ASHINGTON — Five years ago, the phrase “right to try” wasn’t yet an inkling in the minds of its staunchest advocates. Today, the pithy shorthand for the campaign to get dying patients access to experimental treatments has been slapped on bumper stickers, emblazoned on T-shirts, and uttered by some of the most powerful figures in Washington.
At the White House, President Trump has made clear he sees the issue as a priority. And on Capitol Hill — otherwise largely paralyzed on health care policy — Republicans are working on legislation that could fundamentally alter the way in which terminally ill patients can gain access to drugs.
Whether and when a bill will ultimately land on the president’s desk, or even what it will look like, is anyone’s guess. But the past three months have made one thing clear: The modern “right-to-try” campaign — and the scrappy libertarian movement behind it — is closer to legislative reform on this issue than ever before.
Its advance has come despite objections that any legislation could do more harm than good, leaving patients who are desperate for a cure vulnerable to exploitative claims or overhyped therapies.
“Something’s going to get passed,” said Art Caplan, a professor of bioethics at New York University’s Langone Medical Center and a critic of right-to-try laws. “The question is, can you do it in a way that doesn’t do damage to patients [and] that doesn’t take desperate people and leave them just as poorly off as they were before all this?”
The Senate unanimously passed a right-to-try bill last year, and just this week, a once-reluctant House Republican leader told STAT he will introduce new legislation on the issue within the month.
But the story of the five-year fight over “right to try” actually starts at a small lunch meeting of Republican health care experts in Phoenix. It then winds through more than three dozen state legislatures and into the stately meeting rooms of Capitol Hill, where pharmaceutical company lobbyists, Food and Drug Administration officials, and libertarian leaders are still pushing to shape or even upend the ultimate package.
In September 2012, a group of executives from the Cancer Treatment Centers of America reached out to the Goldwater Institute, a small, libertarian think tank named for the former senator and presidential candidate.
Goldwater — which has in past years accepted support from major conservative organizations like the Charles Koch Foundation and Donors Capital Fund — had taken on a wide-ranging libertarian agenda that included activism on campus free speech and school choice. The for-profit hospital chain CTCA also has its own ties to the Koch family.
CTCA and, ultimately, the Goldwater experts believed the FDA’s existing expanded access program — through which the agency approves some 99 percent of requests from dying patients who can’t get into clinical trials but want to try experimental treatments — was too cumbersome.
It was time to do something about it, and a consultant with CTCA, Chuck Warren, coined the term “right to try,” recalled Starlee Coleman, a Goldwater senior adviser.
The advocates decided to start in the states — and their ideas spread like wildfire.
A little over a year after their meeting with CTCA, Goldwater’s model legislation was signed into law in Colorado, a largely Democratic state with a Democratic governor. Thirty-nine other state legislatures eventually followed, many without a single dissenting vote.
“That’s the reason why we are where we are, because it’s common sense for people and there are patients everywhere who have really gone out of their way to tell their personal stories to lawmakers,” Coleman told STAT. “We’re asking [lawmakers] to let people make their own decision. It costs nothing, it hurts no one, no one who isn’t fully informed of the risk is engaged.”
“This is an expression of frustration from very sick people. We’ve given them a voice and a vehicle.”
That view, however, has not been not universally shared.
The movement’s opponents have argued the effort, though well-intentioned, could undermine the FDA’s existing oversight of the clinical trials process. Plus, they argued, it is not the FDA that constrains access to experimental therapies, but drug companies. Nothing in any pending legislation, state or federal, compels them to provide samples of the unproven therapies.
“We have a very good track record of approving or allowing requests for compassionate or expanded access to proceed,” former FDA commissioner Margaret Hamburg told NPR after the Colorado law passed. “I actually don’t believe that we are the barrier in these instances.”
It would be several years before the movement gained traction in the marbled halls of Congress. Before Donald Trump’s election, key House and Senate committees did little more than talk about the issue.
But in 2016, Sen. Ron Johnson (R-Wis.) — locked in a bitter reelection campaign without much support from the national party — picked up the right-to-try torch. He touted the issue on the campaign trail and in emotional videos online — and when he shocked Washington with a November victory, he pledged to deliver on his promise to pass a federal law modeled on the state legislation.
Repealing and replacing the Affordable Care Act dominated the first eight months of Trump’s presidency, and Johnson largely shelved his interest in the effort. But in August, when the Senate was poised to take up separate legislation that sets the rates pharmaceutical companies pay the FDA for drug reviews, Johnson saw an opening.
He threatened to hold up the separate package, even as a major deadline loomed, unless the Senate would pass his bill at the same time. That bill would solidify the idea, now enshrined in so many state laws, that patients who have exhausted other options and who aren’t eligible for existing clinical trials can sidestep the FDA’s expanded access program and ask drug companies for access to experimental treatments.
After a flurry of last-minute dealmaking, largely led by Sens. Lamar Alexander (R-Tenn.) and Patty Murray (D-Wash.), the chairman and ranking member of the Senate health committee, Johnson got his wish: the Senate unanimously passed a new, watered-down version of his bill. (Alexander and Murray helped to add protections to ensure the FDA would still receive information about adverse events and some clinical outcomes related to drugs accessed through right to try, among other changes, according to congressional aides.)
