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 22, 2017

We Need A Fact-Checker

This week I listened to several presentations on ALS.

Over many frustrating years in the fight against ALS, I've learned to listen for the devilish details. Sometimes I think we need an ALS fact-checker.

CDC Grand Rounds

First up on Tuesday was the CDC presentation for their Grand Rounds.  The topic was of particular interest to me -- "National Amyotrophic Lateral Sclerosis (ALS) Registry - Impact, Challenges, and Future Directions."

Early in the presentation there was an assertion of the applicability of this project to other conditions. But how?  The basis of using their algorithm to locate cases of ALS was based on the quick entry of people with ALS into government health coverage systems such as Medicare and the VA.  That doesn't happen with other conditions. Sure, this Registry has a mother lode of lessons learned, but how would another disease find value in its basic design?

We heard that the Registry speeds up clinical trial recruitment and expands ethnic and geographic diversity (without any data to back up that claim).  Sending out emails to the patients you located on a small fraction of enrolling trials doesn't necessarily advance any of those noble goals (and might actually have the opposite effect on trial diversity).  Who knows?

And there was the importance of the  "partners."  It seems to me that if there is any flow of money to or from any "partners," that should be transparent.  There are some "partners" in this slide who have been paid millions of dollars in this project. And exactly for what?

There was a mention that the large national organizations cited in the partner list represent a majority of people with ALS in the US.  How do we know that?

There was a vague reference that the Registry is providing biospecimens to scientists. How many scientists are involved and how many patients' specimens have actually been distributed?

Did I hear correctly that there were 60,000 risk factor questionnaires completed for 17 separate surveys (giving an average of 3529 responses per survey)?

A woman in the audience asked what percentage of people in the US with ALS are actually in the Registry.  That most important question wasn't answered.  We got a tapdance about the challenges of completeness and ongoing statistical studies and local studies and that you'll never find them all.  But we ask again, what percentage of people in the US with ALS are actually in the Registry?

The Registry continues to make generalizations about ALS being white guys' disease based on the people with ALS who are in the Registry.  What do the people with ALS who are not in the Registry look like?  And how many are there?

One presenter who has been involved in the project annual meetings made an off-the-cuff reference to ALS as a disease of 30,000 people.  Wait.  What happened to the 15,908 number that the Registry published?

And we had a lot of interesting information from one of the clinicians who has worked with the CDC on the Registry.  He had data citations in his presentation, none of which seemed to come from the CDC Registry. Oh, well.

ALS Research Update

Thursday evening there was an ALS research update directed to some college students.  Naturally it started with some basics about ALS. Take a look at the slide on the left.   But wait, what does "affects" mean?  And "two cases per 100,000?"  Is that prevalence?  How does that jibe with the "5 cases per 100,000" on the CDC's slide?  Are global numbers that much different from US numbers?

We again saw a lot of interesting data, but none of the citations seemed to involve use of data from the obscenely expensive US CDC Registry.

Families dealing with ALS don't have time to deal with devilish details.  And they shouldn't have to.   These mixed and vague messages are not acceptable in a world where data and science are so important.  And when precious resources are poured into projects, we need to get the facts right and make sure that their value is in proportion to their cost.

Wednesday, April 5, 2017

"I'm Smarter Than You Think I Am"

A few months ago a television news show had a feature on a wonderful teacher.  Each year she had an assignment for every student -- Write down something about yourself that I don't know that I should know.

The responses were at the root of her understanding her students, and they told her of family situations or interests or hobbies or pain or joy.

And there was one child who wrote an especially important message to her -- "I'm smarter than you think I am."


On an ALS message board I just saw a link to a presentation given by a respected, world-famous
ALS clinical researcher on the importance of clinical trials.  She prefaced her message by talking about the real difficulties of doing clinical research in a world of uncurated information on the internet and fake news.  She used terms like "anti-science," "poor education," "ignorance fighting proper science."

And all I wanted to say was, "We're smarter than you think we are."

Saturday, April 1, 2017

In The Middle Of A Roundabout

"The Patient Is At The Center Of Everything We Do"

But are you really patient-centric?

