A framework for best practices in clinical outcome assessment (COA) concept mapping: a case study.

Purpose: Mapping or matching the items in a clinical outcome assessment (COA) to concepts that define a condition is a common method for evaluating a COA's concept coverage. The purpose of this research was to address the lack of formal guidance for conducting this task by developing a framework for best practices in COA concept mapping and applying it to a case study.

Methods: To develop the framework, we examined the literature and created a draft set of best practices which was then reviewed by experienced researchers through focus groups before being finalized.

Patient-Reported Outcome Measure Development and Validation: A Primer for Clinicians.

A comprehensive definition of health includes the assessment of patient experiences of a disease and its treatment. These patient experiences are best captured by standardized patient-reported outcome (PRO) instruments. A PRO is reported directly by the patient (or caregiver) and provides the patient's perspective into how a disease and its treatment impact their lives.

SF-36v2 and FACIT-Fatigue quality of life improvements with organ-specific SELENA-SLEDAI response and belimumab treatment in patients with systemic lupus erythematosus.

Objective: Explore organ-specific SLE burden by assessing health-related quality of life (HRQoL) and fatigue changes associated with Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus Disease Activity Index (SELENA-SLEDAI) organ system response (score improvement) and belimumab treatment.

Methods: Data from four phase III belimumab trials were pooled for post hoc analysis (GSK Study 217382): BLISS-52 (NCT00424476), BLISS-76 (NCT00410384), BLISS-SC (NCT01484496) and EMBRACE (NCT01632241). Patients with baseline organ system involvement were classed as organ system responders if SELENA-SLEDAI scores for that organ system decreased at any post-baseline visit.

Meaningful score changes for SF-36v2, FACIT-fatigue, and RASIQ in rheumatoid arthritis.

Background: Interpretation thresholds for patient-reported outcome (PRO) scores are of crucial importance, particularly when interpreting treatment benefit. This study was designed to determine the within-patient meaningful improvement (WPMI) thresholds for the Short-Form 36 Health Survey version 2 (SF-36v2), the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue), and the novel Rheumatoid Arthritis Symptoms and Impact Questionnaire (RASIQ) among patients with rheumatoid arthritis (RA).

Methods: In this post-hoc analysis, anchor-based and supportive distribution-based methods were used to derive WPMI based on blinded data from all treatment arms in two Phase 2 RA trials with otilimab.

Parenteral Vancomycin in the Treatment of MRSA-Associated Diabetic Foot Infections: An Unnecessary Risk.

Diabetic foot infections (DFIs) are a common and costly complication of diabetes. Soft tissue and bone infections in DFIs frequently lead to amputation and/or sepsis which can be costly for both the patient and the healthcare system. is the most commonly identified causative agent in DFIs, and people with diabetes may have an increased risk of infection with methicillin-resistant (MRSA).

Content Validation of the Movement Disorder-Childhood Rating Scale (MD-CRS) for Dyskinetic Cerebral Palsy.

Background: Dyskinetic cerebral palsy (DCP), a lifelong neurological disorder beginning in early childhood, manifests with hyperkinetic movements and dystonia. The Movement Disorder-Childhood Rating Scale (MD-CRS) is a clinician-reported outcome measure assessing the intensity of movement disorders and their effect on daily life in pediatric patients. Content validity of clinical outcome assessments is key to accurately capturing patient perspective.

Content validity and psychometric properties of the inFLUenza Patient-Reported Outcome Plus (FLU-PRO Plus) instrument in patients with COVID-19.

Purpose: A well-defined and reliable patient-reported outcome instrument for COVID-19 is important for assessing symptom severity and supporting research studies. The InFLUenza Patient-Reported Outcome (FLU-PRO) instrument has been expanded to include loss of taste and smell in the FLU-PRO Plus, to comprehensively cover COVID-19 symptoms. Our studies were designed to evaluate and validate the FLU-PRO Plus among patients with COVID-19.

Validation of the PROFAD-SSI-SF in Patients with Primary Sjögren's Syndrome with Organ Involvement: Results of Qualitative Interviews and Psychometric Analyses.

Introduction: The Profile of Fatigue and Discomfort-Sicca Symptoms Inventory-Short Form (PROFAD-SSI-SF) is a 19-item patient-reported outcome (PRO) measure to assess pain, fatigue, and dryness in patients with primary Sjögren's syndrome (pSS). This analysis identified concepts important to measure, and evaluated the content validity and measurement properties of the PROFAD-SSI-SF, in patients with pSS.

Methods: Qualitative analyses (GSK Study 208396) used transcripts from an online concept elicitation (CE) discussion forum with patients with pSS and interviews with key opinion leaders (KOLs) to finalize a disease model depicting important concepts for patients with pSS.

Deciding Between SF-6Dv2 Health States: A Think-Aloud Study of Decision-Making Strategies Used in Discrete Choice Experiments.

Objective: This study aimed to gain insight into decision-making strategies individuals used when evaluating pairs of SF-6Dv2 health states in discrete choice experiments (DCEs).

Methods: This qualitative, cross-sectional, noninterventional study asked participants to use a think-aloud approach to compare SF-6Dv2 health states in DCEs. Thematic analysis focused on comprehension and cognitive strategies used to compare health states and make decisions.

Considerations When Selecting Patient-Reported Outcome Measures for Assessment of Health-Related Quality of Life in Patients With Pulmonary Hypertension: A Narrative Review.

It is well established that pulmonary hypertension (PH) places a substantial burden on patients' health-related quality of life (HRQoL). As more effective treatments have been developed for this condition, evaluating treatment benefit based on experiences reported by patients regarding their well-being and physical, social, and emotional functioning has increased. A review of the published literature and clinical trials in PH was conducted to identify and evaluate patient-reported outcome measures (PROMs) that assess PH-specific HRQoL for use in clinical studies.

