Mortality among US veterans with a physician-documented diagnosis of pyruvate kinase deficiency.

Real-world studies of pyruvate kinase (PK) deficiency and estimates of mortality are lacking. This retrospective observational study aimed to identify patients with PK deficiency and compare their overall survival (OS) to that of a matched cohort without PK deficiency. Patients with ≥1 diagnosis code related to PK deficiency were selected from the US Veterans Health Administration (VHA) database (01/1995-07/2019); patients with a physician-documented diagnosis were included (PK deficiency cohort; index: date of first diagnosis code related to PK deficiency). Patients in the PK deficiency cohort were matched 1:5 to patients from the general VHA population (non-PK deficiency cohort; index: random visit date during match's index year). OS from index was compared between the two cohorts. Eighteen patients in the PK deficiency cohort were matched to 90 individuals in the non-PK deficiency cohort (both cohorts: mean age 57 years, 94% males; median follow-up 6.0 and 8.0 years, respectively). At follow-up, patients in the non-PK deficiency cohort had significantly longer OS than the PK deficiency cohort (median OS: 17.1 vs. 10.9 years; hazard ratio: 2.3; p = 0.0306). During their first-year post-index, 75% and 40% of the PK deficiency cohort had laboratory-confirmed anemia and iron overload, respectively. Among patients who died, cause of death was highly heterogeneous. These results highlight the increased risk of mortality and substantial clinical burden among patients with PK deficiency. While the intrinsic characteristics of the VHA database may limit the generalizability of the results, this is the first real-world study to characterize mortality in patients with PK deficiency.

Burden of illness for patients with cholangiocarcinoma in the United States: a retrospective claims analysis.

BACKGROUND: Advanced cholangiocarcinoma (CCA) is associated with considerable morbidity and mortality. Novel second-line treatments for advanced CCA underscore the need to understand treatment patterns and economic burden of illness in clinical practice. METHODS: This retrospective, claims-based study using Optum's de-identified Clinformatics® Data Mart Database [2007-2019] selected patients with CCA who experienced failure of a line of therapy containing either gemcitabine or fluorouracil. The index date was defined based on evidence of treatment failure: date of last administration of the gemcitabine- or fluorouracil-based regimen plus 28 days, or initiation date of the next-line systemic therapy. Treatment patterns, healthcare resource use (HRU), costs, and survival were assessed during the follow-up period (index until death or end of eligibility). RESULTS: A total of 1,298 patients met inclusion criteria and had a mean age of 69.1 years. There were 958 patients (73.8%) with intrahepatic and 275 patients (21.2%) with extrahepatic CCA. Average follow-up was 7.5 months. Almost 40% of patients did not receive another line of therapy after the index date. Among the 784 patients who received another line of therapy, 40.3% used fluorouracil-based therapy, 30.7% used gemcitabine-based therapy, and 29.3% used capecitabine-based therapy. Total mean per patient per month CCA-related healthcare costs were $7,743, with medical services ($6,685) a larger driver of monthly costs relative to treatment costs ($1,058). Median overall survival (OS) was 5.3 months among all patients. CONCLUSIONS: Many patients with advanced CCA do not initiate additional therapy after failure of gemcitabine or fluorouracil treatment, and there is considerable variation in treatments among those who do. This study highlights the high costs and unmet need for a standard of care in this patient population.

Higher Medication Adherence and Lower Opioid Use Among Individuals with Autoimmune Disease Enrolled in an Adalimumab Patient Support Program in the United States.

INTRODUCTION: Opioid use is prevalent among patients with autoimmune conditions, despite not being a recommended treatment. Tumor necrosis factor inhibitor (anti-TNF) therapy is an effective treatment for these autoimmune conditions, and patient support programs (PSPs) have been developed to help patients manage their prescribed treatments. This study was conducted to evaluate the impact of PSPs on anti-TNF adherence and opioid use using data on adalimumab (ADA), an anti-TNF. METHODS: The study used insurance claims data linked to ADA PSP data on patients who initiated ADA after 01/2015, were commercially insured, and had data coverage for 1 year before and after (i.e., during the follow-up period) ADA initiation. Patients with opioid use in the 3 months before ADA initiation were excluded. PSP patients enrolled in the PSP within 30 days of ADA initiation and had 2+ PSP nurse ambassador interactions; non-PSP patients had no PSP engagement. ADA adherence [proportion of days covered (PDC), persistence], opioid initiation, 2+ opioid fills, and opioid supply during follow-up were compared between cohorts using regression models that controlled for patient characteristics. RESULTS: Results were obtained for 1952 PSP and 728 non-PSP patients. PSP patients demonstrated better adherence to ADA than non-PSP patients, including higher PDC and persistence (all p < 0.001). PSP patients were 13% less likely to initiate opioids and 26% less likely to have at least 2 fills than non-PSP patients, and they had fewer days of opioid supply (all p < 0.01). CONCLUSIONS: This study supports the benefit of PSPs and suggests that the ADA PSP is associated with improved adherence and potentially lower opioid use.

