In November, Arizona voters will decide whether terminally ill patients should be allowed to take drugs that are in the early stages of testing but haven’t yet been approved by the U.S. Food and Drug Administration. Similar “right to try” laws have been enacted this year in Colorado, Louisiana, and Missouri.
The laws aren’t a response to demands from patients or lobbying by big drug companies. They’ve been promoted by the libertarian Goldwater Institute, named for Senator Barry Goldwater, the late Arizona Republican. “Our objective is to look at different problems out there and come up with solutions that are liberty-based solutions,” says Victor Riches, the group’s vice president for external affairs. “These individual patients have a right to try a medication to try to save their own life.”
The FDA’s modern drug approval process dates to 1962, when Congress began requiring companies to conduct rigorous clinical trials to prove new medicines were safe and effective. That law was passed amid outrage over thalidomide, a sedative prescribed to pregnant women suffering from morning sickness that caused thousands of birth defects in Europe, Canada, and the U.S.
Free-market absolutists have long said the FDA does more harm than good by standing between the public and medical advances that could save lives. In a 1973 Newsweek column, the economist Milton Friedman argued for repealing the 1962 law, saying “the cost of delaying a beneficial innovation is something like 10 to 100 times the value of avoiding a thalidomide-type mistake.”
Friedman suggested abolishing the FDA. The Goldwater Institute has instead focused its campaign on cutting bureaucratic red tape for patients. It’s hard for a politician to object to giving terminally ill people access to potentially lifesaving treatments. No state lawmaker has voted against the right-to-try bills in the three states where they’ve passed. The Goldwater Institute deliberately picked a Democratic-controlled state, Colorado, a Republican one, Louisiana, and one with a divided government, Missouri, for the initial wave of legislation. Its most vocal ally in the medical industry is Richard Garr, chief executive officer of Neuralstem, a biotech company working on a stem cell treatment for ALS, or Lou Gehrig’s disease. Garr, who describes himself as “a lifelong knee-jerk liberal Democrat,” says the right-to-try laws don’t undermine the work of the FDA, because they apply only to therapies that have passed the first phase of human testing in FDA-approved trials.
Regulators and others in the pharmaceutical industry say sidestepping the later stages of testing exposes patients to unknown hazards. The first stage, usually involving only a few dozen subjects, is designed to learn whether a compound is safe for consumption. The laws would give patients medicines before rigorous second- and third-phase trials have established their safety and efficacy compared with placebos. Those later-stage studies help scientists determine proper dosages and understand how a drug affects sick patients who may be taking other medications. “These products have serious risks, and maybe even more concerning, we don’t even know what the risks are,” says Coleen Klasmeier, a former attorney for the FDA who represents drug companies at Sidley Austin. “You can’t just say, ‘Sure, take it. It might kill you, but vaya con Dios.’ ”
The new laws can’t compel pharmaceutical companies to make their newest drugs available before they’ve been vetted. It takes about 10 years and $1 billion to win FDA approval for a compound, and only 16 percent of treatments that begin clinical trials ultimately hit the market. Giving an experimental drug to terminally ill patients could tarnish the safety profile of a new drug out of the gate. “We have serious concerns with any approach to make investigational medicines available that seeks to bypass the oversight of the Food and Drug Administration and clinical trial process,” Sascha Haverfield, vice president for scientific and regulatory affairs at the Pharmaceutical Research and Manufacturers of America, wrote in an e-mail.
It’s not clear whether any patients have taken advantage of the right-to-try laws. People seeking experimental treatments can already enroll in clinical trials sanctioned by the FDA, though people with advanced disease may not qualify. The agency also has a “compassionate use” process to get early-stage drugs to people who don’t qualify for trials. About 1,000 people receive treatment under that program each year, says the FDA, which has not taken a position on the right-to-try laws. “While the agency is supportive of patients’ access to experimental new treatments, FDA believes that the drug approval process represents the best way to assure the development of safe and effective new medicines for patients,” spokesman Jeff Ventura wrote in an e-mail.
The Goldwater Institute’s Riches says the existing process is too cumbersome. “These are patients with terminal illnesses,” he says. “It still takes a number of months to get access to medications.” Whatever the outcome of the vote in Arizona, Goldwater says it plans to push right-to-try policies in more states next year. In the meantime, the campaign may add to pressure on the FDA to shorten the time it takes to get new drugs from the laboratory to the pharmacy counter—a goal the drug industry and patients share.