The White House is making a major push for a House vote soon on a bill that would let terminally ill patients try experimental drugs.
President Trump called for Congress to pass "right to try" legislation in his first State of the Union address last week as well as at the Republicans' retreat afterward. The endorsement comes as Vice President Mike Pence has pressed to get legislation passed in the House after the Senate approved it last year.
The House Energy and Commerce Committee is working on a version of “right to try” legislation, but there is no timetable on when the committee will vote on it.
The federal legislation would allow a patient with a life-threatening disease to use an experimental drug if all other options failed, bypassing Food and Drug Administration requirements.
Trump in his State of the Union address endorsed the idea, saying "patients with terminal conditions should have access to experimental treatments that could potentially save their lives."
"People who are terminally ill should not have to go from country to country to seek a cure — I want to give them a chance right here at home. It is time for the Congress to give these wonderful Americans the 'right to try.'"
Pence approved a state version of right-to-try when he was governor of Indiana, one of 38 states to do so. However, the laws don't have any impact, because they cannot circumvent federal law.
Rep. Greg Walden, R-Ore., chairman of House Energy and Commerce, told the Washington Examiner that he met with Pence on the legislation.
“We are making good progress,” he said. “We have been working with the FDA and vice president directly trying to get it to a good place.”
Pence tweeted Jan. 18 that he met with FDA Commissioner Scott Gottlieb on the importance of passing the legislation.
“It’s about restoring hope and giving patients with life-threatening diseases a fighting chance,” he tweeted.
Walden didn’t offer a timeframe for when the committee hopes to bring up the bill. He said committee members are trying to get the legislation to a place where it can get “bipartisan support in the House."
The bill passed the Senate in August through unanimous consent.
Conservative groups have been pushing for a House vote on the legislation.
“We are trying to strike when there is an opportunity,” said David Barnes, policy director for Generation Opportunity, a political advocacy group funded by the Koch brothers. He added that he hopes to get a vote on the bill by the end of June.
The pharmaceutical industry has not opposed the legislation after working with the Senate's main sponsor, Sen. Ron Johnson, R-Wis.
"We believe the revised right-to-try legislation that passed the Senate includes important protections for patient safety and the clinical trial process," said Andrew Powaleny, spokesman for the Pharmaceutical Manufacturers and Researchers of America.
Under the Senate legislation, a drug must have completed an early phase I clinical trial. Normally, a drug must complete a phase III trial to be considered for FDA approval.
But patient safety groups say a phase I trial doesn’t provide enough information on the drug.
“Phase I typically involves testing a single dose of the drug and trying to establish the highest dose that is too toxic,” said Michael Carome, director of Public Citizen’s health research group. “They provide no meaningful data on effectiveness and very preliminary data on safety.”
Critics also question the need for the legislation since the FDA already has a “compassionate use” program that allows use of an experimental drug outside of a clinical trial. The agency says it approves 99 percent of the roughly 1,000 “compassionate use” requests it receives each year. The FDA and at least one member of a panel of experts called an institutional review board must approve the use of the drug.
The Senate legislation would let patients bypass that program, which can help monitor the safety of the drug's use.
Carome also argues that the bill creates false hope for patients because the drugs may not work, since they need more testing to ensure they are effective.
Barnes said the bill does not circumvent the FDA because any drug to be considered would need to pass the safety phase of agency testing.
He said the bill comes down to removing bureaucratic barriers that prevent a doctor and patient from being allowed to make a decision about whether to try an experimental treatment if all others have failed.
But the manufacturer can be a major hurdle for getting access to the drug. Neither the "right to try" legislation nor the "compassionate use" program forces a company to provide the treatment.
“Companies may only have limited supply of a drug for just testing on clinical trials,” Carome said.
Another major concern for drugmakers that could prompt them to withhold experimental treatments is that a death or injury could jeopardize full agency approval.
People who seek experimental treatments under “compassionate use” typically are in a more advanced stage of the disease than clinical trial participants, Gottlieb said in an October 2017 blog post. The FDA does not comment on pending legislation.
“FDA recognizes that these factors make it more difficult to determine the cause of an adverse reaction,” he wrote.
So, the FDA clarified when a drugmaker needs to report adverse events. Previously, a drugmaker had to report every death or serious injury if it occurs during a clinical trial or through “compassionate use."
However, Gottlieb said in October that a drugmaker has to report a death or injury that occurs outside of a clinical trial only if “there is evidence to suggest a causal relationship between the drug and the adverse event.”
That means a drugmaker would have to report a death or injury only if it is related to the drug, not if the patient dies from another cause.
Gottlieb said the clarification is part of an effort to entice drugmakers to provide more experimental drugs for “compassionate use.”