The Economic Value of Improved Productivity from Treatment of Chronic Hepatitis C Virus Infection: A Retrospective Analysis of Earnings, Work Loss, and Health Insurance Data.

INTRODUCTION: Patients with chronic hepatitis C virus infection (HCV) may incur significant indirect costs due to health-related work loss. However, the impact of curative HCV therapy on work productivity is not well characterized. We estimated the economic value of improved productivity following HCV treatment. METHODS: Adults diagnosed with HCV infection (Optum Healthcare Solutions data; Q1 1999 to Q1 2017) were stratified into two cohorts: (1) treated cohort, patients who received HCV therapy and (2) untreated cohort, therapy-naïve patients. For the treated cohort, the index date was set at the end of the post-treatment monitoring period, assumed to be 6 months after the end of treatment for patients with cirrhosis or for those treated with interferon-based therapy, and 3 months after the end of treatment for patients without cirrhosis who received interferon-free therapy. For the untreated cohort, an index date was randomly selected post-HCV diagnosis. Time from the index date to the first work-loss event was assessed using time to event analyses. An economic modeling approach was used to monetize the improved productivity from reduced risk of work-loss event in the 4 years post-index. RESULTS: Patients in the treated cohort had a lower risk of experiencing a work-loss event compared to untreated patients [unadjusted and adjusted hazard ratios and 95% CI 0.72 (0.61-0.86), and 0.68 (0.55-0.85), respectively; p < 0.001 for both]. The mean cumulative added productivity value associated with HCV treatment was US$4511 (CI $2778-$6278) at 1 year post-index and $21,429 (CI $12,733-$30,199) at 4 years post-index. CONCLUSION: HCV treatment reduces the risk of work loss resulting in productivity gains for employers and employees. The monetary value associated with these productivity gains is substantial, and, after about 4 years, it is comparable to the wholesale acquisition cost of some direct-acting antiviral regimens in the United States. Employers may derive economic benefits from adopting HCV elimination strategies.

Economic Outcomes of Inflammatory Bowel Disease Patients Switching to a Second Anti-Tumor Necrosis Factor or Vedolizumab.

Background: Anti-tumor necrosis factor (TNF) therapies have been the mainstay of inflammatory bowel disease (IBD) treatment for nearly 2 decades. Therapies with novel mechanisms of action have been recently developed. This study compared healthcare resource utilization (HRU) and costs incurred while switching from an initial anti-TNF to another anti-TNF versus switching to vedolizumab. Methods: Adults with IBD who switched from initial anti-TNF to another anti-TNF or vedolizumab were identified from Truven MarketScan claims database (January 1, 2000-September 30, 2017). Patient characteristics were assessed during the 6-month period before the initiation date of the switched-to treatment (index date). Adjusted analyses of all-cause and disease-related HRU and costs during the 6-month period after the index date (study period) were performed. Anti-TNF and vedolizumab switchers with Crohn's disease (CD) and ulcerative colitis (UC) were separately compared. Results: A total of 502 vedolizumab, 1708 adalimumab, 755 infliximab, and 703 other switchers with CD and 461, 428, 311, and 148 with UC, respectively, were identified. Patient demographics were similar across cohorts. Total all-cause costs were significantly higher for vedolizumab than adalimumab, infliximab, and certolizumab switchers in the CD cohort and adalimumab and infliximab in the UC cohort. In both cohorts, adalimumab and other switchers had fewer all-cause and IBD-related outpatient visits than vedolizumab switchers. Conclusions: CD/UC patients who switched to vedolizumab from initial anti-TNF had higher total and treatment costs than patients who switched to another anti-TNF, except for UC patients who switched to golimumab. Prospective studies should be conducted to confirm these findings.

Economic Burden of Illness Among Commercially Insured Patients with Systemic Sclerosis with Interstitial Lung Disease in the USA: A Claims Data Analysis.

INTRODUCTION: Interstitial lung disease (ILD) is a common manifestation of scleroderma/systemic sclerosis (SSc). However, the direct and indirect economic burdens of SSc-ILD remain unclear. This study assessed and compared healthcare resource utilization (HRU), direct healthcare costs, work loss, and indirect costs between patients with SSc-ILD and matched controls with neither SSc nor ILD in the USA. METHODS: Data were obtained from a large US commercial claims database (2005-2015). Patients (at least 18 years old) had at least one SSc diagnosis in the inpatient (IP) or emergency room (ER) setting or at least two SSc diagnoses in another setting, and at least one diagnosis of ILD in the IP or ER setting or at least two diagnoses of ILD in another setting. Controls with neither SSc nor ILD were matched 5:1 to patients with SSc-ILD. Comparisons were conducted using Wilcoxon signed-rank and McNemar's tests and adjusted odds ratios (ORs) and incidence rate ratios (IRRs). RESULTS: A total of 479 SSc-ILD patients and 2395 matched controls were included (52 SSc-ILD patients and 260 matched controls for work loss and indirect cost analyses). Patients with SSc-ILD had significantly higher HRU and costs, IP admissions (adjusted IRR = 5.6), IP hospitalization days (adjusted IRR = 12.0), ER visits (adjusted IRR = 2.8), OP visits (adjusted IRR = 3.1), and days of work loss (adjusted IRR = 4.5). The adjusted difference in annual direct healthcare costs was $28,632 (SSc-ILD, $33,195; controls, $4562) and that in indirect costs was $4735 (SSc-ILD, $5640; controls, $906) (all p < 0.0001). CONCLUSION: SSc-ILD patients had significantly higher HRU, work loss, and direct and indirect costs compared to matched controls with neither SSc nor ILD. FUNDING: Boehringer Ingelheim Pharmaceuticals, Inc.

