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. To conduct the case study, we extracted data from a systematic review of knee osteoarthritis (KO) symptoms and impacts and used the framework to map relevant concepts to items in the SF-36v2® Health Survey (SF-36v2). RESULTS: The framework guides researchers in defining the purpose of and data sources for the mapping, establishing guiding principles and decision-making thresholds, and conducting the mapping exercise. The results of the case study demonstrate the usefulness of the framework in identifying 27/36 items (75%) in the SF-36v2 that addressed concepts that define KO. CONCLUSION: This case study illustrates how the framework for best practices in COA concept mapping may be used, highlighting how establishing clear concept definitions and guiding principles and following a structured process throughout can help produce consistent, reliable, and reproducible results. The results from this rigorous approach can provide valuable evidence to support decisions about the appropriateness of a COA for the intended patient population.

In health-related quality of life research, mapping items in a clinical outcome assessment (COA) to concepts that define a health condition is one way to evaluate an instrument's content validity (or, how well the instrument addresses the concepts it intends to measure). Without formal guidance on how to do this mapping, researchers can be inconsistent. This article describes the development of a framework for best practices in COA concept mapping. Informed by the literature and input from researchers with expertise in COA development and evaluation, the final framework guides researchers through the mapping process from start to finish, from helping to define the purpose of the task and identify the data sources, to establishing guiding principles and decision-making thresholds, conducting the mapping, and displaying the results. A case study­in which items from the SF-36v2^® Health Survey were mapped to concepts from a systematic review of knee osteoarthritis symptoms and impacts­shows the framework in action, demonstrating how following the best practices can lead to consistent results that can support the evaluation of an instrument's content validity.

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. RESULTS: Participants (N = 40) used 3 main strategies when completing DCEs: (1) trading, (2) reinterpretation, and (3) relying on previous experience. Trading was the most common strategy, used by everyone at least once, and involved prioritizing key attributes, such as preferring a health state with significant depression but no bodily pain. Reinterpretation was used by 17 participants and involved reconstructing health states by changing underlying assumptions (eg, rationalizing selecting a health state with significant pain because they could take pain medications). Finally, some (n = 13) relied on previous experience when making decisions on some choice tasks. Participants with experience dealing with pain, for instance, prioritized health states with the least impact in this dimension. CONCLUSIONS: Qualitatively evaluating the decision-making strategies used in DCEs allows researchers to evaluate whether the tasks and attributes are interpreted accurately. The findings from this study add to the understanding of the generation of SF-6Dv2 health utility weights and the validity of these weights (e.g., reinterpreting health states could undermine the validity of DCEs and utility weights), and the overall usefulness of the SF-6Dv2. The methodology described in this study can and should be carried forth in valuing other health utility measures, not just the SF-6Dv2.

Understanding the Symptom Burden and Impact of Myasthenia Gravis from the Patient's Perspective: A Qualitative Study.

INTRODUCTION: Patients with generalized myasthenia gravis (gMG) experience functional impairment due to MG symptoms. This study aimed to assess, from the patient perspective, the symptoms, impacts, and treatment goals of individuals diagnosed with gMG. METHODS: Semi-structured, in-depth concept-elicitation interviews were conducted with 28 individuals diagnosed with gMG in the United States. RESULTS: Participants reported gMG symptoms that affected many body regions and functions, with an average of 16 symptoms per participant. The most frequently reported symptoms were eyelid drooping (93%), physical fatigue (89%), symptoms affecting the legs (82%), difficulty breathing (82%), and difficulty holding head up (82%). Nearly all participants (96%) reported fluctuations in symptoms and severity. Participants' most bothersome symptoms were blurry/double vision (43%), breathing difficulties (36%), all-over fatigue (36%), and swallowing problems (29%). Impacts on physical functioning included an inability to participate in hobbies/sports, need for increased planning, and difficulties performing activities of daily living. All participants reported emotional impacts and impacts on their work and finances. Their treatment goals included reduced fatigue and weakness, improved symptom stability, and minimization of the impact of symptoms, in particular the emotional impact. CONCLUSIONS: The fluctuating and unpredictable nature of gMG symptoms was found to have a substantial impact on patients' emotional, social, and economic well-being. Participants' goals for symptom management suggest that greater focus is needed to help them quickly resume a normal lifestyle by achieving symptom stability. Impacts of fluctuating and unpredictable symptoms are difficult to measure, but it is important to consider symptom fluctuation as well as ongoing symptomatology when making treatment decisions, and to recognize the impact of uncontrolled symptoms on patients, their partners/caregivers, and family/friends. These factors are often not reflected in burden/cost-of-illness studies.

The aim of this study was to understand­from the patient's point of view­the range of generalized myasthenia gravis (gMG) symptoms that they experience, which symptoms bother them most, and which symptoms have the greatest impact on everyday life, as well as patients' treatment goals. Researchers asked these questions in one-on-one interviews with 28 people in the US who have gMG. Study participants reported living with symptoms that come and go, and are sometimes severe, making it difficult to lead a normal life. The most frequently reported symptoms were eyelid drooping (reported by 93% of study participants), physical fatigue (89%), symptoms affecting the legs (82%), difficulty breathing (82%), and difficulty holding head up (82%). The symptoms that bothered patients most were difficulties with vision (43%), problems breathing (36%), all-over fatigue (36%), and trouble swallowing (29%). Participants reported that gMG symptoms affected physical functioning, making it hard to participate in hobbies/sports, increasing the amount of planning needed to conduct their daily lives, and hindering their ability to do day-to-day activities (like driving, eating, and bathing). All participants reported that they were affected emotionally, and that the symptoms of gMG impacted their ability to work and their financial well-being. Participants' treatment goals included reducing fatigue and weakness, making symptoms more stable, and reducing the impact of symptoms, particularly emotional impacts. These responses reveal the extensive effects of gMG symptoms on patients' daily lives and highlight that symptom stability is especially important to people with gMG.

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. METHODS: Two forms of a stand-alone SF-6Dv2 HUS were developed for evaluation. Form A had 6 questions with 5-6 response choices, while Form B used 6 headings and 5-6 statements describing the health levels within each domain. The two forms were evaluated by 40 participants, recruited from the general population. Participants were randomized to debrief one form of the stand-alone SF-6Dv2 HUS during a 75-min interview, using think-aloud techniques followed by an interviewer-led detailed review. Participants then reviewed the other form of SF-6Dv2 and determined which they preferred. Any issues or confusion with items was recorded, as was as overall preference. Data were analyzed using Microsoft Excel and NVivo Software (v12). RESULTS: Participants were able to easily complete both forms. Participant feedback supported the comprehensibility of the SF-6Dv2 HUS. When comparing forms, 25/40 participants preferred Form A, finding it clearer and easier to answer when presented in question/response format. The numbered questions and underlining of key words in Form A fostered quick and easy comprehension and completion of the survey. However, despite an overall preference for Form A, almost half of participants (n = 19) preferred the physical functioning item in Form B, with more descriptive response choices. CONCLUSION: The results support using Form A, with modifications to the physical functioning item, as the stand-alone SF-6Dv2 HUS. The stand-alone SF-6Dv2 HUS is brief, easy to administer, and comprehensible to the general population.