The House is set to take up legislation aimed at giving critically ill patients greater access to experimental drugs beyond the Food and Drug Administration's current compassionate use process — a movement known as right to try.
Shortly after midnight on March 10, House Energy and Commerce Committee leaders unveiled an updated version of the right-to-try legislation that passed the Senate in August 2017 by a voice vote. The lawmakers, along with Majority Leader Kevin McCarthy, R-Calif., confirmed March 11 that the bill would be formally introduced the next day, with a vote scheduled for March 13.
"We are a resilient and resourceful nation, and when faced with long odds, Americans should be given the opportunity to harness our country's vast medical innovation by accessing experimental treatments," McCarthy stated.
He noted that if the legislation was adopted by the House, the bill would need to go back to the Senate before it could be signed by President Donald Trump.
The White House has been putting pressure on the House since last summer to take up the bill.
Trump used his Jan. 30 State of the Union address to publicly back the right-to-try legislation.
Vice President Mike Pence also chimed in on Twitter, telling Congress on Jan. 18 "Let's get this done."
At the Republicans' Feb. 1 retreat, Trump insisted FDA Commissioner Scott Gottlieb was heading up the administration's effort on the legislation, putting the agency chief in a tricky spot.
Gottlieb had criticized the Senate's bill at an Oct. 3, 2017, hearing, saying it contained flawed criteria, which he said could expose more people unnecessarily to potentially dangerous side effects. He called for changes to the bill.
The new House bill appears to address some of Gottlieb's concerns — defining eligible patients as those with a "stage of a disease or condition in which there is reasonable likelihood that death will occur within a matter of months" or would result in "significant irreversible morbidity that is likely to lead to severely premature death."
At a Feb. 6 briefing, Gottlieb told reporters that the FDA had been providing "technical assistance" to Capitol Hill. He said he thought the agency would be able to find a solution with Congress.
Gottlieb followed that with a series of tweets on Feb. 11, in which he said the FDA was "committed to working closely [with] Congress to advance right-to-try legislation that gives terminal patients [a] chance to access promising treatments as part of our commitment to promote public health options."
But critics have argued that the right-to-try legislation would cut the FDA out as a gatekeeper, leaving desperately ill patients potentially exposed to "snake oil" salesmen peddling false hopes.
In a Feb. 6 letter to House leaders, 38 advocacy groups said the right-to-try legislation, in the end, would not give patients greater access to experimental therapies and "would likely do more harm than good."
More than three dozen states have passed their own right-to-try bills.
But in a March 11 op-ed published in The Hill, Cognition Therapeutics CEO Kenneth Moch, Isaac Foundation Executive Director Andrew McFadyen and New York University bioethics professor Arthur Caplan insisted those state laws have actually increased the risk to patients and the biopharmaceutical development process by creating a path to circumvent the FDA regulatory and oversight processes by permitting access to experimental medicines directly from drugmakers.
"No ethical biopharmaceutical company would allow its experimental medicine to be used without FDA oversight and involvement," they wrote.
They also noted that the FDA has approved about 99% of all of the compassionate use applications it has received.
Drugmakers cannot be forced to provide their experimental treatments to patients under the FDA's compassionate use program or the right-to-try legislation.
The legislation would, however, provide liability protection to drug companies, physicians, clinical investigators and hospitals that participate in the existing compassionate use program and the new alternative pathway, unless there is reckless or willful misconduct, gross negligence, or an intentional tort.
The House bill would also require pharmaceutical manufacturers to provide more statistical information about their right-to-try requests, provisions and outcomes, "which is a good thing," Holly Fernandez Lynch, an assistant professor of medical ethics and health policy at the University of Pennsylvania, tweeted March 11.
While the bill would require right-to-try requests to be approved by institutional review boards, Fernandez Lynch insisted that is no substitute for the FDA's oversight, although perhaps those independent panels would seek the U.S. regulatory agency's guidance.
Still, she argued the legislation provides nothing more than a right to ask for access to experimental treatments, "selling patients false hope that could hurt them."
Azar sheds some light
After Health and Human Services Secretary Alex Azar last week warned healthcare providers, insurers, pharmacies and drugmakers that they needed to be more transparent or the government would use its "levers" to force a change, the agency chief decided it was time he also shed some light on his own plans.
During a 30-minute March 8 afternoon briefing with reporters, Azar took questions on a range of topics — from Virginia's fight over Medicaid expansion to whether the Centers for Disease Control and Prevention could study gun violence or use the terms "science-based" and "evidence-based."
Azar was also asked about whether he supported guaranteed-renewability of short-term health insurance plans — sometimes called "skimpy plans" or "junk insurance" by opponents.
The administration on Feb. 20 unveiled a new proposed rule that would expand short-term plans from the current three-month limit to a full year.
Azar noted that one of the questions the administration was seeking public feedback about was whether his agency has the legal authority to allow renewability of those plans.
"We would like to be able to do renewability of these plans for individuals because we think they're low-cost options," Azar told reporters. "If it's right for that person in their situation, we would like for them to have the ability to have renewability."
But if the administration determines it lacks the power or it is too vague, "we are working with Congress to see if we might be able to get clearer authority of renewability if they're considering a package around various reforms," he said.
"We'd like to see the ability to give people the option of renewability in whatever form we could have it," Azar said.
Asked whether he backed the White House's criticism of the Virginia Democrats' attempt to expand Medicaid, Azar said he was "not going to comment on any individual state's process of their legislation and what they may or may not be thinking about in terms of driving forward."
"I have enough fish to fry with actual waivers and requests that come in," he told reporters, adding that he was leaving Virginia's decision up to its legislators.
Azar was also asked about reports that former CDC Director Brenda Fitzgerald had tried to ban certain terms at her agency, like "evidence-based" and "science-based" — a claim she denied.
"We are a science and evidence organization," Azar said, adding that those terms do not have any ideological overtones.
"We use science and evidence as we make our decisions," the health chief said.
He acknowledged, however, that data can be challenged and said he wanted his agency to be a "very robust debating organization."
"I think a healthy organization has that kind of debate and challenging among scientists or even policymakers — challenging assumptions, challenging data, really probing, pushing to make sure we're getting to the right answers," Azar said. "We owe the American people to get to the right answers, the best answers and keeping an open mind as data changes or as new studies come in. That's the approach that we will follow."
Azar reiterated and further clarified remarks he made during a Feb. 15 House hearing about permitting the CDC to study gun violence — a topic the agency has shunned for the most part of the past 20 years for fear of violating the 1996 Dickey Amendment.
The idea that there is a ban on the CDC or any other U.S. health agency conducting research on violence involving guns is "an incorrect narrative," Azar told reporters.
The Dickey Amendment is about expenditure of funds for promotion or advocacy of gun control, not research, he said.
He noted the National Institutes of Health "has had many projects dealing with violence, including gun violence."
Azar emphasized, however, that while there are no legal restrictions on studying gun violence, the CDC has limited research funds and many "competing priorities," like infectious diseases such as pandemic flu, Zika and Ebola.
