In a major win for rare-disease patients in Oklahoma, Gov. Kevin Stitt has signed the Goldwater Institute’s model Right to Try for Individualized Treatments Act, a cutting-edge law that empowers patients to seek personalized treatments designed just for them. Sponsored by state Sen. Brenda Stanley and state Reps. Marilyn Stark and Mark Lepak, Senate Bill 933 expands Goldwater’s original Right to Try law to include individualized, custom treatments that could potentially save patients diagnosed with life-altering diseases.
Oklahoma is the 18th state to allow individuals facing rare and ultra-rare diseases to work with their physicians to seek highly specialized treatments that are as unique as they are. The Sooner State joins Arizona, Arkansas, Colorado, Georgia, Iowa, Indiana, Kansas, Louisiana, Maryland, Mississippi, Nevada, New Hampshire, North Carolina, South Dakota, Tennessee, Texas and West Virginia in championing this crucial legislation.
Why Do We Need It?
This new law marks a profound shift toward personalized medicine and a much-needed acknowledgment of the limitations within the traditional federal regulatory framework. The Right to Try for Individualized Treatments builds upon the success of the original Right to Try, a reform created by the Goldwater Institute and enacted in 41 states before it was signed into federal law in 2018. The original Right to Try allows terminally ill patients to access investigational treatments that have completed Phase I clinical trials but are not yet approved by the Food and Drug Administration—and it is saving lives around the country.
But today, the latest innovations in medicine are made specifically for each patient based on their genetics, and by definition cannot go through the FDA’s outdated regulatory processes in a timely manner. After all, the FDA’s “one-size-fits-all” clinical trial system is designed for large patient populations and ill-suited for the complexities of rare diseases. With 95% of rare diseases lacking FDA-approved treatments, the need for reform is urgent.
The federal government’s slow pace in adapting its regulatory framework has left many patients with limited options. States like Oklahoma are stepping up to fill this void, ensuring patients are not denied access to potentially life-saving treatments due to bureaucratic obstacles. As Sen. Stanley stated, “This bill broadens access to innovative treatments, offering hope to terminally ill patients who have exhausted all approved options and cannot participate in clinical trials.”
Who Will This Law Help?
For 23-year-old Elijah Stacy, who has suffered from a rare genetic, progressive muscle-wasting disease called Duchenne muscular dystrophy since he was 6, the need for the law is as clear as can be.
“Somebody that doesn’t even know me has written in a law which is stopping me from being able to take a drug today that could potentially totally change the course of my life,” Elijah says, underscoring the frustration and desperation faced by countless individuals battling rare diseases.
Similarly, the stories of families like the Rileys of Arizona, who were forced to seek treatment abroad when their infant was diagnosed with a rare genetic brain disease, highlight the desperate measures families must take when faced with limited options.
What Comes Next?
This victory represents a leap forward for individuals with rare diseases, reducing barriers to the most innovative and promising treatments without needing permission from the federal government. In the words of Rep. Stark, “For patients facing serious diagnoses, this creates an opportunity to explore those options closer to home and alongside their doctor.”
The Goldwater Institute will continue to urge lawmakers across the nation to adopt these necessary reforms, recognizing the profound impact of the Right to Try for Individualized Treatments on patients and their families. The Institute extends our thanks to Gov. Stitt and the members of the Oklahoma legislature for their resounding support of SB 933.
You can read more about the Right to Try for Individualized Treatments here.
Taylor Walker is the State Affairs Associate at the Goldwater Institute.
