Joint Assessment of Intended and Unintended Effects of Medications: An Example Using Vascular Endothelial Growth Factor Inhibitors for Neovascular Age-Related Macular Degeneration

Objective. To estimate the net health benefits of pegaptanib and ranibizumab by considering the impact of visual acuity and unintended effects (cardiovascular and hemorrhagic events) on quality-of-life among persons with neovascular age-related macular degeneration. Methods. We designed a probabilistic decision-analytic model using published data. It employed 17 visual health states and three for unintended effects. We calculated incremental net health benefits by subtracting the harms of each medication from the benefit using the quality-adjusted life year (QALY). Results. In a hypothetical cohort of 1,000 75-year olds with new-onset bilateral age-related macular degeneration followed for ten years, the mean QALYs per patient is 3.7 for usual care, 4.2 for pegaptanib, and 4.3 for ranibizumab. Net benefits decline with increasing baseline rates of unintended effects. Interpretation. Net health benefits present a quantitative, potentially useful tool to assist patients and ophthalmologists in balancing the benefits and harms of interventions for age-related macular degeneration

Understanding patient and caregiver perspectives using a dyad approach for data collection: A systematic review of the literature

Treatments outside of a clinical setting may be managed independently by the pediatric patient, independently by a caregiver, or by the patient and caregiver together. Best practices for pediatric clinical outcome assessment (COA) recommend patient-reported outcome (PRO) and/or observer-reported outcome (ObsRO) measures to assess the patient experience of a condition or its treatment. However, a dyad approach where patients and caregivers can complete assessments together may be useful for assessing a shared treatment experience that may not be adequately captured by a PRO and/or an ObsRO. A systematic, targeted literature review of empirical literature was conducted to identify and describe published studies detailing dyad patient-caregiver outcome reporting approaches. The search was run in the MEDLINE®, Embase, and PsycINFO® databases using the OvidSP platform and was limited to English-language studies published within 10 years of the conducted search on 28 September 2021, and 13 articles were selected for full-text review based on pre-specified criteria. Advantages and disadvantages for use of a dyad data collection approach are discussed. Though not appropriate for all settings, dyad data collection may be useful for situations where the best practice approach to measurement does not capture all relevant perspectives, or the use of PRO and ObsRO also does not comprehensively capture all relevant concepts. In following, it may offer a pragmatic solution that can minimize the use of proxy assessment and limit missing data, particularly in research involving a shared patient and caregiver treatment experience. Experience Framework This article is associated with the Staff & Provider Engagement lens of The Beryl Institute Experience Framework (https://www.theberylinstitute.org/ExperienceFramework). Access other PXJ articles related to this lens. Access other resources related to this lens

Evaluation of the American-English Quality of Life in Short Stature Youth (QoLISSY) questionnaire in the United States.

BACKGROUND: The European Quality of Life in Short Stature Youth (QoLISSY) is a novel condition-specific instrument developed to assess health related quality of life (HrQoL) in children/adolescents with short stature from patient and parent perspectives. Study objective was to linguistically validate and psychometrically test the American-English version of the QoLISSY instrument. METHODS: Upon conversion of the British-English version to American-English, content validity and acceptance of the questionnaire were examined through focus group discussions with cognitive debriefing in 28 children/adolescents with growth hormone deficiency (GHD) or idiopathic short stature (ISS) and their parents. In the subsequent field test with 51 families and a re-test with 25 families the psychometric performance of the American-English version was examined and compared with the original European dataset. RESULTS: Pilot test results supported the suitability of the American-English version. Good internal consistency with Cronbach\u27s Alpha ranging from 0.84 to 0.97 and high test-re-test reliabilities were observed in the field test. The QoLISSY was able to detect significant differences according to the degree of short stature with higher HrQoL for taller children. Correlations with a generic HrQoL tool support the QoLISSY\u27s concurrent validity. The scale\u27s operating characteristics were comparable to the original European data. CONCLUSION: Results support that the QoLISSY American-English version is a psychometrically sound short stature-specific instrument to assess the patient- and parent- perceived impact of short stature. The QoLISSY instrument is fit for use in clinical studies and health services research in the American-English speaking population

Cost-Effectiveness Model for Neovascular Age-Related Macular Degeneration: Comparing Early and Late Treatment with Pegaptanib Sodium Based on Visual Acuity

AbstractObjectiveTo compare the cost-effectiveness of pegaptanib and usual care within three distinct cohorts of subfoveal neovascular age-related macular degeneration (NV-AMD) patients, that is, those with early, moderate, and late disease, using a comprehensive economic model.MethodsA Markov framework was used to model lifetime movement of a subfoveal NV-AMD cohort through health states based on visual acuity. The model takes a US payer perspective of patients over the age of 65 years. Clinical efficacy was based on published results for the 0.3 mg pegaptanib and usual care groups. Expert interviews were conducted to determine adverse event treatment patterns and vision rehabilitation resource use. Incidence and costs of comorbidities such as depression and fractures associated with the effects of declining visual acuity were based on our previously published analysis of Medicare data. Transition probabilities were derived from published clinical trial data for each 3-month cycle. Utilities were derived from published sources. Three runs of the model were conducted with cohorts of newly diagnosed patients. Patients were classified as having early, moderate, or late NV-AMD defined as visual acuity in the better-seeing eye of 20/40 to more than 20/80, 20/80 to more than 20/200, and 20/200 to more than 20/400, respectively. Costs and outcomes were discounted 3.0% per annum.ResultsIncremental costs per vision-year gained and per quality-adjusted life-year (QALY) gained for early NV-AMD patients were approximately one-third those of patients with late disease ($15,279 vs.$57,230 and $36,282 vs.$132,381, respectively). On average, patients treated early with either pegaptanib or usual care incurred lower lifetime total direct costs than those treated later. Sensitivity analysis showed that base-case incremental costs per QALY gained for pegaptanib versus usual care were relatively robust.ConclusionsFor patients with subfoveal NV-AMD, treatment with pegaptanib should be started as early as possible to maximize the clinical and economic benefits

A Diagnostic Aid For Detecting Multiple Mental Disorders In Primary Care: The Symptom Driven Diagnostic System For Primary Care (sdds-pc*).

