The lived experience of positive airway pressure therapy in patients with obstructive sleep apnea across the lifespan: a qualitative study.

STUDY OBJECTIVES: Although treatment of obstructive sleep apnea (OSA) with positive airway pressure (PAP) therapy is effective, adherence is often poor. Understanding the patient perspective is needed to inform adherence-promoting interventions. This qualitative study assessed the experiences, preferences, facilitators, and barriers surrounding PAP therapy for the management of OSA in patients from adolescence to older adulthood. METHODS: Eligible participants ages 19 and older were identified from administrative health care claims; adolescent participants ages 12-18 and their parents/caregivers were identified via electronic health records of a tertiary sleep specialty clinic at a large children's hospital. Forty English-speaking patients and 10 parents of adolescents diagnosed with OSA and prescribed PAP therapy completed semistructured 60-minute telephone interviews conducted by a trained facilitator. Common themes and illustrative quotes were identified. RESULTS: Themes around OSA diagnosis, initiating OSA treatment, learning about OSA/PAP, decision to start PAP, PAP benefits and challenges, and reasons for nonadherence were identified. Participants suggested design and delivery changes to improve PAP devices. Issues unique to adolescents and their parents were discussed. CONCLUSIONS: The unique perspectives of patients regarding PAP therapy should be taken into consideration when developing interventions to increase PAP adherence and improve clinical care. Based on identified themes, opportunities for intervention may exist at all stages of care, from diagnosis to treatment initiation. Involving partners, parents, and other caregivers in PAP therapy may be beneficial for optimizing adherence. CITATION: Simon SL, Stephenson JJ, Haynes K, et al. The lived experience of positive airway pressure therapy in patients with obstructive sleep apnea across the lifespan: a qualitative study. J Clin Sleep Med. 2024;20(3):407-416.

Health related social needs and whole person health: Relationship between unmet social needs, health outcomes, and healthcare spending among commercially insured adults.

Commercially-insured adults comprise a majority of health plan members but are least likely to be surveyed about their social needs. Little is known, consequently, about health-related social needs (HRSNs) in this population. The primary aim of this study was to assess the prevalence of HRSNs and health among commercially-insured adults and estimate their relationship with health outcomes and spending. This cross-sectional study used survey data from a representative sample of Elevance Health commercially insured members residing in Georgia and Indiana (U.S.) Adult members reported on HSRNs across nine different domains. Survey data were linked to medical claims data, and regression models were used to estimate the relationship between HRSNs and self-reported health, emergency department visits, three major health outcomes, and healthcare spending (medical and pharmaceutical). Of 1,160 commercially insured adults, 76 % indicated ≥ 1 HRSN, and 29 % reported > 3 HRSNs, (i.e., "high" HRSN). Each HRSN was associated with 2.2 (95 % CI, 1.84-2.55) additional unhealthy days per month, 3.0 percent (95 % CI 1.36 - 4.57) higher prevalence of anxiety/depression, 2.2 percent (95 % CI 0.88 - 3.50) higher prevalence of hypertension, 3.9 more ED visits per 1,000 member-months (95 % CI, 0.29-7.42), and $1,418 higher total healthcare spending (95 % CI, $614.67-$2,220.39) over a 12-month period. The widespread prevalence of HRSNs among commercially insured adults demonstrates the importance of screening all health plan members for HRSNs-not just Medicare and Medicaid members. Commercially insured members who experience high HRSN are at significantly higher risk for worse health, even after controlling for income and demographic characteristics.

Handling Missing Data in Health Economics and Outcomes Research (HEOR): A Systematic Review and Practical Recommendations.

BACKGROUND: Missing data in costs and/or health outcomes and in confounding variables can create bias in the inference of health economics and outcomes research studies, which in turn can lead to inappropriate policies. Most of the literature focuses on handling missing data in randomized controlled trials, which are not necessarily always the data used in health economics and outcomes research. OBJECTIVES: We aimed to provide an overview on missing data issues and how to address incomplete data and report the findings of a systematic literature review of methods used to deal with missing data in health economics and outcomes research studies that focused on cost, utility, and patient-reported outcomes. METHODS: A systematic search of papers published in English language until the end of the year 2020 was carried out in PubMed. Studies using statistical methods to handle missing data for analyses of cost, utility, or patient-reported outcome data were included, as were reviews and guidance papers on handling missing data for those outcomes. The data extraction was conducted with a focus on the context of the study, the type of missing data, and the methods used to tackle missing data. RESULTS: From 1433 identified records, 40 papers were included. Thirteen studies were economic evaluations. Thirty studies used multiple imputation with 17 studies using multiple imputation by chained equation, while 15 studies used a complete-case analysis. Seventeen studies addressed missing cost data and 23 studies dealt with missing outcome data. Eleven studies reported a single method while 20 studies used multiple methods to address missing data. CONCLUSIONS: Several health economics and outcomes research studies did not offer a justification of their approach of handling missing data and some used only a single method without a sensitivity analysis. This systematic literature review highlights the importance of considering the missingness mechanism and including sensitivity analyses when planning, analyzing, and reporting health economics and outcomes research studies.

