In a life-changing victory for patients with rare and ultra-rare diseases, South Dakota Gov. Larry Rhoden this week signed the Goldwater Institute’s Right to Try Individualized Treatments Act. The nonpartisan reform offers hope to those who need it most, allowing suffering patients to work directly with their physician to safely seek the most groundbreaking of investigational personalized care options.
In enacting this innovative reform, South Dakota joins a growing national movement to empower Americans facing rare and ultra-rare diseases to work with their physicians to seek highly specialized treatments that are as unique as they are. South Dakota is now the ninth state since 2022 to champion this crucial expansion, joining Louisiana, Maryland, Mississippi, Arizona, Nevada, North Carolina, Arkansas, and Tennessee.
The Urgent Need: 95% of Rare Diseases Lack FDA-Approved Treatments
This new law marks a profound shift toward personalized medicine and a much-needed acknowledgment of the limitations within the traditional 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 (FDA)—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medicine are made specifically for each patient, based on their genetics, and by definition cannot go through the FDA’s outdated regulatory processes in a timely manner.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, there’s an urgent need for reform.
South Dakota Joins Growing National Movement
The federal government’s slow pace in adapting its regulatory framework has left many patients with limited options. States like South Dakota are stepping up to fill this void, ensuring patients are not denied access to potentially life-saving treatments due to bureaucratic obstacles.
For 23-year-old Elijah Stacy, who has suffered from a rare genetic, progressive muscle-wasting disease called Duchenne muscular dystrophy since he was six, 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 are forced to take when confronted with limited options.
Personalized Treatments, Not Bureaucratic Barriers
This victory in South Dakota represents a significant step toward a future where individuals with rare diseases can access the most innovative and promising treatments without begging the federal government for permission.
“As a co-founder of a rare diseases company, the unanimous passage of this bill warms my heart. It was an honor to sponsor a bill that expands Right to Try for individuals with rare and ultra-rare diseases in South Dakota,” said Rep. Bobbi Andera, who sponsored the legislation in the House. “This smart legislation uses evidence-based practices to address the needs of our most vulnerable populations. I would like to thank the Goldwater Institute for their dedication and teamwork, as it was an absolute pleasure working on this legislation.”
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. We extend our heartfelt gratitude to Rep. Andera, Governor Larry Rhoden, and all the dedicated advocates in South Dakota who championed this life-affirming legislation.
Taylor Walker is the State Affairs Associate at the Goldwater Institute, where she works integrally within the legislative processes of numerous states and at the federal level, engaging and educating policymakers to advance free-market solutions nationwide.
