Tag: FDA


4 Historical Orphan Drug Development Barriers, Have We Broken Through?

Both understanding the role of patient advocacy groups in building better clinical trials and improving the research process mean first taking a look at the history of orphan drug development in the United States and Europe. An “orphan drug” is a drug that treats a condition that fewer than 200,000 people have. Because rare diseases...

Medical and Regulatory Affairs

Navigating Regulatory Pathways to Address Unmet Medical Needs

It would be hard to overstate the need for new therapies that target unmet medical needs, especially in rare disease. After all, there are only about 400 approved “orphan drugs,” meaning that 95 percent of rare diseases lack a single approved treatment. Now the good news: U.S. and European regulators, recognizing the size and severity...


NORD Summit, a Rare Disease Regulatory Wonderland

Rare disease is one of our main areas of focus here at Premier, so you’ll often see my colleagues and me talking about rare, through our webinars and white papers, and basically, almost any phone conversation with us. Last week, I attended the NORD Summit in Arlington, VA, and as usual, I found myself inspired...