EVALUATING CLINICAL OUTCOME ASSESSMENTS IN LOW-RISK MYELODYSPLASTIC SYNDROMES THROUGH DEVELOPMENT OF A COMPREHENSIVE CONCEPTUAL MODEL BASED ON TARGETED LITERATURE REVIEWS AND CLINICIAN INTERVIEWS
Author(s)
Anna P. Cardellino, BS, MPH1, FULYA SEN NIKITAS, MSc2, Shiyuan Zhang, BSc, MSc3, Mariel Alcantara Fernandez, MD4, Shweta Shah, PhD5, Betsy N. Williams, PhD6, Julie R. Bailey, MBA7, Valeria Santini, MD8, Andrew Brunner, MD9, Amer Zeidan, MD10;
1GSK, Philadelphia, PA, USA, 2GSK, LONDON, United Kingdom, 3GSK, Rockville, MD, USA, 4IQVIA, Mexico City, Mexico, 5IQVIA, Lenexa, KS, USA, 6IQVIA, Ridgewood, NJ, USA, 7IQVIA, New York, NY, USA, 8University of Florence, Florence, Italy, 9Massachusetts General Hospital, Boston, MA, USA, 10Yale, New Haven, CT, USA
1GSK, Philadelphia, PA, USA, 2GSK, LONDON, United Kingdom, 3GSK, Rockville, MD, USA, 4IQVIA, Mexico City, Mexico, 5IQVIA, Lenexa, KS, USA, 6IQVIA, Ridgewood, NJ, USA, 7IQVIA, New York, NY, USA, 8University of Florence, Florence, Italy, 9Massachusetts General Hospital, Boston, MA, USA, 10Yale, New Haven, CT, USA
OBJECTIVES: The aim of this study was to develop a preliminary conceptual model (PCM) for low-risk myelodysplastic syndromes (LR-MDS) and evaluate the ability of current clinical outcome assessments (COAs) to adequately capture patient experiences.
METHODS: Two targeted literature reviews (TLRs) were conducted to (1) develop a PCM documenting a comprehensive set of signs/symptoms and impacts specific to LR-MDS and (2) evaluate the landscape of COAs used in LR-MDS clinical trials and regulatory labels based on searches of OVID/ClinicalTrials.gov/PROQOLID/PROLABELS. A gap analysis compared the identified COAs against items in the PCM, considering content validity and psychometrics. Three expert clinician interviews were conducted to refine the initial PCM and prioritize concepts important to patients.
RESULTS: A total of 46 signs/symptoms and 40 impacts were included in the initial PCM based on TLR of 8 peer-reviewed articles and 7 patient blogs; fatigue, dyspnea, anemia, and social impacts were most common. Among the 10 COAs identified, 5 demonstrated moderate coverage of the PCM (FACT-An, 45% concepts covered; EORTC QLQ-C30, 38% concepts covered; QOL-E, 26% concepts covered; MDASI- AML/MDS, 31% concepts covered; and QUALMS, 26% concepts covered). Among these 5, QUALMS and QOL-E demonstrated moderate to strong evidence of content validity and psychometric properties in patients with MDS, which included some with LR-MDS. Clinician interviews refined the PCM to include a total of 88 items. Clinicians endorsed 14 signs/symptoms and 15 impacts relevant to LR-MDS and confirmed the PCM as comprehensive. Current COA challenges and the need for an LR-MDS-specific tool were discussed. Ongoing patient interviews will guide appropriate COA selection.
CONCLUSIONS: Identified COAs did not comprehensively cover the LR-MDS PCM developed. There is a need for de novo COA tools, modification of existing tools, or a combination of multiple tools to ensure coverage of prioritized concepts that capture metrics tailored to LR-MDS disease risk and management strategies.
METHODS: Two targeted literature reviews (TLRs) were conducted to (1) develop a PCM documenting a comprehensive set of signs/symptoms and impacts specific to LR-MDS and (2) evaluate the landscape of COAs used in LR-MDS clinical trials and regulatory labels based on searches of OVID/ClinicalTrials.gov/PROQOLID/PROLABELS. A gap analysis compared the identified COAs against items in the PCM, considering content validity and psychometrics. Three expert clinician interviews were conducted to refine the initial PCM and prioritize concepts important to patients.
RESULTS: A total of 46 signs/symptoms and 40 impacts were included in the initial PCM based on TLR of 8 peer-reviewed articles and 7 patient blogs; fatigue, dyspnea, anemia, and social impacts were most common. Among the 10 COAs identified, 5 demonstrated moderate coverage of the PCM (FACT-An, 45% concepts covered; EORTC QLQ-C30, 38% concepts covered; QOL-E, 26% concepts covered; MDASI- AML/MDS, 31% concepts covered; and QUALMS, 26% concepts covered). Among these 5, QUALMS and QOL-E demonstrated moderate to strong evidence of content validity and psychometric properties in patients with MDS, which included some with LR-MDS. Clinician interviews refined the PCM to include a total of 88 items. Clinicians endorsed 14 signs/symptoms and 15 impacts relevant to LR-MDS and confirmed the PCM as comprehensive. Current COA challenges and the need for an LR-MDS-specific tool were discussed. Ongoing patient interviews will guide appropriate COA selection.
CONCLUSIONS: Identified COAs did not comprehensively cover the LR-MDS PCM developed. There is a need for de novo COA tools, modification of existing tools, or a combination of multiple tools to ensure coverage of prioritized concepts that capture metrics tailored to LR-MDS disease risk and management strategies.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
MSR12
Topic
Methodological & Statistical Research
Topic Subcategory
PRO & Related Methods
Disease
SDC: Oncology