Earlier this week, I attended a listening session with the Commissioner of the FDA, Dr. Robert M. Califf. As a new employee in the NORD Policy Office, this was my first meeting at the FDA, and as such I was… Read More
The purpose of this Patient-Focused Drug Development meeting is to obtain input on the symptoms and other impacts that matte most to patients with AATD. FDA also intends to seek patients’ perspectives on current approaches to treating this disorder. FDA expects that this information will come directly from patients, caregivers,… Read More
Today, the FDA released a blog post about a new tool for drug developers. The FDA writes, “With the vast majority of rare diseases still without FDA-approved treatments, we have recently released a new resource for drug developers — draft guidance document — designed to help them… Read More
Reprinted with permission from FDA Matters: By Steven Grossman
The Orphan Drug Act (ODA) turned 30 this month, demonstrating that good laws really can have an enduring impact. Amidst the celebrations, a reporter asked me a provocative question: Can we afford more orphan drugs costing hundreds of… Read More