Patient Perspectives on the Burden of Heart Failure with Preserved Ejection Fraction in a US Commercially Insured and Medicare Advantage Population: A Survey Study.

Background: Patient-reported health related quality of life (HRQOL) is not routinely assessed in clinical practice. Little is known about health status outcomes reported by patients with heart failure with preserved ejection fraction (HFpEF) in non-clinical trial settings. Purpose: To better understand patient burden of HFpEF in terms of HF-specific functional and symptom status, HRQOL, healthcare resource utilization (HCRU) and costs in a US-based commercial and Medicare Advantage insured population. Patients and methods: We conducted a cross-sectional survey of patients with HFpEF and linked their survey and administrative claims data. Consenting, eligible patients completed a survey that included the 23-item Kansas City Cardiomyopathy Questionnaire (KCCQ-23) and the PROMIS Global Health-10 (GH-10) questionnaire, as well as clinical and demographic questions. HF medication use, HCRU and costs during the 12-month baseline period before the survey were determined from claims data. Generalized linear regression was used to assess the associations between baseline characteristics and the KCCQ-23 overall summary score. Results: Of 598 survey respondents with survey and claims data, 54.7% were female with mean age 74.0 years. The KCCQ-23 overall summary and clinical summary scores were 64.8 and 63.0, respectively, and the GH-10 physical and mental health summary scores were 39.9 and 45.5. Factors related to lower KCCQ-23 overall summary scores were HF treatment and symptom changes during the past 4-weeks before the survey, hospital admission during the past year, low household income, high comorbidity index, and morbid obesity (BMI>40). Total all-cause healthcare costs were $38,243 during the year prior to the survey, of which 42% were HF-related. Conclusion: Patient-reported outcome measure scores indicated impairment due to HF symptoms and physical limitations in this real-world sample of patients with HFpEF, highlighting a need to assess patient-reported outcomes as well as the clinical and economic outcomes traditionally assessed by clinicians, health systems and payers.

Treatment Experiences with CDK4&6 Inhibitors Among Women with Metastatic Breast Cancer: A Qualitative Study.

PURPOSE: To describe patients' perspectives on the use of and potential challenges and barriers with adherence/persistence to cyclin-dependent kinase 4 and 6 inhibitors (CDK4&6i's) to treat metastatic breast cancer (MBC). METHODS: This qualitative study consisted of 60-minute semi-structured telephone interviews with patients with MBC in the US who were either current or recent CDK4&6i users, identified from administrative claims of survey-eligible commercial and Medicare Advantage patients in the HealthCore Integrated Research Database between November 1, 2018 and November 1, 2019. Patients were recruited by email and/or mailed letter. The 60-minute telephone interviews were conducted by a trained facilitator using a study-developed interview discussion guide that included topics impacting treatment choice and adherence/persistence. Interviews were audio-recorded, transcribed, and thematically analyzed. RESULTS: All 462 eligible patients were sent a recruitment email and/or letter to which 36 patients responded, consented to participate, and met study inclusion criteria; 25 patients scheduled interviews, and 24 completed them. Study participants were predominately white, non-Hispanic (96%) with a mean age of 59.5 years. Participants reported a largely positive experience and mentioned very few adherence/persistence issues. They further reported appreciating the ease and convenience of oral oncolytics, coped with side effects, had strong medical and social support, and experienced few cost issues. CONCLUSION: The few adherence/persistence issues reported by participants contrasts with other findings of suboptimal oral oncolytic use. Interview themes indicated several factors that likely contributed to the lack of adherence/persistence issues: trusted relationship with oncologist, belief in importance of medication, positive medication views, strong medical and social support, and minimal personal drug cost. Future research should focus on whether and how much these factors impact adherence/persistence in more diverse populations. If adherence/persistence issues are identified in these populations, then it would be appropriate to study the development of interventions that target factors associated with better adherence/persistence.

Patient Knowledge of Safe Use of ER/LA Opioid Analgesics Following Implementation of the Class-Wide REMS: A Survey Study.

