This is the first piece in a four-part series about Right to Try at the federal and state levels and the response from the private sector. The second piece discusses the passage of federal Right to Try legislation. The third piece talks about the effects of Right to Try on the private sector. The fourth piece discusses Right to Try 2.0, which would expand the law to cover patients with rare diseases who need individualized treatment.
It’s a revolutionary reform born out of an appalling reality: Dying Americans are routinely blocked from potentially lifesaving treatments unless they receive a government permission slip. And that permission can take months, sometimes years—time dying patients do not have.
But thanks to Right to Try—signed into federal law in 2018 after 41 states passed their own versions of the reform—patients are beginning to wake up from this bureaucratic nightmare. Right to Try allows terminally ill Americans to access treatments that have received basic safety approval from the federal Food and Drug Administration (FDA)—and that are being given to patients in ongoing clinical trials—but that have not yet received final government approval for sale.
Right to Try is much more than a new law. It’s a bipartisan, grassroots movement of patients, doctors and activists—all of them fed up with having to navigate a half century of life-threatening regulations. It’s a declaration that in a free country, people should be able to decide for themselves, in consultation with their doctors, whether to try medicines that could prolong or even save their lives. It’s a pronouncement that Americans shouldn’t be forced to travel to other countries to obtain the lifesaving treatments they need because their own government bars them from getting care at home. It’s an acknowledgement of a basic constitutional right—a basic human right: the right to fight for one’s life.
Every year, thousands of people in the U.S. suffer and die while potentially helpful treatments await the outcome of a slow, bureaucratic process. On average, it takes 14 years and a billion dollars before these potentially lifesaving medicines can receive final government approval for sale.
The FDA evaluates treatments under a clinical trial process that consists of three or more phases: the first for basic safety evaluations, the second to assess efficacy in addition to safety and the third to test the drug against placebos and the currently available treatments. For some drugs, there is yet another phase of clinical trials. And until the years-long—sometimes decades-long—process is complete to the FDA’s satisfaction, pharmaceutical manufacturers may not sell these treatments, and doctors may not prescribe them.
This broken process results in horrifying stories like that of Jenn McNary, whose sons Austin and Max were diagnosed with Duchenne muscular dystrophy—an incurable, fatal, degenerative muscle disorder. Jenn tried to enroll both boys in a clinical trial for a promising treatment, but Austin’s disease had progressed too far for him to qualify. Jenn was forced to watch as one son’s condition improved significantly under treatment, while her other son’s condition worsened until he could no longer dress or use the restroom without help. Thirteen-year-old Max became 16-year-old Austin’s caregiver. While Austin did receive the treatment eventually (it is now approved), the delay has likely had irreversible effects on his health.
Laws and regulations that determine how and when a person can receive treatment are remarkably arbitrary. For instance, people are allowed to take dangerous medicines (cancer-treating chemotherapy is essentially poison, and even acetaminophen kills more than 400 people per year) or, in some states, to end their lives with a physician’s help. The federal government even allows healthy volunteers to be paid to test medicines that could be fatal to them. But it prohibits dying patients from trying investigational medicines that the FDA has deemed safe enough for human testing—medicines that might cure them or alleviate their suffering—but that may be ineffective or have unknown side effects.
In other words, the U.S. system does not entirely prohibit patients from taking investigational or even unsafe medications. Rather, it permits exceptions on a limited and arbitrary basis, rather than allowing access on a single, principled foundation. Right to Try establishes another pathway to treatment—one that doesn’t force patients to get a government permission slip. It extends to everyone the same permission to use treatments that is already enjoyed by those fortunate enough to get into a clinical trial, obtain a special exception or afford to travel outside the U.S. for treatment.
Thus, in some ways, Right to Try is not even all that radical—it’s equitable. It applies only to terminally ill patients and only to medicines that have passed the FDA’s Phase 1 safety testing and are being administered to patients in FDA-approved clinical trials as part of the subsequent phases of testing. If a treatment is withdrawn from FDA-approved clinical trials, it also becomes unavailable under Right to Try. And if a treatment has not received initial safety approval, it isn’t eligible. Right to Try also protects manufacturers and doctors from liability if a treatment does not work, so long as they act with requisite care. In short, Right to Try allows patients to seek the best care without having to first beg the government’s permission.
