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.

Sunday, May 31, 2015

We Need A Webinar - The Business 101 of Clinical Trials

It's confusing.  It's a business.  We all need to be better consumers.  I think we have a big information gap that is just crying to be filled.

Clinical trials are critical to finally finding a treatment for ALS.  We all know that they are expensive, but how expensive are they?  And who pays whom to do what?  And how does the whole business process work?

If we understand the roles and the business and responsibilities and compensation better, every patient, caregiver, donor, and advocate will better understand how and why things are done.  And with that, we'll all be able to set more realistic, responsible expectations for change.

Some things that I would love to understand better --

  • Who gets paid to design clinical trials?  How do they go about it?  How does a budget play into trial design?
  • Who draws up all of the consent forms for a trial?
  • Are clinical trial sites usually paid per patient?  Are they still paid when patients drop out?
  • Why can patient travel reimbursements vary from site to site for the same trial?
  • Who is paid to be the keeper of the data?
  • Who is paid to be the analyzer of the data?
  • What is a CRO?  
  • Do for-profit CROs differ from not-for-profit CROs in what they do or how much they charge?
  • Are investigators paid individually or are their institutions paid to conduct trials?
  • Is any drug developer willing to share with us how much an actual ALS clinical trial cost both in internal expenses and in contracted services?
  • Do charitable donations play a role in clinical trials for drug candidates that are owned by private companies?
  • Do you have to pay fees to the FDA if you are conducting a clinical trial?

I obviously have  lot to learn.  Many of us do.  I hope someone will be able and willing to educate us so that we can be better informed.  Clinical trials and drug approvals are a hot topic right now, and some basic business information will certainly raise the quality-bar in the discussions.

Thanks to whomever will help teach us the Business 101 of Clinical Trials.



Monday, May 25, 2015

Meet Two Jimmy Clarks

Meet Jimmy Clark

Yesterday at the Indy 500 they celebrated the 50th anniversary of Jimmy Clark's victory with a pre-race lap of his beautiful winning car.  Jimmy Clark was special.  We called him "The Flying Scotsman." He wasn't flashy.  He was just fast. He looked more like an accountant than a race car driver, but he could drive. He used new technology to chase his dreams of winning.  He assumed risk with eyes wide open and he achieved great success in all kinds of racing.  We applauded his many victories and mourned when we lost this great talent and gentleman in a racing accident in 1968.


Meet Jimmy Clark

In March at the first Rally for Faster Drug Approval I met another Jimmy Clark.  He is special, too.  He is loved by his family and loves life.  He also has ALS.  That puts a bad wrench in things.  All he asks is that he be allowed to assume some risk with eyes wide open so that he might chase his dreams, too.  He wants to be allowed to take risks that are minuscule compared to those that are taken every weekend by those who participate in auto racing.  He wants to advance technology and science just as race car drivers are allowed to do.  He also wants to live for a family that is far more important than a race or a trophy.  This gentleman simply wants us to let him try an unproven, investigational drug with a good safety profile.

Why not, I ask.  Why not?  Where are our priorities?







Friday, May 22, 2015

Your Hands Aren't Really Clean

You don't want blood on your hands.  Nobody does.

If someone were to die because of an allowed, yet unproven ALS treatment, that would leave blood on the hands of the FDA.  Nobody at the FDA wants that.

IF someone were to die from untreated ALS, that's an act of God, right?  No blood ends up on anyone's hands, right?

Wrong!  It's on all of our hands.

Not acting doesn't wash our hands of responsibility to try to stop deaths from ALS.

Finding excuses and reasons to delay does not mean our hands are clean.

Regulators, scientists, advocates, physicians, and bloggers share a new permanent stain with every life lost to ALS.  Our hands are not cleaned by inaction.  You can't wash it off by letting ALS take its course.

Saturday, May 16, 2015

Just Read The Transcript

On February 25, 2013, dozens of people with ALS, caregivers, scientists, drug developers, ALS
organization employees, and clinicians made a trip (many at their own expense and with a great deal of difficulty) to testify to the FDA.

They gave a gold mine of ideas for speeding up drug development and approval.  It's a big gold mine because people testified all day in six-minute segments.

They talked about risk, biomarkers and endpoints, trial design, FDA processes, natural history, controls, and on and on.

Today we hear that the FDA guidance document that was indicated at that meeting is only now being started and that dozens of people with ALS, caregivers, scientists, drug developers, ALS organization employees, and clinicians will soon be convened to give their input.

