House Rejects Bill to Give Patients a ‘Right to Try’ Experimental Drugs

WASHINGTON — In a surprising rebuff to President Donald Trump and Republican leaders, the House derailed a bill on Tuesday that would have given patients with terminal illnesses a right to try unproven experimental treatments.

The bill was considered under special fast-track procedures that required a two-thirds majority for passage, and it fell short. When the roll was called, 259 House members supported the bill, and 140 opposed it.

Most of the opposition came from Democrats, who said the bill gave false hope to patients and could actually endanger people dying of incurable diseases, because it would undermine protections provided by the Food and Drug Administration.

“By defeating this bill tonight, we protected patients and supported FDA’s continued role in approving experimental treatments that may help save a patient’s life,” said Rep. Frank Pallone Jr. of New Jersey, the senior Democrat on the Energy and Commerce Committee. “This bill should have never been on the House floor in the first place since it was only introduced today.”

Republican leaders rushed the bill to the floor using a procedure normally reserved for uncontroversial legislation. But the bill ignited passions among supporters and critics.

The Senate passed a similar bill last year, and Trump supported the legislation.

The House Republican leader, Kevin McCarthy of California, said the bill was not dead. “We will try again, pass legislation and bring hope to those whose only desire is the right to try to live,” he said.

The bill could be brought up again under rules that allow passage with a simple majority.

Going into the debate on Tuesday, supporters of the bill expressed confidence it would win approval.

“Our bill will give some relief to terminally ill patients who have no further options left to extend their lives,” said Rep. Andy Biggs, R-Ariz., where a state right-to-try measure won 78 percent of the vote in a 2014 referendum. “All Americans have the right and freedom to try to save their lives.”

But many doctors and advocates for patients oppose the legislation.

“Supporters of this legislation talk as if effective treatments are being withheld from patients,” said Dr. Robert M. Califf, who was the commissioner of the FDA under President Barack Obama. “The vast majority of experimental therapies are toxic or ineffective. The only way we find out is through controlled studies where we measure the effects.”

In a letter to House leaders, more than 75 patient advocacy groups, including the lobbying arm of the American Cancer Society, opposed the bill.

The American Medical Association said it “does not believe that the bill will substantially improve patient access to investigational therapies.” And the American Society of Clinical Oncology, representing cancer doctors, said the bill “could do more harm than good for patients with life-threatening illnesses” because it would remove the FDA from the evaluation of the risks and potential benefits of some treatments.

Drug companies can already provide experimental medicines to patients outside clinical trials under a program known as expanded access or compassionate use. The FDA says it approves about 99 percent of compassionate use requests. In emergencies or other urgent cases, the agency says, it approves requests for the use of experimental medicines within hours or days of being contacted by a doctor.

Rep. Jan Schakowsky, D-Ill., said the bill would have created “a dangerous back door around the FDA approval process.” Vice President Mike Pence, who signed a right-to-try bill in 2015 as the governor of Indiana, pushed for the federal legislation.

Trump endorsed the idea in his State of the Union address in January. “People who are terminally ill should not have to go from country to country to seek a cure,” he said. “I want to give them a chance right here at home. It’s time for Congress to give these wonderful, incredible Americans the right to try.”

The drug industry was uncharacteristically quiet. The main lobbies for the industry — the Pharmaceutical Research and Manufacturers of America and the Biotechnology Innovation Organization — said they had not taken a formal position on the legislation.

The drive for right-to-try legislation was spearheaded by the Goldwater Institute, a public policy organization that advocates free markets and limited government.

Starlee Coleman, a policy adviser at the institute, said 38 states had adopted right-to-try laws, often with broad bipartisan support.

The House and Senate bills would establish a new pathway providing access to unapproved prescription drugs for certain patients who had exhausted other treatment options. Under the Senate bill, patients with “a life-threatening disease or condition” could obtain unapproved drugs, but eligibility under the House bill was more narrowly defined.

To qualify under the House bill, a patient would have to have some kind of terminal illness: a condition that is likely to cause death “within a matter of months” or “irreversible morbidity that is likely to lead to severely premature death.”

The patient would have to provide informed consent in writing. The manufacturer would have to notify the federal government within seven business days of providing the drug to a doctor for use by a patient, and the doctor must immediately report any “serious adverse events” to the drugmaker.

If patients take an experimental drug under the right-to-try legislation and if it proves unhelpful, the FDA could not use the data to “delay or adversely affect” federal approval of the drug — unless the information was “critical to determining the safety” of the medicine.

The bill would also shield pharmaceutical companies, doctors and hospitals from some of the legal risks of providing drugs that have not been approved. Doctors and hospitals would generally be protected unless they engaged in gross negligence or willful, reckless or criminal misconduct, or they intentionally harmed a patient.

Nothing in the bill would require drug companies to provide experimental drugs to patients who want them, and companies turn down requests for various reasons. In some cases, they have only a limited supply of a product, which they want to use in clinical trials.

But some patients could benefit. “These are real people facing their mortality with no hope,” said Sen. Ron Johnson, R-Wis., the author of the bill passed by the Senate in August.