THE EXACT IN IDIOPATHIC PULMONARY FIBROSIS (IPF)- CONTENT VALIDITY OF AN EXISTING PATIENT-REPORTED OUTCOME MEASURE FOR USE IN A RARE DISEASE
Author(s)
Murray L*1;Vernon M2;O'Quinn S3;Martinez F4;Parker JM3, Leidy NK1 1United BioSource Corporation, Bethesda, MD, USA, 2United BioSource Corporation, London, United Kingdom, 3MedImmune, Gaithersburg, MD, USA, 4University of Michigan, Ann Arbor, MI, USA
OBJECTIVES: IPF is a rare, progressive and ultimately fatal, pulmonary disease. New treatments are needed to relieve symptoms. Characteristic symptoms of IPF include breathlessness on exertion and cough. No instrument to quantify the nature, severity, and pattern of daily respiratory symptoms in IPF patients or the effect of treatment on these outcomes exists. The EXACT, a PRO instrument, assesses respiratory symptoms in patients with chronic obstructive pulmonary disease. This study examined the extent to which the EXACT might be useful as a symptom outcome measure in IPF. METHODS: Individual interviews were performed in two phases. During phase one, participants were recruited through IPF patient support groups and associations (n=20). In phase two, patients with high resolution computer tomography and/or biopsy-confirmed IPF were enrolled through an academic pulmonary clinic (n=10). All patients completed a telephone interview, including concept elicitation of daily IPF symptoms followed by a cognitive interview with the EXACT. RESULTS: The study included 30 IPF patients in the US; mean (SD) age=68.0 (8.56) years; 40% female; 90% white. Mean (SD) FVC % predicted=57.5 (9.37); years since IPF diagnosis=2.5 (1.84). Across all interviews, respiratory symptoms identified during concept elicitation mapped to each of the respiratory items comprising the EXACT; no new symptoms were identified. An unexpected result was the prevalence and relevance of sputum production as a symptom. During cognitive interviews, participants found the items clear, interpretable, and relevant. CONCLUSIONS: Results of this qualitative study suggest EXACT items may be suitable for assessing respiratory symptoms in clinical studies of IPF patients. Next steps include quantitative research to determine structure and scoring of the EXACT-IPF and score reliability, validity, and responsiveness. Adapting an existing PRO instrument can be an efficient and effective method for overcoming challenges associated with instrument development in rare disease populations.
Conference/Value in Health Info
2013-05, ISPOR 2013, New Orleans, LA, USA
Value in Health, Vol. 16, No. 3 (May 2013)
Code
PRS40
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Respiratory-Related Disorders