By definition, a disease is considered rare if it affects fewer than 200,000 people in the United States. However, an estimated one in ten Americans has a rare disease and about one third of all new drugs approved by FDA are now for rare diseases. In fact, in 2017 the FDA approved a record 80 new treatments for are diseases.
Whenever an FDA advisory committee is convened as part of the approval process, the stakes are high and there can be communication challenges. However, for applicants preparing for an advisory committee that is for a rare disease, the challenges are unique.
Read the full article written by Neelu Agrawal, expert in high stake meeting preparations, including FDA Advisory Committees, Oral Explanations, and Scientific Advisory Groups, to learn about some of those challenges, along with key factors for success.
By Neelu Agrawal – Principal, NDA Group/PharmApprove