A PRACTICAL FRAMEWORK FOR INCLUDING DIETARY DATA IN REAL-WORLD EVIDENCE STUDIES: LESSONS FROM INFLAMMATORY BOWEL DISEASE AND PEDIATRIC RARE DISEASES
Author(s)
Neil R. Brett, PhD1, Garthiga Manickam, PhD2, Payal Pozin, PhD3, Elizabeth Donahue, BSc4, Susan A. Oliveria, MPH, ScD3, John S. Sampalis, PhD1.
1PPD™ Observational Studies, Thermo Fisher Scientific, Montreal, QC, Canada, 2PPD™ Evidera™ Health Economics and Market Access, Thermo Fischer Scientific, Montreal, QC, Canada, 3PPD™ Observational Studies, Thermo Fisher Scientific, New York, NY, USA, 4PPD™ Observational Studies, Thermo Fisher Scientific, Boston, MA, USA.
1PPD™ Observational Studies, Thermo Fisher Scientific, Montreal, QC, Canada, 2PPD™ Evidera™ Health Economics and Market Access, Thermo Fischer Scientific, Montreal, QC, Canada, 3PPD™ Observational Studies, Thermo Fisher Scientific, New York, NY, USA, 4PPD™ Observational Studies, Thermo Fisher Scientific, Boston, MA, USA.
OBJECTIVES: Dietary intake influences disease activity, treatment response, and symptom burden in inflammatory bowel disease (IBD) and many pediatric rare diseases, but prior literature reviews showed dietary data are infrequently collected in real-world evidence (RWE) studies assessing treatment outcomes. We developed/applied a practical framework to guide future dietary data inclusion in RWE studies, using examples from IBD and pediatric rare diseases.
METHODS: Targeted literature reviews in IBD and pediatric rare diseases, together with methodological/operational considerations from nutrition science, clinical practice, and RWE design, informed a five-pillar framework for RWE dietary data collection. Eight studies (1 IBD, 7 pediatric rare disease, 2010-2024) collecting treatment outcomes and dietary data were identified and evaluated against the framework by two independent reviewers using predefined high, moderate, and low alignment criteria, with discrepancies resolved by consensus.
RESULTS: The framework comprises five pillars: (1) Objective alignment—matching dietary variables to research questions; (2) Data source strategy—leveraging existing data and supplementing when needed; (3) Fit‑for‑purpose method selection— choosing variables collection methods; (4) Operational feasibility/burden minimization—reducing patient/caregiver burden while considering demographics, dietitian support; and (5) Technology‑enablement—using guided data entry platforms, voice transcription, AI‑assisted image recognition, etc. For studies, alignment varied across pillars: objective alignment (8/8 high), data source strategy (5/8 low), methods selection (7/8 high), operational feasibility (5/8 moderate), and technology‑enablement (8/8 low). Operational complexity was greater in pediatric rare disease studies than the IBD study because of parent/caregiver-reported data; however, some rare-disease settings offer opportunities to leverage dietary data already captured in medical records.
CONCLUSIONS: This practical framework provides a simple structure to help guide designing dietary data collection in IBD and pediatric rare disease studies, and potentially can apply to other therapeutic areas. Results highlight a need for further developing the framework, and integrating data collection technology into future studies, to lower patient/caregiver burden and increase data quality.
METHODS: Targeted literature reviews in IBD and pediatric rare diseases, together with methodological/operational considerations from nutrition science, clinical practice, and RWE design, informed a five-pillar framework for RWE dietary data collection. Eight studies (1 IBD, 7 pediatric rare disease, 2010-2024) collecting treatment outcomes and dietary data were identified and evaluated against the framework by two independent reviewers using predefined high, moderate, and low alignment criteria, with discrepancies resolved by consensus.
RESULTS: The framework comprises five pillars: (1) Objective alignment—matching dietary variables to research questions; (2) Data source strategy—leveraging existing data and supplementing when needed; (3) Fit‑for‑purpose method selection— choosing variables collection methods; (4) Operational feasibility/burden minimization—reducing patient/caregiver burden while considering demographics, dietitian support; and (5) Technology‑enablement—using guided data entry platforms, voice transcription, AI‑assisted image recognition, etc. For studies, alignment varied across pillars: objective alignment (8/8 high), data source strategy (5/8 low), methods selection (7/8 high), operational feasibility (5/8 moderate), and technology‑enablement (8/8 low). Operational complexity was greater in pediatric rare disease studies than the IBD study because of parent/caregiver-reported data; however, some rare-disease settings offer opportunities to leverage dietary data already captured in medical records.
CONCLUSIONS: This practical framework provides a simple structure to help guide designing dietary data collection in IBD and pediatric rare disease studies, and potentially can apply to other therapeutic areas. Results highlight a need for further developing the framework, and integrating data collection technology into future studies, to lower patient/caregiver burden and increase data quality.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
SA48
Topic
Epidemiology & Public Health, Patient-Centered Research, Study Approaches
Disease
Gastrointestinal Disorders, Nutrition, Pediatrics, Rare & Orphan Diseases