Treatment-Free Interval: A Novel Approach to Assessing Real-World Treatment Effectiveness and Economic Impact Among Patients with Irritable Bowel Syndrome with Diarrhea.

INTRODUCTION: Objective assessment of treatment effectiveness using real-world claims data is challenging. This study assessed treatment-free intervals (TFI) as a proxy for treatment effectiveness, and all-cause healthcare costs among adult patients with irritable bowel syndrome with diarrhea (IBS-D) treated with rifaximin or eluxadoline in the USA. METHODS: Adult patients (18-64 years) with IBS-D and ≥ 1 rifaximin or eluxadoline prescription were identified in the IQVIA PharMetrics® Plus database (10/01/2015-12/31/2021) and classified into two mutually exclusive cohorts (i.e., rifaximin and eluxadoline). Index date was the date of rifaximin or eluxadoline initiation. Entropy-balanced baseline characteristics, TFI (periods of ≥ 30 consecutive days without IBS-D treatment), and healthcare costs were reported. Healthcare costs were compared between cohorts using mean cost differences. RESULTS: There were 7094 and 2161 patients in the rifaximin and eluxadoline cohorts, respectively. After balancing, baseline characteristics (mean age 44.1 years; female 72.4%) were similar between cohorts. A higher proportion of patients treated with rifaximin achieved a TFI of ≥ 30 days (76.2% vs. 66.7%), ≥ 60 days (67.0% vs. 47.0%), ≥ 90 days (61.0% vs. 38.7%), ≥ 180 days (51.7% vs. 31.0%), and ≥ 240 days (47.7% vs. 27.9%) compared to eluxadoline. Among patients with a TFI ≥ 30 days, mean TFI durations were 8.3 and 6.0 months for the rifaximin and eluxadoline cohorts. Mean all-cause healthcare costs were lower for rifaximin vs. eluxadoline ($18,316 vs. $23,437; p = 0.008), primarily driven by pharmacy costs ($7348 vs. $10,250; p < 0.001). In a simulated health plan of one million commercially insured lives, initiating 50% of patients on rifaximin instead of eluxadoline resulted in total cost savings of $2.1 million per year or $0.18 per-member-per-month. CONCLUSIONS: This real-world study suggests that TFI is a meaningful surrogate measure of treatment effectiveness in IBS-D. Patients treated with rifaximin had longer treatment-free periods and lower healthcare costs than patients treated with eluxadoline.

Impact of living with an adult with depressive symptoms among households in the United States.

BACKGROUND: The effect of depressive symptoms on individuals has been widely studied but their impact on households remains less explored. This study assessed the humanistic and economic impact of living with an adult with depressive symptoms on adults without depressive symptoms among households in the United States (US). METHODS: The Medical Expenditure Panel Survey (MEPS) Household Component database was used to identify adults without depressive symptoms living in households with ≥1 adult with depressive symptoms (depression household) and adults without depressive symptoms living in households without an adult with depressive symptoms (no-depression household). Weighted generalized linear models with clustered standard errors were used to compare total income (USD 2020), employment status, workdays missed, quality of life (QoL), and healthcare resource utilization (HRU) between cohorts. RESULTS: Adults without depressive symptoms living in a depression household (n = 1699) earned $4720 less in total annual income (representing 11.3% lower than the average income of $41,634 in MEPS), were less likely to be employed, missed more workdays per year, and had lower QoL than adults without depressive symptoms living in a no-depression household (n = 15,286). Differences in total annual healthcare costs and for most types of HRU, except for increased outpatient mental health-related visits, were not significant. LIMITATIONS: Data is subject to reporting bias, misclassification, and other inaccuracies. Causal inferences could not be established. CONCLUSION: The economic and humanistic consequences of depressive symptoms may extend beyond the affected adults and impact other adult members of the household.

Impact of rifaximin use following an initial overt hepatic encephalopathy hospitalization on rehospitalization and costs.

AIM: To assess the impact of rifaximin (± lactulose) use following discharge of an initial overt hepatic encephalopathy (OHE) hospitalization on OHE rehospitalizations and healthcare costs in a real-world setting. METHODS: Adults (18-64 years) with an OHE hospitalization were identified from MarketScan® Commercial claims (Q4'15-Q2'20), classified into two mutually exclusive treatment cohorts (i.e. rifaximin and no rifaximin treatment), and further stratified into four subgroups based on decreasing quality of care (QoC; i.e. Type 1 - rifaximin without delay post-discharge; Type 2 - rifaximin with delay post-discharge; Type 3 - lactulose only post-discharge; Type 4 - no rifaximin/lactulose treatment post-discharge). The impact of rifaximin use on 30-day and annualized OHE hospitalizations and healthcare costs were assessed between cohorts and by the QoC subgroup. RESULTS: Characteristics were similar between the rifaximin (N = 1,452; Type 1: 1,138, Type 2: 314) and no rifaximin (N = 560; Type 3:337, Type 4: 223) treatment cohorts. The 30-day risk of OHE rehospitalization was lower for the rifaximin vs. no rifaximin treatment cohort (odds ratio 0.56, p < .01) and increased with decreasing QoC. The annual rate of OHE hospitalizations was 59% lower for the rifaximin treatment cohort (incidence rate ratio 0.41, p < .01) and increased with decreasing QoC. Compared to the no rifaximin treatment cohort, the rifaximin treatment cohort had higher pharmacy costs, lower medical costs, and no difference in total healthcare costs. LIMITATIONS: This was a claims-based study subject to common data limitations such as billing inaccuracies or omissions in coded claims. Total healthcare costs were reported from a payer's perspective, which do not capture indirect costs associated with patient burden. CONCLUSIONS: Initiation of rifaximin after an OHE hospitalization was associated with reduced OHE hospitalizations both in the 30-days following and annually. Further, reduced medical costs offset increased pharmacy costs, and no annual cost differences were observed between cohorts.

