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 14, 2017

There Are Elephants In The Room

A little over a week ago we got the wonderful news that a drug had been approved for ALS in the US.  Since then we have had multiple news reports and webinars and communication from ALS organizations and MT Pharma America about the drug, Radicava (edaravone).

Don't get me wrong.  I ecstatic about having another tool in the toolkit to treat ALS.  There are just these two elephants bothering me.

Elephant 1 - The Awkward Age Of Precision Medicine

This is a drug that was developed in Japan in the late 1980s and was approved for use in strokes there in 2001.  Based on later ALS studies in Japan, it has been used there for ALS since 2015.

I sensed the first elephant right away.  We've been so conditioned of late to the heterogeneity of ALS and the genetic differences that I wondered how people with ALS in Japan differ from those in the United States.  What are the most common ALS genes in Japan compared to the the US? Is there anything in the Japanese data that will help us predict which people in the United States are most likely to respond?

The elephant sat quietly until I listened to Friday's webinar when Dr. Palumbo from MT Pharma America showed a slide modeling comparable blood levels between people of Japanese ethnicity and Caucasians.  I think that showed us that the drug gets to where it needs to be in both groups, but it really doesn't tell us anything about how the groups responded to the drug, does it?

The concepts of precision medicine today constantly drive us to know more about individual cases of ALS and what interventions are likely to work in specific individuals.  New concepts are to know everything possible that distinguish an individual case of ALS.  Can old clinical trial data shed that kind of light? The elephant emerges. How can we figure out which people in the United States are likely to respond (and as important, not respond) to Radicava?

Elephant 2 - The Price

I'm the person in the room who normally defends drug pricing.  New drugs are expensive to discover and develop into commercial products.  As expensive as pharmaceutical products are, they usually provide results that are both cost effective and beneficial to our lives.  I'm pretty immune to drug price sticker shock.

Right after the Radicava approval announcement when some pricing information was revealed, an ALS caregiver asked me what I thought about the price.  I said that I thought if they had a slam-dunk product and could say that people with specific flavors of ALS would receive significant therapeutic benefit, that the extremely high price was probably in the right ballpark.  Unfortunately, the data that we've seen don't make it likely that this is a slam-dunk product for anyone.  It will be something to consider and try, and it may help, and it may not help, and it may be hard to perceive the difference individually.  Elephant 2, meet Elephant 1.

People with ALS will try it and I'm glad they can.

It's a fact that people who are not going to be responders to this product are going to pay the astronomical price, and we don't have data to minimize the size of that group. It's not like Costco where you get your money back later if it doesn't work.

The webinars we've heard have been great about explaining the process of getting payers to pay for new indications.  If it's likely that Medicare will pay for Radicava and its administration for people with ALS, that will be good.

That doesn't mean that we shouldn't talk about the price elephant.  We absolutely should.  It's not harmless for the government to overpay for something, especially when we may be on the verge of more product approvals for ALS in the US.

Over the past couple of years I have gained huge respect for the way DMD parents advocate for approval of therapies for their kids and then for access to those therapies.  We could learn from them about not ignoring elephants.

Tuesday, May 9, 2017

We Need A Toolkit

Dad was an engineer.

He taught us that you always need a good basic set of Craftsman tools, and every tool has its own use.  Never use a screwdriver when you should use an awl.  Never mess up a bolt with the wrong sized wrench.  Never hammer with anything but a hammer.  And duct tape has no correct application in plumbing.

On Friday we got the wonderful news that MT Pharma America was granted FDA approval for
Radicava (edaravone) for ALS.  There is joy in hearing that there is finally a new tool in a barren toolbox.
We can't stop building a toolkit with one new wrench of unknown size.  We need to figure out for whom Radicava works, and we need to keep complementing the toolkit with new options.  This is one tool.  We need that Craftsman set to address a nasty, heterogeneous disease like ALS.

Edaravone has been used in Japan for a number of years for stroke, and it has been approved and in use there for ALS for almost two years.  I acknowledge that this is fast approval by FDA standards, but two years is a lifetime for many with ALS.  We are grateful for Friday's news.  We must forge ahead more quickly and more aggressively.

We have a toolbox to fill with the right tools for the right people.

Friday, May 5, 2017

It's An Expensive Trip To G12.21

Every person with ALS can tell about an expensive journey to get to the diagnosis.

There are millions of dollars worth of wrong diagnoses along the way.  There are tests.  There are surgeries.  There are physical therapies.  There is lab work. There are speech therapies. There are more wrong diagnoses.  There are MRIs.  There are drugs.  There are scans.  There are psychiatrists.  There are more doctors.  There are braces. There are more wrong diagnoses.  All the while, the person whose ALS is not yet diagnosed continues to decline.

Finally, one day, often a year or more after the start of the expensive journey, a physician says, "You have ALS."  G12.21 is encoded on a medical record.

Mom had one of those long, expensive journeys. She declined terribly along the way. She didn't get her ALS diagnosis until just four months before the ALS took her life.  But for the initiative of a sharp speech therapist, the diagnosis may never have happened.

Some cases of ALS are fast.  Most cases of healthcare delivery are slow.  They are even slower for people who don't have good insurance coverage.

How many people die from ALS without ever finding out that they had ALS?  I guarantee that problem will get worse if yesterday's AHCA legislation moves forward.

Monday, May 1, 2017

The Path From Gingerbread to Disrespect

Since the CDC launched its ALS Registry website in 2010, it has been fraught with gingerbread.

Early on, they installed a "Service Locator."  At the time I thought it was a poor decision.  They
already had links to ALSA and MDA sites where chapter and clinic locators are available and kept current.  Why would the CDC want to maintain with its own databases of chapters and clinics?

The Service Locator is on the lower left of the main page at 

Through the years it stopped working for me.  I've asked others to test in their browsers, and nobody I know has been able to make it work for many months.

We went from an unnecessary piece of gingerbread to a broken piece of gingerbread.

People seeking ALS services are typically in the throes of a disease that consumes all of their time and energy.  They don't need to type their zip codes into a trusted site only to find out that nothing happens.

We went from an unnecessary piece of gingerbread to a broken piece of gingerbread that is disrespectful of those who try to use it.

And it's not that hard to make broken gingerbread disappear from a website.