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, April 16, 2021

Were They Just Doodling?

 For over a decade I and others (many of whom have died from ALS) have gone to FDA meetings to give  thoughts on the need for change for ALS.  There have been Patient Focused Drug Development meetings.  There has been an all-day FDA hearing on ALS.  There have been public hearings on innovative trial designs.  There was an all-day ALSA workshop  so that the FDA could hear our thoughts.  I was not a paid invitee.  This was a volunteer effort that I and others thought was important and would make a difference.  

For over 10 years we have worked with pharmaceutical and biopharmaceutical companies that have asked for volunteer patient and caregiver voices.  I have spent countless hours in meetings that many of us  thought would make a difference.

Over the same decade, we have talked to ALS researchers about how we can improve clinical research from the patients' and caregivers' perspectives.  Again, we thought we could add a valuable perspective and make a difference.

I and others spend long hours doing our homework so that we can be informed contributors.  We learn regulatory and industry perspectives that need to be considered.  We know that there were once good reasons for rules that we would like to see changed.  We have been driven to make Expanded Access and faster determination of scientific truth realities.

Today I think we've been had.  Was anyone even listening?  While we spoke, were they just doodling in those meetings, humoring us but not really planning to change a thing? 


Events of the past few weeks have shown that absolutely nothing has changed.

We've been had.


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