The message is loud and clear: Americans of all political stripes overwhelmingly back the right of patients with rare diseases to access potentially lifesaving gene-based treatments without having to beg the government for permission.
In fact, 90% of respondents to a new national survey for the Goldwater Institute say it is important or very important for patients with terminal illnesses or rare diseases to have access to the most promising, cutting-edge treatments available, even if those treatments haven’t yet been approved—or simply can’t be approved in a timely manner—by the U.S. Food and Drug Administration.
The survey makes certain that Goldwater’s Right to Try for Individualized Treatments Act, or Right to Try 2.0, which was introduced in Congress last month, is not only a win for rare-disease patients, but it’s a political winner as well. Supermajorities of Republicans, Democrats, and independents—86% overall—say it’s time for the FDA’s approval process to be updated to accommodate personalized treatments based on a patient’s unique genetic profile, according to the survey of 1,000 registered voters conducted June 15-17 by the Fabrizio Ward polling firm.
“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. “This survey shows that politicians who put up roadblocks to those patients do so at their own political peril.”
The groundbreaking Right to Try for Individualized Treatments Act was introduced in Congress in June by U.S. Rep. Diana Harshbarger (R-TN), Rep. Andy Biggs (R-AZ), and Sen. Ron Johnson (R-WI). It builds on the success of the original Right to Try, which was enacted in federal law in 2018, allowing terminally ill patients to access investigational treatments that have completed Phase I clinical trials but are not yet FDA-approved.
Over the last decade, medical science has shifted toward a new frontier of individualized medicine. Unfortunately, these highly specific new therapies—often designed for a single patient—frequently clash with the FDA’s outdated, one-size-fits-all regulatory frameworks.
Right to Try 2.0, which has already been passed in 18 states, modernizes federal law by establishing a clear legal pathway for patients with rare and ultra-rare diseases to access personalized medical breakthroughs tailored to their unique genetic profiles.
Elijah Stacy, who was diagnosed with a fatal and incurable disease at age 6, knows firsthand why passing Right to Try 2.0 is so critical. Elijah suffers from Duchenne muscular dystrophy, a rare genetic, progressive muscle-wasting disease which typically takes patients’ lives by the time they reach their mid-twenties. Elijah’s younger brother passed away from Duchenne when he was just 14.
“All Americans deserve medical freedom and the right to try cutting-edge, individualized treatments—my life literally depends on it,” Elijah said. “No politician should stop me from being able to take a drug today that could potentially totally change the course of my life.”
Respondents to the Goldwater Institute’s survey overwhelmingly agree that patients like Elijah should have the right to try cutting-edge treatments close to home, near their families and support systems. Updating the law to allow for individualized treatments is not lowering the bar for safety but recognizing that science has advanced in ways the FDA’s original approval system was not designed for, the respondents agree.
The bottom line: the American people demand federal reform to allow rare-disease patients to finally benefit from modern scientific advances. Political leaders who deliver for them will be rewarded.
The following are some additional findings from the survey.
