Exploring the Experience of Living with and Managing Presbyopia.

SIGNIFICANCE: Presbyopia typically occurs around 40 years of age and affects approximately one-quarter of the global population. Up to October 2021, there were no approved pharmacotherapies for presbyopia, and common treatments, such as glasses, can have disadvantages for individuals' health-related quality of life. PURPOSE: This study aimed to document the experience of living with and managing presbyopia, identify perspectives on treatment options, and determine whether there is an unmet need in the treatment landscape. METHODS: Coded transcripts of concept elicitation (CE; n = 20) and cognitive debriefing (n = 20) interviews with presbyopic individuals, originally conducted for development of patient-reported outcome instruments, were reanalyzed to identify salient concepts describing participants' experiences with presbyopia treatments. Qualitative ranking exercises assessed participants' preferences for a potential pharmacotherapy vs. existing treatments. RESULTS: Because most concepts were identified with the CE interviews, data reflect CE findings unless otherwise noted. Average age across CE/cognitive debriefing interviews was 49.4 years; a vast majority of participants used glasses for presbyopia treatment. Four themes related to treatment with glasses were identified with the interviews: inconvenience during daily activities, negative physical sensations around the eyes/head, limitations, and undesirable impacts on daily life (e.g., psychosocial). Most commonly, participants reported inconveniences related to forgetting glasses and psychosocial impacts (e.g., feeling/looking older). Strained/tired eyes and limited ability to see at varying distances were also reported. Among participants with near-vision glasses who provided data, two-thirds expressed interest in alternative treatments. In addition, almost three-quarters of the participants ranked hypothetical eye drops as their first or second preferred option, vs. reading glasses, contact lenses, magnifying glasses, and surgery. CONCLUSIONS: This study explored the experience of living with and managing presbyopia and identified limitations and negative impacts of current treatments. Pharmacological development (e.g., eye drops) may fulfill an unmet need in the presbyopia treatment landscape.

Healthcare Resource Utilization and Costs in Patients with Geographic Atrophy Secondary to Age-Related Macular Degeneration.

PURPOSE: Geographic atrophy (GA) is an advanced form of nonexudative age-related macular degeneration (AMD) that lacks treatment options. With considerable interpatient variability in the rate of GA progression due to lesion characteristics, information characterizing the disease burden is limited. The aim of this study was to describe the healthcare resource utilization (HCRU) and costs associated with increasing severity levels of GA. PATIENTS AND METHODS: A retrospective analysis was conducted using claims data from IQVIA's PharMetrics Plus database. Patients with a prevalent GA diagnosis were identified between October 1, 2016 and June 30, 2017 and classified by disease severity and laterality. Disease-specific HCRU and costs by disease severity were assessed during the 12-month follow-up period, with multivariable analyses performed adjusting for baseline characteristics. RESULTS: A total of 28,773 GA cases were identified (mean age = 68.7; 58.5% female), of which 24% and 76% had unilateral and bilateral GA, respectively, with varying levels of recorded severity (in increasing order): early or intermediate (EI) AMD, GA without subfoveal involvement (GAwoSF), and GA with subfoveal involvement (GAwSF). Patients with greater baseline severity in the bilateral group had a significantly higher number of outpatient (OP) visits per year (1.98 EI AMD; 2.57 for GAwoSF; 2.63 for GAwSF). Increasing disease severity was associated with higher patient-related costs in the outpatient setting (mean [SD] of $82 [$157], $110 [$559] for unilateral EI AMD and GAwSF, respectively, and $56 [$94], $64 [$97], $59 [$85] for bilateral EI AMD, GAwoSF, GAwSF, respectively). Similarly, higher payer-related costs were seen in patients with bilateral GAwSF compared to bilateral EI AMD (mean [SD] $280 [$325]; $198 [$262]). CONCLUSION: Study findings demonstrate that patients, with more severe GA at baseline, experience greater HCRU and costs in the outpatient setting. Further research should explore specific contributing factors to the long-term economic burden of GA.

Development of the Presbyopia Impact and Coping Questionnaire.

