CONTENT VALIDITY EVALUATION OF SELECTED PATIENT REPORTED OUTCOME MEASURES FOR PAIN, FATIGUE, AND PHYSICAL FUNCTIONING IN ADULTS, ADOLESCENTS AND CHILDREN LIVING WITH OSTEOGENESIS IMPERFECTA
Author(s)
Elizabeth Gibbons, MSc1, laura kelly, DPhil1, Savita Bakhshi Anand, PhD1, STEPHANIE BASCLE, PharmD2, Joshua Maher, PhD3, Tracy Hart, MSc4, Cemre Robinson, PhD5, Ethen Cheng, PhD6, Ingunn Westerheim, MSc7.
1Evidera Thermofisher, London, United Kingdom, 2SANOFI RESEARCH & DEVELOPMENT, Gentilly, France, 3None, None, United Kingdom, 4OIF, Gaithersburg, MD, USA, 5SANOFI RESEARCH & DEVELOPMENT, London, United Kingdom, 6SANOFI, London, United Kingdom, 7OIFE, OSLO, Norway.
1Evidera Thermofisher, London, United Kingdom, 2SANOFI RESEARCH & DEVELOPMENT, Gentilly, France, 3None, None, United Kingdom, 4OIF, Gaithersburg, MD, USA, 5SANOFI RESEARCH & DEVELOPMENT, London, United Kingdom, 6SANOFI, London, United Kingdom, 7OIFE, OSLO, Norway.
OBJECTIVES: We aimed to evaluate the content validity of selected patient-reported outcome (PRO) instruments focusing on pain, fatigue, and physical functioning among adults (≥18 years), adolescents (12 to 17 years), and children (8 to 11 years) living with osteogenesis imperfecta (OI).
METHODS: A patient advisory board (PAB) informed the research design and data interpretation. Participants living with OI in the US and Spain were recruited through patient advocacy groups and qualitative interviews were conducted. PRO instruments debriefed included: Patient-reported Outcomes Measurement Information System (PROMIS)-29 (adults); Pediatric Quality of Life Inventory (PedsQL) Pediatric Pain Questionnaire, Teen Form (13 to 18 years); PedsQL Pediatric Pain Questionnaire, Child Form (8 to 12 years); PROMIS Pediatric Pain Interference Short Form v2.0 (8 to 17 years). Anonymized interview transcripts were analyzed using ATLAS.ti©.
RESULTS: Seven adults, seven adolescents, and six children participated in the interviews. Adults understood the PROMIS-29 instructions and items were considered relevant to their OI experiences. However, the 7-day recall period was not always adhered to and some indicated there was little distinction between "somewhat" and "a little bit" response options for the fatigue domain. Feedback from children and adolescents regarding the PedsQL Pediatric Pain Questionnaires suggested that, while the questionnaire items were generally well understood, younger children found it challenging to complete independently. Responses on the PROMIS Pediatric Pain Interference Short-Forms indicated that the forms appear to be a pragmatic solution to capture the impact of pain in this population.
CONCLUSIONS: The PROs were generally understandable and relevant to participants. However, issues relating to the recall period for the PROMIS-29 and younger children needing support and clarity when completing the PedsQL Questionnaires should be carefully considered when including people living with OI in clinical trials. Engagement with the PAB ensured that the perspectives of individuals and families affected by OI were incorporated.
METHODS: A patient advisory board (PAB) informed the research design and data interpretation. Participants living with OI in the US and Spain were recruited through patient advocacy groups and qualitative interviews were conducted. PRO instruments debriefed included: Patient-reported Outcomes Measurement Information System (PROMIS)-29 (adults); Pediatric Quality of Life Inventory (PedsQL) Pediatric Pain Questionnaire, Teen Form (13 to 18 years); PedsQL Pediatric Pain Questionnaire, Child Form (8 to 12 years); PROMIS Pediatric Pain Interference Short Form v2.0 (8 to 17 years). Anonymized interview transcripts were analyzed using ATLAS.ti©.
RESULTS: Seven adults, seven adolescents, and six children participated in the interviews. Adults understood the PROMIS-29 instructions and items were considered relevant to their OI experiences. However, the 7-day recall period was not always adhered to and some indicated there was little distinction between "somewhat" and "a little bit" response options for the fatigue domain. Feedback from children and adolescents regarding the PedsQL Pediatric Pain Questionnaires suggested that, while the questionnaire items were generally well understood, younger children found it challenging to complete independently. Responses on the PROMIS Pediatric Pain Interference Short-Forms indicated that the forms appear to be a pragmatic solution to capture the impact of pain in this population.
CONCLUSIONS: The PROs were generally understandable and relevant to participants. However, issues relating to the recall period for the PROMIS-29 and younger children needing support and clarity when completing the PedsQL Questionnaires should be carefully considered when including people living with OI in clinical trials. Engagement with the PAB ensured that the perspectives of individuals and families affected by OI were incorporated.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PT13
Topic
Clinical Outcomes, Health Policy & Regulatory, Patient-Centered Research
Topic Subcategory
Instrument Development, Validation, & Translation, Patient Engagement, Patient-reported Outcomes & Quality of Life Outcomes
Disease
Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal), Pediatrics, Rare & Orphan Diseases