The Economic Burden of Adults with Major Depressive Disorder in the United States (2019).

INTRODUCTION: Previous societal burden estimations for major depressive disorder (MDD) often fail to account for several hidden cost components. This study provides a comprehensive evaluation of societal costs for adults with MDD in the United States (USA) in 2019. The potential impact of a more effective, rapid-acting MDD therapy vs standard of care on the economic burden of MDD was estimated to illustrate the utility of such a framework in evaluating new interventions. METHODS: This study used a prevalence-based human capital approach. Incremental costs (2019 US dollars) per individual with MDD were derived from national survey inputs and published literature and included incremental healthcare costs and indirect costs. For each cost component, the societal costs were extrapolated by multiplying the per-patient costs by the number of individuals with MDD. The impact of a more effective, rapid-acting novel therapy on the economic burden of MDD was then simulated on the basis of these inputs. RESULTS: In 2019, the number of adults with MDD in the USA was estimated at 19.8 million (62.7% female; 32.9% severe MDD), and the incremental societal economic burden of MDD was estimated at $333.7 billion ($382.4 billion in 2023 US dollars), or $16,854 per adult with MDD. The primary cost drivers were healthcare costs ($127.3 billion; 38.1%), household-related costs ($80.1 billion; 24.0%), presenteeism ($43.3 billion; 13.0%), and absenteeism ($38.4 billion; 11.5%). In the simulated scenario, a hypothetical novel therapy with a 50.0% early response rate was associated with a 7.7% reduction in the economic burden of MDD relative to standard of care over 12 months. CONCLUSIONS: The economic burden of MDD is substantial and extends beyond healthcare costs, underscoring the impact of MDD across multiple aspects of life. Such a broad societal perspective should be considered in assessing the impact of the advent of effective, rapid-acting MDD therapies.

Treatment Patterns Among Patients with Attention-Deficit/Hyperactivity Disorder and Comorbid Anxiety and/or Depression in the United States: A Retrospective Claims Analysis.

INTRODUCTION: Patients with attention-deficit/hyperactivity disorder (ADHD) often have psychiatric comorbidities that may confound diagnosis and affect treatment outcomes and costs. The current study described treatment patterns and healthcare costs among patients with ADHD and comorbid anxiety and/or depression in the United States (USA). METHODS: Patients with ADHD initiating pharmacological treatments were identified from IBM MarketScan Data (2014-2018). The index date was the first observed ADHD treatment. Comorbidity profiles (anxiety and/or depression) were assessed during the 6-month baseline period. Treatment changes (discontinuation, switch, add-on, drop) were examined during the 12-month study period. Adjusted odds ratios (ORs) of experiencing a treatment change were estimated. Adjusted annual healthcare costs were compared between patients with and without treatment changes. RESULTS: Among 172,010 patients with ADHD (children [aged 6-12] N = 49,756; adolescents [aged 13-17] N = 29,093; adults [aged 18 +] N = 93,161), the proportion of patients with anxiety and depression increased from childhood to adulthood (anxiety 11.0%, 17.7%, 23.0%; depression 3.4%, 15.7%, 19.0%; anxiety and/or depression 12.9%, 25.4%, 32.2%). Compared with patients without the comorbidity profile, those with the comorbidity profile experienced a significantly higher odds of a treatment change (ORs [children, adolescents, adults] 1.37, 1.19, 1.19 for those with anxiety; 1.37, 1.30, 1.29 for those with depression; and 1.39, 1.25, 1.21 for those with anxiety and/or depression). Excess costs associated with a treatment change were generally higher with more treatment changes. Among patients with three or more treatment changes, annual excess costs per child, adolescent, and adult were $2234, $6557, and $3891 for those with anxiety; $4595, $3966, and $4997 for those with depression; and $2733, $5082, and $3483 for those with anxiety and/or depression. CONCLUSIONS: Over 12 months, patients with ADHD and comorbid anxiety and/or depression were significantly more likely to experience a treatment change than those without these psychiatric comorbidities and incurred higher excess costs with additional treatment changes.

Treatment patterns among children and adolescents with attention-deficit/hyperactivity disorder in the United States - a retrospective claims analysis.

