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.

Tuesday, September 14, 2021

Dear Chair DeLauro and Ranking Member Granger


Dear Chair DeLauro and Ranking Member Granger,  

I write you as a concerned advocate for those with ALS (we lost Mom to ALS in 1997), a person who believes in the value of epidemiological data in science, and a taxpayer.

ATSDR has administered a CDC ALS Registry since we worked hard to have the ALS Registry Act  passed into law in 2008. It has disappointed in many respects -- undercounting cases yet publishing reports as if counts were complete, terribly late reporting of data (we just saw 2016 data for the first time), falling into the distracting temptations of scope creep, and entangling itself in financial relationships that prevent objective and candid oversight.

You wisely inserted language into the recent appropriations bill --

https://docs.house.gov/meetings/AP/AP00/20210715/113908/HMKP-117-AP00-20210715-SD003.pdf

Amyotrophic Lateral Sclerosis Registry.—The Committee directs CDC to present a plan to the Committee within 90 days of enactment of this Act to enhance the oversight and advisory process for the National ALS Registry to increase transparency and better reflect the needs of the ALS community. This plan should consider ways to translate Registry findings to human application that can lower the risks and disease burden of ALS, make disease reporting more representative, more accurate, and more inclusive, and include a range of estimates rather than point estimates of ALS prevalence. Equally important are updates to the survey process to ensure the data they collect are impactful and respectful of participant burden. Finally, the plan should ensure that the ALS community can view as much Registry information as possible without compromising the privacy of participants.

Two weeks ago the CDC ATSDR Registry folks held an annual meeting. It has never been a true oversight meeting.  Two weeks ago it was more of a closed, clubby celebration of questionable achievements (from what little I was able to see).  The public could only watch part of the first day.  It takes an invitation to be a full, two-day participant with the ability to unmute and ask questions and make comments.  We're not sure what it takes to get an invitation. A man with ALS, Stephen Finger, PhD, who has participated for several years and asks good questions was not invited back this year.  He seems to have outlived his welcome.  Good for him, but his exclusion was revealing.  

ATSDR refers to its many "partners" at the meeting.  Those are largely paid contractors, including the ALS Association (ALSA), the Muscular Dystrophy Association, and Les Turner ALS Foundation who together get around $1,000,000 per year in no-bid contracts for some vague "education and outreach."  ALSA is also the lead advocacy organization that lobbies you for the $10,000,000 annual budget for the project.

Add to that a number of grant recipients and potential grant recipients at the meeting, and we have a pretty good recipe for a love-fest rather than any kind of objective oversight.

Please insist that they bring to you an oversight plan that is composed of third parties with knowledge of epidemiology and payer data and information systems who have no financial ties to this project.

Thank you very much.





Saturday, August 28, 2021

The Ketchup Was Always Worth the Wait


Anticipation.  Those were the great Heinz ketchup ads of the 20th Century.  The glass bottle with the
narrow neck, the thick, quality ketchup, always worth the frustrating wait while we hummed along with Carly Simon.

We've had a lot of anticipation and waiting since we worked hard to have the ALS Registry Act passed in 2008.  Has it been worth the wait?

We're close to the annual CDC ALS Registry meeting on August 31 and September 1, 2021.  It's not an oversight meeting.  The description is more of a celebration -- "Update stakeholders on the progress and achievements of the National ALS Registry."   Please register and tune in if you are able.  You've paid well over $100 million for this Registry.  Has the ketchup been worth the substantial waits?

The primary purpose of the Registry as written into the law is "to better describe the incidence and prevalence of ALS in the United States."  Everything else is mustard or relish.

Back in the 20th Century we were promised a passive data mining technique that would find 85% of ALS cases via this data mining, and then the other 15% could be added by a patient self-enrollment portal.   We would get annual reports after a time lag necessitated by the wait for public payer files.  That was the promise.

The waiting and the ketchup have been particularly troublesome in the last three years. 

The Waits

We have not received any semblance of annual reporting.  The cavalier attitude about due dates really puts the value of the output into question.  The 2016 data were just published this week.


Heinz knew when to completely redesign that ketchup bottle.  We now use 21st Century bottles that eliminate the wait.  Perhaps it's time for a significant 21st Century bottle change for the Registry. 

