Arizona has made headlines for school choice and occupational licensing reform, but it's also a leader in allowing terminal patients access to promising treatments that have yet to be approved by the Food and Drug Administration (FDA).
Such Right-to-Try laws are now in place in about half of all states, but they don't address advances in medical technology creating treatments that can't pass through traditional regulatory channels because they're tailored for specific patients. Last week, Arizona Gov. Doug Ducey signed legislation expanding the right to try to include personalized medicine.
"The governor signed S.B. 1163, which will help Arizonans get the care they need sooner," Ducey's office announced on August 9. "The legislation builds on 'Right to Try'—a historic, national policy that was developed in Arizona at the Goldwater Institute. The policy ensures that individuals with terminal illnesses are able to seek out investigational medication when their approved treatment options have been exhausted."
The original Right-to-Try legislation was and is groundbreaking, giving desperate patients hope of help from treatments that are still working their way through the FDA's interminable clinical trials.
"Right-to-try laws are state-level reforms that aim to allow terminally ill patients to gain access to experimental drugs without the permission of the Food and Drug Administration (FDA). Under the current model, access to experimental drugs is controlled by the FDA, which must give its approval after it receives an application from a patient's physician," notes BallotPedia. "Right-to-try laws allow patients and physicians to approach a drug manufacturer directly to ask for access to the drug."
Right to Try was adopted by 27 states by mid-2016, adds BallotPedia. Counterpart federal legislation became law in 2018. The legislation doesn't guarantee patients access to treatments, but it partially gets the government out of the way of patients and physicians seeking to try medications that have passed Phase I clinical trials.
But medical technology is taking an increasingly customized approach, seeking to address ailments as they appear in individual patients and not across populations. The legacy regulatory system isn't really set up to deal with customized modern medicine.
"Rapid medical innovations have made it possible to take an individual's genetic information and create a treatment for that individual person," notes the Right-to-Try website, sponsored by the Goldwater Institute. "More patients, especially those with rare and ultra-rare illnesses, will pursue these treatments when they have exhausted other options. Unfortunately, the FDA's current regulatory scheme is not designed to handle these kinds of individual treatments, and that will keep life-saving medication out of the hands of patients unless reforms are adopted."
Goldwater highlights the Riley family of Ahwatukee, Arizona, two of whose daughters, Olivia and Keira, were diagnosed with Metachromatic Leukodystrophy, a rare and degenerative brain disease that robs victims of their abilities before taking their lives. The condition of Olivia, now three, was rapidly progressing and beyond the reach of even experimental treatments. However, newborn Keira was still asymptomatic and could potentially benefit from newly developed gene therapy. But that personalized treatment doesn't qualify for the FDA's usual approval process or for the original Right-to-Try shortcut. That left the family scrambling.
"This treatment for gene therapy is only available in Milan, Italy, because it wasn't yet FDA approved," Kendra Riley, the girls' mother, comments in a video about their plight. "Unfortunately, we had to gather the community to help us and raise hundreds of thousands of dollars to move to Italy for six months during the global pandemic."
Not every experimental treatment works, of course. But when you're out of options and trying to rescue your children from a terminal illness, you do what you can.
"They literally took her as an individual, looked at her DNA and how they could help change it and modify it to make it better so that her body would then function as it should have to begin with," Kendra adds.
Now back home in Arizona, the Riley family reports that Keira is developing normally, having apparently benefitted from the therapy. Her older sister, Olivia, unfortunately, is not expected to survive childhood.
While Right to Try 2.0 expands patients' options, it's far from perfect. Both the original legislation and the updated version are limited carve-outs from a vast regulatory apparatus that slows the approval of medical treatments to a crawl, with the cost measured not only in money, but in lives.
"Drug lag costs lives because people suffer and die from disease that might be treatable, if only there were more investment in finding a cure," argued Jessica Flanigan, an associate professor at the University of Richmond, in her 2017 book, Pharmaceutical Freedom: Why Patients Have a Right to Self-Medicate. "Requirements that raise the cost of development make it less likely that they will succeed. Premarket testing conditions also cost lives because patients with conditions that could be treated or cured by unapproved drugs suffer and die while they are waiting for approval."
Like others, Flanigan recommends restricting the FDA and other regulatory agencies, if they are permitted to have any role at all, to informational certification. Patients and physicians could follow or disregard such guidance as they please.
But that's a major reform for another day, though not too far in the future, we can hope. In the meantime, we're limited to nibbling away at barriers regulators place in the way of desperate patients seeking treatments for intractable illnesses. Right-to-Try laws, both versions 1.0 and 2.0, are part of that effort to give patients just a little more leeway. So are efforts by Sens. Cory Booker (D–N.J.) and Rand Paul (R–Ky.) to allow patients limited access to Schedule 1 drugs, including MDMA and psilocybin, that have been through Phase 1 trials.
"As a physician, I know how important Right to Try is for patients facing a life-threatening condition." comments Paul. "Unfortunately, the federal bureaucracy continues to block patients seeking to use Schedule I drugs under Right to Try."
The federal bureaucracy continues to block hopeful patients from doing a lot of things they would do if free to exercise their discretion. Right to Try 2.0 is only a small improvement. But a small improvement is better than none, and the Riley family is strong evidence that such reforms are worth the effort.