The House failed to pass a bill Tuesday backed by President Donald Trump that would give terminally ill patients the right to seek drugs that remain in a clinical trial phase and are not yet fully approved by the Food and Drug Administration.
The House failed to get the two-thirds vote it needed, in large part because Democrats complained the bill, which was released over the weekend, didn’t go through regular order or get a hearing. They also argued the bill did not do enough to protect patient rights.
GOP aides expect they’ll bring it back up for a vote under a rule allowing it to pass with a simple majority.
The bill, however, doesn’t mandate that companies must provide the drugs to those who ask for them.
The Senate passed a similar bill by unanimous consent last year, and the House made changes to it, meaning it will need to go back to the Senate for another vote if the House passes it Tuesday. Patients would be allowed to access drugs that have completed a Phase 1 clinical trial.
Proponents of the bill argue that it gives patients another chance to pursue treatment after exhausting other options or if they don’t qualify for clinical trials due to inclusion criteria. So-called “Right to Try” laws already exist in 38 states, and backers argue a federal law is needed to ensure greater continuity across state lines.
Trump brought renewed attention to the effort in January when he mentioned the proposal in his State of the Union address, saying “patients with terminal conditions should have access to experimental treatments that could potentially save their lives.”
After it was passed by the Senate, the House Energy and Commerce Committee worked to add changes to address concerns about the bill. The House version, which was released over the weekend, seeks to narrow the scope of eligibility to patients in which there is “reasonable likelihood that death will occur within a matter of months” or “a disease or condition that would result in significant irreversible morbidity that is likely to lead to severely premature death.”
The Senate version included a broader definition of eligibility that could have included chronically ill patients.
The House version also requires that patients get the same informed consent details about the drug as a patient who’s already undergoing clinical trials. And it includes real-time adverse event reporting and a standard to notify the FDA when a company provides an unapproved drug to a “Right to Try” patient.
The bill has long-time support from Vice President Mike Pence as well as conservative groups like Freedom Partners and Americans for Prosperity, two groups backed by the conservative donors David and Charles Koch.
The FDA already has a program in place, the Expanded Access Program, that grants terminally ill patients access to investigational drugs. Scott Gottlieb, commissioner of the FDA, said at a congressional hearing in October that the FDA authorized 99% of the more than one thousand requests “in recent years.”
“Emergency requests for individual patients are usually granted immediately over the phone and non-emergency requests are generally processed within a few days,” he said.
But “Right to Try” advocates argue that patients can save even more time by bypassing FDA permission and certain regulations.
While the bill cleared the Senate easily, it faces stronger opposition in the House. The top Democrat on the Energy and Commerce Committee, Rep. Frank Pallone, issued a statement Monday blasting the bill as needless legislation that undermines the FDA’s approval process and gives patients “false hope,” since it doesn’t require manufactures to actually provide the drugs to the patient.
Critics also fear the legislation could carry broader implications and lead to a slippery slope of bypassing FDA regulations for less urgent cases, such as those with chronic illnesses, for example.
“It’s all well and good and it seems like a nice thing to do for patients, but it’s actually probably not going to open up much access to them than what the FDA already approves,” said Holly Fernandez Lynch, a professor of medical ethics at the University of Pennsylvania. “The bigger challenge is this attack on the FDA’s very reason for existence.”
Marilyn Mongiello, a “Right to Try” advocate along with her husband, Frank, who was diagnosed with ALS and whose name is in the title of the House bill, said those who want to try experimental drugs know they’re not getting guaranteed access to the medicine. She acknowledged the bill won’t require companies to provide the drugs, but she said it removes one more hurdle for the opportunity to access them.
“There is no such thing as false hope. Because while there is breath, there is hope,” Frank Mongiello, who is unable to use his voice, said in an email using eye tracking technology.
It’s unclear yet just how much Democratic resistance the bill will face Tuesday. A House Democratic leadership aide said “there are serious concerns among Democrats about how the bill threatens patient safety, undercuts the drug development process, and provides no legal recourse for patients harmed by unapproved drugs.”
For those who oppose the bill, however, it could be a politically tough vote in an election year.
“Are Democrats really going to deny critically ill patients every opportunity to find treatment? This is state law in 38 states,” said a senior Congressional Republican aide. “Are they going to go against their own constituents?”