The process was clever enough.
ALS Clinic Neurologist: You need to see Dr. Specialist.  Debbie will make an appointment for you on your way out. 
Caregiver to Debbie: May I make the appointment with Dr. Specialist, please?  My schedule is a little difficult, sometimes.  I have a meeting at work on Thursdays and Monday mornings are out and my loved one with ALS can't be at an appointment before 10 am, and after 2 pm he gets really weak. 
Debbie:  We'll just call them to make a first-available appointment.  That way Dr. Specialist knows that you are our referral.  Then when you get the appointment notice, you can call them and change it to whenever you want.

Clever?  Yes.

Patient-centric?  Hardly.

The patient in this case can't communicate easily.  The caregiver is trying to hold down some semblance of employment while being a more-than-full-time caregiver.  Both are dealing with a demanding disease.  Both have very reasonable preferences for all appointments that should be easily accommodated without wasted time and calls.

Sometimes a healthcare delivery system sincerely believes that it is being patient-centric, but to the patients, it's like being at the center of a roundabout with healthcare providers going around them in circles to their own destinations.

Saturday, March 18, 2017

Selective Urgency?

ALS advocates received an email and social media alerts this week:

Urgent, eh?

It was urgent when we discovered that the ALS Registry annual meeting last summer was being blacked out.

It was urgent in December when we asked when we asked for an ETA on a detailed report from that meeting and got an incredibly arrogant "when it's ready" governmental response.

It was urgent weeks ago when we asked the ALS Association, the ALS Registry's chief lobbying organization, for verification of the cumulative cost to-date of the project (and we got crickets).

It was urgent when the second annual report was not published on time and we were kept in the dark.

It has been urgent every year when we have asked how the new proposed appropriation would be spent.  Some details of a budget are urgent for any responsible steward asking for funds. We get none.

It is urgent that we be given the courtesy of information that we need in order to be informed, responsible advocates.

That's urgent.

Thursday, March 2, 2017

Solutions Don't Have To Be Complicated Or Expensive

Goal:  To encourage participation in ALS clinical research.

This morning there are 79 recruiting ALS trials at About half of them are interventional.


Pay an impecunious college student (preferably one with a parent with ALS) $15 per day to post a daily tweet and Facebook post featuring one of the trials.

That is not hard.  It takes effort and a reliable student and a budget of around $5000 per year.  It provides a precious very part-time work opportunity for a student in need of cash.

This will cycle information about every recruiting trial to those dealing with ALS every 2-3 months.


Thursday, February 23, 2017

Partnership, Indeed

From an old ALS Association web page on government partnerships:

"The Association also has worked with Congress to secure $58 million for the registry, including $10 million in 2016."

Today ALS Association staff are on Capitol Hill for #ALSHillDay17 requesting $20 million more from our legislators for the CDC ALS registry.

That's a grand total of $78 million.  

$78 million for a project that isn't accomplishing its core mission. 

The CDC blacked out the invitation-only annual meeting and has failed to produce a public transcript or recording of that meeting before this additional appropriation request.  That in itself is revealing.

Think about that.

This "partnership" is out of control.  This is wrong.

Saturday, February 18, 2017

It's Time For Some Hard Facts

If ever a project should be dealing in facts, it's one that is supposed to be gathering data on the prevalence and incidence of ALS -- the CDC's ALS Registry.

The project has not delivered on its core mission well, and in the past few years the claim of success has been in an area of its scope creep.

Let's look at some facts.

This morning there were 76 open, recruiting clinical trials for ALS at
Of those, 39 are interventional trials and the rest are observational.

Here is the list of the 25 total trials included in the ALS Registry's email notification in the last four years -- .

Fact:  Just seven (9%) of the 76 currently recruiting trials are in the CDC notification.
Fact:  Just two (5%) of the 39 currently recruiting interventional trials are in the CDC notification.

Fact:  This is not a success story.

Tuesday, February 7, 2017

Spirit's Prayer

Spirit’s Prayer

Lord let me be simple, let me be true
A child of God, yes, that’ll do.

Lord let me be - some unblinding light
Colored image, piercing night.

Lord let me be - your hands, your feet
Clapping rhythm, dancing beat.

Lord let me see - your frame, your gold
Smiles singing, stories told.

Lord let me say – Love rules the world!
Amen, I say – “What a wonderful world”.

- Barry Crist -

Today I went to Jerry's funeral (see post below).

Some of his many friends told stories of Jerry that made us smile and laugh and appreciate the gift we had in Jerry.  Sometimes I go to a funeral and really don't recognize the person being eulogized.  That was not the case today.  It was the Jerry we all knew and loved.

