Assessing the content validity of patient-reported outcome measures in adult myositis: A report from the OMERACT myositis working group.
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ABSTRACT: Objective
To investigate the content validity of several patient-reported outcome measures (PROMs) in patients with idiopathic inflammatory myopathies (IIM).Methods
Seven individual PROM instruments were selected by the Outcome Measures in Rheumatology (OMERACT) Myositis Working Group relating to the following domains: pain, fatigue, physical function and physical activity. Twenty patients from the Johns Hopkins Myositis Center were selected for one-on-one face-to-face or phone interviews for cognitive interviewing of individual PROMs to assess comprehension and content validity. Additionally, patients were asked if they thought muscle symptoms, an area originally identified in qualitative studies, were encapsulated by the other four domains.Results
The majority of patients (>70%) felt that each of the instruments was clear, easy to read and understand, and could be used for assessment of its domain. Two-thirds (66%) of patients felt that 'muscle symptoms' were captured by the other domains.Conclusions
We provided evidence to support adequate content validity for several PROMs. Further research is needed to determine whether 'muscle symptoms' warrant a separate domain.
SUBMITTER: Esfandiary T
PROVIDER: S-EPMC7646936 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:BACKGROUND:Evolving regulatory guidelines recommend routine assessment of the acceptability of pediatric oral medicines throughout clinical development processes. However, such assessment is problematic owing to a lack of standard methods or criteria that define acceptability for children and their caregivers. This research aimed to identify the attributes of acceptability for targeted oral formulation types that are important to children, and to develop content-valid patient- and caregiver-reported outcome acceptability measures for use in the context of clinical drug development. METHODS:A concept-focused literature review and two advisory panel meetings involving researchers, clinicians, and measurement scientists were conducted to identify acceptability attributes that may be relevant to children taking targeted oral medicine formulations. The Pediatric Oral Medicines Acceptability Questionnaires (P-OMAQs), including patient (P-OMAQ-P) and caregiver (P-OMAQ-C) versions, were drafted to assess these attributes. Qualitative concept elicitation (CE) and cognitive debriefing (CD) patient and caregiver interviews were conducted to confirm key acceptability attribute concepts for measurement and to evaluate patient and caregiver ability to understand and respond to the questions. RESULTS:A full-text review of 40 articles identified 24 acceptability attributes that were categorized into 10 overarching domains and organized into a preliminary conceptual model. Feedback from the advisory panel refined the preliminary model. In total, 14 attributes were reported during the CE phase of the interviews (n?=?23 pediatric patients, n?=?13 caregivers); six attributes were included in the final model. The draft P-OMAQ was refined over four waves of CD interviews (n?=?31 pediatric patients, n?=?48 caregivers). The final version of the P-OMAQ-P is a 12-item questionnaire designed for young people aged 8-17?years. The P-OMAQ-C is a 19-item questionnaire designed for adult caregivers of young people aged 6?months to 17?years. There are two versions of each questionnaire: one with a 24-h recall period and one with a 7-day recall period. All items are answered on a 5-point numerical rating scale. CONCLUSIONS:This research supports the content validity of the patient and caregiver versions of the P-OMAQ. Both questionnaires appropriately assess the acceptability of oral medicine formulations from the perspective of pediatric patients and their caregivers.
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