Development of a Conceptual Model for the Patient Experience of Immunoglobulin A Nephropathy (IgAN): A Qualitative Literature Review.

INTRODUCTION: Immunoglobulin A nephropathy (IgAN) is a kidney disorder that can lead to progressive kidney disease. Currently, there lacks a comprehensive overview of the symptoms and impacts experienced by those living with IgAN that would help inform the selection or development of fit-for-purpose clinical outcome assessments (COA) to be used in clinical trials. The aim of this study was to develop a conceptual model of the adult and pediatric patient experience of IgAN, including disease signs and symptoms, treatment side effects, and impact on functioning and well-being. METHODS: This study comprised a systematic review and thematic analysis of qualitative studies with adults and children diagnosed with IgAN. Data sources were identified through an electronic database search of journal articles (MEDLINE, Embase, PsycINFO; June 2021), hand-searching of conference proceedings, patient advocacy group websites, and gray literature. Non-English articles were excluded. Identified data (patient/caregiver quotes, author summaries, and interpretations of patient experiences) were extracted from articles. Extracted data were qualitatively analyzed, aided by ATLAS.ti v7. Codes were applied to data; concepts (i.e., symptoms) were identified, named, and refined. A conceptual model was developed by grouping related concepts into domains. RESULTS: In total, five sources were identified for analysis: two journal articles, two online anthologies of patient stories, and one patient organization-sponsored "Voice of the Patient" meeting report. Conceptual model symptom domains included swelling/puffiness (edema), pain/aches/discomfort, fatigue, weight gain, sleep problems, urinary problems, and gastrointestinal problems. Impact domains included emotional/psychological well-being, physical functioning/activities of daily living, social functioning, work/school, and relationships. CONCLUSIONS: Secondary analysis of published qualitative literature permitted development of a novel conceptual model depicting the patient experience of IgAN; however, its depth is limited by a lack of available literature. Further qualitative research is recommended to refine and/or confirm the concepts and domains, determine any relationships between them, and explore the outcomes that are most meaningful to patients. The refined model will provide a useful tool to inform the selection, development, and/or amendment of COAs for use in future IgAN clinical trials.

Development of a Conceptual Model for the Patient Experience of Focal Segmental Glomerulosclerosis (FSGS): A Qualitative Targeted Literature Review.

INTRODUCTION: Focal segmental glomerulosclerosis (FSGS) is a leading cause of kidney disease and can progress to end stage kidney disease (ESKD). An overview of symptoms and impacts of the disease experienced will help inform the selection or development of fit-for-purpose clinical outcome assessments (COA) to be used in FSGS clinical trials. This study aimed to develop a conceptual model (CM) of the adult and pediatric patient experience of FSGS including disease signs/symptoms, treatment side-effects, and impact on functioning and wellbeing. METHODS: This study comprised a systematic review and thematic analysis of qualitative studies with adults and pediatric patients diagnosed with FSGS. Data sources were identified through an electronic database search of journal articles (Medline, Embase, PsycINFO; June 2021) and hand-searching of conference proceedings, patient advocacy group websites, and gray literature. Non-English articles were excluded. Identified data (patient/caregiver quotes, author summaries, and interpretations of patient experiences) were extracted from the articles. Extracted data were qualitatively analyzed aided by ATLAS.ti v7. Codes were applied to data and concepts (symptoms/impacts) were identified, named, and refined. A CM was developed by grouping related concepts into domains. RESULTS: In total, 12 sources were identified for analysis: 6 journal articles and 6 series of patient testimonials. Salient sign/symptom/side-effect domains included swelling/puffiness (edema), pain/aches/discomfort, fatigue, weight changes, skin problems, respiratory problems, and sleep problems. Salient impact domains included emotional/psychological wellbeing, physical functioning/activities of daily living, social functioning, and work/school. CONCLUSION: Secondary analysis of published qualitative literature permitted development of a CM describing the adult and pediatric experience of FSGS. Concept elicitation interviews are recommended to refine the CM, confirm the salient/most bothersome concepts, and confirm the extent of impact on daily life. The refined CM will provide a useful tool to inform the selection, development, and/or amendment of COAs for use in future FSGS clinical trials.

Key measurement concepts and appropriate clinical outcome assessments in pediatric achondroplasia clinical trials.

