By Deborah Borfitz

January 18, 2022 | Next year, a pan-European pilot study gets underway comparing the performance of hybrid and fully decentralized clinical trial models with conventional brick-and-mortar settings where the endpoints are operational metrics such as recruitment speed and diversity of enrolled populations. The focus of the study—the cornerstone of the broader Trials@Home project (Trials@Home – Centre of Excellence Remote and Decentralised Clinical Trials (trialsathome.com) within the Innovative Healthcare Initiative—is to see what decentralized approaches are possible across a continent with a mix of different languages, regulatory authorities, and privacy laws, says Megan Heath, head of Sanofi’s clinical studies unit in Europe.

Parallel projects have been done in the U.S. by organizations that include the Clinical Trials Transformation Initiative (CTTI) and TransCelerate BioPharma, she says. But the lessons learned and subsequent recommendations, specifically from Europe, could prove invaluable.

A progress report on Trials@Home, and how stakeholders are implementing decentralized trials and best-fit criteria for their use in Europe, will all be covered during a live presentation at the 2022 Summit for Clinical Ops Executives (SCOPE). Speakers include Heath and her Sanofi colleague Kim Hawkins, head of global clinical project operations & dossiers delivery.

Major goals of the Trials@Home project include the development of standards as well as recommendations and tools for operationalizing decentralized trials, says Hawkins. Although not explicitly stated, the consortium members are also expecting to walk away with a better understanding of the overall environment for decentralized approaches in Europe.

The public-private Trials@Home consortium itself has roughly three dozen partnering organizations, half academic institutions and smaller companies and the other half member companies of the European Federation of Pharmaceutical Industries and Associations, says Hawkins. Everyone knows going in that they’re committing to a higher-than-normal level of precompetitive information-sharing around issues of mutual concern.

Practical Realities

Sanofi nominated Trials@Home as a topic of interest and is co-lead of the project with the University Medical Center Utrecht (Netherlands). Trials@Home is a five-year project, launched late in 2019, with which at least 20 Sanofi employees are involved to some extent, says Hawkins.

The three-arm RADIAL (Remote and Decentralized Innovative Approaches to Clinical Trials) study, enrolling people with type 2 diabetes who have reached the stage of requiring basal insulin, is the hub of six workstreams constituting Trials@Home, says Heath. The other five specialty workstreams are best practices, technology, ethics, and regulations, communications and training, and project management, collectively covering the external environment for decentralized trials.

Their activities include seeking input from patients, site staff, and regulators on the ability to pivot from traditional operational approaches to decentralized approaches, Heath continues. Members on the regulations workstream helped map out which countries to include in the pilot study, based on prior experience and receptivity to the model as well as ensuring a “good spread of patients from across the European region to be able to draw some reasonable conclusions.”

Much of the groundwork for the study was completed prior to the pandemic, followed by experiences with decentralized trials during their forced introduction earlier last year, Heath adds. RADIAL moves into execution mode early this year.

The reality is that the decentralized model is not a “one-size-fits-all” proposition, says Hawkins. “There may be some clinical trials that can run in a completely decentralized fashion, but most studies will likely have some decentralized aspects. It’s going to depend on the disease and the endpoints being studied.”

Decentralization in some cases will focus less on patients’ home than leveraging their local healthcare providers and surroundings, she adds. This might alleviate the burden on people who would otherwise have to travel to a large academic medical center several hours or even a plane ride away.

Takeaways from the Trials@Home project should have value across the globe, Heath says. Decentralized trials are sometimes assumed to be faster, but operational metrics are needed to measure these impacts and benefits.

Harmonizing Efforts

The RADIAL pilot study will recruit type 2 diabetes patients over a six-month period and treat them for six months. At the end of the study, the key performance indicators (KPIs) will be evaluated across the three operational models (conventional, hybrid, and fully remote), says Heath. “We’re still defining what the conventional arm will look like in detail, especially given the complexity of the operational environment in Europe at the moment.”

The study will analyze recruitment as well as patient and site satisfaction to gauge acceptance and adoption across the various stakeholder groups. RADIAL will be the first time a fully decentralized clinical trial has been conducted in Europe to assess operational KPIs of the model, she points out.

While the pilot study proceeds, the Trials@Home team will be keeping tabs on the broader environment for decentralized trials and what specific aspects of the model make sense to incorporate into its portfolio of studies and under what conditions, Hawkins says. The “BEST” working group of the Trials@Home project will also continue looking at case studies. The Trials@Home project will roll out some final recommendations from those combined efforts and possibly also tools to help facilitate their practical application.

That BEST workstream has already published criteria defining what is and is not a best fit for the decentralized model on the Trials@Home website, along with an initial set of recommendations based on a review of about 20 previously run decentralized clinical trials. The group is currently in the process of grading those case studies and elaborating on what could have been done differently or better, reports Hawkins, with some new materials expected to be posted early next year.

Published recommendations focus on “keeping patients and clinical sites at the center of everything,” she adds. “As an industry, we tend [sometimes] to act quickly and throw technical solutions into the mix and that’s not always the best way to go.”  

In the U.S., Hawkins says, CTTI came out with its own recommendations back in 2018. A new global industry coalition known as the Decentralized Trials & Research Alliance (DTRA) is now aiming to consolidate the work that has been done across multiple, time-limited projects.

The three groups are talking to each other, Hawkins says, but more collaborative work is needed to minimize duplication of effort and leverage past learnings. “We have regular calls with CTTI, and I presented on a few topics at the DTRA annual meeting [in November].”

Editor’s Note: Megan Heath and Kim Hawkins will be presenting on the Trials@Home project at SCOPE on Thursday, Feb. 10, and welcome questions from session attendees.