In the past 5 years:

• 130+Clinical Trials
• 50+Indications
• 2,500+Sites
• 13,000+Subjects

Rare disease clinical trials pose a number of unique challenges: difficulty in diagnosis, finding homogenous populations, obtaining clean data, staying on top of evolving regulatory requirements, and addressing the difficulty and strain of these diseases on patients themselves. These are significant trial challenges for people whose diseases place them alone in a crowd.

At Synteract, we specialize in studies for diseases that affect a small portion of the population or that are otherwise “forgotten” by other treatments. In the past 5 years alone, we have conducted more than 130 trials in rare and orphan disease, covering a wide range of therapeutic indications.

We understand that both rare and orphan indications most often come with unique characteristics. Recruitment can be challenging, because a high number of study sites are required to enroll a very small number of patients. With many rare diseases genetic in nature, it can be emotionally tough for parents to make decisions about a trial affecting one or more members of their family. Case report forms are more complex, and there may be scientific limitations. Statistical significance may be limited as well, so analysis may hinge on individual patients and historical comparison. Working with regulatory authorities throughout the process is critical.

Read this case study to discover how we helped a clinical-stage biopharmaceutical client obtain orphan drug and First PRIority MEdicines (PRIME) Designation in Europe.

For all these reasons and more, proactive recruitment and mental agility are required by investigators who understand what it takes to bring rare/orphan patients into the clinical trial process. We don’t want to lose a valuable patient due to lack of proximity to an investigative site. We have a strong history of working with and guiding investigative sites and patients through these unique scenarios. Strong relationships with patient advocacy groups have been critical in linking us to these hard-to-find patient populations.

“…For those of us who came into the industry with the desire to provide a service
and give hope to patients with unmet medical needs,
the rare disease area is the most exciting place to be.”

Lisa Dilworth, VP, Rare and Orphan Diseases at Synteract

We have the operational expertise and regulatory acumen to build a study program that works, integrating patients, sites, advocacy groups and the sponsor in bringing clinical trials to life and new medicines to market!

Contact us today to find out how we can help you bring your rare disease clinical trial to life.