Congress Passes Right-to-Try Bill

Kerry Dooley Young

Disclosures

May 22, 2018

WASHINGTON — House lawmakers passed a "right-to-try" bill today, clearing the path for President Donald J. Trump to sign it.

The bill is intended to make it easier for people with life-threatening conditions to access experimental treatments outside of clinical trials. Many advocacy groups argue the measure will erode needed protections and put patients at risk for harm from unproven and potentially unsafe therapies.

The House voted 250-169 on a version of the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017, which the Senate approved last year by unanimous consent.

The vote is a victory for Trump, who has been pressuring GOP lawmakers to send him a bill on the issue. Today he retweeted Vice President Mike Pence's tweet celebrating the vote. "This important policy will give hope to the those who need it most — by allowing seriously ill patients a fighting chance to access experimental treatment," Pence said.

Backers of the bill say the US Food and Drug Administration's (FDA) expanded access program for experimental medicines represents too high a hurdle for many patients, although the agency approves about 99% of such requests. They say another pathway for people to get medicines when they cannot enroll in a clinical trial was needed.

"FDA is constantly striving to make improvements to its expanded access program. Some patients and their families, however, still have challenges accessing investigational treatments," the White House had said in a Monday statement in support of the bill. The Trump administration "believes that treatment decisions for those facing terminal illnesses are best made by the patients with the support and guidance of their treating physicians."

Groups such as Americans for Prosperity and the Goldwater Institute have been urging lawmakers to create a new pathway for people to access experimental drugs outside of clinical trials. Americans for Prosperity in April announced its plan for a national television and targeted digital ad campaign that called on Congress to pass right-to-try legislation.

"Millions of Americans who have been told they are out of options and it's time to get their affairs in order, are closer to having the opportunity for one last treatment, without having to get permission from the federal government first," said Victor Riches, president and chief executive of the Goldwater Institute, in a statement about the House's Tuesday vote.

"Dangerous" Path

Critics see the right-to-try bill as an attempt to undermine the authority of the FDA. The bill's opponents include more than 100 medical and consumer groups that signed a May 21 letter spearheaded by the American Cancer Society (ACS) Cancer Action Network, asking House leaders to abandon plans to move the Senate version of the bill. The groups include the American Society of Clinical Oncology (ASCO), the National Comprehensive Cancer Network, the Association of American Medical Colleges, the National Organization for Rare Disorders, and the American Lung Association.

In a separate statement on Tuesday, the consumer watchdog group Public Citizen noted that four former FDA commissioners and hundreds of bioethicists also asked lawmakers to drop their efforts to clear this bill.

The legislation "opens the gate to a dangerous, uncharted pathway for accessing experimental medications that have not been shown to be safe or effective," said Michael A. Carome, MD, director of the Health Research Group at Public Citizen, in a statement. "The bill passed today will expose vulnerable patients to risks of serious harm, including dying earlier and more painfully than they otherwise would have, without appropriate safeguards."

Carome urged drugmakers to stick with the FDA's expanded access program as a "much safer" route for helping those who can't enter clinical trials. The drugmakers' trade group, the Pharmaceutical Research and Manufacturers of America (PhRMA) , has not previously taken a public position on right-to-try bills.

In a Tuesday statement, the group said that its member companies "take the issue of patient access to investigational medicines seriously and have advocated for criteria to consider when to grant access to an experimental treatment outside of a clinical trial.

"As set forth in PhRMA's Principles on the Conduct of Clinical Trials, we believe criteria should include that the patient has a serious or life-threatening illness, the investigational drug remains under active clinical development and the patient is ineligible for, or otherwise unable to participate in a clinical trial," the group said.

Patient Definition

Opponents of the right-to-try bill were particularly disappointed that House GOP leaders abandoned their chamber's own version of this legislation in favor of the Senate's.

The House in March passed its own version of a right-to-try bill. That bill specified that an eligible patient must face "a stage of a disease or condition in which there is reasonable likelihood that death will occur within a matter of months," or have a disease or condition "that would result in significant irreversible morbidity that is likely to lead to severely premature death."

The Senate bill defines patients' eligibility more broadly as those who have been diagnosed with a life-threatening disease or condition and who have exhausted approved treatment options and are unable to participate in a clinical trial involving a desired eligible investigational drug.

Rep. Frank Pallone Jr (D-NJ), the ranking Democrat on the House Energy and Commerce Committee, said that backers of the Senate version of the right-to-try bill downplayed the size of the population that would become eligible for experimental treatments.

The term "life-threatening disease or condition" could sweep in chronic and often manageable afflictions, such as diabetes or chronic heart failure, Pallone said in a speech from the House floor. As a result, a bill depicted as offering hope for a few desperate patients could have a major effect on medical research, he said.

"Supporters of this bill would have you believe this legislation is targeted to those with terminal illnesses, but that is simply not the case," Pallone said. "This exposes an even greater number of patients to risk, and undermines our clinical trial program by diverting patients from trials that could support full approval to the alternative pathway."

The ACS Cancer Action Network, ASCO, and other signers of the May 21 letter to House leaders also questioned the decision to use the Senate's version of the bill.

"While we did not support the recent House passed version of this legislation, the House legislation includes improved patient safeguards compared to the Senate version," they 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," they added.

Still, the coalition of medical groups stressed the shortcomings in the House right-to-try bill as well as the Senate one.

"Additionally, both versions prohibit FDA from halting access to these experimental therapies short of placing a clinical hold on all clinical research on the therapy in question," the groups wrote. "Both House and Senate versions would also remove FDA's consultation on dosing, route of administration, dosing schedule, and other important safety measures available under FDA's current expanded access program."

Rep. Michael C. Burgess, MD (R-TX), chairman of the House Energy and Commerce's health subcommittee, said he was proud to have supported his chamber's version of the bill, which "currently remains idle in the Senate." But he said he was pleased that the House was proceeding with the Senate-passed bill to try to give terminally ill patients a "chance, or maybe a second chance, at life.

"These patients are our constituents. They could be someone we know. Let us take this

opportunity to improve access to experimental treatments for them and give them renewed hope," Burgess said before the vote, urging his colleagues to send the "ground-breaking legislation to President Trump's desk."

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