Proponents praised the measure as a way to give patients more options, while critics warned the move could unleash proverbial 'snake oil salesmen' on seriously ill patients.
A so-called "right-to-try" bill is on its way to President Donald Trump's desk, where it is expected to be signed into law, giving seriously ill patients greater leeway to undergo treatments that have yet to receive final approval from the Food & Drug Administration.
The House voted 250-169 on Tuesday in favor of the measure, which the Senate approved last August. Nine members did not vote.
“Terminally ill patients—and their loved ones—deserve the opportunity to try whatever option is available that may offer a chance for a longer life," Speaker Paul Ryan said in a statement. "For patients who may not qualify for certain trials—or who have tried all other options—this bill will allow them to access experimental treatments and therapies."
Under the legislation, patients with life-threatening conditions would be able to request permission from drug manufacturers to undergo treatments in active clinical trials. The treatments must have completed preliminary testing, and patients must have exhausted their other options.
- Signature to come: Trump is expected to sign the bill into law because he endorsed the proposal, in principle, during his State of the Union address in January. Additionally, the White House gave the bill a boost early last year, as The Wall Street Journal reported.
- Existing FDA channel: During testimony before a House subcommittee last October, FDA Commissioner Scott Gottlieb, MD, expressed a willingness to work with a federal right-to-try law. But he noted the FDA's Expanded Access program already approves 99% of applications seeking permission to use investigational drugs and biologics in patient treatment. The FDA receives more than 1,000 such applications annually, he said. In an interview with STAT last week, Gottlieb said right-to-try would make the FDA's job harder.
- Some question existing FDA channel: "Although FDA authorizes over 99 percent of expanded access requests, that figure is probably misleading," Roger D. Klein, MD, JD, a consultant with The Federalist Society's Regulatory Transparency Project, wrote in an op-ed for The Hill, arguing that a burdensome application process and discouragement from agency officials keep the number of applications quite low. Klein argued the federal right-to-try law would be an improvement.
- Some question those who question the existing FDA channel: "Rather than prioritizing what’s best for the terminally ill, some leaders of the right-to-try movement may be more invested in chipping away at the FDA’s authority," Helen Knight, who graduates this week with an MD from the Johns Hopkins University School of Medicine, wrote in an op-ed for The Baltimore Sun arguing against the right-to-try law. "The movement’s champions, including Americans for Prosperity and the Goldwater Institute, have worked to reduce federal regulatory authority on other fronts. Indeed, the Goldwater Institute is concurrently campaigning to diminish FDA authority by advocating for legislation to rollback restrictions on how companies communicate about off-label uses of drugs."
—Steven Porter is an associate content manager and online news editor for HealthLeaders, a Simplify Compliance brand.