Americans have secured an important right: the freedom to choose medicine and treatments that may extend their lives.
The popular and bipartisan Right to Try movement scored a big win on Wednesday when President Trump signed into law the Right to Try Act (S. 204). The new law will enable people with a life-threatening disease or condition, with approval from their doctors, the ability to access investigational drugs and treatments that are undergoing clinical trials in the U.S. Food and Drug Administration’s approval process.
Prior to 1962, new drugs entering the market were only required to participate in safety trials by doctors and patients. Since then, when the FDA’s monopoly greatly expanded, nearly all drugs have had to pass through the FDA’s costly and burdensome approval process. In recent decades, the drug-to-patient pipeline has been clogged with increasingly more rules and restrictions, resulting in a drug approval process that now spans more than a decade.
Under the current model, even before the most promising drugs are permitted to enter the three-phase clinical trials, they must pass the FDA’s preclinical trial phase. This can take up to six years and requires the submission and approval of an Investigational New Drug application.
While drugmakers work their way through the FDA’s approval regime, people die and/or suffer in the interim. How many? Sam Kazman, general counsel at the Competitive Enterprise Institute, estimated in the Journal of American Physicians and Surgeons that hundreds of thousands of lives have been lost due to ever-increasing FDA clinical demands. Resources that pharmaceutical companies must use to move a drug through the bottleneck approval process are resources that cannot be used to conduct additional research and development of other promising treatments.
Because the average cost to bring a new drug to market now exceeds $2.5 billion and may take longer than a decade, pharmaceutical companies will often only put those drugs into the approval pipeline that have a comparably low risk of being rejected by the FDA.
The grim reality of the costs, deadly costs, of a too-long, too-regulated approval pipeline has forced the hands of some patients to create their own medicinal concoctions in an attempt to replicate the chemical composition of non-accessible investigational drugs.
For example, former physicist Ben Harris was stricken with ALS, also known as Lou Gehrig’s disease. Because Harris was ineligible to participate in clinical trials, he, along with others in the ALS community, self-administered a homebrew version of the experimental pharmaceutical drug NP001. Harris and others suffering with ALS injected or drank a mix of distilled water and sodium chlorite, a main compound in NP001. Although Harris did experience improvements in his ability to swallow after consuming the sodium chlorite mixture, it wasn’t effective enough to hold off the neurodegenerative disease’s progression. Two years after being diagnosed with ALS, in August 2013, he passed away at the age of 46.
Critics of RTT cite the FDA’s already existing expanded access, or “compassionate use,” program that began in 2009, which gives patients with life-threatening illnesses access to investigational treatments without having to participate in a clinical trial. Getting expanded access to these experimental treatments is in many cases the difference between life and death for these patients, because no other comparable therapies can treat their serious illnesses and they do not meet the conditions necessary to participate in clinical trials or do not live near a clinic center. However, the program has significant flaws. Apparently, “compassion” entails dying patients and physicians filling out 100 hours of required paperwork. Further, pharmaceutical companies are hesitant to use their investigational drugs in the program because they fear the FDA may use negative outcomes to justify scrapping the drug altogether.
It’s also worth noting that the Right to Try Act does not replace the expanded access program; it merely establishes an additional alternative pathway for patients. As the act makes clear, it is an expansion of “individual liberty and agency” and does not add layers of red tape to the existing bureaucratic drug approval process.
Some critics say experimental drugs might accelerate death for terminally ill patients, but preventing these patients from taking experimental drugs guarantees they will pass away. Not-yet-fully-approved drugs could be the solution to extend the lives of patients who are deteriorating with each passing day they go without access to effective treatment.
The days of mandating dying patients endure a court battle or international travel for the right to try experimental medicines are now over. The long overdue passage of this act is right in line with the Right to Try legislation that has already passed in 40 states, and with the overwhelming support of the public.
Regulators must realize that with risks come rewards. They must ensure rules do not inhibit drug developers and patients from taking life-savings risks, lest rewards be lost. The passage of RTT is just the first step in giving patients access to the newest life-saving drugs sooner and at a lower cost. The larger and more robust Free to Choose Medicine movement would extend freedom of choice to all patients, not just those terminally ill, and should be championed at the federal and state level to build on this momentum.
As Ben Harris declared, “We simply don’t have time to wait for the results of [clinical trials]. Our life spans are much shorter than the [Food and Drug Administration] approval process.”
Tim Huelskamp is president and CEO of The Heartland Institute. Arianna Wilkerson is a state governments relations coordinator at The Heartland Institute.
