Rare Diseases: The Path Less Chosen - Boston Children's Hospital | Innovation Summit 2014

Rare Diseases: Lessons from the Path Less Chosen

Rare diseases pose challenges on many levels. By definition, they affect fewer than 200,000 patients; yet, collectively, 1 in 10 Americans has a rare disease. Many are children. Rare diseases can be exceedingly difficult to diagnose. Experts and treatments can be hard to come by. But we have entered a new era of focus on rare diseases. The FDA has launched a rare pediatric disease priority review voucher (PRV) program to address barriers in the approval process.

Moderator: David Meeker, MD, President and CEO, Genzyme

Panelists:

Alan H. Beggs, PhD, Director, The Manton Center, Boston Children’s Hospital
Marlene Haffner, MD, President and CEO, Haffner Associates
Peter L. Saltonstall, President and CEO, National Organization for Rare Disorders (NORD)
William Gahl, MD, Director,MD, Undiagnosed Diseases Program, NIH
Robert (Skip) Nelson, MD, PhD, Deputy Director and Senior Pediatric Ethicist, Office of Pediatric Therapeutics, FDA
Albert Seymour, PhD, SVP, Head of Global Research and Non-Clinical Development, Shire Pharmaceuticals