President Trump infused momentum into policy that would allow terminally ill patients quick access to experimental medication, but lawmakers are still working out how many patients should be given this choice.
The promise to circumvent bureaucracy in order to save the lives of the terminally ill will likely sound appealing to voters. Turning Trump’s endorsement into policy, however, won’t be an easy task for lawmakers—even if they generally support the idea.
Sen. Ron Johnson, who championed a right-to-try bill through the Senate, said Trump’s endorsement in the State of the Union sparked momentum around the issue. Johnson added that he is having discussions with House Republican leaders about moving the bill forward. On Monday, 42 lawmakers also wrote to House Speaker Paul Ryan and House Majority Leader Kevin McCarthy asking that Johnson’s bill be brought to the floor.
“This bill safeguards any pharmaceutical company that may wish to participate in Right to Try, but it in no way requires participation to begin with. We are not seeking to impose a mandate on anyone. Instead, we are merely seeking to give patients who have no other options yet another possibility to try to save their own lives,” they wrote.
But policy differences are likely to arise, particularly around how expansive the effects of the legislation should be.
This issue was raised in October when Food and Drug Administration Commissioner Scott Gottlieb appeared before the Energy and Commerce Committee on the topic. He said the Senate version of right-to-try could allow more patients than intended access to investigational products, and recommended narrowing the eligibility from patients who face a “life-threatening disease or condition” to those with a “terminal illness.”
“Many chronic conditions are life-threatening, but medical and behavioral interventions make them manageable without recourse to unapproved therapies,” he told lawmakers in his written testimony.
Johnson expressed concern that changes to the legislation would limit the number of people who could benefit. “I want to make sure that it maintains the freedom for as many individuals in that situation as possible; I don’t want to limit the number of Americans who can take advantage of that,” he told National Journal. “Some of the tweaks I think would dramatically limit it. I mean, for example, maybe a young boy with Duchenne muscular dystrophy could be narrowed out of that definition, which I think would be a real shame.”
But energy committee Chairman Greg Walden said they are looking to improve the bill. “We’re very close on language that will make it even better and a more usable bill,” he said, adding that they are looking at who would benefit from the legislation.
“Remember, this is for terminally ill patients, I mean that’s what we’re told. The language that came over from the Senate says people with incurable diseases, so that would include things like rheumatoid arthritis, diabetes … We’re looking at a lot of those issues,” said Walden.
The right-to-try initiative, backed by the libertarian Goldwater Institute, began at the state level. According to the institute, 38 states have passed laws aiming to cut the red tape around experimental therapies that could help seriously ill patients.
In the process, however, it would also sidestep FDA protections. The FDA has asserted that it already has the ability to quickly bring unapproved medicines to ill patients through the expanded access pathway. Expanded access, or compassionate use, is for patients who are seriously ill and cannot enroll in a clinical trial. The agency says it authorizes 99 percent of these requests, but it cannot require the companies making the therapy to participate.
The Goldwater Institute argues that this process is not working. “[A]n investigation by the Goldwater Institute shows that the entire system for gaining access to an unapproved medication is so rigged with bureaucracy and disincentives that it is bound to fail in most cases,” the group states on its website.
But according to a spate of patient-advocacy groups, the barrier is typically the company denying the request. “When access to a therapy is denied to a patient, it is generally the company that denies the request, and for reasons that appear to be reasonable, such as a determination that the benefits do not outweigh the risks, an unavailability of sufficient product to offer outside of clinical trials, costs, or concerns about adversely affecting clinical trial enrollment,” the groups wrote in a letter to House leaders Tuesday urging against Johnson’s legislation.
The issue moved to the national level when the legality of these state laws were called into question. Johnson has told the FDA in the past that his legislation would clear up these uncertainties.
“It would clarify that the federal government cannot prohibit doctors from treating terminal patients in accordance with state law if certain requirements are met,” he wrote to the agency in a 2016 letter. “The bill includes liability protections for doctors and manufacturers who provide treatment under federal and state right to try laws. It also requires that the FDA not use adverse outcomes of treatments used under right to try laws when reviewing extant Investigational New Drug applications.”
Now, the president has generally backed the idea, naming Gottlieb as the person who would lead this initiative.
“So important—right-to-try. You know, those drugs, they sit in there for 12, 13, 14 years. And a person is terminally ill, they have two months left, and under the old system they don’t want to give them even an experimental medicine because they’re afraid they’re going to be hurt. Well, they’re not going to be around for two months,” the president told GOP lawmakers at their retreat last week.