Financial toxicity in patients with multiple myeloma participating in clinical trials: A U.S. Food and Drug Administration pooled analysis.

Abstract

e19370 Background: Financial toxicity (FT) is a major concern for patients receiving standard cancer treatment, and FT can lead to worse cancer outcomes. However, little is known about the FT of patients enrolled in clinical trials (CT). While investigational treatment may be provided by sponsors free of charge, patients are subject to increased clinic visits (ie, missed workdays, travel costs) and still bear the cost of usual care. This analysis evaluates patient-reported FT in multiple myeloma (MM) trials submitted to US FDA and explores the relationship between baseline FT and overall response rate (ORR). Methods: We pooled data from 9 MM registration CTs submitted to the FDA that included the EORTC Quality of Life questionnaire (QLQ-C30). The QLQ-C30 includes an item asking patients “Has your physical condition or medical treatment caused you financial difficulties?”. We looked at proportion of patients at baseline reporting any FT and their ORR. We also report prevalence, incidence and change from baseline FT at 3 and 6 months. Results: 5,667 patients answered the FT item. Mean age of patients was 65 years, 55% were male, 85% were white and 6% were enrolled in the US. Approximately a third of patients reported experiencing any FT at baseline, 3 and 6 months. When compared to their baseline, 69% of patients had unchanged FT and 14% had worse FT at both 3 and 6 months. The incidence of new cases of FT at 3 months was 17% and at 6 months 7%. The ORR in patients who reported any FT at baseline was 69% compared to 72% in patients with no baseline FT, and was similar regardless of the degree of FT. Conclusions: Few studies have investigated FT in a large sample of CT participants. In this pooled analysis of patients enrolled in MM CTs, approximately a third of patients reported FT associated with their physical condition or medical treatment at baseline and throughout the trial. A small percentage of patients experienced worsening FT during the trial and a small percentage went from no FT to reporting any FT. The impact of baseline FT on ORR was small. Limitations of this preliminary analysis include missing patient level or group level insurance status, socio-economic status and other important determinants of health.