The Ketchup

The hit-and-miss prevalence reports have questionable value.  The undercount is significant.  The demographics continue to be published.  It is not clear that these are merely the people that the Registry finds, and there may be just as many people with ALS out there whom they missed.  Two top NIH officials at a recent Congressional said that according to the CDC there are 16,583 people with ALS in the United States.  We repeatedly see the CDC number as being the number.  First do no harm.  These prevalence reports are doing harm by misleading the public about the number of people living with ALS.

A recent study by Miller et al used some different data sources to estimate prevalence that was starkly different from the CDC's.  And the authors were able to be much more timely than the CDC.  

If the ketchup coming out of the bottle isn't what the consumer expects, Heinz knows to change the recipe.

The Recipe

The "FAQ" document that came with this week's publication of the 2016 prevalence report causes major concerns about the recipe.

They are cooking in excuses for even more delays.  That's not acceptable, especially with the trail of inconsistent excuses we've had in the past.

They are cooking in excuses for a flawed design.  They knew full well that ALS was not reportable or notifiable back when they made promises and designed the algorithm for passive data mining and the patient portal.  

They suggest buying data from more sources and adding them to the recipe.  Whoa!  It's time to step back and do some test recipes first.  Figure out what gaps new data sources can or cannot fill.  Design before you start buying ingredients.  We've been asking for years for their "partner"/contractors to supply counts on the numbers of unique people with ALS they served in the past year along with counts of how many of those were new.  Every year this has been completely ignored.  Finally, the CDC seems curious.  Look at the numbers first, please, before you enter into more entangling contractual relationships that may not actually be the best solution for the completeness problem!  They've never been in a rush before.  Why rush into implementation without a smart analysis?  Perhaps there are even some prepared products that could replace some of the old infrastructure.

They also are suggesting that the capture/recapture approach will be the magic spice to make the ketchup better.  That's not necessarily true.  A 2013 report challenges the magic for this project if you read it carefully. 

The Other Condiments

The ketchup hasn't been good and certainly hasn't been worth the waits.  

When insights into incidence and prevalence are questioned, the project fathers often change the focus to all of the other things that a generously funded government project has done.  The mustard and relish become more important when the ketchup isn't good.  How do they measure success of every add-on?  Number of emails sent does not make a great trial notification system.  Number of biospecimens collected does not make a great Biorepository unless we have tissue and data representative of the diverse population of people with ALS.  

It's quite possible that the external research grants are the most valuable contribution that this project makes; however, the grant making process is opaque to the public and we can't even find out how much of the $10,000,000 that this project costs taxpayers is used by these grants.

Nobody at the annual meetings seems curious about the expenditure in general, let alone on functions like trial notification or on the Biorepository or the CME or the prevalence reporting.  

The Ketchup Consumer

Some consumers have questions that never seem be asked at the annual meeting.  We pay $10,000,000 per year even if the ketchup isn't delivered and we pay regardless of the quality that finally comes out of the bottle.  We should be asking questions.

1. How many unique people with ALSA, MDA, LTALS serve last year?  How many of those were new cases?

2. CDC has given ALS, MDA, LTALS annual contracts that total around $1,000,000 (10% of the annual appropriation) for "education and outreach."  How does the CDC come up with those contract amounts?  How does CDC measure those contractors and deliverables?  What is the cost/benefit evaluation? 

3. Does ATSDR have any idea how much the Biorepository costs every year?  The trial notification? The grants?  How do they measure the cost versus benefit?

4. Is ATSDR not concerned about the incomplete prevalence count that has been taken out of context in public media?  People, even top NIH professionals, have quoted the 16K number as the number of people in the US with ALS.  Is this not a concern?

5. The Miller paper underwritten by MT Pharma found half of its people with ALS in private payer files.  That's pretty much the smoking gun that the surveillance of public payer files will not be successful. How did that  paper's authors get data in a much more timely manner than ATSDR? They reported data through 2017 in June of this year.

6. Earlier this year ATSDR received a suggestion to just publish tables of data in a regular annual process so that the report and publication process aren't delaying things.  Again, how about simplifying the process and just providing data tables in a regular and more timely manner? 

7. How many people are represented by the Biorepository?  How many of them are living?  What are the demographics of people represented?

8. How often are Registry data matched with the death index data?  There are reports that deceased people continue to get clinical trial notifications.  That is painful and does not speak well to the matching capabilities of this rudimentary emailer.

9. A 2017 report raised concerns about disparities, equity, and inclusion in Registry data.  What tangible actions have been taken to address and measure these?

