Patients and families rally for expanded access to experimental drugs in Washington, D.C. on May 11, 2015. (Courtesy of Lina Clark)
With the enactment of a new “Right to Try” law, California joins 31 other states that have already passed legislation to support patients’ efforts to access experimental drugs.
As of Jan. 1, state agencies and licensing boards will not penalize California doctors or hospitals that want to help patients directly petition pharmaceutical companies for the investigational drugs.
Proponents of the “Right to Try” movement had contended that doctors could be hesitant to help such patients because of the risk of using drugs or devices that haven’t passed FDA tests for safety and effectiveness.
The new law makes it clear that California doctors can proceed without fear of censure from the state’s medical board. It also eliminates other possible regulatory obstacles on the state level and creates processes for patient consent and data collection.
"The idea is to kind of get government out of the way in this situation and allow people to just go at it with a doctor to try to get access to these potentially life-saving medications,” said the law’s sponsor Ian Calderon, the Democratic majority leader in the California Assembly.
Supporters of “Right to Try” laws sometimes frame them as the logical counterpart to “Right to Die” laws, which allow physician-assisted suicide for terminally ill patients. California enacted such a law in 2016.
"The patient community is saying, ‘We are smart, we’re informed, we feel it is our right to try some of these therapies, because we’re going to die anyway,’” said Lina Clark, a patients’ rights activist in San Diego.
The FDA already offers a process, called “expanded access” or “compassionate use,” for some patients to obtain experimental drugs. But critics say it’s too slow and cumbersome, even though the FDA approves more than 99 percent of requests that come through that pipeline.
“It just takes so long, it’s inefficient, it’s ridiculous,” Calderon said of the FDA process. “I don’t understand.”
As the state laws proliferate, clinicians and medical ethicists are growing increasingly concerned about the potential safety and financial risks to patients.
“If we take the FDA out of it, how do we protect people from physicians or drug companies that will want to sell them things and will want to prey on their desperation?” asked Dr. R. Adams Dudley, director of UCSF’s Center for Healthcare Value.
Dudley says the FDA and the clinical trial process were put in place for a reason.
"Have we somehow gotten better at managing snake oil salesmen now, than we were back when we decided we needed an FDA in the first place?" Dudley said.
Under the new law, all that patients and doctors can really do is ask for an experimental drug. Drug companies don’t have to give it to them, and insurance companies don’t have to pay for it.
Dudley fears that family members of a patient might exhaust their life savings trying a drug that hasn’t been proven to work -- and could even speed up death or increase end-of-life suffering.
“It basically turns patients -- terminally-ill patients -- into guinea pigs,” said Don Nielsen, the CNA's director of government relations.
“There’s really no demonstrated need for this,” Nielsen said. “This so-called "Right to Try" movement is nothing more than boilerplate legislation promoted by the far-right Goldwater Institute.”
(While the Arizona-based Goldwater Institute has taken an active role in promoting the laws, in California and other states, Calderon insists he came upon the issue independently, while researching physician-assisted suicide for California.)
It’s unclear how effective the state laws have been. There’s no data yet on how many patients have used the state laws, compared to the FDA’s expanded access process.
Dudley predicts the state-based Right to Try laws may eventually face a court challenge and be declared unconstitutional. That’s because federal law gives the power to regulate pharmaceuticals to the FDA, not the states.
But Clark thinks Congress might be open to amending that authority.
“It’s kind of parallel with what’s going on with medical marijuana,” Clark said. “It’s legal at the state level, but we still have a federal Drug Enforcement Agency to whom it is illegal. It’s not an ideal situation. It’s kind of similar to that. We ultimately need a federal law.”
A federal version of Right to Try was introduced in the Senate last year. In the meantime, a recently passed federal law addressing medical research, the 21st Century Cures Act, contains a provision that requires drug companies to be more transparent about how they decide who gets access to experimental drugs and how long it will take.
Editor's note: The original version of this article incorrectly stated the California "Right To Try" law allowed access to an experimental drug at any stage. The law actually only allows a patient to request a drug that has passed through Phase I of the FDA clinical process, when a candidate drug is tested for toxicity on otherwise healthy subjects.