The inclusion of health-related quality of life and other patient-reported outcomes in landmark trials for Food and Drug Administration oncology drug approvals: An analysis of cancer drug approvals between 2006 and 2022.

Abstract

1569 Background: How patients feel and function are essential elements of the clinical benefit of interventions which is often not captured by traditional measures of clinical benefit such as progression-free survival and overall survival. Health-related quality of life, functional status, symptom burden, and other patient-reported outcomes (PROs) assess the impact of interventions from the patient’s point of view. The aim of this study is to assess the inclusion of PROs in landmark trials leading to FDA oncology drug approvals. Methods: This is a retrospective cross-sectional study of pivotal trials leading to FDA oncology approvals between January 2006 and December 2022 based on the FDA oncology approval notifications. The trial protocols of all pivotal trials were evaluated for inclusion of health-related quality of life or other PROs as an outcome measure. The approved therapies were classified into first-line, adjuvant/maintenance, and second or later line of treatment. Chi-square tests were used to assess the relationship between the line of treatment and the inclusion of PROs in the trial protocol. Results: There were 420 oncology approvals during the study period with the highest number of approvals in a single year in 2020 (56) and the least in 2007 (7). Lung cancer has the highest number of approvals (61, 14.5%) followed by lymphoma (50, 12%), leukemia (49, 11.7%), genitourinary malignancies (48, 11.4%), breast cancer (36, 8.6%), gastrointestinal malignancies (32, 7.6%), plasma cell disorders (30, 7.1%) melanoma (20, 4.8%), gynecological malignancies (20, 4.8%), hepatobiliary malignancies (14, 3.3%) and head/neck cancers (14, 3.3%). The approved therapies were first line (146, 34.8%), second or higher line (226, 53.8%), and adjuvant/maintenance therapies (48, 11.4%). PROs were included in 40.7% of the pivotal trials. There is a rising trend in the inclusion of PROs from 5.5% of pivotal trials in 2006 to 31% in 2022. Trials on first-line therapies are more likely to include PROs compared to second or higher-line therapies (44.5% vs 36.7%; p = 0.13). Conclusions: Although there is a rising trend in the inclusion of health-related quality of life and other PROs in pivotal trials for FDA oncology drug approvals, most pivotal trials did not include PROs as outcome measures.[Table: see text]