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Right-to-try experimental drugs bill on verge of becoming law

Legislation that would give U.S. patients greater access to experimental drugs beyond the U.S. Food and Drug Administration's current compassionate-use process is on the verge of becoming law after the House Rules Committee cleared the bill's way for debate and a vote on the chamber's floor.

The so-called right-to-try bill, which was adopted in the Senate in August 2017, is expected to be voted on in the House as early as May 22 after a one-hour debate.

Republicans ditched a different version of the legislation that passed the House in March in a 267-149 vote, albeit after two attempts.

Some Republicans had blamed Democrats for the House bill stalling in the Senate, but the chamber's majority leader, Sen. Mitch McConnell, R-Ky., has made no attempt to bring it to the floor.

The lead author of the Senate right-to-try bill, Sen. Ron Johnson, R-Wis., tried on March 23 to slip the House bill in for a unanimous consent vote in the middle of the night just before Congress adjourned for its spring recess, but Minority Leader Chuck Schumer, D-N.Y., quickly objected to the move, arguing neither Republicans nor Democrats in the chamber had had a chance to scrutinize it.

House bill better

During a May 21 session of the House Rules Committee, Republicans said their chamber's bill was a better product than Johnson's legislation, which he co-sponsored with Indiana Democrat Sen. Joe Donnelly.

But Senate Republican leaders were unwilling to make another move to bring the House bill to their chamber floor, noted Rep. Michael Burgess, R-Texas, chairman of the House Energy and Commerce Health Subcommittee.

"I'd prefer the House bill," he said, adding that the Energy and Commerce Committee worked with FDA Commissioner Scott Gottlieb and his staff to iron out certain issues and avoid the safety problems of the Senate bill.

But Burgess said passing the Senate bill was better "than doing nothing at all."

"We pass this on the floor of the House tomorrow and it goes down to be signed before the end of the week. People want to see that," the Texas Republican said.

Burgess, a doctor, said he hears from "parents of kids with muscular dystrophy all the time about lack of access to compounds or treatments that they think will help their children" after they had participated in a clinical trial, but the FDA determined the product was not ready for U.S. marketing approval.

"Why shouldn't they have the ability to use that?" Burgess asked.

No guaranteed access

Rep. Frank Pallone, D-N.J., ranking member on the Energy and Commerce Committee, noted there is no guarantee that patients can access experimental drugs under the right-to-try legislation, and biopharmaceutical companies cannot be forced to provide their medicines.

In addition, there is no guarantee insurance companies would cover the investigational products, and there is a risk patients could lose coverage if they use an unapproved medicine, he added.

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

The FDA approves 99% of the 1,000 compassionate-use program 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, he said.

"That process is working," Pallone said. "When there's a denial, it's not from the FDA. It's from the pharmaceutical company that decides they don't want to give it to you. And they don't have to."

He said it is also "not a good idea to take the FDA out [of the process], because if you do, then that oversight or those safeguards that they might put in play if they grant the approval disappear."

Rep. Joe Barton, R-Texas, who supports the Johnson bill, agreed that "the FDA rarely says no" to compassionate-use requests.

He said his own brother, who had liver cancer, had gone through the FDA process to obtain an experimental drug and received it. But in the end, the drug ended up accelerating his death, rather than helping him.

But Barton said he believed the Johnson bill was "an improvement over current law," though he said "It's not much."

"I don't really see a reason to oppose it because it could help. It could be a little bit faster," Barton said.

No evidence of helping, but it may harm

Even though about 40 states have passed their own right-to-try bills, Pallone said there was no evidence those measures have helped any patients obtain access to experimental medicines that they did not already achieve under the FDA's compassionate-use program.

The New Jersey lawmaker also noted that Gottlieb testified in October 2017 that under the Johnson bill, patients with potentially less severe conditions would be exposed to risks that should only be considered for people with terminal illnesses.

Gottlieb had urged the House last fall to adopt a narrower definition of patients diagnosed with a terminal illness than the broader "life-threatening disease or condition" criteria in the Johnson bill — a request the chamber met in the bill it passed.

The FDA chief, however, has reversed himself, declaring last week he now fully supports the Johnson bill, even though the language in the legislation has not changed from when he objected to it in his congressional testimony.

Pallone noted that at least 100 patient advocacy groups have opposed the Johnson right-to-try bill, warning that the legislation could make it easier for "bad actors and fly-by-night outfits to promote snake oil" to desperately ill and dying patients.

He said he doubted any legitimate biopharmaceutical companies would go outside the FDA process under the Johnson bill.

Closed-rule process

Pallone also raised objections to the House taking the Johnson bill to the floor for a vote without any committee consideration and markup.

Rep. Jim McGovern, D-Mass., ranking member on the Rules Committee, also complained that the Republicans were breaking their own guidelines by voting on a closed-rule process, under which no amendments could be added to the Johnson bill and only limited debate will be permitted on the legislation when it comes to the House floor.

McGovern said the Rules Committee hit a milestone of having its 84th vote on a closed rule.

"The 115th Congress is the most closed Congress ever," McGovern said, calling the situation "discouraging" and adding to an already "dysfunctional" Capitol Hill.