Development of the sporadic inclusion body myositis physical functioning assessment (sIFA)
DeMuro, C., Lewis, S., Lowes, L., Alfano, L., Tseng, B., & Gnanasakthy, A. (2016). Development of the sporadic inclusion body myositis physical functioning assessment (sIFA). Muscle & Nerve, Advance Online Publication. DOI: 10.1002/mus.25079
INTRODUCTION: Sporadic inclusion body myositis (sIBM) is a progressive idiopathic inflammatory myopathy characterized by atrophy and weakness of proximal and distal muscle groups resulting in a loss of independence and the need for assistive devices and supportive care. To assess treatment benefit of new therapies, a patient-reported outcome measure of physical function was developed. METHODS: The tool was rigorously developed in accordance with the U.S. Food and Drug Administration (FDA) Patient Report Outcomes (PRO) Guidance. A single-visit, observational study was conducted. Standard qualitative analytical methods were employed to analyze interview data and generate questionnaire items. RESULTS: Twenty concept elicitation and 19 cognitive debriefing interviews were conducted, and 6 expert physicians were consulted. The tool consists of 11 items scored on a 0-10 numerical rating scale. Subjects completed the questionnaire utilizing either paper or electronic administration. DISCUSSION: We have developed a PRO tool in alignment with FDA PRO Guidance for use in the functional assessment of treatment benefit in sIBM. This article is protected by copyright. All rights reserved.