INTRODUCTION: Presbyopia is a progressive, age-related visual condition that is characterized by reduced ability to focus on near/close objects, causing impacts on individuals' daily function and health-related quality of life. The Presbyopia Impact and Coping Questionnaire (PICQ) is a new patient-reported outcome (PRO) instrument that assesses presbyopia impact and use of coping behaviors among presbyopic individuals. METHODS: To document the impacts of presbyopia and associated coping behaviors, concept elicitation (CE) interviews were conducted with 20 presbyopic participants. Results from the CE interviews were used to develop draft items for additional testing. Following item generation, the draft PICQ was cognitively debriefed with 20 participants. Data from a phase 2 controlled clinical trial were used for psychometric analyses of the PICQ. The PICQ was administered at site visits throughout a 28-day treatment period. Confirmatory factor analysis (CFA) methods were used to guide the development of the scoring algorithm. The reliability (internal consistency, test-retest), construct validity (convergent and discriminant validity, known-groups methods), and responsiveness (Guyatt's responsiveness statistic [GRS]) of the PICQ scores were evaluated. Finally, anchor-based and distribution-based methods were used to inform thresholds for interpreting meaningful within-patient change. RESULTS: CE interviews identified the important and relevant presbyopia-related impacts and coping behaviors and 22 items were drafted and cognitively debriefed. Following minor revisions and item addition/deletion, a version of the PICQ including 23 items was subjected to psychometric testing. The analysis sample included 151 participants. The CFA established two PICQ domain scores, Coping and Impact, on 0-to-4 scales that demonstrate good model fit (root mean square error of approximation = 0.06, comparative fit index = 0.98, Tucker-Lewis index = 0.98, standardized root mean square = 0.07). Cronbach's alphas for the Coping and Impact scores were 0.89 and 0.84, respectively. Test-retest intraclass correlation coefficients were 0.77 for Coping and 0.67 for Impact. The pattern of results assessing construct validity was acceptable for the PICQ Coping and Impact scores, with the magnitude of correlations and effect sizes generally meeting a priori expectations. The corresponding GRS effect sizes for the PICQ Coping scores were -1.23 (i.e., large) for Patient Global Impression of Change (PGIC) and -0.72 (i.e., medium) for uncorrected near visual acuity (UNVA). The GRS effect sizes for the PICQ Impact scores were -0.60 (i.e., medium) for PGIC and -0.35 (i.e., small) for UNVA. Across three sets of anchor-based analyses for interpreting individual-level change, a responder threshold of -1.00 was identified for both PICQ Coping and PICQ Impact scores. CONCLUSIONS: The totality of evidence from the qualitative and quantitative research establishes that the PICQ scores produced are valid and reliable measures of presbyopia impacts and coping behaviors that are important and relevant for assessing presbyopia treatment outcomes. CLINICALTRIALS. GOV IDENTIFIER: NCT02780115; date of registration May 19, 2016.

Development of the Near Vision Presbyopia Task-based Questionnaire for use in evaluating the impact of presbyopia.

BACKGROUND: Presbyopia is a progressive condition that reduces the eye's ability to focus on near objects with increasing age. After a systematic literature review identified no existing presbyopia-specific patient-reported outcome (PRO) instruments meeting regulatory guidance, a new PRO instrument, the Near Vision Presbyopia Task-based Questionnaire (NVPTQ), was developed. RESULTS: To explore the patient experience with presbyopia, concept elicitation interviews were conducted with 20 presbyopic participants. The most frequently reported impacts were difficulty with reading menus/books/newspapers/magazines, reading on a cell phone/caller ID, and reading small print. Based on these results, a task-based PRO instrument (the NVPTQ) was developed instructing participants to complete four near-vision, paper-based reading tasks (book, newspaper, nutrition label, menu) under standardized settings, and subsequently assess their vision-related reading ability and associated satisfaction. The draft NVPTQ was cognitively debriefed with a sample of 20 presbyopes, which demonstrated that most participants interpreted the items as intended and endorsed the relevance of the concepts being assessed. After the qualitative research, the draft instrument was psychometrically tested using data from a Phase 2 study. Based on item-level analyses, all items in the NVPTQ demonstrated expected response option patterns and lacked substantial floor or ceiling effects. The reliability, validity, and responsiveness of the NVPTQ Performance and Satisfaction domain scores were assessed. All domains scores had large Cronbach's coefficient α values and good test-retest statistics, indicating that the scores are internally consistent and produce stable values over time. The pattern of correlations with a concurrent measure of visual functioning (National Eye Institute Visual Function Questionnaire 25) demonstrated that the NVPTQ domain scores were related to an alternative assessment of near-vision activities. The NVPTQ domain scores were able to distinguish between groups that were known to differ on the clinical outcome of uncorrected near visual acuity, supporting the construct validity of these scores. The NVPTQ domain scores showed evidence of responsiveness to change by being able to distinguish between groups defined as improved and not improved based on patient-reported and clinical outcomes. CONCLUSIONS: This research has resulted in a content-valid and psychometrically sound instrument designed to evaluate vision-related reading ability and satisfaction with vision-related reading ability. TRIAL REGISTRATION: ClinicalTrials.gov NCT02780115. Registered 23 May 2016, https://www.clinicaltrials.gov/ct2/show/NCT02780115?term=NCT02780115&draw=2&rank=1.

Incremental direct medical expenditures associated with anxiety disorders for the U.S. adult population: evidence from the Medical Expenditure Panel Survey.

Data from the 2009 and 2010 Medical Expenditure Panel Survey (MEPS) were used to estimate the annual incremental healthcare expenditures associated with anxiety disorders, for the ambulatory adult population of the U.S. Individuals 18 years and older, who reported a diagnosis of, or had a medical event associated with anxiety disorder(s), were classified as anxiety population. Multivariate regression analyses, using generalized linear models, were conducted to calculate incremental costs associated with anxiety disorders. 8.74% of adult respondents reported being diagnosed with anxiety disorder(s). The annual overall healthcare expenditure associated with anxiety disorders was estimated at $1657.52 per person (SE: $238.83; p<0.001). Inpatient visits, prescription medications, and office-based visits together accounted for almost 93% of the overall cost. Given the prevalence of self-reported anxiety disorders in MEPS, their total direct medical expenditure is estimated at approximately $33.71 billion in 2013 US dollars, meaning this category of mental illnesses absorbs a significant portion of the U.S. healthcare resources.