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.
I attended the meeting at the FDA, testified orally, and submitted written comments. NORD attended but did not testify orally – their comments were submitted only in writing. Personally, I think they missed a great opportunity to speak representing and addressing the rare disease community – and I passed this opinion on to them. NORD’s written comments were comprehensive and not disease specific – it’s not reasonable for them to pick specific diseases.
I testified asking for some careful thought as to how the 20 diseases are selected … and so did most of the others that gave oral testimony. Of course most thought their own disease was a good vehicle for addressing more general issues but those in the rare disease community nearly all spoke about the share challenges of rare disease research, trials and approvals.
I respectfully disagree about the “firestorm” label NORD is applying to the rare disease community. The oral comments were very reasonable with some specific disease lobbying, but I felt the overall attitude was quite respectful. There was no bickering or hostility – just a good group of advocates sharing perspective. We all know that every one of the 7,000+ rare disease cannot fit into a 20 meeting schedule and were trying to make sure that above all, rare disease was represented to address the unique issues of research with very small disease knowledge and small patient communities.
Please do not mistake passion as a firestorm. We are working together and the FDA is listening. Let’s hope their listening turns to good action and NORD’s voice becomes more positive.