New Hampshire patients with rare diseases are one step closer to accessing innovative medical treatments designed just for them. This week the New Hampshire House of Representatives unanimously advanced HB 701, sponsored by Representative Lisa Mazur—a nonpartisan bill that empowers patients with rare and ultra-rare diseases to work with their physicians to seek highly specialized treatments that are as unique as they are.
The legislation also reforms civil liability and provides regulatory clarity so providers can offer treatment without fear of litigation or punishment, as long as the provider has not engaged in any willful misconduct.
HB 701 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). 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medicine are made specifically for each patient, based on their genetics, and by definition cannot go through the FDA’s outdated regulatory processes in a timely manner.
Indeed, nearly 80% of rare diseases are estimated to be genetic in nature, yet fewer than 5% of rare diseases currently have an FDA-approved treatment, largely due to cost constraints and an antiquated regulatory regime.
As a result, people with rare life-threatening or severely debilitating illnesses face significant—often insurmountable—hurdles to get the care they need. HB 701 aims to rectify the regulatory hurdles faced by patients and incentivize more innovative companies to offer investigational treatments in the Granite State.
HB 701 is the natural next step in the Right to Try movement: it expands access to investigational individualized treatments tailored around an individual’s unique genetic makeup and provides civil liability protections so good-faith providers can get patients the innovative treatments they need and deserve.
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.
“When I was diagnosed, there were zero FDA-approved treatments available for me. Today, there’s more treatments coming online, becoming FDA approved, but even for me as an individual, OK, there’s not really any treatments that I can take as of right now,” Elijah says.
“If the patient wants it, if the company’s willing to give it, and most importantly, in my opinion, the physician agrees with it, it’s something that makes sense,” he adds. “And really the only reason I can’t do it is because of the current law that’s very frustrating. 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.”
New Hampshire is not alone in pursuing to expand its right to try laws to include individualized treatments. Eight states have already enacted this Right to Try expansion, and more legislatures are considering it in 2025.
The Goldwater Institute is proud to support HB 701 and looks forward to the New Hampshire Senate acting on this important legislation.
Read more about the Right to Try for Individualized Treatments here.
Brian Norman is the Director of State Affairs at the Goldwater Institute, where he assists in the development and implementation of Goldwater’s national legislative affairs strategy.
