This year’s campaign to allow terminally ill patients to use unapproved drugs in hopes of improving their lives got its start with an Arizona advocacy organization, so it’s fitting that the year’s last push goes through the state.
Arizona will vote on Proposition 303 in November, which would allow dying patients to bypass the Food and Drug Administration (FDA) process if a manufacturer is willing to provide an unapproved drug. Colorado was the first to approve such a law earlier this year, doing so with overwhelming legislative support. Missouri and Louisiana soon followed, and Michigan’s legislature approved a similar law earlier this month. Gov. Rick Snyder’s office didn’t return a message asking whether he’ll sign it, but the main force behind these laws, Arizona’s conservative Goldwater Institute, says legislators in Michigan have indicated Snyder backs the measure.
“When pushing [the bill] through the Michigan legislature, we were close to bill sponsors, who’d talked to the governor about a signing ceremony,” said Victor Richards, the Goldwater Institute’s vice president for external affairs. “My expectation is when those things are happening it’s a pretty positive sign.”
Goldwater spent this year finding sympathetic lawmakers to shepherd experimental drug bills through legislatures with a high success rate, and next year the group plans to launch efforts in about 10 new states, including Texas and Florida, depending on the outcomes of November’s legislative races.
Advocates argue that dying patients can’t wait on FDA clinical trials that can take a decade, and existing programs to allow patients to obtain unapproved drugs are still too slow for someone with little time left.
The laws allow for access to unapproved drugs, but with some caveats. The drug must have passed initial toxicity and dosage testing under the FDA’s clinical trial process. Doctors also can’t prescribe an unapproved drug unless the patient has exhausted other options. Manufacturers are under no obligation to provide the drugs and insurers aren’t required to pay for them.
While the laws have received bipartisan support in state legislatures, they’re not without detractors. Critics in academia and medicine argue they’re premised on false hope, because less than 10 percent of drugs actually make it through clinical trials and few manufacturers will risk defying the FDA by giving out unapproved drugs. Also, critics say, the laws could undermine clinical trials by discouraging participation, in which some participants receive placebos instead of the drug under examination.
“These laws are based on the fantasy that there are all these drugs out there that are just waiting to help these patients if it weren’t for the jackboots of the FDA keeping it from them,” said David Gorski, a doctor who specializes in breast cancer and edits the Science-Based Medicine blog, in a previous interview.
Supporters, including Arizona’s top newspaper, dispute that the laws could hurt the clinical trials that evaluate a drug’s effectiveness because terminally ill patients don’t typically qualify for participation anyway. “People who, for lack of a better way to put it, are too far along are generally ineligible for clinical trials,” Richards said. “If you’re in a situation when you’re down to your last two or three months of life, the clinical-trial process is not going to be of much use to you at that point.”
The FDA has a program known as “compassionate use” that allows for experimental drugs for even seriously ill patients, and the program rarely denies an application, but “right-to-try” supporters again argue that the process is too complex and slow, as evidenced by application figures averaging about 1,000 a year.
Arizona is the first state to refer legislation to a popular vote. What little polling there is on the issue comes from a conservative firm that’s sympathetic to Goldwater’s cause, but it shows overwhelming popular support. While Arizona Democrats mostly opposed the measure in the House, unlike in other states, there’s little organized opposition to the measure or money to put it down. That puts the chances of success in favor of the Goldwater Institute, but the group isn’t taking it easy.
“We’ve seen a lot of times where people [here] got complacent and what seemed like a foregone conclusion ended up failing,” Richards said.