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.

Friday, March 29, 2013

This Is Almost As Good As A T-Shirt

Well, maybe not as good as a t-shirt, but it's not bad.

Last year for ALS Advocacy Day I had some nice buttons made up for PALS who had self-enrolled in the CDCs National ALS Registry or who were in clinical trials (or both!).  The idea was for them to let their peers and others know of the good deeds they had done.  This encourages others to participate and starts some good conversations.  It was nice to see so many wearing them when we went to Capitol Hill.  They are large and readable buttons.

I still have some buttons left and would love for them to find PALS who will wear them proudly.  I would also love for them to find PALS and support group leaders who will help spread them to other PALS and will help spread the word on the importance of Registry self-enrollment and in clinical trial participation. 

All I ask in return is that they be put to work and worn by those in the Registry or a trial.  If anyone would send me some pictures of PALS wearing them, you can be assured that they'll be posted.

Please send a snail mail address and how many Registry buttons and how many clinical trial buttons you need to alsadvocacy at gmail dot com.  You can be assured that your address will be deleted after the buttons are sent.  I'm just an individual trying to spread Registry and clinical trial awareness.

While supplies last.  Please help put these to good use!  If I win the lottery, there will be t-shirts ;-)

Thanks, all.


Wednesday, March 27, 2013

Patient-focused? Fiddle Dee Dee

I submitted an online request for a prescription refill last week.  It seemed like such an efficient way to give the doctor's office what they needed in writing so that they could process at their convenience.

Their online request form was a little awkward for the many people whose health plans expect the use of a mail-order pharmacy.


It was pre-coded "I will pick up at pharmacy."  I simply entered the name and out-of-state address of the well-known mail-order pharmacy along with their fax number.

My phone rang on Sunday morning (overtime?).  It was the nurse from the physician's office.  She wanted to know why I had said that I would pick up the prescription at an out-of-state mail-order pharmacy.  Perhaps she was concerned that I'm losing my marbles.  She clearly had never looked at the prescription renewal form that has "I will pick up at pharmacy" precoded.

That transaction that should have been extremely efficient was much more complicated and expensive than it needed to be, and it certainly wasn't patient-focused.

The problem wasn't a doddering patient.  It was a poorly designed form and a healthcare deliverer who wasn't familiar with it.

I wonder if anyone acted on that experience to fix the problems... or will they leave that to the customer satisfaction survey people?

Saturday, March 23, 2013

It's Still Nuts

About a year ago after having received some chest-thumping appeals from multiple ALS organizations, I wrote this blog entry about how nuts it is that we are kept so uninformed about our ALS research investment portfolio.

A few days ago I listened to a  compelling segment on The Diane Rehm Show regarding choosing effective and efficient charities.

We need to be able to take a serious look at our ALS research investment portfolio, and that can't be done with the selected and selective bits and pieces of information that our ALS charities and our government agencies give us today.  Take a look at some examples --

ALSA
ALSRP
ALSTDI
MDA
PRIZE4LIFE
PROJECT ALS


The investors are left pretty much in the dark about the business basics -- who gets how much, start dates, end dates, and outcomes.  The investors simply aren't given essential information to make informed decisions about the investments they are constantly asked to fund.

Organizations, please don't fly off and slap together competing, duplicate tools to give us this information.  That would be neither effective nor efficient.  We need a single, common tool that every grantmaking ALS organization and government agency keeps current.  It simply needs to include things like we see today on your websites, but with dollars granted, start dates, end dates, and outcomes.  We also need more than active projects.  The history is extremely valuable.  It all needs to be searchable, shared, and accessible from your websites.

We need a common tool that tells your investors the common-sense basics.   You need to make the commitment to keep the content current.  Your investors are tired of being kept in the dark and fighting through scattered bits and pieces of information and your 990s.

One tool...  One way to show that ALS organizations are efficient and effective at spending ALS research dollars... One place to shine some light for your investors.

If you want us to believe that you can figure out a complicated disease like ALS, then you can surely solve this simple business problem for us.  It would be efficient and effective, and that's what we expect of you.


Sunday, March 17, 2013

Perhaps Some Smart, Energetic College Students Could Solve Some Problems

For over a decade military service has been associated with increased risk of ALS.  Yes, over a decade.

We have some big problems now.  Perhaps some bright and creative college students could find some solutions.


Marketing 101,  Political Science 101
The message about ALS in the military has not stuck.  Every year advocates go to Washington and talk with legislative staff members.  They use the story of ALS in the military as a logical lead-in to get the listener's interest and commitment for some modest DOD research investments.  Every year the staff members and legislators looked surprised and act interested.  Every year.  Year after year.   ALS in the military should be more than a quickly forgotten factoid.  This should be a source of national outrage and meaningful action.

