Give and Take: Fostering Greater Participant-Centricity In A Digital Decentralized Trial Of Rare Disease Patients (Preprint)

Abstract

The acceleration of decentralized and digital methodologies for study data collection has created an opportunity to employ more participant-centric approaches in clinical research. Many studies now include digital and non-digital outcome measures that capture symptoms, daily functioning and health-related quality of life measures in ways that are meaningful to and interpretable by patients. However, study patients are rarely privy to the benefits and insights that clinical research yields. In particular, there are two barriers to providing patients with the benefits of participating in research: studies that produce data that are inaccessible to participants (“unidirectional” data), and studies that create results that are incomprehensible to participants (“unilingual” data). To be both effective and engaging for the participant, clinical research can no longer be one-sided. Decentralized clinical studies leverage technology to ease the burden of data collection on participants, allowing them to contribute their data remotely without having to travel to specific study sites, which can lead to a more equalizing and empowering approach to patient-centric research. Our experience of these benefits is illustrated by a case study of a direct-to-participant, prospective, observational decentralized study with myasthenia gravis patients (Trial Registration: ClinicalTrials.gov NCT04590716), including an improved overall trial experience; increased adherence levels to study tasks and assessments; and an opportunity for participants to gain a greater understanding of their symptoms, such as recognizing trigger factors for disease exacerbations.