Trump, who forcefully pushed for the legislation in his State of the Union address and recent drug pricing speech, will likely sign the bill into law.
“The Administration believes that these patients and their families should be able to seek access to potentially life-saving therapies while those treatments are still under review by the FDA,” an administration policy statement said.
The legislation, formally known as the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017, passed via unanimous consent in the Senate last August. That passage came after Johnson threatened to hold up the FDA Reauthorization Act of 2017, which extended FDA user fees and was considered must-pass legislation.
The drugmaker lobby has been noticeably neutral towards the effort, though some individual companies have backed the existing FDA expanded access program.
More than 100 patient advocacy groups, including The American Cancer Society Cancer Action Network, Friends of Cancer Research and the National Organization for Rare Disorders, urged House Speaker Paul Ryan, R-Wis., and Minority Leader Nancy Pelosi, D-Calif., to reject the bill in a letter Monday. The groups noted that while they did not support the previous House-passed bill either, it contained several safeguards compared to the Johnson legislation.
“The Senate version of the legislation is less safe than the pathway proposed in the House version and is dangerous compared to the current expanded access process,” the groups wrote. “The Senate’s bill would allow unproven therapies to be given to patients without FDA notification for up to a full year and would not establish any standards for informed consent.”
Senior House GOP leadership praised the bill as a leap forward for patients.
“It’s a bill that really cuts through some of the red tape and bureaucracy at the FDA, and allows people that are terminally ill to have real tools that they need, and real drugs and access to the things that they need to have a chance to save their life,” House Majority Whip Steve Scalise, R-La., told reporters Tuesday.
But House Energy & Commerce Committee Ranking Member Frank Pallone, D-N.J., slammed the Senate version on the floor, pointing to Gottlieb’s October testimony before the committee and FDA’s expanded access program as evidence that the bill is dangerous and unnecessary.
Gottlieb previously raised concerns over multiple aspects of the Johnson legislation, saying the language used to describe the eligible population could accidently drag in many chronic conditions that are life-threatening and inhibit FDA’s ability to properly regulate under the Federal Food, Drug, and Cosmetic Act.
“If enacted without changes, sponsors and other persons providing eligible investigational drugs to eligible patients under the specified conditions of the bill would not be subject to a number of sections of the FD&C Act and FDA regulations related to clinical trials, premarket approval and labeling. The current language in S. 204 may therefore preclude FDA from taking enforcement actions based on those provisions,” Gottlieb testified last October.
Rachel Sherman, FDA principal deputy commissioner, and Peter Marks, director of the Center for Biologics Evaluation and Research, have also publicly warned Right to Try may hinder FDA’s ability to protect patients.
FDA’s expanded access program already works to provide experimental treatments to terminally ill patients, Pallone noted. In fiscal year 2017, the vast majority of expanded access requests were approved by FDA: the Center for Drug Evaluation and Research allowed 1,632 to proceed out of 1,637, while CBER approved 199 out of 205 requests.
Despite reservations over the Senate bill, both Gottlieb and E&C Chair Greg Walden, R-Ore., fell into party line in the days prior to the House vote. “It is time for the House to do what Senate Democrats won’t and send a right-to-try bill to the President’s desk, bringing hope to terminally-ill patients across the country,” Walden said in a statement upon the vote’s announcement.