House Passes Right-to-Try Bill Over Many Groups' Objections

Kerry Dooley Young

March 22, 2018

The US House of Representatives passed a bill last night that many Republican lawmakers say will make it easier for people with terminal diseases to access experimental therapies. Opponents of the bill, including many well-known medical groups, say it would put desperately ill people at risk.

This is the second attempt by the House after failing last week to pass a right-to-try bill under a procedure known as the suspension of rules, which sets a higher threshold for success. GOP leaders returned this bill to the floor under a rule allowing for passage with a simple majority vote.

This time, the House passed the bill, called the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2018 (HR 5247), by a vote of 267 to 149.

There was both bipartisan support and bipartisan opposition to the measure in the Wednesday vote, with 35 Democrats backing the bill and two Republicans rejecting it, according to the House clerk's tally.

The bill differs from a right-to-try bill (S 204) that the Senate passed last year by unanimous consent. On March 20, the Wall Street Journal's editorial board urged that the House simply pass the Senate's version of the bill and thus quickly send it to President Donald J. Trump for his signature. The newspaper noted that a single member of the Senate could hold up a bid to clear the House's version of the bill in that chamber, adding that this outcome "is probable now that right-to-try has attracted opposition from House Democrats."

Strong Headwinds

Opponents of the House bill already are making an appeal to block the measure in the Senate.

"We urge the Senate to reject this legislation and instead continue to engage with the patient community in constructive discussion on ways to meaningfully improve patients' ability to access new treatments while preserving critical patient protections," said Chris Hansen, president of the American Cancer Society Cancer Action Network (ACS CAN), in a statement after the House vote.

ACS CAN said about 80 other medical and consumer groups joined it in opposing the House bill. The American Society of Clinical Oncology, the Leukemia and Lymphoma Society, and the National Comprehensive Cancer Network were among the signers of a March 12 letter spearheaded by the ACS CAN to congressional leaders.

Critics of the right-to-try bill note that the US Food and Drug Administration (FDA) already has an expanded access program that allows terminally ill people to obtain medicines outside of normal clinical trials. The investigative arm of Congress, the Government Accountability Office, last year reported that of the 5800 expanded-access requests submitted to the FDA from fiscal year 2012 through 2015, the agency allowed 99% to proceed.

FDA Commissioner Scott Gottlieb, MD, has said the agency authorizes these requests quickly. "In the case of emergencies, FDA will typically provide authorization over the phone in a matter of hours," he wrote in a blog last year.

In his Wednesday statement, ACS CAN's Hansen said the House bill would remove the FDA "from consulting on drug dosing, scheduling, and administration, among other important safety measures that the agency is uniquely qualified to understand.

"Patients will not likely see increased access to treatments under this bill but would instead face increased risks from weakened oversight and safety measures," Hansen said.

Four recent past commissioners of the FDA criticized the House and Senate right-to-try bills through a joint statement given to the Washington Post and confirmed by Medscape Medical News. "There is no evidence that either bill would meaningfully improve access for patients, but both would remove the FDA from the process and create a dangerous precedent that would erode protections for vulnerable patients," said Robert M. Califf, MD, and Margaret Hamburg, MD, who led the FDA during the Obama administration, and Mark B. McClellan, MD, PhD, and Andrew von Eschenbach, MD, who served under President George W. Bush.

Support for the Bill

Supporters of the bill argue that it creates another pathway for people who don't qualify for clinical trials to secure experimental drugs. In a joint statement, Greg Walden (R-OR), who is chairman of the House Committee on Energy and Commerce, and Rep. Michael C. Burgess, MD (R-TX), the chairman of Energy and Commerce Committee's health subcommittee, said the right-to-try bill would establish a "robust" informed consent policy to allow access to unapproved drugs and guard patients from misbranding or mislabeling of drugs by manufacturers. The measure also would provide liability protections for manufacturers, physicians, clinical investigators, and hospitals that participate in the existing expanded access program and the new alternative pathway, except in cases of reckless or willful misconduct or gross negligence, the lawmakers said in a statement.

"With the House's passage, we're one step closer in delivering for patients with no other treatment options who are desperately seeking the right to try investigational treatments and therapies," Burgess and Walden said in the joint statement.

Trump and Vice President Mike Pence are strong backers of the right-to-try approach, as is the conservative Phoenix, Arizona–based Goldwater Institute. The institute, which has been spearheading right-to-try laws at the state level to build support for the concept, called the House vote on Wednesday a "win for patients."

"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.

Counteroffer Rebuffed

Before passing the right-to-try bill on Wednesday, the House rejected in a 182 to 233 vote what amounted to a counteroffer from Rep. Frank Pallone Jr (D-NJ). Pallone, the ranking Democrat on the House Energy and Commerce Committee, said the text of his motion shouldn't have been a surprise for either Rep. Walden or Rep. Burgess.

"It is the bipartisan proposal that our staffs were negotiating prior to the introduction of the current Republican bill," Pallone said.

Pallone's motion to recommit would have struck the GOP proposal and would have replaced it with a call for new federal guidance on the use of clinical outcomes associated with the use of investigational drugs that were outside of normal trials.

Pallone said his measure would improve on the FDA's current expanded access program, building off suggestions previously made to the Energy and Commerce Committee at an October hearing. The FDA has been wrestling for some time with demands that it help people who face terminal illness more easily gain access to experimental therapies.

Pallone said his measure would have addressed specific issues that have been raised about the FDA's expanded access program, which include concerns about liability and about how patients' experiences will affect bids to win regulatory approvals.

Under his proposal, the FDA would be asked to issue guidance about how and when it would consider clinical outcomes and when a company could request consideration of these outcomes when applying for regulatory approval. This approach would give companies more clarity about how providing drugs to more patients outside of clinical trials might affect the chances for subsequent approval of these medicines, he said. The proposal also would provide liability protection to physicians, manufacturers, and clinical investigators in hospitals if they are in compliance with the current law and regulations for expanded access, Pallone said.

Pallone argued that the FDA must maintain its current active role in deciding which patients should have access to experimental drugs in order to protect them from treatments that might do more harm than good. He also noted the objections of medical groups such as the ACS CAN to the Republican measure.

"The supporters of this bill claim to be helping desperate patients who are looking for hope," Pallone said. "If this is such a patient-centered bill, why does every major patient organization overwhelmingly oppose it?"

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