President Donald Trump won the legislation he had pushed Congress for months to pass that would permit Americans to seek experimental treatments without going through the U.S. Food and Drug Administration's compassionate-use process.
Critics, however, have argued the real intent of the so-called right-to-try bill is to weaken the FDA.
The head of the agency, Scott Gottlieb, who last fall had expressed concern about the legislation during congressional testimony — saying it risked "undermining a regulatory process that has been carefully crafted over many years to strike a very careful balance" — now supports the bill, even though its language has not changed from when the commissioner earlier objected to it.
The House on May 22 passed the right-to-try bill in a 250-169 vote. It now heads to Trump's desk for his signature. The legislation, however, was not the bill the House had worked on for months and had passed in March. Instead, it was a measure authored by Sen. Ron Johnson, R-Wis.
The Senate passed the Johnson bill in August 2017 as part of a deal to get another piece of legislation critical to the FDA — the Food and Drug Administration Reauthorization Act of 2017 — out of the chamber, which the Wisconsin lawmaker had threatened to hold up unless he got a vote on his right-to-try measure.
At an Oct. 3, 2017, House hearing, Gottlieb had objected to certain elements of the Johnson bill, telling members of the Energy and Commerce Health Subcommittee the legislation's "life-threatening disease or condition" criteria could be interpreted broadly and "could sweep in a whole range of conditions for which we didn't intend," such as chronic conditions, like diabetes.
He said some patients could be unnecessarily exposed to side effects under the Johnson bill.
Gottlieb urged the House last fall to adopt a narrower definition of patients diagnosed with a terminal illness.
While the House fulfilled that request in the bill it passed in March, Senate leaders never attempted to bring the measure before their chamber for consideration.
So, House Republicans said their only option was to try to pass the Johnson bill, even though some of those lower-chamber lawmakers said they preferred the legislation they adopted in March over the Senate's measure.
An about-face?
Gottlieb surprised many on May 17 when he publicly declared on Twitter he was now "comfortable" with the Johnson bill — making the declaration only days after Trump called him out at a White House event, telling the commissioner, "Right to try is happening, right? You have that moving?"
"To be clear, FDA supports the Johnson bill moving to the House floor and stands ready to implement it in a way consistent with the intent of Congress to promote access and protect patients," Gottlieb wrote in another tweet.
Ahead of the May 22 House vote, Gottlieb tweeted that "I stand ready to implement" the bill "in a way that achieves Congress' intent to promote access and protect patients, and build on the FDA's longstanding commitment to these important goals."
Later in the day as the Johnson bill was being debated on the House floor, Gottlieb again publicly expressed support. "I'm certainly comfortable with it," he said during a Washington forum hosted by The Wall Street Journal.
Even though he had earlier testified that FDA lawyers said the agency would be bound by the Johnson bill's broad criteria and that he did not think "we'd be able to, as a matter of our own interpretation of the law, further narrow that," Gottlieb told the forum he would have "some latitude" in addressing the agency's earlier concerns.
"I think a lot of the issues that we provided feedback on, at least some of them, we can address in implementation and still maintain the spirit and the letter of the intent of Congress in how we promulgate either regulation or guidance to implement this," Gottlieb said.
Weakening the gatekeeper
Under the right-to-try bill, patients would be able to bypass the FDA's expanded access program, also called compassionate use, which allows regulators to help terminally ill patients obtain and use investigational medicines outside of the clinical trial process.
Gottlieb had noted during the October 2017 hearing that the FDA approves 99% of the 1,000 compassionate-use program applications it receives annually from patients, with most given the go-ahead within a few days and emergency requests generally granted immediately over the phone.
He acknowledged the FDA cannot force drugmakers to provide their experimental medicines to patients.
The Johnson bill would not change that situation, said Rep. Frank Pallone, D-N.J., ranking member on the House Energy and Commerce Committee.
While the experimental drugs patients seek under right to try must have at least completed the phase 1 testing process, those studies only check for toxicity, not safety or efficacy.
"The main reason this bill is being pushed is to chip away at FDA's authority to ensure the safety and effectiveness of our drugs," Pallone said on the House floor just before the vote on the legislation.
He was among the critics who argued cutting the FDA out as a gatekeeper would leave desperately ill patients potentially exposed to "snake oil" salesmen peddling false hopes.
Pallone noted that more than 100 patient advocacy groups had objected to the Johnson bill.
"The bill that passed the House today is dangerous," the National Organization for Rare Disorders said in a statement.
"As a terminal cancer patient, I know the importance of having hope," former biotech executive Michael Becker, president and founder of MDB Communications LLC, told S&P Global Market Intelligence.
"But dangling the word hope to advance redundant legislation that harms the terminally ill is insulting to me and others like me. The passage of right to try will have broad and devastating consequences. Patients will still be denied access to drugs by their developers or asked to pay for the cost to provide them."
"Worse yet, the already financially frail may lose health insurance coverage if they are able to access drugs via right to try and be left paying out of pocket for hospital and other medical costs as a result," Becker added.
"And when they are forced to seek legal remedies, right to try patients will find those have been taken away through this legislation. My heart breaks to think of this future and how carelessly it has come to fruition."