BACKGROUND/RATIONALE: The US Food and Drug Administration (FDA) approved a Risk Evaluation and Mitigation Strategy (REMS) for extended release/long-acting (ER/LA) opioids in 2012. The purpose of this study was to assess patient knowledge of the safe use of these products following implementation of the REMS and to determine possible effects of the REMS, including impact on medication access. OBJECTIVE: To assess patient knowledge of safe use of ER/LA opioids and use of REMS patient education tools such as the Medication Guide (MG) and Patient Counseling Document (PCD). METHODS: This was a cross-sectional survey of commercially insured (Commercial) and Medicare Advantage-insured (Medicare) adults with ≥1 pharmacy claim for an ER/LA opioid (10/01/2015 - 02/28/2017) in the HealthCore Integrated Research Database and Medicaid-insured (Medicaid) adult members of a research panel, about their knowledge of safe use of ER/LA opioids and receipt/comprehension of the MG and PCD. RESULTS: Survey respondents consisted of 382 Commercial, 43 Medicare and 40 Medicaid adults. While ≥95% of respondents received and read the MG, fewer were aware of the PCD (Commercial: 47%, Medicare: 65%, Medicaid: 53%). Almost 75% of the knowledge questions were answered correctly by ≥80% of all respondents; fewer respondents recognized that use of opioids as directed can lead to death (Commercial: 73%, Medicare: 56%, Medicaid: 63%), the MG should be read at each dispensing (Commercial: 78%, Medicare: 53%, Medicaid: 75%), opioids should not be stored in the medicine cabinet (Commercial: 77%, Medicare: 79%, Medicaid: 58%), missed doses should not be taken as soon as possible (Commercial: 56%, Medicare: 51%, Medicaid: 50%), and pills should not be crushed (Commercial: 85%, Medicare: 67%, Medicaid: 52%). CONCLUSION: Although most respondents reported reading and understanding the MG and exhibited knowledge of safe use of ER/LA opioids, providers' use of the PCD and increased understanding of safe use core messages need reinforcement.

Rate of preventative vaccine use and vaccine beliefs among a commercially insured population.

Immunization is an important component of preventive healthcare services. By recognizing and understanding factors associated with suboptimal vaccination compliance, healthcare providers can better approach at-risk populations and target efforts at reinforcing the vital importance of immunizations. The objective of this study was to understand the factors associated with adherence, beliefs and behaviors of influenza, pneumococcal, and herpes zoster vaccines receipt among commercially insured adults. A cross-sectional survey of patients with medical and pharmacy benefits for a 24-month period between August 1, 2014 and July 31, 2016 who were eligible to receive at least one of three adult vaccines (influenza, pneumococcal, and herpes zoster) was completed. Patients were identified as eligible to receive a vaccine based on current guidelines from the CDC ACIP. Health plan members were identified from administrative claims data in the HealthCore Integrated Research DatabaseSM (HIRD). Among the participants, 11% were eligible and up-to-date on all three vaccines; 52% on some and 37% were not up-to-date on any of the three vaccines. Participants with a healthcare provider were more likely to be up-to-date on eligible vaccines: 79.9% for none, 91.3% for some, and 97.8% for all eligible vaccines. The composite Vaccine Myth Belief score was significantly associated with being up to date on eligible vaccines: 45.0%/12.8% for none, 12/5%/30.8% for some, and 8.9%/33.3% for those up-to-date on all eligible vaccines. Despite numerous interventions designed to increase vaccination rates among adults, compliance remains suboptimal. It is evident that patient and provider education is necessary to fill knowledge gaps and misunderstandings; however knowledge by itself is not sufficient to improve immunization practices. Our results highlight a population that could benefit from a multidisciplinary approach, including interventions at the individual and health system levels.

Observational Real-World Study to Assess Clinical Characteristics and Device Satisfaction in Patients with COPD Treated with Glycopyrrolate/eFlow® CS.

Purpose: To assess clinical characteristics and device satisfaction of patients with chronic obstructive pulmonary disease (COPD) treated with glycopyrrolate/eFlow® Closed System (CS) nebulizer (further referred to as eFlow) under real-world conditions. Participants and Methods: Patients with COPD currently using eFlow were identified by the study sponsor. Consenting patients who met study inclusion criteria completed a cross-sectional survey that included a device satisfaction questionnaire. Means, medians, and standard deviations were calculated. Results: Sixty-six patients met inclusion criteria and completed the survey. Participants' mean ± standard deviation age was 64.9 ± 11.9 years and the majority were white (86.4%) and female (59.1%). Almost two-thirds were former smokers. Thirty-nine (59.1%) reported their COPD to be severe/very severe and 38 (57.6%) reported a COPD exacerbation resulting in a hospitalization, ER visit, or medication modification over the past 12 months. Among 55 participants who had previously used another type of nebulizer, 44 (80%) were overall "much more"/"somewhat more" satisfied with the eFlow compared with their previous nebulizer(s). Regardless of prior nebulizer use, 60 (90.9%) participants were "satisfied"/"very satisfied" overall with the eFlow. Assembly and disassembly, operation, and cleaning were perceived as being "easy"/"very easy" by at least 65% of participants. Among all participants, 57 (86.4%) were "confident"/"very confident" of glycopyrrolate administration. On a Likert scale of 1 ("I don't like it") to 7 ("I like it a lot"), mean scores were at least 5.9 for portability, ease of cleaning, size, weight, short administration time, and relative silence of the device. Over 80% of participants said they "probably"/"definitely" would continue to use eFlow. Conclusion: Based on this real-world study, the majority of patients were highly satisfied with, and confident in, using eFlow.

