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.

Monday, July 20, 2015

"The Single Largest Research Project Ever Created"

Those are the words of the ALS Association regarding the CDC's ATSDR ALS Registry.  We believe in its largeness in terms of expenditure.  There are some serious questions about the largeness of the results.

This week the Registry's annual meeting is being held in Atlanta.

This twitter exchange is telling --

And finally after various advocates pressed the issue, the CDC reneged and the meeting will be webcast --

Starting Wednesday, July 22, 8:30 am EDT

I hope that people will listen in. And I hope that we never again have to ask to have any Registry meeting webcast.   There is a lot that needs to be discussed and understood, and sunshine only helps.

I hope that the following questions will be among those answered:

- As you introduce people at the meeting, will you please ask for any business relationships that the participants have with one another?  And who nominated each to participate in the meeting?  And who is paying each's expenses?

- What is the project total expenditure to-date?

- When will we have some completeness studies of the national Registry versus metro studies so that we get some insight in how many people with ALS are being missed by the national algorithm?  The "we don't know what we don't know" problem concerns many of us.

- Two years ago it was pointed out at the annual meeting that HMO data were missing from the Medicare files.  Are those the Medicare "Advantage" plans?  Do we have any reason to believe that PALS use those less than the proportions noted in this Kaiser article?  Is this a significant problem whose backbone is the idea that a very high percentage of PALS will be in government files?

- MDA indicated in the last meeting that they have around 13,000 PALS in their database.  Does that not cause concerns that an organization that sponsors only a portion of clinics has so many PALS compared with the first Registry report and compared with the annual self-enrollments?

- Last year an annual meeting participant wisely pointed out that you can't manage what you don't measure.  Surely ALSA and MDA are able to tell us how many new PALS entered their clinic systems in the past year.  How does that compare with the new self-enrollments in the Registry?

- Have ALSA and MDA ever simply sent out an email survey blast to all their PALS -- Did you self-enroll in the Registry?  If not, why not?

- Have you measured how many PALS actually enrolled in the clinical trials for which they were notified via a Registry email? The importance of this was broached in the last meetings discussion and I hope has been measured since.

- In an OMB filing, the "annual cost to the government" for the ALS Registry was given as $1,100,000 annually.  Why does that differ so from the $10 million that we request every year?

- Could we get a ballpark breakdown of the annual budget by these categories, please?

- There was a public comments period for a new outbound telephone plan within the Registry project to move non-clinic neurologists' behavior to increase self-enrollment.  What data show that telemarketing like this will change neurologists' behavior?

- Is the premise in the telemarketing plan that non-clinic neurologists are a significant factor in low PALS self-enrollment not at odds with the special status that ALSA and MDA have been given in this project because of their clinic systems?

- Would you discuss Stephen Finger's article on Registry completeness and the first prevalence report, please?

- Would you discuss Stephen Finger's comment related to the recent survival data publication from the Registry, please?

- Would you discuss why Stephen Finger was not invited to participate in the annual Registry meeting, please?

- This observation was first brought up by a PALS at the advocacy conference in May, I believe.  Metro studies are done in areas with much larger minority populations than the general U.S. population, yet Metro studies are showing more ALS than the general U.S. population.  Does that not refute the conclusions of the first national Registry report that ALS is more common in white males?

- Why are we not getting a second annual report?

- The recently published report from the biorepository study group had some votes regarding the future of this part of the Registry.  The report said that ATSDR and McKing employees were not allowed to vote. Could we find out who the 12 voters were, please?

- Could any of the collected tissue samples potentially be used to develop iPS models?

- A recently published article associated formaldehyde exposure with ALS, and funeral directors were spotlighted as being at possible risk.  When something like this is published, how long does it take for ATSDR to review the Registry self-enrollment data to see if the Registry occupational data support or refute a theory?

- If an ALS researcher requests information from the self-enrollment portal, how will those data be released?  Will any of those data be released that have not yet been matched with the government files that are several years behind?

We have advocated hard and at our own expense to get this project more than enough taxpayer dollars to fill its mission and we look forward to the discussions on Wednesday and Thursday.

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