The Relative Value of Anti-Obesity Medications Compared to Similar Therapies.

Purpose: To demonstrate a need for improved health insurance coverage for anti-obesity medications (AOMs) by comparing clinical and economic benefits of obesity treatments to covered medications for selected therapeutic areas. Methods: Using a grey literature search, we identified and prioritized therapeutic areas and treatment analogues for comparison to obesity. A targeted literature review identified clinical and economic outcomes research across the therapeutic area analogues. Associated comorbidities, clinical evidence, indirect costs (ie, absenteeism and productivity loss), and direct medical costs were evaluated to determine the relative value of treating obesity. Results: Four therapeutic areas/treatment analogues were selected for comparison to obesity: smoking cessation (varenicline), daytime sleepiness (modafinil), migraines (erenumab), and fibromyalgia (pregabalin). Obesity was associated with 17 comorbidities, more than migraine (9), smoking (8), daytime sleepiness (5), and fibromyalgia (2). Economic burden was greatest for obesity, followed by smoking, with yearly indirect and direct medical costs totaling $676 and $345 billion, respectively. AOMs resulted in cost savings of $2586 in direct medical costs per patient per year (PPPY), greater than that for varenicline at $930 PPPY, modafinil at $1045 PPPY, and erenumab at $468 PPPY; pregabalin utilization increased costs by $924 PPPY. AOMs were covered by 10-16% of United States health insurance plans, compared to 45-59% for the four comparators. Conclusion: Compared to four therapeutic analogues, obesity represented the highest economic burden and was associated with more comorbidities. AOMs provide greater cost savings compared to selected analogues. However, AOMs have limited formulary coverage. Improved coverage of AOMs may increase access to these treatments and may help address the clinical and economic burden associated with obesity and its comorbidities.

Penile surgery for patients with Peyronie's disease initially treated with collagenase clostridium histolyticum or surgery: a claims database analysis.

Collagenase clostridium histolyticum (CCH) is an injectable therapy targeting collagen present in penile plaques in Peyronie's disease (PD). Data comparing CCH to penile surgery are limited, and long-term therapeutic outcomes are unknown. This retrospective analysis used a US claims database (January 2014-June 2017) to determine the percentage of men with subsequent penile surgery among those who initially received CCH (n = 1227) or surgery (index treatment; n = 620) for PD. Eligible patients were aged ≥18 years with continuous enrollment ≥6 months before and ≥12 months after index treatment date. During 12 months of post-index treatment follow-up, fewer patients with PD initially treated with CCH (4.6% [56/1227]) had subsequent penile surgery versus those initially treated with penile surgery (10.3% [64/620]; p < 0.0001). Mean ± SD time to first subsequent surgery after initial PD treatment was longer in the CCH versus surgery cohort (7.7 ± 3.0 vs 1.7 ± 3.2 months). The likelihood of subsequent surgery varied by initial surgery type: 18.2% after plaque incision or excision with grafting; 11.6% after penile implant; and 8.2% after tunical plication. Patients with PD who received CCH first were less likely to undergo subsequent surgery compared with those who received surgery first within a 12-month post-treatment follow-up.

Development of an Assessment Tool for Completion by Patients with Overweight or Obesity.

INTRODUCTION: Discussions of weight-management strategies between patients and healthcare providers can yield positive outcomes for people with overweight or obesity. Nonetheless, people with overweight or obesity encounter communication challenges and other barriers to pursuing effective weight-management strategies with their healthcare providers. The aim of this study was to develop a new self-completed assessment tool to initiate and facilitate conversations related to weight management between patients and healthcare providers. METHODS: Developing the assessment tool involved a series of steps and draft versions of the tool, based on feedback from key opinion leaders in the field of obesity (N = 4) and input from people with overweight or obesity (N = 18). Three iterative rounds of qualitative interviews were conducted in the USA. A targeted review of prior qualitative research was conducted to identify common and important impacts of obesity on patients' functioning. Standard qualitative analytical methods were used to identify concepts of importance in a concept elicitation exercise during the interviews and were evaluated for potential inclusion in the tool. Potential problems with the tool were flagged during cognitive debriefing of the draft tool. RESULTS: During 18 individual interviews, participants referenced the impact of their weight on their lives, including health and comorbidities, physical function, emotional/mental functioning, social life, and physical appearance. Over the course of the tool's development, 24 common and important impacts of obesity on patients' functioning were reduced to a final set of eight concepts in the final tool that were deemed important and relevant to both patients and key opinion leaders. CONCLUSIONS: The assessment tool is a five-item, self-completed measure expected to foster patient self-advocacy for individuals with overweight or obesity by giving them an opportunity to define their weight-management goals and discuss these, along with various medical interventions, with a healthcare provider.

