Posted on 07/03/2019 9:01:36 AM PDT by SeekAndFind
Americans suffering from debilitating diseases should not have to lobby Congress or Food and Drug Administration (FDA) bureaucrats for special permission to access potentially lifesaving treatments, yet that’s the situation many patients are in.
Consider Jaci Hermstad, a 25-year-old Iowan who suffers from a rare form of ALS. Hermstad has been fighting for her life for months, but has found hope in a groundbreaking molecular therapy developed specifically for her. It seems like common sense that Hermstad should be able to access this innovative, highly specialized treatment before it is too late. However, in the upside-down world of the FDA drug approval process, common sense no longer applies.
It takes, on average, 12 years and $2.9 billion to bring a drug from lab to market. For thousands of Americans, this is far too long.
Even though Hermstad’s life hangs in the balance, FDA’s drug approval process is not currently able to test and approve medicines quickly enough for the new molecular therapy to matter for Jaci. Until just weeks ago, it looked as though Hermstad would almost certainly die while waiting for the FDA to grant her permission to access a procedure that could save her life.
Fortunately for Jaci, she has received overwhelming support from the public and Congress with a recent bill introduced on her behalf, H.R. 2855. This legislation, called “Jaci’s Bill,” would provide access for Hermstad and suffering patients like her to seek molecular therapy by way of a compassionate use request.
The pressure put on FDA by Congress and the public has helped her story reach thousands of people. Thankfully, in the wake of public demands, the FDA has announced it will allow Jaci to access the medicine that could save her life.
However, not everyone is so lucky, and it’s unrealistic to expect every person suffering in pain to create a vast public campaign resembling the effort that led to Jaci’s victory. No patient should be forced to fight for access to a treatment he or she should have every right to access in the first place. Jaci is not the first or the last American to be diagnosed with a terminal or debilitating disease.
Thankfully, there is a public policy solution that would streamline the antiquated drug approval process: Free to Choose Medicine. Under a Free to Choose Medicine track, patients would have the ability to access potentially life-saving medications—at the discretion of the drug manufacturer, doctor, and patient, not FDA bureaucrats.
Free to Choose Medicine would allow thousands of patients to access needed drugs sooner and at a lower cost, all while protecting patients’ right to pursue medications that could improve or save their lives.
It is truly a blessing FDA reacted positively to the public outcry about Jaci’s story, but what about next time? What about the thousands of others suffering while they wait for FDA to act? We must press for reform now, before it's literally too late for dying patients.
The article does not say whether the molecular therapy worked for Jaci.
I nor any member of my family will ever sue anyone if this drug proves to be fatal.
Sign the above statement and go for it.
Shouldn't matter, IMO.
It may take months for the therapy to work.
The "freedom to choose" is the important point.
A few years ago Congress passed something allowing sick people to import non-FDA approved medication. That was a major change from before.
I thought that was sufficient, unless they repealed it later.
Why do we need anything additional, except to make sure US customs does indeed conforms to it.
Sorry, I posted before reading the whole thing.
Now I see that this is about medications that may still be considered experimental, not medications available and imported from other countries.
This should also pass, and leave it to the doctor and patient, at least in real severe situations.
[[Sign the above statement and go for it.]]
Problem is some smarmy lawyer will declare something like “They were under duress due to their illness, and couldn’t make an informed decision as a result, therefore the experimental drug makers are to be held responsible for the death”
You’ll see commercials on Tv saying “Have you or a loved one experienced (fill in the blank) as a result of taking the Drug (fill in the blank) during their trial period? If so, contact us immediately, You may qualify for compensation”
We are running into similar issues of getting Medicine approve by our primary Insurance Company. There no drug approved for Adenoid Cystic Carcinoma of the Trachea by the FDA . The tumor is non-operable due to location at the bottom of the trachea into the left/right main stem bronchus. They prefer to remove to tumor for treatement but there is no replacement tissue for this location.
We did some genome profiling to identifying any existing cancer drug that might work on this tumor. Luckily we have Tricare that will waiver us to use this drug off label.
Without Tricare we would have to pay for the drug out of pocket. Very expensive drug I price it at $92,0000 two years ago, now down to $18000 a year since President Trump took office. If it was not for Tricare we would be flying to Canada every 90 days.
Note: The drug would only run me $350 for a month supply in Canada.
The tumor has stop growing after a year on the drug, ACC will do this sometimes. We are not sure if is working, if not we will be trying another drug and Radiation seed.
Informed consent is the way to go but, like a lot of things in life, the quacksters and con men will creep into this field offering quake cures that will eventually kill or, at least drain the resources of those so afflicted.
Caveat emptor!
Disclaimer: Opinions posted on Free Republic are those of the individual posters and do not necessarily represent the opinion of Free Republic or its management. All materials posted herein are protected by copyright law and the exemption for fair use of copyrighted works.