Then at the end, his good friend, business associate, and lunch buddy read us his poem, "Sprirt's Prayer." 

A good way to end today.  A good way to start tomorrow.

Saturday, February 4, 2017

What Would Jerry Do?

ALS Advocacy is not fun.  The loss of life is relentless.  The funerals are heartbreaking.  The lack of progress is frustrating.

Usually I'm braced a little when we know that a person with ALS has taken a bad turn.  The grief enters and leaves at its own pace.

Last week a different kind of death and grief entered our lives like a shot out of the blue. We lost one of our most effective advocates.  His death was completely unexpected and did not involve ALS.

I use twitter a lot.  I'll tweet to pharmaceutical companies and journalists and politicians to try to shine some light on ALS.  Seldom do the pharmaceutical companies bite.  I'm just an individual tweeter, after all.  A few years ago somebody from the Lilly Open Innovation twitter account exchanged tweets with me. That led to some messages.  That led to a meeting to discuss clinical trial enrollment barriers for those dealing with ALS.  That led to many more meetings and chats at Lilly and Starbucks and Medicine X.  And that led to interactions with all kinds of activist-patients with a variety of diseases.

How did this happen?  Meet Jerry Matczak.  Jerry was paying attention that evening a few years ago on twitter and he wasn't afraid to engage in constructive interactions.  He listened.  Oh, how he listened.  He didn't get defensive.  He never said, "But you don't understand."  He really listened.

And Jerry dreamed as he listened.  He saw the potential of social media and technology to improve clinical research by making patients part of the solution. He saw bigger pictures than most of us.

And then Jerry delivered solutions.

Dozens of e-patients in every disease area have Jerry stories to tell.  He listened.  He dreamed.  He delivered.

Jerry was genuine.  Jerry was a real person who experienced his own loss of a brother to AIDS before there were effective treatments.  Jerry grasped urgency, and he knew the potential of his industry to save lives that are being lost today.

And on Thursday afternoon I received an email from another ALS advocate that Facebook was buzzing that Jerry had died.  No.  That can't have been right.  I just saw him tweet a few days ago.  It must have been some kind of mistake.  No, it was right.

The pharmaceutical industry has a gaping hole to fill now.  The man with the extraordinary gift to listen and engage e-patients and figure out solutions is gone.

My comfort moving forward is that we all have the example and the standard to follow. We just need to ask ourselves, "What would Jerry do?"

Friday, January 20, 2017

It Was Twenty Years Ago Today

Twenty years ago this morning we lost Mom to ALS.

She died from ALS.  She did not succumb to it.

In 1997 it was outrageous that people were still dying without any effective treatment, so long after Lou Gehrig's death.

In 2017 it's even more outrageous.  Over 2,500,000 people have died from ALS since Mom.

Not much has changed in two decades.  We raise funds and walk and support research, but the prognosis for a person today is mostly the same that Mom faced and that Lou faced.

Step back and look.  We have failed.  Individually and collectively we have failed.  Do we ever admit that?

A few days ago there was an ALS Association "Listening Tour" conference call.  I appreciate the opportunity to be heard.  Today I continue to be concerned with a large, influential organization not being able to be introspective and candid about problems and failures.  Dad always said, "Watch you don't break your arm patting yourself on the back."

"We have failed" doesn't make a great fundraising slogan, but we have failed.  We must admit it.  We must do things differently.

Twenty years.  It seems like yesterday.  It seems like forever ago.

Wednesday, January 18, 2017

Welcome to the 21st Century, Clinical-Trial Matching

If you've ever shopped for a clinical trial for ALS, you know that the shopping tools have been stuck in the 1980s.  Clunky.  Incomplete.  Passive.  Redundant in their flaws.

If you've ever shopped for a pair of shoes online, you know how shopping tools should work in the 21st Century.  Inviting.  Effective.  Sensitive to my preferences. Active.

We often hear about how it costs a billion dollars to develop a new drug.  Time is money.  Time is life for people with ALS.  Yet we continue to use archaic search tools or email notifications for clinical research projects.  People with ALS have been issued 1980s tools that are inefficient and ineffective for all stakeholders.

A few years ago I crossed paths with some folks whose work has developed into . Take a look.  They have a business model that addresses a lot of problems that people face in shopping for the right clinical research opportunity.

