Dr. Ivan O. Rosas, M.D.
Assistant Professor of Medicine, Harvard Medical School
Director, Interstitial Lung Disease Program, Brigham and Women's Hospital
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
As a centre of competence for this highly specialised field of medical care, the ZKRD organises the search for donors at a national and international level in order to assure that this complex process operates as quickly, efficiently, transparently and economically as possible. It coordinates the search process and checks all related requests and results for completeness and plausibility.
Its service framework includes coordinating all steps from the initial request to invoicing the cost units. Additionally, the ZKRD fulfils a wide array of consulting and organisational functions.
Along with the U.S. National Marrow Donor Program (NMDP), the ZKRD holds a leading position at an international level. With about 5.5 million donors, it is the second largest registry. If ranking is based on the number of donors identified annually for transplantation, then the ZKRD is one of the most productive registries worldwide.