title: Development of the sporadic inclusion body myositis physical functioning assessment.
abstract: Sporadic inclusion body myositis (sIBM) is a progressive idiopathic inflammatory myopathy characterized by atrophy and weakness of proximal and distal muscle groups that results 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.
  
  
  
  The tool was rigorously developed in accordance with the United States Food and Drug Administration (FDA) patient-reported outcomes (PRO) guidance. A single-visit, observational study was conducted. Standard qualitative analytical methods were employed to analyze interview data and generate questionnaire items.
  
  
  
  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.
  
  
  
  We have developed a PRO tool in alignment with FDA PRO guidance for use in the functional assessment of treatment benefit in sIBM. Muscle Nerve, 2016 Muscle Nerve 54: -, 2016 Muscle Nerve 54: 653-657, 2016.
authors.affiliation: RTI Health Solutions, Research Triangle Park, 200 Park Offices Drive, North Carolina, 27709, USA. demuromercon@rti.org.
authors.forename: Carla
authors.initials: C
authors.lastname: DeMuro
authors.affiliation: RTI Health Solutions, Research Triangle Park, 200 Park Offices Drive, North Carolina, 27709, USA.
authors.forename: Sandy
authors.initials: S
authors.lastname: Lewis
authors.affiliation: Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
authors.forename: Linda
authors.initials: L
authors.lastname: Lowes
authors.affiliation: Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.
authors.forename: Lindsay
authors.initials: L
authors.lastname: Alfano
authors.affiliation: Novartis Pharmaceuticals, Basel, Switzerland.
authors.forename: Brian
authors.initials: B
authors.lastname: Tseng
authors.affiliation: RTI Health Solutions, Research Triangle Park, 200 Park Offices Drive, North Carolina, 27709, USA.
authors.forename: Ari
authors.initials: A
authors.lastname: Gnanasakthy
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D006801:Humans; D008875:Middle Aged; D018979:Myositis, Inclusion Body / Q000175:diagnosis* / Q000503:physiopathology; D010808:Physical Examination / Q000379:methods* / Q000592:standards; D057566:Self Report / Q000592:standards; D011795:Surveys and Questionnaires / Q000592:standards
keywords: development; patient-reported outcomes; physical functioning; qualitative research; sporadic inclusion body myositis