Rare and Orphan Diseases Require Special Treatment


In the past 5 years:

  • 136+ Clinical Trials
  • 50+ Indications
  • 3,000+ Sites
  • 15,000 Subjects

In recent years, there has been a rapidly growing demand for trials in rare and orphan indications partly because of legislation in both the US and the EU that has established incentives to support research in these areas, making it more financially viable. However, the more pressing issue is that patients are waiting!

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 136 trials in rare and orphan disease, covering a wide range of therapeutic indications.

Rare and Orphan Indications Experience:

Synteract Rare/Orphan Pie Chart
  • A - Oncology: 41%
  • B - Respiratory: 15%
  • C - Hematology: 13%
  • D - Metabolic: 11%
  • E - Neurology: 9%
  • F - Gastroenterology: 5%
  • G - Endocrinology: 2%
  • H - Ophthalmology: 2%
  • I - Rheumatology: 1%
  • J - Transplantation: 1%

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. 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.

For all these reasons and more, proactive recruitment and mental agility are required by investigators who understand what it takes to bring these patients with rare and orphan diseases 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. This has also involved having strong relationships with patient advocacy groups critical to linking us to these hard-to-find patient populations. 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!


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