Development and Validation of the Symptom Assessment for Bronchiectasis (SABRE) for the FDA Qualification Program: A Novel Patient-Reported Outcome Instrument to Measure Symptoms of Non-Cystic Fibrosis Bronchiectasis
Author(s)
Kevin C. Mange, MD, MSCE1, Daniel Serrano, PhD2, Kelly McCarrier, PhD, MPH3, Mariam Hassan, PhD, B.Pharm1, Medha Satyal, PhD3, Lauren Podger, MSc2, Sam Parsons, PhD4, Nancy Touba, MPH4, Jui-Hua Tsai, MD3, Charles L. Daley, MD5.
1Insmed Incorporated, Bridgewater, NJ, USA, 2The Psychometrics Team, London, United Kingdom, 3OPEN Health, Bethesda, MD, USA, 4OPEN Health, London, United Kingdom, 5National Jewish Health and the University of Colorado School of Medicine, Denver, CO, USA.
1Insmed Incorporated, Bridgewater, NJ, USA, 2The Psychometrics Team, London, United Kingdom, 3OPEN Health, Bethesda, MD, USA, 4OPEN Health, London, United Kingdom, 5National Jewish Health and the University of Colorado School of Medicine, Denver, CO, USA.
OBJECTIVES: To develop a novel patient-reported outcome (PRO) instrument to measure symptoms in patients with Non-Cystic Fibrosis Bronchiectasis (NCFBE) with and without Non-Tuberculosis Mycobacterial (NTM) lung infection. The study was conducted during the COVID-19 pandemic; challenges posed and solutions employed will be discussed.
METHODS: In Phase 1, qualitative concept elicitation (CE; N=40) and cognitive interviews (CI; N=20) with participants diagnosed with NCFBE were conducted to characterize patient-experienced symptoms, support item generation, and to evaluate the comprehension and relevance of the draft SABRE’s content. In Phase 2, a non-interventional validation study (NIVS) was conducted to evaluate the psychometric properties of the draft SABRE. Modern psychometric methods (MPMs) were employed to test structural validity. Classical methods evaluated the reliability and validity of SABRE scores at baseline (N=179). Test-retest reliability (TRTR) was estimated via intraclass correlation coefficient (ICC[2,1]) among stable participants (no change in patient global impression of frequency [PGI-F] in the 14-day retest interval; N=175).
RESULTS: CE interviews conducted to concept saturation supported the relevance of a daily frequency metric for 5 key NCFBE symptoms: cough, sputum/mucus production, chest congestion, shortness of breath, and fatigue. CIs confirmed the relevance of symptoms, comprehension of item wording, and interpretability of a 5-point verbal response scale (“never” to “almost always”) for all items.MPMs supported a weekly average total sum score for the SABRE. The SABRE demonstrated strong internal consistency (α: 0.78), TRTR (ICC[2,1]: 0.87), and convergent validity (range: 0.68 - 0.79). Known-groups validity was demonstrated across PGI-F groups.
CONCLUSIONS: The qualitative study established the content validity of the SABRE. The NIVS demonstrated that the SABRE has strong reliability and validity for assessing symptoms in patients with NCFBE with and without NTM lung infection. Future research will examine SABRE responsiveness to support SABRE as an endpoint in clinical trials of NCFBE.
METHODS: In Phase 1, qualitative concept elicitation (CE; N=40) and cognitive interviews (CI; N=20) with participants diagnosed with NCFBE were conducted to characterize patient-experienced symptoms, support item generation, and to evaluate the comprehension and relevance of the draft SABRE’s content. In Phase 2, a non-interventional validation study (NIVS) was conducted to evaluate the psychometric properties of the draft SABRE. Modern psychometric methods (MPMs) were employed to test structural validity. Classical methods evaluated the reliability and validity of SABRE scores at baseline (N=179). Test-retest reliability (TRTR) was estimated via intraclass correlation coefficient (ICC[2,1]) among stable participants (no change in patient global impression of frequency [PGI-F] in the 14-day retest interval; N=175).
RESULTS: CE interviews conducted to concept saturation supported the relevance of a daily frequency metric for 5 key NCFBE symptoms: cough, sputum/mucus production, chest congestion, shortness of breath, and fatigue. CIs confirmed the relevance of symptoms, comprehension of item wording, and interpretability of a 5-point verbal response scale (“never” to “almost always”) for all items.MPMs supported a weekly average total sum score for the SABRE. The SABRE demonstrated strong internal consistency (α: 0.78), TRTR (ICC[2,1]: 0.87), and convergent validity (range: 0.68 - 0.79). Known-groups validity was demonstrated across PGI-F groups.
CONCLUSIONS: The qualitative study established the content validity of the SABRE. The NIVS demonstrated that the SABRE has strong reliability and validity for assessing symptoms in patients with NCFBE with and without NTM lung infection. Future research will examine SABRE responsiveness to support SABRE as an endpoint in clinical trials of NCFBE.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
PCR164
Topic
Patient-Centered Research
Disease
SDC: Rare & Orphan Diseases, SDC: Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)