Patient-Centric Structural Determinants of Adherence Rates Among Asthma Populations: Exploring the Potential of Patient Activation and Encouragement Tool TRUSTR to Improve Adherence.

BACKGROUND: Lack of adherence with prescribed medications among the asthma populations exacerbates health outcomes and increases social and economic costs. OBJECTIVES: The proposed study aims to model patient-centric structural determinants of adherence rates among asthma patients and explore the potential of mobile health apps such as the TRUSTR platform to improve adherence using its power of monetary and non-monetary chatbotting and non-non-monetary nudges. Following specific hypotheses are tested: (1) Patient attributes, such as their age and monetary medical condition, have significant effect on their adherence with the prescribed treatment plans. (2) Behavioral nudging with rewards and engagement via mobile health apps will increase adherence rates. METHODS: The patient population (N = 37 359) consists of commercially insured patients with asthma who have been identified from administrative claims in the HealthCore Integrated Research Database (HIRD) between April 1, 2018 and March 31, 2019. Two Structural Equation Models (SEMs) are estimated to quantify direct, indirect, and total effect sizes of age and medical condition on proportion of days covered (PDC) and medical possession ratio (MPR), mediated by patient medical and pharmacy visits. Fourteen additional SEMs were estimated to lateralize TRUSTR findings and conduct sensitivity analysis. RESULTS: HIRD data reveal mean adherence rate of 59% (standard deviation (SD) 29%) for PDC and 58% for MPR (SD 36%). Key structural findings from SEMs derived from the HIRD dataset indicate that each additional year in the age of the patient has a positive total effect on the adherence rate. Patients with poor medical condition are likely to have lower adherence rate, but this direct effect is countered by mediating variables. Further, each additional reward and higher engagement with a mobile app is likely to have a positive total effect on increasing the adherence rate. CONCLUSIONS: HIRD data reveal mean adherence rate of 59% (SD 29%), providing the evidence for the opportunity to increase adherence rate by around 40%. Statistical modeling results reveal structural determinants, such as the opportunity to nudge, are higher among younger patients, as they have higher probability of being non-adherent. Methodologically, lateralization approach demonstrates the potential to capture real-world evidence beyond clinical data and merge it with clinical data.

Basal Insulin Initiation and Maintenance in Adults with Type 2 Diabetes Mellitus in the United States.

OBJECTIVE: A survey of US adults with type 2 diabetes mellitus was conducted to better understand patients' insulin initiation experiences and treatment persistence behaviors. RESEARCH DESIGN AND METHODS: Participants were recruited from consumer panels and grouped by basal insulin treatment pattern: continuers (no gap of ≥7 days within 6 months of initiation); interrupters (gap ≥7 days, resumed treatment); discontinuers (stopped for ≥7 days, not resumed). A quota of approximately 50 respondents per persistence category was set. RESULTS: A total of 154 respondents (52 continuers, 52 interrupters, 50 discontinuers) completed the survey. Mean age was 51.4 years; 51.9% male. Continuers were more likely to report their views being considered during initiation, and less likely to report a sense of failure. Concerns included insulin dependence (64.3% agree/strongly agree), frequent blood glucose monitoring (55.2%), costs/ability to pay (53.9%), fears of or mistakes during self-injection (52.6%), and weight gain (52.6%). Continuers were motivated by benefits of insulin therapy; experienced or potential side effects were notable factors for interruption/discontinuation. Healthcare provider instruction was indicated as a reason for continuing, stopping, and restarting therapy. CONCLUSION: Benefits of basal insulin therapy motivated continuers while side effects impacted interruption/discontinuation. Persistence on basal insulin is often influenced by provider actions. Earlier provider intervention upon signs of treatment discontinuation may promote persistence.

The Association Between Doctor and Pharmacy Shopping and Self-Reported Misuse and Abuse of Prescription Opioids: A Survey Study.

