Elizabeth Nicole Bush

Content Validity of the Female Sexual Function Index (FSFI) in Pre‐ and Postmenopausal Women with Hypoactive Sexual Desire Disorder

INTRODUCTION The Female Sexual Function Index (FSFI) has consistently been shown to have discriminant validity, test-retest reliability, and internal consistency as a measure of female sexual function. However, the content validity (relevance, clarity, comprehensiveness) of the instrument in women with hypoactive sexual desire disorder (HSDD) must also be established. AIM The aim of this study were to assess the content validity of the FSFI, specifically the FSFI desire domain, in pre- and postmenopausal women with HSDD. METHODS Two single-visit content validation studies were conducted in the United States. Eligible premenopausal (both studies) and postmenopausal (second study only) women with HSDD completed the FSFI followed by one-on-one, face-to-face cognitive debriefing interviews including open-ended questions to capture information on their perceptions of the instrument. Information on womens experiences of decreased sexual desire was also captured. MAIN OUTCOME MEASURES The main outcome measures of this study were the womens ratings of the clarity, ease of understanding, comprehensiveness, and relevance of the 19 items of the FSFI. RESULTS Interviews with 15 premenopausal women (first study), and 30 pre- and 31 postmenopausal women (second study), were analyzed. Across the whole sample, most women (80-100%) found every item of the FSFI clear and easy to understand. The majority (53-70%) felt that the FSFI captured all their feelings about decreased sexual desire and other sexual problems, and most (84-90%) indicated that additional questions were unnecessary. Most women in both studies (93-100%) reported that the two items comprising the FSFI desire domain were clear, easy to understand, and were relevant to them. The majority of women thought that a recall period of ≥7 days is most relevant for recall of their sexual desire. CONCLUSIONS These studies establish the content validity of the FSFI in pre- and postmenopausal women with HSDD, supporting the use of this instrument as a measure of sexual function in women with this condition.

Characteristics of hip fracture patients with and without muscle atrophy/weakness: Predictors of negative economic outcomes

Abstract Objective: Hip fractures have negative humanistic and economic consequences. Predictors and sub-groups of negative post-fracture outcomes (high costs and extensive healthcare utilization) were identified in patients with and without muscle atrophy/weakness (MAW). Methods: Truven Health MarketScan data identified patients ≥50 years old with inpatient hospitalizations for hip fracture. Patients had ≥12 months of continuous healthcare insurance prior to and following index hospitalization and no hip fracture diagnoses between 7 days and 1 year prior to admission. Predictors and sub-groups of negative outcomes were identified via multiple logistic regression analyses and classification and regression tree (CART) analyses, respectively. Results: Post-fracture 1-year all-cause healthcare costs (USD

Qualitative Research on Fatigue Associated with Depression: Content Validity of the Fatigue Associated with Depression Questionnaire (FAsD-V2)

31,430) were higher than costs for the prior year (

One Programme, Four Stakeholders: An Overview of the Utilisation of Patient-Reported Outcomes in Intervention Development to Meet the Needs of Regulators, Payers, Healthcare Professionals and Patients

18,091; p < 0.0001). Patients with MAW had greater post-fracture healthcare utilization and costs than those without MAW (p < 0.05). Greater post-fracture costs were associated with a higher number of prior hospitalizations and emergency room visits, length of index hospitalization, Charlson Comorbidity Index (CCI), and discharge status; diagnosis of rheumatoid arthritis, osteoarthritis, or osteoporosis; and prior use of antidepressants, anticonvulsants, muscle relaxants, benzodiazepines, opioids, and oral corticosteroids (all p < 0.009). High-cost patient sub-groups included those with MAW and high CCI scores. Conclusions: Negative post-fracture outcomes were associated with MAW vs no MAW, prior hospitalizations, comorbidities, and medications.

