Published Jun 13, 2022 in biopharmadive.com

A decentralized clinical trial (DCT) is where some or all patient visits occur outside the traditional clinic or hospital setting. It can employ wearables and sensors, telehealth, smartphone-based apps, in-home visits or other methods to make the trial more patient-centric and remote from the site. Before COVID-19, DCTs were becoming more common, however, the pandemic shifted the paradigm and motivated sponsors to look at decentralized and hybrid trials in a different light and realize the benefits these new models will deliver.

Although not all therapeutic areas or indications are appropriate for a decentralized approach, when they can be used, they can increase patient recruitment, retention and diversity and deliver high-quality data, all while making it easier for patients to participate. This can be particularly important for rare disease studies, where a relatively low number of appropriate patients may be more geographically spread across the country or indeed the globe.

Considerations

Emily Mitchell, Executive Director, DCT Operations at ICON, outlined a number of key considerations in determining whether a decentralized trial is appropriate. These include safety profile of the drug or therapeutic area, delivery method of drug, study phase (late phase trials are typically better suited to decentralized elements) patient profile and geographical distribution.

Safety profile and therapeutic area

For example, if the therapy being evaluated must be given as an infusion under the supervision of a physician, a DCT is going to be difficult. Also if the therapy has significant potential side effects, such as cytokine storms or severe immune and inflammatory reactions as seen with some immuno-oncology products, DCTs might not be appropriate, although a hybrid approach may be possible.

“There’s definitely interest in the oncology space,” Mitchell said, “but there are different dynamics that you need to think about from an infusion perspective. For instance, if it is chemotherapy, you need to assess the severity of adverse events that are likely to occur. On the other hand, we’ve seen great interest and have been able to adopt fully decentralized approaches in some of the more benign treatment areas in general medicine such as dermatology or women’s health. Treatments for conditions such as hot flashes align well with the characteristics of being fully decentralized.”

In rare diseases, where relatively few patients are geographically spread, a DCT or hybrid may work well to increase the patient pool and diversity.

Similarly, if the patient population is less mobile or needs more caregiver support such as children or elderly patients, there may be a strong case to reduce burden by increasing in-home visits and assessments.

Applying innovation to make a difference

Mitchell doesn’t see where these considerations change for a small biotech company versus a large pharma company. “I think where we’re seeing differences is dependent on the organizational hunger to transform clinical trials by applying innovation for better outcomes.”

She also added that increasingly, organizations’ sustainability strategies have emerged as a consideration. “A lot of companies I’ve spoken to recently are evaluating how they can help reduce their carbon footprint. Offering a decentralized trial approach has the potential to contribute to the reduction in the carbon footprint of the organization.  The more trials sponsors are running, the larger the company, the bigger the impact.”

Infrastructure for decentralized clinical trials

Decentralized clinical trials can be conducted using a variety of approaches, depending on the indication and the resources of the organization. Services applied include telehealth for doctor/patient visits, patient portals, wearable devices and in-home visits.

One such service and a key differentiator for ICON is the Concierge Support and Coordinating Centre. According to Mitchell, the support center provides the support for truly patient-centric trials. “This group provides the support throughout the patient journey; allowing patients to call in and ask questions, helping them to schedule and coordinate calls with the physicians or scheduling the in-home health nurse to visit the participant’s home. They provide the glue that keeps all the components together and the patient engaged.”

Three key factors for success

Although best practices are still being developed for DCTs, Mitchell cited three key factors for success.

1. Upfront planning. “Getting it right from the very beginning is absolutely critical,” Mitchell said, “because a lot of times we have clients sending us protocols asking how to decentralize a trial. Well, it’s already too late. At that point, the sponsor has already written the protocol and how the assessments are supposed to be conducted. Instead, right at the beginning do a full review and ask, ‘Is this the right fit for decentralization?’ And if yes, then how decentralized do we want to take it knowing the patient population and all the other factors. Then build that protocol around that approach.”

2. Understand the burden. Participating in a clinical trial is never going to be burden-free, whether it is traditional, hybrid or fully decentralized. “It’s just determining where we can shift the burden so that it’s not falling fully on the participant,” Mitchell said. “If it does, they aren’t going to want to participate in that or future trials.”

That may require placing some of the burdens on infrastructure, such as the DCT Concierge Support and Coordinating Centre; or on the central investigator, who can conduct oversight for safety concerns; or for remote patient monitoring so it doesn’t all fall on the care physician or the patients themselves.

3. Maintaining patient safety. And finally, Mitchell noted that the priority at all times is going to be maintaining patient safety, “making sure that we’re engaged with them, that we have the right level of monitoring set up. We need to ensure that we are putting appropriate checks in place so participants feel that their safety is not compromised.”

With appropriate planning and the right partner, decentralized clinical trials can deliver more patient-centric trials and will increase patient recruitment, engagement and retention.