In a life-affirming victory for rare disease patients, West Virginia became the seventeenth state to enact the Goldwater Institute’s Right to Try for Individualized Treatments—a groundbreaking, nonpartisan new law that empowers patients to seek cutting-edge personalized treatments designed just for them. Championed by Delegate Mike Hornby and signed into law by Gov. Patrick Morrisey, HB 4610 expands Goldwater’s original Right to Try law to potentially lifesaving treatments that are designed specifically for individual patients.
In enacting this innovative reform, West Virginia 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. The Mountain State joins Texas, Colorado, Iowa, Louisiana, Maryland, Mississippi, Arizona, New Hampshire, Nevada, North Carolina, Arkansas, Tennessee, South Dakota, Kansas, Indiana, and Georgia 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medicine are made specifically for each patient, based on their genetics, and by definition cannot go through the FDA’s outdated regulatory processes in a timely manner.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, there’s an urgent need for reform.
The federal government’s slow pace in adapting its regulatory framework has left many patients with limited options. States like West Virginia are stepping up to fill this void, ensuring patients are not denied access to potentially life-saving treatments due to bureaucratic obstacles.
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 in West Virginia 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.
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 its heartfelt gratitude to coalition partners Americans for Prosperity – WV and the Cardinal Institute whose efforts made this victory possible, as well as Delegate Hornby, Gov. Morrisey and the members of the West Virginia legislature for their unanimous support of HB 4610.
You can read more about the Right to Try for Individualized Treatments here.
Brian Norman is the Director of State Affairs at the Goldwater Institute.