Evaluation of the Measurement Properties of Four Performance Outcome Measures in Patients with Elective Hip Replacements, Elective Knee Replacements, or Hip Fractures

BackgroundFatigue is one of the most common symptoms of major depressive disorder (MDD). The Fatigue Associated with Depression Questionnaire (FAsD) was developed to assess fatigue and its impact in patients with MDD. The current article presents the qualitative research conducted to develop and examine the content validity of the FAsD and FASD–Version 2 (FAsD–V2).MethodsThree phases of qualitative research were conducted with patients recruited from a geographically diverse range of clinics in the US. Phase I included concept elicitation focus groups, followed by cognitive interviews. Phase II employed similar techniques in a more targeted sample. Phase III included cognitive interviews to examine whether minor edits made after Phase II altered comprehensibility of the instrument. Concept elicitation focused on patients’ perceptions of fatigue and its impact. Cognitive interviews focused on comprehension, clarity, relevance, and comprehensiveness of the instrument. Data were collected using semi-structured discussion guides. Thematic analyses were conducted and saturation was examined.ResultsA total of 98 patients with MDD were included. Patients’ statements during concept elicitation in phases I and II supported item development and content. Cognitive interviews supported the relevance of the instrument in the target population, and patients consistently demonstrated a good understanding of the instructions, items, response options, and recall period. Minor changes to instructions for the FAsD–V2 did not affect interpretation of the instrument.ConclusionsThis qualitative research supports the content validity of the FAsD and FAsD–V2. These results add to previous quantitative psychometric analysis suggesting the FAsD–V2 is a useful tool for assessing fatigue and its impact in patients with MDD.

Response scale selection in adult pain measures: results from a literature review

For a new intervention to achieve commercial success, regulators (to provide marketing authorisation), payers (for reimbursement and formulary placement), healthcare professionals (HCPs; to prescribe) and patients (to adhere and persist) must all consider the intervention to be valuable. These stakeholders are increasingly using the patient’s perspective on a condition and its treatment—measured through patient-reported outcome (PRO) instruments—to define the value of a treatment. However, there is common confusion about the most appropriate way to incorporate the patient’s perspective into the clinical development of pharmaceutical interventions in a way that will resonate with these four key stakeholder groups. This article briefly summarises the perspectives of regulators, payers, HCPs and patients regarding PRO endpoints, and examines how a robust, comprehensive and systematic PRO endpoint strategy can be developed to meet the needs of all stakeholders in a single development programme. Such an endpoint strategy may include PRO assessments of first-order signs and symptoms using a disease-specific measure, health-related quality of life using both a disease-specific measure and generic utility measure, and general quality of life. Other PRO endpoints (e.g. preference/satisfaction, ease of use, HCP contact time, absenteeism) may be useful with some stakeholders to provide further differentiation between interventions.

Patient-Centered Research to Support the Development of the Symptoms of Major Depressive Disorder Scale (SMDDS): Initial Qualitative Research

OBJECTIVES To evaluate the measurement properties of four performance outcome (PerfO) measures (timed up and go, four-step stair climb, long stair climb, and repeated chair stand) in three patient populations(elective total hip replacement [eTHR], elective total knee replacement [eTKR], and hip fracture [HF]). METHODS A cross-sectional and longitudinal design was used to assess the PerfO measurement properties using the US Food and Drug Administration guidance for industry around patient-reported outcome measures to support labeling claims. Patient-reported outcome measures and patient- and clinician-reported global concept items were completed along with four PerfO measures at visit 1 and two follow-up visits. Measurement properties assessed included reliability, construct validity, ability to detect change, and estimates of meaningful change. RESULTS A total of 280 patients (100 eTHR, 105 eTKR, and 75 HF) were recruited, with most (n = 276) providing data at visit 1. Most of the patients were female (64%) and retired (64%), and had at least one comorbidity (91%). Inter-rater and test-retest reliability ranged from good to excellent (0.73 ≤ intraclass correlation coefficient ≤ 0.95) for each PerfO measure. Known-groups validity was demonstrated for all PerfO measures, with those reporting less pain better physical functioning and those who did not use an assistive device having quicker mean completion times. Construct validity and ability to detect change were demonstrated and estimates of meaningful change derived. CONCLUSIONS This study found the measurement properties of four PerfO measures in samples of patients with eTHR, eTKR, and HF to be supported for consideration of future use, and provided estimates for interpretation of change.

