals and growth factors were generally used responsively; antifungals were mainly used prophylactically. Unfit patients with AML require substantial HRU; novel therapies are needed to alleviate this burden and improve outcomes.

Burden of illness and treatment patterns among patients with peripheral T-cell lymphoma in the US healthcare setting

Objective Limited real-world information exists on the characteristics or treatment patterns of patients with peripheral T-cell lymphoma (PTCL). We reported demographics, treatments and direct healthcare resource utilization (HRU) in a large cohort of US patients newly diagnosed with PTCL. Methods Patients aged ≥18 years with a PTCL diagnosis between January 2011 and December 2016 were identified from the Inovalon MORE2 Registry. Continuous medical/pharmacy enrollment 6-months prior to and ≥1-month after the first PTCL diagnosis was required. The main focus of this study was on newly diagnosed patients receiving cyclophosphamide, doxorubicin, vincristine, and prednisone (CHOP) versus other chemotherapy. Results A total 2971 patients with PTCL and chemotherapy information were included in the study; 1706 (57%) received CHOP and 1265 (43%) other chemotherapy. A majority of patients (51.7%) were female; mean (standard deviation) age at index was 61.0 (±16.0), Charlson score was 4.1 (±2.9), and follow-up time was 24.6 (±16.7) months. During the variable follow-up period, HRU was similar for the CHOP and other chemotherapy cohorts; 58.1% and 59.3% had ≥1 all-cause hospitalizations, respectively. The proportion of patients with ≥1 PTCL-related hospitalizations was higher in the CHOP than in the other chemotherapy cohort (40.3% vs. 9.7%, respectively) and mean length of stay was longer (4.6 vs. 3.7 days per patient per month, respectively). Conclusions This retrospective analysis of patients with PTCL revealed high levels of comorbidity and HRU; novel interventions that improve patient outcomes and reduce the HRU burden of PTCL are needed.

Persistence to hypomethylating agents and clinical and economic outcomes among patients with myelodysplastic syndromes

ABSTRACT Objectives To evaluate hypomethylating agent (HMA) persistence in patients with myelodysplastic syndromes (MDS), and examine its association with healthcare resource utilization (HRU) and progression to acute myeloid leukemia (AML). Methods A total of 2,400 adults diagnosed with MDS initiating HMAs were included from IBM MarketScan databases during 1/1/2011–3/31/2018. The index date was HMA initiation following MDS diagnosis. Patients were classified according to their persistence status by the end of a fixed ‘landmark period’ of 4 months post-index. Results Median persistence to HMAs was 5.6 months (95% CI: 5.2, 6.1); HMA non-persistence increased with time. Non-persistent patients had a significantly higher non-HMA-related HRU burden than persistent patients [adjusted incidence rate ratios, outpatient visits: 1.12 (95% CI: 1.10, 1.14); inpatient visits: 1.48 (95% CI: 1.30, 1.69); emergency department visits 1.30 (95% CI: 1.12, 1.50); all p-values < 0.001]. All-cause and HMA-related outpatient visits were lower among non-persistent patients, likely because of fewer HMA administration-related visits. The incidence rate of AML was numerically, although not significantly, higher in non-persistent patients, when starting follow-up at the end of the landmark period. When follow-up began at the index date, non-persistent patients had a significantly higher rate of AML [adjusted hazard ratio, 1.88 (95% CI: 1.53, 2.32); p-value < 0.001]. Conclusions HMA non-persistence, which increased over time, was associated with significantly higher non-HMA-related HRU, and numerically higher AML progression in MDS patients initiating HMAs. Future studies should evaluate predictors of HMA non-persistence in this patient population.

National Comprehensive Cancer Network (NCCN) category I/FDA-approved metastatic pancreatic adenocarcinoma (mPDAC) treatments in commercially insured patients: An analysis of inpatient (IP) and emergency room (ER) admissions.

