Clinical must think outside, and inside the box

From site-in-a-box to virtual/siteless trials: which new model will successfully disrupt clinical research?

Many of us are feverishly working to create a leaner and more efficient R&D model.

We all know the reasons and ramifications of the huge costs in clinical research. Researchers are competing for the same patients and research sites are struggling to meet to demands for trials. Trial recruitment is slow, diversity is low, drop-out rates are high, and delays with knock-on cost hikes are frequent.

The problem is systemic. Sites and CROs are locked into a dated model of working, with incompatible technology, slowing progress. Two emerging models are now gaining traction: decentralized, virtual and ‘site-in-box’ trials, both aiming to cut some of the clinical research intermediaries and inefficiencies by getting closer to the patient.

The question with any trial technology is firstly how effective it is, but just as importantly, how scalable is it?

Decentralized, virtual trials, where technology is used to facilitate research remotely, often in the patient’s home, have long been a utopian ideal. The startup Science37 won headlines in 2017, for a significant advance with this model, after successfully completing a full Phase IIb Trial for acne patients, in collaboration with skin microbiome company AOBiome. According to Science37, they were able to reduce enrolment time, improve diversity and, from the patient’s perspective, deliver a better experience.

Yet scalability was a barrier. For one, siteless trials have limited applications in clinical research. As Joe Kim, Senior Advisor in Translational Technology & Innovation at Eli Lilly, puts it: “No one is pulling their blood on their own. If a trial professional requires an examination to evaluate you, they need to do that in the flesh.” While the reach of this model can be expanded, by deploying service providers to the home, there is a clear limit to expansion possibilities.

Part of what determines what can and what cannot be researched remotely is technological. Indeed, this is an area that Kim is optimistic about for the future of remote research. As wearables improve, and as clinical researchers are able to design trials with digital endpoints in mind, we will be able to expand the scope of remote trials to more treatment areas. At the least, remote research can play a growing role in studies as the technology matures, saving the patient from unnecessary trips to their nearest site.

The site-in-a-box trials model approaches the solution from a different starting point, seeking to improve convenience to a patient of accessing a clinical research site vs. decentralizing research to the patient’s home. They seek to convert regular practicing physicians in hospital systems, or smaller practices with everything they need to run a clinical trial. They offer comprehensive support, including research equipment installation and accreditation to help doctors get up and running.

When we caught up with Ben Liu, the CEO of TrialSpark, an early leader in this space, he pointed out that site-in-a-box models aren’t necessarily vying with siteless trials to be the panacea for R&D. He says, “Siteless vs. sites everywhere is a false dichotomy. We should investigate every visit and ask: what is the best way for this visit to accommodate the research goal? Our model is visit type agnostic. Whether it is at a doctor’s office, or at patients home.” In Liu’s view, virtual trials are just one additional option, a positive progression as industry seeks to better cater to a range of individual patient needs.

While TrialSpark has plans to bring this model across all geographies in the US, so far TrialSpark has focused on select markets on the East Coast, Mideast, and South, where it has signed up more than 400 providers by offering HCPs an option to earn an additional revenue stream, offer clinical trials as a treatment option, as well as focusing on making enrolment a painless process. This coverage has translated into access to a more diverse pool of patient participants – with 49% identifying as Non-White/Hispanic, for example.

Participating patients report that they value being able to stay with a doctor they know, as well as the major reduction in travel time. Liu highlights the connection to the patient as a core strength, which allows TrialSpark to directly change the patient experience. By contrast, under the current model, sponsors are beholden to CROs’ and sites’ willingness, or not, to implement patient experience improvements.

While the TrialSpark approach shows promise, it also faces a structural constraint in that it must often compromise, or cut back on its capabilities in order to integrate with a CRO’s way of working, as part of a larger study. This is a challenge that is as much about scale, and the capacity of a startup like TrialSpark to meet the needs of a study, as it is about distribution and particular sponsor preference.

Kim states, “We don’t pick a set of investigators, we pick individual ones. We can’t always do things a different way to fit the network of a given startup [with this approach]. There are experts who we may want to specifically work with, who aren’t under their umbrellas and they may not want to change their way of working.”

The future is hybrid

If we were able to start with a blank canvas, and design clinical trials from the ground-up, we certainly wouldn’t arrive at where we are today. By adopting ways of working that are designed for today’s world and technology, we could dramatically improve trial efficiency, as well as the patient and site investigator experience. But from where we are now, it will take more than one company to drive the structural change that needs to happen.

Innovators like TrialSpark have without doubt developed a better model for clinical research, but until these networks hit scale, we are still forced to integrate with the current, creaking system.

A lesson can be learned from companies such as GlobalCare Clinical Trials, which has been working for a number of years on scaling services that take clinical research to the patient. In contrast to recent newcomers, its strategy has focused on integrating within the current model of clinical research.

It targets sponsors and then offers participating sites a service, which gives the option for the patient to choose the location of the research. It concentrates less on technology and more optimising an efficient and flexible service model that can scale up and down, as needed. It can scale because it integrates well to the current system of clinical research.

GlobalCare CEO, Gail Adinamis, also highlights a key distinction between technology-driven offerings and service-driven offerings, which she sees as an important driver for scale. “When we look at the first decentralized trials, they were largely unsuccessful because they put too much of a burden on the patient. What we focus on is to go to the patient, at a time that is convenient. When we shifted to a model of home visits we were able to help patients maintain their quality of life, which enabled investigators to recruit from a broader area and retain patients for the duration of the study.”

On the strength of a compelling value proposition for providers, meanwhile, TrialSpark has been expanding quickly and its ability to influence the overall design of clinical research will grow as it scales. Right now, it must be careful not to get stuck in a middle ground.

Our research shows there is a real uplift in determination to overhaul development like never before. Multiple projects are being driven by cross-industry working groups, academia and startups, seeking to build a new digitally-driven clinically research model. Indeed, we will have an entire event devoted to this within our upcoming eyeforpharma Philadelphia Summit.

Designing for the future is difficult and fraught with chicken-and-egg scenarios. We need faith in order to continue investing in a new infrastructure for clinical trials that puts interoperability and user experience first. As new players continue to emerge who are doing just this, and as industry working groups bring results, we are getting closer to a tipping point moment and a new normal.