Following Colorado’s lead, a number of states are considering bills that would allow terminally ill patients to take medications that aren’t yet approved by the U.S. Food and Drug Administration (FDA). But critics say the laws could do more harm than good.
Colorado Gov. John Hickenlooper signed a so-called “right to try” bill into law last month following a unanimous vote in both chambers of the state legislature. With the permission of a doctor and the drug manufacturer (but not the FDA), the law allows dying patients to use unapproved drugs that have passed initial toxicity and dosage testing under the FDA’s clinical trial process.
Minnesota and Missouri have similar measures pending in their legislatures, while Arizona is putting the issue to a vote this fall as a referendum. Louisiana became the second state to enact such a law Monday. But efforts to pass federal legislation have failed to get off the ground in both the Senate and the House, most recently in 2010.
The idea, popularized recently by the film “Dallas Buyers Club,” is to give dying patients who have tried every other viable option and aren't typically eligible for clinical trials an opportunity to take promising new drugs.
“They can’t wait for a clinical trial,” said state Rep. Joann Ginal, a lead sponsor of the Colorado law. “They’ve exhausted all other options and this gives them hope to try new compounds.”
Ginal, a doctor who's overseen clinical trials for pharmaceutical companies, was personally touched by the issue when her brother recently died of blood cancer. He took experimental drugs that she said she believes helped prolong and improve his life.
In that sense, Ginal is like many of the people who have shared their stories before legislative committees and TV cameras, insisting that they know what’s best for their loved ones. To them, waiting years for a drug to complete all phases of an FDA clinical trial is too long and the existing process to obtain experimental drugs through the FDA is too difficult. The length of time varies, but it can take seven years or more to complete clinical trials, according to the FDA.
Drugs typically go through three phases in a clinical trial. The first assesses the appropriate dosage of a drug and dangers of toxicity with a limited number of participants. Under right-to-try laws, patients can get drugs without the FDA's permission after they've passed this phase.
The following phases try to measure the effectiveness through controlled studies. Only about 7 percent of drugs that enter clinical trials make it past the third stage, according to the FDA. The limited number of drugs that actually make it through the process fuels criticism that experimental drugs are actually more likely to offer false hope or even greater pain.
“These laws are based on the fantasy that there are all these drugs out there that are just waiting to help these patients if it weren’t for the jackboots of the FDA keeping it from them,” said David Gorski, a doctor who specializes in breast cancer and edits the Science-Based Medicine blog, in an interview.
Since 2009, the FDA's expanded access program -- sometimes called “compassionate use” -- has allowed patients who are either seriously ill or dying to use experimental drugs as a last resort if they have permission from both a doctor and the pharmaceutical provider. The fact that the FDA's program also includes the seriously ill, not just the terminally ill, makes it more expansive than right-to-try legislation, but the process is also longer.
Since fiscal year 2009, the FDA has gotten 1,037 annual requests, on average, and granted 1,031 on average. Right-to-try supporters say the low demand shows how difficult it is to navigate the FDA program, which requires extensive paperwork and verification from a patient’s doctor.
The Pharmaceutical Research and Manufacturers of America (PhRMA), a trade association, says the issue shouldn’t be taken up on a state-by-state basis. The group says it’s committed to helping patients and doctors understand the importance of clinical trials and better navigate the FDA's existing expanded-use process.
Some critics are concerned that right-to-try laws will eventually extend beyond the terminally ill and could lead to fewer people participating in clinical trials.
“Without patient participation in clinical trials, you cannot generate the safety and efficacy data needed for the FDA to approve new medicines for use by a broad patient population," said Sascha Haverfield, the vice president of Scientific and Regulatory Affairs at PhRMA.
The issue then becomes one of public health versus individual wants.
“The majority of cases I get asking for help are actually not from the terminally ill,” said Arthur Caplan, a bioethicist at New York University. “If those folks get added into the ‘compassionate use’ movement -- and trust me, there are many more of them than there are the terminally ill -- it will threaten the ability of companies to recruit people to their studies.”
The FDA isn’t taking a position on the Colorado law and won’t say whether it directly conflicts with federal law, which could expose participating manufacturers to liability. But there’s little question that the FDA is ultimately responsible for approving the use of experimental drugs, so companies will be hesitant to risk liability or jeopardize eventual approval of a drug that might one day yield huge profits, Caplan said.
“It’s said that the FDA doesn’t have compassion,” he said. “The market doesn’t have compassion. Investors -- angel investors, the stock market -- that’s not a place known for compassion."
Correction: This article previously included Louisiana among the states where legislation is pending. The state enacted a right-to-try law Monday.