BACKGROUND: This study aimed to identify fit-for-purpose clinical outcome assessments (COAs) to evaluate physical function, as well as social and emotional well-being in clinical trials enrolling a pediatric population with achondroplasia. Qualitative interviews lasting up to 90 min were conducted in the US with children/adolescents with achondroplasia and/or their caregivers. Interviews utilized concept elicitation methodology to explore experiences and priorities for treatment outcomes. Cognitive debriefing methodology explored relevance and understanding of selected COAs. RESULTS: Interviews (N = 36) were conducted with caregivers of children age 0-2 years (n = 8) and 3-7 years (n = 7) and child/caregiver dyads with children age 8-11 years (n = 15) and 12-17 years (n = 6). Children/caregivers identified pain, short stature, impacts on physical functioning, and impacts on well-being (e.g. negative attention/comments) as key bothersome aspects of achondroplasia. Caregivers considered an increase in height (n = 9/14, 64%) and an improvement in limb proportion (n = 11/14, 71%) as successful treatment outcomes. The Childhood Health Assessment Questionnaire (CHAQ) and Quality of Life in Short Stature Youth (QoLISSY-Brief) were cognitively debriefed. CHAQ items evaluating activities, reaching, and hygiene were most relevant. QoLISSY-Brief items evaluating reaching, height bother, being treated differently, and height preventing doing things others could were most relevant. The CHAQ and QoLISSY-Brief instructions, item wording, response scales/options and recall period were well understood by caregivers and adolescents age 12-17. Some children aged 8-11 had difficulty reading, understanding, or required caregiver input. Feedback informed minor amendments to the CHAQ and the addition of a 7-day recall period to the QoLISSY-Brief. These amendments were subsequently reviewed and confirmed in N = 12 interviews with caregivers of children age 0-11 (n = 9) and adolescents age 12-17 (n = 3). CONCLUSIONS: Achondroplasia impacts physical functioning and emotional/social well-being. An increase in height and improvement in limb proportion are considered to be important treatment outcomes, but children/adolescents and their caregivers expect that a successful treatment should also improve important functional outcomes such as reach. The CHAQ (adapted for achondroplasia) and QoLISSY-Brief are relevant and appropriate measures of physical function and emotional/social well-being for pediatric achondroplasia trials; patient-report is recommended for age 12-17 years and caregiver-report is recommended for age 0-11 years.

Validation of patient-reported vaso-occlusive crisis day as an endpoint in sickle cell disease studies.

Individuals with sickle cell disease (SCD) experience vaso-occlusive crises (VOC). Historically, VOC episodes have been assessed through medical utilization, thereby excluding events managed at home. In order to validate a daily patient-reported outcome for patients with SCD to accurately report their VOC status and experience of a pain crisis, a SCD Diary was included in Evaluation of Longitudinal Pain Study in Sickle Cell Disease (ELIPSIS), a longitudinal, six-month, non-interventional study. The daily patient-completed diary included a description of SCD pain crisis, followed by questions on: pain crisis in the past 24 h (VOC Day question; respective response yes or no), worst pain, tiredness, and functioning. Thirty-five patients with SCD participated in ELIPSIS. Analyses were performed to validate the patient-reported VOC Day. Mean symptoms and functioning scores on the first or last VOC Day of a VOC Event were compared using t-tests with the mean of the three non-VOC Days before and after the event. Mean severity of symptoms and functioning scores on all VOC Days compared to all non-VOC Days were higher, with statistically significant mean differences between first/last VOC Days and respective three non-VOC Days (p's < .01). A subset of patients (n = 15) and caregivers (n = 9) were interviewed to evaluate their understanding of the SCD Diary questions. Nearly all confirmed that the pain crisis description accurately described the VOC experience, and participants expressed confidence differentiating SCD crisis pain from everyday pain. These results demonstrate patients can reliably report their experiences with VOC-related pain crises using the SCD Diary.

A Retrospective Study of Clinical and Economic Burden of Focal Segmental Glomerulosclerosis (FSGS) in the United States.

