Advancing Decentralized and Pragmatic Clinical Trials as a Path Toward Improved Representativeness and Greater Generalizability of Clinical Trial Evidence.

Abstract

Despite efforts to improve research equity in clinical trials, a lack of representativeness continues to threaten the generalizability of clinical trial evidence and leads to several ethical and economic consequences. Decentralized clinical trials (DCTs) and pragmatic clinical trials (PCTs), novel clinical trial models that use technology to enable alternative data collection methods and integrate studies into clinical care, hold great promise for addressing representativeness challenges but also face several limitations. Leveraging technology and clinical care settings to conduct trial visits and collect trial data inherently limits participation from people without reliable access to technology and consistent medical care. Further, representativeness needs to be improved across the full spectrum of clinical trials, from trials conducted in early product development phases to trials conducted after a product is approved, but the DCT and PCT elements that can improve representativeness are more likely to be deployed after seminal evidence for a new medical product's regulatory approval has already been generated. We propose three solutions to help ensure the defining aspects of DCTs and PCTs increase representativeness and the generalizability of evidence generated in clinical trials conducted at all phases in the product lifecycle. We suggest that efforts should be centered around collaborative work with regulators to define data standards and validate outcomes when alternative data sources and collection methods are used, the creation of technical support resources and transparent processes for the conduct of trials facilitated by technology, and the incorporation of health equity principles into the design and deployment of technology-based tools used to facilitate DCTs and PCTs.