Navigating the Regulatory Landscape

In Regulation & Government by Freya SmaleLeave a Comment

This guest blog post was provided by Premier Research.

In part two (click here to read about part one) of our four-part panel discussion at the World Orphan Drug Congress in Washington, DC: “Making Rare Disease Clinical Trials Feasible,” the panel turned to the regulatory landscape for those working with rare diseases.

Frank Armstrong (Medical Advisor, Premier Research) kicked off the discussion by highlighting a statistic from a Premier Research survey of groups involved in clinical trials in rare diseases, finding that that 43% of respondents found that securing regulatory approval for trial design was difficult or extremely challenging.

John Orloff (Chief Medical Officer at Novartis) talked about the importance of the Orphan Drug Act in minimizing surprises when designing trials. Angi Robinson (Scientific Account Leader at Premier Research) stressed the importance of being very diligent about the type of data you collect and how clean it needs to be when preparing for a trial design.

Phil Vickers (Senior Vice President and Head of Research and Development, Shire HGT) focused on the benefit to risk aspect when approaching the regulatory landscape. He stressed that no one wants to develop unsafe drugs, but the benefit to risk calculations have to be flexible in the case of rare diseases and that it needs to part of an open discussion with regulators and patients very early, and throughout every step of the process when developing treatment.

We’ll be covering more about what the panel had to say in future posts. In the meantime, we hope you find 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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