10. ALS has a cruel way of imposing term limits on the invited people with ALS who are allowed to speak at your annual meeting.  We have lost Ted Harada, Rob Tison, and Becky Kidd among others, all experienced people who thoughtfully prepared and spoke their minds at your meeting before they died from ALS.  Your decision to change the rules in 2021 so that Stephen Finger could no longer participate  smacks of wanting to eliminate a thoughtful, prepared person with ALS who spoke his mind and perhaps outlived his welcome.  This is not a question.

























Tuesday, July 27, 2021

We'll Have What They're Having

My message for the House Energy Committee Health Subcommittee Hearing coming up on Thursday, July 29, 2021.

______________________

Everyone reading this letter already knows the urgency that ALS demands.

 

ALS has become an orphaned orphan disease.  That’s the worst.  

 

Ever since we lost Mom to ALS in 1997 I’ve written letters and testified to the FDA and written more letters and visited legislators.  We’re still an orphaned orphan disease.  

 

FDA

 

We want the same FDA that oncology has.  They have Project Facilitate to help with Expanded Access.  We don’t.  Why not?  

 

You can find 106 enrolling interventional trials for ALS at clinicaltrials.gov.  How many Expanded Access Programs are there?  Two.  And one of them is highly restricted.  Just two.

 

Something is not right here.  Other diseases have this Expanded Access path that also allows rigorous science to proceed while dying people can get access to therapies.  They even have an 800 number to help.  We in the world of ALS seem to get bogged down in opaque processes that move at the speed of concrete.  

 

NIH

 

We want the same NIH that other diseases have, too.

 

Sure, we appreciate the grants that the NIH makes toward a patchwork of ALS research.  But what about the expertise of the NIH that can make trials smarter and more accessible?  What about the knowledge of the NIH on how to do biorepositories better and more cost effectively than anyone else?  We need the NIH to become engaged with ALS for more than passing out grant money.  Please, we need the NIH that is known for the science that goes on at its headquarters for so many other diseases.

 

 

Is there any question as to why those dealing with ALS and their families feel that they have been orphaned?  We need for our federal agencies simply to do for ALS what they have done for other diseases.

 

That’s all.

 

Thank you.

Friday, April 16, 2021

Were They Just Doodling?

 For over a decade I and others (many of whom have died from ALS) have gone to FDA meetings to give  thoughts on the need for change for ALS.  There have been Patient Focused Drug Development meetings.  There has been an all-day FDA hearing on ALS.  There have been public hearings on innovative trial designs.  There was an all-day ALSA workshop  so that the FDA could hear our thoughts.  I was not a paid invitee.  This was a volunteer effort that I and others thought was important and would make a difference.  

For over 10 years we have worked with pharmaceutical and biopharmaceutical companies that have asked for volunteer patient and caregiver voices.  I have spent countless hours in meetings that many of us  thought would make a difference.

Over the same decade, we have talked to ALS researchers about how we can improve clinical research from the patients' and caregivers' perspectives.  Again, we thought we could add a valuable perspective and make a difference.

I and others spend long hours doing our homework so that we can be informed contributors.  We learn regulatory and industry perspectives that need to be considered.  We know that there were once good reasons for rules that we would like to see changed.  We have been driven to make Expanded Access and faster determination of scientific truth realities.

Today I think we've been had.  Was anyone even listening?  While we spoke, were they just doodling in those meetings, humoring us but not really planning to change a thing? 


Events of the past few weeks have shown that absolutely nothing has changed.

We've been had.


Wednesday, April 7, 2021

This Is The App We Really Need

Suppose the average life-expectancy for an average American adult is 42 years.

Suppose the average life-expectancy for a person with ALS is 3 years.

That's a big difference, and nobody hears the ALS Clock tick as loudly as a person living with it.

Enter the Apple Watch app that runs on the ALS clock.

It would make my watch run 14 times faster than it does today.  Mickey's hands would be flying.  The dates would be changing before my eyes.

If you tell a person with ALS that a project is being delayed a quarter on your healthy calendar, that is like 3.5 years on the ALS Clock.

If you waste a day on pointless meetings, that's like two weeks on the ALS Clock.

How uncomfortable would it be if an hour Zoom call took up 14 hours on your new ALS Clock App?

Pretty uncomfortable, I hope.

And that's what we need.  Turn your watch to the ALS Clock and get to work, faster.