Over the years we have talked.  I have complained.  They have listened.  I have suggested.  They have listened.  They have taken an interest in ALS.  I maintain that ALS can be a stress-test for a lot of areas of healthcare that need improvement.  They get that.

Yesterday on the right panel of this blog below the tweets, I inserted a snippet of code that gives everyone a portal into their newest edition of their clinical trial search tool.  I hope that it is helpful.  Give it a try. You might find something interesting.  Use their contact form to let them know what you think.  Perfect?  No.  A step into the 21st Century?  Absolutely.

So today we see that it is completely possible to have online search tools that are kept continuously up-to-date with the newest data.  That has been a big flaw in many old search tools.  We see a tool that includes all recruiting trials (and not just those from selected sponsors).  We see a tool that unsnarls the data into something readable and usable.  We see a tool that gives you some control of filters.  We see a tool that is easily sharable.

Yes, I still have suggestions -- more control over filters, yelp-like reviews, information like the number of seats left at a trial site.  I have every confidence that we can get there.  Today we see the giant leap we needed to welcome clinical-trial matching into 21st Century.  Thanks,

Note:  I neither seek nor receive any compensation for my work to improve the fight against ALS.

Friday, December 30, 2016

Which Dots Did They Collect?

For over a decade, some of us have worked hard to get a good census of people with ALS.

My theme was, "We'll never connect the dots if we don't start collecting the dots."  I started the website in 2008 to help make the point.

And I worked hard with others to enable the ALS Association and the CDC to deliver what was to have been a population-based US Registry.

So many years and over $70,000,000 later, here we are.  

But did it count all the noses?  No.
Does it know how many noses it missed?  No.
Does it know which ones it missed?  No.
Does it make broad demographic generalizations based on the ones it did locate?  Yes.

Which dots did they collect?
Which dots are missing?
Is the epidemiology of the missed dots the same as of the located dots?

As an ALS neuroscientist so aptly said to me, "We don't know what we don't know."

And that's a problem -- a very expensive problem.

Tuesday, December 20, 2016

We Got Big Fluffy Slippers For Christmas

A long-awaited report has finally emerged from this year's blacked out CDC ALS Registry Annual meeting:

Please take a look.  It's easy reading.

It's a beginner's primer on the basics of the CDC's ALS Registry project.  It contains no specifics on any critical issues that may have been discussed at that meeting. There is no information on any decisions or advice from the participants.  Executive Summary?  Hardly.

We deserve specifics. We deserve metrics. We deserve accountability. We deserve transparency. We deserve results.

We don't deserve fluff.

Wednesday, December 14, 2016

There Are Big Lessons In Small Places

Last week was the big 27th International Symposium on ALS/MND in Dublin.  It is an intense few days.  There were 1100 people registered, mostly neuroscientists, clinical specialists, organizational employees, and a scant number of unaffiliated people with ALS and caregivers.

I learned.  I learned a lot there.

Less than a five-minute walk from the gorgeous Dublin Convention Centre on the Liffey was the small, old church of Saint Laurence O'Toole.

I learned there, too, in the shadow of the grand symposium.

When I arrived at the quiet little church, there were a few elderly folks already waiting for Mass to
start.  A couple of rolling walkers were stored behind their pews. Some younger adults trickled in.  Finally a line of very young lads and their teachers streamed in and sat in the front pews. The priest welcomed the boys studying for their first communions and prepared to start Mass.

I was relieved.  The homily wouldn't be overly long and wouldn't be all about Saint Augustine's or Kierkegaard's arcane philosophy lessons.  This day we were going to be geared for the kids.  It's not that I can't grasp complicated philosophy and theology.  It's that sometimes we need to take the shortest, clearest route from theory to practice, and that's the route that children's liturgies usually take.

And it takes a smart person to distill complicated lessons into those clear, direct paths.

As Mass began, Father also welcomed the shut-ins who were watching and praying with us on the internet.  This little church had a web stream for those who couldn't be there.

Yes, I learned at a tiny, old church in the shadow of the grand symposium of esteemed and ambitious scientists.

We could all learn.

Saturday, December 3, 2016

For the "Ask the Experts" Session Prior To the 27th International ALS MND Symposium

1. At my last count, there are at least a half dozen completely separate "precision medicine" projects for people with ALS.
     a. Has anyone actually established protocols and shared data between any of these silos?
     b. Is anyone concerned about the duplication of resources and infrastructure?

