August 28, 2020
By Christina Sandefur
For decades, the federal U.S. Food and Drug Administration (FDA)’s sluggish and bureaucratic drug approval process has prevented patients from accessing potentially lifesaving vaccines and treatments. And for decades, patient advocates have been demanding a system that focuses less on one-size-fits-all regulations and more on getting individuals the right treatments at the right time.
The growing outrage over this exceedingly inhumane system began to manifest in popular culture, just as a state-based, grassroots movement called Right to Try began driving a national conversation about letting people suffering from terminal illnesses decide—in consultation with their doctors—whether to try drugs that might prolong or save their lives.
In 2013, the Oscar-nominated Dallas Buyers Club starring Matthew McConaughey revealed the toll that the FDA’s drug approval process took on AIDS patients in the 1980s. At one point in that film, a bureaucrat admits “the FDA’s standard procedure to approve a new drug is eight to twelve years.” But patients don’t have that kind of time. “Screw the FDA,” McConaughey’s character retorts. “I’m gonna be DOA.” Unable to wait and desperate to save their own lives, McConaughey’s character began smuggling unapproved drugs into the U.S.
The film was based on real events—intense political pressure from AIDS sufferers that eventually prompted the federal government to take minor steps to improve its process. In 1987, the FDA instituted the “compassionate use” program, also known as expanded access, allowing people to obtain investigational medications outside the traditional (and extremely limited) clinical trial mechanism. But while political pressure changed the law, it did not change the mindset at the FDA. Decades later, the expanded access exception is still applied inequitably, on a case-by-case basis. And the process is so extremely cumbersome and time-consuming that only about 1,200 patients a year are even able to submit requests to the FDA—even though, for example, over half a million Americans die annually of cancer alone.
Two years ago, the CW’s television comedy-drama Life Sentence explored the unfairness and inhumanity of this system. That show featured a young woman named Stella Abbott (played by Lucy Hale) whose terminal cancer is unexpectedly cured by a last-resort investigational treatment she receives in a clinical trial. In the show’s third episode, “Clinical Trial and Error,” Stella is devastated to learn that a young girl dying of cancer and out of options can’t get the treatment that saved her own life, because the clinical trial was shut down for not being effective enough. Stella fights to get the trial reopened because although the treatment is a long shot, it’s the girl’s only hope.
Although that was a work of fiction, it has been an unfortunate reality for many terminal patients. Jenn McNary, mother of two boys with Duchenne muscular dystrophy, experienced the horror of watching one of her young sons improve significantly under treatment he received in a clinical trial, while her other son—who was not admitted into the trial—worsened until he could no longer dress or even use the restroom without help.
Then came Right to Try, a bipartisan grassroots reform that passed in 41 states in only four years and became federal law on May 30, 2018. Right to Try protects the right of terminally ill patients to use safe, experimental treatments that could prolong or save their lives. It’s been helping people across the country, putting these deeply personal, life-or-death decisions back in the hands of patients and their physicians. And it’s continuing to make healthcare more patient-centric.
Earlier this year, Right to Try featured in NBC’s medical drama New Amsterdam (a show about doctors who “go above and beyond for their patients” and “tear up the bureaucracy and provide exceptional care”). In the second season episode “In the Graveyard,” doctors discuss prescribing psilocybin, a psychedelic prodrug compound, to help treat depression in terminal patients. Although that treatment is not yet FDA-approved, one of the characters notes that it’s showing promise in clinical trials and thus could be part of a treatment protocol because “Right to Try laws allow wide latitude in treating terminal patients.”
Of course, real life isn’t Hollywood. But these films and shows aren’t about the details of particular medicines. They’re about a deeper principle—one that everyone, regardless of their political views, should be able to get behind: A patient’s life is his or her own, and that patient—not some bureaucracy—should be making the choices about medical treatment. Our drug approval system is woefully out of date, and Right to Try has offered us one example of a successful solution. As we combat the coronavirus pandemic, it’s time we extend its principle of patient autonomy to as many people as possible.
Christina Sandefur is the Executive Vice President at the Goldwater Institute.