The US FDA has underlined its support for decentralized trials with new draft guidelines designed to encourage industry to study drugs in a more patient-centric way.
The guideline – available here – sets out the agency’s position on decentralized trials and includes several provisions designed to encourage use of the approach. These include permission to obtain lab tests at a local facility rather than a research medical center and license to conduct follow-up “visits” using telemedicine.
The document builds on guidelines in 2020 that were designed to facilitate trial decentralization in response to the COVID-19 public health emergency and its associated disruptions such as quarantines, site closures and travel limitations.
Decentralized trials will play an important role in drug research going forward according to US FDA commissioner Robert Califf, who set out the benefits of the approach in a statement marking the draft guideline’s release.
“As we seek to improve our evidence generation system, decentralized clinical trials may enhance convenience for trial participants, reduce the burden on caregivers, expand access to more diverse populations, improve trial efficiencies, and facilitate research on rare diseases and diseases affecting populations with limited mobility.”
Industry response
In general, the draft guidelines were welcomed as a positive step by the trials sector and – according to experts from decentralized technology developer Medable – is likely to encourage the use of site-less drug studies.
Chief Scientific Officer Pamela Tenaerts told Clinical Insider “The FDA’s Draft Guidance will help create a level of comfort in DCTs for sponsors, sites, CROs, and other stakeholders. I applaud the requests for more upfront planning and making sure workflows and communications are in place. This fits well within the quality by design frameworks that are being adopted by regulators and ICH.”
She added, “Fundamentally, the FDA isn’t issuing anything new in its latest Draft Guidance – yet it has breathed new, sustaining life into modern clinical trials. The FDA has infused credibility into tech-enabled trials, crystallized some of the operational rules (though, there is much more work to be done here), and legitimized the DCT space. What was once thought to be a temporary band-aid to get the industry through the COVID-19 pandemic, is here to stay. And, finally, a clinical trial is just a clinical trial – not decentralized or hybrid or any other characterization.”
Kevin Potgieter, the firm’s vice president of regulatory affairs, was also positive about the guideline.
“The FDA Draft Guidance further validates the DCT model and provides additional regulatory clarifications around its implementation and operation. We expect sponsors will study all FDA guidance on DCT very closely and continue to increase adoption of hybrid and fully decentralized studies,” he said.
Quality concerns
Although she welcomed the guidance, Tenaerts did raise some questions about the guidelines, particularly in relation to quality control standards.
While certainly not a pervasive sentiment in the new Draft Guidance, I would urge the FDA to avoid holding DCTs to a higher standard than brick and mortar trials…There is just as much potential for variability between investigators and site personnel collecting data in traditional trials as there is in DCTs and this statement may indicate that this must be more controlled than ever for decentralized clinical trials.”
Similarly, Potgieter called on the FDA to add more details, adding “I think it would be helpful to see examples of completed Form 1572, perhaps one for a fully decentralized study leveraging local HCPs entirely, and another for a hybrid design.
“Also, it would be helpful for the FDA to provide guidance in a Q&A format for sponsors who may like to know about the new content and structure for the DCT-relevant content FDA will be requiring in IND submissions going forward – something akin to the guidance for ongoing trials that came out during COVID-19 to create a working document.”
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