e16739 Background: There are currently four NCCN category 1 systemic regimens approved in the United States for the treatment of mPDAC: FOLFIRINOX (FFX), gemcitabine+nab-paclitaxel (gem+nab-P), gemcitabine monotherapy (gem), and liposomal irinotecan + 5-fluorouracil/leucovorin (5-FU/LV) following progression with gem-based therapy. There is limited real-world research on the IP admissions and ER visit healthcare resource utilization (HRU) of patients receiving these treatments. Methods: Using the IQVIA PharMetrics Plus administrative claims database, data were analyzed for adult patients with mPDAC treated with NCCN category 1 regimens in first through fourth line of therapy between January 1, 2014 and May 31, 2019. For each line of therapy, continuous treatment was defined as the time from first administration of a therapy until the last administration. Mean all-cause and mPDAC-related IP admissions, ER visits, inpatient length of stay (LOS) during treatment were assessed. Results: Of the 2,731 patients with mPDAC included in the study, 101 (3.7%) were treated with a liposomal irinotecan based regimen, 1,316 (48.2%) were treated with gem+nab-P, 612 (22.4%) with FFX, and 624 (22.8%) with gem in any treatment line. The mean number of IP admissions was 1.2 for liposomal irinotecan treated patients, 1.5 for gem+nab-P, 1.5 for FFX, and 1.2 for gem. Among patients with at least one IP admission the mean LOS was 4.5 days for liposomal irinotecan, 5.4 days for gem+nab-P, 3.8 for FFX, and 5.1 for gem treated patients. Patients treated with liposomal irinotecan had a mean of 1.3 ER visits during treatment. Gem+nab-P, FFX, and gem-treated patients experienced 1.7, 1.4, and 1.8 mean ER visits, respectively. Mean mPDAC-related IP admissions ranged from 1.1 – 1.5, ER visits ranged from 1.1 – 1.7, and mean LOS ranged from 3.8 – 5.5 days. Conclusions: In this descriptive retrospective study patients receiving liposomal irinotecan, across all treatment episodes, generally experienced numerically lower mean IP admissions and ER visits. LOS was similar across all regimens. Further studies are necessary to characterize the IP and ER HRU burden among mPDAC patients treated with approved regimens.

Burden of illness among patients with severe aplastic anemia who have had insufficient response to immunosuppressive therapy: a multicenter retrospective chart review study.

This study assessed treatment patterns and healthcare resource utilization (HRU) of patients with severe aplastic anemia (SAA) with insufficient response to immunosuppressive therapy (IST). A retrospective chart review was conducted at Dana-Farber Cancer Institute (DFCI), United States, and Hôpital Saint-Louis (HSL), France. Eligible patients were ≥ 18 years old, diagnosed with acquired SAA between January 1, 2006, and July 31, 2016, had insufficient response to IST, and had ≥ 12 months of follow-up post-diagnosis. Overall survival (OS) was estimated using the Kaplan-Meier method. Among the 40 patients, mean age at diagnosis was 44 years and 53% were women. Median follow-up time after SAA diagnosis was 48.3 months. Ninety-five percent of patients received antithymocyte globulin (ATG) as primary therapy prior to hematopoietic stem cell transplant (HSCT). Most common secondary SAA therapies prior to HSCT were eltrombopag (28%) and androgens (15%). Seventy-five percent of patients received HSCT. Prior to HSCT, patients received an average of 2.7 red blood cell (RBC) and 3.3 platelet transfusions per month; patients had 0.9 hospitalizations, 0.4 emergency room visits, and 12.8 office visits per year. Five-year OS was 75%, with infection as the primary cause of death. Additionally, this study provides information on the subgroup of patients receiving eltrombopag which was the most common secondary therapy. This study quantified transfusion and HRU burden associated with SAA and demonstrated high 5-year survival in a recently treated cohort.

An observational study of pediatric healthcare burden in Angelman syndrome: results from a real-world study

BackgroundThe objective of this study is to describe variations in the healthcare resource utilization (HRU) among individuals with Angelman syndrome (AS) over the first 12 years of life. Data for this study were drawn from the AS Natural History study (ASNHS), which is an observational study on the developmental progress, behavior, and medical morbidity of individuals with AS conducted over eight years. Caregiver-reported information on hospitalization, surgery, and medication utilization was used to assess HRU. Repeated measures mixed effect models were used to assess the relationship between age and probability of hospitalization, surgery, and prescription medication utilization.ResultsMean age at study enrollment was 6 years of age and both sexes were equally represented. The mean number of visits per participant was three. Results from this study suggest that individuals with AS have a high HRU burden. Hospitalization and surgery burden were highest in the first year of life. Use of medications for seizures and sleep disturbance increased over time.ConclusionsThe study highlights the significant healthcare burden among individuals with AS. Future studies that estimate cost and caregiver burden associated with AS are needed to assess the lifelong economic impact of AS on families and healthcare system.

