This guest blog post was provided by Premier Research.
Last November, we were pleased to host a panel discussion at World Orphan Drug Congress Europe in November 2013, where four leading specialists shared practical examples of their hands-on experience designing feasible studies.
Study Design Considerations is the first of five video segments that provide highlights from that discussion. In it, our panelists, including senior executives from Aegerion and Lumena Pharmaceuticals, discuss what goes into study design. These considerations include:
- Why you need to talk to patients and their families early on, and what you need to learn from them.
- How to select end points that satisfy physicians, patients and regulators – all of whom may have different interests.
- Why you may have to throw out the norms – and what to do instead.
- How to share the challenges with regulators early on, so they become part of the solution.
Our panel includes unique perspectives from four leading specialists:
Dr. Colin Hayward, Vice President, Medical Affairs, Premier Research
Dr. Susan Tansey, Medical Director, Pediatrics, Premier Research
Dr. Massimo Boriero, President for Europe Middle East and Africa, Aegerion Pharmaceuticals
Dr. Ciara Kennedy, Chief Operating Officer, Lumena Pharmaceuticals
And, remember, this is just the first of several WODC videos, so make sure to check back here soon for part 2, How to Successfully Engage Regulators.