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Legislation would allow 'right-to-try' trial drugs

February 5, 2015

Originally published in USA Today

A person with a terminal illness may hear of a drug that could be promising only to hear it has not yet received government approval for use. Unless that person can find his or her way into a clinical trial for that compound, he or she will most likely not benefit from it.

Now a number of states, including Indiana, are trying to change that dynamic with “right-to-try” legislation that would allow those with a terminal illness to use a drug that has gone through the first stage of approval, as long as they had the blessing of their doctor and the drug manufacturer.

Four states — Michigan, Colorado, Missouri, and Louisiana — have passed right-to-try bills. Arizona put a similar measure in place via a voter referendum.

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This year, another 22 states, including Indiana, are considering similar legislation, which would allow the use of medicines that have cleared basic safety tests.

“This isn’t to help drug companies. This is all patient-driven, because we have heard stories of people who are dying and there’s drugs out there that could possibly save them and they’re not allowed to take them and they’re like, ‘What have I got to lose?’ ” said Republican state Rep. Wes Culver, sponsor of a right-to-try bill in the Indiana General Assembly.

But some raise concerns about such efforts, saying that gaining access to early-stage drugs may not actually benefit those in the final stages of life.

Dr. Paul Helft, an oncologist and director of the Charles Warren Fairbanks Center for Medical Ethics at IU Health, said that right-to-try bills, if passed, could inadvertently wind up hurting some patients.

“I think these are mostly coming out of the illusory thinking that meaningful benefit is likely,” said Helft, also a professor of medicine at Indiana University School of Medicine. “Two, these come out of the sense there’s nothing to lose, you’re going to die anyway. But there are things that you could lose: You might die faster or might spend time getting sick from the side effects of the drug.”

Although most of those who might exercise their “right to try” have terminal cancers or diseases like Lou Gehrig’s disease, Culver did not have those in mind and had never heard of the phrase when he first came up with the idea, he said.

Ebola, the lack of approved medicines for the disease, and fears of a worldwide outbreak inspired his bill, Culver said. Only after proposing it, did he realize he had tapped into a national movement.

The Goldwater Institute, a conservative think tank based in Phoenix, has taken up the cause, writing model right-to-try legislation for states, a version of which was proposed in the Indiana Senate. The institute sees the issue as protecting “the fundamental right of people to try to save their own lives.”

“The people that we hear from are talking about drugs that have shown very significant efficacy in clinical trials but they end up having to wait years for the approval, and meanwhile they die.”

Frank Burroughs, co-founder of the Abigail Alliance for Better Access to Developmental Drugs

Frank Burroughs wishes that option had been open to his daughter, Abigail, who died from head and neck cancer at age 21 in 2001. After her death, he co-founded the Abigail Alliance for Better Access to Developmental Drugs in her name. The Lawton, Va., organization advocates for earlier access to drugs for terminal patients.

During the last months of Abigail’s life, her doctor told her about a drug in development that was performing well in trials at the time. It had targeted the same type of cancer Abigail had, but in the colon, not the neck, so she was not eligible to participate.

“What this is saying is that there are promising drugs that you can identify as drugs that need much earlier approval,” Burroughs said. “The people that we hear from are talking about drugs that have shown very significant efficacy in clinical trials but they end up having to wait years for the approval, and meanwhile they die.”

Other drugs that the Abigail Alliance has supported have been approved for use in Europe but are still awaiting a nod from the U.S. government.

While the Abigail Alliance supports state efforts for right-to-try legislation, it focuses much of its energy on the national level, trying to persuade Congress and the Food and Drug Administration to speed up approval of promising drugs.

Patients do have other avenues they can pursue to obtain investigational drugs. They can enroll in a clinical trial or ask their doctors to file an application with the FDA for a single investigational or compassionate use.

None of these is easy, however, supporters of right-to-try legislation argue.

Only about 3 percent of those who have the medical conditions that would make them eligible for clinical trials are accepted, said Kurt Altman, national policy adviser and general counsel for the Goldwater Institute.

Still, passing right-to-try legislation could also detract from participation in clinical trials, said IU’s Helft. And clinical trials exist in a very different context than individual use, he added

“I have no problem with patients enrolling in early-phase trials to get drugs, because those trials are done in a systematic way so we actually get to learn something and they’re done with informed consent,” he said. “If patients have relatively unfettered access to drugs that could interfere with future trial participation … it would be much easier to go out and get the drug rather than participating in a clinical trial.”

But Burroughs says the idea is not to supplant clinical trials but to offer an alternative to those who, like his daughter, cannot get into a study and have no other way to access the drug.

Four and a half years after Abigail died, the drug she sought did receive approval for use in treating the type of cancer that she had.



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