Despite Right to Try’s broad and overwhelmingly bipartisan support, it was not without its critics, who largely consisted of university bioethicists and current and former government officials. Critics contended that patients are unable to make decisions for themselves because they are desperate and underinformed and thus need a “neutral” decision-maker. But the FDA has its own biases. In fact, the agency is exceedingly risk averse. If the FDA approves a bad treatment, it gets punished, and if it blocks a bad treatment, it gets rewarded. But it doesn’t get rewarded for approving good treatments or punished for delaying them (and putting countless lives in jeopardy). So the FDA has every incentive to delay approval rather than hasten it.
And no one has all the information necessary to make the “correct” treatment decision—not even the FDA. After all, “unsafe” drugs routinely receive FDA approval. In 1997, the FDA removed fenfluramine after 25 years on the market when reports associated it with heart conditions. In fact, almost one-third of FDA-approved drugs in 10-year period were later flagged or removed from the market due to safety concerns. And the FDA itself recognizes that “there is never 100% certainty when determining reasonable assurance of safety and effectiveness.”
No system will eliminate all risks. The real question is: Who should ultimately decide what level of risk is “acceptable” to a patient—government officials or patients themselves? People will sometimes make choices that strike others as foolish or misguided. But that’s already true today, in the case of both FDA-approved medications and products that are sold without FDA oversight, such as herbal supplements. We trust people to make their own complicated and risky decisions in other areas of life, from buying a car to investing in the stock market to skydiving. Why should decisions about their own healthcare be any different?
Some bioethicists argued against Right to Try because it isn’t a panacea; there will still be patients who can’t afford treatment. While drug companies sometimes provide investigational treatments at no cost, and federal law prohibits companies from earning a profit off medicines that aren’t FDA approved, some lifesaving treatments (especially those that are brand new) may still be prohibitively expensive. But there are charities, including Right to Try-specific organizations, that help patients pay for treatments.
But even if some patients are still unable to pay, it makes little sense to address this disparity by denying access to everyone. For decades, the U.S. healthcare system favored the wealthy and well connected, who had the means to travel overseas to get treatments that poorer patients couldn’t access in the U.S., or the political or economic influence to obtain treatment prior to full FDA approval. Right to Try simply extends that option to everyone.
Detractors also argued that government can’t let patients and doctors make their own choices, because they might choose medicines that ultimately don’t work, succumbing to “false hope.” It’s true that some investigational medicines may not work. That’s also true of FDA-approved treatments. But one thing that surely doesn’t work is a bureaucracy that deprives ill people of the freedom to make their own medical choices.
Finally, bureaucrats argued that Right to Try was unnecessary, since the FDA already has a “compassionate use” program (officially titled the “expanded access” program), under which desperate patients may beg the government for “compassion” to permit them access to medicines that are still going through clinical trials. Before Right to Try, the FDA insisted it was already approving 99% of all “compassionate use” requests. But this statistic is an illusion: It ignores how many patients never even submit compassionate use requests because the approval process is so cumbersome.
By the FDA’s own admission, the initial paperwork for expanded access would take a doctor 100 hours to complete. And doctors have to abide by burdensome protocols and data-reporting requirements, essentially making them responsible for overseeing a mini-clinical trial for that one patient. Also, an Institutional Review Board (a separate committee at a medical facility) must weigh the ethical considerations associated with the patient’s use of the treatment—and many such committees meet infrequently. In practice, “compassionate use” is so tangled in red tape that before Right to Try, only about 1,200 patients per year were able to submit compassionate use requests to the FDA—even though over half a million Americans die annually of cancer alone. The real “false hope” is the government’s compassionate use program, not Right to Try.
Right to Try is not a silver bullet—it doesn’t guarantee patients a cure, and it doesn’t fix all the problems in the American drug approval system. It’s impossible to tell how many lives will be saved because of the federal Right to Try law. But for patients who are turned away from clinical trials and are unable to navigate the FDA’s complex expanded access process, Right to Try gives them a new chance—and the right to hope.
A “permission first” system like the one that existed before Right to Try prevents the right treatment from getting to the right patient at the right time when a life hangs in the balance. And a system that prioritizes the preferences of bureaucrats and politicians at the expense of individuals’ rights to shape their own destinies is fundamentally inconsistent with the principles of a free society. Right to Try takes the most intimate, personal decisions out of the hands of bureaucrats and puts them back where they belong: with patients and their healthcare providers. Terminal patients are already fighting for their lives; they shouldn’t have to fight the government too.
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