Thank Heavens These People Didn't Design Our Interstate Highway System

Imagine a highway system where the people who wanted a highway from New York to Chicago built one.  And the people who wanted a highway from Cleveland to Saint Louis built themselves a road.   And the people who wanted a highway from Kansas City to Denver poured their own pavement.  And the people who needed to get from Dallas to Los Angeles constructed their own road.  And the people in Seattle who wanted to get to Miami blazed a long and impressive trail.

A mess?  Indeed.
An expensive mess?  Yep.
An unbelievable premise?  Sure.

Today we have a very expensive ALS "Registry" project that was implemented by the CDC.  We know little of its status because of the cone of silence that has enveloped those who are invited to participate in its annual meetings.  We know that it has passive surveillance of government records of ALS cases at its backbone, and we know that there is some self-enrollment that complements the mined data.

We also have a PRO-ACT database provided by NEALS that houses clinical trial data on many of the same people who are in the CDC's registry.

And the MDA has recently announced the launch of its own patient "registry" that will capture data related to patient care and natural histories.  We assume that this will house information on some people with ALS who have fragments of information in the CDC's "Registry" and the PRO-Act database, too.

And at the recent Team Gleason Summit, leading ALS researchers spoke of the need for some "big data" concepts of holistic views of patients so that every patient can be a research patient.  Oh, and nobody present involved with the CDC project or the MDA project mentioned those within earshot of the webcast.

A mess?  Indeed.
An expensive mess?  Yep.
An unbelievable premise?  We're living it.  Oh, and we're paying for it.

Lou Knows

Ability is what you're capable of doing

Motivation determines what you do 

Attitude determines how well you do it 

The fight against ALS has failed to deliver a treatment. We must face that we collectively and individually have failed. Feel-good pats on the back for effort don't save lives. We have not won the fight.

Is it our ability?

Is it our motivation?

Is it our attitude?

We must fix all three.  Lou knows.

This Is Putting Heads Together?

1. A few weeks ago Team Gleason assembled an unprecedented group of scientists, clinicians, and organizations (at Team Gleason expense) to put their heads together and fix the fight against ALS.  The MDA was among the organizations represented.
2. Throughout the Team Gleason Summit, conversations touched on information needs and the data concepts that would be central to speeding the development of therapies.  It was a persistent thread.  
3. As I listened on the webcast, I didn't hear a peep from the MDA about information or data.  Not a word.  Nothing.
4. Today we see this big announcement from the MDA --  http://mda.org/media/press-releases/quintiles-selected-muscular-dystrophy-association-develop-us-disease-registry

So much for putting heads together.  I'm not sure that Team Gleason got its money's worth.

The Difference Between Hope and Hype Is A Single Letter

We learned a lot from the discussions at ALSTDI's conference today.

One of the afternoon panelists made the statement that stuck with many of us -- The difference between hope and hype is just one letter.

I've longed for some kind of Pinocchio-meter for all of the "promise" touted in the fight against ALS.  Perhaps we can use a Hope-Hype image that fades to the correct vowel when time reveals the truth.

Please tame the hype.

It's Time To Dump The "Pick A Leader And Get In Line" Fight Against ALS

Occasionally a big light bulb turns on in my dim mind.  This week I experienced a lot of lumens as I worked in an online group trying to plant some new seeds in the fight against ALS.

People waste a lot of time and energy vying for so-called "leadership" in this fight.

When people experience ALS, it is overwhelming.  You have to pick your battles and you have to learn to trust people who can help you.   You also have the urge to make a difference.  It's a delicate equation -- trying to find your leader who has blazed trails before and trying to help the next person.

Add the whole instinct to survive which drives you to those leaders with "hope" written on their backs.

And there is a constant stream of rookie recruits.

And the disease awareness is so poor and the disease demands are so high that resources are scarce.

For years we've operated under a pick-your-leader-and-march mentality.  It hasn't exactly worked well.  Organizations and individuals vie for followers, and people in lines aren't exactly using their creative juices well.

We need to smarten up.  We need to recognize strengths and weaknesses in organizations and people.  We need to break up the lines and learn how gang up on problems and not each other.  We need to stop meandering behind leaders.

Oh, and those who think they were born to lead us need to stop spinning stories to attract followers.  Let the data speak.  We'll figure out when and where and whether to follow today.