BACKGROUND: Attention-deficit/hyperactivity disorder (ADHD) is a common neurobehavioral disorder affecting approximately 10.0% of children and 6.5% of adolescents in the United States (US). A comprehensive assessment of the current treatment landscape is warranted to highlight potential unmet needs of children and adolescents with ADHD. Therefore, this study described treatment patterns and healthcare costs among commercially insured children and adolescents with ADHD in the US. METHODS: Children and adolescents with ADHD initiating pharmacological treatment indicated for ADHD were identified from IBM MarketScan Commercial Database (2014-2018). A treatment sequence algorithm was used to examine treatment patterns, including discontinuation (≥ 180 days following the last day of supply of any ADHD treatment), switch, add-on, and drop (discontinuation of an agent in combination therapy), during the 12-month study period following the index date (i.e., first observed ADHD treatment). Total adjusted annual healthcare costs were compared between patients with and without treatment changes. RESULTS: Among 49,756 children and 29,093 adolescents included, mean age was 9 and 15 years, respectively, and 31% and 38% were female. As the first treatment regimen observed, 92% of both children and adolescents initiated a stimulant and 11% initiated combination therapy. Over half of the population had a treatment change over 12 months-59% of children and 68% of adolescents. Treatment discontinuation over 12 months was common in both populations-21% of children and 36% of adolescents discontinued treatment. Healthcare costs increased with the number of treatment changes observed; children and adolescents with treatment changes (i.e., 1, 2, or ≥ 3) incurred an incremental annual cost of up to $1,443 and $2,705, respectively, compared to those without a treatment change (p < 0.001). Costs were largely driven by outpatient visits. CONCLUSIONS: Over a 12-month period, treatment changes were commonly observed and were associated with excess costs, highlighting the unmet treatment needs of children and adolescents with ADHD in the US.

The Economic Burden of Posttraumatic Stress Disorder in the United States From a Societal Perspective.

Objective: To estimate the economic burden of posttraumatic stress disorder (PTSD) in the United States civilian and military populations from a societal perspective.Methods: A prevalence-based and human capital approach was used to estimate the total excess costs of PTSD in 2018 from insurance claims data, academic literature, and governmental publications. Excess direct health care costs (pharmacy, medical), direct non-health care costs (research and training, substance use, psychotherapy, homelessness, disability), and indirect costs (unemployment, productivity loss, caregiving, premature mortality) associated with PTSD were compared between adults with PTSD and adults without PTSD, or the general population if information was not available for adults without PTSD.Results: The total excess economic burden of PTSD in the US was estimated at $232.2 billion for 2018 ($19,630 per individual with PTSD). Total excess costs were $189.5 billion (81.6%) in the civilian population and $42.7 billion (18.4%) in the military population, corresponding to $18,640 and $25,684 per individual with PTSD in the civilian and military populations, respectively. In the civilian population, the excess burden was driven by direct health care ($66.0 billion) and unemployment ($42.7 billion) costs. In the military population, the excess burden was driven by disability ($17.8 billion) and direct health care ($10.1 billion) costs.Conclusions: The economic burden of PTSD goes beyond direct health care costs and has been found to rival costs for other costly mental health conditions. Increased awareness of PTSD, development of more effective therapies, and expansion of evidence-based interventions may be warranted to reduce the large clinical and economic burden of PTSD.

Opioid-Induced Constipation: Cost Impact of Approved Medications in the Emergency Department.

INTRODUCTION: Opioid-induced constipation (OIC) prescription medications (OIC-Rx) like methylnaltrexone subcutaneous (SC) have shown efficacy in treating OIC in the emergency department (ED). This study aimed to describe and compare healthcare resource utilization (HRU) and healthcare costs in ED patients with OIC receiving OIC-Rx versus those not receiving OIC-Rx. METHODS: Adult patients with OIC during an ED encounter were identified from a hospital-based ED encounters database (2016-2019) and classified on the basis of receipt of OIC-Rx (OIC-Rx versus No OIC-Rx cohorts). Entropy balancing was used to reweight characteristics of the two cohorts. HRU and healthcare costs were measured and compared during the ED encounter and 30-day post-discharge period. RESULTS: Among 11,135 patients in the OIC-Rx cohort (21,474 in the No OIC-Rx cohort), 93% received methylnaltrexone SC. Patients in the OIC-Rx cohort had 0.7 fewer inpatient days per OIC ED encounter and 64% decreased odds of being hospitalized versus the No OIC-Rx cohort (both p < 0.001). During the post-discharge period, the OIC-Rx cohort had 35% decreased odds of any re-encounter (p < 0.001). The OIC-Rx cohort had a $732 reduction in costs per OIC ED encounter versus the No OIC-Rx cohort (p < 0.001), driven by larger hospitals and patients with Medicare or Commercial insurance. During the post-discharge period, the OIC-Rx cohort had a $421 reduction in costs associated with any re-encounter versus the No OIC-Rx cohort (p = 0.004). CONCLUSION: Patients receiving OIC-Rx in the ED had decreased odds of being hospitalized and fewer re-encounters in the 30-day post-discharge period versus patients who did not receive OIC-Rx, resulting in cost savings for insurance agencies and healthcare providers.

