Value of DCT Approaches in Rare Disease Trials
Clinical trials for rare disease therapies pose unique challenges. Rare disease studies tend to be complex, due to a lack of historical data, well-defined clinical endpoints and available comparators. Rare conditions, inherently, mean that the pool of potential study candidates will be small which causes challenges to recruitment, as does the fact that these participants are often managing debilitating conditions. All of this increases pressure on the study team to keep enrolled participants engaged and to try to prevent dropouts.
Elements of decentralized clinical trials (DCTs) can be employed to mitigate these challenges and increase the odds of study success.
Recruitment and Retention
A recent study revealed that approximately 25% of all rare disease trials terminate due to insufficient recruitment.1 To answer this challenge, we can implement technologies that make it easier for patients and families to find and get into studies. DCT approaches can expand how we engage with these participants. For example, rare disease patients are often highly engaged with patient advocacy groups. These groups can help promote the study while adding built-in credibility for their audience. Patients visiting these advocacy groups online can be directed to a web page where they can find out more about the study, self-pre-screen, and be directed to a participating site. All this can help get qualified candidates enrolled quickly.
Additionally engaging these same patient advocacy groups early in the protocol design stage can help develop protocols that have the patient voice built-in and make participation as easy as possible. DCT elements also aid retention in other ways. Remote data collection can allow data collection from participants’ homes. Technology also facilitates in-home visits with clinicians via telehealth. These approaches help reduce participant burden by reducing the need for some in-person site visits, a major benefit for chronically ill individuals and those with mobility challenges. The ability to remain in the home more, while maintaining a link to their clinicians can help keep many participants enrolled.
Addressing Site-Related Challenges
The term rare also extends to clinical trial sites. Fewer rare disease studies compared to common diseases means there are fewer sites with the available clinical expertise. Further, the lower number of studies equates to less drug development experience for sites. Lack of experience means that sites may not be as familiar with elements such as good clinical practice (GCP) nor the extra systems and necessary regulatory requirements that need to be in place when compared to large study centers.
DCT elements can ease study processes for less-experienced or even research-naïve sites, but they will need support. Options like remote data collection provide frequent updates on patients that sites can monitor in near real time. This allows them to make informed decisions to support patients quickly. It also means that necessary site visits can be more focused – by eliminating the need for sites to collect ALL the data during in-person visits, they can instead focus on providing support for patients.
Rare disease trials are complex; the potential pool of study candidates and trial sites is small. DCT approaches can help by allowing for remote data collection and streamlining processes for sites. By reducing the burden for rare disease study participants and giving sites the tools they need to manage these complicated studies, DCTs can facilitate better study experiences and more successful rare disease trials. To read more on the subject of DCT approaches in rare disease studies, take a look at our recent report, Challenges in Rare Disease Studies and How Decentralized Trials Can Support.
Joyce Moore
Director, Strategy and Partnerships
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.