Find Uncommon Success in Rare Disease Studies

In Clinical Development by Freya Smale

Planning, readiness, and high-quality data are vital to any clinical trial, but the stakes are much higher in studies involving rare and ultra-rare diseases. Because they’re few in number, study participants are like gold dust, and every data point counts.

That’s how three experts from Premier Research and Alexion Pharmaceuticals described the rare disease environment in our latest webinar, “Rare Disease Studies: How to Run Successful Programs,” available now for download.

Rare disease trials are uniquely challenging, but recent successes are cause for encouragement. 2014 saw a 53 percent increase in orphan drug approvals over the previous year, with 49 new drugs OK’d by the FDA. Industrywide, designation requests jumped by one-third.

In this webinar, you’ll hear from Angi Robinson, Executive Director, Rare Disease and Pediatrics for Premier Research; Patricia Nowowieski, Head of Global Clinical Operations – Cambridge for Alexion Pharmaceuticals; and Mallory Bissett, Associate Director of Global Clinical Operations for Alexion Pharmaceuticals.

Among the topics:

Getting it right the first time. The number of possible participants in rare disease trials is inherently small, so studies can’t be duplicated. “More is more” when it comes to vetting participants, scrutinizing every detail, and building in quality through rigorous attention to inspection readiness.

Managing relationships. Engaging investigators is critically important, and so is fostering close collaboration among the sponsor, the trial sites, and the CRO.

Involving patient groups and social media. Early and frequent involvement of patient advocacy groups is vital when working with small populations, and social media plays an expanding role as well. Study-specific Facebook pages are becoming more common, and so is promotion of disease awareness via YouTube.

We’ve learned a lot about rare diseases over the course of more than 100 studies and want to share what we know. In addition to checking out the resource center, see us at the World Orphan Drug Congress, April 23-24 in Washington, DC. Premier Research is a platinum sponsor and will have a speaker and two roundtable presentations.

For more information on Premier Research, visit or contact us at

Premier Research is joining us at World Orphan Drug Congress USA 2015. It is not too late to meet them there. Simply visit the website by clicking on the image below.