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.
To qualify for Miracle Flights' assistance to fly to non-research medical appointments, families must provide a letter from both a referring and an accepting physician and have a low to moderate income. This program is for children less than 18 years of age. They are allowed to be accompanied by two parents or the child's eligible caregiver. The completed request must be made no more than 60-90 days and no later than 14 days before the appointment. International patients can also qualify to be flown into the U.S. For more information, call 800-359-1711 or click the website link.
Julia's Wings Foundation is a non-profit organization, certified 501(c)(3), that provides financial assistance to families of children with the life threatening hematological diseases, aplastic anemia, MDS and PNH. The fund has a budget of $75,000 per calendar year for the purpose of providing financial support to these families to cover costs of medicines not covered by insurance, travel, lodging, and other living expenses for qualified participants. If you are a parent or guardian of a child with aplastic anemia and have been forced to incur significant financial burdens in the process of getting your child the medical treatment they require, you may be eligible to receive financial assistance from the Julia’s Wings Foundation. We offer short-term financial assistance to families of children with aplastic anemia, particularly those in the midst of treatment or prolonged hospitalizations. See above link for more information and click here for the application process.