In the past three years, 33 U.S. states have passed laws aimed at helping dying people get easier access to experimental treatments. Supporters say these patients are just looking for the “right to try” these treatments.
Such laws may sound compassionate, but medical ethicists warn they pose worrisome risks to the health and finances of vulnerable patients.
California’s right-to-try law was enacted in January. It protects California doctors and hospitals who want to prescribe any medicine that has successfully made it through a Phase 1 drug trial. That’s the first stage of human testing required by the Food and Drug Administration — the sort of study that focuses merely on a drug’s safety, not its effectiveness.
Ian Calderon, a Democrat from Southern California and majority leader in the state’s Assembly, was one author of the law. He said that if he had just received a terrible diagnosis, he would want to try anything possible to live.
“My thought would be, ‘What do I have to lose?’” Calderon said. “I have an opportunity to potentially find a cure. Or at least find something that prolongs my life — find something that could help me.”
He said the law seemed to him the logical next step, after California passed a law in 2016 permitting physician-assisted death.
“It’s inhumane to have a law on the books that allows you to end your own life, but no law on the books that allows you to fight to extend it,” he argued. “That just seems counterintuitive.”
Proponents contend that some doctors have been hesitant to help dying patients, for fear of being penalized for using drugs or devices that don’t have FDA approval.
California’s law ensures that doctors can help patients petition to get an investigational medicine from drugmakers without fear of censure from the state’s medical board. It eliminates regulatory obstacles on the state level and creates processes for patient consent and data collection.
Lina Clark and her husband David Huntley on vacation in Croatia in 2014. Before he died of complications from ALS in 2015, Huntley’s illness prompted them both to become activists, lobbying for California’s right-to-try law. (Courtesy of Lina Clark)
David Huntley, a San Diego State University professor who died from ALS, or Lou Gehrig’s disease, in 2015, was among the patients who fought for California’s law. Before he died, Huntley testified in favor of the bill from his wheelchair.
His widow, Lina Clark, founder of the patients’ advocacy group HopeNowforALS, said her husband completely understood what was at stake.
“The patient community is saying: We are smart, we’re informed, we feel it is our right to try some of these therapies, because we’re going to die anyway.”
It’s a compelling argument, but there are serious risks, according to doctors and medical ethicists.
“We know some people try to take advantage of our desperation when we’re ill,” said Dr. R. Adams Dudley, director of the Center for Healthcare Value at University of California, San Francisco. “If we take the FDA out of it, how do we protect people from physicians or drug companies that will want to sell them things and will want to prey on their desperation?”
Dudley said the FDA and the clinical trial process were put in place for a reason — not just to shut out would-be snake oil salesmen, but to ensure that manufacturers are producing a safe product and not cutting corners.
“If you say there’s a path that’s not through the FDA,” he said, “then there are billions of dollars out there to be made by skipping the important steps that we’ve developed.”
The new state laws are called “right to try,” Dudley said, but all that patients can really do is ask for an experimental medicine. Drug companies don’t have to give them the medicine, and insurance companies don’t have to pay for it.
David Huntley testified in favor of California’s right-to-try bill in April 2015 in Sacramento. He died three months later. (Courtesy of Lina Clark)
Dudley said patients could spend huge amounts of money trying a drug that hasn’t been proven to work. And the patient may also be giving up their hopes for a controlled, peaceful death at home.
“Instead, you try a drug and you get very severe lung problems,” he said, “and you end up on a breathing machine in a hospital. That could cost hundreds of thousands of dollars.”
Although nearly three dozen right-to-try laws are now on the books, researchers at New York University who’ve been looking for evidence of the laws’ usefulness haven’t yet found a single substantiated case of a patient getting a drug using a state law.
That’s partly, perhaps, because the FDA already has a process to help patients and their doctors apply for the use of experimental drugs (and such requests are nearly always approved). Still, Calderon and others point out that the process entailed in these “compassionate use requests” is much too slow and cumbersome for many patients who are dying.
A new federal research law might help change that. The 21st Century Cures Act requires drug companies to be more transparent about how they decide who gets experimental access to promising medications, and how long it will take.
This story is part of a partnership that includes KQED, NPR and Kaiser Health News.