ALS Advocacy

ALS Advocacy
Lou Gehrig's Disease - Motor Neuron Disease - Amyotrophic Lateral Sclerosis
Thought it had been cured by now? Still no known cause. Still no cure. Still quickly fatal. Still outrageous.

Friday, October 26, 2012

Runners Left on Base. We Need To Drive Them Home!

Yesterday at the FDA meeting for Patient-Focused Drug Development disease areas, three gentlemen with ALS had solid base hits.  Now it's up to the rest of us to take some swings to bring them home.

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.

Batter Up

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
http://www.regulations.gov/#!documentDetail;D=FDA-2012-N-0967-0001

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. 

5 comments:

  1. You hit it out of the park with this one. People living every minute of the day understand the urgency, and the need. Who do the big groups advocate for? It doesn't seem like it the people with ALS.

    I hope everyone who reads this blog, goes the FDA website and posts a comment requesting that ALS be one of the 20 diseases for patient focused drug development. All the Allstars who live with ALS deserve it.

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  2. Meeting was a perfect example of why patients speak best for patients. Organizations get distracted by other agendas when they have the microphone.

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  3. No victory lap yet. Mission not accomplished yet. Please send more messages to the FDA. Please tell them that people with ALS at the table will help them. ALS must be one of the 20 disease areas. People with ALS have too much to offer to miss this opportunity!!!

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  4. A thought I had at the big FDA meeting with lots of disease advocacy organizations present -- If people had a chance to walk in each other's shoes, nobody would pick the ALS shoes. Ever.

    Perhaps that's a good reason why the unique perspective of people with ALS will be of great value to the FDA and they figure out patient-focused drug development.

    Nah, nobody would touch those ALS shoes.

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  5. Wow. The person that said I would rather have any one of those other diseases. What a powerful statement that is.
    We have so much to offer. ALS is a MND, and I think that there are many forms(not yet named or discovered). What this means is, I feel that our patient centered help could be much more beneficial to a wide variety of patients with MND.
    I have commented before and will do so again. Thanks to all for their tireless efforts in this matter !

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