REGULATORY/HTA FRAMING PATIENT-REPORTED OUTCOME INSTRUMENTS IN ULCERATIVE COLITIS CLINICAL TRIALS: FREQUENCY OF USE AND PSYCHOMETRIC EVIDENCE
Author(s)
Kevin Kallmes, MA, JD1, Devendra Patil, MS2, Chong H Kim, MPH, MS, PhD3.
1Nested Knowledge, St. Paul, MN, USA, 2Massachusetts College of Pharmacy and Health Sciences, Boston, MA, USA, 3Gilead, Foster City, CA, USA.
1Nested Knowledge, St. Paul, MN, USA, 2Massachusetts College of Pharmacy and Health Sciences, Boston, MA, USA, 3Gilead, Foster City, CA, USA.
OBJECTIVES: This targeted literature review (TLR) aimed to identify patient-reported outcome (PRO) instruments used in ulcerative colitis (UC) clinical trials (2021-2026) and to evaluate psychometric validation evidence for newly identified PRO instruments.
METHODS: A TLR was conducted on the Nested Knowledge platform across two streams. Stream 1 searched ClinicalTrials.gov for interventional UC clinical trials (all phases, 2021-2026). Studies included if they enrolled adults with UC, evaluated a pharmacological intervention, and reported at least one PRO endpoint; 51 studies met final inclusion criteria. Stream 2 conducted targeted PubMed searches for psychometric validation studies for each newly identified PRO instrument; 17 studies were included. Data extraction covered study design, phase, drug class, UC severity, sample size, demographics, and PRO instruments. Validation data captured content validity, construct validity, reliability, responsiveness, and MCID/MID.
RESULTS: Twenty-four unique PRO instruments were identified across 51 UC clinical trials. The most frequently used were PRO-2/Mayo PRO sub-scores (n=32, 63%), IBDQ (n=18, 35%), Abdominal Pain NRS (n=7, 14%), U-NRS (n=6, 12%), and FACIT-Fatigue (n=5, 10%). Bowel urgency emerged as a key patient-centric endpoint, assessed via U-NRS and BUF across multiple Phase 3 trials. Five novel UC-specific PROs were identified: U-NRS, UC-PROSS, TUMMY-UC, PRO-UC Diary, and BUF. The U-NRS was the most comprehensively validated new instrument (5 studies; ICC=0.768-0.877; MCID ≥3 points). PROMIS-Fatigue SF-7a demonstrated full psychometric evidence including MCID ≥7 points. Critical evidence gaps were identified for Abdominal Pain NRS (7 trials; no UC-specific validation) and BUF (no formal psychometric studies).
CONCLUSIONS: Twenty-four unique PROs were identified across 51 UC clinical trials from 2021-2026. PRO-2 and IBDQ remain the most widely used instruments. Bowel urgency has emerged as a priority outcome domain, with the U-NRS now comprehensively validated. Important validation gaps remain for Abdominal Pain NRS and BUF, representing priority areas for future PRO qualification work in UC.
METHODS: A TLR was conducted on the Nested Knowledge platform across two streams. Stream 1 searched ClinicalTrials.gov for interventional UC clinical trials (all phases, 2021-2026). Studies included if they enrolled adults with UC, evaluated a pharmacological intervention, and reported at least one PRO endpoint; 51 studies met final inclusion criteria. Stream 2 conducted targeted PubMed searches for psychometric validation studies for each newly identified PRO instrument; 17 studies were included. Data extraction covered study design, phase, drug class, UC severity, sample size, demographics, and PRO instruments. Validation data captured content validity, construct validity, reliability, responsiveness, and MCID/MID.
RESULTS: Twenty-four unique PRO instruments were identified across 51 UC clinical trials. The most frequently used were PRO-2/Mayo PRO sub-scores (n=32, 63%), IBDQ (n=18, 35%), Abdominal Pain NRS (n=7, 14%), U-NRS (n=6, 12%), and FACIT-Fatigue (n=5, 10%). Bowel urgency emerged as a key patient-centric endpoint, assessed via U-NRS and BUF across multiple Phase 3 trials. Five novel UC-specific PROs were identified: U-NRS, UC-PROSS, TUMMY-UC, PRO-UC Diary, and BUF. The U-NRS was the most comprehensively validated new instrument (5 studies; ICC=0.768-0.877; MCID ≥3 points). PROMIS-Fatigue SF-7a demonstrated full psychometric evidence including MCID ≥7 points. Critical evidence gaps were identified for Abdominal Pain NRS (7 trials; no UC-specific validation) and BUF (no formal psychometric studies).
CONCLUSIONS: Twenty-four unique PROs were identified across 51 UC clinical trials from 2021-2026. PRO-2 and IBDQ remain the most widely used instruments. Bowel urgency has emerged as a priority outcome domain, with the U-NRS now comprehensively validated. Important validation gaps remain for Abdominal Pain NRS and BUF, representing priority areas for future PRO qualification work in UC.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HTA330
Topic
Health Policy & Regulatory, Health Technology Assessment, Patient-Centered Research
Topic Subcategory
Decision & Deliberative Processes
Disease
Biologics & Biosimilars, Gastrointestinal Disorders, Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)