Study protocol to develop a patient-reported outcome measuring disability associated with unilateral vocal fold paralysis: a mixed-methods approach with the CoPE collaborative

Abstract

IntroductionPatient-reported outcome (PRO) measures are increasingly developed with multisite, representative patient populations so that they can serve as a primary endpoint in clinical trials and longitudinal studies. Creating multisite infrastructure during PRO measure development can facilitate future comparative effectiveness trials. We describe our protocol to simultaneously develop a PRO measure and create a collaborative of tertiary care centres to address the needs of patients with unilateral vocal fold paralysis (UVFP). We describe the stakeholder engagement, information technology and regulatory foundations for PRO measure development and how the process enables plans for multisite trials comparing treatments for this largely iatrogenic condition.Methods and analysisThe study has three phases: systematic review, measure development and measure validation. Systematic reviews and qualitative interviews (n=75) will inform the development of a conceptual framework. Qualitative interviews with patients with UVFP will characterise the lived experience of the condition. Candidate PRO measure items will be derived verbatim from patient interviews and refined using cognitive interviews and expert input. The PRO measure will be administered to a large, multisite cohort of adult patients with UVFP via the CoPE (vocal Cord Paralysis Experience) Collaborative. We will establish CoPE to facilitate measure development and to create preliminary infrastructure for future trials, including online data capture, stakeholder engagement, and the identification of barriers and facilitators to participation. Classical test theory psychometrics and grounded theory characterise our approach, and validation includes assessment of latent structure, reliability and validity.Ethics and disseminationOur study is approved by the University of Wisconsin Health Sciences Institutional Review Board. Findings from this project will be published in open-access journals and presented at international conferences. Subsequent use of the PRO measure will include comparative effectiveness trials of treatments for UVFP at CoPE Collaborative sites.

Multicenter Development and Validation of the Vocal Cord Paralysis Experience (CoPE), a Patient-Reported Outcome Measure for Unilateral Vocal Fold Paralysis–Specific Disability

“…The analytical plan is consistent with reporting standards for health questionnaire measurement and is described in the published protocol Table 1. contains the definitions for each psychometric analysis and measures used, including changes to the analysis made since publication of the protocol.…”

“…The conceptual framework was informed by a systematic review of existing literature on UVFP in conjunction with interview data from patients with UVFP . We include identified risk factors (eg, sociodemographic characteristics and comorbidities), surgical risk factors (eg, specific procedures, complications), and anatomical characteristics that have the potential to increase UVFP risk.…”

“…Details of the study protocol, inclusion criteria, study design, item development, cognitive interviews, and conceptual model have been published . Consequently, the methods are described in brief, and we focus on results of analyses.…”

How Do Academic Otolaryngologists Decide to Implement New Procedures Into Practice?

“…Such collaboratives are increasingly common, thereby conferring significant opportunity to disseminate best practices, encourage multidisciplinary collaboration, and improve care. [12][13][14][15][16][17][18][19] Formation of research consortia suggests a cultural trend toward collaborative dissemination of new information. However, little research describes how subspecialty surgeons adopt new procedures and whether they decide to sustain those changes.…”

“…Because otolaryngology communities are small, comparative effectiveness research often requires multi‐institutional collaboration to attain analytic sample sizes. Such collaboratives are increasingly common, thereby conferring significant opportunity to disseminate best practices, encourage multidisciplinary collaboration, and improve care 12‐19 . Formation of research consortia suggests a cultural trend toward collaborative dissemination of new information.…”