If the promise by congressional leaders that the right-to-try legislation would save lives fails to be borne out, Health and Human Services Secretary Alex Azar and Food and Drug Administration Commissioner Scott Gottlieb cannot be blamed, a top White House official said.
"By the very nature of this, the patients who will be afforded this opportunity are those who are terminally ill, so I think it would be hard to hold the secretary accountable for if they're not fully recovered," Marc Short, White House director of legislative affairs, said May 25 in response to S&P Global Market Intelligence's questions during a conference call with reporters.
The right-to-try legislation, which passed the House on May 22 and is now headed to President Donald Trump's desk for his signature, would permit Americans to seek experimental treatments without going through the FDA's longstanding compassionate-use process.
Short said Trump plans to hold a signing ceremony next week on the bill.
Gottlieb had initially expressed concern that the legislation's "life-threatening disease or condition" criteria was too broad and "could sweep in a whole range of conditions for which we didn't intend," such as chronic conditions, like diabetes.
At an Oct. 3, 2017, hearing the FDA chief said the legislation could result in more people unnecessarily being exposed to dangerous side effects.
Gottlieb had called for the bill's criteria to be narrowed to patients with terminal illnesses.
But the legislation that has now passed the Senate and the House, authored by Sen. Ron Johnson, R-Wis., does not do that and remains broad. Gottlieb has since thrown his full support behind the bill, even though the language in the measure has not changed since he testified.
No guaranteed access
In sending the bill, known as the Trickett Wendler, Frank Mongiello, Jordan McLinn and Matthew Bellina Right to Try Act of 2017, or S. 204, to Trump, House Speaker Paul Ryan, R-Wis., said that patients would be able to get access to experimental treatments under the legislation.
But at the October 2017 House hearing on the Johnson bill, Gottlieb acknowledged that the FDA does not have the authority to force drugmakers to provide their experimental medicines to patients, and the legislation does not change that situation, nor does it provide a guarantee of access.
While the experimental drugs patients seek under the right to try bill must have at least completed the phase 1 testing process, those studies only check for toxicity, not safety or efficacy. Phase 1 trials also consist of small numbers — often a few dozen — of usually healthy people, unless the products are being tested in cancer patients. Few drugs that come out of early stage testing demonstrate later that they save lives.
Yet Rep. Greg Walden, R-Ore., chairman of the House Energy and Commerce Committee, on May 24 said that "this right-to-try legislation will save lives."
"It will give people hope that they otherwise would not have," Walden added.
But opponents of Johnson's bill said it cuts the FDA out as a gatekeeper, leaving desperately ill patients potentially exposed to "snake oil" salesmen peddling false hopes.
More than 100 patient advocacy groups objected to the Johnson bill and the National Organization for Rare Disorders called the legislation "dangerous."
Dangling hope
"Dangling the word hope to advance redundant legislation that harms the terminally ill is insulting to me and others like me," said former biotech executive Michael Becker, president and founder of MDB Communications LLC, who has been diagnosed with terminal cancer. "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."
But the White House's Short said the bill was "a huge step forward to provide [patients] opportunity and hope."
"We certainly believe that there should be a level of mercy that's afforded them and not being tied up in a bureaucratic morass to get drugs available to them," Short said. "Roughly, a million Americans die from a terminal illness every year and many of these patients spend years searching for a potential cure or even just to get into a clinical trial."
Last fall, Gottlieb testified that the FDA's lawyers told him 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."
But this week, he said he would have "some latitude" in addressing the agency's earlier concerns.
"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 during a May 22 healthcare forum hosted by The Wall Street Journal.
The FDA commissioner has committed to making changes to the FDA's expanded-access, also known as compassionate-use, program, which already approves 99% of the 1,000 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.
When asked, the White House's Short did not specify how the administration would distinguish between the already planned changes to the compassionate-use program from the actions taken to implement the right-to-try legislation and how it would gauge the success of saving lives — as Walden and other members of Congress promised it would.
"We look forward to sharing with you positive results in the years ahead," Short said.
