President Trump makes extraordinary claims about the success of the Right to Try law, which was designed to give terminally ill people access to experimental medical treatments before they’ve been approved by the Food and Drug Administration. “I tell you, it’s a miracle — so many people have been saved,” he said at a rally in Lake Charles, La., last month, describing how desperately ill people were, thanks to him, able to access the work of the “best doctors and labs, technicians in the world.” When he signed the law in May 2018, he used even more sweeping language: “We will be saving … thousands and thousands, hundreds of thousands” of lives. The Trump 2020 campaign has used the law to promote the idea that the GOP is “the party of health care.”
Unfortunately, Right to Try appears to be helping very few people. The “incredible” transformation that Trump described in Louisiana is a fantasy: Right to Try mainly succeeds at giving its political backers a cheap public relations victory, even as they exploit the hopes of dying Americans.
Proponents frame the statute as a triumph for the freedom of patients to take sensible risks if they are essentially out of options. Under the law, if a drug has completed a Phase I clinical trial, which determines the highest safe dose of a new intervention (whose effectiveness has yet to be proved), then pharmaceutical companies may make their experimental therapies available to extremely ill people who request them. These patients don’t have to ask the FDA for permission, drug companies can bypass ethics boards that normally monitor drug tests and the companies are shielded from lawsuits.
The idea of letting people take a gamble like that has intuitive appeal. But almost a year and a half after the law’s enactment, scholars and reporters looking for evidence of its effectiveness have turned up only a small number of people who have used the law to obtain treatment. Matt Bellina, an amyotrophic lateral sclerosis (ALS) patient who campaigned for the law, has been receiving an experimental therapy still in clinical testing from BrainStorm, the New York-based company has confirmed. Daniela Bota, a neurologist at the University of California at Irvine, helped secure a nonapproved drug through Right to Try for a patient with aggressive brain cancer, she told STAT News (and confirmed to The Washington Post).
And the Goldwater Institute, which championed the law, recently announced that the Epitopoietic Research Corporation has treated six patients with brain cancer this year under Right to Try and has become the first drug manufacturer to create a formal program for RTT applicants. By law, pharmaceutical companies are required to make annual reports about provision of drugs through Right to Try, which the FDA will then post online, but the agency hasn’t yet set a deadline for these documents. Other RTT patients will no doubt come to light, but if the program were the grand triumph Trump says it is, more people would surely be coming forward with success stories.
One reason for the limited impact of Right to Try thus far is that its proponents vastly exaggerated the barriers that the FDA placed between terminally ill patients and experimental drugs. The agency already had an “expanded access” pathway for experimental treatments. To get treatment through that route, a patient must have a serious illness, lack a way to get the treatment via a clinical trial and lack a legally available alternative treatment. (Patients might be shut out of a clinical trial because of previous participation in a different trial or because their disease has progressed too far, among other reasons.) The main distinction between expanded access and Right to Try is that, in the former, the FDA is more in the loop: Companies must submit safety reports to the agency that include copious data about efficacy, side effects and “adverse events” (the most grave side effects). This information is used in determining whether a drug should be approved for general use.
Many patients get drugs through expanded access. In 2018, for example, the FDA processed more than 1,500 applications (which can include multiple individuals) for expanded access to unproven treatments — and approved more than 95 percent of them, typically within hours to days.
Both before and after passage of Right to Try, the main barrier to patients getting experimental treatments hasn’t been the government. It’s been drug companies. They have little incentive to provide unapproved drugs. Yes, Right to Try gives pharmaceutical manufacturers liability protections, a shield they had lacked. And while there’s less mandatory data collection, the FDA can still use the most severe “adverse events” to evaluate drugs’ safety and efficacy; companies are therefore motivated to administer them through their own carefully controlled clinical trials, rather than via Right to Try.
The law also prohibits charging more than is necessary to recover costs for providing access to these treatments, meaning there is no direct financial incentive to make them available. Nor are insurance companies required to reimburse patients or drugmakers for these costs. Consider BrainStorm, which has received “countless” inquiries about its experimental cell therapy from patients with ALS, according to its chief medical officer, Ralph Kern. But the company decided not to make the drug available (except to Bellina, a one-off exception) through either the expanded-access program or Right to Try because there is “no funding mechanism,” Kern said.
Of course, even if a patient gains access to an unproven treatment that has gone through Phase I clinical trials, that’s hardly a guarantee of a positive health outcome. Most experimental treatments at this stage will never be proved safe and effective and brought to market. According to one study, only 13.8 percent of all drugs that make it through Phase I will be approved — and rates are lower in the areas of particular interest to terminally ill patients, such as cancer treatments (3.4 percent, says the same study). What’s more, those figures say nothing about the overall effectiveness of the drugs.
Despite all this, Trump continues to present Right to Try as a centerpiece of his health-care agenda. He often cites Natalie Harp as an example of RTT’s “truly spectacular results.” Harp, a Trump supporter who has joined the advisory board of the president’s 2020 campaign, speaks and writes often about Trump and health care. As she has explained on social media and in interviews, she has a rare form of bone cancer and was harmed by a medical error in 2015 (intravenous delivery of “sterile water” instead of saline) that left her close to death. She was at first offered only palliative care, she says, but then was able to access an immunotherapy drug through Right to Try — and her health significantly improved. As she puts it in an interview on the Trump campaign’s YouTube site: “I have cancer, I’ve survived a medical error, I want to get better, I don’t want to die, and they didn’t offer me that opportunity. The Democrats didn’t, doctors didn’t, Donald Trump did. . . . Because with Right to Try, he’s like, ‘You never give up.’ ” Appearing with the president at a Faith and Freedom Coalition conference in June, she called him “my Good Samaritan.”
But looking closely at her accounts of her illness, it’s hard to see how she could be a beneficiary of Right to Try. The effects of the life-threatening medical error she suffered were treated before the law’s passage, for instance. As she sought help for her cancer, she says, she was ultimately given “an FDA-approved immunotherapy drug for an unapproved use” — which made the difference. However, off-label or unapproved use of FDA-approved drugs is common and permitted under rules that long predate Trump’s tenure. Right to Try specifically allows access to drugs that have not received FDA approval. (Harp didn’t respond to requests for an interview made through various social media channels, cellphone messages and Trump 2020 spokespeople.)
But setting her case aside, if Right to Try were the transformative policy that Trump describes, there ought to be many verifiable examples of its success. Instead, we’re left mostly with Trump’s boasts.
It’s true that the “expanded access” pathway isn’t perfect. Ethical-review boards sometimes raise unnecessary objections to allowing people to access experimental drugs; the standards they apply could be made more consistent, and extending legal protections to drugmakers that allow such access makes sense. But Right to Try has done little, if anything, to increase access to investigational drugs, and it’s a cruel falsehood to tell terminally ill people that the law has led to hundreds or thousands of “miraculous” recoveries.