Challenges In Rare Disease Studies And How DCTs Can Support

Rare disease studies tend to be more complex than most.

This is due to a variety of factors that can include diversity in clinical disease progression, the lack of well-established clinical endpoints, as well as a limited number of potential patients who are thus geographically dispersed. Compounding these challenges, the FDA and EMEA often use different criteria for assessing rare disease studies, causing sponsors to create protocols that attempt to meet both sets of requirements.

While these protocols are, of course, designed to achieve the required safety and efficacy data, the procedures and visit schedules can place a high degree of burden on patients and their families.

THREAD is excited to share our report 'Challenges In Rare Disease Studies And How Decentralized Trials Can Support'.

Download to learn:

• Why enhanced site training and support is needed for rare disease studies
• How decentralized clinical trials (DCTs) can minimize the patient burden
• Where DCTs can best be applied in the drug development pipeline