Patient-Reported Outcomes Strategy in Oncology Drug Development Trials: Any Consistencies in the Approach to Evaluate Patient’s Quality of Life Between Head and Neck Cancer and Multiple Myeloma?

OBJECTIVES: To identify consistencies and differences in Health-Related Quality of Life (HRQoL) Patient Reported Outcomes (PRO) endpoint strategy across clinical trials from two oncology indications: Multiple Myeloma (MM) and Head and Neck Cancer (HNC).

METHODS: IQVIA’s COA Accelerator platform was used to retrieve HRQoL PRO endpoints based on existing literature from 2006 to 2023 in both indications. COA Accelerator data allowed the comparison and evaluation of consistencies and differences across HRQoL PRO endpoints between the two indications.

RESULTS: Across the two indications, 237 HRQoL PRO endpoints were identified: 152 for MM and 85 for HNC. We found that HRQoL PRO endpoints were often included as secondary endpoints for both indications (>80%). Also, these endpoints mostly explored change from baseline (>62%). Furthermore, three questionnaires were predominantly employed: the EORTC QLQ-C30 and the generic EQ-5D (3L and 5L). They were used to measure over 73% and 59% of the HRQoL PRO endpoints for MM and HNC, respectively. Disease-specific questionnaires were used less consistently to evaluate HRQoL, representing only around 26% and 40% of endpoints measured. These were mostly represented by the EORTC disease-specific questionnaires (EORTC QLQ-MY20 and EORTC QLQ-H&N35), and to a lesser extent by the FACT disease-specific questionnaires (FACT-MM and FACT H&N).

CONCLUSIONS: Overall, we found a clear consistency in the HRQoL PRO endpoint strategy across both indications with HRQoL being measured as a change from baseline at a secondary endpoint level. Moreover, the oncology-specific EORTC QLQ-C30 and generic EQ-5D questionnaires were more frequently used to measure HRQoL compared to disease-specific questionnaires. Further exploration should be conducted to understand why generic HRQoL questionnaires are this oftenly used, despite the existence of validated disease-specific questionnaires.