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.

Saturday, May 16, 2015

Just Read The Transcript

On February 25, 2013, dozens of people with ALS, caregivers, scientists, drug developers, ALS
organization employees, and clinicians made a trip (many at their own expense and with a great deal of difficulty) to testify to the FDA.

They gave a gold mine of ideas for speeding up drug development and approval.  It's a big gold mine because people testified all day in six-minute segments.

They talked about risk, biomarkers and endpoints, trial design, FDA processes, natural history, controls, and on and on.

Today we hear that the FDA guidance document that was indicated at that meeting is only now being started and that dozens of people with ALS, caregivers, scientists, drug developers, ALS organization employees, and clinicians will soon be convened to give their input.

Ok, everybody.  How about just reading the transcript from that FDA hearing?  You have the materials to start a draft FDA guidance document right under your noses.  Let's get moving.  Spending buckets of money on unnecessary meetings isn't a sign of an understanding of urgency.  Nearly 300,000 more people have died from ALS since that hearing.  Urgency?

You can watch.
http://www.fda.gov/Drugs/NewsEvents/ucm339833.htm

You can read the whole thing.
http://www.regulations.gov/#!documentDetail;D=FDA-2013-N-0035-0271

And you can read the FDA's summary and reaction.
http://www.regulations.gov/#!documentDetail;D=FDA-2013-N-0035-0272

As an example, one of the ideas presented that day is that people with ALS have a right to know who is doing what during the clinical trial and FDA back-and-forth in the approval process.  Are we waiting for statisticians?  Are we waiting for an FDA decision?  Are we waiting for more data to be submitted?  What's going on?  Are we waiting for somebody on vacation?  Think about how much more clearly the Accelerated Approval application for GM604 might be perceived if we simply had this kind of transparency policy.  Any successful modern business has project teams that know what's going on in the scrum today, yet the veil of secrecy continues to obstruct the view of the most important people in the drug development and approval process -- those with ALS and their caregivers.

And there are hundreds of more examples of ideas that can have a direct impact on speeding up drug development and approval.

Just read the transcript.


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