The very act of using right-to-try therapies can render them ineligible for health insurance or hospice care when they need it most.
By almost all standards, President Trump’s State of the Union was light on policy prescriptions, but the one bill that he did choose to name-drop has led to a flurry of conversation. That would be the federal “right to try” bill, which was passed in the Senate last August but has since stalled in the House. In his address, Trump explained it by asserting that “patients with terminal conditions should have access to experimental treatments that could potentially save their lives.” The idea has already gained considerable traction at the state level, and when framed in this way, the case for right to try is undeniably a compelling one.
The right-to-try movement first gained national attention in 2001 with the case of Abigail Burroughs, who died of cancer after being denied access to an experimental drug, Erbitux, which her physicians had believed might have saved her life. Her father went on to found the Abigail Alliance for Better Access to Developmental Drugs, which sued the Food and Drug Administration, arguing that terminally ill patients have a constitutional right to such unapproved therapies. A divided panel of the U.S. Court of Appeals for the District of Columbia Circuit at first sided with the alliance, but a year later, in 2007, the case was reheard and the ruling was reversed, favoring the FDA. Ultimately, the court held that “although terminally ill patients desperately need curative treatments … their deaths can certainly be hastened by the use of a potentially toxic drug with no proven therapeutic benefit,” and they couldn’t claim a constitutional right to experimental treatments that might only serve to worsen their conditions. Having failed to gain ground in the courts, the right-to-try movement eventually turned to state legislatures, and in 2014, Colorado became the first to pass a right-to-try law.
But in the years since the trial, something crucial had happened: The Federal Food, Drug, and Cosmetic Act was expanded in 2009 and now grants patients access to experimental treatments when enrollment in clinical trials is impossible. The expanded access program is necessarily a last resort, but it’s far from the bureaucratic nightmare many right-to-try advocates make it out to be, and it’s been made more accessible and efficient over time. Today, the form takes 45 minutes to complete, and the FDA will reply to emergency requests within no more than 24 hours—with a 99 percent approval rate. So the problem with Trump’s rallying cry for right to try is not that the people shouldn’t have it, but that such a law already exists—and if anything, the right-to-try bill would end up undermining patients’ access to care rather than affording them viable alternatives.
The form takes 45 minutes to complete, and the FDA will reply to emergency requests within no more than 24 hours—with a 99 percent approval rate.
It’s worth asking why the FDA requires the form, if it ends up granting 99 percent of requests anyway. The reason helps explain that the process isn’t just a formality. In about 10 percent of cases, the FDA will make safety-oriented changes, adjusting, say, the dosage or the frequency with which a patient receives the treatment. Crucially, these decisions are often made on the basis of confidential information—information that physicians who might sign off on access to a drug under right to try don’t have. By bypassing this step—a consultation of the scientists best placed to know about and therefore minimize the risks involved in taking an experimental drug—right to try could end up putting patients in harm’s way.
In addition, the proposed federal right-to-try law will only require the successful completion of a Phase I study, which isn’t enough to ensure efficacy or safety on its own. Contrary to our unfounded optimism about medical progress that insists that new drugs must be good drugs, fewer than 10 percent of drugs that enter Phase I end up being approved; for oncology, that figure falls to 5.1 percent. The FDA is aware of these rates, and the expanded access program is designed accordingly. Its broad requirement is “sufficient evidence of safety and effectiveness,” which usually means relying on data from Phase III trials or very compelling data from Phase II trials. It is also occasionally willing to draw on more preliminary results if the disease is immediately life-threatening.
Again, this caution isn’t solely a matter of procedure. There are numerous examples of experimental therapies that looked promising in Phase I, only to prove useless or actively dangerous further along on the road to FDA approval. Trials for semagacestat, which was developed to improve cognitive function in patients with Alzheimer’s, were terminated early after recipients of the treatment were found to have “worsened cognitive and functional status and an increased risk of skin cancer compared to patients who received placebo.” Phase III trials for torcetrapib, a drug intended to treat heart disease that was at one point seen as a miracle cure that “would probably have been the largest-selling pharmaceutical in history,” were discontinued when death rates turned out to be higher in the branch of the trial taking the drug compared with the placebo.
These tragedies make it clear that patients who exercise their right to try before further studies have been done may end up worse off than they were before—yet the very act of using right-to-try therapies can render them ineligible for health insurance or hospice care when they need it most. Of the right-to-try laws now on the books in 38 states, half allow insurers to deny patients hospice coverage should they require it after the use of right-to-try drugs. Several have made it clear that health insurers are not obligated to cover the costs of any complications that may arise, and the law in Colorado goes even further: For the duration of an experimental therapy secured under right to try—and even six months after their last doses—patients are without vital protections.
The very act of using right-to-try therapies can render patients ineligible for health insurance or hospice care when they need it most.
Right to try is also perhaps better characterized as the right to ask. Drug companies can and do refuse to grant access even where right-to-try legislation exists. Janssen Pharmaceuticals has made it explicit that it will not evaluate right-to-try requests, “as those mechanisms do not provide for FDA input, which we consider critical for ensuring patient safety.” Instead, it encourages patients to continue to make requests via existing channels—namely, the FDA’s expanded access program. In the case of Jordan McLinn, who became the face of the movement in Indiana (and is likely the reason it’s now a pet issue for Vice President Mike Pence), the manufacturer of the experimental therapy sought by his mother declined to make it available under right to try. He was instead enrolled in a clinical trial—an option available to all qualifying patients without right-to-try legislation of any kind. McLinn’s condition has improved since he was enrolled in the trial, according to his mother.
That’s great news, but it’s important to realize that McLinn didn’t benefit from state-level right-to-try legislation, nor would the federal bill have compelled pharmaceutical companies to provide experimental therapies. As a stunning report from researchers at NYU School of Medicine noted, “In the more than two and a half years since the first law was signed, there have been no documented cases of anyone receiving access, because of a right to try law, to an experimental product that would not have been available via the FDA’s Expanded Access Program.”
The existing expanded access program grants the very freedoms right-to-try advocates claim to seek, and does so quickly, readily, and without stripping terminally ill patients of their health care. State-level legislation has proven toothless and doesn’t target areas that are actually in need of reform. So why has right to try become such a hobby horse for the right?
In recent years, the driving force behind the movement has been the Goldwater Institute, a conservative and libertarian think tank. Curtailing the FDA’s influence might be an attractive prospect for these states’ rights–oriented Republicans, which might explain the institute’s peddling of this inflammatory (and, again, unnecessary) law. But a hands-off approach here could have severe consequences both physically and financially for desperate, vulnerable individuals. If giving patients their best chance of recovery is really the priority, legislators should be raising awareness of and continuing to improve upon the expanded access program, not introducing a new law that seeks to undercut it. Passing this bill might protect insurers and pharmaceutical companies, but it won’t protect the people.
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