Economic Burden among Commercially Insured Patients with Systemic Sclerosis in the United States.

OBJECTIVE: To quantify healthcare resource utilization (HRU), work loss, and annual direct and indirect healthcare costs among patients with systemic sclerosis (SSc) compared to matched controls in the United States. METHODS: Data were obtained from a large US commercial claims database. Patients were ≥ 18 years old at the index date (first SSc diagnosis) and had ≥ 1 SSc diagnosis in the inpatient (IP) or emergency room (ER) setting, or ≥ 2 SSc diagnoses on 2 different dates in the outpatient (OP) setting between January 1, 2005, and March 31, 2015; continuous enrollment was required during the followup period (12 months after the index date). Individuals with no SSc diagnoses were matched 1:1 to patients with SSc. Wilcoxon signed-rank and McNemar tests were used for comparisons and regressions with generalized estimating equations for adjusted OR (aOR) and incidence rate ratios (IRR) between 2 cohorts. RESULTS: There were 2192 pairs of patients with SSc and matched controls included (mean age 57.6 yrs; 84.3% female); of these, 233 were eligible for work loss/indirect cost analyses. Compared to matched controls, patients with SSc had significantly higher HRU and costs during the 1-year followup period, IP admissions (adjusted IRR = 2.4), IP hospitalization days (adjusted IRR = 3.1), ER visits (adjusted IRR = 2.0), OP visits (adjusted IRR = 2.3), and days of work loss (adjusted IRR = 2.6). The adjusted difference in annual direct and indirect costs was US$12,820 and $3103, respectively (all p < 0.0001). CONCLUSION: Patients with SSc had a high direct and indirect economic burden postdiagnosis.

Clinical and Economic Burden of Patients with Chronic Hepatitis C with Versus Without Antiviral Treatment in Japan: An Observational Cohort Study Using Hospital Claims Data.

INTRODUCTION: Japan has one of the highest prevalence rate of chronic hepatitis C (CHC) in the industrialized world. However, the burden of CHC treatment is poorly understood. Thus, the healthcare resource utilization and costs of treated versus untreated patients, and patients with early versus delayed treatment initiation, were assessed in Japan. METHODS: Adult patients with ≥ 2 CHC diagnoses were identified from the Medical Data Vision hospital claims database (1 April 2008-31 May 2016). The presence or absence of antiviral treatment claims was used to form the treated and untreated cohorts, respectively. Among treated patients, the presence of a cirrhosis-related diagnosis was used as an indicator of delayed treatment. The index date was defined as the date of the first antiviral claim for treated patients and randomized to any date with a medical visit for untreated patients. Annualized total healthcare costs and costs associated with hepatic manifestations (HMs) or extrahepatic manifestations (EHMs) were evaluated from the index date to the last observed medical visit. RESULTS: Of 100,125 patients with CHC, 12,984 were treated (early: 8104, delayed: 4880) and 87,141 were untreated. After adjusting for covariates, untreated patients had ¥613,034 ($5456 USD; ¥1 = $0.0089) higher annual medical costs compared with treated patients (P < 0.001), a difference driven by higher inpatient costs. Between 65% (treated patients) and 70% (untreated patients) of medical costs were EHM-related and between 14% (untreated patients) and 15% (treated patients) were HM-related. Patients in the delayed treatment cohort had ¥114,347 ($1018) higher annual medical costs (P < 0.001) versus those in the early treatment cohort. About 95% of these costs were EHM-related, and 64% were HM-related. CONCLUSION: Withholding or delaying antiviral treatment initiation for Japanese patients with CHC increases the clinical and economic burden associated with HMs and EHMs. FUNDING: AbbVie.

Short-term costs associated with non-medical switching in autoimmune conditions.