INTRODUCTION: Information on the economic burden of focal segmental glomerulosclerosis (FSGS) is sparse. This study characterized health care resource utilization (HCRU) and costs in patients with FSGS, and evaluated the impact of nephrotic range proteinuria on these outcomes. METHODS: This retrospective, observational cohort study used administrative claims data from the Optum Clinformatics Data Mart Database from October 2015 to December 2019. Patients with FSGS (n = 844; first claim = index event) between April 2016 and December 2018 were matched on index date, age, sex, and race to non-FSGS controls (n = 1688). FSGS nephrotic range (urine protein/creatinine ratio >3000 mg/g or albumin/creatinine ratio >2000 mg/g) and non-nephrotic subpopulations were identified. Baseline comorbidities, 12-month post-index all-cause HCRU and costs (per patient per year [PPPY]), and immunosuppressant prescriptions were compared between matched cohorts and between FSGS subpopulations. RESULTS: Comorbidity burden was higher in FSGS. Of 308 patients with available urine protein/creatinine ratio/albumin/creatinine ratio results, 36.4% were in nephrotic range. All-cause HCRU was higher in FSGS across resource categories (all P < 0.0001); 50.6% of FSGS and 23.3% of controls were prescribed glucocorticoids (P < 0.0001). Mean total medical costs were higher in FSGS ($59,753 vs. $8431 PPPY; P < 0.0001), driven by outpatient costs. Nephrotic range proteinuria was associated with higher all-cause inpatient, outpatient, and prescription costs versus nonnephrotic patients (all P < 0.0001), resulting in higher total costs ($70,481 vs. $36,099 PPPY; P < 0.0001). CONCLUSIONS: FSGS is associated with significant clinical and economic burdens; the presence of nephrotic range proteinuria increased the economic burden. New treatment modalities are needed to reduce proteinuria, help improve patient outcomes, and reduce HCRU and associated costs.

Comparison of readmissions among hospitalized nonvalvular atrial fibrillation patients treated with oral anticoagulants in the United States.

Objectives: To compare the risks of 1-month all-cause, major bleeding (MB)-related and stroke-related readmissions and the associated hospital resource use and costs among patients previously hospitalized for nonvalvular atrial fibrillation (NVAF) and treated with warfarin, rivaroxaban, and dabigatran vs apixaban. Methods: Adult patients hospitalized with NVAF (any discharge diagnosis position) who received apixaban, warfarin, rivaroxaban, or dabigatran during hospitalization were identified from the Premier database (1 January 2013-30 June 2017) and grouped into respective cohorts. Propensity score matching was used to generate cohorts with similar characteristics. In regression analyses the risk of readmissions that occurred within 1 month of discharge were evaluated and the associated length of stay (LOS) and costs compared. Results: NVAF patients treated with warfarin vs apixaban had significantly greater risk of all-cause (odds ratio [OR] = 1.05; confidence interval [CI] = 1.02-1.08; p < .001), MB-related (OR: 1.28; CI: 1.16-1.42; p < .001), and stroke-related (OR: 1.33; CI: 1.11-1.58; p = .002) readmissions; for all readmission categories, average LOS was significantly longer and costs significantly higher for warfarin treated patients. NVAF patients treated with rivaroxaban versus apixaban had significantly greater risk of all-cause (OR: 1.06; CI: 1.02-1.09; p = .001) and MB-related (OR = 1.62; CI = 1.44-1.83; p < .001) readmissions, but not stroke-related readmission; for MB-related readmissions average LOS and costs were higher for rivaroxaban treated patients. Significant differences in risks of all-cause, MB-related, and stroke-related readmissions were not observed between the apixaban and dabigatran cohorts. Conclusion: In this retrospective real-world analysis of NVAF patients, apixaban treatment was associated with better clinical outcomes than warfarin or rivaroxaban and lower hospital resource burden.

Validation of obesity coding among newly treated nonvalvular atrial fibrillation patients using an integrated electronic medical record and claims database.

Objective: To evaluate the validity of diagnosis codes for identifying obesity and morbid obesity among newly treated nonvalvular atrial fibrillation (NVAF) patients.Methods: An integrated electronic medical record (EMR) and claims database (1 January 2013-31 March 2018) was used. Adult patients with ≥1 claim for an oral anticoagulant (OAC) from 1 January 2014-30 September 2017 were identified (index date). Patients were required to have ≥1 atrial fibrillation diagnosis, no OAC use or valvular disease during the 12 months before index date, ≥12 months of continuous enrollment before and ≥6 months after index date, and ≥1 BMI measurement 6 months before or after index date. Patients with BMI ≥30 kg/m2 and BMI ≥40 kg/m2 were classified as obese and morbidly obese, respectively. Sensitivity, specificity and positive predictive value (PPV) were calculated to assess the validity of diagnosis codes for obesity and morbid obesity.Results: A total of 7501 patients met all selection criteria. Forty-six percent of patients had BMI ≥ 30 kg/m2, of whom about one-quarter had a BMI ≥ 40 kg/m2. Twenty-five percent and 10% of patients had a diagnosis code for obesity or morbid obesity, respectively. Sensitivity, specificity and PPV for obesity diagnosis codes were 48.67% (95% CI: 47.00%-50.35%), 95.24% (94.54%-95.88%) and 89.78% (88.32%-91.12%), respectively, and 62.75% (59.30%-66.11%), 96.46% (95.99%-96.89%) and 67.93% (64.43%-71.29%) for morbid obesity diagnosis codes, respectively.Conclusion: Among newly treated NVAF patients, obesity diagnosis codes had high PPV, high specificity and modest sensitivity. Morbid obesity diagnosis codes also had high specificity, but modest PPV and sensitivity. These findings have implications for case selection and control for obesity as a confounder in studies using a claims database.

