States move to give terminally ill 'right-to-try' drugs

A growing number of states are considering legislation to allow terminally ill patients to gain access to experimental drugs, before the U.S. Food and Drug Administration deems them market-ready.

"Right-to-try" laws were passed in five states last year — Arizona, Colorado, Louisiana, Michigan and Missouri, according to the National Conference of State Legislatures. Twenty-five more have proposed similar legislation.

The legislation is modeled after a proposal drafted by the Goldwater Institute, a conservative Arizona-based think tank. It allows patients who are ineligible or unable to participate in clinical trials — and have exhausted all other treatment options — to gain access to experimental drugs as long as they have a physician's recommendation.

Not all experts in the medical community agree that this legislation could be beneficial. Arthur Caplan, founding director of the Division of Medical Ethics at NYU Langone Medical Center's Department of Population Health, said that loosening restrictions on experimental treatments can lead to as much harm as good.

"Experimental drugs can make you die quicker," he said. "You can die much more miserably and much more quickly if the drug is bad."

Although the FDA does have expanded access provisions, which allow terminally ill patients to receive experimental treatments, Kurt Altman, the national policy adviser and general counsel of the Goldwater Institute, said that the process can be burdensome and overlong.

Earlier this month, the FDA announced that its expanded access form will only require 45 minutes for a physician to complete. Previously, the form required about 100 hours to fill out. The length of the process was such that it could take three or four months for a patient to get a decision from the FDA, Altman explained. With right to try however, it should only take three to four weeks.

The purpose of the legislation then, he said, is to give people who have exhausted all methods of treatment the hope of seeing another day.

"It brings the control (over medical decisions) back to a local level," Altman said. "Just between a patient and their doctor."

David Gorski, managing editor of Science-Based Medicine agreed that while the FDA's process may be cumbersome, it is still a viable method.

"The 'ineffectiveness' of the FDA's expanded access program is greatly exaggerated by proponents of right to try," said Gorski, a surgical oncologist. "While it's true that the paperwork burden could really use some pruning, it is uncommon for expanded access requests to be denied."

Caplan said that he doubts the effectiveness of the legislation however, because it does not require drug companies to agree to provide the drugs. In this regard, right to try is similar to the FDA, which also cannot force drug manufacturers to provide their products.

"These laws have been around for six months and I don't know anybody who has gotten a drug yet," Caplan said. "If that's true, that's not much of a law."

Similar laws have been proposed before, such as the Compassionate Access Act which failed to gain the necessary support to make it past its introduction to the U.S. House of Representatives. None of them have garnered the amount of attention the right-to-try laws have recently amassed. Of course, with the added attention comes increased scrutiny.

"It opens the door to physicians who want to rip you off. They can give you a quack cure and you wouldn't know it," Caplan said. "There's too grave a risk for irresponsible doctors when you take the FDA out of the picture."

Altman counters this complaint by citing the fact that under right to try, drugs must have cleared the FDA's phase I testing and remain in ongoing tests.

"We have built-in protections from doctors selling 'magic elixirs' to desperate patients," he said. "It's not just some drug that a doctor can get in a dark alley from a guy in a trench coat."

According to Gorski, merely having passed phase I trials is not enough to determine whether a drug is safe, since these trials are based on small sample sizes, often of less than 30 patients, and are only meant to determine the maximum tolerated dosage of the drug, as well as any obvious side effects.

"Most drugs that pass phase I never make it to market, and it's not at all infrequent for serious toxicities and side effects to be identified in phase II and III trials," Gorski said.

He also pointed out that the legislation would still make experimental treatments inaccessible to many patients, as it does not obligate either the drug manufacturers or insurance companies to help provide funding for patients. It also prevents patients from suing doctors for malpractice if the recommended drug has an adverse effect on their condition, as the legislation explicitly prevents any action to be brought against physicians for their recommendations, Gorski explained.

"The patient seeking right to try is completely on his own," he said. "The patient has to pay for experimental drug completely on his own, which means only those who are either quite well off or sufficiently savvy at social media fundraising to raise the enormous amounts of cash necessary will have any chance to have access to experimental drugs under right to try."

Caplan said since the right-to-try laws already in place are state laws attempting to bypass the FDA's authority, they can be ruled unconstitutional by the federal government.

"They're feel-good laws," Caplan said. "They do draw attention to the issue, but they don't do anything to get you the drugs."