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

To accurately evaluate the benefit of new cancer treatments, clinical trials must assess health-related quality of life (HRQOL) measures, as well as safety and antitumor efficacy.HRQOL measures reflect the physical and mental health of patients and are particularly important in hematological malignancies, in which some interventions have modest survival benefits but significant toxicities.Thus, studying how various hematologic malignancies and their treatment affect HRQOL is crucial to determining the best management strategies. 1 Here, we analyzed the extent to which the field appreciates the value of HRQOL in pivotal clinical trials by calculating how often trials for drugs to treat hematological malignancies that resulted in US Food and Drug Administration (FDA) approval assessed patient QOL.We obtained data on HRQOL from study protocols (https://clinicaltrials.gov/) and publicly available product labeling at Drugs@FDA (https://www.accessdata.fda.gov/scripts/cder/daf/).We analyzed drugs approved between January 2016 and May 2020 to reflect the most recent hematologic malignancies drug approvals.The studied hematologic malignancies included multiple myeloma, leukemias, non-Hodgkin lymphomas, Hodgkin lymphoma, and myelodysplastic syndrome.We reviewed primary, secondary, and exploratory outcome measures in each protocol.If any QOL measurement was included as part of the primary, secondary, or exploratory outcomes in the study protocol, we deemed this study to assess HRQOL.Only 69% of clinical trials had some results reported at clinicaltrials.gov.Therefore, for the studies that collected data on HRQOL, we systemically searched of PubMed, Cochrane Central Register of Controlled Trials, Scopus, and the bibliographies of the relevant articles using the National Clinical Trial number, study title/subtitle, authors names, and/or studied drug/s to ascertain whether the published studies that supported FDA drug approval reported QOL measures.To assess reporting of HRQOL, we analyzed all publications available by 10 February 2021 (our data analysis cutoff point).Each drug approval had at least 1 available publication or abstract presentation.The frequency of HRQOL assessment and subsequent reporting of HRQOL measures were analyzed.