Switching to Another Oral Anticoagulant and Drug Discontinuation Among Elderly Patients With Nonvalvular Atrial Fibrillation Treated With Different Direct Oral Anticoagulants.

We compared the risks of switching to another oral anticoagulant (OAC) and discontinuation of direct oral anticoagulants (DOACs) among elderly patients with nonvalvular atrial fibrillation (NVAF) who were prescribed rivaroxaban or dabigatran versus apixaban. Patients (≥65 years of age) with NVAF prescribed DOACs (January 1, 2013 to September 30, 2017) were identified from the Humana research database and grouped into DOAC cohorts. Cox regression analyses were used to evaluate whether the risk for switching to another OAC or discontinuing index DOACs differed among cohorts. Of the study population (N = 38 250), 55.9% were prescribed apixaban (mean age: 78.6 years; 49.8% female), 37.3% rivaroxaban (mean age: 77.4 years; 46.7% female), and 6.8% dabigatran (mean age: 77.0 years; 44.0% female). Compared to patients prescribed apixaban, patients prescribed rivaroxaban (hazard ratio [HR]: 2.08; 95% confidence interval [CI], 1.92-2.25; P < .001) or dabigatran (HR: 3.74; 95% CI, 3.35-4.18, P < .001) had a significantly higher risk of switching to another OAC during the follow-up; compared to patients prescribed apixaban, the risks of discontinuation were also higher for patients treated with rivaroxaban (HR: 1.10; 95% CI, 1.07-1.13, P < .001) or dabigatran (HR: 1.29; 95% CI, 1.23-1.35, P < .001).

Continuation with apixaban treatment is associated with lower risk for hospitalization and medical costs among elderly patients.

Objective: To compare the risk of hospitalization and costs associated with major bleeding (MB) or stroke/systemic embolism (SE) among elderly patients with nonvalvular atrial fibrillation (NVAF) who initiated apixaban then switched to another oral anticoagulant (OAC) vs. those who continued with apixaban treatment. Methods: NVAF patients (≥65 years) initiating apixaban were identified from the Humana database (1 January 2013-30 September 2017) and grouped into switcher and continuer cohorts. For switchers, the earliest switch from apixaban to another OAC was defined as the index event/date. A random date during apixaban treatment was selected as the index date for continuers. Patients were followed from index date to health plan disenrollment or 31 December 2017, whichever was earlier. Multivariable regression analyses were used to examine the association of switchers vs. continuers with risk of MB-related or stroke/SE-related hospitalization and healthcare costs during follow-up. Results: Of 7858 elderly NVAF patients included in the study, 14% (N = 1110; mean age: 78 years) were switchers; 86% (N = 6748; mean age: 79 years) were continuers. Apixaban switchers vs. continuers had significantly greater risk of MB-related hospitalization (hazard ratio [HR]: 2.00; 95% CI: 1.52-2.64; p < .001) during follow-up; risk of stroke/SE hospitalization did not differ significantly (HR: 1.36, 95% CI: 0.89-2.06, p = .154). MB- and stroke/SE-related medical costs were higher for switchers vs. continuers, although total all-cause healthcare costs were similar. Conclusion: Elderly patients with NVAF in the US who continued with apixaban treatment had a lower risk of MB-related hospitalization and lower MB- and stroke/SE-related medical costs compared to patients who switched to another OAC.

Comparative clinical outcomes between direct oral anticoagulants and warfarin among elderly patients with non-valvular atrial fibrillation in the CMS medicare population.

