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House rejects Trump-backed 'right-to-try' experimental drugs bill

Legislation failed to garner enough votes in the House to give critically ill patients greater access to experimental drugs beyond the U.S. Food and Drug Administration's current compassionate-use process — dealing a blow to the Republicans who backed the measure, including President Donald Trump and Vice President Mike Pence.

The vote on the right-to-try bill, which had only formerly been introduced earlier in the day on March 13, was 259-140, falling short of the required two-thirds of the 435 House members needed to pass.

Republicans, who control the House, blamed Democrats for the bill's failure, although 32 members of the minority party had voted in favor. Two Republicans voted against the right-to-try measure.

For the most part, Democrats criticized the bill as an attempt to weaken the FDA.

"Supporters of this legislation have claimed that it will provide seriously ill patients who have exhausted all of their available treatment options access to experimental therapies free from the barriers of FDA oversight," Rep. Frank Pallone, D-N.J., ranking member of the House Energy and Commerce Committee, said on the chamber floor. "While it is understandable that someone suffering from a disease that has no more options would want to try anything that could help them fight their disease, this legislation delivers the false hope to patients and their families that they will receive a cure to their underlying disease or condition."

Pallone noted that nothing in the right-to-try legislation compels a drug manufacturer to grant access on request.

"This legislation provides patients and their families nothing more than the right to ask a manufacturer for access to early stage unproven treatments," he said.

Pallone said the FDA approves 99% of the requests it receives for access to experimental medicines under its compassionate-use program, also known as expanded access.

Last year, the FDA received 1,500 requests and only rejected nine of those, he pointed out.

But Rep. Brian Fitzpatrick, R-Pa., who sponsored the House bill, said the FDA's compassionate-use process was too slow and burdensome.

Fitzpatrick argued that the number of requests approved by the FDA last year paled in comparison to the 12,000 people in France who gained access to experimental treatments.

House Majority Leader Kevin McCarthy, R-Calif., said Republicans would try again.

"The House will not let this be the end," he said in a statement.

White House pressure

Trump had publicly thrown his support behind the right-to-try effort during his Jan. 30 State of the Union address.

Pence also had pressed the House to quickly take up the measure, which had been tweaked from a version that was adopted by a voice vote in the Senate in August 2017.

Trump had put FDA Commissioner Scott Gottlieb — who publicly criticized the Senate's right-to-try bill — in a tricky spot by insisting that the agency chief was heading up the administration's effort on the legislation.

During an Oct. 3, 2017, hearing, Gottlieb said the Senate legislation contained flawed criteria, which could expose more people unnecessarily to potentially dangerous side effects. He called for changes to the bill.

But Gottlieb appeared to later fall in line with Trump, telling reporters Feb. 6 that he thought his agency would be able to find some sort of a solution with Congress.

He also posted a series of tweets on Feb. 11 that seemed to support the Republicans' right-to-try effort, saying the FDA was "committed to working closely" with lawmakers to advance the legislation.

House Republicans tried to address some of Gottlieb's concerns — defining eligible patients as those with a "stage of a disease or condition in which there is reasonable likelihood that death will occur within a matter of months" or would result in "significant irreversible morbidity that is likely to lead to severely premature death."

Passionate issue

But critics have argued that the measure would cut the FDA out as a gatekeeper, leaving desperately ill patients potentially exposed to "snake oil" salesmen peddling false hopes.

Former biotech executive Michael Becker, president and founder of MDB Communications LLC, who has been diagnosed with terminal cancer, argued that the right-to-try bill is not a pro-patient measure but a "libertarian Trojan horse designed to weaken the FDA."

"And once this dangerous precedent is set, I fear it could easily be expanded to include patients with less severe diseases," Becker wrote in a March 13 blog.

Gottlieb's predecessor, Robert Califf, took to Twitter on March 13 to express his concerns about the House bill.

"We all want to help people with critical illnesses, but we should get beyond the slogans and hyperbole," Califf tweeted. "I'm worried by what I see."

"Matching an experimental therapy to a patient's biology and illness requires time and skill. The FDA paperwork is the least time issue," the former FDA commissioner said.

Califf noted that 90% of drugs in early safety trials "don't make it to market either because they don't work or [because of their] excess toxicity."

But Rep. Joe Barton, R-Texas, insisted giving "false hope is better than none at all." He questioned why a patient should have to wait for FDA approval "if you are about to die anyway."

Rep. Morgan Griffith, R-Va., argued terminally ill patients should be given the choice of using unapproved drugs.

"They know it's unlikely to succeed, but they are willing and make the decision and the choice to take that chance," the Virginia Republican said on the House floor.

"They want to fight for life," Griffith said. "If I had a terminal diagnosis, I would even consider injecting monkey urine if I thought it would give me a few more months, a few more years with my children."