Mikaela Knapp, 25, died of kidney cancer on April 24, 2014 after being rejected for FDA clinical trials. A petition started by her husband, Keith, to allow her access to promising experimental drugs garnered 481,841 signatures. (Facebook)
Utah Gov. Gary Herbert and Indiana Gov. Mike Pence both signed bills on Wednesday that allow physicians to prescribe “investigational” medication that has made it through the first part of the FDA’s three-phase clinical trials process to terminally ill patients who have exhausted other options.
Joining Pence at the signing ceremony in Indianapolis was five-year-old Jordan McLinn, who has Duchenne muscular dystrophy (DMD), a fatal degenerative disease that has no FDA-approved therapies. However, Laura McLinn, the boy’s mother, said that there were promising new drugs being developed that might help her son.
According to the National Conference of State Legislatures, similar bills have been filed in 32 states and the District of Columbia so far this year.
On March 13, Gov. Asa Hutchinson signed the Arkansas Right to Try Act (SB4), which states that “patients who have a terminal disease do not have the luxury of waiting until an investigational drug, biological product, or device receives final approval” from the FDA.
The law grants immunity to pharmaceutical companies, doctors and hospitals who administer experimental drugs except in cases of “gross negligence or willful misconduct.”
On March 9, Gov. Matt Mead signed the Wyoming Right to Try Act (SF3), which passed both chambers of the state legislature with just two dissenting votes.
The law, which goes into effect July 1, also allows terminally ill patients who have “considered all other treatment options currently approved” by the FDA to be treated with “investigational” drugs or devices that have cleared the first phase of clinical trials. Insurance companies are allowed, but not required, to provide coverage for such treatment.
Last November, 78 percent of Arizona voters also approved Proposition 303, a “right to try” ballot initiative. Colorado was the first state to pass similar legislation in 2014.
“When someone is on their deathbed, the fact that FDA regulations would let them die rather than try has got to be one of the most inhumane policies of the federal government. Every state should nullify the FDA like this,” said Mike Maharrey, communications director of the Tenth Amendment Center, which supports “right to try” laws.
However, critics of “right to try” laws say that untested drugs could do more harm than good.
“They’re far more likely to harm patients than to help them,” Michigan oncology surgeon Dr. David Gorski blogged in November, accusing advocates of “shamelessly…play[ing] on people’s fears of Ebola to promote these bad laws.”
“Having passed phase 1 does not mean a drug is safe…If there’s one thing worse than dying of a terminal illness, it’s suffering unnecessary complications from a drug that is incredibly unlikely to save or significantly prolong your life and bankrupting yourself and family in the process,” Gorski added.
Other critics say the FDA’s job is to protect patients from potentially dangerous or ineffective drugs, and that it already has a mechanism in place that allows individuals who do not qualify for clinical trials access to experimental treatments.
The FDA began its first formal “expanded access” program in 1987 after receiving numerous complaints that only a few hundred out of the thousands of patients diagnosed with AIDS were allowed to participate in clinical trials.
A decade later, the FDA allowed terminally-ill patients to apply for its “compassionate use” program, which received 5,849 single-patient applications between 2010 and 2014, and denied only 33.
The FDA pointed out that it approved more requests for expanded access in 2014 than during any year since 2010, when the agency first began publishing statistics on the program. Last year, 1,843 requests for expanded access were received, the highest number since 2010.
But “right to try” advocates say the application process is so time-consuming and cumbersome that it discourages sick people and their doctors from applying, and many patients die before their applications are approved.
The Arizona-based Goldwater Institute, which developed model “right to try” legislation, published a 2014 policy report stating that “over a half million cancer patients and thousands of patients with other terminal illnesses die each year as the bureaucratic wheels at the FDA slowly turn.”
The criticism prompted the FDA to create a working group last December to “develop policies that would improve access to investigational therapies.”
And FDA Associate Commissioner Peter Lurie also announced in February that the agency would “provide a streamlined alternative” application for individual patients that would take only 45 minutes to complete, “compared to the 100 hours listed on the previous form.”
But Goldwater Institute president Darcy Olsen called the FDA process “an inhumane system that prevents the vast majority of Americans with terminal illnesses from accessing promising investigational treatments.
“Compassionate use should be the rule for everyone, not the exception,” Olsen said.