International multiphase mixed methods study protocol to develop a cross-cultural patient-reported outcome instrument for children and young adults with cleft lip and/or palate (CLEFT-Q)

Abstract

IntroductionPatient-reported outcome (PRO) instruments should be developed according to rigorous guidelines in order to provide clinically meaningful, scientifically sound measurement. Understanding the methodology behind instrument development informs the selection of the most appropriate tool. This mixed methods protocol describes the development of an internationally applicable PRO instrument, the CLEFT-Q, for evaluating outcomes of treatment for cleft lip and/or palate (CL/P).Methods and analysisThe study includes three main phases that occur iteratively and interactively. In phase I, we determine what concepts are important to patients regarding their outcome. A conceptual framework for the CLEFT-Q is formed through a systematic review and an extensive international qualitative study. The systematic review ascertains what concepts have previously been measured in patients with CL/P. The qualitative study employs interpretive description and involves in-depth interviews with patients in high-income and lower-middle income countries. Preliminary items are generated from the qualitative data. Preliminary scales are then created for each theme in the framework. Cognitive debriefing interviews and expert clinician input are used to refine the scales in an iterative process. In phase II, the preliminary scales are administered to a large international group of patients with CL/P. The modern psychometric method of Rasch Measurement Theory analysis is employed to define the measurement characteristics. The preliminary scales are shortened based on these results. In phase III, further tests assess reliability, validity and responsiveness of the instrument.Ethics and disseminationThe study is approved by Research Ethics Boards for each participating site. Findings from this study will be published in open access peer-reviewed journals and presented at national and international conferences. Integrated knowledge translation is employed to engage stakeholders from the outset of the study. Successful execution of the CLEFT-Q will result in an internationally applicable PRO instrument for children and young adults with CL/P.

Psychometric findings and normative values for the CLEFT-Q based on 2434 children and young adult patients with cleft lip and/or palate from 12 countries

“…Full protocol details have been published. 18 Content was developed from interviews with 138 patients from 6 countries. 19 Findings were used to develop a conceptual framework comprising 3 domains; i.e., appearance, health-related quality of life and facial function.…”

Development of a new patient‐reported outcome measure to evaluate treatments for acne and acne scarring: the ACNE ‐Q

“…We used a modern psychometric approach called Rasch Measurement Theory (RMT) . Figure shows our multiphase iterative mixed‐methods approach . This paper describes phases I and II.…”

“…Flow diagram showing the multiphase mixed‐methods protocol for developing the ACNE ‐Q (reproduced with permission from Wong Riff et al .) . QUAN , quantitative; QUAL , qualitative.…”

How to Integrate Lived Experience Into Quality-of-Life Assessment in Patients Considering Facial Transplantation

“…22 The definition of QoL used in these measures evolved through a phased approach, beginning with systematic review of extant literature and qualitative methods (including in-depth patient interviews) and progressing to development of scales, field testing, and psychometric validation and evaluation. 23,24 This approach has helped identify meaningful, replicable outcome measures, 22,23 and published minimum standards help promote appropriate uses of these PRO measures. 25 This approach has also been applied in developing and validating scales for a number of facial conditions, including the FACE-Q for aesthetic facial procedures 6,7,26 and the CLEFT-Q for cleft lip and palate.…”

“…25 This approach has also been applied in developing and validating scales for a number of facial conditions, including the FACE-Q for aesthetic facial procedures 6,7,26 and the CLEFT-Q for cleft lip and palate. 24 Some have suggested that the FACE-Q might eventually be adapted for use in FT, 27 but existing PRO measures, such as those used in FACE-Q, have not been validated in the FT population due in part to small numbers of FT candidates and recipients. Eventually, as more procedures are undertaken, PRO measures might be developed for FT. A standardized PRO instrument for FT would help support the procedure's eventual transition from research to standard of care and would help facilitate reimbursement by third-party payers.…”