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.
Development of the sporadic inclusion body myositis physical functioning assessment (sIFA)