Mindy Waizer

Siteless Clinical Trial Design Is Here — Are You Ready?

The era of siteless clinical trials for drug development is here.

Also known as virtual, remote and digital trials, siteless trials build on the “direct-to-patient” trend in clinical trial design.

Meeting patients where they are physically located instead of requiring them to visit clinical trial sites has been a step forward in patient-centered care. It is also a cost-saver. One study published in the journal Clinical Trials estimated that site monitoring costs comprise between 9 and 14 percent of the total trial cost.

A great advantage of the siteless clinical trial is convenience. Because many qualified patients may not live near enough to the location where a study is conducted, they are often unable to participate. Requiring patients to make frequent visits to a clinical trial site presents a significant barrier to recruitment and retention and limits the diversity of trial participants, according to a study in Clinical Therapeutics.

Currently, some trials recruit patients’ local providers or use visiting nurses to help patients participate in decentralized trials. Some require only limited trial site visits while supplementing with telemedicine technology. In these hybrid models, patients connect to the clinical research organization (CRO) remotely for a significant portion of the trial.

The next leap forward in clinical trial design is to empower patients to participate entirely from their homes with the use of telehealth, mobile technology and digital medical devices.

In fact, the revolution has already begun.

Web-Based Tools and Telehealth Led the Way

Early attempts at incorporating remote trial elements used web-based tools for screening and consent. Other studies then began to use telehealth to connect patients with researchers for remote monitoring.

Pfizer was the first to initiate a feasibility study with all-digital management in 2011. It used web-based and telehealth tools for its Research on Electronic Monitoring of Overactive Bladder Treatment Experience (REMOTE) study. Online forms were used to screen applicants and obtain consent. The study drugs were sent directly to patients. While an overly cumbersome process and forms that were less than user-friendly contributed to its early termination, this study formed the basis of future attempts at siteless trials.

A European collaborative’s success with a feasibility study of wireless blood glucose meters for glucose profiling set the bar for web-based studies in 2016. Sanofi, in partnership with eClinicalHealth, Langland and Mendor, recruited participants in the VERKKO remote online phase 4 clinical trial for diabetes through Facebook. Patients registered online, consented electronically and received materials for the study in their homes. Results showed that compared with a similar traditional-style study, compliance improved 18 percent, and coordinating the study took 66 percent less time.

Many clinical trials have begun to incorporate mobile health (mHealth) technology to capture real-world data, giving researchers a more detailed picture of patients’ overall health. Feedback from mobile health devices can be recorded and accessed by the care team in real time, greatly improving data quality and access.

Building the Tech – And the Support

Companies like IQVIA, a provider of analytics, technology solutions, and contract research services, and Parallel16, a clinical platform for enrollment, engagement and management, are developing tools to enable trial administration from a distance.

The research organization Science 37 is making it happen. Its first completed siteless study was a Phase 2b interventional randomized placebo-controlled trial for an AOBiome acne medication. Science 37 shipped all study materials directly to patients and monitored them through a telemedicine app. Patients received an iPhone and a data plan to ease communication with the study team.

Science 37 reported that it screened more than 8,000 people online and enrolled 372 patients in less than seven months — approximately 50 percent faster enrollment than with a traditional model. It also reported a significant rise in diversity of participants, with a 41 percent non-Caucasian population compared to the 10 percent a traditional clinical trial at a research-focused medical facility might receive.

Pharma giant Novartis is working with Science 37 to develop 10 trials using decentralized models, with the goal of scaling down to siteless trials.

Newer studies are expanding the role of the pharmacist to ensure adequate oversight from afar. One siteless CRO, Center Point Clinical Services, has strategically grown the role of the clinical trial research pharmacist (CTRP) to ensure that patients remain engaged with a qualified advisor who is trained in patient counseling. These CTRPs access real-time participant data and teleconference with patients in their homes.  

The growing role of the pharmacist factors into Spencer Health Solutions’ system as well. Pharmacists prepare pre-packaged medications in unit doses to be dispensed by the spencer® Smart Hub device, removing the possible element of human error in administering the trial drugs. spencer connects patients and caregivers with pharmacists, clinicians and others on the care team through a videoconferencing function. It also syncs with mobile health devices and reports secure data to the care team through HIPAA-approved methods.

Moving Forward

Some challenges to using a siteless format still exist, such as concerns around data security, adequate oversight and accurate patient reporting. But with ever-improving tools, it will soon become a model of choice. Siteless design has the potential to transform the landscape of drug development, saving investigators and patients time and money while increasing data quality and engagement.

Christine Mackay, Site Development Director, University of Kansas Cancer Center, provided background information for this article.

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