Economic burden of attention-deficit/hyperactivity disorder among children and adolescents in the United States: a societal perspective.

OBJECTIVE: To provide a comprehensive evaluation of the economic burden associated with attention-deficit/hyperactivity disorder (ADHD) among children and adolescents from a US societal perspective. MATERIALS AND METHODS: Direct healthcare costs of children (5-11 years) and adolescents (12-17 years) with ADHD were obtained using claims data from the IBM MarketScan Research Databases (01/01/2017-12/31/2018). Direct non-healthcare and indirect costs were estimated based on literature and government publications. Each cost component was estimated using a prevalence-based approach, with per-patient costs extrapolated to the national level. RESULTS: The total annual societal excess costs associated with ADHD were estimated at $19.4 billion among children ($6,799 per child) and $13.8 billion among adolescents ($8,349 per adolescent). Education costs contributed to approximately half of the total excess costs in both populations ($11.6 billion [59.9%] in children; $6.7 billion [48.8%] in adolescents). Other major contributors to the overall burden were direct healthcare costs ($5.0 billion [25.9%] in children; $4.0 billion [29.0%] in adolescents) and caregiving costs ($2.7 billion [14.1%] in children; $1.6 billion [11.5%] in adolescents). LIMITATIONS: Cost estimates were calculated based on available literature and/or governmental publications due to the absence of a single data source for all costs associated with ADHD. Thus, the quality of cost estimates is limited by the accuracy of available data as well as the study populations and methodologies used by different studies. CONCLUSION: ADHD in children and adolescents is associated with a substantial economic burden that is largely driven by education costs, followed by direct healthcare costs and caregiver costs. Improved intervention strategies and policies may reduce the clinical and economic burden of ADHD in these populations.

Economic burden of attention-deficit/hyperactivity disorder among adults in the United States: a societal perspective.

BACKGROUND: Attention-deficit/hyperactivity disorder (ADHD) is associated with substantial clinical burden as individuals transition to adulthood, including higher rates of comorbidities, mortality, incarceration, and psychiatric hospitalizations than in individuals without ADHD. These higher rates likely contribute to substantial economic burden as well. OBJECTIVE: To provide a comprehensive evaluation of the economic burden associated with ADHD in the US adult population. METHODS: Direct health care costs were obtained by using claims data from the IBM MarketScan Research Databases (January 1, 2017, through December 31, 2018). Direct non-health care costs and indirect costs were estimated on the basis of the literature and government publications. Excess costs incurred by adults with ADHD during 2018 were evaluated from a societal perspective; per-patient costs were extrapolated to the national level. RESULTS: An estimated 8.7 million adults live with ADHD in the United States, resulting in a total societal excess cost attributable to ADHD of $122.8 billion ($14,092 per adult). Excess costs of unemployment ($66.8 billion; 54.4%) comprised the largest proportion of the total, followed by productivity loss ($28.8 billion; 23.4%) and health care services ($14.3 billion; 11.6%). CONCLUSIONS: ADHD in adults is associated with substantial economic burden. DISCLOSURES: This study was funded by Otsuka Pharmaceutical Development & Commercialization, Inc. (Otsuka). The study sponsor contributed to and approved the study design, participated in the interpretation of data, and reviewed and approved the manuscript. Schein is an employee of Otsuka. Gagnon-Sanschagrin, Davidson, Kinkead, Cloutier, Guérin, and Lefebvre are employees of Analysis Group, Inc., a consulting company that provided paid consulting services to Otsuka to develop and conduct this study and write the manuscript. Adler has received research support from Shire/Takeda, Sunovion, and Otsuka; consulting fees from Bracket, Shire/Takeda, Sunovion, Otsuka, the State University of New York (SUNY), the National Football League (NFL), and Major League Baseball (MLB); and royalty payments (as inventor) from New York University (NYU) for license of adult ADHD scales and training materials. Childress has received research support from Allergan, Takeda/Shire, Emalex, Akili, Ironshore, Arbor, Aevi Genomic Medicine, Neos Therapeutics, Otsuka, Pfizer, Purdue, Rhodes, Sunovion, Tris, KemPharm, Supernus, and the US Food and Drug Administration; was on the advisory board of Takeda/Shire, Akili, Arbor, Cingulate, Ironshore, Neos Therapeutics, Otsuka, Pfizer, Purdue, Adlon, Rhodes, Sunovion, Tris, Supernus, and Corium; received consulting fees from Arbor, Ironshore, Neos Therapeutics, Purdue, Rhodes, Sunovion, Tris, KemPharm, Supernus, Corium, Jazz, and Tulex Pharma; received speaker fees from Takeda/Shire, Arbor, Ironshore, Neos Therapeutics, Pfizer, Tris, and Supernus; and received writing support from Takeda/Shire, Arbor, Ironshore, Neos Therapeutics, Pfizer, Purdue, Rhodes, Sunovion, and Tris. Part of the material in this study was presented as a poster at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 2021 Virtual Meeting; May 17-20, 2021.

Healthcare costs among patients with macular oedema associated with non-infectious uveitis: a US commercial payer's perspective.

