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.

Thursday, June 18, 2020

Just A Little Bit

Respect.

In the past two years I have been fortunate to meet and work with some remarkable people with ALS and caregivers.  They are smart.  They work hard.  They study hard.  They listen and they speak up.  They have found an important way to contribute more than their money and their friends' money.  They contribute their creativity and their time and their thoughtful talents within I AM ALS.  I sit in on Zoom with them almost every day. Their books aren't on a shelf behind them.  They open them and read a lot.  I, a healthy person, accomplish a small fraction of what they are able to do to move public policy and industry.  They are a refreshing bunch who seem to be moving gigantic barriers that have deep footings in the fight against ALS.

Yesterday the ALS Association issued an official statement regarding H.R. 7071, a bill to provide some funding for small biopharms for Expanded Access Programs and in addition would establish an FDA Center of Excellence for Neurodegenerative Diseases, modeled after what was done for oncology several years ago.

The ALSA statement got off to a condescending start by calling H.R. 7071 "well-intentioned" before providing a short litany of what I consider to be off-target objections.

Respect my I AM ALS friends, please.

They have studied and worked hard to make Expanded Access Programs an expectation for all clinical trials.  One barrier has been a lack of planning by small biopharms that raised their capital four or five years ago.  The funding in H.R. 7071 is intended to help bridge that gap.  It's not forever.  By 2025 small biopharms will plan for EAPs as they raise capital because they will have figured out that they can't afford not to.

You see, EAPs can actually help speed up their paths to approval.  It gives them a chance to gather informative data on a broader population of people with ALS (beyond the narrow groups they pick  with best chances to be statistically successful).  The data they can gather are priceless.

There seems to be misunderstanding among some about the purpose of the legislation's funds.  They are not intended to spread unproven treatments to as many people as possible like expensive experimental peanut butter.  They are intended to provide a lifeline to small biopharms so that they can provide responsible EAPs.

And lest we all become hung up on arithmetic exercises, remember that in an EAP the sponsor is allowed to recover at most the cost of product, and typically the products are provided gratis for good business reasons.

Open Label Extensions were mentioned in the ALSA retort as being preferable.  It's not either-or, folks.  OLE should be demanded of every clinical trial design, and it is being demanded by my friends at I AM ALS. I'm glad that ALSA has finally decided that OLEs are important, too.

At More Than Our Stories in February, we had a long, and spirited discussion on such public funding for EAPs.  There was legislative, policy, and research expertise involved in the discussion, including some who authored H.R. 7071.  We talked about the importance of not doing whack-a-mole with other important NIH funding.  We talked about new sources of funding raising the tides. We wrestled with supporting small biopharms versus all sponsors.  Honestly, in my opinion, the ALSA concern that the EAP funding might detract from other research funding is specious.  We need to stop acting like we'll eat each other's lunch.

For years it has been clear that "the oncology FDA" has been more forward-thinking about using all available tools to speed development, approval. and access to therapies than "the neurology FDA."   It's like two different FDAs.  If you search for the use of Expanded Access Programs, you'll find plenty in oncology.  You're lucky to find one or two in ALS.  To ask for a FDA Center of Excellence to get things done for neurodegenerative diseases they way they get things done for oncology is hardly the bureaucratic threat that ALSA, an organization that itself is intimately familiar with bureaucracy, describes.

I ask you to pay attention to the people with ALS and caregivers of I AM ALS who are getting things done.  I ask you to respect them for the smart and hard-working and thoughtful people they are.  And I ask you to help them to move public policy forward.

Thank you.



Monday, April 6, 2020

How to Get Nowhere

Ask us.  We know.

Consider an experimental drug for a quickly fatal disease.  One person says it's the greatest hope ever.  Another person says we shouldn't accept it without sufficient data.  Meanwhile, back at the ranch, people are dying while forces row like crazy in exact opposite directions.

Yesterday I saw the President again tout one experimental treatment for Covid-19.

On the same newscast. I saw the head of the American Medical Association say that she would not prescribe it off-label for her patients.