Cost-effectiveness analysis of semaglutide 2.4 mg for the treatment of adult patients with overweight and obesity in the United States.

BACKGROUND: The rising prevalence and associated public health burden of obesity has led to advancements in pharmaceuticals for weight management. Semaglutide 2.4 mg, an anti-obesity medication (AOM) recently approved by the US Food and Drug Administration, has demonstrated clinically relevant weight loss in its phase 3 clinical trials. Economic evaluation comparing semaglutide 2.4 mg with other available weight management therapies is essential to inform payers for decision-making. OBJECTIVES: To assess the cost-effectiveness of semaglutide 2.4 mg in the treatment of adult patients with obesity (ie, body mass index [BMI] ≥ 30) and adult patients who are overweight (ie, BMI 27-29.9) with 1 or more weight-related comorbidities from a US third-party payer perspective. METHODS: A cohort Markov model was constructed to compare semaglutide 2.4 mg with the following comparators: no treatment, diet and exercise (D&E), and 3 branded AOMs (liraglutide 3 mg, phentermine-topiramate, and naltrexone-bupropion). All AOMs, including semaglutide 2.4 mg, were assumed to be taken in conjunction with D&E. Changes in BMI, blood pressure, cholesterol level, experience of acute and chronic obesity-related complications, costs, and quality-adjusted life years (QALYs) were simulated over 30 years based on pivotal trials of the AOMs and other relevant literature. Drug and health care prices reflect 2021 standardized values. Cost-effectiveness was examined with a willingness-to-pay (WTP) threshold of $150,000 per QALY gained. Sensitivity analyses were conducted to test the robustness of the cost-effectiveness results to plausible variation in model inputs. RESULTS: In the base-case analysis, treatment with semaglutide 2.4 mg was estimated to improve QALYs by 0.138 to 0.925 and incur higher costs by $3,254 to $25,086 over the 30-year time horizon vs comparators. Semaglutide 2.4 mg is cost-effective against all comparators at the prespecified WTP threshold, with the incremental cost per QALY gained ranging from $23,556 to $144,296 per QALY gained. In the sensitivity analysis, extended maximum treatment duration, types of subsequent treatment following therapy discontinuation, and weight-rebound rates were identified as key drivers for model results. The estimated probability of semaglutide 2.4 mg being cost-effective compared with comparators ranged from 67% to 100% when varying model parameters and assumptions. CONCLUSIONS: As a long-term weight management therapy, semaglutide 2.4 mg was estimated to be cost-effective compared with no treatment, D&E alone, and all other branded AOM comparators under a WTP threshold of $150,000 per QALY gained over a 30-year time horizon. DISCLOSURES: Financial support for this research was provided by Novo Nordisk Inc. The study sponsor was involved in several aspects of the research, including the study design, the interpretation of data, the writing of the manuscript, and the decision to submit the manuscript for publication. Dr Kim and Ms Ramasamy are employees of Novo Nordisk Inc. Ms Kumar and Dr Burudpakdee were employees of Novo Nordisk Inc at the time this study was conducted. Dr Sullivan received research support from Novo Nordisk Inc for this study. Drs Wang, Song, Wu, Ms Xie, and Ms Sun are employees of Analysis Group, Inc, who received consultancy fees from Novo Nordisk Inc in connection with this study.

Real-world HIV diagnostic testing patterns in the United States.

OBJECTIVES: To understand real-world implementation of the updated CDC HIV diagnostic testing algorithm. STUDY DESIGN: Retrospective database analysis. METHODS: Using data from Quest Diagnostics, we identified patients with at least 1 HIV-1/HIV-2 antibody differentiation test (BioRad Geenius HIV 1/2 Supplemental Assay [Geenius]) between January 1 and December 31, 2017. Study measures included Health Insurance Portability and Accountability Act-compliant patient demographics, test results, test frequency, and sequence relative to the CDC HIV diagnostic algorithm, including HIV-1 RNA Qualitative Assay (Aptima) or HIV-2 nucleic acid test (NAT). RESULTS: A total of 26,319 patients were identified (mean [SD] age, 40.7 [14.3] years; 66.4% male), with 28,954 Geenius tests, 7234 Aptima tests, and 298 HIV-2 NATs. In 26.4% of test sequences, the Geenius results were indeterminate or negative and required subsequent confirmatory NATs. A total of 8.5% of patients had more than 1 Geenius test in 2017, and 11.2% of the time, results of the first and second tests differed. A total of 74.2% of test sequences matched the CDC-recommended algorithm. CONCLUSIONS: Our study findings suggest that the CDC HIV diagnostic algorithm is complex and may pose suboptimal testing efficiency. Opportunities to improve diagnostic efficiency by reducing indeterminate results and repeat tests are warranted.