BACKGROUND/RATIONALE: Little is known about the reasons for visiting multiple doctors/pharmacies, known as doctor/pharmacy shopping, to obtain opioids. OBJECTIVE: To investigate patients' self-reported reasons for doctor/pharmacy shopping and assess whether doctor/pharmacy shopping behavior can be used as a surrogate measure of opioid abuse/misuse. METHODS: We conducted a cross-sectional web-based survey among adult patients with ≥2 pharmacy claims for immediate-release or extended-release/long-acting opioids between 7/1/2015 and 12/31/2016, identified from a large United States (US) commercial claims database. Patients were classified into no, mild, moderate, or severe shopping categories based on their claims. Reasons for doctor/pharmacy shopping and opioid abuse/misuse were determined from patient responses to the Prescription Opioid Misuse and Abuse Questionnaire. RESULTS: A random sample of 10,081 patients was invited to participate in the survey and 1085 (11%) completed surveys. The most frequently reported reasons for doctor/pharmacy shopping were convenience, availability, price, and multiple morbidities requiring pain management. Among patients in the no, minimal, moderate, and severe shopping categories, only 7.8%, 8.5%, 11.8% and 12.6% reported opioid abuse/misuse, respectively. CONCLUSION: In this commercially-insured population, patient-reported reasons for doctor/pharmacy shopping do not suggest opioid abuse/misuse. Less than 15% of patients with shopping behavior in the past 3 months reported any reasons attributable to opioid abuse/misuse, indicating that shopping behavior in this population may not be a good surrogate for abuse/misuse.

Disconnected: a survey of users and nonusers of telehealth and their use of primary care.

OBJECTIVE: The study sought to assess awareness, perceptions, and value of telehealth in primary care from the perspective of patients. MATERIALS AND METHODS: We conducted a cross-sectional, Web-based survey of adults with access to telehealth services who visited healthcare providers for any of the 20 most-commonly seen diagnoses during telehealth visits. Three groups were studied: registered users (RUs) of telehealth had completed a LiveHealth Online (a health plan telehealth service provider) visit, registered nonusers (RNUs) registered for LiveHealth Online but had not conducted a visit, and nonregistered nonusers (NRNUs) completed neither step. RESULTS: Of 32 831 patients invited, 3219 (9.8%) responded and 766 met eligibility criteria and completed surveys: 390 (51%) RUs, 117 (15%) RNUs, and 259 (34%) NRNUs. RUs were least likely to have a primary care usual source of care (65.6% vs 78.6% for RNUs vs 80.0% for NRNUs; P < .001). Nearly half (46.8%) of RUs were unable to get an appointment with their doctor, and 34.8% indicated that their doctor's office was closed. Among the 3 groups, RUs were most likely to be employed (89.5% vs 88.9% vs 82.2%; P = .007), have post-high school education (94.4% vs 93.2% vs 86.5%; P = .003), and live in urban areas (81.0% vs 69.2% vs 76.0%; P = .021). CONCLUSIONS: Telehealth users reported that they relied on live video for enhanced access and were less connected to primary care than nonusers were. Telehealth may expand service access but risks further fragmentation of care and undermining of the primary care function absent better coordination and information sharing with usual sources of patients' care.

Bridging the Gap Between Self-Reported and Claims-Derived Adherence Measures for Basal Insulin Among Patients with Type 2 Diabetes Mellitus.

INTRODUCTION: Complex or personalized insulin regimens challenge traditional adherence measures. Our objective was to develop an improved basal insulin (BI) adherence measure using both patient-reported and administrative claims data, resulting in a more complete measure. METHODS: Patients' self-reported BI utilization over the previous 12 months was linked with their claims data for the same period. Hybrid medication possession ratio (MPR) was derived by calculating expected days of insulin supply [total dispensed insulin units from claims over 12 months divided by self-reported total daily dose (TDD)]. The hybrid MPR was compared against traditional claims-based MPR, adjusted claims-based MPR, and patient-reported MPR. For all MPR measures, the adherence threshold was ≥ 0.8. A logistic model was used to predict non-adherence per hybrid MPR. The predicted model-based MPR was compared with existing measures in a larger cohort. RESULTS: The study sample consisted of 296 patients. TDD derived from claims was higher than self-reported TDD [77.9 (71.8) vs. 57.7 (38.3)], implying average dispensed insulin would last longer than claims-based days supply. Correspondingly, hybrid and MPRs adjusted for package size (56% and 71%, respectively) were higher than claims-based MPR (50%). Age, total claims-based days supply, retinopathy, adjusted MPR-based adherence, and non-insulin injectable use were key predictors of hybrid MPR-based adherence. Applying the claims-based prediction model to a larger cohort to test validity showed high correlations with predicted and adjusted MPR-based adherence. CONCLUSIONS: Traditional claims-based MPR underestimated adherence while adjusted MPR overestimated adherence when self-reported total daily dose was taken as benchmark insulin dose. The predicted model may help identify patients with poor basal insulin adherence. More research is needed to further confirm the findings. FUNDING: Eli Lilly and Company, Indianapolis, IN, USA.

Real-world Clinical Characteristics, Treatment Patterns, and Exacerbations in US Patients With Asthma Newly Treated With Omalizumab.

