Many experts say that patients facing death should get to try last-ditch experimental medications, but there’s disagreement over how exactly to accomplish that.
Since 2014, 30 states have passed “right-to-try” measures allowing drug companies to give terminally ill patients new medications before they are fully approved by the Food and Drug Administration, treatments otherwise available only to patients in clinical trials.
Pioneered by the libertarian Goldwater Institute, the bills allow dying patients to go around the FDA approval process and try out drugs that have completed only the first of three clinical trials.
Right-to-try is aimed at patients who have exhausted their treatment options, but who don’t qualify for a clinical trial for one reason or another. Only a small minority of terminally ill patients, about 3 percent, gain access to experimental treatments through clinical trials, supporters note.
Washington Republicans have gotten in on the movement, introducing federal bills that would block the FDA from overriding the state right-to-try laws. Sen. Ron Johnson of Wisconsin has introduced a Senate measure and Rep. Matt Salmon of Arizona is sponsoring a House version.
“I believe that the right-to-try is a component of the God-given right to life,” Salmon said at a rally the Goldwater Institute held this month. “It offers hope to those who have nowhere else to turn, and affords the possibility of the terminally ill benefiting from groundbreaking research that has yet to make it to market.”
Yet critics of the measures say they hold little practical value for patients. The legislation allows, but doesn’t require, pharmaceutical companies to provide patients with experimental drugs. If a drugmaker does give a patient an unapproved medication and the patient dies, the FDA might pause before approving it.
Alison Bateman-House, an ethicist at New York University’s medical school, says right-to-try measures give patients false hope they will get a lifesaving medication, as she doubts that most pharmaceutical companies would expand access under the new laws.
“There is no incentive for a company to give out a drug under right-to-try, because all it will do is poison their relationship with the FDA,” said Bateman-House, who sits on a committee of experts formed by NYU that has been examining the issue.
Bateman-House said that to really make experimental drugs available to the terminally ill, the FDA would have to assure drugmakers — formally, in writing — that the agency wouldn’t hold it against them if their medication doesn’t work outside the clinical trial setting.
“Right-to-try is a very naive attempt to give access to patients,” she said. “What you need is a more far-reaching type of agreement between the FDA and [the drug industry].”
Unsurprisingly, the drug industry opposes right-to-try too, saying it doesn’t approve of creating a precedent for bypassing FDA approval pathways.
“Any legislation should protect the integrity of clinical trials and the FDA oversight of expanded access to maintain the best interests of patients,” said Pharmaceutical Researchers and Manufacturers of America spokesman Andrew Powaleny.
But to the Goldwater Institute and other advocates of right-to-try laws, the important thing is to open the door, even if few drugmakers actually walk through it.
Goldwater spokeswoman Starlee Coleman acknowledged that some drugmakers won’t make their new medications available to patients outside clinical trials, but insisted that “the government shouldn’t stand in their way” if they want to.
“Not every company will participate, but there will be some that do — and who already are — and they should be free to do so without government red tape,” Coleman said.
While the FDA already has a “compassionate use” program whereby terminally ill patients can apply to try an unapproved drug, advocates of right-to-try see it as too lengthy and cumbersome. Some patients die while trying to complete the application process, which formerly took doctors as long as 100 hours.
The agency acknowledged the problem last year and issued draft guidance for a simpler application form that would take less time to complete, only about 45 minutes.
Federal right-to-try bills have languished in committee so far, but the bipartisan medical cures bill the House passed last year contains a similarly aimed provision requiring companies to provide the FDA with more information about how they would respond to patients requesting experimental drugs through compassionate use.
Goldwater has rallied support for right-to-try by telling stories of terminally ill patients hoping for a last-minute cure, inviting patients suffering from muscular atrophy and Lou Gehrig’s Disease to speak at a recent rally on Capitol Hill.
But critics charge that despite the hope that right-to-try laws generate, few patients if any have gained access to experimental drugs under the new laws. Still, state lawmakers have appeared eager to move forward on right-to-try, making the institute’s campaign hugely successful.
“It sounds horrible that I’m not going to vote for a patient to have a right to try,” Bateman-House said.
While California Gov. Jerry Brown vetoed a right-to-try law in the fall, 30 states have right-to-try on the books, and the legislation has been introduced in 18 more states, according to the group.