The Incidence, Prevalence, and Associated Costs of Anemia, Malignancy, Venous Thromboembolism, Major Adverse Cardiovascular Events, and Infections in Rheumatoid Arthritis Patients by Treatment History in the United States.

OBJECTIVE: Comorbidities in rheumatoid arthritis (RA) can influence treatment selection, impact treatment persistency, and increase health care costs. This study assessed the magnitude of comorbidity burden via epidemiology (incidence and prevalence) and associated costs of select comorbidities in RA patients: anemia, malignancy, venous thromboembolism (VTE), major adverse cardiovascular events (MACE), and infections, stratified by history of disease-modifying antirheumatic drug (DMARD) exposure. METHODS: From the IQVIA PharMetrics® Plus database, we selected adult patients with RA (2 or more RA diagnostic codes at least 30 days apart) at initiation of a new DMARD (DMARD-naïve), after the first conventional synthetic DMARD (csDMARD) or after the first biologic DMARD (bDMARD). We assessed pre-index prevalence (percentage) and on-treatment incidence (per 100 patient-years [P100PY]) of the aforementioned comorbidities. For patients with versus without incident conditions, we compared total all-cause health care costs as unadjusted and adjusted for baseline characteristics and health care costs. RESULTS: Prior to initiating a new treatment, among DMARD-naïve patients (N = 28,201), csDMARD switchers (N = 7,816), or bDMARD switchers (N = 4,656), the overall prevalence ranged from 14.1% to 16.2% (anemia), from 1.3% to 5.2% (malignancy, evaluated in csDMARD and bDMARD switchers), from 1.5% to 2.1% (VTE), from 1.8% to 2.9% (MACE), and from 66.6% to 76.1% (infections). Once on index treatment, overall incidence (P100PY) among the cohorts ranged from 6.9 to 8.9 (anemia), from 2.0 to 2.3 (malignancy), from 0.7 to 0.9 (VTE), from 1.6 to 2.0 (MACE), and from 77.4 to 87.7 (infections). The incident comorbidities (except herpes zoster) were associated with increased adjusted health care costs. CONCLUSION: Anemia, malignancy, VTE, MACE, and infections affect patients with RA at all stages of their treatment journey and are associated with increased health care costs.

Effect of a Patient Support Program for Idiopathic Pulmonary Fibrosis Patients on Medication Persistence: A Retrospective Database Analysis.

INTRODUCTION: In 2015, Boehringer Ingelheim (BI) created a support program for patients with idiopathic pulmonary fibrosis (IPF) treated with nintedanib, to help patients obtain their prescription, learn about their disease and medication, and provide support in the management of their IPF. The purpose of this study was to measure the impact of the program on nintedanib persistence among patients with IPF newly treated with the medication. METHODS: A retrospective cohort analysis of BI Pharmaceuticals, Inc.'s Specialty Pharmacy (SP) database was conducted. Patients at least 18 years of age, newly treated with nintedanib from April 1, 2015 to January 31, 2018, and with at least one diagnosis of IPF were included in the study; earliest nintedanib prescription was the index date. Patients were classified into two mutually exclusive cohorts: enrolled in the patient support program within 60 days of index or not enrolled in the program at any time. The cohorts were compared in terms of patient characteristics, time to nintedanib discontinuation (a gap of more than 60 days between refills), and proportion of persistent patients at 6, 12, 18, and 24 months after index. Time to discontinuation was compared between the cohorts using Kaplan-Meier analysis. A multivariable Cox proportional hazards model assessed the impact of program participation on time to discontinuation within the first 12 months. RESULTS: A total of 3114 enrolled and 9388 non-enrolled patients were identified. The proportion of patients persistent on nintedanib was higher among enrolled patients throughout the post-index period (57.8% vs. 49.7% at 6 months, 34.7% vs. 28.9% at 12 months; p < 0.05). In adjusted analyses, being enrolled in the program was associated with a 21% decreased hazard of discontinuing nintedanib over the first-year post-index [hazard ratio (HR) = 0.79, 95% CI 0.75-0.83, p < 0.05). CONCLUSION: Real-world evidence suggests a persistence benefit for patients with IPF treated with nintedanib who are enrolled in the patient support program.

