Written by Lisa Sencen on November 19, 2018
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:
- Increase understanding of the expanded access program
- Identify and discuss strengths, challenges… Read More
Written by Lisa Sencen on August 9, 2018
Dear Reader,
I want to invite you to join me and 700 others from the rare disease community at our annual Summit. This year, NORD’s 2018 Rare Diseases and Orphan Products Breakthrough Summit will take place October 15-16 in Washington, D.C.
We expect this year’s Summit to again be the… Read More
Written by Lisa Sencen on July 11, 2018
Dr. Scott Gottlieb was sworn in as the 23rd Commissioner of Food and Drug Administration in 2017, and provided a keynote speech at the NORD Rare Summit shortly thereafter. 2018 marks his second year at the Summit, and we look forward to… Read More
Written by Mary Dunkle on August 31, 2017
In an action the rare disease community has been eagerly awaiting, the Food and Drug Administration (FDA) has announced the first-ever approval of a gene therapy in the U.S. The product is Kymriah, and it is approved to treat certain children and young adults who have a cancer of blood… Read More
Written by Lisa Sencen on August 15, 2017
It has been an interesting year for healthcare reform and the Orphan Drug Act, and as I’m sure you are aware; the conversation isn’t over yet. Major changes to healthcare and tax reform continue to be discussed and that is why it is so important that now, more than ever, rare… Read More