2. Are there any specific novel clinical trial design features that you expect to see implemented in the next 12 months as a result of the Airlie House guidelines or the ALSA FDA draft guidance work?

Thank you.


Information about "Ask the Experts" can be found at

We are grateful for the live stream this year and hope that more of the symposium activities will be live streamed in the future for those who are interested in the newest and best science but cannot travel.

Thursday, December 1, 2016

Stop, Look, Listen!

After a lively day of sausage-making, a 21st Century Cures Act passed the House yesterday and now it's the Senate's turn to make some things happen. I think that this can provide some good opportunities to advance the fight against ALS.  Increased NIH funding, FDA resources, and the Precision Medicine Initiative are all very important in my opinion.

But one item gave me pause when I saw the list of ingredients left in the House version:

Sure, a neurological disease surveillance system makes ultimate sense when there are so many devastating and unsolved neurological diseases.  But wait... "a neurological disease surveillance system coordinated by the CDC..." Been there, done that, and it has not gone well.

Please, stop, look, and listen to us.  We have an opportunity to do something great or to be part of a train wreck.

Today we have a tremendously expensive ALS Registry -- a neurological disease surveillance system coordinated by the CDC.  It has failed to live up to its potential, and it has cost us taxpayers over $70 million to date.  It has let us down on many levels.  It has generated scant reports whose numbers nobody wants to  use.  It has been fraught with scope creep and gingerbread of questionable value and unrelated to delivering accurate and timely disease surveillance.  And perhaps the most revealing problem of all has been its lack of transparency.  This year the invitation-only annual meeting was blacked out and we have not see a  transcript yet.  The CDC doesn't not seem to want to talk about it except to the voluntary health agencies that lobby annually for its funding (and in a cozy arrangement receive hundreds of thousands of dollars in no-bid contracts).

So please, stop, look, and listen to us.  Let's fix the CDC ALS Registry problem and move forward with something well-designed, nimble, transparent, and focused to provide valuable information on all neurological diseases.  Please learn from our journey and don't be part of a 21st Century train wreck.

Wednesday, November 23, 2016

We The People Get The Message

We simply don't accept it.

This year's CDC ALS Registry annual meeting was August 3-4, 2016.  It was blacked out.  No webstream was provided this year.

Here is some background:

Over three months later, we still have no public transcript, no minutes, no recording, no report from this meeting.

The project has been expensive.  We think that the cumulative total of our investment as taxpayers is close to $80 million.

The project results have been sparse and questionable.

Participants at the annual meeting are handpicked by those being compensated for work on the project.

The chief lobbying agency for the project, the ALS Association, is also a well-paid contractor on the project.  In a convenient cycle of silence, they are approaching time to have another $10 million etched into next year's appropriations without any discussion with the hundreds of advocates they like to send to Capitol Hill with their requests.

Our questions go answered.  We are kept in the dark.  We're tired of this.  The project and money are too important to be on a $10 million annual autopay without accountability and transparency and substantial results.

We the people do not accept this.

Thursday, November 10, 2016

The Principal Sets The Tone For The School

I learned a big lesson as a child.  It has served me well in life.

I'm a Boomer. Our grade school was in an area that experienced astronomical growth of families and homes.  Our classes were large and the students and families were serious about education in spite of diverse challenges.

We were blessed with excellent principals who were all business about providing quality life and
academic educations... except for a couple of years.  She was a well-intentioned but not very good principal.  The new policies were nutty.  Changes were ill-conceived and short-sighted.  We could have unraveled as a school.  We didn't.

It took strong, engaged parents who picked their battles carefully and taught their kids that the principal isn't always right, but she's the principal.

It took strong, competent teachers who cared more about teaching than administrivia.

It took students who could see what was going on but not let a principal stand between them and a good education.

Our parents addressed concerns quietly and directly and effectively.

It only lasted a couple of years. We survived.  It was a good life lesson for me.

I have since seen schools and organizations and businesses where the tone set at the top has not been good.  It takes a lot of work in the trenches to overcome that and not unravel completely.  We can't keep our eyes off of what's important... not for a second.

Thursday, November 3, 2016

Phone a Friend for a Lifeline

If you are not familiar with edaravone (Radicut or Radicava), here is some important background:

MT Pharma recently filed a New Drug Application (NDA) with the US FDA, asking for a "priority review."  That priority review would have been the FDA's commitment to review the application in six months rather than ten.  For some reason, the FDA declined the priority request and now we can't expect its decision until June, 2017.  That's four extra months for people with a disease that kills 500 Americans every month.