Treatment Patterns and Healthcare Outcomes with Collagenase Clostridium Histolyticum vs Surgery in Peyronie's Disease: A Retrospective Claims Database Analysis.

INTRODUCTION: Treatments for Peyronie's disease (PD) include surgical management and collagenase clostridium histolyticum (CCH). AIMS: To evaluate PD treatment trends after CCH approval and compare clinical outcomes in CCH- and surgery-treated cohorts. METHODS: Patients newly diagnosed with PD between January 2011 and December 2017 were identified in a U.S. claims database. Cohorts initiating treatment with CCH or surgery between January 2014 and June 2017 were included. Patients were continuously enrolled ≥6 months before and ≥12 months after index date. Post-treatment penile complications and analgesic use were compared 1 year after procedure in propensity score-matched cohorts. MAIN OUTCOME MEASURES: The main outcome measures of this study were treatment patterns, penile complications, and analgesic use. RESULTS: In the newly diagnosed PD cohort, 1,609 patients received CCH and 1,555 patients had surgery. Overall CCH or surgery treatment rate/year increased from 9.8% in 2014 to 15.5% in 2017, with <1% receiving verapamil or interferon. Initial treatment ratios of CCH to surgery increased from approximately 1:1 (2014) to 2:1 (2017). In the unmatched CCH (n = 1,227) and surgery (n = 620) cohorts, more (P < .05) surgery-treated patients received analgesics (particularly opioids), oral PD therapies, vacuum erection devices, and phosphodiesterase-5 inhibitors before the index date. After propensity score matching (n = 620/cohort), newly occurring postprocedural complications during the follow-up period were higher in the surgery cohort (25.3% vs 18.4%, P = .003). The surgery cohort had significantly (P < .05) higher rates of erectile dysfunction (65.0% vs 44.8%), penile pain (17.9% vs 8.9%), and penile swelling (8.1% vs 5.2%) and was more likely to be prescribed opioids (93.3% vs 38.9%; P < .0001) or non-steroidal anti-inflammatory drugs (27.0% vs 20.3%; P = .006). CONCLUSION: CCH demonstrated fewer complications and less analgesic use than surgery and was used as the initial therapy for PD twice as often as surgery. L Trost, H Huang, X Han, et al. Treatment Patterns and Healthcare Outcomes with Collagenase Clostridium Histolyticum vs Surgery in Peyronie's Disease: A Retrospective Claims Database Analysis. Sex Med 2021;9:100321.

The Impact of a Forced Non-Medical Switch of Inhaled Respiratory Medication Among Patients with Asthma or Chronic Obstructive Pulmonary Disease: A Patient Survey on Experience with Switch, Therapy Satisfaction, and Disease Control.

PURPOSE: Budesonide/formoterol pressurized metered-dose inhaler (pMDI) was removed from a Medicare Part D formulary, and patients switched to fluticasone-based dry powder inhaler (DPI) therapies. This study describes the experience, satisfaction, and disease control among patients with asthma or chronic obstructive pulmonary disease (COPD) who switched due to removal from the formulary. PATIENTS AND METHODS: A patient survey was conducted among adults with asthma or COPD who used budesonide/formoterol pMDI for ≥3 months prior to the formulary block and the new medication for ≥3 weeks after switching, recruited by providers in a research panel. Survey comprised both validated instruments (PASAPQ, OEQ, ACQ-6, and CAT) and stand-alone questions. Patient characteristics, switch experience, device and treatment satisfaction, onset of effect, and disease control were compared between disease (asthma and COPD) and medication (once and twice daily) cohorts. Minimal significance for group differences: P≤0.05. RESULTS: Among 100 patients, 93% received communication from their doctor or nurse about the switch and 73% received training on using the new inhaler. Patients used their new treatment for an average of 7 months prior to completing the survey. Patient satisfaction with the new therapy was high (PASAPQ; mean overall satisfaction: 6.2 for asthma; 6.0 for COPD; P=0.338). However, asthma was not well controlled (ACQ-6) in 62% of patients with asthma, and 56% of patients with COPD reported high/very high impact of their illness on their lives (CAT). Sixty-eight percent and 70% of patients with asthma and COPD, respectively, required reliever medication (≥3 puffs) most days during the week prior to the survey. There were no significant differences in disease control (ACQ-6, CAT) between once-daily and twice-daily treatments (P>0.05 for both asthma and COPD). CONCLUSION: Even when reporting satisfaction with their new medication, objective measures showed substantial morbidity, regardless of DPI device or dosing regimen.

