Urge Congress To Support Free To Choose Medicine
My name is Vincent Galbiati. Last year with the guidance of my advisors and board of directors, I started a nonprofit called Tomorrow’s Cures Today. Our objective is to promote a national agenda for speeding the discovery of medical cures and communicate the importance of restructuring the way therapeutic drugs are developed, tested and delivered.
We did this because of heart rending stories like Jenn McNary’s.
Jenn is the mother of two boys diagnosed with Duchenne muscular dystrophy, the most lethal genetic disorder among children. It slowly weakens a patient’s muscles, leading to paralysis and early death. Until recently, there was no cure. But now there is some hope. Jenn’s younger son Max was accepted into a clinical trial, where he received Eteplirsen, a new drug being tested to stop the progression of the disease. Her older son Austin, however, was not accepted into the trial and, because of Food and Drug Administration rules, was denied the right to use the same drug. Max is now walking, leading a normal life, while his brother is confined to a wheelchair and continues to deteriorate. During an interview, Jenn stated the painfully obvious—“Both my sons deserve to live.”
She’s right. No one should have to endure her nightmare.
Austin wasn’t allowed to participate in a “randomized clinical trial” because the stage of his disease was more advanced than the other participants. But that’s how trials have to be run in order to satisfy the FDA. And this problem shows why many patients are demanding an alternative to the FDA’s single, isolated and protected drug approval system.
Tomorrow’s Cures Today is now petitioning Congress to create a new drug development and testing system—one that would allow Austin and thousands of patients like him to try not-yet-approved drugs in a more open and transparent process, and which could result in approvals based on what’s learned from these observations of treatment outcomes for patients with widely varying health conditions. We call this new system, Free To Choose Medicine.
There are three key principles to Free To Choose Medicine.
1. Some patients and their doctors only want medicines tested and approved in the traditional way, and for them nothing would change. But others are not just willing, but eager to try not-yet-approved drugs. They should not be denied the right to choose a viable medical treatment. So, after safety testing is completed, patients should be free to choose a not-yet-approved drug if its developer is willing to make that drug available. After all, patients, under care of their physicians, and with access to detailed reports describing all prior test results, are in a better position than government bureaucrats to decide whether the risks are worth bearing.
2. To ensure patients are fully informed, developers offering not-yet-approved drugs on the Free To Choose Medicine track would have to make all prior testing data available in a publicly accessible online database. And every patient taking a Free To Choose drug would have to agree to let his or her doctor submit reports on an anonymous basis about their progress and any side effects. This “observational data” collected from real world use would provide valuable information to patients and let them make informed and meaningful choices.
3. As more and more patients use Free To Choose drugs, the value of information available to other patients will grow. Drugs that show severe side effects or little therapeutic benefit will be abandoned, although the data generated will be useful to drug developers. But drugs with positive experience will become more popular and the amount of observational data will grow even more. Technological advances in collecting and analyzing health data will make it possible for doctors and researchers to see who is benefiting the most from individual drugs and who is at higher risk. This “big data” collection will be superior to what’s collected in clinical trials, where researchers try to limit enrollment to patients who are all alike. With enough patients, the FDA could even approve new drugs on the basis of observational data alone.
By signing this petition you are supporting the right of patients to choose drugs that have not yet been approved by the Food and Drug Administration; establishing an alternative drug approval system using observational data and open source data collection; providing a means of making new drugs available faster and less expensive.
We need Congress to authorize Observational Approval to help patients like Austin McNary.
Please visit www.tomorrowscurestoday.org for more information about Free To Choose Medicine and consider participating with Tomorrow’s Cures Today in convincing Congress and the President that it is time for Free To Choose Medicine.
Vincent Galbiati, President
Tomorrow’s Cures Today Foundation
Urge Congress To Support Free To Choose Medicine
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