USE OF DISEASE-SPECIFIC EORTC SCALES IN SMOLDERING MULTIPLE MYELOMA: EVIDENCE FROM PATIENT INTERVIEWS AND ALIGNMENT WITH THE EORTC ITEM LIBRARY STRATEGY
Author(s)
Susanne Huschens, PhD1, Katharine S. Gries, PharmD, PhD2, Monika Bullinger, PhD3.
1Janssen-Cilag GmbH, Neuss, Germany, 2Patient Reported Outcomes, Johnson & Johnson, Raritan, NJ, USA, 3University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
1Janssen-Cilag GmbH, Neuss, Germany, 2Patient Reported Outcomes, Johnson & Johnson, Raritan, NJ, USA, 3University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
OBJECTIVES: Patient‑reported outcomes (PROs) are essential for assessing patient‑relevant effects in benefit assessment procedures. Targeted selection of items over a full PRO instrument allows for minimizing burden to patients by eliminating irrelevant scales/items. The objective of this work was to examine and justify the PRO strategy using empirical patient evidence and to contextualize the EORTC methodology permitting the selective use of items and scales.
METHODS: Qualitative interviews were conducted with 15 patients diagnosed with smoldering multiple myeloma (SMM) and 6 patients with multiple myeloma (MM) following a prior SMM diagnosis. Patients were asked to describe the most relevant symptoms and disease impacts experienced during the course of SMM, followed by cognitive debriefing of items. The selection of EORTC scales capture both disease‑specific symptoms and the psychological burden of disease progression. Item analysis was conducted to assess change over time.
RESULTS: Frequently reported symptoms included fatigue, weakness, pain, balance problems, tingling, appetite loss, constipation, and anemia. Patients described emotional burden (worry, stress, anxiety) and limitations in daily activities, physical functioning, cognitive abilities, and social participation. The EORTC QLQ‑C30 items and EORTC QLQ‑MY20 scales Disease Symptoms and Future Perspective items were consistently endorsed by patients as relevant. Patients confirmed that the selected items accurately reflected their experience, and no relevant concepts were identified as missing. Selective use of EORTC QLQ‑MY20 scales is consistent with the EORTC Item Library strategy, which allows for individual scales/items alongside the EORTC QLQ‑C30 without requiring administration of the complete disease‑specific module, provided that the conceptual structure of the scales is preserved. Comparable approaches have previously been accepted by health technology assessment bodies.
CONCLUSIONS: The findings support the use of selecting items or scales from the EORTC item library to target meaningful concepts of patient‑relevant morbidity and health‑related quality of life as part of the overall PRO strategy.
METHODS: Qualitative interviews were conducted with 15 patients diagnosed with smoldering multiple myeloma (SMM) and 6 patients with multiple myeloma (MM) following a prior SMM diagnosis. Patients were asked to describe the most relevant symptoms and disease impacts experienced during the course of SMM, followed by cognitive debriefing of items. The selection of EORTC scales capture both disease‑specific symptoms and the psychological burden of disease progression. Item analysis was conducted to assess change over time.
RESULTS: Frequently reported symptoms included fatigue, weakness, pain, balance problems, tingling, appetite loss, constipation, and anemia. Patients described emotional burden (worry, stress, anxiety) and limitations in daily activities, physical functioning, cognitive abilities, and social participation. The EORTC QLQ‑C30 items and EORTC QLQ‑MY20 scales Disease Symptoms and Future Perspective items were consistently endorsed by patients as relevant. Patients confirmed that the selected items accurately reflected their experience, and no relevant concepts were identified as missing. Selective use of EORTC QLQ‑MY20 scales is consistent with the EORTC Item Library strategy, which allows for individual scales/items alongside the EORTC QLQ‑C30 without requiring administration of the complete disease‑specific module, provided that the conceptual structure of the scales is preserved. Comparable approaches have previously been accepted by health technology assessment bodies.
CONCLUSIONS: The findings support the use of selecting items or scales from the EORTC item library to target meaningful concepts of patient‑relevant morbidity and health‑related quality of life as part of the overall PRO strategy.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PCR133
Topic
Health Technology Assessment, Methodological & Statistical Research, Patient-Centered Research
Topic Subcategory
Instrument Development, Validation, & Translation, Patient-reported Outcomes & Quality of Life Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology