While the COVID-19 pandemic has accelerated innovation in many areas, including clinical trial design, many recent advances were already in motion before 2020 as sponsors and CROs sought to improve the patient journey and encourage trial participation. Traditional trials, especially those for pediatric populations, often place enormous burdens on patients and their families.
A busy family may find it prohibitively difficult to overcome barriers related to transportation, child care, time off from school and work, and other considerations. Such issues in turn lead to longer recruitment timelines, less diverse patient populations, and lower retention and compliance rates. Many of these problems can be alleviated by the implementation of decentralized or hybrid clinical studies.
Planning of decentralized clinical trials
Decentralized clinical trials (DCTs) are conducted partially or entirely in a location other than a clinical brick-and-mortar site. Visits are conducted by a home health nurse and typically take place in the homes of the patients involved. For pediatric studies, they also could occur in a school nurse’s office or even a daycare center. Executing a DCT requires significant planning and outside-the-box thinking, but the convenience it affords significantly reduces the burden on patients and their families.
When planning a DCT, sponsors should account for several considerations unique to these studies:
Principal investigator oversight: The sponsor must prove to the FDA that there has been adequate oversight of the visits conducted in remote locations.
Consistency and data accuracy: Given the number of locations and people involved, the sponsor must ensure that visits are being carried out and data are being collected in a consistent manner. Making adequate training materials available to the home healthcare nurses is key.
Identification of study sites: For a hybrid study with diverse geographic locations, the sponsor must carefully select sites that have the necessary capabilities for the study and that are accessible to the participating patients.
Additional vendor management and costs: While the sponsor can spend less on investigator fees with a DCT, the costs of technology vendors, home healthcare services, direct-to-patient shipping, and other considerations are higher than for traditional clinical trials.
One of the great advantages of conducting a DCT is the availability it affords a sponsor to use modern technology to aid in recruitment. Since there are fewer geographical restrictions, sponsors can make greater use of social media campaigns and patient advocacy groups to inform patients and attract a more diverse population.
Furthermore, many modern advancements allow clinical trial data to be collected with little or no disruption to a patient’s or family’s routine. Wearables allow up-to-the-minute patient information to be collected. Parents already using apps to track a child’s health can seamlessly transition to doing so for a clinical trial. Adolescents who would be reluctant to comply with study treatment and visit requirements can track and report their progress on their phones, a familiar medium.
Such advancements also allow for real-time study monitoring and better training for those conducting home visits. For example, the Premier Consulting team has incorporated glasses with attached video cameras into the study plans for a growth monitoring and safety study for full-term healthy newborns. The glasses, worn by home healthcare nurses during visits, provide a hands-free way for nurses to evaluate and measure the infants, while the study’s principal investigator watches in real time. In this way, the principal investigator can address any issues that arise and ensure consistent and accurate data across all locations.
Benefits of decentralized clinical trials
While DCTs are not appropriate for every development program, sponsors who are able and willing to conduct them can reap several clear benefits.
Patient recruitment, retention, and compliance: The reduced burdens for patients and lessened geographical constraints lead to shorter recruitment times, more diverse patient populations, and higher rates of participant retention and compliance.
Higher-quality data: As noted above, wearables, apps, and other technologies can ensure that researchers collect accurate, real-time data that can inform patients’ treatment plans. Furthermore, home healthcare nurses may spot environmental factors in a patient’s residence that could affect the study data but that would not be apparent if the visit were conducted at a traditional study site.
Better patient experience: At the heart of each clinical trial are the patients who will benefit from the therapy being developed. Choosing to implement a DCT is one way a sponsor can improve the patient journey for its development program and reduce the burden on families and caregivers.
Conducting a pediatric DCT requires a deep understanding of the pediatric research space and the ability to think creatively to overcome the many unique challenges a sponsor will face. Contact us to find out how we can support your pediatric clinical program.