A Republican-led effort to allow so-called Right to Try legislation failed to move forward late Tuesday in the House of Representatives, amid opposition — both muted and loud — by regulators, patient groups and the some in the pharmaceutical industry.
President Donald Trump gave his support during his State of the Union for the idea, which aims to provide access to experimental, unproven treatments to treat life-threatening illnesses when patients have run out of options. Vice President Mike Pence and the GOP funders the Koch brothers are also prominent backers.
The bill vote was 259-140, but that fell seven votes short of the two-thirds majority needed for passage under the legislative maneuver used to bring the bill to a House floor vote.
The high-profile backing and years of pressure from the libertarian Goldwater Institute fueled momentum for the legislation to provide access to drugs that have passed just Phase 1 trials.
But warnings from patient groups, researchers, key Democrats and even Trump's own Food and Drug Administration Commissioner Scott Gottlieb and other top FDA staffers may have helped derail the bill.
Many argued an existing FDA program aimed at providing unapproved treatments to desperate patients, dubbed expanded access or compassionate use, already serves the role. Those opposed to the Right to Try bill added that the risk of sidelining the FDA’s oversight role is too high.
Less than a month after Trump made his remarks in the State of the Union, the FDA released figures showing that in fiscal year 2017 the vast majority of expanded access applications were allowed to proceed.
FDA’s Center for Drug Evaluation and Research allowed 1,632 expanded access applications to proceed out of 1,637 in the last fiscal year, while FDA’s Center for Biologics Evaluation and Research reported that out of 205 requests, 199 were approved.
In October, the House Energy & Commerce health subcommittee held a hearing on a Senate-passed version of the bill sponsored by Sen. Ron Johnson, R-WI.
Gottlieb raised concerns over that bill’s scope, saying the language could drag in many chronic conditions that are life-threatening, yet remain manageable without unapproved therapies.
"The term used in the bill for ‘terminal illness’ would benefit from a clear definition. We recommend defining it as ‘a stage of disease in which there is a reasonable likelihood that death will occur within a matter of months,'" Gottlieb testified.
Rachel Sherman, FDA principal deputy commissioner, and Peter Marks, director of the Center for Biologics Evaluation and Research, have also both publicly raised concerns that Right to Try may sidestep FDA’s current system to the detriment of patients.
"Expanded access is something the agency has done … we almost never deny a single patient, but there is a safety check," Sherman said at one recent conference. "We don’t think responsible companies would be terribly interested in this, our primary concern, our only concern in this bill is that patients be protected."
House Majority Leader Kevin McCarthy, R-CA, on Twitter said that the defeat in the House would not be the end for Right to Try. "We will try again, pass legislation, and bring hope to those whose only desire is the right to try to live," McCarthy tweeted.
It's unclear if the House will attempt to attach Right to Try legislation to a larger vehicle such as the looming government funding omnibus, or if it will attempt to pass the Senate-passed Johnson bill.
Last minute lobbying on definitions
According to a lobbyist familiar with last-minute negotiations that occurred late last week, the new E&C bill first rolled out to stakeholders would have narrowed the definition even further, but that tighter definition was pulled back at the last minute after Goldwater lobbying. With a narrow vote to defeat the bill last night, it's unclear if the effort to expand the eligible population cost the legislation House passage.
The original House language shown to groups Thursday night defined an 'eligible illness' as: "(A) a stage of a disease or condition in which there is reasonable likelihood that death will occur within a matter of months; or (B) a rapidly progressive pediatric disease or condition, or progressive neurological disease or condition, where there is significant irreversible morbidity that is likely to lead to severely premature death," according to the source.
But that was changed and clause (B) instead referred to "a disease or condition that would result in significant irreversible morbidity that is likely to lead to severely premature death," in the final House bill brought to a vote last night.
It was unclear if FDA backed that final language, with the agency telling BioPharma Dive that it did not have a comment on the change shortly before the vote.
"We at FDA share the strong desire to help patients facing terminal illnesses access promising experimental treatments in their fight. We have been working closely with legislators to provide technical assistance as Congress works on this important issue to help ensure patients are also protected," FDA said in a statement.
"FDA will continue to work closely with Congress as they advance Right to Try legislation that gives terminal patients the chance to more safely access experimental treatments as part of our commitment to protecting patients while promoting public health," the agency added, before the vote.
A GOP spokesperson emphasized to BioPharma Dive that the final language still would have narrowed the definition from the Senate-passed bill.
Democrats and many patient groups argued that the bill provided false hope to patients, noting that the legislation does nothing to compel manufacturers to provide the investigational treatments.
"I strongly oppose this bill that puts vulnerable patients at risk by completely removing the Food and Drug Administration from the review or oversight of access to investigational therapies. This legislation simply is not needed – 99% of expanded access requests are already approved by FDA under the current system," E&C Committee Ranking Member Frank Pallone, D-NJ, said in a statement.
On their side were major disease-focused groups such as the American Cancer Society and American Lung Society.
Starlee Coleman, senior policy advisor at Goldwater, argued that Right to Try does not take the place of FDA’s expanded access, "we’re not striking that program and replacing it with a new program ... this is in addition to it."
Consumer watchdog Public Citizen also opposed the House legislation, saying that although it improves on the Senate-passed bill, it still has "fundamental flaws that would put vulnerable patients at risk and undermine their rights."
“The bill also would broadly immunize from liability manufacturers, sponsors, physicians, clinical investigators and hospitals for a wide range of conduct related to the provision of experimental medications to patients, including provision of such medications under the FDA’s existing Expanded Access program," Michael Carome, director of Public Citizen’s Health Research Group, said in a statement. "In this way, the bill would bar suits in a variety of situations in which state law might reasonably impose liability and would eliminate a strong incentive for manufacturers and health care providers to act responsibly and in the best interests of patients."
America’s Health Insurance Plans told Healthcare Dive that they do not have a formal position on Right to Try. PhRMA has also walked a careful line, urging Congress to maintain FDA’s oversight role in any Right to Try proposal.
But one major drug company appears hesitant to utilize any Right to Try law. Johnson & Johnson's biotech arm Janssen says in its pre-approval policy for investigational drugs that it will continue to use FDA’s expanded access program.
"We support these requests through our established review and evaluation processes, which includes independent review by the FDA to assure full consideration of available safety data of which the FDA may be uniquely aware," the company policy states.