Correction to: Real-World Treatment Patterns, Adverse Events, Resource Use, and Costs Among Commercially Insured, Younger Patients with Chronic Lymphocytic Leukemia in the USA: A Retrospective Cohort Study.

In the original article, it has been noticed that the abbreviation ''CLL'' is incorrectly published throughout the paper as the abbreviation "CCL". The correct abbreviation is "CLL".

Real-World Treatment Patterns, Adverse Events, Resource Use, and Costs Among Commercially Insured, Younger Patients with Chronic Lymphocytic Leukemia in the USA: A Retrospective Cohort Study.

INTRODUCTION: Amidst a changing treatment landscape, real-world evidence on the burden of chronic lymphocytic leukemia (CLL) is limited. The purpose of this study was to describe treatment patterns, adverse events (AEs), and economic burden among treated patients with CLL. METHODS: A retrospective cohort study was conducted with IQVIA PharMetrics® Plus. Patients at least 18 years old with CLL treatment between November 1, 2013 and May 31, 2018 were identified; index date was first observed CLL treatment. Patients had at least one CLL diagnosis pre-index and a second diagnosis anytime during the study period, at least 1-year pre- and at least 30-day post-index continuous enrollment and no pre-index CLL treatment. Analyses focused on patients receiving one of the four most common regimens observed. Outcomes included treatment patterns, frequency of incident AEs, and healthcare resource use and costs. Multivariable logistic regression and generalized linear modelling were used to evaluate risk of hospitalization and all-cause costs per patient per month (PPPM). RESULTS: A total of 1706 patients were included in the study (median [interquartile range] age 58 [55-62] years, 66% male, median Charlson Comorbidity Index 2 [2-3], median follow-up 16 [8-28] months). Common regimens, irrespective of treatment line, were bendamustine-rituximab (B-R, 27%), ibrutinib monotherapy (I, 27%), rituximab monotherapy (R, 19%), and fludarabine combined with cyclophosphamide and rituximab (FCR, 16%); 59% had at least one incident AE (B-R, 62%; I, 60%; R, 25%; FCR, 79%). Mean total all-cause healthcare cost over follow-up was $13,858 ± 14,626 PPPM. Increased number of AEs was associated with increased odds of hospitalization (odds ratio = 2.9; 95% confidence interval [CI] 2.5-3.4) and increased mean cost PPPM (cost ratio = 1.2; 95% CI 1.1-1.2). CONCLUSION: This study highlights the treatment toxicity and associated economic burden among patients with CLL in the USA. As novel therapies are increasingly used, further research examining outcomes will inform the risks, benefits, and value of novel agents to prescribers and patients.

Real-world treatment patterns for patients with metastatic head and neck squamous cell carcinoma treated with immuno-oncology therapy.

BACKGROUND: Real-world use of immuno-oncology (IO) therapies (nivolumab and pembrolizumab) in metastatic head and neck squamous cell carcinoma (mHNSCC) has not been well studied. METHODS: mHNSCC patients treated with an IO therapy were identified from a large US claims database from 2016 to 2017. Treatment patterns before and after initiation of IO therapy (index date) were described. RESULTS: Among 416 mHNSCC patients, 85% had ≥1 regimen prior to IO therapy. Ninety-seven percent of patients initiated IO as monotherapy and 3% initiated IO combined with another systemic treatment. One hundred seventeen (28%) patients had a subsequent regimen, usually chemotherapy (n = 58, 50%) or IO monotherapy (n = 27, 23%), of which 22 patients restarted the same IO therapy and 5 switched to another IO monotherapy. CONCLUSION: The majority of mHNSCC patients initiated IO as a monotherapy. Approximately half of patients with a subsequent regimen received chemotherapy and one-fourth received IO monotherapy.

Real-World Economic Outcomes of Brexpiprazole and Extended-Release Quetiapine Adjunctive Use in Major Depressive Disorder.

