CNS Summit: Discussions on Virtual and Hybrid Clinical Trials

The CNS Summit was recently held in Boca Raton, Florida with the focus on its mission, Collaborating for Novel Solutions. The summit brought together 750 leaders in biopharma, contract research organizations, regulatory officials and academics to discuss innovation in drug development, including virtual clinical trials, patient-centricity, and regulatory challenges, among the many topics.

Jonathan Cotliar, chief medical officer of Science 37, a leader in virtual clinical trials, took time to speak with BioSpace about virtual and hybrid clinical trials, the CNS Summit, and Science 37.

“We know that drug development is a very costly and timely endeavor and that participants are largely Caucasian, middle-aged males,” Cotliar said. “There’s a very notable history of clinical research and the lack of diversity among participants involved in clinical trials. But if you can bring a clinical trial to patients in their homes, you eliminate those barriers and you get a trial participant population that is more likely to reflect the demographics within a given locale.”

To that end, a virtual clinical trial utilizes telemedicine, sensor and wearable technology, and at-home patient visits. At the all-virtual end, it moves completely away from the brick-and-mortar clinic or hospital-based clinical trial, with that traditional brick-and-mortar trial at the other end of spectrum. In between is a model that is growing rapidly, the hybrid approach.

Cotliar’s company, Science 37, was founded about five years ago by two physician-scientists on the premise that if you make clinical trials more patient-friendly or patient-centric, you could speed up the drug development and approval process. In that period, Cotliar notes, they have conducted, are actively conducting, or finalized 27 clinical trials. Of those 27, 22 are industry sponsored. And of those 22 industry-sponsored trials, 16 were “interventional trials that really represent a lot of complex procedures and assessment that we were able to coordinate and initiate in patients’ homes. We were able to deploy our mobile nurses and the technology piece we’ve built that enable a lot of these activities. And we’ve done five validation studies in addition to the 22 industry-sponsored trials.”

He notes that other organizations are increasingly becoming interested in virtual and hybrid clinical trials and are beginning to “dip their toes in.”

One of the panels at the CNS Summit Cotliar participated in with other executive leaders involved virtual trials. “What is really exciting is we have more tell-ins and organizations that recognize the value of virtual trials and a more patient-centric way of thinking about research. More organizations are committing resources to conducting virtual trials, which is better for the entire industry.”

Another topic of discussion at the summit related to virtual clinical trials was the idea of digital endpoints. When a sponsor company submits a New Drug Application to the U.S. Food and Drug Administration (FDA), it has to demonstrate that the therapy has a clinically meaningful effect for patients. There is a long history of established traditional clinical endpoints, whether it’s a cholesterol level, a hemoglobin A1C level, or specific metrics regarding cancer survival and tumor shrinkage.

A body of digital endpoints is not as well established. But increasingly, for certain types of clinical trials, sensor data that might be supplied by a smartwatch like an Apple Watch, Garmin or Fitbit, is playing a part in virtual and hybrid clinical trials.

Recently, the nonprofit Digital Medicine Society established a crowdsourced library of digital endpoints. This library describes 38 unique industry-sponsored trials that evaluated digital endpoints in new therapies or new uses for existing medical products.

Cotliar said, “These digital endpoints are sort of aggregations of everyday activities that we don’t traditionally think about as surrogates for the functional status of certain diseases, especially chronic diseases. As wearables and sensors become more integrated into our daily lives, we can tweak algorithms to better reflect specific data points to highlight whether someone is doing ‘good’ or ‘not good’ for a specific disease and whether a medical intervention can change those data points that are surrogate markers for a specific disease. They aren’t that far from a traditional clinical endpoint but can be integrated into the more traditional clinical endpoints.”

He notes that both the digital endpoints and the sensors and wearables themselves are stimulating discussions in the industry on how they can be used to assess disease severity and activity and how they may influence behaviors. “It’s all going to change as these sensors and wearables become more ubiquitous and data scientists tease out more data points. This is exciting, a future state where the concept of a randomized placebo-controlled trial will be more of an outlier than rule because of these unique disease data points and the way we can generate data compared to what we’ve typically been limited by in traditional clinical trials.”

One of the things Cotliar observed at the summit was “it was clear to me that the acceptance of industry-wide virtual trials was only going to be the normal route in clinical research. It’s not only been accepted, but there’s a velocity of that acceptance. The tenor of the discussion compared to similar meetings in the last couple years was significantly different. Having CROs calling themselves virtual trial providers is a testament to that fact.”

Another is machine learning, artificial intelligence (AI) and voice as enablers of clinical trial efficiency. Cotliar said their use, “is becoming more and more solidified, with specific solutions incorporating AI, voice and machine learning. It is not just concepts, but they are being deployed in real ways as solutions for clinical trials.”

And as that process expands throughout the industry, Science 37 is seeing the opportunities increase. “We used to get pilot studies of 10 or 20 patients,” Cotliar said, “as a virtual trial cohort as part of a larger study. But now we’re seeing a willingness to have us do a larger and larger percentage of the enrollment, if not all of the enrollment. That’s exciting. We’re poised to get into the oncology space with some projects we plan to announce by the end of the year about growing oncology trials and how they can be conducted and enabled with the virtualization of the protocol design.”