Oct. 5, 2016
Posted by Jennifer Huron
Washington, D.C., October 5, 2016 – NORD, the leading independent nonprofit organization representing the 30 million Americans with rare diseases, today announced the three keynote speakers for its 2016 Rare Diseases & Orphan Products Breakthrough Summit to be held Oct. 17-18 in Arlington, VA.
This year’s event will feature FDA Commissioner, Dr. Robert Califf; parent advocate and co-founder of CureBatten.org, Kristen Gray; and senior policy analyst at Prevision Policy, Kate Rawson. For more information and to register, visit https://rarediseases.org/summit-overview/.
Robert Califf, Commissioner of the U.S. Food and Drug Administration (FDA), will present a day one keynote address that will focus on FDA priorities and how FDA is preparing for the next generation of medical advances. One-third of the new drugs approved by FDA each year are orphan drugs to treat rare diseases.
Kristen Gray is the co-founder of CureBatten.org, also known as the Charlotte and Gwenyth Gray Foundation, a NORD member organization. In 2015, she and her husband, Hollywood producer Gordon Gray (The Rookie, Miracle), learned that their two young children had Batten disease, a genetic, degenerative and fatal brain disorder that at the time had no treatment or cure. One year later, their eldest daughter, 5-year-old Charlotte, became the first patient in the world to enroll in a clinical trial for an experimental gene therapy for Batten that could save her life and the lives of many others. In the opening keynote of the Summit, Kristen will discuss her family’s journey from first noticing subtle symptoms, to pursuing a diagnosis, seeking testing for younger daughter, finding researchers, raising funds, working with FDA, finally obtaining gene therapy treatment in an investigational setting, and their unwillingness to accept “there is no cure” as an answer for their daughters and other children with rare diseases.
Kate Rawson is a Senior Editor at Prevision Policy LLC, a firm that provides health care policy analysis. In her day two keynote, Kate will present on national election implications for the rare disease community. She has nearly 20 years of experience covering FDA-regulated healthcare industries, most recently pharmaceutical and biotechnology. She is a senior editor for The RPM Report and writes frequently about health care policy, FDA regulation, and reimbursement coverage. Rawson also co-founded the FDA/Centers for Medicare and Medicaid Services Summit for Biopharmaceutical Executives, an annual conference in Washington. Previously, she was a senior editor for “The Pink Sheet,” published by FDC Reports.
The keynote speakers join a roster of experts that include 20 top leaders from FDA, including Director of the Center for Drug Evaluation Janet Woodcock and Director of the Center for Biologics Evaluation Peter Marks, the National Institutes of Health (NIH), and a poster session that will feature original research, innovations and advancements under the theme of “Life-Transforming Treatments.” A few highlight sessions include:
- Telemedicine and Rare Diseases
- Advancing Pediatric Research and Treatment
- The Challenge of Drug Shortages for the Rare Disease Patient
- Potential Advances through Genetic Innovation
- The Challenge of Orphan Drug Pricing
- Externally-Led Patient Focused Drug Development Meetings
- Collaborating across Borders and Examining Rare Diseases as a Global Health Challenge
- Predicting the 2017 Pipeline of Orphan Product Development
- Strategies to Address Patient Challenges
For more than 30 years, NORD, a 501(c)(3) independent charity, has been the nation’s voice on rare diseases and at the forefront of patient advocacy. NORD’s annual Breakthrough Summit is the largest multi-stakeholder gathering of the rare disease community.
Media Contact: Members of the press are welcome to attend and must register in advance by contacting Jennifer Huron at [email protected] or 203.304.7258.
Learn about NORD’s other events here.