OBJECTIVES: To estimate short-term costs associated with non-medical switch (NMS) from originator biologics to biosimilars among stable patients with autoimmune conditions in rheumatology, gastroenterology, and dermatology from a US provider's and third-party payer's perspective. METHODS: An economic model was constructed to estimate switching costs related to physician time and healthcare resource utilisation (HRU) at the initial NMS visit and over 3 months. The proportion of patients with relevant conditions treated with originators and expected NMS rate, physician time, HRU, and payer reimbursement were derived from a physician survey. Switching costs were estimated for a practice of 1,000 patients with relevant conditions by therapeutic area and for an insurance plan with 1 million individuals by therapeutic area and all areas combined. Switching cost drivers were assessed with one-way sensitivity analyses. RESULTS: Physicians expected extra 6 minutes for the NMS visit and 22 minutes over 3 months; NMS rates of 14.4%, 15.5%, and 17.7%; and 11.3%, 16.2%, and 33.2% of time not reimbursed for gastroenterology, rheumatology, and dermatology, respectively. The total switching costs for payer's were $771,460 (for n = 3,609 patients with an NMS rate of 16.6%), mostly due to follow-up visits and additional laboratory tests/procedures. In sensitivity analyses, the NMS rate was the main cost driver. Increasing the NMS rate to 25% and 50% increased payer's total switching costs to $1.19 and $2.39 million, respectively. CONCLUSIONS: Originator-to-biosimilar NMS in stable patients with autoimmune conditions could result in considerable switching costs for both providers and payers.

The long-term direct and indirect economic burden among Parkinson's disease caregivers in the United States.

BACKGROUND: Parkinson's disease is a progressive, disabling neurodegenerative disorder associated with significant economic burden for patients and caregivers. The objective of this study was to compare the direct and indirect economic burden of Parkinson's patients' caregivers with demographically matched controls in the United States, in the 5 years after first diagnosis of Parkinson's disease. METHODS: Policyholders (18-64 years old) linked to a Parkinson's disease patient (≥2 diagnoses of Parkinson's disease; first diagnosis is the index date) from January 1, 1998 to March 31, 2014, were selected from a private-insurer claims database and categorized as Parkinson's caregivers. Eligible Parkinson's caregivers were matched 1:5 to policyholders with a non-Parkinson's dependent (controls). Multivariable regression adjusted for baseline characteristics estimated direct costs (all-cause insurer cost [medical and prescription] and comorbidity-related medical costs; patient out-of-pocket costs) and indirect costs (disability and medically related absenteeism costs). Income progression was also compared between cohorts. RESULTS: A total of 1211 eligible Parkinson's caregivers (mean age, 56 years; 54% female) were matched to 6055 controls. In adjusted analyses, Parkinson's caregivers incurred significantly higher year 1 total all-cause insurer costs ($8999 vs $7117) and medical costs ($7081 vs $5568) (both P < 0.01) and higher prescription costs (range for years 1-5, $2506-2573 vs $1405-$1687) and total out-of-pocket costs ($1259-1585 vs $902-$1192) in years 1-5 (all P < 0.01). Parkinson's caregivers had significantly higher adjusted indirect costs in years 1-3 (range for years 1-3, $2054-$2464 vs $1681-$1857; all P < 0.05) and higher cumulative income loss over 5 years ($5967 vs $2634 by year 5; P for interaction = 0.03). CONCLUSIONS: Parkinson's caregivers exhibited higher direct and indirect costs and greater income loss compared with matched controls. © 2018 International Parkinson and Movement Disorder Society © 2018 The Authors. Movement Disorders published by Wiley Periodicals, Inc. on behalf of International Parkinson and Movement Disorder Society.

Incidence of catheter-related complications among Japanese patients with central venous catheters as well as patients with short bowel syndrome.

PURPOSE: Short bowel syndrome (SBS) is a serious rare disorder that is usually managed with parenteral nutrition. Central venous catheter (CVC)-related complications are known to occur, but their incidence rates (IRs) in Japan are unknown. The aim of this study was to estimate the incidence of complications in Japanese patients with CVCs, including patients with SBS using CVCs. PATIENTS AND METHODS: A retrospective cohort study in 64,817 patients with CVCs, including 81 patients with SBS, between April 2008 and October 2016 using a claims database in Japan was conducted. IRs of complications were calculated as total events divided by total catheter-days among all patients with CVCs and among patients with SBS. RESULTS: The majority (>90%) of patients in the CVC and SBS cohorts were ≥18 years old. Overall, IRs of any type of complication were numerically higher in patients with SBS compared with all patients with CVCs (2.68 vs 1.95 cases per 1,000 catheter-days, respectively). Among patients with SBS, septicemia, infection, and inflammatory reaction were the only complications observed. The complications with the highest incidence were catheter-related bloodstream infections (CRBSIs) in both the overall CVC cohort and the SBS cohort with IRs of 1.35 and 2.68 cases per 1,000 catheter-days, respectively. The IR of any CVC-related complication was numerically higher in patients with SBS with cancer vs without cancer (3.44 vs 1.86 cases per 1,000 catheter-days, respectively). CONCLUSION: Our study quantifies the incidence of complications in patients with CVCs, including those with SBS, in Japan.

Economic Burden of Patients with Inadequate Response to Targeted Immunomodulators for Rheumatoid Arthritis.