Real-World Clinical and Economic Outcomes and the Role of Bevacizumab in Patients With Non-Small-Cell Lung Cancer With Liver Metastases.

Liver metastases are associated with poor outcomes in patients with advanced non-small-cell lung cancer (aNSCLC). Nevertheless, the vasculature in the liver microenvironment may be conducive to the use of antiangiogenesis inhibitors to potentially improve outcomes. Limited real-world clinical and economic data are currently available for this patient subpopulation. Two retrospective cohort analyses were conducted using data from an electronic health record (n = 14,209) and a claims database (n = 9,017). Patients with aNSCLC with and without liver metastases were identified in each database. Patients with baseline liver metastases in the electronic health record database were further categorized into two subgroups-those who had or had not received bevacizumab-containing regimens. Multivariable Cox proportional hazards regression models were used to adjust for baseline characteristics to evaluate the effect of treatment of bevacizumab. Liver metastases were associated with significantly poorer survival (median overall survival, 6.3 months v 10.1 months) and higher costs and health care resource utilization-total per-patient-per-month costs of $27,589 versus $19,607. Cost differences were primarily driven by inpatient costs, including a two-fold increase in hospitalizations and a 1.7-fold higher mean length of stay. Treatment with bevacizumab was associated with improved survival. Whereas overall survival improved in patients with and without baseline liver metastases who received bevacizumab, the relative survival benefit was greater in patients with liver metastases (hazard ratio, 0.63 [95% CI, 0.50 to 0.81] v hazard ratio, 0.80 [95% CI, 0.74 to 0.86]). Patients with aNSCLC with liver metastases have poorer survival and a higher cost and health care resource utilization burden. Bevacizumab was associated with a survival benefit in patients with aNSCLC with liver metastases.

A Real-World Claims Analysis of Costs and Patterns of Care in Treated Patients with Glioblastoma Multiforme in the United States.

Patients with glioblastoma multiforme (GBM) have a poor prognosis and high likelihood of recurrence. Routine care for incident cases in the United States involves surgical resection, followed by radiation therapy (RT) with concurrent and adjuvant temozolomide. Real-world data reporting the treatments and health care burden associated with GBM are limited. To assess patterns of care, health care resource utilization (HCRU), and costs associated with treatment of GBM in the United States. This study is a retrospective claims database analysis. Adult patients with a GBM diagnosis (index date) between January 1, 2010, and June 30, 2016, who had undergone brain surgery within 90 days of the index date, had received temozolomide and/or RT up to 90 days after index date, and had at least 6 months of continuous enrollment before the index date, were identified. Patients were excluded if they had (a) another primary cancer within 6 months pre-index, (b) secondary brain metastases, or (c) received temozolomide and/or RT pre-index. Baseline characteristics, treatments, HCRU, and costs were reported. First-line therapy began upon first receipt of RT and/or temozolomide after index date; second-line therapy began when a new drug was added > 28 days after initiation of first-line therapy or when there was a treatment gap > 90 days. Treatment regimens, duration of treatment (corrected group prognosis method), HCRU, and costs were reported descriptively in the 0- to 6-month and 7- to 12-month periods following initiation of first-line and second-line therapy. Baseline characteristics were comparable between patients receiving temozolomide and/or RT. Patients receiving RT without chemotherapy tended to be older, be retired, and have more baseline comorbidities. Of the 4,071 patients receiving first-line therapy for GBM, most (73.0%) received temozolomide + RT; 24.4% received RT; and 2.5% received temozolomide monotherapy. Of those receiving first-line therapy, 1,283 (31.5%) patients subsequently received second-line therapy: 39.4% received bevacizumab monotherapy; 28.9% received bevacizumab combination therapy (temozolomide, 45.2% of patients; irinotecan, 24.3%; and temozolomide + lomustine, 15.4%); 15.5% received temozolomide monotherapy; and 13.7% received other systemic cancer therapies. The proportion of patients with hospitalizations increased from 2.9% (4-6 months pre-index) to 20.8% in the 3 months before the index date (likely due to diagnostic procedures) and 28.1% in the first 6 months after index (likely due to surgery) and then decreased to 13.3% in the 7- to 12-month period after index. Mean total per-patient costs at 6 and 12 months were $117,325 and $162,550 (first line) and $126,128 and $243,833 (second line). Costs in all time periods were largely driven by costs of RT/systemic cancer therapy. Most patients with newly diagnosed GBM received treatment according to recommendations. However, relatively few patients received second-line therapy, and the HCRU burden and costs associated with both lines of therapy were substantial. Novel therapies for GBM are required to improve treatment options and outcomes in these patients. This study was funded by Bristol-Myers Squibb (Princeton Pike, NJ). Neither honoraria nor payments were provided for authorship. Norden received consultancy fees relating to this study from Bristol-Myers Squibb. Dastani, Korytowsky, Le, Singh, and You are employees of Bristol-Myers Squibb. Dastani and Korytowsky are shareholders of Bristol-Myers Squibb. Bobiak was an employee of Bristol-Myers Squibb at the time of this study. Preliminary data from this study were previously presented at the International Society for Pharmacoeconomics and Outcomes Research 22nd Annual International Meeting in Boston, MA, May 20-24, 2017.

