When the Patient-Focused Drug Development (PFDD) Initiative was first announced as part of the 2012 PDUFA reauthorization, many patient organizations expressed concern that the initiative’s public meetings would focus on just 20 groups of diseases. However, FDA has now provided a pathway for extending the model to many other diseases.
In a blog published this week, Theresa Mullin, PhD, writes that FDA has found the PFDD public meetings to be very helpful, providing a first-hand understanding of the needs and wishes of patients. As a result, FDA is now inviting patients with diseases not covered by the original public meetings to organize their own, following the PFDD model.
It’s a great opportunity for patient organizations representing people with rare diseases to share their hopes, challenges and concerns directly with FDA. Interested groups can find more information about this on the FDA website and in the Federal Register posting.