Abstract
Over the past 13 years, there have been advances in characterizing the patient experience in oncology trials, primarily using patient-reported outcomes (PROs). This review aims to provide details on the PRO measures and analyses used in multiple myeloma (MM) registrational trials. We identified registrational trials supporting MM indications from 2007 to 2020 from FDA databases. Trial protocols, statistical analysis plans, and clinical study reports were reviewed for PRO measures used, collection methods, statistical analyses, baseline and instrument completion definitions, and thresholds for clinical meaningfulness. Twenty-five trials supporting 20 MM indications were identified; 17 (68%) contained submitted PRO data. Of the 17 trials, 14 were randomized controlled trials and the remainder were single-arm trials. All but one trial were open label trials. Seven trials collected data electronically and five in paper format. The majority of trials evaluated at least two PRO measures (82%) with two trials (12%) utilizing four measures. Nine unique PRO measures were used, most commonly the EORTC QLQ-30 (87%), EQ-5D (65%), and QLQ-MY20 (47%). All 17 (100%) trials provided descriptive summaries, 10 (59%) carried out longitudinal mixed model analysis, 9 (53%) conducted responder analysis, and 2 (12%) did a basic inferential test. We noted substantial heterogeneity in terms of PRO collection methods, measures, definitions, and analyses, which may hinder the ability to effectively capture and interpret patient experience in future MM clinical trials. Further research is needed to determine the most appropriate approaches for statistical and analytical methodologies for PRO data in MM trials.
Highlights
The US Food and Drug Administration (FDA) has approved multiple drugs and biologics for the treatment of patients with multiple myeloma (MM) based on clinician-assessed primary endpoints of progression-free survival or overall response over the past 13 years
We identified pivotal trials supporting approved MM indications submitted to the FDA between January 2007 and January 2020
Fiero and colleagues [19] describe the five attributes of the estimand framework for patient-reported outcomes (PROs) in oncology stressing the need for defining the treatment, the study population, the endpoint, the intercurrent events, and the summary statistic
Summary
The US Food and Drug Administration (FDA) has approved multiple drugs and biologics for the treatment of patients with multiple myeloma (MM) based on clinician-assessed primary endpoints of progression-free survival or overall response over the past 13 years. There have been 15 oncology and malignant hematology approvals that resulted in PRO inclusion in the USPI This represents a very small share of oncology and malignant hematology approvals, approximately half (7 of the 15 instances) of PRO in malignant hematology and oncology labeling were for approvals that occurred since 2017, likely due to improvements in the collection and analysis of this type of clinical data [4]. Despite the increase in MM approvals over the past two decades, no MM product labels contain PRO information in the USPI. We aim to provide details on the variety of instruments used, the diversity of the statistical analyses plans in analyzing the data, inconsistencies in data analyses and presentation, and recommendations that may facilitate robust collection and analysis of PRO data in the clinical trials
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