And sometimes the one who appears to be the least among us at the back of that line has the idea or the common sense that we really need to follow today.

Let's Have The Most Painful Meeting Ever

Since this seems to be the year of meetings to defeat ALS, maybe this concept of two serial meetings would actually move us forward together.

Let's start with a virtual room full of people with ALS, caregivers, some retired pharmaceutical executives, and some retired FDA officials.

Why the retirees?  They know their businesses, yet they have no horses in this race.  They don't have to worry about walking on eggshells around people who are dying.  They can be brutally honest.  They can be informative and candid.

Stakeholders need to stop trying to assume that they know what the others want or need.  They need to talk.

Meeting 1:

OK, let the first meeting begin.  Let's talk about investment in ALS drug development.  Let's talk about the business of bringing ALS drugs to market.  Let's talk about the short-term and long-term financial incentives.  Let's talk about the approval process as it really is.  Skip the Ph I,I,III basics. We hear those over and over but seldom get past them.  Let's talk about breakthrough status and expanded access programs and markets for drugs. Let's talk about how drugs are paid for. Let's talk about how a program that is good for a rare cancer drug may be not so good for an ALS drug.  Let's hear the heartburn that each special program brings to each person in that room.  Let's learn from one another.  Let's tell it like it is.

That's a pretty painful meeting for all parties, but it would be tremendously constructive.  Today patients dream dreams about programs that they think could bring drugs to them.  Are they even viable?  Are they conducive to industry investment in ALS?  Are they products of an FDA that today doesn't have good "special" programs designed for an ALS?  Are there paths we've not considered.  What do patients really want?  What do drug developers really want?  What do investors want, today and tomorrow?  What does the FDA need? What does everyone need?  

All the cards and reality need to be on the table.  We need to be candid with one another.  That's a constructive, uncomfortable, painful meeting that has never happened before.

Meeting 2:

Ah, now we're ready to design some solutions.  Now that we've stopped assuming and mind-reading, we can start the creative process.  Let's break through the maze and find a path to get therapy candidates to patients more quickly.  Let's not try to bend existing programs to fit ALS if they don't really serve all the stakeholders well.  Let's move the fight forward creatively on solid ground.

We've had far too many variations of Meeting 2 already this year.  We need a Meeting 1.

Just The Facts, Please

History is an essential asset, yet in the fight against ALS, we do a very poor job of retaining it.

The nature of ALS is that the people dealing with it don't have memories of what happened five or ten or twenty years ago.  They are slapped with a diagnosis and are largely on their own to find information and to figure out whom to trust.

We need to do a better job of retaining what really happened two or five or ten or twenty years ago.  We need to make the fact-finding easier for those with ALS who seek a ray of hope.

Occasionally those of us who have been paying attention to matters ALS for more than a decade see or hear someone spouting revisionist history.  It may be innocent, or it may be manipulative.  In either case, it's wrong and it does not help the fight move forward.

Today's technology could give us an excellent electronic archive of historical facts.  It's time for organizations and individuals to contribute facts lest we forget or are led astray or repeat the mistakes of the past.

Pick Your Game Of Musical Chairs

The same number of people will be added to each game continuously.  There are a lot more chairs in the blue game than in the red.

The undertaker removes people who don't have chairs when the music stops.

Which game would you rather join?

This shows the flaw in using prevalence (the number of chairs) as the measure of a disease's impact.

The small number of ALS chairs doesn't mean that it's a small problem.  It means that it's a huge problem!

Aren't There Ever Any Scouts At The Table During Trial Design?

Envision a person with ALS who has volunteered for a clinical trial -- a trial designed in much the same way that researchers have traditionally designed trials for rashes or cholesterol control or male pattern baldness.  There is trial period with the drug or placebo, and when that's over, everyone waits while results will be evaluated and the drug company will decide if and how to proceed to another trial phase.

For ALS trials, it seems like the trial designers never seriously consider what happens to their trial volunteers if this stuff really works.  It's not like they'll just have to scratch an itch or watch their diets or comb over while they wait to see what's next.  They decline quickly. They die.

People with ALS who have volunteered for trials and who perceive improvements during the trials are left hanging out to dry.  This all could be avoided with some creative trial design that gives them options to continue on the drug (or the placebo... if it works, they really don't care) before they sign on the dotted line.