Even then, however, the effort looked poised to die quietly in the House.
FDA Commissioner Scott Gottlieb testified about the Johnson bill in October, and while he stopped short of signaling opposition to the bill, he made it clear he believed the language needed further changes.
House Energy and Commerce Committee Chairman Greg Walden — an Oregon Republican with outsize power over the path forward for the legislation — also voiced concerns about the Johnson bill. As late as mid-December, he told STAT he still had major questions about the utility of any legislation on the topic, asking aloud whether any patients had benefited from one of the dozens of state laws on the books.
“If indeed the state laws are not being used very much, then the last thing I want to do is give false hope to families and dying patients that somehow they’ll have a new access when in fact they may not,” he said.
At one point, he even ducked into a part of the Capitol that is off limits to reporters to avoid a STAT reporter’s question about his plans for the legislation.
Since January, however, Walden has been far more eager to get something done. He’s spoken directly with Trump about the push, pledging repeatedly to unveil new legislation that tweaks the Johnson bill “very soon.”
Gottlieb, too, has offered support for the effort in principle. Last month, he tweeted that the “FDA shares the administration’s goal to help patients facing terminal diagnosis access promising treatments in their fight,” and promised more changes to the administration’s existing program.
Behind the scenes, according to several advocates, there was a key player helping to keep up pressure on the issue, particularly with Walden: Vice President Mike Pence.
Pence, like Johnson, had seen firsthand the power of a poignant patient story. As governor of Indiana, he met Jordan McLinn, a young Hoosier with Duchenne muscular dystrophy who, along with his mother, Laura, became a vocal advocate of the right-to-try law that Pence ultimately signed into law.
Pence and McLinn have remained close — enough so that Pence and his staff occasionally invite the young patient and his mom to the White House when they are in Washington. And enough so that Pence hasn’t forgotten about right to try.
Pence and his staff started ramping up their outreach to both the FDA and Capitol Hill on the issue in late December, several individuals familiar with the movement told STAT. He’s taken meetings with Gottlieb and Walden and the president, too — and continued to increase the pressure on the House to get something done.
The ultimate proof of that campaign? Trump called for action at the State of the Union.
“People who are terminally ill should not have to go from country to country to seek a cure — I want to give them a chance right here at home,” Trump said. “It’s time for the Congress to give these wonderful, incredible Americans the ‘right-to-try.’”
He would later press the issue again at a Republican retreat in Greenbrier, W.Va., and in a private February meeting with Walden.
All eyes are now on Walden and the package he is pulling together this month. He says the bill will represent a serious effort to compromise between backers of the Johnson bill and those who have raised concerns about its scope.
He and other lawmakers will also have to confront the concerns of the powerful pharmaceutical and biotech industries.
While drug companies have largely avoided any public positions on the right-to-try movement, they are working with lawmakers behind the scenes to ensure the FDA maintains an oversight role in the process and to narrow the scope of who can rely on any right-to-try pathway, congressional aides in both chambers and several industry lobbyists told STAT.
“They’re reluctant to say, organize opposition,” NYU’s Caplan said. “They’re quietly in opposition. They don’t really want to lose FDA involvement after a decision to make something available. But it’s quiet and behind the scenes, not the usual [activism]. But they’re not going to be vocal about that, because they might offend some patient groups.”
The issue is also fraught with both legal and political considerations about the cost of providing the drug, the liability and impact on ongoing clinical trials, and the potential for bad press.
“For the traditional pharma and biotech companies, the last thing they want is chaos. The requirements for good evidence protect them from charlatans,” said Dr. Robert Califf, a former FDA commissioner now at Duke Health.
PhRMA spokesman Andrew Powaleny told STAT the trade group “has always believed it is crucial any Right to Try policy proposal protects patient safety and the integrity of the clinical trial process along with the [FDA’s] oversight role,” adding that the group continues to engage with Johnson and other key leaders in the House, Senate, and administration.
Should Walden’s new legislation clear the committee, there’s no reason to think it wouldn’t be able to pass the House, advocates said.
But a revised package would have to undergo new scrutiny in the Senate later this year — a timeline that worries some supporters.
Supporters warn, too, that they will demand a “clean” vote on the Johnson bill if they view Walden’s package as too narrow. One lobbyist said Walden was eyeing reforms to the existing expanded access program, and not an alternative right-to-try pathway — and that might not be enough to win the support of the advocates. It could also alienate Pence and the president, who have backed Johnson’s broader bill.
“We’re still pretty hopeful we’ll get a vote on House floor on the direct Senate language,” said
David Barnes, the policy director of Generation Opportunity, which is allied with Freedom Partners. “We’re calling on House leadership step up to the plate and let it get a huge majority.”
Walden, for his part, declined to get into specifics about how his bill will differ from Johnson’s, but suggested he had two concerns about making sure the federal law is “written correctly.”
“Obviously I have sympathy for people with terminal diseases, and as long as they can safely access some of these potential medicines the right way, then A, the pharmaceutical companies would participate, because there’s no requirement that they do, so you’ve got to get that piece right. And B, we didn’t want to do anything that could harm somebody, so you’ve got to get that piece right,” he said. “I think we’re pretty close.”
Correction: this story originally misstated the year of Goldwater’s first meeting with CTCA.