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Research & Commentary: Suffering patients need the hope that cutting-edge individualized medicine can offer, but the FDA is still decades behind

March 15, 2022

In this Research & Commentary Matt Dean looks at new compassionate care access that picks up where right to try left off

Federal Right-to-Try legislation was signed into law on May 30th, 2018. Right-to-Try is a compassionate idea that is rooted in the fact that people who have perhaps months to live, cannot wait additional years for the FDA to complete phases of trials on a potentially lifesaving drug that has been found to be safe, but whose efficacy has not yet been proven.

Now the states are leading again. In Arizona, Senator Barto has introduced a bill that picks up where Right-to-Try left off. SB1163 recognizes that advances in research offering individualized treatments to patients just doesn’t work in the current FDA approval process that was designed for one-size-fits-all drug approval.

FDA is a bureau set up to protect the safety of the public by testing drugs to make sure that they are safe and effective. The FDA has done a good job in our country that develops and approves more drugs than any other country in the world. The FDA regulates 20 cents out of every dollar spent by the American consumer including 20,000 prescription drugs and 78% of the food supply.  

However, when approved treatments do not work for suffering patients, FDA is simply too slow to respond with other options. The current emergency expanded authorization approval process was only approving around 1,000 cases/year. Those not included in a clinical trial or expanded access, are left without hope.

Right to Try has offered that hope for those who face a life-threatening disease, have exhausted all treatments, and do not qualify for a clinical trial. This bill takes the next step and offers hope to millions more by expanding the available patients to those who could be helped through individualized treatments.

The FDA by its nature amasses very large cohorts and looks for cures and treatments that are safe for all. However, as research continues to accelerate, new treatments and potential cures are being found for smaller cohorts.

It’s unrealistic to expect the FDA to respond as quickly as technological innovation demands, but Right to Try has offered the flexibility to offer suffering patients a way forward. The FDA was intended to be so to protect the greatest number of people by protecting them from unproven drugs. However, that approval process was designed at a time when the human genome was not yet mapped, and the idea of individual DNA mapping was science fiction.

The Mayo Clinic’s “All of Us” research initiative aims at building a bank of one million deidentified genetic profiles. This will allow researchers to design patient-specific therapies to cure and treat common and rare disease. Researchers at Mayo recently diagnosed Araea Sell with a rare genetic disorder that had gone undiagnosed for seven years. The thirteen-year-old is responding to therapy designed for just her thanks to targeted treatment through a clinical trial at Mayo. "I'm very thankful and I feel much better," says Araeya, who loves camping, crafts and hanging out with friends. "I have a lot more energy, and I'm not so tired all the time." But only a lucky few like Araeya have access to clinical trials.

As research builds, more patients will have the opportunity to have that chance. States are shaping that with important legislation like Senator Barto’s legislation. 

 

The following articles provide more information about right to try.

 

The FDA does allow limited expanded access to nonapproved drugs

Expanded Access | FDA

Right to Try passed and implemented nationally

Right to Try | FDA

The Goldwater Institute looks at the Right to Try movement and progress

What is Right to Try | Right to Try - National Movement

State legislatures begin the next step in the right to try movement

http://righttotry.org/rtt2-0/

FDA at a glance

https://www.fda.gov/about-fda/fda-basics/fact-sheet-fda-glance

Individualized Medicine at Mayo Clinic

 https://individualizedmedicineblog.mayoclinic.org/2022/02/28/young-girl-with-rare-disease-gets-life-changing-diagnosis-and-targeted-therapy-at-mayo-clinic/

https://www.mayo.edu/research/centers-programs/center-individualized-medicine

Mayo Clinic’s “All of Us” program is collecting a one million-person genetic bank to help discover specialized and individualized treatment plans for unique patients.

https://newsnetwork.mayoclinic.org/discussion/nih-all-of-us-research-program-highlights-progress-next-steps-in-building-largest-most-diverse-health-research-effort/#:~:text=Mayo%20Clinic%20is%20one%20of%20more%20than%20100,May%206%2C%202018%2C%20several%20milestones%20have%20been%20reached.

Science Direct looks at the FDA’s Expanded Use program

https://www.sciencedirect.com/science/article/pii/S2452302X17302917

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Nothing in this Research & Commentary is intended to influence the passage of legislation, and it does not necessarily represent the views of The Heartland Institute. For further information on this and other topics, visit the The Heartland Institute’s website

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Article Tags
Health Care
Author
Matt Dean is Senior Fellow for Health Care Policy Outreach at The Heartland Institute.
mdean@heartland.org