Ok, everybody.  How about just reading the transcript from that FDA hearing?  You have the materials to start a draft FDA guidance document right under your noses.  Let's get moving.  Spending buckets of money on unnecessary meetings isn't a sign of an understanding of urgency.  Nearly 300,000 more people have died from ALS since that hearing.  Urgency?

You can watch.
http://www.fda.gov/Drugs/NewsEvents/ucm339833.htm

You can read the whole thing.
http://www.regulations.gov/#!documentDetail;D=FDA-2013-N-0035-0271

And you can read the FDA's summary and reaction.
http://www.regulations.gov/#!documentDetail;D=FDA-2013-N-0035-0272

As an example, one of the ideas presented that day is that people with ALS have a right to know who is doing what during the clinical trial and FDA back-and-forth in the approval process.  Are we waiting for statisticians?  Are we waiting for an FDA decision?  Are we waiting for more data to be submitted?  What's going on?  Are we waiting for somebody on vacation?  Think about how much more clearly the Accelerated Approval application for GM604 might be perceived if we simply had this kind of transparency policy.  Any successful modern business has project teams that know what's going on in the scrum today, yet the veil of secrecy continues to obstruct the view of the most important people in the drug development and approval process -- those with ALS and their caregivers.

And there are hundreds of more examples of ideas that can have a direct impact on speeding up drug development and approval.

Just read the transcript.


Thursday, May 14, 2015

Some Stream-of-Consciousness Thoughts From #ALSAdvocacyDay2015


In no particular order, I reflect on my short trip to Washington, DC, this week.

  • For the first time ever, I'm grateful for being held captive on a plane, waiting for a jetway.  My Senator across the aisle could not have been more gracious and concerned as we talked about ALS, drug approvals, and the FDA.  Thanks, USAirways.
  • The Rally for Faster Drug Approvals held by www.HopeNowForALS.org did not disappoint -- enlightening speeches, thoughtful PALS and CALS, good information on FDA and drug approvals.  It was hot, but the PALS, CALS there did not wilt.  And there were signs and cameras, and heads on the street turned.
  • A sign taught me that "We are the change that we have been waiting for." I love it.
  • The evil ALS continues to pick some remarkably talented individuals and families.
  • Double-blind, placebo-controlled trials don't need to be the "gold standard."  Shoot, the gold standard is no longer the "gold standard," a PhD economist with ALS explained.
  • You listen to the SGD, but you look at the PALS delivering a speech, and it's amazing.
  • Open and diverse discourse can teach us all new things, and God knows, we need new ideas.
  • The status quo is simply unacceptable.  Same old same old isn't good enough.
  • Denial of hugs is one of the big evils of ALS.
  • Bragging rights should not be an unspoken priority.
  • PALS, CALS are their stories and so much more. So much more.
  • The Abigail Alliance has a the most refreshing IRS Form 990 that I've ever read.
  • The ship's photographer can be annoying in a legislative meeting.
  • Advocacy is year-round work.  Many of us take that seriously. All the more reason to give advocates a heads-up when a HR number is changed and prior co-sponsors need to re-up on the new HR. 
  • Legislators are very frustrated with the FDA, too.
  • I love Hoosiers.
  • Dormant Therapies provision is important in 21st Century Cures, but there is a lot more than needs to be in there that will have a direct impact on getting therapies to PALS faster.  Is anyone looking out for PALS?  Really looking out?
  • We all don't have to agree.
  • What do those ALSA people who randomly morph into your legislative meetings really do?
  • Money doesn't buy a great advocacy experience.
  • A chance encounter with your other gracious Senator on Pennsylvania Avenue can cap off a good day of advocacy.  I'll always remember talking about 21st Century Cures with him directly across the street from where I held a sign at the Rally for Faster Drug Approvals a day earlier.
  • My favorite hashtag for the fight against ALS is #notquietly . 




Monday, May 11, 2015

We Need To Put The Sign Back Where It Belongs

Quickly.

A year ago we talked to legislators about some Medicare rules being implemented that were a crazy trip back to 1980.  They were denying people with ALS access to modern, economical technology related to speech generating devices.  When we talked to legislators and staff members, they were 100 percent in agreement that the rules were antiquated and wrong.