BACKGROUND: Targeted immunomodulators (TIMs), including biologic disease-modifying antirheumatic drugs (DMARDs) and JAK/STAT inhibitors, are effective therapies for rheumatoid arthritis (RA), but some patients fail to respond or lose response over time. This study estimated the real-world prevalence of RA patients with inadequate responses to an initial TIM (nonresponders) in the United States and assessed their direct and indirect economic burden compared with treatment responders. METHODS: Administrative claims data (January 1999-March 2014) from a large private-insurer database were used, which included work-loss data from a subset of reporting companies. Eligible patients (classified as responders and nonresponders) had ≥ 1 claim for a TIM approved for the treatment of RA and ≥ 2 RA diagnoses in the claims history, with continuous pharmaceutical and medical benefit eligibility for 6 months before (baseline) and 12 months after (study period) the date of the first TIM claim (index date). All-cause and RA-related health care resource use (HCRU) and costs, work loss, and indirect costs during the study period were compared for responders versus nonresponders. Multivariable regression was used to adjust for baseline covariates. Sensitivity analyses of HCRU and direct costs were conducted for patients with index dates before and after 2008 to account for different approval dates of TIMs. RESULTS: Of 7,540 eligible patients with RA, 2,527 (34%) were classified as responders, and 5,013 (66%) were classified as nonresponders; 407 and 723 had work-loss data, respectively. After adjusting for baseline covariates, nonresponders had significantly higher HCRU, including inpatient admissions (incidence rate ratio [IRR] = 1.94), outpatient visits (IRR = 1.19), emergency department visits (IRR = 1.53), and number of prescription fills (IRR = 1.09; all, P < 0.001). Nonresponders also had significantly higher adjusted all-cause ($12,868 vs. $9,621, respectively) and RA-related ($5,740 vs. $4,495; both, P < 0.001) medical costs compared with responders. In addition, nonresponders reported significantly more days of work lost compared with responders (22.1 vs. 16.7 days, respectively; IRR = 1.21; P = 0.007) and higher indirect costs ($3,548 vs. $2,890; P = 0.002). Sensitivity analyses of HCRU and direct costs by index date (before and after 2008) were consistent with the full sample. CONCLUSIONS: A large portion of patients with RA had inadequate responses to their initial TIM therapy with significantly higher economic burden, including higher HCRU, medical costs, and indirect costs due to work loss, compared with TIM therapy responders. DISCLOSURES: Funding for this research was provided by AbbVie, which was involved in all stages of the study research and manuscript preparation. Tundia and Fuldeore are employed by AbbVie. Song and Macaulay are employed by Analysis Group, which received grants from AbbVie to conduct this study. Strand reports grants and personal fees from AbbVie, Amgen, AstraZeneca, Bayer, Boehringer Ingelheim, Celltrion, Corrona, Crescendo, Genentech/Roche, GSK, Janssen, Lilly, Novartis, Pfizer, Regeneron, Samsung, Sandoz, Sanofi, and UCB outside the submitted work. Study concept and design were contributed by Tundia, Song, and Macaulay, along with other authors. Data analyses were designed and conducted by Song and Macaulay. All authors contributed to data interpretation. Writing of the manuscript was led by Tundia, Song, and Macaulay, with revisions by all authors. A synopsis of the current research was presented at the American College of Rheumatology/Association of Rheumatology Health Professionals meeting, which took place in Washington, DC, during November 11-16, 2016.

Healthcare resource utilization and costs by age and joint location among osteoarthritis patients in a privately insured population.

AIMS: To compare healthcare resource utilization and costs between patients aged 18-64 years with osteoarthritis (OA) and matched controls without OA in a privately insured population. METHODS: Patients with OA were selected from de-identified US-based employer claims (Q1:1999-Q3:2011). The index date was defined as the first OA diagnosis indicated by ICD-9-CM codes. One year before and after the index date were defined as the baseline and study periods, respectively. A second OA diagnosis during the study period was also required. Patients with OA were matched one-to-one on age, gender, index date, and minimum length of follow-up to controls without OA. Baseline characteristics and study period resource utilization and costs (2016 USD) were compared between cohorts. RESULTS: This study identified 199,539 patients with OA (knee: 87,271, hip: 19,953, hand: 15,670, spine: 12,496). The average age was 54 years, and 58% were female. OA patients had higher healthcare resource utilization than matched controls in inpatient, emergency room, and outpatient settings (p < .001 for all). Further, patients with OA had 4-times the excess total medical costs of their matched controls ($14,521 vs $3,629; p < .001). Patients with hip OA had the highest medical costs among all joint locations. Outpatient and pharmacy costs were similar among patients with knee, hip, and hand OA, but higher in patients with spine OA. In sub-group analyses, older patients (45-64 years old) had higher costs. LIMITATIONS: This sample, obtained using claims data, only includes patients who were actively seeking care for OA and were likely symptomatic. Asymptomatic patients would likely not be captured in this analysis. CONCLUSIONS: Patients with OA incur greater healthcare resource utilization and costs than patients without OA, with substantial variation by joint location.

Inpatient Admissions and Costs Associated with Persistent Use of Dalfampridine Extended-Release in Multiple Sclerosis: A Claims Database Analysis.

