Reporting of Health-Related Quality of Life Outcome Measures in Genotype based Targeted Therapies for Advanced Cancers

Background Genotype based matched targeted therapies (MTT) trials have shown clinical benefit in patients with advanced cancer however health-related quality of life (HRQoL) outcomes are often inconsistently reported in these trials. In this study, we evaluated the overall reporting and quality of HRQoL based randomised controlled trials (RCTs). Methods We searched various biomedical databases (CENTRAL, MEDLINE, EMBASE) from 2000 to 2024, for eligible RCTs evaluating biomarker based MTTs (alone or combined with standard of care systemic standard-of-care [SOC]) versus SOC or placebo in patients with advanced cancers that had progressed after at least one prior systemic therapy. We evaluated inclusion of HRQoL endpoints, timing of reporting, tools used, and adherence to selected CONSORT-PRO guidelines. Results A total of 208 studies were assessed for eligibility. No studies reported HRQoL as a primary endpoint. Among 61 RCTs (n = 21,103), HRQoL was included as a secondary or exploratory endpoint in 35 (57.4%) trials. Only 17 (27.9%) trials reported HRQoL in the primary publication. Inclusion and reporting declined over time, with only 44.0% of trials after 2020 including HRQoL and just 8.0% reporting it in the primary publication. Trials with positive primary outcomes reported HRQoL more frequently and earlier. Compliance with CONSORT-PRO was poor: no trial stated HRQoL in their hypothesis or described handling of missing data. Common instruments included EORTC QLQ (n = 20), FACT (n = 14), and EQ-5D (n = 10). Conclusion HRQoL is significantly under-reported in MTT based trials, with declining inclusion and poor adherence to reporting standards. This is particularly important as MTT do not significantly improve overall survival in this cohort and therefore quality of life becomes paramount for these patients.