W

ASHINGTON — 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.

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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.

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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.”

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  • These pseudo-intellectual pinheads should stay in their taxpayer-funded ivory towers and keep their mouths shut before they piss off one too many people and the taxpayers tear them from their lofty perches by cutting off their funding.

  • The FDA should be shut down and food safety give to the Ag department. I think it is an open question whether they have killed more people than they have saved. The FDA is at this time nothing but a gatekeeper suffering from an acute case of regulatory capture.

  • So, if I’m terminally ill these guys don’t want to give me a chance to roll the dice because they are worried that I might happen to roll snake-eyes? I say “no thank-you” to the intelligentsia who want to take the dice out of my hands. If they want to give me copious warnings about the potential dire consequences before I roll them, that would be fine, but self-determination is a basic freedom.

  • you lost me with the first sentence,
    If I was dying and there was nothing “approved” that would stop me from dying you can bet your ass I would want the option to “try” an experimental treatment.
    You talk of possibly harming a dying patient,are you kidding me they are dying and probably in a lot of pain so what if the trial treatment doesn’t work it at least has a chance to work which gives the possibility of curing the patient and if it doesn’t it would at the very least give the health industry information that they didn’t have before.

  • So the dying should just shut up and die because you wouldn’t want to “weaken confidence in the FDA”?!

    Could swamp creatures be more repulsive?! Just die, because we wouldn’t want to diminish the self value of bureaucrats?! This country can’t end soon enough.

  • “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.” HOW STUPID is that???? These people are DYING already, they have little or no “health” to harm, and nothing to lose… Absolutely NO REASON to not let them TRY to save their lives…

  • Terminally ill pt.’s support this Bill because so few pt’s get a chance at getting these new , life saving drugs. The opponents constantly cite the ” 99 % FDA approval rate “, but never give you the numbers . 2500 – 3,000 compassionate use approvals granted per year is pathetically low , a tragedy and embarrassing in a country of 314 Million that leads the way in developing 21st Century life saving drugs every year . Drugs just out of the reach of tens of thousands of dying pt.’s every year . The trials are small , and incredibly slow , at least 5 years , often 7 – 10 years. RTT has already caused the FDA to review and shrink their hours long paperwork that obstructed compassionate use for years . Admitting a problem . Noone cared then . The opponents still don’t seem to care about this broken system . The FDA shouldn’t delay for one day a dying persons’ last chance. I say revise the RTT bill slightly- requiring a pt., dr. and co. to notify the FDA of compassionate use , then let the FDA step in if they choose. That would speed up most of the requests. Then true reformers, invested in actually helping these dying pt.’s , can move on to tackle the other obstacles to compassionate access- like the fact that a too busy oncologist and a dying pt. have to design an IND – individual ” trial ” protocol , to request compassion . That takes hours and hours that they don’t have. By the way , my wife would have died in 2011 from metastatic bc , she couldn’t get through the FDA paper work , but found an expanded access clinic in DC , 450 miles from our home near Boston. It saved her life and she is cancer free 6 years. She was the first pt. to testify for RTT in Ariz. and Colo. Please view ” Lorraines’ Story ” on the Goldwater Institute RTT website

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