OBJECTIVE: To describe patient characteristics and healthcare costs associated with uveitic macular oedema (UME) in US clinical practices from a commercial payer's perspective. METHODS AND ANALYSIS: The IBM MarketScan Commercial Subset (1 October 2015-31 March 2020) was used to identify patients with non-infectious uveitis (NIU), with or without UME. Patients with UME at any time were further classified into subgroups of patients who received a UME diagnosis during the study period and those who received a UME diagnosis and local steroid injection (LSI) during the study period. Demographic and clinical characteristics, NIU-related treatments and healthcare costs were described for each cohort and subgroup during the most recent 12 months of continuous health plan enrolment. Healthcare costs were also described by vision status among all patients with NIU. RESULTS: A total of 36 322 patients with NIU were identified, of whom 3 301 (9.1%) had UME and 33 021 (90.9%) had no UME. Patients with UME more frequently received NIU-related treatment compared with those without UME (64.6% vs 45.0%), particularly LSI treatment (12.5% vs 0.7%). Mean total all-cause healthcare costs per-patient-per-year (PPPY) were higher among patients with UME ($19 851) than patients without UME ($16 188) and were especially high among those with bilateral UME ($24 162). Further, vision loss was more commonly observed in those with UME versus those without UME (5.7% vs 2.2%) and a trend of increasing healthcare costs with increasing vision loss was observed. CONCLUSION: NIU is associated with substantial clinical and economic burden, particularly when UME is present.

Treatment patterns among adults with attention-deficit/hyperactivity disorder in the United States: a retrospective claims study.

OBJECTIVE: To assess treatment patterns in adults with attention-deficit/hyperactivity disorder (ADHD) and associated healthcare costs in a real-world US setting. METHODS: Claims data from the IBM MarketScan Commercial Subset (Q1/2014-Q4/2018) was used to identify adults diagnosed with ADHD who newly initiated on ADHD treatment (index date). Treatment sequences were defined using an algorithm; for each sequence, the regimen comprised all ADHD-related agents observed within 30 d of the first agent during the 12-month study period. Treatment changes included discontinuation, switch, add-on, and drop. Treatment characteristics were described for the first treatment regimen observed. Total adjusted annual healthcare costs were compared between patients with no treatment change and patients with 1, 2, and ≥3 treatment changes. RESULTS: Among 122,881 adults with ADHD, the majority initiated a stimulant (95.1%) as their first treatment regimen observed; 9.3% of patients initiated combination therapy of ≥2 ADHD-related agents, and 34.9% of patients had psychotherapy. After an average first treatment regimen duration of 7.1 months, 50.2% of patients experienced a treatment change (22.5% discontinued, 17.5% switched, 5.3% had an add-on, and 4.6% had a treatment drop). Among those who discontinued, 44.8% did so within the first month of initiation. Mean annual healthcare costs were higher among patients with at least 1 treatment change compared to those with no treatment changes; excess costs increased with each additional treatment change. CONCLUSIONS: Treatment changes were commonly observed and were associated with excess healthcare cost, emphasizing the unmet treatment needs of adults with ADHD in the US.

Excess healthcare costs in patients with autosomal dominant polycystic kidney disease by renal dysfunction stage.

AIM: To build upon previous outdated studies by comprehensively assessing the direct healthcare burden of autosomal dominant polycystic kidney disease (ADPKD). MATERIALS AND METHODS: Patients with ≥2 diagnoses for ADPKD (ADPKD cohort) were identified in the US fee-for-use IBM Truven Health Analytics MarketScan Commercial Claims and Encounters and IBM Truven Health Analytics MarketScan Medicare Supplemental databases (01 January 2015-31 December 2017) and matched (1:3) to controls without ADPKD (non-ADPKD cohort). The index date was the last calendar date followed by 12 months continuous enrollment (study period). Patients with ADPKD were stratified into one of seven mutually exclusive groups based on chronic kidney disease (CKD) stages (I-V), end-stage renal disease requiring renal replacement therapy (ESRD-RRT), and unknown stage. RESULTS: During the 12-month study period, patients with ADPKD incurred significantly higher total healthcare costs than those without ADPKD (mean cost difference = $22,879 per patient per year [PPPY]; p < .001). Besides CKD stages I and II, total healthcare cost differences increased as patients progressed beyond CKD stage III, with the greatest difference observed among patients with ESRD-RRT. Total healthcare cost differences between cohorts were more pronounced in subgroups of patients with hypertension ($29,347) and with high risk of rapid progression ($39,976). Similar results were observed in the Medicare Supplemental population, with a total mean cost difference of $42,694 PPPY (p < .001); cost difference was also higher in the hypertension ($46,461 PPPY) and high risk of rapid progression ($45,708 PPPY) subgroups. LIMITATIONS: Results may not be representative of the overall ADPKD US population; CKD stage was based on diagnosis and procedure codes; criteria used to identify ADPKD at risk of rapid progression did not rely on laboratory values; there may be billing inaccuracies and omissions in health insurance claims data. CONCLUSIONS: This study demonstrated the substantial healthcare costs associated with ADPKD, which increased as patients progressed through more severe CKD stages.

