Integrating Decentralized Trial Design Methods to Explore Clinical Trial Landscape in Rare Genetic Diseases: A Scoping Review

Authors

• LM Edgar
• L Koehly

Abstract

The clinical trial environment is changing with novel concepts such as decentralized clinical trial (DCTs) designs. The COVID-19 pandemic disrupted clinical studies, hence DCT elements are appropriate for research designs. In place of in-person assessments and on-site monitoring, DCT design elements such as electronic consent and telemedicine can minimize participant burden as opposed to centralized trial designs that limit participation for vulnerable minority populations with chronic and rare diseases. Rare patients are more likely to be geographically separated, hence such designs may increase rare patient and clinical trial collaborations. FDA's draft guidelines about DCTs mention its potential with improving recruitment and retention among underrepresented racial and ethnic groups. This review aims to synthesize the literature in the context of rare genetic disease and whether DCT elements are associated with racial composition of such trials. The goal is to contribute to existing guidance and address gaps in understanding of how DCT designs foster minority participation within the rare genetic disease trial landscape. The Joanna Briggs Institute methodology will be used to conduct the scoping review along with adherence to Preferred Reporting Items for Systematic reviews and Meta-Analyses extension for Scoping Reviews criteria. Decentralized elements and participant demographics will be extracted from papers of randomized clinical trials conducted within the rare genetic disease landscape between January 2004 and December 2023 and limited to English only. This project will suggest novel approaches toward the literature and insights related to DCT elements and their participant demographics among the rare genetic disease landscape.

Scientific Focus Area: Clinical Research

This page was last updated on Tuesday, August 6, 2024