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.

Thursday, February 25, 2016

They Were Only Separated By 1.8 Miles, Yet They Were A World Apart Today

Today there were simultaneous events of huge importance to those trying to speed up access to therapies for ALS.

The Senate Hearing on Connecting Patients to New and Potential Life-Saving Treatments started at the exact moment that the White House Precision Medicine Summit convened.

Boy, were they ever worlds apart.

I had great expectations for the Senate Hearing.  I know a number of people took time to write informed letters to that Committee on better paths to FDA approval and the concept of risk versus benefit with ALS.

What we got was an infomercial for Right to Try Laws, and not a very informative one at that.  You see, the FDA wasn't even there.  If they weren't invited, then shame on the Committee.  If they didn't show up, then shame on the FDA.  Either way, it was disrespectful to the many who took time to submit testimony.  It was maddening enough that the February 25, 2013, FDA ALS Hearing turned out to be nothing more than a dog and pony show.  Three years later we only got the dog.  No pony.  What a disappointment on one end of Pennsylvania Avenue.  We honestly have had far superior discourse on Right to Try and other access mechanisms on the Diane Rehm Show.  She remembers to include the FDA in the discussion.

On the other end of Pennsylvania Avenue we had substance and commanding arguments for change.  Many familiar faces from Medicine X spoke to their rare disease experiences and the importance of information and precision medicine to address life-threatening conditions.  It's always a treat to hear from Dr. Collins. Then when the President of the United States arrived, he didn't step up to the podium.  He sat in as a contributor on a panel discussion, and he sure hit nails on the head when he chimed in.  The information silos must go in healthcare and in research.  The regulatory processes that gave us access to penicillin don't work for today's methods and needs. People should own their own data and be able to share them, and technology providers should enable that.  I only wish that more ALS researchers were paying attention.

It was a bad day at one end of Pennsylvania Avenue and a great day at the other.  I'm thankful for those who led and contributed to the #precisionmedicine summit!  And today made me extra proud to have been a MedX e-patient scholar.  Medicine X rocked the White House today.


1 comment:

  1. One reason i didn't write to Sen. Johnson was my utter disappointment with 2013 fda meeting. My wife and i made huge effort to get there, only to have two commissioners sleep through my presentation. Why waste my time with Johnson?

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