RIGHT-TO-TRY HEADS TO PRESIDENT TRUMP

Johnson Right to Try bill clears House after Walden, Gottlieb fall in line
By David Lim
• May 22, 2018

Dive Brief:

The House of Representatives Tuesday voted 250 to 169 to pass Sen. Ron Johnson’s, R-Wis., Right to Try legislation, sending the bill to President Donald Trump’s desk. The vote is the culmination of a multi-year campaign by the Goldwater Institute for the legislation, which aims to provide access to unapproved experimental drugs under review at the Food and Drug Administration to patients facing life-threatening illnesses when they have no further options.

The Senate bill, which covers a much broader population than an earlier House-passed version, is set to be signed by Trump. The president has been a vocal advocate for the legislation despite warnings from multiple patient groups, researchers, key Democrats and top FDA staffers including Commissioner Scott Gottlieb that it may have unintended effects and potentially hinder FDA’s authority without changes.

Conservative groups including Freedom Partners and Americans for Prosperity backed the Senate bill, calling attention to the fact that 40 states have passed versions of Right to Try, a strategy employed by Goldwater to drum up momentum for the bill.

Dive Insight:

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," a Statement of Administration Policy 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 on Aug. 3, 2017. The Senate passage of the Right to Try bill came after Johnson threatened to hold up the FDA Reauthorization Act of 2017, which extended FDA user fees and was considered must-pass legislation.

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.