Communications 101,  Instructional Design 101
Veterans with ALS are entitled to service-related disability benefits. Period.  Parts of the VA system didn't seem to get the memo. Stories of veterans who have difficulty getting the help to which they are entitled continue.  Time is important when you have ALS.  Energy is precious when you have ALS.  They need a low-hassle VA that delivers for them efficiently.

Pre-Med 101
Veterans continue to report having difficulty getting basic medical services related to ALS.  Bi-Pap and DPS are allowed by insurance and Medicare for civilian ALS patients.  Why would there be any question for veterans?  Civilians with ALS have access to one-stop clinics where they can see a number of specialists.  It's actually quite efficient and effective.  Why don't veterans have access to such healthcare.

Management 101
Consistency with handling of veterans' benefits often doesn't happen until an appeal is filed.  That wastes precious time for veterans dealing with the ticking ALS clock.  That wastes tax dollars.  Why can't the VA get things right the first time, every time?

Social Work 101, Common Sense 101 
We all know that military families carry a large part of the burden of military service, yet we as a nation often forget when it's time to actually do something.  When many soldiers get back home, the healing for the whole family begins.  For those with ALS, it's a different story.  Dealing with ALS is huge.  It's the most physically and emotionally difficult job that you can imagine, and spouses and children rise to the role; however, we need to give them some help.  That's just common sense.  When a young Army officer forced into retirement by ALS has to open a Paypal plea to friends and strangers to get some overnight caregiver assistance for his family, something is very wrong.  Much is revealed about the character of a nation in how it treats those who have kept its people safe.


Are there any bright students out there who can swarm on a project and come up with some clever solutions to problems of a bureaucratic morass?  We need you.

Friday, March 15, 2013

We Have Ten Days

The FDA has asked the public for comments related to drug development and ALS.  So far very few have taken them up on their offer to listen.

We need everyone who has ever lost a loved one to ALS to speak up.
We need everyone who has ever donated a dollar to an ALS charity to speak up.
We need everyone who has ever been employed in the battle against ALS to speak up.
We need everyone who has ever volunteered for an ALS organization to speak up.

Around 60 people gave their six-minute comments in person to the FDA on February 25.  Only 393 more have submitted comments to the FDA online as of today.  That is just not enough. The FDA is bombarded with requests every day.  We need to make sure they know that ALS needs to move to their front burner.

Don't know what to say?  It doesn't have to be technical or fancy or long.  Ask the FDA to listen directly to patients and caregivers.  Ask the FDA to be willing to let people with ALS take some risks.  Ask the FDA to help come up with some clever ways to move their approval process faster for ALS therapy candidates.  Ask the FDA to let us know what they need to do a better job for ALS.  Simply remind the FDA that the status quo is not acceptable.  Let them know that they're protecting people with ALS to death.

Please speak up.  Nobody speaks on your behalf as well as you do yourself.

Here's the link --

The deadline is March 25.

They have asked.  It would be a crime for us to fail to respond.

Sunday, March 10, 2013

Monday Morning Quarterbacks, Where Were You When The Trial Was Designed?

It's easy to criticize clinical trial design after the fact.

We need smart input up front from more stakeholders, including people with ALS and caregivers.

We need open standards for data sharing.

We need more smart statistical eyes on the data.

You know what they say -- If you're not part of the solution, ...

Friday, March 8, 2013

Meanwhile, Back At The Ranch

On Monday, February 25, dozens of people with ALS and their caregivers got themselves to the FDA for the historic full-day hearing on ALS.  Dozens of scientists left their labs to speak to the FDA, too. Our ALS not-for-profits pulled out all their stops to be there.  For a full day, stakeholders gave persuasive and urgent messages to the FDA on the need to speed up drug development for ALS.  The FDA panel listened for almost eight hours.

There was only one "industry" speaker that day, the CEO of a self-described "baby biotech."  He asked for specifics that would help him bring his company's candidate to and through clinical trials.

It was disappointing that there were no others from industry who chose to speak that day.  At the time I thought that there were probably good reasons since they are regulated by the FDA and I'm sure they need to be careful about what they say to a regulator.  There are some pharmaceutical companies who have put significant dollars into bringing an ALS drug to market, and we are grateful for that.  In addition, there are a lot of reasons why making drug development and approval for ALS would be a wonderful pilot project for a lot of disease areas.  We wish they had spoken, but at the end of the day, their actions will be more important than words.

Then came yesterday's bombshell from Bloomberg News.  On the very day we were at the FDA, a big lawsuit was being filed...
http://www.bloomberg.com/news/2013-03-07/biogen-asked-in-suit-to-return-als-biosamples-to-knopp.html

That could explain some reticence to talk at the FDA.

