Challenges In Rare Disease Studies And How Decentralized Trials Can Support

Rare diseases cover a wide spectrum of conditions affecting different organ systems with varying severity, age of onset, and prevalence, each with their own individual nuances and challenges. Getting a diagnosis can be a lengthy process involving many different medical specialties and, as such, these patients tend to be well-informed on their disease. That said, their disease can also have progressed significantly prior to finding a suitable treatment pathway or research study.¹

Many of those suffering from rare diseases possess symptoms that affect their mobility, increasing the burden of traveling to clinical sites – both for standard care and for research study purposes. Limitations from patient symptoms can make clinical trial participation particularly challenging for this already limited number of patients, in turn increasing the difficulty for sponsors seeking to conduct rare disease therapy studies.

With the use of digital platforms and supporting processes, decentralized clinical trials (DCT), can move many aspects of clinical trial participation from the traditional research site to a remote location, most often the participant’s home.

DCT approaches that allow for remote data collection can help to improve how rare disease studies are conducted in several ways. Find out how utilizing technology to capture and submit data from the patients’ homes helps to reduce the number of necessary clinical visits and lighten the study burden for patients and caregivers.