Evaluating the Content Validity of Four Performance Outcome Measures in Patients with Elective Hip Replacements and Hip Fractures

BackgroundThe purpose of this literature review was to examine the existing patient-reported outcome measurement literature to understand the empirical evidence supporting response scale selection in pain measurement for the adult population.MethodsThe search strategy involved a comprehensive, structured, literature review with multiple search objectives and search terms.ResultsThe searched yielded 6918 abstracts which were reviewed against study criteria for eligibility across the adult pain objective. The review included 42 review articles, consensus guidelines, expert opinion pieces, and primary research articles providing insights into optimal response scale selection for pain assessment in the adult population. Based on the extensive and varied literature on pain assessments, the adult pain studies typically use simple response scales with single-item measures of pain—a numeric rating scale, visual analog scale, or verbal rating scale. Across 42 review articles, consensus guidelines, expert opinion pieces, and primary research articles, the NRS response scale was most often recommended in these guidance documents. When reviewing the empirical basis for these recommendations, we found that the NRS had slightly superior measurement properties (e.g., reliability, validity, responsiveness) across a wide variety of contexts of use as compared to other response scales.ConclusionsBoth empirical studies and review articles provide evidence that the 11-point NRS is likely the optimal response scale to evaluate pain among adult patients without cognitive impairment.

Developing and Implementing Performance Outcome Assessments: Evidentiary, Methodologic, and Operational Considerations

BackgroundContent valid, patient-reported outcome (PRO) measures of major depressive disorder (MDD) symptoms are needed to assess MDD treatment benefit. While a range of questionnaires are currently available to evaluate aspects of depression from the patient’s perspective, their comprehensiveness and qualitative development histories are unclear.ObjectiveThe objective of this study was to describe the process and results of the preliminary qualitative development of a new symptom-based PRO measure intended to assess treatment benefit in MDD clinical trials.MethodsQualitative interviews were conducted with adult MDD patients in the USA who recently experienced a major depressive episode. Experienced interviewers conducted concept elicitation (CE) and cognitive interviews using semi-structured interview guides. The CE interview guide was used to elicit spontaneous reports of symptom experiences along with probing to further explore and confirm concepts. The cognitive interview guide was developed to evaluate concept relevance, understandability, and structure of the draft items, and to facilitate further instrument refinement.ResultsForty patients participated in the CE interviews. A total of 3022 symptom codes, representing 84 different concepts were derived from the transcripts. Data from the CE interviews were considered alongside existing literature and clinical expert opinion during an item-generation process, leading to development of a preliminary version of the Symptoms of Major Depressive Disorder Scale (SMDDS). Fifteen patients participated in three waves of cognitive interviews, during which the SMDDS was further refined.ConclusionsThe SMDDS is a 35-item PRO measure intended for use as an endpoint in MDD clinical trials to support medical product labeling. The SMDDS uses a 7-day recall period and verbal rating scales. It was developed in accordance with the US Food and Drug Administration (FDA)’s PRO Guidance and best practices. Qualitative interviews have provided evidence for content validity. Future quantitative studies will confirm the SMDDS’s measurement properties and support FDA qualification.

The Potential Role of Individual-Level Benefit-Risk Assessment in Treatment Decision Making: A DIA Study Endpoints Community Workstream

OBJECTIVES To assess the content validity of performance outcome (PerfO) measures for use with patients undergoing hip fracture (HF) surgery and elective total hip replacement (eTHR). METHODS This study was a substudy of a broader evaluation of measurement properties of PerfO measures. The PerfO measures assessed were timed up and go (TUG), four-step stair climb (4SC), long stair climb (LSC), and repeated chair stand (RCS). For this substudy, HF and eTHR participants were interviewed to evaluate the relevance and difficulty of each PerfO measure. Qualitative analysis was conducted on interview transcripts, and summaries of coded data were produced to assess saturation. RESULTS All 18 HF participants related the PerfO measures (TUG, 4SC, and RSC) to activities they completed in daily life, with slight variations in some specific aspects. For the eight eTHR participants, the correspondence between the PerfO measures (TUG, 4SC, and LSC) and activities in daily life varied: all participants saw similarity in the movements for the TUG; most undertook short stair climbs in daily life, but most did not regularly undertake LSC in daily life. Nevertheless, all HF and eTHR participants reported that the PerfO measures were relevant and had a level of difficulty similar to daily activities. CONCLUSIONS This study contributes novel methods that adapt US regulatory guidance for patient-reported outcome measures to the evaluation of PerfO measures. A structured approach was used to explore specific details of each measure and correspondence to everyday life. This study demonstrates how content validity of PerfO measures can be meaningfully assessed.