3 Tips for Applying DCT Approaches in Rare Disease Trials
Decentralized clinical trial (DCT) approaches can be extremely useful in helping to facilitate clinical research participation for rare disease patient populations. That said, many sponsors new to conducting studies that include DCT elements may need some help getting started. Following are some tips based on THREAD’s experience helping sponsors to select and execute DCT approaches for their rare disease studies.
Tip #1 – Consider the Unique Needs of Study Participants
The primary reason it can be so difficult to recruit and retain rare disease study participants is right there in the name – the disease is rare. When we are able to find eligible study candidates, it is important that we make study participation as simple and accessible as possible to enroll them and keep them engaged over the course of a trial. For example, many rare disease sufferers possess symptoms that make day-to-day tasks more difficult. Challenges to mobility, memory or the presence of chronic pain can be non-starters for potential participants, especially if a study requires a huge list of assessments. Understanding the difficulties of the people likely to enroll in a study can help you choose DCT elements that will remove barriers to engagement. For patients with mobility challenges, DCT approaches like virtual visits and wearable sensors allow them to stay at home while still providing study teams with the necessary data. Managing a rare disease can often be demanding and taking part in a clinical trial more so. DCT applications that remind the participant or their caregiver to take medications, fill out eCOAs, or perform other study tasks can take that away some of that management burden.
Tip #2 – Ease the Pathway to Participation for Research-Naïve Sites
Because rare diseases are, well, rare, there are limited study sites with deep experience in clinical research. This likely means that sponsors investigating new rare disease therapies will need to turn to sites that have limited trial experience. To avoid creating new burdens for these sites, sponsors can choose technology platforms that help them to manage the study process every step of the way. Solutions that provide real-time analytics into study performance go a long way in helping study teams stay on top of participants. Simplified dashboards can provide immediate insights while allowing study team members to dive down into specific performance areas (e.g. eCOA submissions, trends in sensor data, etc.) to ascertain how participants are doing and plan any necessary interventions. The right study management platforms can help get new sites up and running faster while expanding access to new groups of potential study candidates.
Tip #3 – Make Complex Trials Simple
All clinical trials are complex. Rare disease studies can be more complicated than others and, because they often lack a good understanding of disease progression or clinical subgroups, they also can lack well-defined endpoints. Adding DCT elements, while making studies more accessible to participants, can ramp up the complexity if sponsors and sites have limited DCT experience. It is important to work with partners that truly know how to conduct successful DCTs. The right partners can provide trial design inputs that serve to simplify the experiences for participants, caregivers, and sites. Any technologies (smart device apps, mobile medical devices, wearable sensors, etc.) implemented need to be intuitive and as easy to use as possible. Further, there needs to be a support system in place that is easy to engage with because even the best-designed technologies will have hiccups or raise user questions.
Making complex rare disease studies less intimidating through well-designed DCT approaches can make a significant difference for sponsors, particularly in terms of participant retention and compliance with study responsibilities. By employing participant-first thinking and building trials that make the experience smoother for all involved, sponsors can set the stage for success in their rare disease studies. For more information about the use of DCT approaches in rare disease trials, check out THREAD’s recent report on the topic, or visit THREADresearch.com.
Joyce Moore
Head of Rare Disease Solutions
Joyce brings over 22 years of clinical drug development expertise where she has worked effectively across multiple disciplines, therapeutic areas, and clinical stakeholders. For the last 16 years, she has focused on patient engagement and has a deep understanding of the Rare Disease and pediatric clinical trial space.