Mental health impact on healthcare workers due to the COVID-19 pandemic: a U.S. cross-sectional survey study.

Background: The COVID-19 pandemic has impacted the mental health and well-being of health care workers (HCWs). This study examined mental health outcomes and COVID-related stress impacts among a diverse sample of ambulatory HCWs, including clinicians and support staff, as well as the associations between mental health outcomes and work impairments in this population. Detailing these results can help in designing interventions to alleviate this burden.

Development of the SF-6Dv2 health utility survey: comprehensibility and patient preference.

Background: The SF-6Dv2 classification system assesses health states in six domains-physical functioning, role function, bodily pain, vitality, social functioning, and mental health. Scores have previously been derived from the SF-36v2® Health Survey. We aimed to develop a six-item stand-alone SF-6Dv2 Health Utility Survey (SF-6Dv2 HUS) and evaluate its comprehensibility.

Development, psychometric evaluation and cognitive debriefing of the rheumatoid arthritis symptom and impact questionnaire (RASIQ).

Background: Rheumatoid arthritis (RA) is a chronic inflammatory disease often associated with persistent pain. There is a need for a patient-reported outcome measure (PROM) that is rooted in the patient experience and psychometrically validated. We describe the development of the Rheumatoid Arthritis Symptom and Impact Questionnaire (RASIQ), a novel PROM with potential to record key symptoms and impacts of RA with a 24-h recall period.

Qualitative and psychometric approaches to evaluate the PROMIS pain interference and sleep disturbance item banks for use in patients with rheumatoid arthritis.

Background: Patients with rheumatoid arthritis (RA) commonly experience pain despite the availability of disease-modifying treatments. Sleep disturbances are frequently reported in RA, with pain often a contributing factor. The Patient-Reported Outcomes Measurement Information System (PROMIS) Pain Interference and Sleep Disturbance item banks were initially developed to provide insights into the patient experience of pain and sleep, respectively, though they were not specifically intended for use in RA populations.

Responder analysis for neuropathic impairment and quality-of-life assessment in patients with hereditary transthyretin amyloidosis with polyneuropathy in the NEURO-TTR study.

Objective: Hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) is a rare disease characterized by rapid neuropathic progression. In pivotal studies of gene-silencing treatments, the modified Neuropathy Impairment Score + 7 tests (mNIS + 7) and Norfolk-Quality of Life (QOL)-Diabetic Neuropathy (DN) questionnaire assessed treatment impact on neuropathic progression. Establishing responder definition (RD) thresholds for these measures would enable evaluation of clinically meaningful treatment benefit.

Psychometric properties of FACIT-Fatigue in systemic lupus erythematosus: a pooled analysis of three phase 3 randomised, double-blind, parallel-group controlled studies (BLISS-SC, BLISS-52, BLISS-76).

Background: Fatigue is a key symptom in patients with systemic lupus erythematosus (SLE), and regulatory bodies recommend its assessment in clinical trials of SLE therapies.

Methods: This post hoc pooled analysis of the three BeLimumab In Subjects with Systemic lupus erythematosus (BLISS) Phase 3 randomised, double-blind, parallel-group controlled trials evaluated the measurement properties of the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue. Patients (N = 2520) completed the FACIT-Fatigue every 4 weeks from baseline until the end of each study period.

Achieving clinical response in postpartum depression leads to improvement in health-related quality of life.

Objective: To evaluate the health-related quality of life (HRQoL) burden associated with postpartum depression (PPD), determine the extent to which clinical response impacts HRQoL, and estimate the impact of PPD and clinical response on healthcare resource utilization (HRU) and productivity.

Methods: Patient data ( = 127) from two multicenter, randomized, double-blind, placebo-controlled phase 3 clinical trials evaluating the safety and efficacy of brexanolone injection in adults with PPD were employed for these posthoc analyses. HRQoL and health utility was assessed with the SF-36-v2 Health Survey (SF-36v2) acute version.

A structured review evaluating content validity of the Asthma Control Test, and its consistency with U.S. guidelines and patient expectations for asthma control.

Objective: To assess whether the content of the Asthma Control Test (ACT) served as a valid measure of asthma control (i.e., content validity) by mapping ACT items to the National Heart, Lung and Blood Institute (NHLBI) guideline asthma control definitions, and to language used by patients to describe their asthma.

Heart dose and coronary artery calcification in patients receiving thoracic irradiation for lung cancer.

Background: Thoracic irradiation (TIR) is associated with an increased risk of coronary artery disease (CAD) and coronary-related death. Lung cancer patients receive considerable doses of TIR, making them a high-risk population that may benefit from post-therapy surveillance. Coronary artery calcium (CAC) is a known biomarker of CAD development and may serve as a useful indicator of disease progression in this population.

Assessment of the COPD Assessment Test Within U.S. Primary Care.

Rationale: Uptake of the COPD Assessment Test (CAT) is not yet widespread in patients with chronic obstructive pulmonary disease (COPD) within U.S. primary care and its alignment with other assessments has not been evaluated in U.

Impact of chronic thrombocytopenia on in-hospital outcomes and healthcare resource utilization after transcatheter aortic valve replacement.

Background: There is little data on the impact of chronic thrombocytopenia (CTP) on outcomes after transcatheter aortic valve repair (TAVR). Most studies are from single centers and mostly focused on postprocedure thrombocytopenia.

Objectives: This study sought to report on the impact of CTP (>1 year) on in-hospital outcomes and healthcare resource utilization after TAVR.