Thank you all for your response to my recent request to send letters to the FDA on the Patient-Focused Drug Development initiative last week. Quite a number of you (dozens) responded on behalf of rare disease in general and many on behalf of MLD or lysosomal disease specifically.
As I mentioned in my prior post, we will always be partial to MLD because of our roots – but it’s really the more general rare disease issues that are important to me in most of these policy discussions. Those that know me well know I have a real passion for the rare disease community.
I wanted to address one issue that has come up a couple of times including a recent blog from NORD. They have twice described the engagement of rare disease advocates in the process as creating a “firestorm” at the FDA.