WASHINGTON, D.C. — Patients desperately waiting to access cutting-edge, life-saving treatments have new hope today with the introduction of the Right to Try for Individualized Treatments Act—landmark legislation that establishes a legal pathway for patients to access gene-based therapies without having to beg the government for permission.
U.S. Representative Diana Harshbarger (R-TN) and Senator Ron Johnson (R-WI) introduced the groundbreaking legislation, also known as Right to Try 2.0. The bill builds on the success of the original Right to Try by modernizing federal law and establishing a clear legal pathway for patients with rare and ultra-rare diseases to access cutting-edge, personalized medical breakthroughs tailored to their unique genetic profiles.
Under the Goldwater Institute’s original Right to Try, which was enacted into federal law in 2018, terminally ill patients were able to successfully access investigational treatments that had completed Phase I clinical trials but were not yet FDA-approved. Since that time, 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 Unfortunately, these highly specific new therapies—often designed for a single patient—frequently clash with outdated, one-size-fits-all regulatory frameworks at the FDA. That leaves patients unable to access lifesaving therapies.
“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 Right to Try for Individualized Treatments opens the door to the latest advances in medical treatment and brings the federal government into the 21st century.”
The original Right to Try law has a proven track record of providing options to those who had none, and the Right to Try for Individualized Treatments builds on that legacy by protecting the next generation of medical breakthroughs. By empowering patients and their doctors to pursue individualized gene and cell therapies without unnecessary government interference, the act seeks to uphold the fundamental principle of medical autonomy and provide options for the most vulnerable families.
Incredibly, other countries’ regulatory systems are outpacing the United States, forcing patients with rare diseases to travel overseas to get the best medical treatments. That’s why, from coast to coast, Americans across party lines are demanding reform. Already, nearly 20 states have passed protections for accessing personalized medicine, and it’s time to make the Right to Try for Individualized Treatments the law of the land. The Riley family of Arizona knows firsthand why this reform is so critical.
“When our daughters were diagnosed with metachromatic leukodystrophy, we were 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,” said Kendra Riley, a mother and leading patient advocate. “No family should have to cross an ocean and raise astronomical sums of money just to save their child’s life when the technology to cure them exists right here at home. This legislation is the lifeline that rare disease families have been desperately praying for.”
While medical technology has evolved at a breathtaking pace, regulatory systems remain stuck in an era of mass-produced drugs. The Right to Try for Individualized Treatments Act 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. It is time to move past a one-size-fits-all regulatory model and put patients back in the driver’s seat of their own healthcare.
The introduction of the federal Right to Try for Individualized Treatment serves as an important step in the ongoing effort to align federal policy with the states, providing a clear benchmark for advocates working to highlight and dismantle the administrative barriers that currently prevent patients from accessing the next generation of healthcare.