BACKGROUND: While the clinical benefits of dalfampridine extended-release (D-ER) have been established in patients with multiple sclerosis (MS) through multiple clinical trials, there is limited real-world data on D-ER use, in particular the persistent use of D-ER, and associated acute care resource utilization and costs. OBJECTIVE: To examine the real-world association of D-ER use and inpatient admissions and costs among patients with MS. METHODS: This study was a retrospective observational claims analysis of the MarketScan database (April 2009-March 2014). Eligible patients consisted of adult enrollees aged 18-64 years who had (a) 12 months of continuous private plan enrollment preceding (baseline) and following (follow-up) the first D-ER claim; (b) ≥ 2 MS diagnosis codes with ≥ 1 during the baseline period; (c) ≥ 2 consecutive D-ER claims; and (d) no alternate gait-impairing etiologies during the baseline and follow-up periods. Patients were separated into 2 D-ER cohorts in the main analysis: persistent (≥ 360 days of D-ER supply) and nonpersistent (< 360 days of supply) users. Sensitivity analyses were conducted, examining additional breakdowns of days of supply within the nonpersistent cohort. Inpatient admissions (all-cause and MS-related) and health care expenditures were calculated and compared between the cohorts during follow-up using Wilcoxon rank-sum and chi-square tests. Regression models were conducted, controlling for age, sex, MS relapses, comorbidities, disease-modifying therapy use, and other baseline factors, including inpatient admissions and costs. RESULTS: Of 1,598 eligible patients, 719 (45.0%) were persistent D-ER users, and 879 (55.0%) were nonpersistent D-ER users. The 2 cohorts had similar demographic and clinical characteristics, with mean (SD) ages of 51.0 (8.4) and 50.6 (8.6) years and were 71.3% and 66.6% female, respectively. Compared with nonpersistent D-ER use, persistent D-ER use was associated with lower odds of all-cause inpatient admissions (OR = 0.58, P = 0.010) and MS-related inpatient admissions (OR = 0.50, P = 0.004). Persistent use was also associated with lower inpatient expenditures for all-cause admissions ($669 vs. $1,515, P = 0.002) and MS-related admissions ($388 vs. $891, P = 0.008). CONCLUSIONS: Persistent D-ER use was associated with significantly lower rates of all-cause and MS-related inpatient admissions and costs. DISCLOSURES: Funding for this research and medical writing assistance was provided by Acorda Therapeutics. The study sponsor was involved in all stages of the study research and manuscript preparation. Guo and Niyazov were employees of Acorda Therapeutics at the time of this study and may own stock/stock options. Wu, Macaulay, Terasawa, and Schmerold are employees of Analysis Group, which received consultancy fees from Acorda Therapeutics for this project. Krieger was a consultant for Acorda Therapeutics for this project and has the following additional financial interests to report: consulting/advisory board work with Bayer, Biogen, EMD Serono, Novartis, Genentech, Genzyme, and Teva. Study concept and design were contributed by Guo, Niyazov, Macaulay, and Wu. Macaulay, Terasawa, Schmerold, and Wu helped prepare the data, and data interpretation was performed by Krieger, Guo, Niyazov, and Macaulay, along with Terasawa and Wu. The manuscript was written by Terasawa and Schmerold, along with Macaulay, and revised by all the authors. A portion of the current research was presented in poster format at the 2106 American Academy of Neurology Annual Meeting, which took place in Vancouver, BC, Canada, on April 15-21, 2016.

Prevalence of Noninfectious Uveitis in the United States: A Claims-Based Analysis.

IMPORTANCE: Noninfectious uveitis (NIU) is a collection of intraocular inflammatory disorders that may be associated with significant visual impairment. To our knowledge, few studies have investigated NIU prevalence overall or stratified by inflammation location, severity, presence of systemic conditions, age, or sex. OBJECTIVE: To estimate NIU prevalence using a large, retrospective, administrative claims database. DESIGN, SETTING, AND PARTICIPANTS: This analysis used the OptumHealth Reporting and Insights database to estimate 2012 NIU prevalence. Analysis was conducted in September 2016. The large administrative insurance claims database includes 14 million privately insured individuals in 69 self-insured companies spanning diverse industries. Included in the study were patients with NIU with 2 or more uveitis diagnoses on separate days in 2012 and continuous enrollment in a health plan for all of 2012 and categorized by inflammation site. MAIN OUTCOMES AND MEASURES: We estimated overall NIU prevalence by inflammation site, severity, sex, and age. Patients with anterior NIU were categorized by the presence of systemic conditions. RESULTS: Of the approximately 4 million eligible adult patients, approximately 2.1 million were women, and of the 932 260 children, 475 481 were boys. The adult prevalence of NIU was 121 cases per 100 000 persons (95% CI, 117.5-124.3). The pediatric NIU prevalence was 29 cases per 100 000 (95% CI, 26.1-33.2). Anterior NIU accounted for 81% (3904 cases) of adult NIU cases (98 per 100 000; 95% CI, 94.7-100.9) and 75% (207 cases) of pediatric NIU cases (22 per 100 000; 95% CI, 19.3-25.4). The prevalences of noninfectious intermediate, posterior, and panuveitis were, for adults, 1 (95% CI, 0.8-1.5), 10 (95% CI, 9.4-11.5), and 12 (95% CI, 10.6-12.7) per 100 000, respectively, and for pediatric patients, 0 (95% CI, 0.1-1.1), 3 (95% CI, 1.8-4.1), and 4 (95% CI, 2.9-5.6) per 100 000, respectively. The prevalence of NIU increased with age and was higher among adult females than males. Application of these estimates to the US population suggests that NIU affected approximately 298 801 American adults (95% CI, 290 512-307 324) and 21 879 children (95% CI, 19 360-24 626) in 2015. CONCLUSIONS AND RELEVANCE: The estimated prevalence of NIU was 121 cases per 100 000 for adults (95% CI, 117.5-124.3) and 29 per 100 000 for children (95% CI, 26.1-33.2). Prevalence was estimated using administrative claims from a commercially insured population, which may have a different prevalence than other segments of the US population. A better understanding of the prevalence of NIU will help to determine the number of patients affected.

