Decentralized Clinical Trials
Gail A. Van Norman · JACC Basic to Translational Science · 2021
T he Covid-19 pandemic disrupted many clin- ical trials that were potentially bringing new therapeutics to market-an additional untallied cost of the pandemic in lives and quality of life owing to delays in releasing potentially beneficial therapeutics to patients in need.A separate sideeffect of the pandemic has been swift adoption of virtual interactions between physicians and patients to provide continuity of care while maintaining social distancing.This comes at a time of rapid advancement of technology permitting those interactions, such as enhanced internet connectivity, electronic health records, real-time video conferencing, smartphone health applications, and remotely connectable health monitoring devices that are becoming both more accurate, practical, and affordable.Interest in decentralized clinical trials (DCTs) that use "virtual elements" like these has grown in parallel with acceptance of "virtual medicine," accelerating shifts in clinical trial design that many feel are long overdue.DCTs-also termed "direct-to-participant trials" or "virtual" studies-are characterized by less dependence on traditional research facilities or specialist intermediaries for data collection.DCTs leverage "virtual" tools, such as telemedicine, sensory-based technologies, wearable medical devices, home visits, patient-driven virtual health care interfaces, and direct delivery of study drugs and materials to patients' homes.In a fully decentralized clinical trial, subject recruitment, delivery and administration of study medication, and acquisition of trial outcomes data all proceed without involving in-person contact between the study team and the patient/subject.Currently, clinical trials for drug approval often already include decentralized elements, and DCTs often incorporate traditional design with decentralization of the patient/subject interactions (Central Illustration).ADVANTAGES OF DCTs: SMALLER, FLEETER, CHEAPER, MORE DIVERSE?DCTs may improve the logistics of conducting a clinical trial by improving recruitment and retention of subjects (1).Patients who would otherwise face daunting challenges from centralized study trials may be able or willing to enroll in DCTs, because remote monitoring and data collection minimize obstacles to participating, such as logistical difficulties in accessing the trial location-e.g., travel costs, nonacceptance of job absences for study activities, and mobility challenges posed by medical comorbidities.A collateral effect is improvement of trial access for participant populations that are currently most underrepresented in current traditional trials: the elderly, the poor, those living in remote locations, and many ethnic minorities.Recruitment times are also likely to improve, as are generalizability of results across diverse populations.DCTs can potentially decrease trial sample size by enabling the development of individualized thresholds for measuring treatment effects.Digitalized tools such as biometric sensors may allow more objective methods of measuring pain, quality of life, functional status, and cognitive function, permitting better understanding of individual responses to treatment and individualized patient toxicities.