Patients with rare and ultra-rare diseases could soon access personalized gene therapies without waiting for FDA approval under new federal legislation introduced June 8, 2026. U.S. Representative Diana Harshbarger (R-TN) and Senator Ron Johnson (R-WI) introduced the Right to Try for Individualized Treatments Act—also called Right to Try 2.0—which builds on a 2018 federal law that allowed terminally ill patients to access investigational drugs. The new bill aims to create a legal pathway for individualized gene and cell therapies that are tailored to a single patient's genetic profile, treatments that often clash with what the Goldwater Institute calls "outdated, one-size-fits-all regulatory frameworks."
Nearly 20 states have already passed protections for accessing personalized medicine, according to the Goldwater Institute, which championed the original Right to Try law. That 2018 federal law allowed terminally ill patients to access investigational treatments that had completed Phase I clinical trials but weren't yet FDA-approved. The new legislation targets a different category: highly specific therapies designed for individual patients based on their unique genetic makeup, treatments that medical science has shifted toward since 2018.
"No American should be forced to beg the government for permission to try to save their own life, and no bureaucrat should prevent a patient from accessing cutting-edge therapies," said Victor Riches, President and CEO of the Goldwater Institute. The institute says other countries' regulatory systems are now outpacing the United States, forcing American patients with rare diseases to travel overseas for treatments. Kendra Riley, an Arizona mother whose daughters were diagnosed with metachromatic leukodystrophy, told the institute her family "was forced to raise half a million dollars and temporarily relocate our entire family to Italy just to access a gene therapy that could save our youngest daughter's life."
The bill responds to what its supporters describe as a mismatch between rapid advances in personalized medicine and regulatory systems built for mass-produced drugs. According to the Goldwater Institute, the legislation will "ensure that our laws keep pace with modern innovation, removing the bureaucratic barriers that stand between patients with rare diseases and the personalized care that could save their lives." The institute frames the act as empowering patients and doctors to pursue individualized treatments "without unnecessary government interference" while upholding medical autonomy. By establishing what it calls "a clear legal pathway" at the federal level, the bill aims to align federal policy with the nearly 20 states that have already moved to protect access to personalized therapies.
The Right to Try for Individualized Treatments Act serves as "an important step in the ongoing effort to align federal policy with the states," the Goldwater Institute says, and provides "a clear benchmark for advocates working to highlight and dismantle the administrative barriers that currently prevent patients from accessing the next generation of healthcare." For families like the Rileys—who had to cross an ocean and raise extraordinary sums just to access existing gene therapy technology—the legislation represents what Kendra Riley calls "the lifeline that rare disease families have been desperately praying for." The bill puts patients "back in the driver's seat of their own healthcare," the institute concludes, in an era when life-saving treatments exist but remain out of reach because of regulatory hurdles.