PURPOSE: The objective of this study was to examine patient characteristics, treatment patterns, and exacerbations among patients with asthma newly treated with omalizumab. METHODS: Data for this study were obtained from administrative claims and medical records. The index date was the date of the first claim for omalizumab. All patients had ≥12 months of continuous health plan eligibility before and after the index date. Demographic and clinical characteristics were obtained 12 months before the index date. Treatment patterns of asthma medications, including omalizumab, and asthma exacerbations were evaluated in the preindex and postindex periods. FINDINGS: The study included 1564 patients. Asthma-related medication use decreased from the preindex to the postindex periods (oral corticosteroids, 83.3%-66.4%, P < 0.001; inhaled corticosteroids [ICSs], 33.1%-26.8%, P < 0.001; long-acting ß2-adrenergic agonists [LABAs], 6.6%-5.2%, P = 0.009; ICS-LABA combination, 69.3%-64.3%, P < 0.001; leukotriene modifiers, 67.8%-59.7%, P < 0.001). The proportion of patients with any asthma exacerbations decreased by 33.6% (66.6%-44.2%, P < 0.001). Notably, the relative decreases in hospitalization and emergency department exacerbations were 79.3% and 72.2%, respectively. A total of 930 patients (59.5%) discontinued omalizumab treatment during the entire postindex period (maximum, 3400 days [approximately 9 years]), with 353 (38.0%) restarting omalizumab treatment. IMPLICATIONS: In this real-world analysis, patients newly initiating omalizumab therapy for allergic asthma used fewer concomitant asthma medications, while experiencing significant reductions in asthma exacerbations, especially hospitalization- and emergency department-specific exacerbations, from pre- to post-omalizumab treatment initiation periods.

Real-World Characteristics and Treatment Patterns in Patients with Urticaria Initiating Omalizumab in the United States.

BACKGROUND: Omalizumab is indicated for the management of chronic idiopathic urticaria (CIU) in patients aged 12 years or older with persistent hives that are not adequately controlled by H1 antihistamines. While its safety and efficacy in CIU patients have been evaluated in multiple clinical trials, real-world use of omalizaumab in CIU has not been well characterized. OBJECTIVE: To assess demographics, clinical characteristics, and treatment patterns of CIU patients who initiated omalizumab to better understand the usage of this agent in CIU management in the real world. METHODS: This retrospective cohort study used medical and pharmacy claims data in the United States from the HealthCore Integrated Database to identify patients with CIU newly treated with omalizumab (≥ 4 omalizumab claims within 6 months of the initial claim) between March 21, 2014, and October 31, 2015 (study intake period). The index date was defined as the date of the first claim for omalizumab during the study intake period. Demographic and clinical characteristics were described for patients treated with omalizumab, as were treatment patterns associated with omalizumab and concomitant medications associated with CIU treatment. Descriptive and inferential statistics were reported. The Kaplan-Meier method was used to examine omalizumab treatment patterns. RESULTS: This study included 298 omalizumab-treated patients (mean [SD] age of 43.5 [13.64] years; 70.8% female); approximately 84% were seen by an allergist/immunologist. All patients had ≥ 12 months of continuous enrolment and a subset of 138 patients had ≥ 18 months of follow-up. For patients with ≥ 12 months of post-index follow-up, 12.1% (n = 36), 28.5% (n = 85), and 32.9% (n = 98) discontinued omalizumab within the 6-month, 12-month, and the entire post-index periods (mean 530 days), respectively; the mean number of days patients were continuously treated with omalizumab was 443.1 (95% CI = 425.0-461.3); the probabilities of continuous treatment (95% CI) were 0.879 (0.836-0.911), 0.711 (0.656-0.759), and 0.647 (0.585-0.703) for the 6-, 12-, and 18-month post-index periods, respectively. For the 98 patients who discontinued omalizumab during the entire post-index period, 28.6% restarted omalizumab after the first discontinuation within the post-index period (mean time from first discontinuation to first restart=329 days). Use of medications such as oral corticosteroids, montelukast, cyclosporine, and prescription H1 and H2 antihistamines decreased during the 1- to 6-month and 7- to 12-month post-index periods compared with those within the 6-month pre-index period. CONCLUSIONS: In this cohort of CIU patients who were newly prescribed omalizumab, the majority were treated by allergists/immunologists as expected, and approximately 60% of patients continued on therapy beyond 18 months. Concomitant medication use decreased after omalizumab initiation. These data on the real-world use of omalizumab for CIU may help to better inform decision-making processes for health care payers by quantifying omalizumab and concomitant medication treatment patterns over a longer time frame relative to previous studies. DISCLOSURES: This study was sponsored by Novartis Pharmaceuticals, which provided funding support for the conduct of the study. Kavati, Ortiz, and Paknis are employees of Novartis Pharmaceuticals. Ke, Wertz, Huang, Wang, Willey, and Stephenson are employees of HealthCore, an independent research organization that received funding from Novartis Pharmaceuticals for the conduct of this study. Beck is an employee of the University of Rochester Medical Center, who was under contract with Novartis Pharmaceuticals to provide consulting services to this study, and reports grants from Genentech, outside the currently submitted work. Bernstein is affiliated with Bernstein Clinical Research Center, which was under contract with Novartis Pharmaceuticals to provide consulting services to this study, and reports receiving grants and personal fees from Novartis Pharmaceuticals, grants and personal fees from Genentech outside of the submitted work, and is an author on the Joint Task Force for Practice Parameters for Urticaria and the GALEN international guidelines for urticaria under preparation. Selected study data were presented in a poster at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 22nd Annual International Meeting on May 20-24, 2017, in Boston, MA. A poster based on this dataset was presented at the 2017 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting on October 26-30, 2017, in Boston, MA.

