In another victory for rare-disease patients, New Hampshire Gov. Kelly Ayotte signed legislation this week expanding the state’s Right to Try Act to include access to investigational individualized treatments.
New Hampshire is the 16th state to enact legislation modeled in-part after Goldwater’s Right to Try for Individualized Treatments Act — a groundbreaking, nonpartisan law that empowers patients to seek cutting-edge, personalized treatments designed just for them.
Championed by state representatives Lisa Mazur, Erica Layon, Steven Kesselring, and Wayne MacDonald, HB 701 expands Goldwater’s original Right to Try law to include potentially lifesaving treatments designed specifically for individual patients. The bill also reforms civil liability and provides regulatory clarity so providers can offer treatment without fear of litigation or punishment.
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 Act 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medicine are made specifically for each patient based on their genetics and by definition cannot go through the FDA’s outdated regulatory processes in a timely manner. 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 New Hampshire are stepping in 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 six, the need for the law is clear.
“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.
What Comes Next?
This victory in New Hampshire 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 Act on patients and their families.
We extend our gratitude to Cornerstone Action; representatives Mazur, Layon, Kesselring, and MacDonald; Gov. Ayotte; former state representatives John Lewicke and Michael Yakubovich; Americans for Prosperity-New Hampshire; and all the dedicated advocates who championed this life-affirming reform in New Hampshire.
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.