So now we wait while people die.  Or maybe we don't.

Normally I'm not a fan of Expanded Access Programs (EAPs) that the FDA allows for drugs that are not yet approved.  They can be terribly unattractive for drug developers, especially if their entire market for a brand new drug is an unmet-need disease like ALS.  They can be secretive programs where data are lost.  The right solution is to get drugs to market approval faster (where everybody has access and payers are paying),

Maybe in this case we shouldn't let the perfect accelerated approval concept get in the way of some possible benefits for the 4000 Americans with ALS who will die before the FDA review is completed and for the 4000 Americans who are about to be diagnosed with ALS in that same period.

Why is this case different?

  • We believe that MT Pharma America may be amenable to doing some kind of Expanded Access Program in the United States.
  • The product has already been approved and marketed for ALS in Japan since 2015.
  • The product had been approved and marketed for stroke in Japan since 2001.
  • The product has a good safety profile so far.
  • If people with ALS who might participate in an EAP would voluntarily self-report data, perhaps at a place like, all might learn more about the drug and potentially which subgroups of people with ALS it may help.
There has already been talk of medical tourism for people with ALS who have the substantial means to go to Japan.  There have already been shadowy internet schemes suggested to buy the product in other countries.  Neither of these paths of desperation is the right answer for Americans with ALS.  A A transparent and legitimate EAP would make so much more sense.

An EAP wouldn't be easy. This is an infusion product and the process won't be cheap.  But ALS isn't easy, and it is anything but cheap.

If you have any interest at all in seeing MT Pharma America provide an Expanded Access Program for edaravone, they need to hear from you.  If nobody asks, they will have no reason to go down that path.  Here is the contact information: 1-888-292-0058 (typo in phone number corrected Nov. 3, 2:30pm EDT... I apologize for the error) or email 

Some grass-roots ALS advocates started calling yesterday.  MT Pharma America has contractors fielding phone calls.  They will ask your name, phone number, email address for followup, and relationship to one with ALS.  Please take a few minutes and call or email them to let them know the importance that providing early access through an EAP could mean to the thousands of people with ALS who have no great options today.  And thank them for their consideration.

It's worth asking.  We certainly can't demand it.  This case may be different enough that an EAP might be possible for the drug developer to offer, could give people with ALS an option, and might even advance some scientific understanding more quickly.

Please consider and call today. This could be a viable lifeline for people with ALS.

Tuesday, October 18, 2016

Actions >> Words

Yesterday I was involved in an interesting twitter exchange on the difference between the words we hear in the fights against diseases and the actions actually delivered.

And an apt example had played out on twitter just a few minutes earlier yesterday.

We got word from an ALS not-for-profit that they are trying to gather 1000 voice samples quickly for a research project.  The voices both of people with ALS and of healthy volunteers are needed. I did my own recording and found the process to be very simple, fast, and unobtrusive.  This seemed like a good job for social media.  1000 recordings should be a piece of cake, especially since some organizations have a wonderful social-media reach.   I posted the link and some requests for people and organizations to retweet.

Not one ALS organization retweeted.  Not one.  Individuals did, but not one ALS organization.  No action.

Here is one of the words that bothers me when I see this kind of pointless apathy and inaction -- "collaboration."  The use of that word has been pretty constant since the windfall of the ice of 2014.  There have been tens of thousands of dollars spent on talking about "collaboration."  Meetings and talk and hollow words don't solve problems.  Simple actions do.

When it came to actually doing something simple yesterday to fill one organizations's ALS study with quick voice samples, "collaborate" was just a word.  It didn't generate any action.  It meant nothing.

Talk's cheap.  Show us what you do.

Monday, October 10, 2016

A Study in Customer Experience in Clinical Trial Inquiry in the United States

Following is a link to the full paper written as a part of my epatient participation in the Precision Medicine track at
Stanford's Medicine X 2016:

"A Study in Customer Experience in Clinical Trial Inquiry in the United States"

A brief summary was also given in an Ignite! presentation at Medicine X, and results will be presented in a poster session at the 27th International Symposium on ALS/MND in December.

Many thanks to all with ALS and their loved ones who have ever sought to participate in clinical trials.  I hope that the information in this study will make an easier path in the future.

