In DepthPatient Advocacy

‘Right to Try’ laws bypass FDA for last-ditch treatments

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Science  20 Jun 2014:
Vol. 344, Issue 6190, pp. 1329
DOI: 10.1126/science.344.6190.1329

Colorado Governor John Hickenlooper signed the first Right to Try law in May.

PHOTO: ALEX WONG/GETTY IMAGES

Several state legislatures, prodded by desperately ill patients and a libertarian think tank, are defying federal regulators to make experimental drugs available outside of clinical trials. The new laws, called Right to Try, allow terminally ill patients to request unapproved drugs after early-stage testing. Last month, Right to Try went on the books in Colorado and Louisiana; a similar law awaits the governor's signature in Missouri, and Arizona will put the issue to a vote in November.

The movement taps into long-standing frustration over what some view as the sluggish pace of clinical trials. Although the laws are framed as a boon for dying patients, critics raise a long list of caveats. Companies, they say, will often be reluctant to offer a drug outside of its clinical trials—and the untested therapies will rarely work. Only about one in 10 drugs in phase I clinical trials ever demonstrate efficacy to win Food and Drug Administration (FDA) approval. “It sounds like, ‘We're throwing you a life preserver, and you'll have your shot at being cured,’” says Arthur Caplan, a medical ethicist at New York University's Langone Medical Center in New York City. “I can't tell you how rare that scenario is.”

Rare it may be, supporters say, but not impossible. “Any patients alive today with ALS will most likely be dead by the time we're finished with our trials,” says I. Richard Garr, president of Neuralstem Inc. in Germantown, Maryland, whose stem cell treatment for the fatal neurodegenerative disease is in phase II clinical trials. His company plans to take advantage of Colorado's Right to Try law to make its unapproved treatment available to ALS patients, well ahead of any FDA decision.

Right to Try laws are being spearheaded by the Goldwater Institute, a libertarian group based in Phoenix. The laws require that patients be terminally ill, have considered other available treatment options, and be unable to participate in a clinical trial. The institute describes the laws as a “complement” to an existing expanded access program at FDA, which allows patients with serious or life-threatening diseases to try experimental drugs if they've exhausted their other options. But with expanded access, an institutional review board must also approve each request, and the patient's doctor must report follow-up data to FDA.

According to FDA's submission receipts report for fiscal year 2013, the agency granted all but three of nearly 1000 expanded access requests it received. When patients can't access experimental treatments, the holdup is often the drug developer, not FDA, Caplan says. Companies cannot be forced to supply an experimental treatment outside a clinical trial, and often they don't wish to do so. Manufacturing more of a drug can strain a company's resources, says Emil Kakkis, CEO of the biotech company Ultragenyx Pharmaceutical Inc. in Novato, California, and president of the EveryLife Foundation for Rare Diseases, which promotes innovation for rare disease treatments.

Companies also worry that a bad outcome from FDA's expanded access will torpedo a therapy, even though the agency maintains that this has never happened.

The fervor over Right to Try has some patient advocates worried that existing options for dying patients will be overlooked. A one-person expanded access request through FDA is an uphill battle, but the system can accommodate larger groups, says Jess Rabourn, director of the ALS Emergency Treatment Fund in San Jose, California, which helps biotech companies apply for and fund expanded access programs.

Legally, Right to Try raises major questions. Federal law limits moving drugs or their components across state lines without first applying to FDA, notes Meredith Manning, a lawyer specializing in the FDA approval process at Hogan Lovells in Washington, D.C. “If FDA were to assert” that the federal law takes precedence, “it would have a good argument,” she believes. So far, FDA has not taken a public position on Right to Try, and agency officials declined to comment on the laws for this story.

“I don't think that patient populations and foundations and advocates should be duped into thinking that this is something that it's not,” Rabourn says. “It's not something that changes the federal law, and that's the law of the land that governs drug development. It's what any shareholder-owned drug company has to abide by.”

Garr says that if FDA forbids the company from offering its unapproved treatment, an injection of neural stem cells into a patient's spinal cord, he'll follow the rules. Still, Garr, who accepted an invitation to join the Goldwater Institute's Right to Try National Advisory Council, says he believes in offering the treatment to patients who want it. The inclusion criteria will be almost identical to those of the ongoing trial: Patients must be in the early stages of ALS. So far, no other company has said it will follow Neuralstem's lead.

Meanwhile, Garr says, “we're already getting tremendous numbers of people writing, saying they're ready to move to Colorado tomorrow.”

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