An overwhelming 90% of Americans say it's important or very important for patients with terminal illnesses or rare diseases to access cutting-edge treatments even before FDA approval, according to a new national survey released by the Goldwater Institute. The poll of 1,000 registered voters, conducted June 15–17 by Fabrizio Ward, reveals broad bipartisan support for expanding patient access to personalized, gene-based therapies that often can't be approved quickly under current federal rules. The findings come one month after Congress introduced the Right to Try for Individualized Treatments Act, legislation designed to let patients with rare diseases access treatments tailored to their unique genetic profiles.
The survey shows that 86% of respondents—including supermajorities of Republicans, Democrats, and independents—believe the FDA's approval process should be updated to accommodate personalized treatments based on a patient's genetic makeup. Support for the principle cuts across party lines, with Americans of all political stripes backing the right of patients to access potentially lifesaving gene-based treatments without having to seek government permission. The poll tested attitudes toward treatments that haven't yet been approved or simply can't be approved in a timely manner by the FDA, and found near-universal agreement that patients should have access close to home, near their families and support systems.
"Americans understand that government bureaucrats should never stand in the way of patients seeking access to lifesaving treatments," said Victor Riches, President and CEO of the Goldwater Institute, in the report. The survey indicates that "politicians who put up roadblocks to those patients do so at their own political peril," according to Riches. Respondents overwhelmingly agreed that updating the law to allow individualized treatments isn't lowering the bar for safety but recognizing that science has advanced in ways the FDA's original approval system wasn't designed for, the report states.
The survey's findings underscore a disconnect between medical innovation and regulatory frameworks, the report explains. Over the last decade, medical science has shifted toward individualized medicine, but these highly specific new therapies—often designed for a single patient—frequently clash with the FDA's outdated, one-size-fits-all regulatory approach. The Right to Try for Individualized Treatments Act, introduced in Congress in June by Rep. Diana Harshbarger, Rep. Andy Biggs, and Sen. Ron Johnson, builds on the original Right to Try law enacted in 2018, which allowed terminally ill patients to access investigational treatments that completed Phase I clinical trials but weren't yet FDA-approved. The new legislation, already passed in 18 states, would establish a clear legal pathway for patients with rare and ultra-rare diseases to access personalized medical breakthroughs. The report features Elijah Stacy, diagnosed at age 6 with Duchenne muscular dystrophy—a fatal muscle-wasting disease that typically claims patients' lives by their mid-twenties—whose younger brother died from the condition at just 14.
The bottom line, according to the Goldwater Institute: the American people demand federal reform to allow rare-disease patients to finally benefit from modern scientific advances. The survey confirms that the Right to Try for Individualized Treatments Act isn't just a win for rare-disease patients but a political winner as well, and political leaders who deliver for them will be rewarded.