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.

Monday, February 22, 2016

Time To Speak Up and #CutRedTape !

The United States Senate Committee on Homeland Security and Governmental Affairs is having a hearing on Thursday, February 25, on "Connecting Patients to New and Potential Life Saving Treatments."

http://www.hsgac.senate.gov/hearings/connecting-patients-to-new-and-potential-life-saving-treatments

It is significant that this committee's hashtag is #CutRedTape !

You are encouraged to submit testimony to the Committee via Josh McLeod josh_mcleod@hsgac.senate.gov .  Even one or two sentences on the topic will help.

My submitted comments follow:
February 20, 2016

Senator Ron Johnson
Chairman Senate Committee on Homeland Security and Governmental Affairs
328 Hart Senate Office Building
Washington, DC 20510

Regarding the February 25 Hearing – Connecting Patients to New and Potential Life Saving Treatments

Dear Chairman Johnson,
 
Three years ago to the day of this hearing, the FDA conducted an all-day hearing on ALS.  Dozens of people with ALS and caregivers made a difficult trip to Silver Spring to testify.  Dozens of scientists and organizational representatives testified, too.  It was a day when FDA officials sat face-to-face with dying people who were begging for the right to take some risks.  It was a day when caregivers gave the FDA some concrete suggestions for changing the drug approval process to save their loved ones.  It was a day when a mother of a young man with ALS asked that this not turn out to be a dog-and-pony show.  Three years later, it is clear that it was nothing more than a dog-and-pony show. 
Three years. 20,000 American funerals later.  A half million global funerals later.  We all deserved more than a dog-and-pony show. 
I realize that the FDA has a huge responsibility to keep Americans safe. I appreciate that.  They would rightly feel blood on their hands if something were approved that did more harm than good.  Why don't they feel the blood on their hands today when people die from ALS, protected to death from experimental drugs?  Why don't they feel the blood on their hands today when people with ALS die from the FDA-approved morphine that makes them more "comfortable" in death? 
A basketball game is played differently in the last 60 seconds, especially when you're behind.  The last two minutes of a football game can be a magnificent display of adapting to the situation.  Yet our game-planners for drug access and approval have one playbook.  Today the drug approval process for a simple therapy for a relatively healthy child is substantially the same as for a possible drug for that child's parent dying from ALS.  No Hail-Marys are allowed for that child's Mom or Dad. That's just wrong. 
I personally think that Right-to-Try laws are not the answer.  We need a strong FDA at the center of drug development.  We shouldn't be building networks of drug access that only work for people of substantial means.  Yet those Right-to-Try laws have brought an important conversation to this hearing, and I am grateful for that. 
The Legislative Branch has provided the FDA with a path of Accelerated Approval that is designed to let them change the game plan for terminal, unmet-need diseases.  Why doesn't the FDA use it for ALS?  Is it liability?  Is it concern over payers (which I don't believe is the responsibility of the FDA)?  What is the problem?   
And please don't try to tell us that Expanded Access Programs are the answer.  They simply aren't viable when the entire market for a new drug is the unmet-need disease and the science is dreadfully expensive.  They don't work for ALS.  We need to encourage drug developers, not discourage them. 
We learned at that FDA hearing three years ago that drug developers are frustrated because they don't get to collaborate with the FDA.  They submit. The submission is accepted or rejected. If it's the latter, they have to start over.  Are drug developers afraid to propose new ideas for fear they will be rejected?  
I'm a Boomer.  I'm old enough to remember when we all had access to drugs that had just been tested for safety but not for efficacy.  As an adult today blessed with good health, I appreciate the FDA changes of the 1960s that require efficacy testing of the drugs my doctor prescribes. But as the daughter of a woman lost to ALS, I also know that she should have been given a chance to try something -- something that may or may not have helped her.  Something she could have accepted with eyes wide open about risk.  Something that could have advanced the science more quickly for another mother. 
Last year Gregg Doyel, a talented sports journalist for the Indianapolis Star, wrote a column about his friend, Maureen, who was dying from ALS.  She was a beautiful human being.  She was a mother and wife.  She wanted access to try something.  She wanted a chance at a Hail Mary.  Gregg's article ended with some words that I hope stick with everyone in this hearing room.  Actually, I hope they sting, too.  -- "For god's sake, you coldhearted bureaucrats, they're dying."


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