The Right to Try

‘Right to Try’ laws would allow dying patients access to non-FDA-approved treatments

February 21, 2014

A bill allowing the terminally ill to use experimental drugs not yet approved for use by the Federal Drug Administration has advanced to the full House in Arizona.

Arizona lawmakers passed the bill out of committee and in the next three weeks both chambers of the legislature could vote on it. It would then need to be approved by voters in November.

The Right to Try law would give those who have exhausted all other treatment options the use of non-FDA approved drugs. According to the Goldwater Institute, similar laws are under consideration and in different stages of the legislative process in seven other states.

Supporters of the bill are optimistic it will become law in Arizona and that other states will follow suit.

"This is a fundamental right for people to have," said Christina Corieri, a health care policy analyst at the Goldwater Institute, which helped craft the law. "They have a fundamental right to save their own life, and there shouldn’t be a bureaucrat in the picture."

Corieri said patients themselves should have the right to decide if they want to take these drugs, which may or may not have potential side effects.

"We know the side effect that will occur if they don’t access these drugs—they are going to die," said Corieri.

Frank Burroughs, cofounder of the Abigail Alliance, which advocates for wider access to developmental drugs, said Arizona’s actions are a hopeful sign.

"I am very hopeful right now," said Burroughs. "At times, this business could feel very hopeless."

Burroughs’ daughter, Abigail, died at the age of 21 after exhausting all FDA approved drugs. Her access to the drug Erbitux was denied, even though it was showing promising signs in early clinical trials.

The FDA approved Erbitux three-and-a-half years after Abigail’s death.

Burroughs said he believes Abigail would be alive today if she had access to Erbitux.

"She had a significant chance," he said, noting the drug was showing efficacy early on in Phase II trials and that the data was promising.

Every drug for cancer and other life-threatening illnesses that Burroughs’ group has pursued for earlier access for the last decade has been approved by the FDA. He said many lives could have been saved or extended if there had been earlier access to these drugs.

Jenn McNary’s sons, Austin, 15, and Max, 12, both have Duchenne Muscular Dystrophy (DMD), a disease that reduces life expectancy to the late teens and early 20s. Max took part in the clinical trials for the drug eteplirsen when he was expected to be wheelchair bound within six months.

Max is now playing soccer and "running to his friend’s house," McNary said.

Austin, who didn’t qualify for the trial, is in a wheelchair. His condition is deteriorating.

"The fact is he is dying anyway," McNary said, explaining why he should have access to eteplirsen.

The drug is now in Phase II trials and numerous experts attended a congressional briefing on February 10 in Washington saying eteplirsen is extremely promising and an "amazing feat."

"Austin is angry," McNary told the Free Beacon. "He told me, ‘The FDA is heartless and they don’t care that I need the drug right now.’ "

"He knew it worked. He looked at his brother and saw it worked," McNary said. "When you have a terminal illness, you have nothing to lose."

"This is their last chance," McNary said. "If we don’t get these boys on this drug, it’s a death sentence for our kids."

When contacted for comment, an FDA spokesperson said in an email statement they are prohibited by law from discussing any drug application under review or the status of any investigational drug.

"The FDA is aware that DMD is a serious, rare genetic disorder that severely affects patients, often at a very young age. Currently, there are no FDA approved therapies to treat this disease," the statement read. "Since eteplirsen is an unapproved drug, we cannot share information about the status of any clinical trials or applications for eteplirsen. However, please know that we will work diligently to review any request made to expedite its review."

"The FDA has a long history of approving drugs for rare and life-threatening diseases, and has many processes in place to speed access to promising new treatments," the statement said.

If eteplirsen is not accelerated for approval by the FDA, it could be 2018 before it is available for use.

"These people have a ticking clock above their heads. They don’t need bureaucrats standing in the way," Corieri said.

Published under: FDA