We’re delighted to announce that Acting FDA Commissioner, Norman E. “Ned” Sharpless will deliver the keynote address at this year’s Rare Diseases & Orphan Products Breakthrough Summit. Sharpless, a physician and scientist, became Acting Commissioner of FDA earlier this year and previously served… Read More
Washington, DC, July 19, 2019—The National Organization for Rare Disorders (NORD)®, the leading independent nonprofit organization representing the over 25 million Americans with rare diseases, announces the appointment of four new members to its Board of Directors, effective immediately.
Joining the Board of… Read More
Real-world evidence and compelling case studies bring to life the challenges facing the estimated 30 million Americans fighting to live better with rare disorders
Washington, DC, June 3, 2019—The National Organization for Rare Disorders® (NORD), the leading independent nonprofit… Read More
Building on the Reagan-Udall Foundation for the FDA’s work with FDA and other stakeholders to develop the Expanded Access Navigator, the Reagan-Udall Foundation is convening stakeholders from government, industry, academia and patient groups to discuss process issues that include:
Washington, D.C., October 18, 2018— Despite a record-breaking number of new approvals, orphan drugs remain a nominal part of overall drug spending, accounting for only 9.6% of total sales in the U.S. in 2017, according to a new study released today by the IQVIA Institute. The study… Read More
