Georgians with rare diseases are one step closer to accessing innovative medical treatments designed just for them. Today the Georgia Senate unanimously advanced the Hope for Georgia Patients, sponsored by Senator Matt Brass – a nonpartisan law 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.
Senate Bill 72 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medicine are made specifically for each patient, based on their genetics, and by definition cannot go through the FDA’s outdated regulatory processes in a timely manner. As a result, people with rare life-threatening or severely debilitating illnesses face significant – often insurmountable – hurdles to get the care they need.
SB 72 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 expands patient eligibility beyond terminal illnesses to include patients with life-threatening or severely debilitating conditions.
The legislation provides for multiple layers of patient protections, such as thorough informed consent requirements and review by a federally approved facility’s Institutional Review Board to ensure the treatment is safe and ethical.
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.”
Georgia is not alone in pursuing this important reform. Six states have already enacted this Right to Try expansion, and more legislatures are considering it in 2025.
With nearly 80% of rare diseases estimated to be genetic in nature, policymakers should prioritize empowering rare disease patients to access innovative, individualized gene therapies. Sadly, fewer than 5% of rare diseases currently have an FDA-approved treatment, largely due to cost constraints and an antiquated regulatory regime.
“Whether it’s a mom with breast cancer or a child suffering from a rare disease, everyone’s journey is different. Thanks to advances in medical technology, we can now help each patient where they are, using treatments that are more effective, have fewer side effects, and are more likely to help people recover and lead a full life,” says Senator Matt Brass, who sponsored SB 72 in the Georgia Senate. “I am proud to sponsor the Hope for Georgia Patients Act and want to thank Lt. Governor Burt Jones and my colleagues in the Georgia senate for their support. Working together, passing this legislation, we can give hope and effective healthcare to the people of Georgia.”
The Goldwater Institute is proud to support SB 72 and looks forward to the House of Representatives 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.