Instead we have traditional trial designs and the subsequent cry for Expanded Access Programs after the fact.  Perhaps we should get in front of the problem and have trial designers, including patients, talk about what happens to trial volunteers if this stuff really works.  Be prepared.

Stuck In The Middle WIth You

The organizational fight against ALS loves the middle.

At the recent Team Gleason Summit or February's FDA Hearing, who was dreaming big, daring to change the basic ways that business is done to fight ALS?

Who was dreaming small, willing to step up and deliver some simple basic changes that would tear down silos that make the armies fighting ALS so inefficient?

The battlefield against ALS is so comfy in that middle where "promising" projects become expensive and big and not game-changing... and the deliverables are poorly measured and nobody is really held accountable.

Pffft.

Pick Your Bus

Here are the ground rules.

• There is one empty seat on each bus.  Take your pick.
• Each bus will pick up five people today.  Five riders will have to give up their seats to make room for the new riders and will be taken off the bus by the Grim Reaper.

One bus has 15 seats.

The other bus has 65 seats.

Which bus would you pick?

The smaller bus is a much bigger threat, eh?  Who would ever pick it unless you were anxious to get kicked off to meet the Grim Reaper?

This demonstrates the myth that smaller disease prevalence (the size of the bus) means smaller disease impact.

ALS is the Grim Reaper Express Microbus.  It's a lot worse than a big bus.

I Was Hungry...

They say that the bitterest words at final judgment will be, "I was hungry, and you formed a committee."

Perhaps a fitting corollary to that will be, "I was dying, and you had a meeting."

Food for thought in this, a big year for big ALS meetings.

What Are The Barriers?

A few months ago I was discussing ALS with some people far smarter than I.  A gentleman asked me a thoughtful question -- "What are the barriers between you and a treatment for ALS?"

Wow.  I had to think about that.  I had a diverse list of answers.

What if various stakeholders in the fight against ALS put heads together and were candid with each other about the barriers they perceive?

We all might learn a few things.  We all might be able to move a few of those heavy sandbags.

Happy Physicist Taught Us All A Few Things

A gentleman with ALS who went by the online name "Happy Physicist" died yesterday. 

HP taught us all much. 

He was a scientist and is best known for his constant study and experimentation on himself to see if he could unlock a clue to treating ALS.  He understood science and he knew what ALS would do to his future if he sat back and watched.  And HP encouraged all people with ALS who tried things (and they all do) to share their data.  "If it is done in secret, then it is done in vain."

There is another mark that he left that some may not remember, so I take this sad day to remind us all.

HP studied available clinical trials and signed up for one he thought had promise -- Neuraltus NP001.  HP also knew that a day waiting to fill a clinical trial was as precious as a day waiting for the FDA or for scientists.  He took matters into his own hands to get that clinical trial filled quickly.   He used every social media device known to man to let people with ALS know about the trial.  He was a gazelle.

HP was from Bloomington, Indiana, and he traveled to Lexington, Kentucky, for his clinical trial infusions.  That was a long drive but worth it to HP. On one trip, HP got online and found the ALS Association Lexington support group meeting was happening while he was there.  He showed up at the support group meeting to try to recruit his peers to give the NP001 trial a whirl.  Here's the shocker -- they had no idea of this clinical trial opportunity in their own back yard.  Several signed up right away.

So the next time clinical trial sponsors or organizations or research scientists complain about patients not willing to enroll in clinical trials, look upward and remember HP, and tell them that perhaps someone should simply tell the patients about the trials.

Thank you, God, for HP.

Prevalence Is A Red Herring

For years we have been told by our largest ALS organization that disease prevalence is the "gold standard" by which the public sector measures the relative impact of diseases.  That's just the way it is.

Questions from Congresspeople or Senators about constituents with ALS are typically answered with a logical guess based on a number that has been couched and hedged for years -- "As many as 30,000 Americans have the disease at any moment in time."  Carefully worded, eh?

Everybody knows somebodies who lost somebodies to ALS.  Oh, it's so easy to want that prevalence number to be higher so that ALS will rank higher on America's priority list.

That's a red herring -- a big stinky red herring.

We believe that as many people are diagnosed with ALS as MS every year.  That's incidence.  

The number of people living with ALS at any moment (prevalence) is a tiny fraction of those living with MS.  What happened to all the people who were diagnosed with ALS?  Hint:  It's not good.

