Right to Try for Individualized Treatments Act Introduced in Congress to Expand Access to Gene Therapies for Rare Disease Patients
Key Insights
The Right to Try for Individualized Treatments Act (Right to Try 2.0) was introduced in Congress by Rep. Diana Harshbarger (R-TN) and Sen. Ron Johnson (R-WI) to create a legal pathway for patients to access personalized gene-based therapies.
The legislation builds on the original 2018 federal Right to Try law, which allowed terminally ill patients to access post-Phase I investigational treatments not yet FDA-approved.
Nearly 20 states have already passed similar protections, but current FDA regulatory frameworks are described as outdated and unable to accommodate highly individualized treatments designed for single patients.
U.S. Representative Diana Harshbarger (R-TN) and Senator Ron Johnson (R-WI) introduced the Right to Try for Individualized Treatments Act in Congress this week—landmark legislation designed to establish a clear legal pathway for patients with rare and ultra-rare diseases to access cutting-edge, personalized gene-based therapies without requiring government permission.
The bill, also known as Right to Try 2.0, builds on the Goldwater Institute (search)'s original Right to Try legislation, which was signed into federal law in 2018. That law allowed terminally ill patients to access investigational treatments that had completed Phase I clinical trials but were not yet FDA-approved. However, the new legislation addresses a critical gap: today's frontier of individualized medicine—including therapies designed for a single patient—cannot fit through the FDA's traditional, one-size-fits-all regulatory frameworks in a timely manner.
"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 (search). "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."
A Growing State-Level Movement
Already, nearly 20 states have passed Right to Try 2.0 protections, allowing patients to access personalized medicine at the state level. The introduction of the federal legislation represents an effort to align national policy with these state-level reforms, providing a uniform benchmark for patients across the country.
The Goldwater Institute (search) noted that other countries' regulatory systems are outpacing the United States, forcing patients with rare diseases to travel overseas for treatment. The Riley family of Arizona exemplifies this burden.
"When our daughters were diagnosed with metachromatic leukodystrophy (search), 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."
Modernizing Outdated Regulatory Frameworks
The legislation addresses a fundamental mismatch between the pace of medical innovation and the structure of federal regulation. While medical technology has advanced rapidly toward individualized treatments—gene and cell therapies tailored to a patient's unique genetic profile—the regulatory system remains designed for mass-produced drugs. Under current frameworks, highly specific therapies that by their nature cannot go through traditional FDA approval pathways leave patients without viable options.
The Right to Try for Individualized Treatments Act seeks to remove these bureaucratic barriers by empowering patients and their doctors to pursue individualized gene and cell therapies without unnecessary government interference. The act upholds the principle of medical autonomy and aims to provide options for the most vulnerable families.
The original Right to Try law has a demonstrated track record of providing options to patients who previously had none. Right to Try 2.0 extends that legacy by protecting the next generation of medical breakthroughs, ensuring that laws keep pace with modern innovation and that patients remain in the driver's seat of their own healthcare decisions.