NOVEL MEASUREMENT STRATEGIES FOR COLLECTION OF CLINICAL OUTCOME ASSESSMENTS IN A PEDIATRIC POPULATION
Author(s)
Turner-Bowker D1, Krohe M1, Yaworsky A1, Kelly M1, Padilla B1, Chaston E1, Radin A2, Mastey V2, Nadeau KC3, Jalbert J2
1Adelphi Values, Boston, MA, USA, 2Regeneron, Tarrytown, NY, USA, 3Stanford University, Stanford, CA, USA
Presentation Documents
OBJECTIVES: To describe a novel measurement strategy for clinical outcome assessments (COAs) developed to assess side effects of peanut oral immunotherapy treatment (OIT) in pediatric patients. METHODS: Qualitative patient interviews were conducted as part of a broader study to develop a content-valid electronic COA, the Side Effects of Peanut OIT Diary (SEPOD), to assess daily allergic side effects of peanut OIT for desensitization of peanut allergy in children (6-17 years). We conducted 2 waves of in-person interviews with pediatric patients with clinically-documented sensitivity to peanut allergen. Patients aged 6-12 years were interviewed with their caregiver. All interviews included a cognitive debriefing (CD) component evaluating the patient’s ability to understand and respond to the SEPOD. The overall COA measurement strategy and SEPOD revisions were considered after each wave. RESULTS: The initial measurement approach tested 2 SEPOD versions: a patient-reported outcome (PRO) version for children 12-17 years and a caregiver-administered PRO (CarePRO) version for children 6-11 years. While items were identical, the CarePRO version included caregiver instructions for questionnaire administration and verbatim instructions to be read to the child. The pediatric patient was expected to respond independently in both versions. Results from CD with 24 patients indicated that some younger children (≤8 years) had difficulty understanding questions even when read aloud. To address this, a caregiver-administered outcome (CareO) version was developed for children 6-8 years. The CarePRO and CareO are similar except that the CareO allows for caregiver (observer) report when needed and appropriate and records who (child or caregiver) responded to diary questions. CONCLUSIONS: Measurement strategies offering easily adaptable solutions, such as alleviation of reading burden and options for observer-reported outcome assessment, can benefit pediatric COA.
Conference/Value in Health Info
2019-05, ISPOR 2019, New Orleans, LA, USA
Value in Health, Volume 22, Issue S1 (2019 May)
Code
PIH8
Topic
Clinical Outcomes, Methodological & Statistical Research, Patient-Centered Research
Topic Subcategory
Clinical Outcomes Assessment, Instrument Development, Validation, & Translation, Patient-reported Outcomes & Quality of Life Outcomes, PRO & Related Methods
Disease
Pediatrics