WASHINGTON — Dozens of doctors, medical ethicists, and lawyers are warning Congress that legislation to allow Americans with life-threatening conditions access to unapproved, experimental drugs risks harming patients’ health.
The letter was drafted by Alison Bateman-House, associate professor of medical ethics at NYU Langone Health, along with some of her colleagues. It is addressed to the leaders of the House Committee on Energy and Commerce, the committee currently considering a so-called “right-to-try” bill. The letter was circulated for online signatures on Thursday, and organizers said they planned to send the letter on Feb. 5.
In August the Senate passed a right-to-try bill sponsored by Sen. Ron Johnson, which is now sitting in a House committee. The bill would allow patients with life-threatening conditions access to drugs that have not been approved by the Food and Drug Administration. FDA Commissioner Scott Gottlieb has expressed reservations about the bill, but President Trump seems to support it.
The 40-plus signatories of the draft letter, however, say they are strongly opposed.
“This legislation sells vulnerable patients and families false hope at the expense of weakening the FDA’s critical role in making sure that all Americans can have confidence in the safety and effectiveness of our medical products,” the draft letter read on Thursday afternoon.
The FDA already has a pathway for patients to be able to use experimental treatments outside of clinical trials, which is called “expanded access” or “compassionate use.” According to an FDA study, 99 percent of requests made from 2005 to 2014 for these experimental drugs were approved.
But a right-to-try bill would allow doctors and patients to skirt around the FDA, leaving them more vulnerable if something goes wrong.
“Patients with terminal conditions who access unapproved therapies outside of clinical trials may be at risk of hastened death or reduced quality of the life that they have left, and deserve protections similar to patients taking part in clinical trials,” the authors wrote.
Andrew Powaleny, a spokesperson for the pharmaceutical industry organization PhRMA, which hasn’t taken a firm stance on the legislation, said, “It is crucial that any right-to-try policy proposals protect patient safety and the integrity of the clinical trial process along with U.S. Food and Drug Administration oversight. PhRMA appreciated the opportunity last fall to work with Sen. Johnson on his proposal and is engaged in an ongoing dialogue with his office and Energy and Commerce Committee Chairman Walden.”
Trump doubles down
Meanwhile, in a speech to Republican lawmakers on Thursday, Trump reiterated his support for congressional action on the subject. He first spoke publicly in favor of right-to-try legislation in his State of the Union address on Tuesday.
“I hope you folks can approve it, and I hope you agree with it,” Trump told Republican members of Congress at a Thursday retreat in Greenbrier, West Virginia.
Trump’s remarks implied that FDA Commissioner Scott Gottlieb was on his side, saying that Gottlieb was “heading it up.”
However, Gottlieb has actually expressed concern about the current bill that the House is considering, testifying at an October hearing that it would undercut the agency’s authority to protect patients from unsafe drugs.
The FDA declined to comment Thursday.
Bateman-House testified at the October hearing alongside Gottlieb. She said that she has not been in touch with the committee since the hearing, and that she thought after the hearing the bill had “more or less died.”
But Vice President Pence has been vocal recently on the issue, proclaiming his support in January. He signed a similar bill in Indiana as governor in 2015. Thirty-eight states now have such laws.
“I think his heart’s probably in the right place,” Bateman-House said of Pence. “I just wish he would put all this time and energy [into legislation] that was actually going to help patients.”
This article is reproduced with permission from STAT. It was first published on Feb. 1, 2018. Find the original story here.