Saying some chance is better than none, a House panel voted Thursday to let terminally ill patients get drugs that have not yet been approved for use.
The 5-3 vote by the House Committee on Reform and Human Services came after testimony from Steven Walker about the cancer death of his wife, Jennifer McNeillie more than a decade ago. Walker, co-founder of the Abigail Alliance for Better Access to Developmental Drugs, said she was denied access to drugs then considered experimental but since approved by the U.S. Food and Drug Administration.
“The FDA was much more focused on statistical minutiae,” he told lawmakers. “They were more focused on the process than delivery.”
Rep. Phil Lovas, R-Peoria, told his colleagues they can see a real-world example for themselves just by watching “Dallas Buyers Club,” a movie which shows how terminally ill AIDS in the 1980s patients had to go outside the country to find the medications to save their own lives.
“We hear stories of Americans going overseas to get treatment,” said Lovas, sponsor of HCR 2005. “Unfortunately, this is restricted very many times to the very wealthy who can afford that treatment and can afford the travel costs to do so.”
Thursday's vote is just the first step in the process. Aside from still needing full House and Senate approval, it is crafted to give voters the last word: The measure would be placed on the November ballot.
Lovas said he is not critical of the FDA, saying it is doing its job of ensuring that drugs are safe and effective. But he said the process can take up to 15 years.
“Terminally ill patients don't have that time to wait,” he said.
Rep. Sally Gonzales, D-Tucson, said she is sympathetic, having had a father and two brothers who died of cancer and being a cancer survivor herself.
She cautioned, though, against opening the door. She said many of these drugs in early approval process never get beyond that because they're found either to be ineffective or perhaps even outright dangerous. And Rep. Juan Mendez, D-Tempe, warned against giving patients “false hope.”
But Rep. Kelly Townsend, R-Mesa, said she thinks that the legislation, with a bit of tweaking, can be narrowed so that these drugs are available only to those terminally ill patients who have tried other available options without success. And she brushed aside concerns that an experimental drug might cause harm.
“Obviously, they don't have much time to live anyway,” she said. “So what's the alternative?”
Nor was Townsend persuaded by concerns of giving patients “false hope.”
“The person has no other hope,” she said.
“They should have that ability to make that decision on their own to go ahead and make that last-ditch effort,” Townsend said. “That's their human right to do that.”
Kurt Altman, an attorney for the Goldwater Institute, assured lawmakers that there would be no liability to the state for allowing terminally ill patients to take experimental drugs that might actually harm them. He said existing laws already require there be "informed consent'' by a patient or caregiver before any drug can be prescribed.
The measure is limited to those who have a “terminal illness.” That is defined in the legislation as “a disease that, without life-sustaining procedures, will result in death in the near future or a state of permanent unconsciousness from which recovery is unlikely.”
It also would not permit an individual to get – or a drug company to provide – just any drug that is being evaluated. They would had to have completed at least what is called Phase 1, meaning the manufacturer has tested it on a small group of people to evaluate safety, determine a safe dosage and identify side effects.
Mendez, quoting a paper by Dr. Dean Gesme, a Minnesota oncologist, said measures like this generate false hopes in patients.
“The reality is, less than 10 percent of drugs beginning Phase 1 trials are eventually adopted as viable treatment,” Mendez said. He also said that providing this access would hamper cancer research.
“Patients would have little incentive to enter Phase 2 and Phase 3 clinical trials which are our only means to determine side effects and efficiency of new drugs,” he said.
Walker dismissed the idea that there are other options for terminally ill patients, like trying to get into one of the experimental studies. Aside from entry being “highly restrictive,” he said the aim of those studies is not to help participants.
“And we know that's true because a lot of those patients end up going into control arms where they get nothing but a sugar pill,” Walker said. “So these trials are not for patients. They're for learning.”