A rapid evidence assessment of bleed-related healthcare resource utilization in publications reporting the use of direct oral anticoagulants for non-valvular atrial fibrillation

Objective: Non-valvular atrial fibrillation (NVAF), a common cardiac arrhythmia, is associated with high morbidity and carries a substantial economic burden. Historically, vitamin K antagonists (VKAs; e.g. warfarin) have been used for therapy of NVAF, but recently several direct oral anticoagulants (DOACs) have been approved for prevention of stroke in patients with NVAF. This review summarizes the real-world evidence (RWE) for healthcare resource utilization (HRU) in patients receiving oral anticoagulants (VKAs and/or DOACs) for therapy of NVAF.Methods: A PRISMA-compliant literature search assessed Medline® and Embase® databases from 1 January 2011 to 4 May 2017, and the National Health Service Economic Evaluation Database from 1 January 2011 to 31 December 2015. Publications were included if they reported observational data from real-world use of one or more anticoagulant therapies. Outcomes of interest included hospitalizations, length of stay (LOS), mortality and costs.Results: Twenty-eight publications were included. Apixaban and dabigatran were associated with fewer bleed-related hospitalizations than warfarin. Bleed-related LOS were generally longer for warfarin than for DOACs. Bleed-related treatment costs were lower for patients receiving apixaban or receiving dabigatran than patients receiving rivaroxaban or receiving warfarin. Bleed-related mortality in patients receiving oral anticoagulation for treatment of NVAF were low across all DOACs and warfarin.Conclusions: The limited available evidence for HRU burden among patients receiving oral anticoagulation for NVAF suggests that DOACs (particularly apixaban and dabigatran) offer some degree of benefit in terms of HRU outcomes, compared with warfarin. Further work is required to understand HRU outcomes in patients receiving DOACs.

Health care burden and treatment patterns in commercially insured children with chronic idiopathic/spontaneous urticaria: A real-world study in the United States.

Chronic idiopathic urticaria (CIU)/spontaneous urticaria (CSU) is defined by the presence of wheals, angioedema, or both for ≥6 weeks, with or without an identifiable trigger. Real-world health care data among children with CIU/CSU remain scarce. To describe treatment patterns, health care resource utilization (HRU), and costs in pediatric patients with CIU/CSU (<12 years old) and to compare these with pediatric patients without CIU/CSU. A commercial administrative claims data base (September 2013 to June 2016) was used. The CIU/CSU cohort included pediatric patients with either two or more claims for a diagnosis of urticaria ≥6 weeks apart or one or more claims for a diagnosis of urticaria and one or more claims for a diagnosis of angioedema ≥6 weeks apart (index was defined as the first claim). The control cohort comprised pediatric patients without urticaria or angioedema (index randomly assigned). Patients with <6 months of eligibility before and after the index date were excluded. HRU and costs were compared between the cohorts during the observation period after propensity score matching. A total of 6109 pediatric patients with CIU/CSU were selected, and 6107 were 1:1 matched with controls. The patients with CIU/CSU who had a mean ± standard deviation age of 4.58 ± 3.36 years, and 47.9% were girls. CIU/CSU-related medication use increased after diagnosis (e.g., baseline versus 6-month follow-up, 2.2 versus 8.0% for nonsedating prescription H1 antihistamines; 7.4 versus 17.4% for oral corticosteroids). Relative to the controls, the patients with CIU/CSU had higher rates of HRU (incidence rate ratios of 1.71, 2.39, and 2.07 for inpatient, emergency department, and outpatient visits, respectively; all p < 0.01), and higher all-cause per patient per year costs (mean cost differences of $2090, $1606, and $483 for total, medical, and pharmacy costs, respectively; all p < 0.01). This study highlighted unmet needs in pediatric patients with CIU/CSU who had increased medication (e.g., oral corticosteroids) and HRU burden after a diagnosis for CIU/CSU, and higher rates of HRU and costs relative to those without CIU/CSU.