A high rate of undiagnosed mental disorders in primary care has been well documented for nearly three decades. (Shepherd et al 1966; Regier et al 1978,1993; Goldberg et al 1980; Van Hermert et al 1993; Olfson and Klerman, 1992; Barrett et al 1988). Failure to recognize mental illness has been shown to lead to undertreatment, greater impairment, and a longer duration of illness (Ormel et al 1991). The few efforts to change the diagnostic practice of primary care physicians, either through providing information from a patient screen completed prior to the physician visit or through physician education, have provided equivocal results. (Higgins 1994; Rand et al 1988; Goldberg 1980). The effects of improved physician recognition on decreasing patients’ health care use have also been equivocal, probably because there are many steps between recognition and patient outcome. These steps include accurate diagnosis, proper treatment, patient compliance, and timely follow-up with adjustment in treatment, as needed. Efforts to improve detection of mental disorders have concentrated on developing patient screens, which usually includea list ofpsychiatric symptoms independent of specific diagnoses, (Goldberg et al 1980; Borgquist et al 1993; Von Korff et al 1987) or which screen for one diagnosis; depression or alcoholism (Selzer 1971; Babor et al 1992). The former approach is limited because symptoms are not directly related to a treatable disorder, the latter, because patients may have more than one treatable psychiatric disorder (Kessler et al 1994). Moreover, screens are not widely used in primary care (Nelson and Berwick, 1989). Because most primary care visits are 15 minutes long or less, there is a need to develop rapid assessment methods that can be incorporated into routine care (Barrett 1991; Mitchell et al 1988; Anderson and Mattsson 1989)

Clinical risk factors for age-related macular degeneration: a systematic review and meta-analysis

BACKGROUND: Age-related macular degeneration (AMD) is the leading cause of blindness in Western countries. Numerous risk factors have been reported but the evidence and strength of association is variable. We aimed to identify those risk factors with strong levels of evidence which could be easily assessed by physicians or ophthalmologists to implement preventive interventions or address current behaviours. METHODS: A systematic review identified 18 prospective and cross-sectional studies and 6 case control studies involving 113,780 persons with 17,236 cases of late AMD that included an estimate of the association between late AMD and at least one of 16 pre-selected risk factors. Fixed-effects meta-analyses were conducted for each factor to combine odds ratio (OR) and/or relative risk (RR) outcomes across studies by study design. Overall raw point estimates of each risk factor and associated 95% confidence intervals (CI) were calculated. RESULTS: Increasing age, current cigarette smoking, previous cataract surgery, and a family history of AMD showed strong and consistent associations with late AMD. Risk factors with moderate and consistent associations were higher body mass index, history of cardiovascular disease, hypertension, and higher plasma fibrinogen. Risk factors with weaker and inconsistent associations were gender, ethnicity, diabetes, iris colour, history of cerebrovascular disease, and serum total and HDL cholesterol and triglyceride levels. CONCLUSIONS: Smoking, previous cataract surgery and a family history of AMD are consistent risk factors for AMD. Cardiovascular risk factors are also associated with AMD. Knowledge of these risk factors that may be easily assessed by physicians and general ophthalmologists may assist in identification and appropriate referral of persons at risk of AMD

Cross-Cultural Equivalence of the Patient- and Parent-Reported Quality of Life in Short Stature Youth (QoLISSY) Questionnaire

Background: Testing cross-cultural equivalence of Patient Reported Outcomes requires sufficiently large samples per country, which is difficult to achieve in rare endocrine paediatric conditions. We describe a novel approach to cross-cultural testing of the Quality of Life in Short Stature Youth (QoLISSY) questionnaire in five countries by sequentially Taking-One-Country-Out (TOCO) from the total sample and iteratively comparing the resulting psychometric performance. Methods: Development of the QoLISSY proceeded from focus group discussions over pilot testing to field testing in 268 short statured patients and parents. In exploring cross-cultural equivalence the iterative TOCO technique was used to examine and compare validity, reliability and convergence of QoLISSY patient- and parent-ratings in the field test data set, and to predict QoLISSY scores from clinical, socio-demographic and psychosocial variables. Results: Validity and reliability indicators were satisfactory for each sample after iteratively omitting one country. Comparisons with the total sample revealed cross-cultural equivalence in internal consistency and construct validity for patients and parents, high inter-rater agreement and a substantial proportion of QoLISSY variance explained by predictors. Conclusion: The TOCO technique is a powerful method to overcome problems of country specific testing of PRO instruments. It provides an empirical support to QoLISSY´s cross-cultural equivalence and is recommended for future research.This study was funded by Pfizer Limited. Andreas Pleil is at the time of this study full-time employee of Pfizer Inc; Hartmut Wollmann was a full-time employee of Pfizer Inc. No authors received compensation for their contributions to the manuscript