Genpharm video

Approaches to Clinical Trial Design

In Clinical Development by Freya Smale

This guest blog post was provided by Premier Research. In April, as a part of the World Orphan Drug Congress in Washington DC, we hosted a four-part panel discussion: “Making Rare Disease Clinical Trials Feasible.” Phil Vickers (Senior Vice President and Head of Research and Development, Shire HGT) and John Orloff (Chief Medical Officer at Novartis) sat down with our own Frank Armstrong (Medical Advisor) and Angi Robinson (Scientific Account Leader) to discuss the ins and outs of the rare disease clinical trials. In part one of this four-part video series, “Approaches to Clinical Trial Design,” the panel discusses the challenges of designing clinical trials and what can be done to improve the process. Shire’s Phil Vickers addresses Metachromatic Leukodystrophy (MLD) and the challenges associated with developing trials for a rare disease with so much heterogeneity and the importance of accelerated approval using biomarkers. In particular, he also shares some ethical questions we face when deciding whether or not to run a placebo trial when running trials on children, especially when the disease can be so destructive to children. Furthermore, with such a small rare disease patient pool, and every bit of data is useful, and the practicality of placebo trials also comes into question. John Orloff of Novartis approaches clinical trial design and the importance of using longitudinal measurements to take advantage of as much data as possible and maximize the amount useable information. 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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