Monday, October 3, 2016

If You Can Only Go To One Conference, Make it Medicine X

That's advice that a respected friend gave me three years ago.  Boy, was he ever right.

This was my third experience at Stanford University's Medicine X  .  It was my second as an e-patient.  I thought after last year's experience that this year might be a letdown.  Boy, was I ever wrong.

Pre-Conference 1 -- "Providers are from Mars, Patients are from Venus: A workshop to improve patient-provider communication."
Faculty and staff of the Stanford Medical School facilitated a workshop that opened eyes to challenges and pitfalls of difficult provider-patient communications today.  Actors who work in the medical school gave excellent portrayals of the challenges.  I left with hope that healthcare delivery will improve, and I appreciate the insights into the training and challenges of the physician.  Thanks to Boehringer Ingleheim for this workshop.

Pre-Conference 2 -- "Shared Decision Making"
Eli Lilly and Company decision science specialists facilitated a workshop centered around a painfully difficult decision that a young couple faced regarding genetic testing and Alzheimer's disease.  The process of sorting out the elements of decisions was enlightening.  Thanks, Lilly.

And I was certainly glad to learn the California Duck Protocol.

E-patient Dinner
The night before the official start of Medicine X, the e-patient delegates are invited to a group dinner where we met a talented and diverse group of movers and shakers, many of whom are dealing with huge health challenges.  It's a special group and I'm grateful to have been included.

Medicine X Day 1

The speakers who work in our government were such refreshing highlights -- truly people who are able and willing to break the bureaucratic ruts that hold us back.  Claudia Williams, LaVerne Council, Susannah Fox -- the sharpest tacks in the healthcare toolkit as far as I'm concerned.

And then Eric and Terri Amador came on stage to teach all of healthcare through their ALS experience.  And Day 1 was good, so good.

Medicine X Day 2

As distracted as I was over my afternoon Ignite! presentation, it was again a productive day.  The
people you meet, the conversations you have, the presentations you didn't expect to attend are all some of the best things about Medicine X.

My Ignite! talk went fine, and I had questions about the clinical trial secret shopper project afterward, and I can't thank Louise Schaper enough for making a challenging 5 minutes fun and bringing out the best in every presenter.

It was a football Saturday at Stanford, and as I walked back to my hotel after a very long day, the campus quiet was almost eerie as tens of thousands of people were consumed with football in a far corner of the campus.

Medicine X Day 3

Just when I thought it couldn't get better, it did.  I attended the Million Veteran Project presentation in
the PMI tent.  They have half a million veterans' genetic typing completed and histories completed already.  They are looking for things to do with this rich set of data.  

They are looking at a long list of diseases that are often connected with those who have served in the military -- heart disease, PTSD,... Have they looked at ALS, I asked?  They did not know that there is double the incidence of ALS in US military veterans than in the general population.  They do now, and it is on their radar.  Think of the clues that all of the genomic data may hold related to the ALS-military mystery!

Thank YOU, MVP.

Then I went to a session on the project in Portugal.  For years we have been wanting a resource where the McGyver hacks that people invent to outsmart ALS can be archived and referenced.  Is this it?  I think it may be.

Finally I attended a timely session presented by Joe Gulfo on the FDA we need in the 21st Century, certainly a timely topic for me.  We do need a strong regulatory body and Joe's insights into its history and weaknesses today were interesting.   Ironically this was the day before the FDA Sarepta announcement.  And I am better armed today to march forward with ideas to shorten the path to approval for experimental ALS treatments.

Thanks very much to Dr. Larry Chu and Sarah Kucharski and Zoe and all who made this a valuable and energizing conference for me again.

Monday, September 26, 2016

Feed the Monster

I've been going to (yes, actually going to) Walks to Defeat (nee D'Feet) ALS for 15 years.

Something has bothered me for 12 years.

ALS leaves a large, terrible wake of loved ones behind every time someone dies from the disease, and the deaths are constant.  Why doesn't the attendance at walks grow at a constant pace every year to reflect that wake?  Why isn't the crowd that raises awareness and makes noise in communities huge by now?  It seems like the size of the crowd remains pretty flat year after year.  It's large, and that's wonderful, but where is everyone with loved ones left behind who used to come to the walk three or four or five or more years ago?