PURPOSE: Major depressive disorder (MDD) is a chronic mental disorder with a substantial clinical and economic burden. Despite the efficacy of adjunctive atypical antipsychotics (AAP) for augmentation in patients with major depressive disorder (MDD) who failed first-line antidepressant therapy (ADT), little is known of the impact of AAP choices on healthcare resource use and costs in real-world practice. Therefore, this study compared real-world healthcare utilization and costs in patients with MDD treated with brexpiprazole or extended-release (XR) quetiapine as adjunctive treatment to ADT. PATIENTS AND METHODS: Adults with MDD starting adjunctive treatment with brexpiprazole (n=844) or extended-release (XR) quetiapine (n=688) were identified in the adjudicated health plan claims data (07/2014 - 09/2016). Resource use and healthcare costs in the 6 months following treatment initiation were compared between non-matched populations, and between propensity score-matched groups, and by multivariable regression analyses. RESULTS: During follow-up, unadjusted all-cause hospitalization (6.6% vs 12.5%) and ED visits (17.0% vs 27.5%) were lower with brexpiprazole compared to quetiapine XR (both p<0.001). Brexpiprazole-treated patients had significantly lower mean medical costs (US$6,421 vs US$8,545, p=0.0123) but higher mean pharmacy costs (US$7,401 vs US$4,691, p<0.0001) than quetiapine XR-treated patients did. Total healthcare costs were not significantly different between the two cohorts. Propensity score-matched comparisons of 397 patients in each cohort showed no statistically significant difference in all-cause hospitalization, ED visits, and total healthcare costs; and significantly lower medical costs (US$5,719 vs US$8,602, p=0.0092) but higher pharmacy costs (US$7,091 vs US$5,091, p=0.0007) in brexpiprazole compared to quetiapine XR. In multivariable regressions, brexpiprazole was associated with 16.1% lower medical costs (p=0.0186) and 9.4% higher total healthcare costs (p=0.0463) as compared to quetiapine XR. CONCLUSION: Significantly lower medical costs were observed in patients with MDD treated with brexpiprazole vs quetiapine XR.

Treatment patterns, adverse events, healthcare resource use and costs among commercially insured patients with mantle cell lymphoma in the United States.

INTRODUCTION: There are limited data on treatment patterns, adverse events (AEs), and economic burden in younger, commercially insured patients treated for mantle cell lymphoma (MCL). METHODS: Adults with ≥1 treatment for MCL between 1 November 2013-31 December 2017 were identified from IQVIA Real-World Data Adjudicated Claims-US; index date was first treatment. Patients carried ≥1 MCL diagnosis, were newly treated, and were enrolled continuously for ≥12 months prior to and ≥30 days following index. Patients receiving the four most common MCL regimens were included. Measures included frequency of incident AEs, resource use, and costs overall and by number of AEs. Adjusted logistic regression and generalized linear modeling evaluated risk of hospitalization and all-cause costs per patient per month (PPPM). RESULTS: Two thousand five hundred and nine treated patients had a drug-specific code and were classified to a specific treatment regimen. Of those patients, 1785 patients received at least one of the four most commonly used MCL regimens (R-CHOP, rituximab monotherapy, B-R, and ibrutinib) at some point over follow-up (median 23 months). R-CHOP was the most common regimen observed in the first line (26%), followed by rituximab monotherapy (19%), B-R (15%), and ibrutinib (5%). The median age was 57 years; median Charlson Comorbidity Index was 0. Among patients receiving the four most common regimens, 63% of patients experienced ≥1 incident AE (R-CHOP 77%, B-R 58%, and ibrutinib 52%). An increasing number of incident AEs was associated with increased hospitalization risk (odds ratio = 2.4; 95% Confidence Interval [CI] 2.1-2.7) and increased mean costs PPPM (cost ratio = 1.1; 95% CI 1.1-1.2). DISCUSSION: This is the largest study describing treatment patterns and clinical and economic impact of MCL treatment. The most common regimens were R-CHOP, rituximab monotherapy, B-R, and ibrutinib. The majority of treated patients experienced at least one incident AE, with hospitalization risk and all-cause costs increasing as the number of AEs increased.

Clinical outcomes associated with pathogenic genomic instability mutations in prostate cancer: a retrospective analysis of US pharmacy and medical claims data.

