Characteristics and research waste of randomized controlled trials in melanoma

Abstract

Abstract Background Numerous large-scale randomized controlled trials (RCTs) have propelled melanoma treatment strategies. Research waste presents a significant challenge in translating the outcomes of RCTs into clinical practice. Currently, research waste has not been reported in melanoma-related RCTs. Objectives To determine research waste in RCTs for melanoma. Methods In January 2024, we searched ClinicalTrials.gov for phase III and phase IV RCTs registered from January 2000 to December 2023, using ‘melanoma’ as the keyword. We recorded the information listed on the website and searched PubMed and Scopus for the publication and citation status of the RCTs. A completed RCT requires at least 47 months of preparation time for publication; hence, RCTs completed after December 2019 but not yet published were excluded from the analysis of publication status. Results In total, 165 RCTs were included in the analysis. Melanoma RCTs primarily studied pharmacological interventions, with the registrations for immunotherapy increasing annually. In the analysis of research waste, 103 RCTs were included, of which 41 (41 of 103, 39.8%) were unpublished. Of the 62 published RCTs, 19 (19 of 62, 31%) reported insufficiently, and 19 had avoidable design flaws (19 of 62, 31%). Ultimately, 64 RCTs (64 of 103, 62.1%) were judged to have research waste. Registration after 2010, conducting studies in multiple countries, using multiple drug interventions, and having survival as the primary outcome were independent protective factors against research waste. Thirty-four RCTs (34 of 62, 55%) were cited by guidelines, and 21 RCTs (21 of 62, 34%) reused their prospective data. Conclusions We describe the characteristics of phase III and phase IV RCTs related to melanoma conducted over the past 2 decades. We identified a substantial degree of research waste. The protective factors against research waste revealed in this study can provide references for the rational and efficient conduct of new RCTs in the future.