There's no question about it: the new law sounds just great. President Donald Trump, who knows a thing or two about marketing, gushed about its name when he signed the “Right to Try” bill into law on May 30. He was surrounded by patients with incurable diseases, including a second grader with Duchenne muscular dystrophy, who got up from his small wheelchair to hug the president. The law aims to give such patients easier access to experimental drugs by bypassing the Food and Drug Administration.
The crowd-pleasing name and concept are why 40 states had already passed similar laws, although they were largely symbolic until the federal government got onboard. The laws vary but generally say that dying patients may seek from drugmakers any medicine that has passed a phase I trial—a minimal test of safety. “We're going to be saving tremendous numbers of lives,” Trump said. “The current FDA approval process can take many, many years. For countless patients, time is not what they have.”
But the new law won't do what the president claims. Instead it gives false hope to the most vulnerable patients. “This is a right to ask, not a right to try,” says Alison Bateman-House, a medical ethicist at New York University and an expert on the compassionate use of experimental drugs. The right to ask was already firmly in place. “If I had a magic wand,” she says, “rather than passing a new law, I would have figured out a way to educate people about the system we already have.”
In fact, for decades pharmaceutical companies have made unapproved drugs available through programs overseen by the FDA. This “expanded access” is aimed at extremely ill patients who, for one reason or another, do not qualify for formal drug studies. A 2016 report shows that the FDA receives more than 1,000 annual requests on behalf of such patients and approves 99.7 percent of them. It acts immediately in emergency cases or else within days, according to FDA commissioner Scott Gottlieb.
Of course, there are barriers to getting medicines that may not be effective or safe. Some patients cannot find a doctor to administer them or an institution that will let them be used on-site. And many of these drugs are simply not made available. Drugmakers cannot be compelled to do so: a 2007 federal court decision found “there is no fundamental right ... of access to experimental drugs for the terminally ill.” The new law changes none of this.
Pharma companies have many reasons to be cautious about providing what is in their labs. A drug in early stages of testing may have risks and benefits that are wildly uncertain. Supplies may be limited and production costly, so a company may wish to save its precious stock for clinical trials. Developers may also be concerned that their drugs will do poorly in fragile, dying patients, word will get out, and the consequences will be dire: patients will be scared away from studies, investors will retreat, stocks will tank. Thus, work on a potentially valuable new medicine might get derailed by acts of compassion.
“If you said patients could just call up and say, ‘I want the drug—give it to me,’ how could you ever run a clinical trial?” asks Kenneth Moch, president and CEO of Cognition Therapeutics in Pittsburgh. “What happens for future patients?” In a long career, Moch has had a hand in making drugs available for compassionate use and holding them back. “There are no simple, monolithic solutions,” he says. He doubts his industry will embrace “Right to Try” and said so at a congressional hearing on the bill: “My comment was that no ethical developer of an experimental medicine I know of would let it be used outside of the FDA's regulatory oversight.”
Unethical companies, however, may find fresh opportunities to prey on desperate patients under the new law. It releases doctors, hospitals and drugmakers from liability. And although it stipulates that manufacturers can charge patients only what it costs to provide the drug, there is no required preapproval of these charges by the FDA, as there is with expanded access. Such issues led dozens of major patient-advocacy groups to oppose the legislation, which was originally drafted and promoted by the Goldwater Institute, a libertarian think tank.
Vibhav Rangarajan, an Illinois cardiologist, had hoped the law would help his two-year-old daughter, Radha, gain access to an experimental drug for a rare disease called metachromatic leukodystrophy that is destroying her nervous system. He wrote movingly about her plight in a recent piece published online in Stat and says he is saddened that this law lacks meaningful incentives for drug companies: “It's not really going to change the landscape.”