Giving terminally ill patients access to not-yet-approved drugs as a last resort seems compassionate, but critics say an Arizona referendum on the issue is not as black and white as it sounds.
Detractors say Arizona Proposition 303, known as the “Terminal Patient’s Right to Try” referendum, could give patients false hope because the chances of doing harm with unapproved pharmaceuticals far exceeds any potential benefit.
Additionally, critics say such a law could open the doors for greedy quacks to prey on desperate patients with expensive medications that don’t work.
And while many opponents agree the federal drug approval process can be frustratingly slow, they also say any state attempt to circumvent federal law is ill-advised.
Proponents say a Right to Try law is a common-sense move to give patients with a terminal diagnosis access to experimental drugs not yet approved by the U.S. Food and Drug Administration (FDA).
Similar laws have already passed in Colorado, Missouri and Louisiana.
Last-ditch treatment with experimental drugs could extend or save lives, said Victor Riches, vice president of the Goldwater Institute, a conservative Phoenix-based group that have given $70,000 toward the campaign to pass Proposition 303.
Supporters say much of the criticism underestimates patients and their families.
The law requires drugs to have gone through Phase One of the FDA process and be part of a clinical trial — so they are not completely untested, Riches emphasizes. The problem is the FDA approval process is long and cumbersome, and patients who want last resort experimental drugs don’t have time on their side, he said.
The proposition, placed on the Nov. 4 ballot by the Legislature, leaves it up to individual insurance companies as to whether they cover investigational drugs, Riches said.
“Our strong belief is that the right to try to save one’s own life is a fundamental constitutional right,” Riches said. “It’s a very simple proposal and it’s just about giving these terminally ill patients the ability to save their own lives.”
Patient’s Right to Try laws are often known as “Compassionate Access,” or “Dallas Buyers Club” laws — a reference to a group established by Dallas resident Ron Woodroof in the 1980s for purchasing unapproved treatments for HIV/AIDS, which became the subject of the 2013 Oscar-winning film.
“There is no scientific data whatsoever that the cocktails they were giving out at the Dallas Buyers Club were helping anybody,” said Joan Koerber-Walker, president and CEO of the Arizona Bioindustry Association. “To tell patients who are terminally ill that they have a right to try and not give them a pathway to succeed is wrong.”
The Arizona Bioindustry Association is not taking an official position, but Koerber-Walker says Prop. 303 “grossly oversimplifies” a very complicated issue.
“Unfortunately under Prop. 303 there are no provisions for oversight, nothing to make sure people aren’t taking advantage of patients,” Koerber-Walker said.
She says there are existing mechanisms for terminal patients that are far more well thought-out, including an accelerated drug-approval process and an existing expanded access program offered by the FDA through an application process, though Prop. 303 supporters say that program is not doing enough for patients, and there’s too much red tape.
While Riches says support is bipartisan, much of the opposition in the Arizona Legislature came from Democrats such as Rep. Sally Ann Gonzales, D-Tucson, who said Right to Try is pre-empted by federal law.
Dr. David Gorski, a surgical oncologist based in Detroit, has spoken up against Right to Try proposals nationwide, including Prop. 303 in Arizona. Gorski, a surgery professor at Wayne State University and managing editor of the “Science-based Medicine” blog, says most investigational drugs that make it past Phase One trials still end up failing.
Gorski writes he finds it hard to be charitable with “right-to-try” supporters since they appear to be mostly ideologically motivated by a dislike of federal regulation, or motivated by a support for quack medicine.
“Never forget that the reason the laws we have exist is to protect the public against drugs that don’t work or are too toxic and, just as importantly, from companies that would sell such drugs with no evidence of efficacy or safety,” Gorski writes.
But Prop. 303 spokesman Gibson McKay says patients and their families would be making up their own minds about whether to try a last-ditch treatment. There is a reason the unapproved treatments are often referred to as “what- the-hell” drugs, because there’s nothing more to lose.
Supporters of Right to Try feature patients in their campaigns.
One such family is the Morris family of Phoenix who sought help for their son’s rare bone cancer by traveling to get a drug treatment available in England. Their son, 13-year-old Diego Morris, is now cancer free.
The drug they wanted for him is called mifamurtide, and while it’s been approved by the European equivalent of the FDA, and other countries such as Mexico and Israel, it is not approved in the U.S.
The family tried to work with doctors in the U.S. and the drug manufacturer to gain access to mifamurtide, which, taken in conjunction with chemotherapy, may reduce the chance of a recurrence of cancer.
But they were unsuccessful and ended up living in England for nine months.
It’s impossible to know whether the mifamurtide worked, but the Morris family is happy they took what they believe is an extra protective measure for Diego.
“We think (Prop.) 303 makes sense. It allows you to make an informed decision to try and save your life,” said Diego’s mother, Paulina Morris. “People in London were shocked finding Americans getting care in another country. They think we have the access to everything.”