There were a number of people and organizations who signed up to make public comments. Because of the number of people asking to comment, each was limited to two minutes. It was mostly organizations. On the speaker list there were four individuals who would speak to ALS (three of whom were patients) plus two organizations -- MDA and ALSA. All were interspersed throughout the long list of commenters. That was good.
After some brief FDA presentations the leadoff hitter was the veteran with ALS who has taken this cause to the patients. He is not able to travel and the FDA read his comment. It was a powerful statement. His closing words were --
I provide that short summary of my ALS story to show the FDA that they may still have something to learn directly from ALS patients. ALS is obviously a complex disease with the potential for many secondary issues like the one I described. The only way the FDA is going really understand the entire ALS disease process, and the unmet medical need is by talking to ALS patients and caregivers directly. The only way the FDA will really understand the risks ALS patients are willing to accept in drug development is by talking directly to ALS patients. I encourage the FDA to change its position, add ALS to the disease list, and make ALS the stand alone topic of the first PDUFA V patient meeting.Solid base hit. One man on base.
After a few other speakers my name came up on the list. I hope that I did no harm and set things up a little for the next ALS patients to speak. My words are in the prior blog post.
A few more speakers talked of their diseases and then came another man with ALS. He made another strong showing. The patients weren't working from an organization's agenda. Each spoke independently and uniquely. The didn't always agree on details, but that is actually a good sign that they'll be productive participants in patient-focused drug development discussions. He gave a strong message that this is a moment that must be seized. http://tmurph58.tumblr.com/post/34364683190/my-statements-at-yesterdays-fda-public-meeting
Another solid base hit. Runners on first and second.
The third patient speaker made quite an impression. He told us that he would give anything to have some of the diseases being discussed rather than ALS. He was motivated and reminded the FDA that nobody was paying him to be there (in contrast to the many paid advocates there). He came across as a man who could add much clarity and common sense to patient-focused drug development discussions.
Another hit. Bases are loaded.
It was time for the two organizations on the agenda to hit their runners home. The ALS speakers had made a strong showing. Unfortunately neither organization adapted its comments to advance what ALS patients had just accomplished.
MDA suggested that all neuromuscular diseases be grouped into one "disease area" for the patient-focused discussions. It was like a parent who doesn't want to pick a favorite child, perhaps. It really didn't serve any of the diseases well in my opinion. It certainly set back the concept that our ALS patients had set up so well.
Strike out. Bases are still loaded.
It felt like ALSA came to a baseball game with a golf club. They mentioned that they have been working with the FDA for two years. They mentioned that ALS incidence is higher in veterans. They mentioned that the FDA's announcement of disease areas was not clear to patients and has caused some "disease wars." They asked for a Part 15 Hearing for ALS for 2013. That's an existing priority for ALSA and a noble cause indeed. Unfortunately that hearing has nothing to do with the Patient-Focused Drug Development meetings and disease areas. A 200 yard drive down the middle, but it was the wrong game. Runners were not advanced.
Two outs. Bases are still loaded.
It's up to us all to bring the three patients on base home. Please continue to submit comments at
There are other posts on this blog to help give you background. We must make a good case for people with ALS to contribute as a disease area in the Patient-Focused Drug Development meetings. We need more solid base hits to bring our base runners home.
Thanks to those who have submitted. Please take another at-bat and submit again. If you've not submitted comments yet, take a few practice swings, and batter up. Please don't leave three all-stars stranded on base. We have until November 1.