Vaso-occlusive crises and costs of sickle cell disease in patients with commercial, Medicaid, and Medicare insurance - the perspective of private and public payers.

AIM: To characterize vaso-occlusive crises (VOCs) and describe healthcare costs among commercially-insured, Medicaid-insured, and Medicare-insured patients with sickle cell disease (SCD). MATERIALS AND METHODS: The IBM Truven Health MarketScan Commercial (2000-2018), Medicaid Analytic eXtract (2008-2014), and Medicare Research Identifiable Files (2012-2016) databases were used to identify patients with ≥2 SCD diagnoses. Study measures were evaluated during a 12-month follow-up period, stratified by annual number of VOCs (i.e. 0, 1, and ≥2). RESULTS: Among 16,092 commercially-insured patients (mean age = 36.7 years), 35.3% had 1+ VOCs. Mean annual total all-cause healthcare costs were $15,747, $27,194, and $64,555 for patients with 0, 1, and 2+ VOCs, respectively. Total all-cause healthcare costs were mainly driven by inpatient (0 VOC = 31.0%, 1 VOC = 53.1%, 2+ VOCs = 65.4%) and SCD-related costs (0 VOC = 56.4%, 1 VOC = 78.4%, 2+ VOCs = 93.9%). Among 18,287 Medicaid-insured patients (mean age = 28.5 years, fee-for-service = 50.2%), 63.9% had 1+ VOCs. Mean annual total all-cause healthcare costs were $16,750, $29,880, and $64,566 for patients with 0, 1, and 2+ VOCs, respectively. Inpatient costs (0 VOC = 37.2%, 1 VOC = 64.3%, 2+ VOCs = 72.9%) and SCD-related costs (0 VOC = 60.9%, 1 VOC = 73.8%, 2+ VOCs = 92.2%) accounted for a significant proportion of total all-cause healthcare costs. Among 15,431 Medicare-insured patients (mean age = 48.2 years), 55.1% had 1+ VOCs. Mean annual total all-cause healthcare costs were $21,877, $29,250, and $58,308 for patients with 0, 1, and ≥2 VOCs, respectively. Total all-cause healthcare costs were mainly driven by inpatient (0 VOC = 47.9%, 1 VOC = 54.9%, 2+ VOCs = 67.5%) and SCD-related costs (0 VOC = 74.9%, 1 VOC = 84.4%, 2+ VOCs = 95.3%). LIMITATIONS: VOCs managed at home were not captured. Analyses were descriptive in an observational setting; thus, no causal relationships can be inferred. CONCLUSIONS: A high proportion of patients experienced VOCs across payers. Furthermore, inpatient and SCD-related costs accounted for a significant proportion of total all-cause healthcare costs, which increased with VOC frequency.

Institutionalization risk and costs associated with agitation in Alzheimer's disease.

INTRODUCTION: Agitation in individuals with Alzheimer's disease (AD) may predict institutionalization. This study assessed the incremental risk and costs associated with agitation in individuals with AD. METHODS: A retrospective analysis of the National Alzheimer's Coordinating Center Uniform Data Set (June 2005-February 2018) was conducted. Incremental risk of institutionalization associated with agitation was estimated and used with the number of institutionalized individuals with AD and agitation and costs of living by residential setting in the United States (literature-based), to estimate incremental institutionalization costs. RESULTS: The analysis included 11,348 individuals with AD: 6603 (58.2%) with and 4745 (41.8%) without agitation. Compared with individuals without agitation, those with agitation were 20% more likely to be institutionalized (odds ratio = 1.20; 95% CI = 1.08-1.33). Total incremental cost of institutionalization associated with agitation was $4.3 billion ($50,588/individual). DISCUSSION: Agitation is associated with a higher risk of institutionalization among patients with AD, which translates into a substantial economic burden.

The Economic Burden of Nocturia on the U.S. Health Care System and Society: A National Health and Nutrition Examination Survey Analysis.

