Assessment and reporting of quality-of-life measures in pivotal clinical trials of hematological malignancies.

Abstract

158 Background: The importance of assessment and reporting of health related quality of life (QoL) has been increasingly recognized in the field of oncology. QoL measures, which reflect patient’s perceived benefits and satisfaction, might be especially important in hematological malignancies clinical trials, where the intervention may not result in cure with modest overall survival benefit. Methods: Data on health related QoL were obtained from studies’ protocols and product labeling available publicly at Drugs@FDA. Drugs approved from 2016 to 2020 were analyzed. On the basis of publicly available study protocols and Food and Drug Administration (FDA) reviews, the authors reviewed the assessment of health related QoL in 53 clinical trials supporting 50 drug approvals from 2016 to 2020. Those trials resulted in approval of medications to treat leukemia, non-Hodgkin lymphoma, Hodgkin lymphoma, myelodysplastic syndrome, and multiple myeloma. Results: A total of 14,819 patients were assessed in the 5 years period after exclusion of unpublished studies. We calculated the frequency of assessment of health related QoL and if QoL measures were reported in the subsequent publications. After exclusion of 7 unpublished studies, we analyzed 46 clinical trials for reporting of HRQOL. Thirty percent of the protocols included assessment of health related QoL with only 43% of them (13% of all the analyzed clinical trials) reported the results of health related QoL. The results were consistent across all subtypes of hematological malignancies and were similar regardless of the studied primary endpoint. Conclusions: QoL measures are under studied in clinical trials of hematological malignancies. Studies that measured health related QoL didn’t report the results in more than half of the time. Although results may be reported in separate future publications, improvement in assessing and reporting of QoL will help to incorporate QoL measures in patient care and therapy choice decision. [Table: see text]