Does smaller prevalence make ALS a smaller problem than MS?  Heavens, no!  It makes it a much deadlier problem.

High incidence and smaller prevalence form a toxic combination.  If you only look at prevalence, you are missing the boat.  Low prevalence does not make ALS a small problem. It makes ALS a huge problem.

So as the ALS Registry numbers unfold, we'll probably find in the data that there are far fewer than 30,000 people living with diagnosed ALS at any moment in the US.  Should we avoid that low-prevalence fact?  We need to stop leaning on a shaky prevalence guess that feels good for fundraising and speak to the data.  High incidence paired with even smaller prevalence mean that we have a large, urgent problem.

It takes a smart hound not to be thrown off by the scent of a stinky fish.  We need to be smart in the fight against ALS.  Leaning on an assumed "gold standard" with a shaky number isn't going to defeat ALS.

This troubling ramble that worships the red herring is from the report of the CDC's 2012 ALS Registry Annual Meeting at http://wwwn.cdc.gov/als/Download/2012%20ALS%20Annual%20Meeting%20Summary%20Report.pdf

While he did not want to hide information, it could possibly come out that there are not 30,000 people 

with ALS and that it is much lower.  There would be a lot of negative connotation about what this 

all means if there is a map with people in each state.  He could hear appropriators and 

Congressman saying, “You only have 2 people in the whole State of Texas who have this 

disease?”  He thought it was very important that consideration be given to everything in terms of 

research projects, because the intention was never to figure out the number of people who have 

the disease.  If incidence is higher in one state, that is important.  While they should not hide 

information, they should also not be distributing information that could be damaging to the 

research tool they wanted to create, particularly in a budget-conscious environment.  That could 

hurt the overall goal of finding a treatment and cure. 

[Hat tip to The ALS Battlefield for all his work.]

Let The Data Speak

What are the barriers between people with ALS and a treatment?

That's an important question.  There are some answers that may be surprising.

Organizational secrecy causes some big barriers, and it serves nobody well except organizations.

Think about it.

Let the data speak.

Please Love Us (And Our Friends) For Our Minds, Too

Dear _____,

It's that Walk time of year.  As a participant in past Walks, we want you to be among the first to sign up and help us raise valuable dollars in this year's Walk.

Please sign in online and donate what dollars you can and tell your friends and ask them to donate as many dollars as they can.  Spread the word.  It makes a difference.  Your contributions advance the valuable work of our organization that is focused on beating ALS.

Thank you for your past contributions and we look forward to your help and the help of your friends and theirs and theirs as you all contribute valuable dollars to help in this Walk to fight ALS.

Sincerely,

________________

Ok, now simply substitute the word "ideas" for "dollars" and substitute "Advocacy Effort" for "Walk."

Get my drift?

Dear People Invited To The CDC ATSDR ALS Registry Surveillance Annual Meeting Today

If the veil of secrecy is required, please explain that requirement to us.

If the veil of secrecy is desired, please explain that to us, too.

Such secrecy is not healthy for good project governance and is clearly not in HHS's spirit of patient-centricity.

Thank you.

Howdy, Partners

We think that the supporters of the CDC's ATSDR National Registry and our major ALS organizations deserve some candid and clear information about who has been paid to do what from the government funds that we fought hard to have approved.

From ALSA's 2012 Annual Report, the most recent on its website, under the heading of "Fighting for the Interests of the ALS Community," there was mention of a "partnership" with the CDC to help raise awareness of the National ALS Registry.  There was absolutely no mention of any kind of contractual or vendor relationship or compensation related to the $6 million which we taxpayer/advocates requested of all taxpayers for the Registry.

 

 

Then we stumbled across this cool million (quite by accident) a few weeks ago at usaspending.gov.

 

 

And what in the world is this about?





 

We understand that the ALS Registry powers that be are meeting in Atlanta this week.  We hope that they'll shine some sunshine on their proceedings and processes and "partnerships."

We Should Have A Large, Angry Crowd By Now

Around 10 million people have died from ALS/MND since Lou Gehrig's diagnosis in 1939.

That's like the entire population of Belgium or Sweden.

That's an outrageous loss of life.

Who can lead the many more loved ones of these 10 million people into a cohesive movement?  The base grows, yet the engaged base doesn't.

Porquoi? Varfor?

We Have Three Very SImple Questions Regarding The CDC's ATSDR National ALS Registry

What percentage of the total patients in the Registry have been identified...