Economic burden of sarcoidosis in a commercially-insured population in the United States

Background: Sarcoidosis is a multi-system inflammatory disorder characterized by the presence of non-caseating granulomas in involved organs. Patients with sarcoidosis have a reduced quality-of-life and are at an increased risk for several comorbidities. Little is known about the direct and indirect cost of sarcoidosis following the initial diagnosis.Aims: To provide an estimate of the healthcare resource utilization (HCRU) and costs borne by commercial payers for sarcoidosis patients in the US.Methods: Patients with a first diagnosis of sarcoidosis between January 1, 1998 and March 31, 2015 (“index date”) were selected from a de-identified privately-insured administrative claims database. Sarcoidosis patients were required to have continuous health plan enrollment 12 months prior to and following their index dates. Propensity-score (1:1) matching of sarcoidosis patients with non-sarcoidosis controls was carried out based on a logistic regression of baseline characteristics. Burden of HCRU and work loss (disability days and medically-related absenteeism) were compared between the matched groups over the 12-month period following the index date (“outcome period”).Results: A total of 7,119 sarcoidosis patients who met the selection criteria were matched with a control. Overall, commercial payers incurred $19,714 in mean total annual healthcare costs per sarcoidosis patient. The principle cost drivers were outpatient visits ($9,050 2015 USD, 46%) and inpatient admissions ($6,398, 32%). Relative to controls, sarcoidosis patients had $5,190 (36%) higher total healthcare costs ($19,714 vs $14,524; p < 0.001). Sarcoidosis patients also had significantly more work loss days (15.9 vs 11.3; p < 0.001) and work loss costs ($3,288 vs $2,527; p < 0.001) than matched controls. Sarcoidosis imposes an estimated total direct medical cost of $1.3–$8.7 billion to commercial payers, and an indirect cost of $0.2–$1.5 billion to commercial payers in work loss.Conclusions: Sarcoidosis imposes a significant economic burden to payers in the first year following diagnosis.

Healthcare resource utilization and work loss in dermatomyositis and polymyositis patients in a privately-insured US population

Background Dermatomyositis and polymyositis (DM/PM) are inflammatory myopathies characterized by muscle inflammation/weakness. Patients with DM/PM have a reduced quality-of-life and are at an increased risk for several comorbidities. Studies have assessed the incidence and prevalence of DM/PM; however, no study has estimated the burden of the diseases in terms of both healthcare resource utilization (HCRU) and work loss incurred by patients.Objective To provide a comprehensive, current estimate of the annual HCRU and work loss in DM/PM patients in the US.Methods All patients (aged 18–64 years) with a first diagnosis of DM/PM between January 1, 1998 and March 31, 2014 (‘index date’) were selected from a de-identified privately-insured administrative claims database. DM/PM patients were required to have continuous health-plan enrollment 12 months prior to and following their index date. Propensity-score (1:1) matching of DM/PM patients with non-DM/PM controls was carried out based on a logistic regression of demographic characteristics, comorbidities, costs, and HCRU to control for these confounding factors. Burden of HCRU and work loss (disability days and medically-related absenteeism) were compared between the matched DM/PM and the non-DM/PM cohorts over the 12-month period after the index date (‘outcome period’).Results Of the 2617 DM/PM patients that met sample selection criteria, 2587 (98.9%) were matched with a non-DM/PM control. During the outcome period, DM/PM patients had significantly increased HCRU across places of service, including 44% more inpatient admissions (3.6 vs 2.5, p < 0.001), increased visits with specialists such as rheumatologists, neurologists and physical therapists, and filled 4.7 more prescriptions (32.2 vs 27.5, p < 0.001) than matched control patients. The increased HCRU led to significantly more medically-related work loss among DM/PM patients than matched controls (p < 0.001).Conclusions DM/PM imposes a substantial increase in healthcare resource use and is associated with statistically significantly greater work loss in the first year following diagnosis.