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Regulatory Focus™ > News Articles > 2021 > 3 > Trials without clinical sites offer chance for improved access, generalizability

Trials without clinical sites offer chance for improved access, generalizability

Posted 05 March 2021 | By Mary Ellen Schneider 

Trials without clinical sites offer chance for improved access, generalizability

The COVID-19 pandemic has fueled the rise of clinical trials without clinical sites and opened the door to continuing these innovative models after the public health crisis has faded. While these trials offer the potential to expand access and the generalizability of results, there are also some limitations to conducting drug trials in this format.
In a special communication in JAMA Internal Medicine, Steven R. Cummings, MD, director of the San Francisco Coordinating Center and research scientist at the California Pacific Medical Center Research Institute, outlined the different designs for trials without clinical sites, the benefits and barriers, and the lingering research questions.
“The pandemic and the [Food and Drug Administration] have enabled people to be innovative and creative. The article is essentially a toolbox to help people think differently,” Cummings said in an interview.
Not all clinical trials without clinical sites are built the same way; several different models already exist. For instance, site-free or remote trials have no clinical sites, while home-based trials rely on at-home specimen collection and often employ mobile nurses to administer treatment. A virtual trial makes use of telemedicine technologies to conduct digital face-to-face visits. Other trials – referred to as decentralized, hybrid, or disseminated – are trials that may have clinical sites as well as remote data collection, virtual visits, and home-based services.
Launching a clinical trial without clinical sites requires rethinking many elements of the process, from recruitment to informed consent to how to administer treatments, with participants taking on more of the functions previously performed by research staff. Many observational trials, as well as studies of behavioral interventions, have been successfully conducted without clinical sites, Cummings noted. However, the process is more complicated when it comes to trials evaluating drug therapies.
Cummings said it is important to consider whether all the essential assessments in the protocol can be done outside of a standard site, either by nurses in the home or at a nearby lab or radiology facility. Also, the protocol and enrollment must be sufficiently simple for the intended patients to complete it at home with minimal or only remote assistance.
One of the most difficult parts of designing and running a trial without clinical sites is creating and maintaining relationships with patients, Cummings said. Telephone calls, emails, texts, and video contact with trial staff can help to compensate for the lack of in-person visits, he said, as can having a “concierge” to guide patients through the process.
While FDA has not been a barrier to conducting trials without clinical sites, the agency has yet to approve any drugs based on findings from these trials. Also, regulatory barriers remain to conducting trials without clinical sites internationally, Cummings noted.  
“Pharmaceutical companies may increase their use of these methods as confidence grows that the FDA will accept data from trials conducted without clinical sites as part of approval of new drugs or new indications for existing ones,” Cummings wrote.
Going forward, Cummings said data are needed about the yield and cost of recruiting participants to trials without clinical sites, retention of patients over long-term trails, and the comparative costs for trials with and without clinical sites.
JAMA Internal Medicine


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