WASHINGTON -- Earlier this week, two Republican congressmen to House Speaker Paul Ryan (R-Wis.) and Majority Leader Kevin McCarthy (R-Calif.) urging them to bring a "right-to-try" bill to a vote in the House as soon as possible.
The bill, called the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act, passed in the Senate last August.
It would allow individuals with life-threatening illnesses to obtain experimental drugs prior to FDA approval. However, the law does not require drug companies to make their products available, and the FDA already has a pathway to allow such access. Industry in general has not sought changes to that pathway. Nevertheless, some in Congress are unsatisfied with the FDA's policies and implementation and are seeking to loosen the reins.
"The fundamental purpose of the Right to Try Act is very simple: it merely allows terminally ill patients who have exhausted all other options to try medications that have passed basic Food and Drug Administration safety protocols but not completed the full, multiyear approval process. This bill safeguards any pharmaceutical company that may wish to participate in Right to Try, but it in no way requires participation to begin with," wrote Reps. Andy Biggs (R-Ariz.) and Brian Fitzpatrick (R-Pa.) in a letter signed by 40 other members and sent to House leadership on Monday.
"It is frustrating that we hit this logjam for months," Sen. Ron Johnson (R-Wis.), lead sponsor of the Senate bill, told the .
Still, he was pleased to hear President Trump pressure Congress to pass a federal Right to Try law in his State of the Union. "People who are terminally ill should not have to go from country to country to seek a cure,'" Trump said.
Right-to-try legislation has already been passed in 38 states, but Biggs and Fitzpatrick argued in their letter that a federal bill is needed because state laws are "being preempted by a lack of guidance at the federal level."
"Moving forward, placing [the right-to-try bill] on the suspension calendar would be the best and most expedient way to proceed, and we have no doubt that such an effort would be successful," they continued.
"[I]t is now time to move forward without delay. We owe nothing less to the millions of patients across the country who are fighting for their lives each and every day," Biggs and Fitzpatrick wrote.
Meanwhile, right-to-try advocates met with Vice President Mike Pence on Tuesday, according to the .
Representatives for the Goldwater Institute, a libertarian think tank and fierce advocates of the bill, argue that the FDA's own data suggest the agency has made the process of accessing investigational drugs too challenging.
"There is no possible way that only [about] 1,000 people per year want to try to save their own lives," a spokeswoman for the institute said. "We just fundamentally do not believe that you should have to apply to the government for permission to try to save your own life."
Despite the enthusiasm of these congressmen and other right-to-try advocates, the legislation has provoked sharp criticism from experts in medical ethics and drug regulation.
Alison Bateman-House, PhD, MPH, of NYU Langone Health in New York City, and several colleagues drafted a letter of their own, this time to House Energy & Commerce leadership, opposing the Trickett Wendler bill and all other right-to-try measures that the committee is currently considering. It was sent with more than 300 signatures.
In the letter, Bateman-House and her colleagues recalled congressional testimony of FDA Commissioner Scott Gottlieb, MD, who in October 2017 worried over the "breadth of the language" in the bill, they wrote.
As critics of right-to-try point out, the FDA has an "expanded access" pathway, which is sometimes called " and 99% of patients' requests to receive experimental treatments for expanded access are approved "within a few days or hours," noted Bateman-House.
"It is important to remember that the current regulatory system for medical products and research in the United States was created as a result of serious patient harm," she said, citing.
"While obtaining unapproved therapies outside of a clinical trial is not about research, the products themselves remain experimental and have not been shown to be safe and effective ... Patients with terminal conditions who access unapproved therapies outside of clinical trials may be at risk of hastened death or reduced quality of the life that they have left, and deserve protections similar to patients taking part in clinical trials," Bateman-House and her colleagues continued.
"Expanded access can be improved, but the right to try approach is misguided and would likely do more harm than good," according to the letter.
At a briefing earlier this week, Gottlieb said that "trying to facilitate access to treatment for patients who face terminal diagnoses is a high priority."
And with regard to the proposals in Congress, the agency is making itself available to work out a solution.
"I think we'll get there," he said.