Patients with life-threatening conditions could soon have a new way to ask drug makers for medicines the Food and Drug Administration hasn’t yet approved, after the House on Tuesday voted to approve a version of “right-to-try” legislation.
The House passed the legislation 250-169. Now, the measure awaits a signature from President Trump, who repeatedly and passionately supported the measure and is expected to sign the legislation into law. The Senate had already passed this version of the legislation last August.
The bill’s passage is a win for conservative and libertarian groups like the Koch-brothers-backed Americans for Prosperity and the Goldwater Institute. It is also the conclusion of a surprisingly and increasingly political saga that pitted those groups against many House Democrats, some drug makers, patient groups, and medical ethicists. Key House Republicans and former FDA commissioners have all raised concerns with the legislation, as has current FDA Commissioner Scott Gottlieb, though he remains supportive of the bill.
“Today’s vote is a beacon of hope for the patients who are desperately seeking the ‘right to try’ investigational treatments and therapies,” said Reps. Greg Walden (R-Ore.) and Michael Burgess (R-Texas), who together chair the Energy and Commerce Committee and its health subcommittee, in a joint statement.
The legislation that passed the House would allow patients with life-threatening conditions ask drug makers to share treatments that haven’t yet been granted FDA approval, though the treatments must be in active clinical trials, and must have cleared initial preliminary testing. Patients who want to use the pathway must have exhausted all their other options, and must be unable to participate in a given clinical trial. The legislation doesn’t obligate drug makers to provide the treatments, and it doesn’t prevent them from charging patients for their associated costs if they do.
The legislation is a broader, earlier version of a policy that House Republicans spent weeks working to refine, with input from supporters, drug makers, and FDA staff. That version would have limited the pathway to patients who might “die within a matter of months.” It also would have given the FDA more insight into the use of the medicines under the new pathway.
Gottlieb acknowledged last week that the bill that passed Tuesday would make it harder for him to protect patients than the more refined version, saying “we’d have to do a little bit more … in guidance and perhaps in regulation.”
A key Republican who helped draft the more refined version, Rep. Greg Walden of Oregon, blamed Senate Minority Leader Chuck Schumer of New York for blocking the Senate consideration of the more refined bill. Because the upper chamber wouldn’t pass that bill, the House decided to take up the Senate’s earlier version instead, in part because of pressure from the administration, several lawmakers and other sources said.
“This is something he’s worked very hard on,” House Majority Leader Kevin McCarthy of California said before the vote Tuesday, referencing the president. “I know of many times the president’s been making phone calls, so this is actually a very big success.”
For the most part, supporters of the movement have backed both bills. They say the legislation is necessary to help patients who are running out of time sidestep the overly cumbersome FDA rules and the agency’s slow-moving timelines.
“The Administration believes that treatment decisions for those facing terminal illnesses are best made by the patients with the support and guidance of their treating physicians,” the White House said in its statement of support Monday.
Opponents, meanwhile, say the legislation will allow “snake oil salesmen” to take advantage of desperate patients and could ultimately weaken or undermine the FDA’s authority to regulate drugs. They point out, too, that the FDA already has a system in place for granting terminally ill patients access to experimental therapies, often within a tight timeframe. Though opponents said the more refined version of the legislation was an improvement, they said it still failed to address many of their concerns.
“The legislation would roll back essential patient safeguards and could result in patients being harmed by unproven, and potentially unsafe, therapies,” a coalition of patient groups, including the American Cancer Society Cancer Action Network, Friends of Cancer Research, and the National Organization for Rare Disorders, wrote in a letter to lawmakers.. “Furthermore, the legislation would significantly restrict FDA’s ability to stop access to an experimental therapy and would remove expert consulting requirements on dosing and other important safety measures currently provided by FDA.”
PhRMA, the main trade group for drug makers, stayed neutral in its statement after the vote, as it has on previous versions of the federal legislation.
“We continue to believe that any legislation involving access to medicines that have not yet been approved must benefit and protect patients and not disrupt the future of clinical trials, U.S. Food and Drug Administration oversight and the research and approval of new medicines,” a spokesman said in a statement.
https://www.statnews.com/2018/05/22/house-vote-right-to-try/
