Is financial toxicity captured in quality of life assessments in oncology randomized clinical trials?

Abstract

Background

Financial difficulties in relation with diagnosis and treatment of patients with cancer affects their quality-of-life (QoL). We aim to characterize how financial toxicity was captured in oncology randomized clinical trials (RCTs), and to estimate how often the study-drug or other expenses were covered by sponsors.

Methods

This was a cross-sectional analysis of articles published in six high impact journals (The New England Journal of Medicine, The Lancet, JAMA, The Lancet Oncology, Journal of Clinical Oncology, and JAMA Oncology). Selected articles needed to report on a RCT published between January 2018 and December 2019, study an anti-cancer drug, and have reported QoL results. We abstracted the QoL questionnaires used; whether the survey was directly assessing financial difficulties; whether a difference in financial toxicity was reported between arms; and whether the sponsor supplied the study-drug or covered other expenses.

Results

For all 73 studies that met inclusion criteria, 34 studies (47%) utilized QoL questionnaires without direct assessment of financial difficulties. The study drug was provided by the sponsor in at least 51 trials (70%), provided according to local rules in 3 trials (4%), and undetermined in the remaining 19 trials (26%). We found 2 trials (3%) with payments or compensation to enrolled patients.

Conclusion

This cross-sectional study found 47% of articles reporting on QoL in oncology RCTs did not use QoL questionnaires directly assessing financial toxicity. Additionnaly, the study drug was supplied by the sponsor in most trials. Financial toxicity occurs in real-life settings when patients have to pay for the drugs and other medical expenses. QoL assessments from oncology RCTs lack generalizability to real-world settings, due to limited querying of financial toxicity.

Policy summary

Real-world evidence could be demanded by regulators as post-requirement studies to ensure QoL results observed in trials will replicate in patients treated outside investigational trials.