Atrial fibrillation (AF) prevalence increases with age; > 80% of US adults with AF are aged ≥ 65 years. Compare the risk of stroke/systemic embolism (SE), major bleeding (MB), net clinical outcome (NCO), and major adverse cardiac events (MACE) among elderly non-valvular AF (NVAF) Medicare patients prescribed direct oral anticoagulants (DOACs) VS warfarin. NVAF patients aged ≥ 65 years who initiated DOACs (apixaban, dabigatran, and rivaroxaban) or warfarin were selected from 01JAN2013-31DEC2015 in CMS Medicare data. Propensity score matching was used to balance DOAC and warfarin cohorts. Cox proportional hazards models estimated the risk of stroke/SE, MB, NCO, and MACE. 37,525 apixaban-warfarin, 18,131 dabigatran-warfarin, and 55,359 rivaroxaban-warfarin pairs were included. Compared to warfarin, apixaban (HR: 0.69; 95% CI 0.59-0.81) and rivaroxaban (HR: 0.82; 95% CI 0.73-0.91) had lower risk of stroke/SE, and dabigatran (HR: 0.88; 95% CI 0.72-1.07) had similar risk of stroke/SE. Apixaban (MB: HR: 0.61; 95% CI 0.57-0.67; NCO: HR: 0.64; 95% CI 0.60-0.69) and dabigatran (MB: HR: 0.79; 95% CI 0.71-0.89; NCO: HR: 0.84; 95% CI 0.76-0.93) had lower risk of MB and NCO, and rivaroxaban had higher risk of MB (HR: 1.08; 95% CI 1.02-1.14) and similar risk of NCO (HR: 1.04; 95% CI 0.99-1.09). Compared to warfarin, apixaban had a lower risk for stroke/SE, MB, and NCO; dabigatran had a lower risk of MB and NCO; and rivaroxaban had a lower risk of stroke/SE but higher risk of MB. All DOACs had lower risk of MACE compared to warfarin.

An empirical tool for estimating the share of unmet need due to healthcare inefficiencies, suboptimal access, and lack of effective technologies.

BACKGROUND: Although there has been growing attention to the measurement of unmet need, which is the overall epidemiological burden of disease, current measures ignore the burden that could be eliminated from technological advances or more effective use of current technologies. METHODS: We developed a conceptual framework and empirical tool that separates unmet need from met need and subcategorizes the causes of unmet need into suboptimal access to and ineffective use of current technologies and lack of current technologies. Statistical models were used to model the relationship between health-related quality of life (HR-QOL) and treatment utilization using data from the National Health and Wellness Survey (NHWS). Predicted HR-QOL was combined with prevalence data from the Global Burden of Disease Study (GBD) to estimate met need and the causes of unmet need due to morbidity in the US and EU5 for five diseases: rheumatoid arthritis, breast cancer, Parkinson's disease, hepatitis C, and chronic obstructive pulmonary disease (COPD). RESULTS: HR-QOL was positively correlated with adherence to medication and patient-perceived quality and negatively correlated with financial barriers. Met need was substantial across all disease and regions, although significant unmet need remains. While the majority of unmet need was driven by lack of technologies rather than ineffective use of current technologies, there was considerable variation across diseases and regions. Overall unmet need was largest for COPD, which had the highest prevalence of all diseases in this study. CONCLUSION: We developed a methodology that can inform decisions about which diseases to invest in and whether those investments should focus on improving access to currently available technologies or inventing new technologies.

Comparison of Drug Switching and Discontinuation Rates in Patients with Nonvalvular Atrial Fibrillation Treated with Direct Oral Anticoagulants in the United States.

