Landscape of Patient-Reported Outcomes Measures (PROMs) in Oncology Trials: Key Insights Into Patient Voice and Value

OBJECTIVES: Patient-reported outcome measures (PROMs) provide a window into patient voice and are critical in oncology studies. This study aimed to characterize the use of PROMs in US-FDA approved oncology therapies in the past decade.
METHODS: We analyzed PROMs used in oncology trials approved by US-FDA over 2015-2024. Data on oncology trials and PROMs were extracted from an internal database of 500 drugs approved since 2015. PROM use was analyzed based on Trial Phase, Cancer type, PROM characteristics and PROM adoption pre-post US-FDA guidance for measuring PROMs in oncology trials (2021).
RESULTS: US-FDA approved 114 oncology therapies were studied in 232 trials (Phase 3 = 43%, Phase 2 = 32%, observational = 10%, Phase 1 = 8%). PROMs were included in 140 trials (60%) of the trials. Generic PROMs such as shortform (SF12 and SF36) and EuroQol 5-Dimension (EQ5D) were used in 40 trials (29%). EQ-5D and SF measures were primarily used in Lung (n= 32 trials), Hematological (n= 27 trials), and Gastrointestinal cancers (n=6 trials). Cancer specific PROMs such as EORTC Quality of Life Questionnaire (EORTC-QLQ-C30) and Functional Assessment of Cancer Therapy (FACT) were used in 99 trials (71%). EORTC-QLQ-C30 was the most used measure in trials for Hematological (n=54), Lung (n=27), Breast (n=35) and Prostate (n=4) cancers. FACT-LYM was used in 9 Hematological cancers trials, whereas FACT-B was used in 8 Breast cancer trials. PROMs for assessing anxiety and depression like Hospital Anxiety and Depression Scale and work productivity like Work Productivity and Activity Impairment were used infrequently in specific cancers (Breast, n=2). PROM utilization increased post the release of FDA guidance in 2021.
CONCLUSIONS: PROMs utilization in oncological pivotal trials is increasing post US-FDA guidance and is proving valuable insights into patient care.