Every not-for-profit needs to figure out a revenue machine that will keep it sustainable.  I get that.  The ALS revenue machine is almost too easy.  There are thousands of newly diagnosed people with ALS every year and their families and friends and coworkers and neighbors want to help.  Enter the multi-level marketing of the walk revenue machine, and those who want to help can donate, and they do.  The constant stream of new diagnoses supplies many new donors and old ones fade away a few years after the funerals.

And getting people to actually show up for the walk itself isn't even a priority for some chapters.  It's all about the revenue machine, and the relentless disease is the fuel that feeds it.

Some people think I'm crazy for going back year after year after year.  I'm not.  If more people did that, we would have more people who see the problem of feeding the monster without changing the fight.  You see, the talk of achievements is recycled year after year.  It doesn't take Einstein to see what has happened.

Saturday, September 17, 2016

Meet Elizabeth Cosby

Today I have the privilege of delivering an Ignite! talk at Stanford University's MedicineX.  I appreciate the opportunity to present a message about ALS and clinical research to so many outstanding people whose mission is to rebel and improve medicine.  Thank you, #medx .

The full paper on this project will be published on this blog before October 1.

The project results will also be presented in a poster presentation at the ALS MND Symposium in December.


The day Mom was diagnosed with ALS, it hit us from out of the blue. There is one thing I remember vividly about that day.
The neurologist pulled me aside and said, ”Get her to someone doing clinical trials because the only thing the least bit “promising” would be experimental.
We were fortunate.
We had an action plan on day 1.

You see, the ice didn’t cure ALS.
There is no effective treatment.
It’s a difficult disease.

That’s EXACTLY why clinical trials are so important to those with ALS.
1. They are a legit Hail Mary
2. They advance the science for others.
You who do clinical research RECRUIT for trials. 

But wait.  You are RECRUITING.  They are SHOPPING.
People looking for a clinical trial are doing what we all do every day when we look for a new car or a great, affordable restaurant in Palo Alto.
They do not seek to be recruited.
They seek the best trials for them.
Businesses use Customer Journey Maps to show the complexity their customers experience to do business with them.
Complexity is never good in customer service. They journey should be easy for the customer.
There are some basic elements of most Customer Journey Maps –
Awareness, Research, Purchase, OOBE (WOW) Disney Apple.
Lets back up to Awareness.  A lot of money is spent on making people aware of clinical trials. Great.
Based on personal experience, I suspect that we are losing people who are already aware of clinical trials in the Research part of their journey, and the problem is poor customer service.
Am I all wet? How could I put some data behind my suspicion?  Meet our secret shopper, Elizabeth Cosby.
Elizabeth is a fictitious woman who came to life a few months ago in a new gmail account.  Elizabeth has a fictitious aunt who was recently diagnosed with ALS. 
Elizabeth tried to help her find a clinical trial.
The first part of the journey was to find all recruiting, interventional ALS trials.
Elizabeth wanted to organize the information for comparison shopping.
That was terribly time-consuming.
Finally on the evening of April 4, Elizabeth emailed inquiries about 39 trials.
There were 5 simple questions in each email – things like is it still enrolling, time commitment, reimbursement…
The first response came back that very evening.  It answered all the questions and was encouraging. Was I all wet about poor customer service?
Unfortunately time told the tale.

Of the 39 trials, Elizabeth got absolutely no response (not even an autoresponder) regarding 11 of them.  Crickets.

39 clinical trials.
28 responses.
11 crickets.
From the 28 responses we a variety of data on the five questions.
Please read my paper at for the full scoop.

This project was as much about the journey as the five questions.
Elizabeth assigned a completeness score to each response.
If you answered all 5 questions, you got a 5.
If you punted or simply referred Elizabeth to a consent document, you got 0.
Average completeness score was 3.  On average, Elizabeth had 3 of her 5 questions answered.
Elizabeth assigned a tone score to the responses (respectful, collaborative, encouraging).  Sure, it’s subjective, but that doesn’t mean it’s not important.
Average 4
It really got interesting when we added the scores together for each response.
13 of the 28 responses got perfect scores of 10.
What we saw was that
when they were good, they were very, very good, and when they were bad, they were horrid.

Good customer service. 
It’s not rocket science.  It’s not neuroscience.  It’s common sense. 
If we fix some easy customer service problems that Elizabeth uncovered, the clinical trial path will be faster and easier.  Everybody will win!