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

Abstract

UNSTRUCTURED Clinical research is becoming more participant-centric by closely involving patients in study conceptualization and design, and shifting the setting of research to the home and community by employing decentralized methodologies for data collection. 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. Our experience through a direct-to-participant, decentralized study with myasthenia gravis patients illustrates that a more equalizing and empowering approach to conducting patient-centric research brings multiple benefits (Trial Registration: ClinicalTrials.gov NCT04590716). These include 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.