Background: Prostate cancer (PC) is a clinically heterogenous disease, and genetic mutations may be useful for patient risk stratification. This retrospective cohort study compared clinical outcomes, pharmacy use, and outpatient resource use in PC patients with and without pathogenic genomic instability mutations, including DNA repair deficiency (DRD) mutations and those in TP53, PTEN, and RB1. Methods: Patients ≥18 years newly-diagnosed with PC between June 2011-March 2016 were identified in medical and prescription claims databases linked to a genomic dataset. All-cause and PC-specific pharmacy use and outpatient resource use (office visits, laboratory tests, radiology examinations, and radiation therapies) over 1, 2, and 3 years and time to evidence of disease progression after PC diagnosis, based on secondary cancer diagnosis codes and treatments received, were evaluated in mutation carriers with ≥1 of 24 gene mutations and in a sub-set of DRD gene mutation carriers, with each compared to non-mutation carriers. Results: Mutation carriers (n = 274) and non-mutation carriers (n = 74) had similar demographic and clinical features. Non-mutation carriers had lower risks of developing metastasis and castration-resistant PC than mutation carriers (hazard ratio [HR] = 0.7, 95% CI = 0.5-0.9; HR = 0.5, 95% CI = 0.3-0.9, respectively) and DRD mutation carriers (HR = 0.5, 95% CI = 0.3-0.7; HR = 0.4, 95% CI = 0.2-0.7, respectively). Compared to non-mutation carriers, mutation carriers had more all-cause pharmacy claims over 2 years of follow-up (74.4 vs 59.1, p = 0.04) and more PC-specific pharmacy claims over 2 years (11.1 vs 6.5, p = 0.01) and 3 years (17.9 vs 9.8, p = 0.01) of follow-up. No differences were observed in outpatient resource use during the follow-up period by mutation status. Conclusion: PC patients carrying ≥1 pathogenic DNA instability mutation, and DRD mutation carriers specifically, had higher clinical burden than non-mutation carriers. Targeted therapies for these patients are needed to reduce clinical burden and associated healthcare resource utilization.

Assessment and effect of a gap between new-onset epilepsy diagnosis and treatment in the US.

OBJECTIVE: To estimate the treatment gap between a new epilepsy diagnosis and antiepileptic drug (AED) initiation in the United States. METHODS: Retrospective claims-based cohort study using Truven Health MarketScan databases (commercial and supplemental Medicare, calendar years 2010-2015; Medicaid, 2010-2014) and a validation study using PharMetrics Plus Database linked to LRx claims database (2009-2014). Persons met epilepsy diagnostic criteria, had an index date (first epilepsy diagnosis) with a preceding 2-year baseline (1 year for persons aged 1 to <2 years; none for persons <1 year), and continuous medical and pharmacy enrollment without epilepsy/seizure diagnosis or AED prescription during baseline. Outcomes included percentage of untreated persons (no AED prescription) up to 3 years' follow-up and comparative outcomes (incidence rate ratio: untreated persons/treated persons), including medical events and health care resource utilization. RESULTS: In the primary study, 59,970 persons met selection (or inclusion) criteria; 36.7% of persons with newly diagnosed epilepsy remained untreated up to 3 years after diagnosis. In the validation study (N = 30,890), 31.8% of persons remained untreated up to 3 years after diagnosis. Lack of AED treatment was associated with an adjusted incidence rate ratio (95% confidence interval) of 1.2 (1.2-1.3) for medical events, 2.3 (2.2-2.3) for hospitalizations, and 2.8 (2.7-2.9) for emergency department visits. CONCLUSIONS: One-third of newly diagnosed persons remain untreated up to 3 years after epilepsy diagnosis. The increased risk of medical events and health care utilization highlights the consequences of delayed treatment after epilepsy diagnosis, which might be preventable.

Comparative effectiveness of teriflunomide and dimethyl fumarate in patients with relapsing forms of MS in the retrospective real-world Teri-RADAR study.

AIM: Head-to-head clinical trials of teriflunomide (TFM) versus dimethyl fumarate (DMF) have not been conducted. OBJECTIVES: To compare the real-world effectiveness of TFM versus DMF. METHODS: Anonymized data were collected from patients with relapsing multiple sclerosis (MS) initiating treatment with teriflunomide (N = 50) or DMF (N = 50). RESULTS: On follow-up magnetic resonance imaging (MRI) compared with baseline, with TFM versus DMF treatment, the proportion of patients with new/enlarging T2 or gadolinium-enhancing lesions was 30.0 versus 40.0% (p = 0.2752). However, median annualized percent whole brain volume change was -0.1 versus -0.5 (p = 0.0212). There were no significant treatment differences on additional MRI and clinical end points and no unexpected safety signals. CONCLUSION: The effectiveness of teriflunomide was superior to DMF on whole brain atrophy and similar to DMF on other MRI/clinical end points.

The impact of copay assistance on patient out-of-pocket costs and treatment rates with ALK inhibitors.

