Part of a continuing series of videotaped sessions at Boston Children’s Hospital’s recent Global Pediatric Innovation Summit + Awards 2014.
Rare diseases offer a lot of opportunity for gene discovery, but getting a drug to market presents many challenges, and costs per patient are high. This 50-minute session explored this complicated landscape from multiple angles. The panelists:
- David Meeker, MD, President and CEO, Genzyme (Moderator)
- Alan H. Beggs, PhD, Director, Manton Center for Orphan Disease Research, 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, Undiagnosed Diseases Program, NIH
- Robert (Skip) Nelson, MD, PhD, Deputy Director and Senior Pediatric Ethicist, Office of Pediatric Therapeutics, FDA
- Albert Seymour, PhD, Senior VP, Head of Global Research and Non-Clinical Development, Shire Pharmaceuticals
Read our day-of coverage of this session along with full coverage of the Summit. We will continue to post more sessions from the Pediatric Innovation Summit over the next two weeks (sessions are also available on YouTube).