Decentralized Clinical Trials in Hematology: the Promise and the Peril

Abstract

Decentralized clinical trials (DCTs) are clinical studies where part or all trial-related activities occur remotely, reducing or eliminating the need for patients to physically visit a research site. By applying a more patient-centered design, DCTs have the potential to enhance patient access to clinical trials, improve enrollment and retention, and increase convenience, while simultaneously decreasing costs. This perspective explores the growing importance of DCTs, including the opportunity to facilitate engagement of patients with rare diseases and their broader implications for improving access to care. It also examines the challenges of implementing a DCT, comprising logistical complexities, reliance on technology, and regulatory considerations. Drawing upon our direct experience designing and executing a DCT for patients with Clonal Cytopenia of Undetermined Significance, we discuss our study design, share key pitfalls encountered and lessons learned regarding technology integration, site coordination, and patient engagement. Based on these experiences, we offer recommendations for optimizing DCT design, addressing challenges, and guiding future trials to fully realize the potential of decentralized models, ultimately fostering more equitable and patient-centric clinical research.