INTRODUCTION: Continuous usage of direct oral anticoagulants (DOACs) among nonvalvular atrial fibrillation (NVAF) patients is essential to maintain stroke prevention. We examined switching and discontinuation rates for the three most frequently initiated DOACs in NVAF patients in the USA. METHODS: Patients who initiated apixaban, rivaroxaban, or dabigatran (index event/date) were identified from the Pharmetrics Plus claims database (Jan 1, 2013-Sep 30, 2016, includes patients with commercial and Medicare coverage) and grouped into cohorts by index DOAC. Patients were required to have a diagnosis of NVAF and continuous health plan enrollment for 12 months prior to the index date (baseline period) and at least 3 months during the follow-up period. Drug switching rates to any other DOAC or warfarin and index DOAC discontinuation rate were evaluated separately with descriptive statistics, Kaplan-Meier analysis, and multivariable Cox regression analysis. RESULTS: Of the NVAF study population (n = 41,864), 37% initiated apixaban (n = 15,352; mean age 62 years), 51% initiated rivaroxaban (n = 21,250; mean age 61 years), and 13% initiated dabigatran (n = 5262; mean age 61 years). During the follow-up period, the unadjusted drug switching rates of patients treated with apixaban, rivaroxaban, and dabigatran were 3.6%, 6.3%, and 11.1%, respectively (p < 0.001 across the three cohorts); while the index DOAC discontinuation rates were 52.8%, 60.3%, and 62.9%, respectively (p < 0.001). After we controlled for differences in patient characteristics, patients treated with rivaroxaban (HR 1.8; 95% CI 1.6-2.0; p < 0.001) and dabigatran (HR 3.4; 95% CI 3.0-3.8, p < 0.001) had a significantly greater likelihood for drug switching than patients treated with apixaban. Also, both rivaroxaban (HR 1.1; 95% CI 1.1-1.2, p < 0.001) and dabigatran (HR 1.3; 95% CI 1.2-1.3, p < 0.001) treated patients were more likely to discontinue treatment. CONCLUSION: In the real-world setting, patients with NVAF newly treated with apixaban were less likely to switch or discontinue treatment compared to patients treated with rivaroxaban or dabigatran. FUNDING: Pfizer and Bristol-Myers Squibb.

A cost-benefit analysis of smoking cessation prescription coverage from a US payer perspective.

INTRODUCTION: Smoking drives substantial direct health care spending, comprising 8.7% ($168 billion) of annual United States aggregated spending. Smoking cessation (SC) prescription use is an effective strategy to improve health outcomes, increase quit rates, and reduce economic burden. However, patient out-of-pocket costs may limit the use. Health care payers play a vital role in driving use through formulary decisions and copayment policies but must consider both the near-term financial investment as well as downstream effects of increased coverage on health care budgets. This study estimates the return on investment (ROI) of providing Affordable Care Act (ACA)-recommended prescription SC coverage. METHODS: A cost-benefit analysis (CBA) estimates the ROI of providing prescription SC coverage, based on pharmacy costs and savings from smoking-attributable medical expenditures among Medicare, Medicaid, and commercial plan enrollees over 10 years. The CBA incorporated national-level population demographics, smoking prevalence estimates, proportion of smokers attempting to quit, and the utilization of SC products. A five-state Markov chain model simulated patterns of quit attempts, relapse, and cessation assuming two quit attempts per year, no patient cost-sharing, and 25.4% utilization of prescription SC aids. Results include number of quitters, annual pharmacy and smoking-attributable medical costs, and ROI. RESULTS: After initial investment in SC treatment, smoking-attributable medical benefits accrue over time, generating a positive ROI by year 4 for commercial (11.3%) and Medicaid (78.4%) plans and by year 3 for Medicare (30.6%). Over 10 years, an average return of $1.18, $2.50, and $3.22 savings per dollar spent on SC prescriptions for commercial, Medicaid, and Medicare plans, respectively, may be realized. DISCUSSION: Given the proven efficacy of SC pharmacotherapy, near-term investments in supporting ACA-recommended SC coverage translate into a positive ROI. As smoking is a leading cause of morbidity and mortality, increased access to prescription SC medications may improve health outcomes and reduce smoking-attributable costs to payers over time.

Mobile App Usage Patterns of Patients Prescribed a Smoking Cessation Medicine: Prospective Observational Study.

BACKGROUND: Cigarette smoking is the leading preventable cause of death and is responsible for more than 480,000 deaths per year in the United States. Smoking cessation is challenging for many patients. Regardless of available treatment options, most quit attempts are unaided, and it takes multiple attempts before a patient is successful. With the ever-increasing use of smartphones, mobile apps hold promise in supporting cessation efforts. This study evaluates the ease of use and user satisfaction with the Pfizer Meds app to support smoking cessation among patients prescribed varenicline (Chantix). OBJECTIVE: Study participants included varenicline users who downloaded and used the app on their personal smartphone. The main objectives were to report mobile app download frequency and usage details and to describe the participant-reported satisfaction with and usefulness of the app over the 14-week follow-up study period. METHODS: Adults aged 18 years or older who had been prescribed varenicline were identified from the Express Scripts Incorporated pharmacy claims database. After meeting privacy restrictions, subjects were sent an invitation letter and second reminder letter with instructions on how to download the Pfizer Meds mobile app. Participants received a push notification to complete a smartphone-enabled survey regarding the utility of the app 12 weeks after downloading the app. Descriptive statistics summarized sociodemographics, use of varenicline, and details of use and satisfaction with the mobile app. RESULTS: Of the 38,129 varenicline users who were sent invitation letters, 1281 participants (3.35%) downloaded the Pfizer Meds app. Of the 1032 users with demographic and other data, 585 (56.68%) were females, and 446 (43.22%) were males; mean age was 46.4 years (SD 10.8). The mean number of app sessions per participant was 4.0 (SD 6.8). The end-of-study survey was completed by 131 survey respondents (10.23%, 131/1281); a large number of participants (117/131, 89.3%) reported being extremely, very, or moderately satisfied with the app. A total of 97 survey respondents (97/131, 74.0%) reported setting up a quit date in the app. Of those, 74 (74/97, 76%) reported quitting on their quit date. CONCLUSIONS: Positive patient engagement was observed in this study based on app download and usage. This study quantified how the Pfizer Meds app performed in an observational real-world data setting. The findings demonstrate the willingness of participants to set a quit date and use the app for support in medication adherence, refill reminders, and information regarding how to take the medication. This study provides real-world evidence of the contribution apps can make to the continued encouragement of smokers to improve their health by smoking cessation.