That was a full year ago.  There has been lots of talk and there have been band-aids, but we learned that we need a law.  The buck can't stop where it belongs for Medicare recipients unless we make a law.  Today people with ALS are denied or lose the very devices that let them be productive and engaged parents, citizens, and patients.

Enter a small charity that couldn't let those people with ALS lose the purpose, productivity, and joy
that the simple ability to communicate provides.  They let the buck stop with them because the need is vital.  In fact, they have spent over $1.4 million bucks in the last few months supplying enabled devices to people with ALS that Medicare should have been providing.  Perhaps the fact that this organization is led by a young man with ALS who uses this very technology is the reason he decided to help in a substantive way... rather than pass the buck.

Charitable contributions should not be expected to cover for a broken government rule.  Government should not be allowed to pass the buck.

But we need a law. Now.

Please write your Member of Congress. Today.  Demand quick support of the Steve Gleason Act.  Here is information and a convenient tool to submit your thoughts --
http://www.medicareadvocacy.org/take-action/

In an ultimate twist of irony, President Johnson signed Medicare and CMS into existence at the Truman Library in 1965.  They should have paid more attention to the sign while they were there.







Sunday, May 10, 2015

Before We Ask For Another $10,000,000.00 For The ALS Registry - Question 2

Which is it, $1.1 million or $10 million?



In the 2013 OMB filing for this project, we see the following:
http://www.reginfo.gov/public/do/DownloadDocument?documentID=396524&version=0
(And a hat tip to the ALS advocate who sent that document to me yesterday.)

14.  Annualized Cost to the Government
Data analysis by ATSDR may result in action taken by the Division of Toxicology and Human Health Studies in response to the required CDC mandate in maintaining preventive health activities and surveillance systems.  The action taken will vary, depending on the analysis.
 The total cost to the federal government for the collection of this information for the three year ongoing project is $3,300,000 as itemized below. Annual ATSDR personnel costs $420,000 Additional expenses will be incurred by ATSDR in order to operate a successful surveillance program/registry.  Four staff will contribute to this program: a Senior Scientist (25% contribution=$75,000), and a program analyst (100% contribution = $100,000)  A contractor will be used to maintain the web portal for case registration and participation in surveys in addition to providing public user support 40 hours per week ($500,000).  Lesser expenses may include computer resources, telephone calls, and recruitment materials (approximately $5,000).
 The estimated annual cost to the government is $1,100,000.

So we're confused.  We ask for another $10 million every year.  Why?

The ALS Registry budgeting process has always been a bit of a mystery.  Last year we learned at the ALSA Advocacy Conference that the original $10 million number was a number that the legislative sponsor thought could be raised. It was not built up from a businesslike budget.  We also got our first glimpse into annual expenditures with some very broad percentage breakouts at that conference --



It would be respectful of those with ALS and caregivers who are going to legislators to ask for another $10 million to know more clearly how that much money would be spent by project component.:

And yes, it continues to be a concern that the advocacy organization that sets the public policy priority that champions $10 million for the Registry has itself been a large, paid contractor on the project.

Why are we asking for $10 million for a $1.1 million project?  More does not mean better.

Thursday, May 7, 2015

Before We Ask For Another $10,000,000.00 For The ALS Registry - Question 1

Next week ALS advocates will be sent to Capitol Hill to ask for another $10 million for another very generous ALS Registry budget.

We have questions.

A PALS described a problem with the first Registry report issued last July -- http://www.huffingtonpost.com/stephen-finger/national-als-registry_b_5620631.html

Q1:  Have any of the local studies that were funded as part of the Registry project shed any light on this significant concern on how large the big "unknown" patient population is?  Are we able to extrapolate how many PALS are likely not found via the Registry's database mining and the online enrollment portal?



Friday, May 1, 2015

These Seats Have An Obstructed View

It's just wrong.  You can't follow a game when you can't see who has the ball.  You can't figure out when to cheer or boo.  It's incredible that in this age of the taxpayer-financed sports palace that fans can be stuck with expensive seats with obstructed views.

Such is also the case with our view of the back-and-forth between Genervon and the FDA.  Who has the ball?

Are we waiting for the FDA to give Genervon something?

Are we waiting for Genervon to give the FDA something?

All we know is that the most important people in the stadium, those with ALS and their caregivers, can see time ticking off on the clock, but they can't even figure out who has the ball.  They have been assigned seats with a conveniently obstructed view.

It's just wrong.