Tuesday, April 2, 2013 - Tuesday, April 2, 2013
8:00 a.m. - 10:00 a.m.
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Monday, April 1, 2013 12:00 p.m.
Where BIOCOM Boardroom
4510 Executive Drive, Plaza 7
San Diego, California 92121
Please note that this event is at full capacity and we are not accepting registrations.
The waitlist is now full.
Driving Orphan Drug Development Success
Rare and genetic diseases affect one in 10 Americans, 30 million people in the United States, and 300 million people globally. Over 6,800 distinct rare diseases exist and approximately 80 percent are caused by faulty genes. The National Institutes of Health estimates that 50% of people affected by rare diseases are children, making rare diseases the most deadly and debilitating diseases for children worldwide. Understanding the regulatory pathway and unique statistical approaches for orphan products are critical to a well-designed drug development program.
Please join us for this informative presentation, which will be followed by Q&A
Tim McDaniel, Ph.D.
Director of Scientific Research, Illumina, Inc.
"A son's story of personal genome sequencing for a rare cancer"
Tim McDaniel will describe a genome project he led for his mother, who suffered from a rare cancer. This very personal story of personalized medicine will be told from his perspective of scientist, patient advocate, and son.
Tim Coté, MD, MPH, Coté Orphan Consulting
Former Director of the FDA Office of Orphan Products Development and CMO of the National Organization for Rare Disorders
"What's new in the orphanage - FDASIA and beyond"
Tim Coté will provide an overview of the orphan drug development landscape. This important presentation will provide insights on the effects of FDASIA on orphan products, including a look into the future of orphan product development.
Elizabeth Ludington, Ph.D.
Vice President of Biometrics, Agility Clinical, Inc.
"Important considerations for successful small clinical trials"
Elizabeth Ludington will describe important elements related to the design, conduct, and analysis of small clinical trials. She will provide specific challenges, potential solutions, and success stories from the industry.