Evaluating Thresholds for Minimal Clinically Important Difference (MCID) in Patient-Reported Outcome Measures (PROMs) in Locally Advanced or Metastatic Urothelial Cancer (la/mUC): a Systematic Literature Review (SLR)
Author(s)
Mairead Kearney, MB BCh, MPH, MBA, MSc. Econ1, Thomas Macmillan, MSc2, Julia Poritz, PhD2, Sherrie Schreiber-Gosche, MSC2, Mihaela G. Musat, PhD2;
1the healthcare business of Merck KGaA, Darmstadt, Germany, 2Cytel, Cambridge, MA, USA
1the healthcare business of Merck KGaA, Darmstadt, Germany, 2Cytel, Cambridge, MA, USA
Presentation Documents
OBJECTIVES: Increased use of PROMs in clinical research and policy-making makes their reliable interpretation imperative. Suitable reference values for MCID thresholds facilitate precise interpretation of changes in patients’ health-related quality of life (HRQOL), guiding treatment decisions and improving care.
METHODS: An SLR of interventional and observational studies reporting PROMs in la/mUC was conducted (May 29, 2024) to identify MCID thresholds used at the item, domain, and total-score levels. We further analyzed HRQOL outcomes of studies meeting MCID thresholds for between-group differences and longitudinal individual-patient changes.
RESULTS: The SLR identified 49 studies (37 clinical trials; 12 RWE studies). MCID thresholds were only reported in 32.7% of trials (11/37) and 0% of RWE studies. EORTC QLQ-C30 and EQ-5D were the most commonly reported PROMs. The MCID threshold for EORTC QLQ-C30 (single items, subscales) was 10 points in nine studies and 5-10 points in one study. Five studies concluded that clinically meaningful change occurred. The MCID for EQ-5D visual analog score was considered 7 points in three studies and 7-12 or ≥15 points in two studies (threshold not met in any study). The reported MCID for EQ-5D utility index was 0.09-0.12 or ≥0.8 points in two studies (threshold not met). Only two studies used MCID thresholds to interpret between-group differences (per EORTC guidelines). Other studies did not distinguish between thresholds for between-group differences and longitudinal individual-patient changes. For the EORTC QLQ-C30 questionnaire, all studies applied the 10-point threshold across domains.
CONCLUSIONS: In addition to underreporting of MCID thresholds, we found inconsistency and heterogeneity in definitions of thresholds for the same PROM when applied in clinical trials, hindering the ability to interpret and compare PROMs between studies. Future research and collaboration should focus on defining and implementing consistent MCID thresholds to reliably capture patient perspective on the effectiveness of different treatments.
METHODS: An SLR of interventional and observational studies reporting PROMs in la/mUC was conducted (May 29, 2024) to identify MCID thresholds used at the item, domain, and total-score levels. We further analyzed HRQOL outcomes of studies meeting MCID thresholds for between-group differences and longitudinal individual-patient changes.
RESULTS: The SLR identified 49 studies (37 clinical trials; 12 RWE studies). MCID thresholds were only reported in 32.7% of trials (11/37) and 0% of RWE studies. EORTC QLQ-C30 and EQ-5D were the most commonly reported PROMs. The MCID threshold for EORTC QLQ-C30 (single items, subscales) was 10 points in nine studies and 5-10 points in one study. Five studies concluded that clinically meaningful change occurred. The MCID for EQ-5D visual analog score was considered 7 points in three studies and 7-12 or ≥15 points in two studies (threshold not met in any study). The reported MCID for EQ-5D utility index was 0.09-0.12 or ≥0.8 points in two studies (threshold not met). Only two studies used MCID thresholds to interpret between-group differences (per EORTC guidelines). Other studies did not distinguish between thresholds for between-group differences and longitudinal individual-patient changes. For the EORTC QLQ-C30 questionnaire, all studies applied the 10-point threshold across domains.
CONCLUSIONS: In addition to underreporting of MCID thresholds, we found inconsistency and heterogeneity in definitions of thresholds for the same PROM when applied in clinical trials, hindering the ability to interpret and compare PROMs between studies. Future research and collaboration should focus on defining and implementing consistent MCID thresholds to reliably capture patient perspective on the effectiveness of different treatments.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
PCR237
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Oncology