Direct Costs in Patients with Celiac Disease in the USA: A Retrospective Claims Analysis.

BACKGROUND: Celiac disease (CeD) is an autoimmune disease triggered by gluten ingestion. AIM: We assessed total direct costs burden associated with CeD in patients with CeD versus patients without CeD using administrative claims data. METHODS: Patients with CeD (cases) with ≥1 occurrences of CeD diagnosis were selected at a randomly chosen date (index date) from the OptumHealth Reporting and Insights database from 01/01/1998 through 03/31/2013. Cases were continuously enrolled throughout baseline (1 year before index date) and study (1 year after index date) periods. Cases were categorized as full remission and partial remission and matched 1:1 based on age, sex, region, index date, company, and employment status. Total all-cause and CeD-related costs were calculated. RESULTS: A total of 12,187 cases were matched with an equal number of controls. Mean total all-cause costs were $12,217 in cases versus $4935 in controls (P < 0.0001). In full remission (N = 10,181 [83.5 %]) and partial remission (N = 2006 [16.5 %]) cases, mean total all-cause direct costs (cases versus controls) were $11,038 versus $4962 and $18,206 versus $4796, respectively. All-cause medical costs ($9839 for all cases, $8723 for full remission cases, $15,499 for partial remission cases) accounted for the majority of all-cause total costs and included outpatient costs ($6675; $6456; and $7785, respectively) and hospitalizations ($2776; $1963; and $6906, respectively). CeD-related medical costs were 13 and 27 % of all-cause medical costs for all cases and partial remission cases, respectively. CONCLUSIONS: Patients with CeD and partial remission of CeD incurred significantly higher (2.5 and 3.8 times) total all-cause costs compared with matched controls.

Economic impact of expanded use of biologic therapy for the treatment of rheumatoid arthritis and Crohn's disease in Argentina, Brazil, Colombia, and Mexico.

OBJECTIVES: To estimate economic impact resulting from increased biologics use for treatment of rheumatoid arthritis (RA) and Crohn's disease (CD) in Argentina, Brazil, Colombia, and Mexico. METHODS: The influence of increasing biologics use for treatment of RA during 2012-2022 and for treatment of CD during 2013-2023 was modeled from a societal perspective. The economic model incorporated current and projected medical, indirect, and drug costs and epidemiologic and economic factors. Costs associated with expanded biologics use for RA were compared with non-expanded use in Argentina, Brazil, Colombia, and Mexico. A similar analysis was conducted for CD in Brazil, Colombia, and Mexico. RESULTS: Accounting for additional costs of biologics and medical and indirect cost offsets, the model predicts that expanded use of biologics for patients with RA from 2012 to 2022 will result in cumulative net cost savings of ARS$2.351 billion in Argentina, R$9.004 billion in Brazil, COP$728.577 billion in Colombia, and MXN$18.02 billion in Mexico; expanded use of biologics for patients with CD from 2013 to 2023 will result in cumulative net cost savings for patients with CD of R$0.082 billion in Brazil, COP$502.74 billion in Colombia, and MXN$1.80 billion in Mexico. Indirect cost offsets associated with expanded biologics use were a key driver in reducing annual per-patient net costs for RA and CD. LIMITATIONS: Future economic projections are limited by the potential variance between projected and actual future values of biologic prices, wages, medical costs, and gross national product for each country. CONCLUSIONS: Increasing biologics use to treat RA and CD may limit cost growth over time by reducing medical and indirect costs. These findings may inform policy decisions regarding biologics use in Argentina, Brazil, Colombia, and Mexico.

Direct and indirect resource use, healthcare costs and work force absence in patients with non-infectious intermediate, posterior or panuveitis.