Non-adherence to basal insulin among patients with type 2 diabetes in a US managed care population: Results from a patient survey.

The aim of this study was to assess insulin non-adherence among patients with type 2 diabetes (T2DM) to better understand relationships between adherence, basal insulin (BI) usage, and patient experiences. A cross-sectional survey of patients with T2DM using BI was conducted. Adherence was measured by the Morisky Medication Adherence Scale 8-Items (MMAS-8). Low adherence (LA) was defined as MMAS-8 score < 6, high adherence (HA) as MMAS-8 score = 8, and medium adherence as MMAS-8 score = 6 to < 8. Patients with MMAS-8 scores = 6 to < 8 were excluded from the analysis. Of 400 completed surveys, 395 patients (98.8%) completed all MMAS-8 items, 112 with LA, 134 with HA. Compared with HA patients, greater proportions of LA patients followed more complex BI dosing patterns (57.1% vs. 39.5%, P = 0.014), had some difficulty calculating their correct BI dose (40.2% vs. 6.8%, P < 0.001), reported having missed ≥1 dose per month (79.3% vs. 12.6%, P < 0.001), and temporarily stopped BI in the past year (23.2% vs. 0.7%, P < 0.001). In conclusion, understanding patients' experiences with BI therapy can help formulate strategies to improve adherence.

Comparison of claims vs patient-reported adherence measures and associated outcomes among patients with nonvalvular atrial fibrillation using oral anticoagulant therapy.

OBJECTIVE: To compare oral anticoagulant (OAC) adherence among patients with nonvalvular atrial fibrillation (NVAF) using patient-reported and claims-based measures, and to evaluate the effect of OAC adherence on health care costs and patient satisfaction with OAC therapy. METHODS: This was a hybrid US observational study consisting of a longitudinal cohort survey followed by linkage and analysis of respondents' administrative claims data. Patients with NVAF receiving warfarin, dabigatran, rivaroxaban, or apixaban completed an initial survey and follow-up surveys at 4, 8, and 12 months. Patient-reported adherence was measured at each survey by Morisky Medication Adherence Scale (MMAS-8) and pharmacy claims-determined adherence by the proportion of days covered (PDC) for the 12-month period following the initial survey date; adherence was defined as MMAS-8 score =8 and PDC ≥80%. Patient satisfaction with OAC therapy was assessed by the Duke Anticoagulation Satisfaction Scale (DASS). RESULTS: Overall, 675 patients completed at least the initial survey (warfarin, n=271; dabigatran, n=266; rivaroxaban, n=128; apixaban, n=10). Fewer than half (47.9%) were PDC adherent, 37.2% were MMAS-8 adherent, and 19.4% were adherent by both measures. Total medical costs of PDC-adherent patients were significantly lower vs PDC-nonadherent patients (US$640 vs $993 per-patient per-month, respectively, p<0.05). MMAS-8-adherent patients reported higher treatment satisfaction; total DASS score was significantly lower among MMAS-8-adherent than MMAS-8-nonadherent patients (37.3 vs 42.9, respectively, p<0.001). CONCLUSION: Using claims-based or patient-reported methods to measure OAC adherence may lead to different results when assessing impact on health care costs and satisfaction with anticoagulation medication. These results underscore the importance of considering both claims-based and patient-reported measures when evaluating treatment adherence in real-world settings.

Real-world treatment patterns and outcomes of omalizumab use in patients with chronic idiopathic urticaria.

