Decentralized clinical trials (DCTs) are lauded for their patient-centric nature and their increased ability to withstand the impact of unexpected influences like pandemics. DCT benefits for adult clinical trials are often discussed, but what benefits do decentralized trial elements bring to pediatric clinical trials? Children and their families stand to gain much from the continued adoption of DCTs.
Children and their families stand to gain much from the continued adoption of decentralized trials. PRA's most recent webinar brought together speakers from diverse stakeholder positions to discuss the benefits and challenges of decentralized clinical trials in the pediatric population.
PRA’s Center for Pediatric Clinical Development recently hosted a webinar with Xtalks titled Decentralized Clinical Trials: Let’s Not Forget Pediatrics! This webinar brought together speakers from diverse stakeholder positions to discuss the benefits and challenges of decentralized clinical trials in the pediatric population.
PRA Health Sciences’ Isaac R. Rodriguez-Chavez, Ph.D., M.H.S., M.S., SVP, Scientific and Clinical Affairs; Head, Global Center of Excellence Strategy for DCTs, began the webinar, reminding us that elements of DCTs are universal and are applicable to pediatric populations as well.
“We know it is critical to maintain children and their families in the places where they live, their homes, their cities, their towns, their countries. So, we minimize disruption to their lives and have that opportunity to increase initial engagement and retention of these participants, mitigating the stress that is related to participation in clinical trials,” said Isaac.
It’s important to intervene with these populations early, which allows them access to medicines they would not have access to otherwise. Isaac specified DCTs can provide access to care for underserved populations, allowing equity of access to care where previously there was none.
Isaac went on to cover some of the considerations a study must account for in pediatrics, such as changes as the child grows, anatomical and physiological differences, energy and maturity differences, and medical procedures specifically designed for children. He also emphasized the importance of family-specific enrollment and retention strategies to account for the influence of the family in decision-making. Additionally, there are logistical considerations, like disruption of family life, and regulatory considerations for DCTs. Pediatric DCTs have the same regulatory expectations as traditional pediatric trials and follow the same guidelines as adult DCTs. For a list of regulatory considerations, please see the full webinar here.
Shelly Barnes, Global Clinical Innovations Lead, UCB Biosciences, provided viewers with the sponsor’s perspective of implementing DCTs in the pediatric population. It was evident from her presentation that UCB is embracing DCTs—and she even shared that an adolescent DCT was one of the few studies at UCB that went unaffected by the COVID-19 pandemic.
The FDA and other agencies are open and supportive of DCT models. The benefit for families and patients is clear. Shelly admitted that DCTs are often met with hesitation by industry professionals who have long been a part of the traditional clinical trial delivery methods. She stressed the importance of educating operations teams about DCTs to alleviate concerns.
In order to make operations teams comfortable with implementing DCTs, educational resources and an open environment for questions are key. UCB has also adopted an early engagement innovation strategy, relying on patient surveys, panels, and early patient input to discover what was valuable to a particular patient population. Because there is no one way to develop a DCT, each can be designed to the appropriate extent of decentralization and should be informed by patient feedback.
Shelly pointed out that DCTs can introduce risk to a trial. Risks need to be carefully considered and minimized as much as possible. There can be risks to individuals and the data collection. Living spaces can be unclean, and location/family dynamics could be unsafe for home health nurses or the child. Close relationships with sites can help a sponsor understand the risk considerations for certain patients and design an approach that mitigates the risk.
It is important to keep in mind that for DCTs, families must administer treatments, use multiple devices, welcome strangers into their homes, etc., in exchange for not traveling to the site. Shelly closed with a strong question: “While all of these interventions facilitate participants not to travel to a site, there is also some transfer of responsibility to the participant. So, our question is, where is the line? At what point does not traveling to a site result in a participant feeling too burdened with the costs of managing the remote interventions?”
Terry Jo V. Bichell, PhD, MPH, Founder/Director, COMBINEDBrain Consortium for Outcome Measures and Biomarkers for Neurodevelopmental Disorders, shared her perspective on DCTs as a mother caring for a child with Angelman’s Sydrome. Her son, Lou, was diagnosed and began participating in clinical trials to manage his symptoms at age one. Since then, he has participated in many clinical trials, including three DCTs, so Terry Jo was able to compare her experience with traditional and DCT models.
When the COVID-19 pandemic hit, Lou was participating in a DCT. Terry Jo said it was a blessing that they were not in a traditional trial at the time. The DCT made participation during the pandemic so much easier on her family and son, as his trial was able to continue without interruption. In previous trials, she had to navigate weekly plane rides round trip in a day while having four other children at home. This travel required her to hire a helper to assist her and Lou and an additional helper to take care of her other kids at home. It was a relief for her and her family to be able to participate in a trial without the extensive burdens they had encountered before. And, the DCT allowed her to keep her family safer from the risk of COVID-19 and ensure that Lou continued to receive his treatment.
Her most salient point was that DCTs allowed more accurate data collection. “Being in a decentralized clinical trial is so much easier on our family, so much easier on my son. You can actually see what he can do when he is in a DCT. If we haul him in a plane, or a car, or a train and arrive in a strange city…and try and get him to perform tasks that he doesn’t want to do, you can’t actually get a clear picture of what he’s actually like.”
Being in a decentralized clinical trial is so much easier on our family, so much easier on my son. You can actually see what he can do when he is in a DCT. If we haul him in a plane, or a car, or a train and arrive in a strange city…and try and get him to perform tasks that he doesn’t want to do, you can’t actually get a clear picture of what he’s actually like.
Terry Jo V. Bichell, PhD, MPH, Founder/Director, COMBINEDBrain Consortium for Outcome Measures and Biomarkers for Neurodevelopmental Disorders
Through the DCT, Lou was able to stay at home—a place where he was comfortable and better able to showcase the progress he made with the therapeutic. He was fully himself and avoided influences of environmental factors. The data captured from those visits was far more representative of the true therapeutic benefit he was receiving from the trial.
“It makes it a lot easier to be in a clinical trial…it’s not nearly as burdensome at all. So better data and easier to get people to participate,” Terry Jo added.
In closing, Terry Jo said that DCTs had been a great thing for her family and Lou’s care. The only disadvantage she felt the model presented was the greater difficulty in building relationships with Lou’s providers.
Our closing speaker was Christoph P. Hornik, MD, PhD, MPH, Associate Professor of Pediatrics with Tenure, Duke University and Duke Clinical Research Institute. As a provider, he offered the site perspective of DCTs. He began by emphasizing the overarching value of a DCT model is there, despite it being too early to establish hard best practices. Sites are valuable partners for designing DCTs—they can help with protocol design, participant assessments, and patient recruitment. For protocols, sites are able to consider the scientific complexity, feasibility, and identify home-based care programs. Sites have relationships with patients and patient communities, which means they can help locate patients for participation.
Christoph believes it’s important to consider how communication is managed, both with trial participants and their families and with patients’ primary physicians. “DCTs are an opportunity to enhance and promote [the patient-physician relationship] by taking some of the responsibility of offering trial participation to a patient off the shoulders of the treating clinician. Instead, we take it on as the DCT-executing entity and offer the information back to the physician to help inform the patient-physician relationship.”
As clinical trials continue to increasingly adopt decentralized aspects to improve clinical trials for all stakeholders, it’s important the same level of innovation extends to pediatric clinical trials. Thank you to our speakers for sharing their valuable insights with us and with our attendees.
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