During a campaign stop in Lancaster, Pennsylvania, in 2016, now–Vice President Mike Pence heard an emotional appeal from an 11-year-old boy whose father was dying of ALS, a fatal neurological disease with no known cure. The boy asked for Pence’s support for federal legislation giving people like his father a “right to try” experimental treatments without oversight from the Food and Drug Administration. Pence promised to “get it done.” Since then, a lobbying effort championed by Pence and recently joined by libertarian groups connected to the Koch brothers has made right-to-try a Republican priority. Trump endorsed the idea in his State of the Union address, and several bills are under consideration in Congress.
The principle behind right-to-try legislation seems difficult to argue with, particularly when it’s conveyed by desperately sick people. Why shouldn’t terminally ill patients who’ve exhausted all legal treatment options be allowed to use, at their own risk, promising drugs that are still working their way through the FDA’s approval process? But the wrenching personal stories obscure a few essential facts—such as, that the FDA already has a program known as “compassionate use” that offers a pathway to obtain experimental drugs. They also gloss over the history of the right-to-try movement, specifically the fact that it is driven by ideological groups interested in curtailing the FDA’s regulatory authority more broadly. Many health-care experts and patient advocates, even those who believe the terminally ill should have access to experimental medicines, argue that the right-to-try bills in Congress now would not actually help patients—rather, they crack open a door that could lead to a deeper gutting of our drug-safety system.
Right-to-try is largely the project of the Goldwater Institute, an Arizona-based libertarian think tank and legal organization. Goldwater works mainly at the state level to roll back what it sees as government overreach—whether that be Obamacare, limits on corporate campaign contributions, or “obstacles that keep medical professionals from providing the care they are trained to give,” in the words of the institute’s senior policy adviser Starlee Coleman. Goldwater sees the FDA as one of these obstacles; the institute argues that the agency is blocking dying patients from obtaining experimental therapies that might save their lives. Since 2013, 38 states have passed right-to-try laws based on model legislation crafted by Goldwater, which would allow patients to skirt the FDA and work solely with their doctors and pharmaceutical companies to obtain drugs in the very early stages of clinical trials. Though the state laws have had limited practical impact, they’ve helped right-to-try become a national movement.
A major problem with such legislation is that its foundational premise—that the FDA is preventing patients with no other options from using experimental drugs—is almost entirely devoid of hard evidence. Established in 1987 and since revised, the agency’s compassionate-use program approves 99 percent of the requests it receives, usually within a few days or 24 hours for emergency applications. While right-to-try advocates often claim that the paperwork takes 100 hours to complete, the FDA has streamlined the process and says the current version takes only 45 minutes. To apply, patients must have exhausted all other treatment options, find a doctor willing to treat them with an experimental drug, and then convince the manufacturer of that drug to provide it.