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, April 28, 2018

Do You Really Want To Be A Silent Partner In This?

Words of concern have clouded the CDC ALS Registry for a decade -- disappointing, incomplete, expensive, opaque.  The concerns have never been enough to be faced seriously by those who set funding priorities. The project gets a generous annual autopay of $10,000,000 that is a recurring top ALS Association priority. Hundreds of sincere advocates just do what they're told every year and support what must be a noble goal.

The project has now crossed a line that demands action. The goal may be noble, but the deliverable is doing harm. Yes, it is doing harm.

In October a report was quietly published that confirmed and quantified the completeness problem, and it showed data that should concern (if not outrage) every ALS advocate. Please read it.  The CDC Registry missed almost as many people with ALS as it found, and the people it missed don't exactly look like the people it counted.

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5815913/



Bias (implicit or explicit) is a big issue in clinical research, and this Registry is a source of bias.

CDC annual reports on ALS assert general disease demographics based on the people with ALS they found, not on the missing 43%.  Think about that.  In a poorly diagnosed disease, this can even be self-fulfilling.

CDC clinical research notification goes to the people with ALS they found, not the overlooked 43%.  So much for diversity in clinical trials.

Do you want to be a party to this?  Will we all look back in a decade and cringe at what this generous funding was enabling?

First, do no harm.  This Registry is doing harm.

Do you want to be a silent partner in this?

Please speak up.  This issue matters.  The 43% matter.



Sunday, April 8, 2018

"Y'all Take Care Of It Yourselves"

"We don't have anything to say right now."


That's the mystifying strategy that a gaggle of large ALS organizations have taken during the most important ALS public policy crisis in decades.

We're in the final week of the comments period on the FDA ALS Draft Guidance.  So far organizations have gone into hiding and engaged in an odd abdication of leadership, telling individuals that it's up to us. They are urging us to submit comments but have not issued any comments themselves.

Keep in mind that this guidance will directly affect clinical trial designs moving forward, and the draft guidance we got does not encourage innovation in trial designs.  If made final, this guidance will in no way speed up drug development or approvals.  

But our largest ALS patient advocacy and research organizations have no official comments.

They turn to us individuals and say, "Y'all take care of it yourselves."

Mystifying.

Disappointing.

Infuriating.

Revealing.