Less than two years after graduating college, Kim Suozzi ran out of options.
Her glioblastoma — a form of brain cancer — had returned after surgery, radiation and chemotherapy. She participated in two clinical trials, but the cancer persisted.
“She just had nothing else to do,” said her boyfriend, Josh Schisler, “but just to die.”
After her death in January 2013, Schisler returned to Jefferson City, where he’s now a legislative aide to Republican Rep. Eric Burlison of Springfield. He hoped to persuade lawmakers to pass legislation giving terminally ill patients the chance to try yet-unproven drugs.
Two bills in the General Assembly would let them take drugs that have only passed the first stage of the U.S. Food and Drug Administration’s approval process. That means they’ve been tested for safety on laboratory animals, but their effectiveness on humans remains unknown.
would allow patients to take such drugs after they’ve given written consent and received a recommendation or prescription from a physician. Arizona, Louisiana and Colorado are also considering right-to-try legislation. No state has passed such a law.
“It would make a big difference for people,” Schisler said, “even if it was just in the form of hope.”
Opponents say right-to-try laws would bypass protections in the federal approval system designed to protect patients from the dangers of untested drugs. Most drugs that clear the first phase of testing later fail to achieve FDA approval, they say. Those pharmaceuticals might prove ineffective or carry dangerous and unintended consequences, they warn, including premature death.
“For every home run you might hit with accessing a drug after just Phase One, you’re going to get a lot of drugs that don’t work that have some terrible side effects,” said Jann Bellamy, a Florida lawyer who has campaigned against right-to-try legislation.
Bellamy said drugmakers supplying experimental substances could find themselves running afoul of FDA rules. And she’s argued that Congress has made drug approval the jurisdiction solely of the FDA.
The Missouri legislation passed the House, has cleared a Senate committee and awaits action in the full Senate.
FDA spokeswoman Stephanie Yao said in an email that the agency held no position on the legislation. The agency sometimes grants patients access to experimental drugs, but only under rigorous rules that require they exhaust alternatives with already-approved drugs. Right-to-try bills would offer a faster route to eligibility for using the experimental drugs.
Sen. Rob Schaaf, a St. Joseph Republican and physician sponsoring a right-to-try bill, called the existing federal process making experimental drugs available to patients “inadequate.”
“Just because there’s the side-stepping process doesn’t mean that it’s completely open,” Schaaf said. “We need to make it so that if you’re terminally ill and you’ve tried everything and your physician has suggested that there’s an experimental drug that provides some hope for you, that you ought to be able to get that.”
The FDA’s expanded-access process is complicated and time-consuming, said Christina Corieri of the conservative Goldwater Institute, an Arizona-based think tank.
“Medical decisions belong to doctors and patients,” Corieri said. “There is no need to have a bureaucrat in that decision-making process.”
Burlison, the chairman of the House committee that passed the bill, said he thinks the FDA has good intentions but wondered “where is the logic when it comes to people that are terminally ill, that have no choices left?”
Efforts to push forward similar legislation started in Congress in 2008, Corieri said, but the legislation has never had a hearing.
Christian Tadrus, president of the Missouri Pharmacy Association, said he is torn by the idea. Pharmacists want to help the sick, he said, but right-to-try could place them in the uncomfortable position of dispensing drugs of uncertain value and safety.
“How do we do this in a way that is helpful for patients,” he said, “but is yet not doing more harm to them?”