Imagine you've been diagnosed with a terminal disease. You exhaust all the treatment options currently available to save your life, but none work. Then you learn of a potential new treatment that's been proven reasonably safe and has shown promise, but hasn't been approved by the federal government for widespread use.
Would you want to try the new treatment? I would. Depending on its relative risk, you might even be willing to try a promising drug that hadn't yet passed safety inspection.
Unfortunately, this isn't a hypothetical. It's a nightmare that many Americans must endure every year as their survival hinges on FDA approval of drugs designed to save people from terminal illness. While new drugs crawl through the FDA approval process, Americans with terminal diseases die waiting.
FDA approval is a three-step process. In Phase I, a potential new drug is tested for safety among a small group of test subjects. In Phase II, the drug is tested for its effectiveness in a small group. Finally, in Phase III, the drug is tested for safety and effectiveness in a large group.
Generally, if you're not in the clinical trial group, you're out-of-luck. And good luck getting into the trial group. According to the Goldwater Institute, an estimated 97 percent of patients with the worst diagnoses are either ineligible because of the stage of their disease or they don't have a way to participate in a clinical trial.
This fundamental injustice was the subject of Dallas Buyer's Club, a recent hit movie starring Matthew McConaughey who plays Ron Woodruff, a desperate AIDS patient who takes and distributes AIDS treatments that aren't approved by the FDA in the 1980s. Woodruff finds a drug that seems to work for him - and then creates a "buyer's club" to help others access the same treatment.
Thanks largely to the efforts of AIDS activists in the 1980s, the FDA tweaked the drug approval process to allow for greater participation in drug trials for people diagnosed with terminal diseases. Today, a desperate patient can petition the FDA for permission to receive a drug outside of a clinical trial. But the procedure is cumbersome and expensive. According to the FDA, a single application from a doctor should take around 100 hours to complete.
Rep. Jim Neely understands the problems with FDA approval of new treatments for terminal patients. A practicing physician who grew frustrated with slow approval of AIDS treatment drugs in the 1980s, Dr. Neely also has personal experience with this unjust bureaucracy. His daughter Kristina is fighting Stage IV colon cancer and has been trying, unsuccessfully, to participate in clinical trials.
With these experiences in mind, Dr. Neely continued the work he's been doing for patients his entire career. He did his best to improve their condition - this time in a different arena - by sponsoring House Bill 1685. Though the bill, unfortunately, cannot force the FDA to change its rules, it removes all barriers in state law for patients with terminal illnesses to gain access to potential life saving drugs that have not yet been approved. It does so by permitting drug companies to sell experimental treatments that have passed Stage I testing to patients with terminal illnesses who have considered all other treatment options, given their informed consent, and received a prescription from their physician. It also insulates physicians who prescribe new treatments from lawsuits or professional discipline as a result of prescribing such drugs unless there's evidence of gross negligence or willful misconduct.
While a stringent FDA approval process makes sense for non-life threatening diseases and widespread sale of new drugs, it defies logic to forbid Americans who are about to die from taking drugs that might work. HB 1685 isn't about Viagra or even more serious qualify-of-life drugs. It's about potential life-savers, and it furthers the foundational premise of our Republic - that we are all endowed by our Creator with an unalienable right to life.
In the last week of session, HB 1685 passed unanimously, making Missouri the first state to adopt what many have called "right to try" legislation. For Missourians caught in these terrible situations, it removes all state-based obstacles. And though its impact is limited to our state and we cannot force the FDA to adopt a more flexible stance, Dr. Neely's bill may be the first step toward what would be more precisely called a "right to try to save your own life" law.
State Rep. Jay Barnes, R-Jefferson City, represents Missouri's 60th District.