Strategically Managing the Logistics and Costs of Rare Disease Clinical Trials

In Clinical Development by Freya Smale

This guest blog post was provided by Premier Research. In the final part of our four-part panel discussion at the World Orphan Drug Congress in Washington, DC: “Making Rare Disease Clinical Trials Feasible,” the panel wraps up their discussion by talking the logistics and costs of running clinical trials in rare diseases. Frank Armstrong (Medical Advisor) starts off the discussion with some interesting statistics. The average cost of a patient in a rare disease trial was about 25 times the average cost of a patient in a chronic trial. Also highlighted from Premier Research’s survey of groups involved in clinical trials in rare diseases, was that about 75% of rare disease trials are outsourced by a sponsor to a CRO. Angi Robinson (Scientific Account Leader at Premier Research) cautions that when preparing trial implementation to be mindful that the number of patients does not equal the amount that you’re going to spend on a trial. It’s very important to take advantage of your preproduction phase, as that is an opportunity to be strategic about managing costs. John Orloff (Chief Medical Officer at Novartis) added that flexibility and simplicity are vital in managing costs. There are many unanticipated costs when going into a rare disease trial and simplifying your endpoints and measurements down to what is absolutely needed can go a long way in bringing down costs. This concludes our coverage of our panel at this year’s World Orphan Drug Congress in Washington. If you missed any of the previous posts you can read about them here: Part One, Part Two, Part Three. We hope you found this video, and the whole series, enlightening. We’re pleased to be a sponsor of the 4th Annual World Orphan Drug Congress , November 14-15, 2013. We’re currently developing our panel discussion, and would love to hear from you about what you’d like to see us cover with our Geneva panel. Let us know in the comments below, or send us an email at

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