OBJECTIVE: To examine treatment patterns, treatment response, and demographic and clinical characteristics of patients with chronic idiopathic urticaria (CIU) newly initiated on omalizumab therapy in real-world practice in the US. METHODS: This retrospective observational cohort study used US claims data from the HealthCore Integrated Research Database (HIRD®) augmented with medical record data to identify CIU patients newly-treated with omalizumab (≥4 omalizumab claims within 6 months of initial claim; index date = first omalizumab claim date) between March 21, 2014 and October 31, 2015 and with ≥6 months pre- and ≥12 months post-index health plan eligibility. Study outcomes were captured from medical records for up to 12 months pre-index and up to 24 months post-index. Descriptive statistics were reported. RESULTS: This study consisted of 88 patients with a mean (SD) age of 43.4 (± 13.46) years, 68.2% were female, and 36 patients had ≥18 months post-index eligibility. Among 69 patients with documented index dose, 75.4% received omalizumab 300 mg and 69.6% remained on index dose throughout follow-up. For 52 patients with documented index dosing frequency, 96.2% had every 4-week dosing frequency. Among 86 patients with omalizumab documentation, 83.7% reported CIU improvement after omalizumab initiation and 24.4% discontinued omalizumab. For all patients, the proportion using oral corticosteroids (OCS) decreased pre- to post-index (52.3% vs 39.8%). Similar results were observed for patients with ≥18 months post-index eligibility. CONCLUSIONS: In this real-world analysis, the majority of patients remained on omalizumab for ≥12 months, and had a positive response. OCS use decreased following omalizumab initiation.

Clinical and economic burden of dyspnea and other COPD symptoms in a managed care setting.

PURPOSE: The degree to which symptoms such as dyspnea affect patients with COPD is individualized. To address the gap between clinical symptom measures and self-perceived disease burden, we investigated the symptom status of adult patients with COPD and followed with an administrative claims analysis of health care resource utilization and costs. METHODS: This was a hybrid US observational study consisting of a cross-sectional patient survey followed by a retrospective analysis of administrative claims data. The primary COPD symptom measures were the modified Medical Research Council (mMRC) Dyspnea scale and the COPD Assessment Test (CAT). RESULTS: A total of 673 patients completed the survey. Of these, 65% reported mMRC grades 0-1 (low symptomatology) and 35% reported mMRC grades 2-4 (high symptomatology); 25% reported CAT score <10 (low symptomatology) and 75% reported CAT score ≥10 (high symptomatology). More patients with high symptomatology (by either measure) had at least one COPD-related inpatient hospitalization, emergency room visit, physician office visit, or other outpatient services, and filled at least one COPD-related prescription medication vs patients with low symptomatology. COPD-related costs were higher for patients with high symptomatology than patients with low symptomatology. In a multivariate analysis, COPD-related costs were also higher in patients reporting severe symptoms. CONCLUSION: Patients with high COPD symptomatology utilized more health care resources and had higher COPD-related health care costs during the 6-month post-survey period than patients with low symptomatology.

Long-term natalizumab treatment is associated with sustained improvements in quality of life in patients with multiple sclerosis.

BACKGROUND: Multiple sclerosis (MS) patients experience lower health-related quality of life (HRQoL) than the general population. In clinical trials, natalizumab significantly improved HRQoL and reduced relapse rates and disability progression in patients with relapsing MS. In a 1-year analysis of patients included in the current study, HRQoL improvement occurred within 3 months of natalizumab initiation and continued for 1 year thereafter. However, natalizumab's long-term efficacy in improving HRQoL has not been studied. METHODS: In this longitudinal, observational, single-arm US study, HRQoL and treatment satisfaction were evaluated in MS patients receiving intravenous natalizumab 300 mg every 4 weeks in clinical settings. Patients completed surveys at baseline and every 6 months for 3 years and reported the following measures: Short Form-12 Version 2 (SF-12v2), Multiple Sclerosis Impact Scale (MSIS-29), and Treatment Satisfaction Questionnaire for Medication. RESULTS: In this study, 120 patients completed ≥3 years of natalizumab treatment. Significant HRQoL improvements were evident from baseline to year 3 by increases in SF-12v2 Physical Component Summary (PCS) and Mental Component Summary scores (P<0.01) and decreases in MSIS-29 physical and psychological scores (P<0.0001). Patients with less physical disability (baseline Disease Steps [DS] 0-2) had significant improvement from baseline to year 3 in SF-12v2 PCS (P<0.05) and MSIS-29 physical scores (P<0.05). Physical HRQoL outcomes in patients with baseline DS 3-6 remained stable over 3 years. Treatment satisfaction increased significantly from baseline to year 1 (P<0.0001) and was maintained in the following 2 years. CONCLUSION: Patients reported physical and psychological HRQoL improvements over 3 years of natalizumab treatment, supporting the long-term efficacy of natalizumab in real-world settings. Lower baseline disease activity and earlier treatment were related to better outcomes, indicating the importance of starting natalizumab early in the disease course. Treatment satisfaction increased after natalizumab initiation and remained high over 3 years of treatment.