PURPOSE: To ascertain resource use, costs and risk of workforce absence in non-infectious uveitis cases versus matched controls. METHODS: In a retrospective claims analysis of employees in the United States, prevalent (N = 705) and incident (N = 776) cases 18-64 years old with ≥2 diagnoses of non-infectious intermediate, posterior or panuveitis were matched 1:1 to controls without uveitis. Persistent prevalent cases (treated for ≥90 days, N = 112) also were analysed. Outcomes were annual direct resource use and costs associated with inpatient stays; emergency department, outpatient and ophthalmologist/optometrist visits; and prescription drugs. Indirect resource use and costs associated with work loss from disability and medically related absenteeism also were compared. Multivariate regression assessed cost differences between cases and controls. RESULTS: Cases had significantly (p < 0.05) more medical resource use versus controls including 0.4 versus 0.2 emergency visits and 16.5 versus 7.6 outpatient/other visits. Cases used more prescription drugs (7.8 versus 4.1) and had more disability days (10.3 versus 4.6), medically related absenteeism days (8.5 versus 3.8), and work loss days (18.7 versus 8.4) than controls (all p < 0.05). Total direct ($12 940 versus $3730) and indirect ($3144 versus $1378) costs were higher in cases than controls (all p < 0.05). Results for persistent cases suggested greater utilization and associated cost and work loss burden. Compared with controls, cases had significantly greater risks of workforce absence, leave of absence and long-term disability (all p < 0.05). CONCLUSION: Non-infectious intermediate, posterior or panuveitis, particularly persistent disease, is associated with substantial medical and work loss costs suggesting an unmet need for more effective treatments.

Comparison of real-world outcomes of adalimumab and infliximab for patients with ulcerative colitis in the United States.

OBJECTIVE: We compared the real-world effectiveness of initiating adalimumab and infliximab among patients in the US who were naïve to tumor necrosis factor (TNF) inhibitors. METHODS: A retrospective chart review was conducted to evaluate the real-world effectiveness among adults with ulcerative colitis (UC) initiating adalimumab or infliximab. Charts of patients with UC were abstracted by treating physicians (randomly selected from a nationally representative panel) in April 2014. Patient eligibility criteria included: adalimumab or infliximab initiation on/after 1 October 2012; no prior anti-TNF therapy, history of Crohn's disease, or colectomy; and ≥6 months of follow-up. Information on clinical outcomes (partial Mayo score, remission rate, physician global assessment (PGA), stool frequency, and rectal bleeding) and treatment patterns (dose escalations, discontinuations, switches, and treatment augmentations) were retrospectively reported by treating physicians. Kaplan-Meier curves and multivariate Cox proportional hazards regression models were used to assess the time to clinical outcomes and treatment changes for each therapy. RESULTS: Overall, 170 physicians participated, contributing data on 380 and 424 patients who initiated adalimumab and infliximab, respectively. Baseline clinical characteristics were similar between groups. Both adalimumab- and infliximab-treated patients showed substantial improvements from baseline to follow-up in effectiveness measures; results of these measures were similar between the adalimumab and infliximab cohorts. Time to remission (p = 0.5241), no rectal bleeding (p = 0.7648), normal stool count (p = 0.9941), and normal PGA (p = 0.7697) showed no significant differences between therapies in unadjusted and adjusted comparisons. Unadjusted and adjusted time to event analysis of discontinuation (p = 0.7151), dose escalation (p = 0.6310), treatment augmentation (p = 0.1209), and switching (p = 0.7975) showed no significant differences between the two cohorts. LIMITATIONS: Retrospective, observational design. CONCLUSIONS: Adalimumab and infliximab were similarly effective in the treatment of moderate-to-severe UC in the real-world clinical setting.

Impact of a Telehealth and Care Management Program on All-Cause Mortality and Healthcare Utilization in Patients with Heart Failure.

BACKGROUND: Telehealth has the potential to improve chronic disease management and outcomes, but data regarding direct benefit of telehealth in patients with heart failure (HF) have been mixed. The objective of this study was to determine whether the Health Buddy Program (HBP) (Bosch Healthcare, Palo Alto, CA), a content-driven telehealth system coupled with care management, is associated with improved outcomes in Medicare beneficiaries with HF. MATERIALS AND METHODS: This was a retrospective cohort study of 623 Medicare beneficiaries with HF offered HBP enrollment compared with a propensity score-matched control group of Medicare beneficiaries with HF from the Medicare 5% sample. Associations between availability of the HBP and all-cause mortality, hospitalization, hospital days, and emergency department visits were evaluated. RESULTS: Beneficiaries offered enrollment in the HBP had 24.9% lower risk-adjusted all-cause mortality over 3 years of follow-up (hazard ratio [HR] = 0.75; 95% confidence interval [CI], 0.63-0.89; p = 0.001). Patients who used the HBP at least once (36.9%) had 57.2% lower mortality compared with matched controls (HR = 0.43; 95% CI, 0.31-0.60; p < 0.001), whereas patients who did not use the HBP had no significant difference in survival (HR = 0.96; 95% CI, 0.78-1.19; p = 0.69). Patients offered the HBP also had fewer hospital admissions following enrollment (Δ = -0.05 admissions/quarter; p = 0.011), which was primarily observed in patients who used the HBP at least once (Δ = -0.10 admissions/quarter; p < 0.001). CONCLUSIONS: The HBP, a content-driven telehealth system coupled with care management, was associated with significantly better survival and reduced hospitalization in Medicare beneficiaries with HF. Prospective study is warranted to determine the mechanism of this association and opportunities for optimization.