Ongoing Challenges and Outlook for Rare Disease Drug Development

There are an estimated 7,000 rare diseases that affect 25 to 30 million people in the United States, more than half of whom are children, with treatments available approximately 5% of these conditions. Many of these diseases are life-threatening, genetic in origin, and only recently are new treatments bringing the prospect of a better life to some patients. This article dives deep into the current situation and future outlook of the world of rare diseases, including scientific innovation requirements, regulatory flexibility and pricing negotiations, and the growth and success of rare disease drug development and commercial launches.