INTRODUCTION: The patient cost burden of oral anticancer medicines has been associated with prescription abandonment, delayed treatment initiation, and poorer health outcomes in the US. Since 2011, several small molecule tyrosine kinase inhibitors have been approved for the treatment of non-small cell lung cancer (NSCLC) patients with rearrangement of the anaplastic lymphoma kinase (ALK) gene. The objective of this study was to measure the impact of copay assistance on patient cost sharing and treatment patterns in patients prescribed oral ALK inhibitors (ALKi's). METHODS: Patterns of claims approval/rejection and payment/reversal, out-of-pocket (OOP) costs, and treatment persistence were reported for patients identified in the IQVIA Formulary Impact Analyzer database from January 2013 to August 2017 linked to a medical claims database. The primary study cohorts were patients with copay assistance, including manufacturer's copay cards, other discount cards, or free-trial vouchers, on the index ALKi claim, and patients without copay assistance at any time during the follow-up period. RESULTS: In total, 3,143 patients were included in analyses related to claim patterns, and 1,685 patients were included in analyses related to treatment persistence. Copay assistance decreased the OOP cost for the first approved ALKi by $1,930, on average. Patients with copay assistance picked up ALKi prescriptions from the pharmacy sooner than patients without copay assistance (2.6 days vs 25.7 days). In adjusted analyses, patients with copay assistance had 88.2% lower risk of abandoning their first approved prescription and 24.3% lower risk of discontinuing treatment with the first observed ALKi (all p < 0.001). CONCLUSION: Copay assistance reduced the patient cost burden for ALKi's and was associated with patients picking up their ALKi prescriptions, beginning ALKi treatment sooner, and remaining on treatment.

Evaluation of patient support program and adherence to long-acting injectable aripiprazole for patients utilizing injection local care centers.

OBJECTIVE: Patient support programs, such as the ASSURE Program for long-acting injectable aripiprazole, are designed to help support access to medications, including long-acting injectable (LAI) antipsychotics for patients with schizophrenia. This study was conducted to evaluate adherence to long-acting injectable aripiprazole among patients utilizing the program local care centers (LCC). METHODS: Data collected from participating LCC between October 2014 and February 2018 were utilized. Characteristics of patients receiving injections at LCC and participating in additional support services of the program, types of program offering utilized and patient cost share for long-acting injectable aripiprazole were described. Adherence, measured as the proportion of days covered (PDC) during follow-up, was estimated in patients utilizing the LCC for 6 months and 9 months. Patients with PDC ≥80% were considered adherent to treatment. RESULTS: Two hundred and thirty-four patients received at least one injection at participating LCC and enrolled in the patient support program. Mean (SD) age was 37.3 (13.5) years; 60.7% were male; 32.5% were covered by Medicare. In total, 157 and 87 patients were actively utilizing the LCC for at least 6 months and 9 months, respectively. PDC of 97% and 98% were reported among patients with 6 months and 9 months of follow-up, respectively, and patients were considered adherent to long-acting injectable aripiprazole during follow-up. CONCLUSION: Patients utilizing the LCC demonstrated high medication adherence, suggesting that injection services provided by the centers may reduce barriers to treatment and help patients with schizophrenia remain on LAI antipsychotic treatment.

Adherence rates and health care costs in Crohn's disease patients receiving certolizumab pegol with and without home health nurse assistance: results from a retrospective analysis of patient claims and home health nurse data.

BACKGROUND: Patient support programs have a positive effect on adherence to therapy. Certolizumab pegol (CZP) is a tumor necrosis factor antagonist for the treatment of Crohn's disease. OBJECTIVES: To assess, using real-world claims data, whether home health nurse assistance had an effect on patients' adherence to CZP and to measure its impact on health care use and costs. METHODS: A retrospective analysis of medical and pharmacy claims data from the IQVIA Real-World Data Adjudicated Claims Database was conducted using data from January 1, 2007 through September 30, 2015. CZP patients with Crohn's disease were eligible to receive self-administration instructions from a nurse or nurse-administered CZP injections, or both. These services were provided by CIMplicity®, a home health nurse program sponsored by UCB Pharma. Cohorts were based on patients with and without nurse assistance and were matched based on gender and categorical age. Adherence to CZP was determined using the medication possession ratio (MPR) and proportion of days covered (PDC). A Kaplan-Meier analysis was performed to compare time to discontinuation of CZP between the two cohorts. Multivariate regression analyses were performed, adjusting for additional covariates to compare the effect of CZP with and without nurse assistance on hospitalization and total health care costs. RESULTS: Patients with at least 12 months of continuous enrollment post-index date were evaluated for adherence to CZP (n=276 in each cohort). The mean and median PDC and MPR values were higher with nurse assistance than without. Time to discontinuation was significantly longer in patients who received CZP with nurse assistance than without (P=0.0004). Results from the multivariate analyses showed a significant reduction in all-cause hospitalization (-55.8%; P=0.0026) and total health care costs (-14.3%; P=0.0045) with nurse assistance. CONCLUSION: This analysis suggests that home health nurse assistance increases adherence to CZP and reduces health care costs in patients with Crohn's disease.