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 12, 2012

We Need To Apply For A Job

The FDA has a help-wanted sign out in its window.

The FDA seeks 20 disease areas that will serve them well in a set of meetings on patient-focused disease development. There is a list of 39 candidate diseases; however, more are welcome to apply. You may submit your "disease resume" in advance. You may attend a live interview either remotely or in person on October 25.

If you don't apply, you'll never get the job. If you don't apply with a focus on what the FDA seeks, you'll never get the job. It's going to be tough. This is a very tight job market.

Do you wish you had some inside scoop on how to pitch ALS for this job? I think we got some by listening to the FDA's process meeting that was held Wednesday. Here's the scoop as I heard it:

There were a number of patient advocacy organizations at the process meeting. It was difficult to hear them all on the remote connection, but I would estimate 20 were there. There were also two individuals with ALS in the room!  In addition there were around a dozen people on the remote phone line, including at least three individuals with a direct interest in ALS.

There will be 20 meetings over the next five years, each of which will focus on one disease area.  The meetings will be an opportunity for the FDA to learn from patients.  The FDA wants hear what it needs to know about a disease from the patients themselves.  The FDA wants to hear the patients' thoughts on the benefits and limits of therapies.  The FDA wants the patient perspectives on benefits and risks.  Should they approve a drug despite risks?  What outcomes do the patients want?  How do patients think success should be measured? Are investigators today measuring the right things when they see if a drug works (or not)?   The FDA wants to enhance its reviewers' ability to find benefits in therapies.

Here's the rub.  We heard that the FDA knows that ALS is the worst of the worst.  The FDA has all process ready for a quick approval of the first thing that comes along with even a small amount of efficacy.  Clearly the FDA has all hands on deck to move on ALS as quickly as a therapy candidate comes along.  As a result, some folks there may not perceive that ALS has anything to add to the patient-focused disease discussions.  They feel that they have you covered when it comes to patient-focus, ALS patients.

We need to make sure that they know what's in it for the FDA by having ALS be one of the 20 patient-focused meeting diseases.  We are fortunate that in early 2013 there will be a completely separate FDA meeting to talk about ALS issues. That is great and all kinds of stakeholders can get things off their chests and learn from one another; however, that's not a patient-focused drug development discussion.  Asking for both is not overindulgence and there is a lot in it for the FDA.

In the last year we have seen people with ALS shake up some traditional thinking about clinical trials and measures and endpoints.  We have seen patient perceptions about measures and the way trials are trying to evaluate efficacy.  Below are the words of a man with ALS
“I wish clinical evaluations were more quantitative and less subjective… Our disease is not well understood, and it seems that every regular clinic visit offers a great opportunity to learn much more about how the disease progresses, including average and variations of measurable changes in strength measurements related to arms, legs, neck and even tongue. I suspect this data could then be used to add more precision to evaluations between treatment and placebo (control) groups during clinical studies, rather than rely on much less precise and much less stratified data from the use of (functional rating) scores and survival.”
Those comments fit the goals of the patient-focused drug development meetings like a glove. 

Do you remember last year when a man with ALS purchased a bunch of strength-grip testers on ebay for his friends with ALS? He thought that was a pertinent measure.  People with ALS have ideas about measures and endpoints and the risks that they are more than willing to take in order to have a shot at life.  People with ALS are motivated to a higher purpose for their limited days and surely their voices would make the FDA reviewers better.

We need to apply for this job.  People with ALS can deliver value for the FDA.  We need to make a good case.  These meetings are about making the FDA better.  We need to show them a disease population that can help.

Nobody ever earned a job offer with a me-oriented application.  Please apply for this job by showing the FDA what's in it for them.  People with ALS will add value to their processes, and perhaps in doing so will spark some new ideas that will result in better therapies for a lot of diseases.

Please submit comments at --

Please spread the word.  Persuasive grass-roots comments will make a difference.
We all need to apply for this job on behalf of all with ALS.  Please do it now.

Then plan to attend and participate in the meeting on October 25, either in person or remotely via the internet.

Thank you.

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