Q1: Both by your passive data mining and by self-enrollment?

Q2: By passive data mining only and not by self-enrollment?

Q3: By self-enrollment only and not by passive data mining?

How else are we to know the effectiveness of the self-enrollment efforts?  Whoops, I guess that's a fourth question (but only if we can't get answers to Q1, Q2, and Q3).

And if we can't get the answers because of some OMB policy, then yet another question is whether the OMB gag order is OMB's alone or whether there was an agreement presented to OMB by the CDC which prevented this disclosure.

Yes, it turned out to be five questions, but there are only three if we get the simple (and important) responses to Q1, Q2, and Q3.

The Greatest Day Ever For ALS Awareness Was July 4, 2009

http://en.wikipedia.org/wiki/Michael_Goldsmith

Through the work of one wise and driven man with ALS, every major network and every major publication carried an ALS feature.

This website gets a pretty steady amount of traffic.  On that July 4 weekend four years ago the traffic was astronomical.  It came from thousands of people searching for words related to ALS.  Never before, never since has there been anything like it.

"4 ALS" wasn't about fundraising.  It wasn't about an organization.  It was about ALS awareness and it worked.

Michael Goldsmith, we miss you.

"Mom Is Still Mom"

If You Could Propose One Idea To Improve Healthcare Delivery...

@phrma asked us on twitter, " If you could propose one idea to help improve health care delivery in the United States, what would it be? - See more at: http://www.phrma.org/phrmapedia/conversations/improve-us-health#sthash.KrzGflZr.dpuf "

If anyone knows where the rough spots are in healthcare delivery, its a person with ALS or a caregiver.

 

This idea is simple. It's incredibly simple. It's low-tech.  Its need reflects a healthcare delivery system that still doesn't get patient-centricity though.

Dear people in the entire healthcare delivery process, I, the patient or caregiver, am a valuable resource for fixing a few things that aren't hard to fix.  Please listen to me.  Really listen.

After waiting for an appointment under the racket of a waiting-room television, I mentioned to the receptionist that it would be nice if there were a quiet area for patients who choose to read or work.  Even my car dealer does that these days so that I can be productive while I wait.  Did that suggestion make it to a staff meeting or a process-improvement team at the doctor's office?  Probably not.  I think a lot of those patient comments are ignored by the front-line folks because the patient can always mention them on the customer satisfaction survey.  Besides, is the receptionist expected to be a waiting-room change-agent?

When I said to the physician, "Gee, the walls are thin in these exam rooms," that was a big clue that I heard things that were none of my business.  What happened to that comment?  Probably nothing.  Yet we spend gazillions on HIPAA forms.

When I as a caregiver mentioned to the doctor that it took me two trips to the BMV to get the handicapped hang tag and if the doctor's office simply kept those forms, it would only have taken one, did anything happen to save the next caregiver a trip?

Healthcare delivery stakeholders, your patients are a gold mine.  Are front-line healthcare deliverers motivated to be part of solutions?  Between it's-not-my-job syndrome and leaving the voice of the customer to the customer satisfaction surveys, we miss the gold mine.  Are patients who make observations and suggestions valued or are they wasting your valuable time?  It's up to you, healthcare delivery.

We Need More Idea-Thons To Defeat ALS

We're so good at walk-a-thons.  Let's get really good at idea-thons.

The idea struck me during the consensus-building final portion of the Team Gleason ALS Research Summit yesterday. www.teamgleason.org/summit

It's harder to do online than they did at the tables, face-to-face, but I think some clever people could come up with a good online format that should include everyone -- rocket scientists down to low-i.q. bloggers.

This would be like speed-dating for ideas.  Get some sparks of creativity out there.  Capture them.  See if they generate other sparks or plop.  Move on.

In an online, chat-like format:

• Pick a general topic
• Have someone float an idea
• People who want to discuss stay in the chat and start chatting
• People who don't want to discuss or who don't like the idea stay on the sideline
• Have at it for fifteen minutes
• Float another idea...

Eventually the idea bag is full and it's time to deliver (thanks, Dreamfinder and Figment), but in the fight against ALS, we've not been very good at the new ideas. The more ideas you have today, the more ideas you'll have tomorrow.

Yesterday we had sparks of creativity and the discussions were all-inclusive. That's good and that's different.