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."