I have no idea who is on the right side of the law in this matter, but I do know that it will slow down the very processes that dozens of ordinary people fighting an extraordinary foe begged for the FDA to speed up.

"See you in court," is not a good drug development strategy from the patients' perspectives.

Please, parties in this suit, sit down in a room and figure out what is right quickly.  There are two quickly ticking clocks -- your attorneys' billable-hours clocks and our patients' ALS clocks.  I'm guessing that the attorneys aren't nearly as sensitive to the latter as they are their own.

If this drags on to a trial, let's fill the courtroom with people with ALS.  It's all about the patients, right?

Monday, March 4, 2013

Think Pink Ink

"The Pink Sheet" is a trade publication for pharmaceutical companies that commands significant attention.  Back in the olden days, the sheets were all pink and were routed around pharma corporate offices with routing slips.  Publications were of a variety of colors back then.  One's inbox could look like Monopoly money, but you always reached for the pink ones when they arrived.  They contained insider news that was pertinent.

In the last month there have been two large articles in "The Pink Sheet" revolving around ALS.  That's huge, if only to have a big unmet need in front of those who can allocate resources to pounce on an opportunity.

Take a look --
http://www.elsevierbi.com/search?q=amyotrophic

Yes, it's an expensive read.  If you know anyone at a pharmaceutical company, you might get them to loan you an e-copy (please make sure that they read the articles and tell all of their associates about them, too).

The fact that ALS is at last getting the attention of the pharmaceutical industry is all good.



March 4 11am EST - LATE FLASH!
ALS appears in "The Pink Sheet" yet again, with a thorough recap of last week's FDA hearing on ALS. Wow.

Sunday, March 3, 2013

Fool Me Once, Shame On You

Fool me twice, shame on me.

There's not much time between an ALS diagnosis and death.  Nobody with ALS has a long memory of things ALS.  They can be fooled.  Yes, they are bright, but they are thrown into a nightmare and are on a search for hope. They and their families are facing mortality in an immediate kind of way, and they want to trust. 

It's easy to stop looking when you hear what you want to hear.  Besides, who can blame a person with ALS for wanting to try things?

For a person with ALS, fool me once, and you won't have a chance to fool me a second time, but there will be somebody new to listen and be trusting and have a chance at being fooled that first time again.  And so it goes, over and over.

There are no good answers with ALS.  From one who has a long memory of things ALS, here are some thoughts for those searching for hope --
  • Google is your friend.  Go through old web pages online along with message boards and read them thoroughly.  It will take time.  It will be painful.  Don't stop reading when you hear what you want to hear.
  • www.waybackmachine.org is your friend.  Look at websites in their past states.  That helps clear the smoke in case you are being told some revisionist histories today.
  • Talk to people who have been around awhile.
  • Use online forums to investigate.  Sure, everybody has an opinion, and you don't have to agree with everyone, but there are people with longer memories than you who can be insightful and helpful as you make decisions.  If anyone belittles ALS message boards, remember that the advice there costs you nothing and there may be someone there who can help you with some point-counterpoint.
  • When you try something, please share your data and experiences in a public place such as the patient profiles at www.patientslikeme.com .  It should raise a big red flag if you are not encouraged to share publicly.
It's often hard to look back through a rear-view mirror, but we all know that things aren't always what they appear to be.






Saturday, March 2, 2013

You Know What They Say About Squeaky Wheels

Only 255 comments have been submitted so far at

How many people are board members of ALS organizations?
How many people are employed by ALS organizations?
How many people raise funds for walks and events?
How many people volunteer for PALS and ALS causes?
How many people blather on online about ALS?
How many people have lost loved ones to ALS?
How many people have lost a co-worker or neighbor or a member of a church to ALS?
How many people sell goods or services to ALS organizations?
How many people work at ALS clinics?
How many people care about people with ALS?
How many people care about the next person to get ALS?



OK... We need for EVERYONE to mobilize and make comments to the FDA. It can be as simple as what so many people said on Monday -- The status quo is unacceptable.

If all the FDA hears are the voices of 60 people on Monday and a few hundred online, they won't feel the heat.

Please, it's not our ALS organizations' job to speak up for you. It's not your walk team captain's job. It's not your supervisor's job. It's not somebody else's job. It's YOUR job. Please submit a comment. Democracy works, but you know what they say about squeaky wheels.

Thanks. Just as you take care of your own oxygen first on the airplane, please make your own comments first, then help spread the word.  Please squeak the FDA into action.

http://www.regulations.gov/#!docketDetail;D=FDA-2013-N-0035