As Pfizer begins enrolling the first-ever FDA-approved “virtual” clinical trial, which allows patients to participate from home using smartphones and computers instead of visiting a clinic, industry executives believe the study has far-reaching implications for improving the productivity of clinical trials in the future.
For the 16-week study, which will assess the safety and efficacy of an overactive bladder drug called Detrol LA, Pfizer will screen potential patients online, ship medicines to their homes and monitor the results remotely. The study, which will include 600 patients from 10 states across the U.S., will compare results with a previously completed phase IV trial on the same drug in the hope of validating the “virtual” clinical approach to research.
Researchers expect this new approach, called either direct-to-patient or patient-centered, will allow for faster study recruitment and enrollment, since patients can participate regardless of their proximity to investigative sites. Pfizer believes allowing patient participation from home also could lower study withdrawal rates, result in more reliable data and increase patient compliance.
“This approach, if proven successful, holds considerable promise in speeding up clinical trials while improving their quality,” said Briggs Morrison, M.D., Pfizer’s senior vice president of worldwide medical excellence. “This program and similar programs that may follow could lead to an entirely new way for patients to participate in trials and contribute to biomedical research.”
The direct-to-patient model also could result in significant cost savings for study sponsors. Anthony Costello, chief operating officer of Mytrus, the San Francisco-based startup running Pfizer’s trial, estimates sponsors could save from 25% to 60% over the traditional costs paid to CROs.
“There is a massive cost savings to the pharma industry in not having to start up dozens or hundreds of sites to enroll patients,” said Costello, who has co-founded several technology startups including Nextrials. “It’s not hard to imagine. If you take a trial that has 50 sites and cut it down to a trial that uses one site, you get away from contracts, IRB submissions, site initiation visits, trainings and payments with 50 organizations and do it with one instead.”
Industry executives already have begun to look at the opportunities the direct-to-patient model offers for improving the productivity of its clinical research process. Costello estimates up to 20% of trials today could be run with this approach. While the model won’t work for all clinical research, Costello said it could be used successfully in post-marketing studies, some late-phase studies, trials required to support a drug’s switch from prescription to over-the-counter status and phase II-III clinical trials that require long-term follow ups.
“When you add it all up, about 15% to 20% of clinical trials—which is thousands of studies a year—don’t have procedures or protocol requirements that necessitate patients go to a site with special equipment to be seen or have the product administered every few weeks,” he said.
With the Pfizer trial underway, Mytrus has begun talks with other large biopharmaceutical companies about opportunities for direct-to-patient trials. Mytrus has two direct-to-patient trials in the institutional review board (IRB) approval process; these trials are expected to begin within the next eight to 10 weeks. Other trials are in the discussion phase and are expected to start before year end.
While the direct-to-patient model holds great potential for improving productivity in clinical development, change may be slow. “We know how long it takes to turn an industry like this one onto a new technology and make it standard, especially those of us who came through the EDC revolution. We don’t think that tomorrow everyone is going to be running direct-to-patient clinical trials,” said Costello.
“What we are seeing so far are early indicators that the industry is starving for some way to recruit patients faster and do the trials more cost effectively,” he said. “Patients out there are also starving for clinical trials that meet their medical needs.”