New FDA Draft Guidance Signals Acceptance of Decentralized Trials (When Done Right)

What has Covid-19 taught us about virtual clinical trials?


image for clinical trial design cover

When regulators speak, we listen.

Naturally risk-averse industries like the life sciences need health authority acceptance before trying new approaches and especially before committing to wholesale operational change, even if the outcome has great potential. Consider decentralized clinical trials (DCTs) – a new model for conducting research. DCTs saw a sudden uptick during the Covid-19 pandemic but they are still not ubiquitous industry wide despite their significant benefits.

The Food and Drug Administration (FDA)’s Decentralized Clinical Trial Draft Guidance issued on May 2 may give DCTs the shot-in-the-arm they need for broader adoption – not because of the specific operational guidelines outlined in the document, but simply because the document now exists. It signals acceptance of decentralized trials when designed fit-for-purpose.

The guidance document is short at 19 pages, and not particularly prescriptive. It covers decentralized clinical trial (DCT) design, remote clinical trial visits and other activities, the use of digital health technologies as enablers of studies, the roles and responsibilities of sponsors and investigators, and how investigational medical products should be delivered. It also lays out the agency’s current thinking on informed consent, ethics oversight, packaging and shipping of investigational medicines and devices, and the factors that should be considered in drawing up safety monitoring plans. Protocols are likely to become longer with the additional expectations of the FDA for clarification and documentation of planning and coordination of various stakeholders.

But, in many areas, the Draft Guidance is still vague, such as on the question of responsibility for PI oversight when trial activities occur outside of the PI’s direct supervision and data is collected away from the site (i.e., home health visits). Further, the industry will need clarity beyond what the FDA has described regarding the completion of Form 1572 for hybrid and fully decentralized study designs (the FDA now encourages a central address where audits can take place even when some trial staff are in various locations). Also, it would be helpful for the FDA to provide guidance in a living Q&A format – a working document for sponsors who want to know about the new content and structure for the DCT-relevant content the FDA will be requiring in IND, IDE, or other submissions going forward – akin to the guidance for ongoing trials that came out during the pandemic.

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The FDA has made a purposeful choice to write in broad strokes, stopping short of detailing ways to execute DCTs. Even so, the Agency is starting to acknowledge – and thereby support – the global shift towards expanded trial models. Essentially, the FDA is implicitly broadening the definition of clinical trials. Soon, we won’t say “decentralized” or “hybrid” or “digital” clinical trials. We will simply say “clinical trials,” recognizing that digital components will be baked into each trial innately. Remember how people in the 1990s used to refer to phones as “cellular phones” and now they are identified as simply “phones?” We are making a similar transition now in clinical research, thanks in part to the FDA.

Advanced trial methodologies have been used sporadically in clinical research for decades, but the FDA has only just started issuing a flurry of draft guidance documents for the use of various methodologies and technology components. In doing so, the FDA is normalizing the use of modern technologies in clinical trials – no longer trying to convince the industry to use technology, but rather, demonstrating how to use it while holding to critical patient safety and data quality clinical trial fundamentals.

Here are a few examples:

This is a global revolution, too. In December 2022, the European Medicines Agency (EMA) published its Recommendation Paper on Decentralized Elements in Clinical Trials, replacing its emergency pandemic guidance with a more robust framework. The paper emphasizes the protection of study participants, data integrity, and clear definition of roles and responsibilities for study sponsors, investigators, and other stakeholders. Like the FDA, the EMA supports the increased use of technology in clinical studies, so long as the fundamental principles of good study conduct remain intact. Additional health authorities around the world will follow suit as major markets make tech-enabled trials the de facto standard for quality, cost efficiency, and patient experience.

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Beyond geography, the new guidance is unique in that it includes all potential investigational medical product centers – Center for Drug Evaluation and Research (CDER), Center for Devices and Radiological Health (CDRH), Center for Biologics Evaluation and Research (CBER), and Oncology Center of Excellence (OCE) – thereby, including all investigational medical products – drugs, biologics, and medical devices. This opens new doors for all market sectors. For instance, device manufacturers benefit from a potentially more efficient way to conduct follow-up studies and post-market clinical follow-up (PMCF) as required in the EU. DCTs enable streamlined methods to capture valuable data about the quality-of-life impact of their innovations over longer timelines and in a way that is nearly burdenless to patients.

Fundamentally, the FDA isn’t issuing anything new in its DCT Draft Guidance – yet it has provided considerable support and helped define the landscape for modern clinical trials when deployed responsibly and fit for purpose. The FDA has infused credibility into tech-enabled trials, crystallized some of the operational rules (though, there is 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 pandemic, is now verifiably here to stay.

We hear you, FDA, and are grateful for the directional support. At the same time, we recognize that the ultimate proof in the pudding comes in the form of more efficient, population-representative research that provides reliable data to drive new therapies to patients faster.
It’s coming soon – get out your spoons.

Photo: exdez, Getty Images


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