Dying patients want quicker access to experimental drugs that could extend their lives, and 37 states, including Minnesota, have enacted laws since 2014 granting that wish.
Supporters say a federal law would strengthen protections, and Congress is now considering the issue. That’s focusing debate on whether such “right-to-try” laws have practical positive effects or just create false hope while undercutting patient safety. The conversation is growing in importance as the U.S. population ages.
The dispute also is an early test of the Food and Drug Administration’s direction under new Commissioner Scott Gottlieb and President Donald Trump.
The laws allow the use of drugs and devices that have passed preliminary FDA safety tests but haven’t been approved for use. Vice President Mike Pence signed Indiana’s right-to-try law in 2015 during his tenure as governor. He met with proponents of a federal law in February, pledged the president’s support and said, “We’re going to get this done.”
At a January meeting with pharmaceutical executives, Trump promised to change “a lot” of FDA rules. “One thing that has always disturbed me, they come up with a new drug for a patient who is terminal and the FDA says, ‘We can’t have this drug used on the patient,’ ” the president said.
Gottlieb, a physician and Hodgkin’s lymphoma survivor, has not taken a position on right to try, said FDA spokeswoman Jennifer Rodriguez. But at his April confirmation hearing, he said that the agency sometimes displays an “unreasonable hunger for statistical certainty” and that it was sometimes too cautious in reviewing new drugs.
Years away from approval
Colorado became the first state with a right-to-try law in 2014. The state laws are based on legislation written by the Goldwater Institute, a think tank based in Arizona that promotes limited government.
It’s unfortunate, said spokeswoman Starlee Coleman, “that so few people in this country have access to medications that are being safely used in clinical trials but are years away from [FDA] approval.”
The FDA now allows patients access to experimental drugs via clinical trials run by drug companies and overseen by federal regulators. It also allows patients to apply for access to experimental medications outside trials. Since 2009, the FDA says, it has approved 99 percent of those applications. But right-to-try proponents say that “expanded access” or a “compassionate use” option is too slow and requires cumbersome paperwork.
“My goal is that at no point is someone told that there isn’t anything that can be done — not because the science says so or that the medication says so but because bureaucrats say no,” said Minnesota state Rep. Nick Zerwas, R-Elk River, who sponsored right-to-try legislation that became state law two years ago. Zerwas survived a serious heart condition because he was able to undergo experimental surgery.
He believes a federal law is needed to add patient protections, clarify how the process works and define how insurance plans are affected.
‘Waiting for government’
Wisconsin state Rep. Patrick Snyder, a Republican who sponsored a right-to-try bill that passed the Assembly and is pending in the Senate, agrees. “Patients at the worst time in their life should not be waiting for government to grant them access to a potentially lifesaving drug,” said Snyder, who has worked with dying patients in a hospice.
Juran Cook, a New Berlin, Wis., nurse, called state and federal laws “essential.” The death last October of her husband, Mitch Cook, 58, from amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease) made her a right-to-try activist.
Education is needed to make patients and their doctors aware of their rights under state laws, she said. Cook rejects critics who say that the laws offer only wishful thinking to dying patients.
“There is no such thing as false hope,” she said. “You either have hope or you’re helpless.”
Her husband found what she called “a glimmer of hope” the month he was diagnosed — three years before his death — when they learned about a trial of an experimental stem cell and gene therapy for ALS at a California hospital. Last October, the month he died, the researcher received FDA approval for the therapy.
The Pharmaceutical Research and Manufacturers of America (PhRMA), an industry group, has not taken a position on right to try, said spokeswoman Caitlin Carroll.
“Any legislation,” she said in a statement, “should protect the integrity of clinical trials and the FDA oversight of expanded access to maintain the best interests of patients.”
Some skeptics call right-to-try laws pointless.
“I see right to try as feel-good, hollow legislation,” said Arthur Caplan, a New York University bioethicist who formerly taught at the University of Minnesota. He’s chairman of a working group on ethical issues involving access to medications before FDA approval.
He described what he sees as a significant flaw in the laws: Drug manufacturers, not the FDA, control access to experimental drugs, and they often don’t want to risk eventual FDA rejection of medications they’ve spent years and billions of dollars developing if something goes wrong.
Removing the FDA from the process for terminal patients, he said, could open the door to “rip-off artists” peddling drugs that they know won’t work.
What’s really needed, Caplan said, are advocates who can help patients figure out what clinical trials are available and help them apply. “One of the ways to help is to think about designing trials that are more inclusive so that more people can get into them,” Caplan said. “Right now, trials exclude those who are too old or too sick.”
Elizabeth Richardson, a managing associate at Duke University’s Margolis Center for Health Policy, said the laws are “tackling something that isn’t really the problem … based on the assumption that the FDA is the real barrier.”
The agency, she said, has accelerated its response to compassionate-use requests.
Like Caplan, Richardson would like to see more support for patients who enroll in drug trials, including guidance on weighing benefits and risks.
Some states are enacting laws that go beyond the standard right-to-try language.
A California law that took effect Jan. 1 requires patients to get a confirming diagnosis by a second doctor, creates a review board for right-to-try requests and asks patients to sign waivers accepting the risks of experimental drugs.
“If I had a terminal illness, I would want every opportunity to swing for the fences to try to save my life,” said the California bill’s sponsor, Rep. Ian Calderon, a Democratic leader in the state Assembly.
Noting that critics say that states can point to few, if any, patients whose lives have been extended under the laws, Calderon said, “One person sometime will benefit. … That makes the bill worth it.”
In Texas, Rep. Drew Springer, a Republican, sponsored a measure that gives patients with chronic or terminal diseases access to “investigational stem cell treatments” that don’t have FDA approval. Adult, not embryonic, stem cells would be used. Republican Gov. Greg Abbott signed the bill last month. It takes effect Sept. 1.
Springer called the bill a logical extension of the state’s right-to-try law and said that he and his cosponsor, Republican Rep. Tan Parker, will lobby former Texas Gov. Rick Perry, now Trump’s energy secretary, to ask the FDA to consider a similar proposal.
Springer’s interest is personal. His wife, Lydia, injured her spine 28 years ago and uses a wheelchair. The bill, he said through tears during the legislative debate, “might give somebody like my wife a chance to walk.”