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.



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