New Decentralized Clinical Trial FDA Draft Guidance Emphasizes the Need for Enhanced Communication for Successful DCTs

On May 2nd, 2023, the US Food and Drug Administration (FDA) took a significant step to support the use of decentralized clinical trials (DCTs) by releasing a draft of guidance fully devoted to DCT practice. The FDA defines DCTs as “clinical trials where some or all of the trial-related activities occur at locations other than traditional clinical trial sites.” The agency acknowledges the potential benefits of DCTs in improving accessibility, increasing diversity, and facilitating drug development in clinical research if implemented appropriately. 

The release of this decentralized clinical trial FDA draft guidance (hereto referred to as FDA draft guidance) is applicable and timely as DCTs become more widely accepted and adopted. DCTs are often attributed to having a patient-centric approach, which can improve diversity, equity, and inclusion in clinical trials; however, it needs to be acknowledged that DCTs aren’t a one-size-fit-all solution. What makes DCTs remarkable is their flexibility: the ability to tune the level of decentralization depending on the study and patient population’s needs. Some protocols might be best suited for full decentralization, while others might be better as hybrid trials that incorporate both decentralized and traditional methods.

Designing and implementing the right level of decentralization into the study protocol is crucial for achieving clinical trial success and improving patient experience. To accomplish this, effective communication and transparency between all key stakeholders is essential for unlocking the full potential of trial decentralization and maximizing the benefit for all patients, sites, and sponsors. The FDA draft guidance provides key insights into when and how adjustments to DCTs are necessary depending on the nature of specific clinical studies. 

Highlights of the FDA Guidance – Dialing Trial Decentralization to Fit Study and Patient Needs

DCTs for Certain Investigational Products 

The suitability of investigational products (IP) for DCTs depends on specific characteristics. According to the draft FDA guidance, DCTs may be appropriate for IPs with a well-characterized safety profile and a less complicated administration process. The draft guidance also notes that IPs with long shelf life and good stability profile are ideal for shipment through direct-to-patient (DtP) methods. It is crucial for sponsors to consider these factors when determining whether DCT and DtP shipments are appropriate for a particular IP. 

DCTs for Certain Trials 

The draft guidance suggests that the effect size observed in a traditional trial may differ from what is observed in a DCT, depending on the hypothesis being tested. For example, the FDA notes in non-inferiority trials, where a new drug is compared to an already known active intervention, it is not safe to assume that the effect size determined in a DCT is the same as the effect size determined using traditional methods. Hence, it would be critical to consult the FDA when planning a DCT for a non-inferiority study to determine the appropriate effect size. 

Healthcare Providers in DCTs

Some trial-related activities can be delegated to local healthcare providers (HCPs) who are geographically close to patients’ homes or their preferred locations; however, the FDA notes that sponsors should take into consideration the risk of variability in data collected by different personnel, as well as the complexity of trial activities when determining whether or not to delegate tasks to local HCPs. For instance, an IP that requires a complex administration process may be more suited for personnels at traditional sites to perform.

Safety Monitoring in DCTs

As in traditional trials, patient safety must be prioritized in DCTs, and a safety monitoring plan should be adapted based on the decentralized nature of this clinical trial. The plan should include the method and frequency of remote-check in to monitor patients’ well-being, as well as clear guidance for patients on what to do and who to contact in the event of adverse events (AE). 

Knowing is Half the Battle

DCT is a powerful tool that could revolutionize the way we conduct clinical trials. As the FDA emphasized in their draft guidance, DCTs have the potential to increase diversity, improve accessibility, and facilitate drug development, thereby accelerating the availability of novel treatments to the market. It must be noted, however, that DCTs are not a one-size-fits-all solution. Trial decentralization needs to be tailored to the specific needs of each clinical trial and patient population. To accomplish this, sponsors must have a deep understanding of the patient population being studied, site capabilities, and the specific purposes for adding decentralized solutions and capabilities.

As the FDA states, ensuring patient safety must be the primary priority when conducting clinical trials, be it traditional, hybrid, or in a fully decentralized model. The benefits must outweigh the potential risks and drawbacks when considering DCTs (or any other clinical trial solution). For the patient population being studied, patient experience must be considered. Some additional questions may include, will DCT capabilities reduce the patient burden? Is there enough representation in the participants recruited through DCTs or through traditional methods? Will DCT improve the diversity of patients participating in clinical trials, hence making the results more generalizable to the intended patient population? 

If DCT solutions are deemed beneficial for patients in your studies, the patient experience must be considered. Not all technology is created with the patient in mind. It is incumbent upon us that we put every effort into making trial tasks seamless and less burdensome as possible for patients, trial sites and sponsors. Disparate systems commonly used by different sponsors may put more burden on sites in the form of administrative hurdles, increased trainings, and reduced patient-physician interactions. These pain points may arise when sites and sponsors are not aligned with their preferred solutions, and ultimately translate to patient burden, retention issues, and poor experiences. This prompts an open dialogue among stakeholders to ensure trial sites’ needs are fulfilled, and push towards using a unified platform that could address these issues for both sites and sponsors. 

Communicate this Plan

The ultimate goal of all stakeholders is to bring novel treatments to the market that improve and extend patients’ lives. By using emerging technologies, all stakeholders must demonstrate strong collaboration and communication to align and determine the most ideal trial decentralization model that fits the needs of each study and patient population. It is crucial to communicate with the FDA to establish a robust guidance and recommendation that can serve as a pillar for designing and implementing effective DCTs that benefit patients in the best possible way. In addition, it is necessary to communicate with sites to understand their capabilities and feasibilities in implementing the trial design. Most importantly, communication with patients is essential to ensure the trial decentralization solutions are easy to understand and follow.

As your trusted DCT solution providers, Medidata is here to help you develop the DCT solution that is the right fit for your trial. We recognize the importance of seamless experience for all patients, sites, and sponsors, as well as weaving in patient voices into all technology solutions. At Medidata, we provide the industry’s only unified platform for all technology solutions, which we continuously develop and enhance by infusing patients’ voices from various backgrounds and therapeutic areas. 

Inspired by patients, powered by data, and delivered to scale. The future of DCTs is bright! Please contact us to help you design and implement the right DCT solution for your trial. 

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