Rare & Orphan Disease Clinical Trials

Rare & Orphan Diseases CRO

In the past 5 years:

  • 195+Projects
  • 75+Different Indications
  • 2,400+Sites
  • >11,000Patients

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. Spanning all phases of development (0-IV), in the past 5 years alone we have conducted over 195+ projects in rare and orphan disease, including over 50 Advanced Therapeutic Medicinal Product (ATMP) studies.

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. 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.

To address the increasing need for knowledge and expertise in this challenging area, we recently launched the Synteract Rare Disease Institute™ - an innovative engagement program that capitalizes on Synteract’s strengths and skillsets in rare and orphan diseases. The program incorporates lectures, webinars, interactive discussions, videos and industry literature. The Synteract Rare Disease Institute covers a wide variety of topics including patient perspectives, bioethics, regulatory requirements, customer engagement, patient advocacy and more.

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.

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!

Learn more about how we bring your Rare & Orphan Disease clinical trials to life!

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Synteract Rare, Orphan, & Pediatric Diseases Clinical Trial Leadership

Lisa DilworthContact us to find out more about our Pediatric and Rare and Orphan Diseases expertise and experience

Lisa Dilworth
VP, Strategic Development, Rare, Orphan and Pediatric Diseases

Contact us to find out more about our Rare, Orphan, & Pediatric Diseases expertise and experience
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