On Election Day, Arizona voters approved a referendum that allows terminally ill patients to receive treatment with drugs and devices that haven’t been approved by the Food and Drug Administration. Arizona became the fifth state to approve a so-called right-to-try law this year.
But critics of the state laws charge that they’re feel-good measures that don’t address some of the real reasons patients don’t receive experimental treatments in the first place.
Nevertheless, legislatures in Colorado, Louisiana, Michigan and Missouri also passed right-to-try laws this year as part of a nationwide effort spearheaded by the conservative Goldwater Institute, which hopes to get right-to-try laws on the books in all 50 states.
The measures generally permit a patient to get access to an experimental drug or device after it has passed the earliest stage of safety study. This initial testing for drugs, often called phase 1, involves a small group of usually healthy people receiving a medicine to get a first look at its safety and side effects. The testing requirements for devices vary, and initial safety may be explored in small clinical studies or laboratory experiments.
“For people with terminal illness, for whom nothing else has proved effective, they don’t have the luxury of waiting four to five months to get through the FDA’s compassionate-use program,” says Victor Riches, vice president of external affairs at the Goldwater Institute. Riches says the delays come from requirements for a lengthy application by the patient’s physician, FDA review and approval of the request, and a federally required review by an institutional review board, a group of medical experts that evaluates the risks and ensures that the patient understands them as well.
Between 2009 and 2013, the FDA received roughly 1,000 expanded-access applications annually and approved virtually all of them. Some patient advocates and policy analysts say that while the FDA process could be sped up, they support the agency’s continued oversight because of its critical role in ensuring safety and effectiveness.
Experimental therapies could help some people. But they are also, by definition, unproved, and they may turn out to be ineffective. They may also carry significant risks, not all of which are known before in-depth clinical testing.
Even if the FDA approves a request for an experimental drug or device, the patient might not get it. Drugmakers and device companies aren’t obligated to provide a therapy to patients who request it.
Right-to-try laws are no different.
Without any assurance of access to an experimental drug or device, and with no financial support to help patients cover the costs, right-to-try laws give patients false hope, say critics of the laws.
“There’s no money in these laws, and no provision for companies to supply anything,” says Arthur Caplan, a medical ethicist at NYU Langone Medical Center. Caplan has been a vocal critic of right-to-try laws.
The degree to which insurers cover drugs and medical services under FDA’s expanded-access program varies. According to the FDA website, some companies charge patients for the experimental drug, and medical services may or may not be covered.
Critics also voice a broader concern about the potential impact on the drug development process.
How do drugmakers or device companies balance the needs of a sick individual against the potential to introduce the therapy to a broader population? Terminally ill individuals who receive experimental treatment may well suffer serious adverse events, potentially setting back the approval process.
In addition, if more people get access to experimental therapies through right-to-try laws, they may be less likely to participate in the clinical trials that are essential to new drug development.