A cost-effectiveness analysis of varenicline for smoking cessation using data from the EAGLES trial.

BACKGROUND: The cost-effectiveness of varenicline has been demonstrated in the US health care setting using the Benefits of Smoking Cessation on Outcomes (BENESCO) model to simulate the lifetime direct costs and consequences of a hypothetical cohort of US adult smokers who make a single attempt to quit. The aim of this study was to undertake an updated cost-effectiveness analysis, using current epidemiology inputs and recently published smoking cessation data from the Evaluating Adverse Events in a Global Smoking Cessation Study (EAGLES), the largest clinical trial of smoking cessation pharmacotherapies conducted to date. METHODS: BENESCO is a Markov model simulating the effect of a single attempt to quit smoking on four smoking-related diseases: coronary heart disease, stroke, chronic obstructive lung disease, and lung cancer. Inputs were updated to include efficacy from EAGLES and newer data for the epidemiology of smoking in the US, the epidemiology and direct treatment costs of the four morbidities, and the costs of the interventions. Analyses compared varenicline, bupropion, nicotine replacement therapy (NRT) patch, and placebo with regard to the incidence of smoking-related morbidity, the incidence of smoking-related mortality, and cost-effectiveness at a time horizon from 2 years to lifetime. RESULTS: The study cohort comprised of 18,394,068 US adult smokers who made a single quit attempt during the first year of the model. For varenicline, there were an estimated 319,730 fewer smoking-related morbidities at the lifetime compared with placebo. Similarly, smoking-related mortality decreased by 198,240 subjects when varenicline was compared with placebo. For the same time horizon, varenicline was more effective and less costly, ie, dominant, compared with all comparators in the cost-effectiveness analysis. CONCLUSION: Based on the BENESCO model, smoking cessation with varenicline results in reduced incidence of smoking-related morbidity and mortality compared with other smoking cessation interventions and remains a cost-effective strategy in the US population.

Smoking Cessation Is Associated With Lower Indirect Costs.

OBJECTIVE: This study quantified differences in indirect costs due to decreased work productivity between current and former smokers. Former smokers were further categorized by number of years since quitting to assess corresponding differences. METHODS: Data on employed individuals were obtained from the 2013 US National Health and Wellness Survey (NHWS; N = 75,000). Indirect costs were calculated for current smokers and former smokers from weekly wages based on age and sex. RESULTS: The annual total indirect costs for current smokers were $1327.53, $1560.18, and $1839.87 higher than for those who quit 0 to 4 years, 5 to 10 years, and more than or equal to 11 years prior, respectively. There were no significant differences in mean total indirect costs between the former smoker groups. CONCLUSIONS: Current smokers showed significantly higher total annual indirect costs compared with former smokers, independently of the number of years since quitting smoking.

Estimating the public economic consequences of introducing varenicline smoking cessation therapy in South Korea using a fiscal analytic framework.