That's it.  Hatfields versus McCoys.  Right versus wrong.  Rowing like crazy in opposite directions.

Neither mentioned joining a clinical trial.  

That's a way to access a therapy that provides exactly the evidence that we all need to figure out whether the stuff works (and for whom).

Rather than rowing in opposite directions, how about promoting the clinical research that can can both provide the experimental therapy to people and help us reach the scientific truth?

And what if the trial isn't near enough people?  There are easy ways to fix that.
And what if the placebo is an issue for some people?  There are easy ways to fix that.
And what if people are too old or too ill to qualify for the trial?  There are easy ways to fix that.

If people would stop arguing and start promoting clinical research that is humane and informative and nimble, we would start getting somewhere.

Ask those in the fight against ALS.  The constant tension between well-meaning forces has gotten us nowhere.

There are ways to do clinical research that will fulfill the goals of both parties who today are rowing in opposite directions.  Both sides need to smarten up if we want to get somewhere.  It applies to Covid-19.  It applies to ALS.

Sunday, February 2, 2020

There Ought To Be A Law

Or should there?

The lack of access to investigational treatments for people with ALS is unconscionable.  The problem isn't new.  We need to fix it now.

What's the best way to fix it?

A law?  More laws?  Fewer laws?  Fewer regulations? More regulations? Public pressure?  Smarter business strategies? Calls to conscience?  Public funding? Private funding? A toolkit of  some of these?

There is a flurry of new legislation currently being proposed to fix various aspects of the problem.

A lesson I learned from the last law intended to fix the problem is that the devil is in the details.  We need to know a lot more than the good intentions and a great name on proposed legislation.

Do we really need a law?  Exactly how would it work?  Examples, please. And details.

Conditional Approval

A proposal that is most interesting to me at the moment is the one on Conditional Approval.

https://www.braun.senate.gov/senator-braun-introduces-conditional-approval-act-patients-fatal-diseases-asks-comments

Senator Braun has provided both the full text as well as an opportunity to comment.  I am grateful for that since we need to polish these proposals as much as possible before moving them forward as legislation.

I have more questions than comments, but I hope that they will be helpful and I know discussion of the answers will enlighten me.

Suppose a therapy is in a Phase 3 trial and the sponsor had intended all along to file for full approval. When the trial is complete and the sponsor sees the data, the sponsor isn't sure that the FDA will be enthusiastic about a broad, full approval.

  • Can the sponsor then ask for Conditional Approval when filing trial results with the FDA? 
  • Can the sponsor suggest the confirmatory studies and data that will be supplied in the future as part of the conditions?  Or is that all up to the FDA?
  • If a sponsor had decided to go ahead and ask for full approval, could the FDA respond with a suggestion that Conditional Approval would be more appropriate?  Or must the sponsor always initiate the request without any prompting from the FDA?
  • Could the FDA suggest confirmatory studies today (without this legislation) after viewing an application for full approval?  Do we need a law?
  • Does the FDA have the resources and teeth to enforce conditions on a Conditional Approval?
  • Are there any precedents for the FDA to accept RWE as part of a full approval?
  • Are there FDA guidances for quality-assurance of RWE?
  • Do we know how government payers might approach covering a Conditionally Approved therapy?

Suppose a therapy is a repurposed drug with full approval in another country.

  • Could Conditional Approval give the FDA a tool to force some confirmatory studies with a US population? 
  • Might Conditional Approval help us find the scientific truth on responder subgroups, making the drug a lot more useful and affordable for people in the US with ALS?

Conditional Approval has worked in Europe.  That's encouraging, but the payer situation is very different there.

Conditional Approval has worked for veterinary products.  That's encouraging, but again, the payer situation is very different.

Can we make this concept work?  I hope we can if it can bring therapies to people with ALS sooner.

And as important, it seems to me that this could give us a way to get to scientific truth without giving broad approval to therapies that work only for subsets of responders.

Thanks to anyone who can advance the discussion.  It's important that we don't stop at the title of the legislation.