BACKGROUND: Nocturia, characterized as waking during the main sleep period to urinate, is a common condition. Persistent nocturia results in sleep fragmentation with deleterious effects on health and well-being. Yet, there are limited data on the economic burden of nocturia in the United States. OBJECTIVE: To assess the association of nocturia with health care resource utilization (HRU), work productivity, and self-rated health while estimating the societal costs of nocturia in the United States in 2017. METHODS: A retrospective cross-sectional study was conducted using data from the National Health and Nutrition Examination Survey (NHANES; 2005-2006 to 2013-2014). Adults aged ≥ 18 years (excluding pregnant women) were stratified into individuals with nocturia (≥ 2 voids/night) and individuals without nocturia (< 2 voids/night), based on the threshold for clinically significant nocturia. Outcomes were self-reported and included HRU (hospitalizations, outpatient visits); work productivity (weekly hours worked, employment); and current health status. Multivariable regression analyses adjusting for age, race, sex, body mass index, insurance status, education level, alcohol use, smoking status, and self-reported comorbid conditions were used to compare the 2 cohorts, overall and stratified by age group (20-44 years, 45-64 years, and 65+ years) to distinguish the effects on different age groups including the Medicare-aged population. Excess direct health care costs and indirect productivity costs associated with nocturia in the United States were then calculated using a prevalence-based approach and available literature (i.e., nocturia prevalence estimates, aggregated unit costs by HRU type, and average hourly earnings in the United States). RESULTS: 22,300 individuals were identified, and 24% had nocturia (≥ 2 voids/night). Median age was 55.2 and 43.2 years among individuals with and without nocturia, respectively, and the proportion of males was 43.3% and 51.3%, respectively. Individuals with nocturia had significantly more HRU, including hospitalizations and outpatient visits, worked significantly fewer hours weekly, and were significantly less likely to be employed when compared with those without nocturia. They were also significantly less likely to report being in very good/excellent health. These comparisons remained statistically significant across age groups. Total excess direct health care costs were $62.9 billion (hospitalization: $47.6 billion; outpatient: $15.3 billion). Total excess indirect productivity costs were $151.7 billion. Altogether, costs were estimated at $214.5 billion, equivalent to $3,491 per individual with nocturia. Individuals aged 20-44 years incurred 23.5% of total excess costs, while those aged 45-64 and 65+ years incurred 48.2% and 28.3%, respectively. Sensitivity analyses based on lower prevalence estimates resulted in costs of $94.0 billion, while those based on higher prevalence estimates reached up to $231.1 billion. CONCLUSIONS: Nocturia is associated with a substantial economic burden in the United States even when evaluated based on lower prevalence estimates. This study underscores the importance of timely diagnosis and management of nocturia patients to alleviate health-related and economic consequences to patients and society. DISCLOSURES: This work was supported by Ferring Pharmaceuticals, which contributed to and approved the study design and participated in the interpretation of data, review, and approval of the manuscript. Gauthier-Loiselle, Gagnon-Sanschagrin, and Wu are employees of Analysis Group, which received consultancy fees from Ferring Pharmaceuticals for work on this study. Jhaveri is a full-time employee of Ferring Pharmaceuticals. Parts of this work were presented as a poster presentation at AMCP Nexus 2018; October 22-25, 2018; Orlando, FL.

A real-world analysis of healthcare costs and relative risk of hyperprolactinemia associated with antipsychotic treatments in the United States.

AIMS: Antipsychotic medications are associated with an increased risk of hyperprolactinemia, but differ in their propensity to cause this complication. This study aimed to assess the economic burden of hyperprolactinemia, and to compare its risk among adult patients using atypical antipsychotics (AAs) with a mechanism of action associated with no/low vs high/moderate prolactin elevation. METHODS: This retrospective cohort study was based on US Commercial and Medicaid claims databases. Healthcare costs were compared between matched hyperprolactinemia and hyperprolactinemia-free cohorts using a two-part model. Risk of hyperprolactinemia was compared between patients receiving AAs with a mechanism of action associated with no/low (no/low prolactin elevation cohort) vs high/moderate prolactin elevation (high/moderate prolactin cohort) using logistic regression. RESULTS: In the commercially insured sample, compared to the hyperprolactinemia-free cohort (n = 499), the hyperprolactinemia cohort (n = 499) was associated with incremental total healthcare costs of $5,732 ($20,081 vs $14,349; p = .004), and incremental medical costs of $3,861 ($13,218 vs $9,357; p = .040), mainly driven by hyperprolactinemia-related costs. In the Medicaid-insured sample, compared to the hyperprolactinemia-free cohort, the hyperprolactinemia cohort was associated with incremental total healthcare costs of $10,773 ($30,763 vs $19,990; p = .004), and incremental medical costs of $9,246 ($20,859 vs $11,613; p = .004), mainly driven by hyperprolactinemia-related and mental health-related costs. The odds of hyperprolactinemia in the no/low prolactin elevation cohort were 4-5-times lower than that in the high/moderate prolactin elevation cohort (odds ratio =0.21; p < .001). LIMITATIONS: Hyperprolactinemia may be under-reported in claims data. CONCLUSIONS: Hyperprolactinemia is associated with substantial healthcare costs. AAs associated with no/low prolactin elevation reduce the risk of hyperprolactinemia by 4-5-times compared to AAs associated with moderate/high prolactin elevation. Treatment options with minimal impact on prolactin levels may contribute to reducing hyperprolactinemia burden in AA-treated patients.

Treatment and Monitoring Patterns Among Premenopausal Women with HR+/HER2- Advanced Breast Cancer.