BACKGROUND AND AIMS: Smoking gives rise to many cross-sectorial public costs and benefits for government. Costs arise from increased healthcare spending and work-related social benefits, while smoking itself provides significant revenue for government from tobacco taxes. To better understand the public economic impact of smoking and smoking cessation therapies, this study developed a government perspective framework for assessing smoking-attributable morbidity and mortality and associated public costs. This framework includes changes in lifetime tax revenue and health costs, as well as changes in tobacco tax revenue, from fewer smokers. METHODS: A modified generational accounting framework was developed to assess relationships between smoking-attributable morbidity and mortality and public economic consequences of smoking, including lifetime tax revenue gains/losses, government social transfers, and health spending. Based on the current prevalence of smoking in South Korean males, a cohort model was developed for smokers, former-smokers, and never-smokers. The model simulated the lifetime discounted fiscal transfers for different age cohorts in 5 year age bands, and the return on investment (ROI) from smoking cessation therapy. RESULTS: Former smokers are estimated to generate higher lifetime earnings and direct tax revenues and lower lifetime healthcare costs due to the reduction of smoking-attributable mortality and morbidity compared to smokers, even after accounting for reduced tobacco taxes paid. Based on the costs of public investments in varenicline, this study estimated a ROI from 1.4-1.7, depending on treatment age, with higher ROI in younger cohorts, with an average ROI of 1.6 for those aged less than 65. CONCLUSIONS: This analysis suggests that reductions in smoking can generate positive public economic benefits for government, even after accounting for lost tobacco tax revenues. The results described here are likely applicable to countries having similar underlying smoking prevalence, comparable taxation rates, and social benefit protection provided to individuals with smoking-related conditions.

Content Validity of a Willingness to Quit Tool for Use with Current Smokers in Clinical Practice.

INTRODUCTION: Despite reductions in rates of smoking in the past decade, smoking remains one of the most significant public health concerns. Quitting smoking can result in reductions in a number of serious health conditions. The brief Willingness to Quit (WTQ) tool can be used in routine clinical practice to assess current willingness to quit and engage a patient-physician dialogue regarding smoking cessation. The overall aim of this study was to validate the content of a WTQ tool for use with current smokers in clinical practice. METHODS: In-depth, qualitative interviews were conducted with 12 current smokers and five physicians. The interview was divided into two sections: concept elicitation (CE) followed by cognitive debriefing (CD). During CE, participants were asked questions exploring the different factors that can impact an individual's willingness to quit smoking. During CD, participants were given a copy of the WTQ tool and asked to comment on their level of understanding and interpretability of the items and the feasibility of completing the tool in clinical practice. RESULTS: All of the current smokers (n = 12) and physicians (n = 5) interviewed indicated that the items were understandable and relevant to assess willingness to quit. The tool was considered simple and suitable for use in clinical practice. CONCLUSION: The WTQ tool is a brief tool to assess willingness to quit and to engage communication between patients and physicians. All smokers should be offered smoking cessation support and facilitating a discussion on willingness to quit further supports a personalized quit plan. FUNDING: Pfizer Inc.

Characteristics of first-time varenicline users - A cross-sectional study in Finnish quitters.

BACKGROUND: Varenicline is an efficacious medicine for smoking cessation (SC) but little is known about the characteristics of varenicline users. This study examined the characteristics of first-time (naïve) varenicline users in Finland and compared those who had previously used SC pharmacotherapy to those who were trying SC pharmacotherapy for the first time. METHODS: A cross-sectional survey was conducted in Finnish community pharmacies between February 2014 and January 2015. Pharmacy customers purchasing a varenicline starter package for the first time ever were asked to complete a questionnaire or to participate in a structured interview conducted by the pharmacist (identical questions). The questionnaire included questions about demographic characteristics, smoking habits, previous cessation attempts and factors associated with varenicline use. RESULTS: Altogether 98 people completed the survey. The majority were daily smokers (96%, n = 94), with a history of over 10 years of regular smoking (94%, n = 92), and a strong/very strong nicotine dependence (67%, n = 66). Half of the participants (54%, n = 53) were trying a SC pharmacotherapy for the first time. Demographic characteristics and smoking habits were similar between first-time and previous users of SC medications (p > 0.05). Health centers (42%, n = 41) and occupational health care clinics (37%, n = 36) were the most common sources of varenicline prescriptions. The majority of participants received the prescription for varenicline after mentioning their desire for quitting to a physician (70%, n = 69). CONCLUSIONS: Considering the relatively large proportion of SC naïve medicine users among new users of varenicline, smokers who have previously been reluctant to quit smoking, to use other pharmacological SC interventions, or perhaps unaware of these options may be interested in attempting cessation with varenicline. Most participants made the initiative to discuss their smoking with the physician, which led to varenicline prescribing. This suggests that physicians may not satisfactorily recognize their patients' nicotine dependence and desire to quit, and they should more actively support patients' smoking cessation.