Amid a tidal wave of Republican election wins, supporters of so-called right-to-try laws claimed victory as Arizona overwhelmingly approved a measure that will allow dying patients to get drugs and medical devices not yet approved by the Food and Drug Administration.
Arizona became the fifth state in the country to approve a right-to-try law, and the first to pass it by voter referendum. Lawmakers in Colorado, Louisiana, Missouri and Michigan have all passed similar laws by overwhelming majorities over the past several months, and the state Legislature in Nevada is expected to consider a bill next year.
The practical effect of the laws is unclear since they contradict federal statutes and regulations, leading the drug industry's leading trade group to oppose the approach. But proponents see Tuesday's outcome as further evidence of the public's support for such measures, which allow drug and device manufacturers whose products are still in the investigational stage to offer those therapies to terminally ill patients once those products have completed the FDA's Phase One clinical trials.
“I think it's really a sign that there's a desire from the public at large to do something to make sure that these terminally-ill patients have access to medication,” said Victor Riches, vice president of external affairs for the Goldwater Institute, a Phoenix-based, not-for-profit conservative research organization that has lobbied state governments across the country to pass similar measures. “I think there's a general consensus in the public that an element of the FDA's process is broken.”
The institute wrote the model legislation (PDF) that has served as the template for the right-to-try bills that have become law in those states. The group is leading a nationwide lobbying effort for states to adopt similar laws. Riches said the campaign has targeted as many as 10 states, including Texas and Florida, for similar measures to be introduced over the next year.
The organization has found that state legislatures are more likely than Congress to pass right-to-try legislation and that a critical mass of states adopting the laws would put pressure on the federal government to loosen its process for approving investigational drugs. But with Republican majorities now in the House and Senate, proponents may see a new opportunity to find lawmakers willing to take up the cause.
Critics see the state laws as little more than political statements about the burden of federal regulation.
“Legally, these laws are not effective at all,” said Henry Greely, a professor of law and director of the Center of Law and the Biosciences at Stanford University. “The state cannot overturn federal law that requires FDA approval for these uses.”
“It's purely an anti-government, political move that imposes no obligations on anyone other than impinging federal law,” Greely said. “Could it in the long term have political effect of leading to pressure to loosen compassionate-use limits of the FDA? Maybe.”
States that have embraced the laws have done so with overwhelming support. Arizona's right-to-try measure, known as Proposition 303, garnered nearly 80% of the vote. The margin was similar in Colorado in May, when the state Legislature passed a right-to-try bill with large bipartisan support, making it the first in the country.
“No one really wants to be seen as in opposition to letting the dying having a shot at a life preserver even though the odds are long,” said Arthur Caplan, director of the medical ethics division at NYU Lagone Medical Center. “Politically, that is not a good place to be, and I think conversely, this is an easy bill to vote for because it doesn't cost anything—it's a feel good bill.”
The FDA already allows for access to investigational therapies for terminally ill patients outside of a clinical trial under expanded or “compassionate” use on a case-by-case basis, and has granted approval in 99% of all cases. The biggest roadblock for patients, Caplan said, is lack of knowledge about that policy and lack of financial resources to pay for the experimental treatments. The right-to-try laws, Caplan said, do nothing to address those issues.
But right-to-try supporters contend the FDA grants compassionate-use permission only after a lengthy review process, which can take up to several months, an amount of time they say terminally ill patients may not have.
The state laws allow patients seeking an investigational drug to get a prescription from a physician after all other approved treatment options are exhausted. Drugmakers and anyone involved in providing and administrating the treatments are shielded from lawsuits and disciplinary actions by state regulators. State officials found to have blocked access to an eligible patient can be found in violation of a misdemeanor offense punishable by up to six months in jail.
The laws do not, however, require pharmaceutical companies to provide unapproved drugs and devices. Nor do they call for insurers to cover the costs of those treatments. In fact, payers would not be under obligation to cover any healthcare costs related to the experimental treatment, meaning patients could have to pay out of pocket for care they need if they have a bad reaction to it.
Few drugmakers have come out in support of right-to-try laws. Most of them, Riches said, prefer to remain neutral. But the Pharmaceutical Research and Manufacturers of America opposes the state laws.
“We have serious concerns with any approach to make investigational medicines available that seeks to bypass the oversight of the Food and Drug Administration (FDA) and clinical trial process, which is not in the best interest of patients and public health,” John Murphy, assistant general counsel for PhRMA said in an e-mail. “Legislation at the state level, however well intentioned, is unlikely to add any meaningful new approaches that can optimize the federal expanded access process overseen by FDA.”
Other concerns raised by critics include that broader access to investigational drugs will make patients less willing to participate in a clinical trial and risk being part of a control group that gets a placebo. Critics also point out that the laws don't compel health plans to pay for the drugs or any treatment patients need if they suffer harmful side effects.
And even though the laws insulate pharmaceutical companies from lawsuits, the manufacturers risk a public-relations nightmare if a patient is injured or killed.
“Successful completion of the clinical trial process is necessary to demonstrate that an investigational medicine is safe and effective, which is required to obtain FDA approval, so that companies may make the medicine available to a broader patient population when clinically appropriate,” Murphy said.
Follow Steven Ross Johnson on Twitter: @MHsjohnson
