By Ann Neuer

March 25, 2016 | Science 37, a two-year old startup based in Culver City, California, was formed by experienced clinicians determined to re-imagine and bring disruptive change to the inefficient clinical trial process.  How about shifting to a more patient-centric, rather than site-centric, approach that could shorten patient enrollment timelines and boost retention, two widely acknowledged intractable problems?  Apparently, the clinical trials-savvy judges agreed with this assessment and named Science 37 one of the winners of the 2016 Clinical Informatics News Best Practices Awards, announced at the recent Summit for Clinical Ops Executives (SCOPE) in Miami, Florida. 

The winning entry detailed Science 37’s so called metasite model, a virtual site that speeds enrollment by allowing patients to participate in trials remotely—from the comfort of their home—via the company’s Network Oriented Research Assistant (NORA) technology platform.  This is a significant advantage as research suggests that less than 30% of patients live within two hours of an investigative site.

Science 37 used the metasite model in the PEMPHIX trial, a Phase III study for a rare condition known as pemphigus vulgaris (PV), an auto-immune skin condition that causes skin to form blisters and sores.  The metasite was the study’s only telemedicine-based site.  Working in conjunction with LA BioMed, a nonprofit research organization affiliated with the David Geffen School of Medicine at UCLA, Science 37 looked to accelerate patient enrollment for the PEMPHIX trial by improving access and the patient experience.  This was an intense challenge, given the rare nature of PV—only 30,000 to 40,000 cases in the US, and found in less than 1/100th of 1% of the global population.

Noah Craft, MD, Ph.D., co-founder and CEO, explains, “We are using technology to remove the geographic barriers and unlock access, an important factor in rare diseases.  A high percentage of people want to participate in clinical trials, but less than 5% do so because of various reasons, including difficulty with access.  Because we are the physicians and researchers ourselves, our model is centered on treating the patient as our primary customer.  This is quite different from how CROs and sponsors operate.”

The patient-centric approach focuses on a simple and engaging experience for patients using NORA technology, which functions as an app easily accessible on their mobile phones.  Once the patient is enrolled in the trial, NORA enables communication with the research team, which is conducted through secure video chats and messaging.  In addition, patients can log into their study, and have direct access to the investigator and study coordinator.  This works by the patient touching the photo of the coordinator, for example, which results in a call to that coordinator’s cell phone 24 hours a day.  Home-based patient visits are conducted by mobile nurses who use iPads to enter source data into the NORA Amazon Web Services cloud-based database.  Coordinators can then enter those data remotely into an electronic data capture system.

This approach seems to be working, as in the first six months of the PEMPHIX trial, the metasite was responsible for enrolling 30% of all enrolled subjects, translating into an enrollment speed that was more than 20 times faster than the projected per site enrollment rate for the study.  This is a significant finding, as according to clinicaltrials.gov, the study is actively recruiting at 60 sites across the globe, and hopes to achieve an enrollment of 124 patients, averaging just two patients per site.  Craft credits the company’s success to both its hi-tech/hi-touch approach and the metasite model providing an extensive geographic reach, recruiting patients from seven states, essentially reaching 23% of the US population from the single metasite. 

The focus on patient engagement and empowerment may be a key factor in the company’s extraordinary 100% retention rate.  So far, in the company’s limited experience, approximately 200 patients have participated in studies using the metasite approach, and “no one has dropped out.  It’s about the experience for patients, which is profoundly different than a normal trial, where they have to go to a site, maybe take time off of work, pay lots of money to park, and maybe not talk to the doctor.  The technology might sound impersonal, but when we ask patients about their experiences, our feedback says otherwise,” Craft notes. 

Science 37 is a young company, but its model is gaining traction, and would be the envy of many organizations.  According to Craft, “We’re enjoying an incredible pipeline, with several more interventional studies starting within the next month, all without any sales or marketing effort.”

This unique approach allows for casting a wide net across the US to speed patient enrollment and retain those patients due to providing a positive experience.  This is telemedicine at its best.