INTRODUCTION: Premenopausal women with hormone receptor positive (HR+) and human epidermal growth factor receptor-2-negative (HER2-) advanced breast cancer (aBC) often present with aggressive tumor types that lead to poor prognosis, high rates of recurrence, and mortality. Although clinical guidelines provide evidence-based recommendations for optimal treatment and monitoring, there is a dearth of information regarding treatment and monitoring patterns in clinical practice. In this study, we describe treatment and monitoring patterns among premenopausal women with HR+/HER2- aBC in real-world practice. METHODS: A large US claims database was used to describe treatment patterns for patients in first, second, and third lines of therapy. Treatment monitoring included complete blood count (CBC), liver function test (LFT), and electrocardiogram (EKG) monitoring, described for the first three lines of therapy, and separately for patients receiving endocrine monotherapy (ET) and chemotherapy. RESULTS: Among 3203 patients, chemotherapy was the most common treatment used in first-line (63.6%) and second-line therapy (66.9%). ET was used in 34.4, 30.1, and 73.6% of patients in first, second, and third lines of therapy, respectively. The two most common treatment sequences were a single line of ET (27.3%), and two consecutive lines of chemotherapy followed by a line of ET (19.3%). Patients receiving chemotherapy were monitored with CBC on average more than two times per month, and for LFT one to two times per month. Patients receiving ET were monitored with CBC and LFT on average once every 2-3 months. Overall, approximately 20% of patients were monitored with an EKG at some point during each line of therapy. CONCLUSION: A considerable proportion of premenopausal women with aBC received first- and second-line chemotherapy, which appears inconsistent with current clinical guidelines. The observed treatment heterogeneity points to a lack real-world consensus on the management of premenopausal women with HR+/HER2- aBC. FUNDING: Novartis Pharmaceuticals Corporation.

Burden of Infections Among Chronic Myeloid Leukemia Patients Receiving Dasatinib or Nilotinib: A Real-World Retrospective Healthcare Claims Study in the United States.

INTRODUCTION: Tyrosine kinase inhibitors (TKI) have been demonstrated to prolong survival in patients with chronic myeloid leukemia (CML). However, TKIs may be associated with an increased risk of infections. This study compared healthcare resource utilization (HRU) and costs among patients with CML receiving dasatinib or nilotinib, with a focus on infection-related economic outcomes. METHODS: Two large administrative databases were used to identify adult patients newly diagnosed with CML who initiated dasatinib or nilotinib as first- (1L) or second-line (2L) therapy and were classified into the following 1L (dasatinib 1L/nilotinib 1L cohorts) or 2L (dasatinib 2L/nilotinib 2L) cohorts based on the initiated 1L/2L TKI therapy. Infection-related HRU and healthcare costs were compared between cohorts, separately for 1L and 2L. RESULTS: Cohorts included 1156 patients in the dasatinib 1L and 677 patients in the nilotinib 1L cohorts, 322 patients in the dasatinib 2L, and 207 in the nilotinib 2L cohorts. In 1L and 2L, infection-related HRU was higher for dasatinib than nilotinib cohorts. Infection-related inpatient (IP) days constituted a larger proportion of all-cause IP days in the 1L/2L dasatinib than 1L/2L nilotinib cohorts (dasatinib 1L/2L: 53%/58%; nilotinib 1L/2L: 50%/46%). Compared to the nilotinib cohort, the dasatinib cohort had higher all-cause total costs per patient per year by US$17,901 in 1L and $28,625 in 2L. Of the total cost difference, infection-related were $6048 (34%) in 1L and $28,192 (99%) in 2L, largely driven by IP cost differences (1L/2L: 96%/98%). CONCLUSIONS: Dasatinib was associated with higher HRU and healthcare costs compared to nilotinib, particularly related to infections. FUNDING: Novartis Pharmaceutical Corporation.

Dosing Patterns and Economic Burden of Palbociclib Drug Wastage in HR+/HER2- Metastatic Breast Cancer.

INTRODUCTION: Targeted therapies have revolutionized the treatment of hormone receptor positive/human epidermal growth factor receptor 2 negative (HR+/HER2-) metastatic breast cancer (mBC). However, as for many oncology drugs, the dose of targeted therapies may need to be adjusted over time, leading to drug wastage when a dose modification is needed but the dose cannot be split or saved. This has been shown to be the case for palbociclib and has led to concerns among payers. This study described palbociclib dosing patterns and estimated the economic burden of the drug wastage associated with palbociclib dose modifications in postmenopausal women with HR+/HER2- mBC. METHODS: A large US claims database was used to identify postmenopausal women with HR+/HER2- mBC who received a palbociclib-based therapy during one of their first three lines of therapy for mBC between February 2015 (palbociclib approval) and December 2015. Dosing patterns (dosing modifications and sequences) were reported; a dose modification was defined as an increase/decrease of at least 25 mg daily compared to the preceding dose. Estimates of drug wastage costs were based on days with overlap in prescription fills for different palbociclib doses. RESULTS: A total of 473 postmenopausal palbociclib-treated women with HR+/HER2- mBC were included (first line 214; second line 157; third line 120). Over an average duration of line of therapy of approximately 4 months, dose modification was observed in 17.8%, 31.2%, and 35.0% of patients in first, second, and third line. Average overlap in prescription fills was 9.2, 9.9, and 5.4 days in first, second, and third line. This potential drug wastage resulted in an average cost of $4376, $4740, and $2592 per patient in first, second, and third line. CONCLUSIONS: This study showed that drug wastage due to palbociclib dose modification results in substantial costs. Treatment options with more flexible dosing may help reduce the costs of drug wastage. FUNDING: Novartis Pharmaceuticals Corporation.