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FDA chief optimistic on solving 'right-to-try' dilemma on experimental drugs

U.S. Food and Drug Administration Commissioner Scott Gottlieb said he thought his agency could find a solution with Congress to allow critically ill patients who have exhausted all other options to have broader access to experimental drugs that have not gained FDA approval.

"I think we will be able to work out a proposal that can accomplish all the goals," Gottlieb said Feb. 6 at an event hosted by the advocacy group the Alliance for a Stronger FDA.

Gottlieb has been up front about his opposition to the so-called right-to-try legislation that passed the Senate last summer and is pending in the House.

But his boss, President Donald Trump, and Vice President Mike Pence have pressed lawmakers in recent weeks to finalize the bill and get it to the White House for enactment.

Trump used his Jan. 30 State of the Union address to publicly back the right-to-try legislation and reiterated his support a few days later at the Republicans' retreat at the Greenbrier resort in White Sulphur Springs, W.Va. He insisted that Gottlieb was heading up his administration's effort on the legislation, putting the FDA chief in a tricky spot.

Gottlieb told lawmakers at an Oct. 3, 2017, hearing that the Senate's right-to-try bill contained flawed criteria, which he said could expose more people unnecessarily to potentially dangerous side effects. He called for changes to the bill.

Compassionate use already in play

The FDA already has a long-running expanded access program, also known as compassionate use, in which regulators help terminally ill patients obtain and use investigational medicines outside of the clinical trial process.

"We've done a lot of work, both on my watch as well as before I got here, trying to improve the expanded access program, make it more accessible to patients, expand opportunities for patients to get drugs on a compassionate-use basis," Gottlieb said at the Feb. 6 event.

Critics of the right-to-try legislation have argued that the Senate bill would cut the FDA out as a gatekeeper, leaving desperately ill patients potentially exposed to "snake oil" salesman peddling false hopes.

Gottlieb said his agency has been providing "technical assistance to everyone who has asked us for technical assistance" on Capitol Hill and has been "working with multiple parties."

He said there were now "multiple proposals from Congress" under consideration, although he did not disclose any details about how those measures may differ from the Senate's bill or if his agency's powers would be weakened in any way — something he publicly pushed back against at last fall's hearing convened by the House Energy Subcommittee on Health.

But Gottlieb said the FDA in recent weeks has "made an effort to make ourselves available to Congress in trying to work for a solution that can work on behalf of patients."

"There's a lot of people interested in this, both in the patient community, and obviously, in the administration and also on Capitol Hill," Gottlieb said.

"I think we'll get there," he said.