Patients with rare diseases in Mississippi are one step closer to accessing innovative medical treatments designed just for them, thanks to unanimous support from Mississippi legislators who have voted to advance the Lifesaving Treatment Act out of the state Senate.
Championed by Sen. Josh Harkins, and modeled after the Goldwater Institute’s Right to Try for Individualized Treatments, Senate Bill 2858 empowers patients with rare and ultra-rare diseases to work with their physicians to seek innovative, highly specialized treatments that are as unique as they are.
With nearly 80% of rare diseases estimated to be genetic in nature, it is no surprise that access to innovative, individualized gene therapies is top of mind for many patients. Fortunately, new technologies and recent advances in modern medicine have made it possible for researchers to tailor treatments to an individual patient using his or her own genetics. If enacted into law, Senate Bill 2858 will protect the right of patients to pursue these customized treatments at the recommendation of their physicians and in concert with strong patient protections.
Mississippi legislators have a long history of leadership when it comes to standing up for patients. In 2015, Mississippi’s legislature approved Goldwater’s Right to Try reform, landmark legislation to protect the right of terminally ill patients to access investigational treatments advancing through the U.S. Food and Drug Administration’s approval process. SB 2858 builds on this successful history and demonstrates the commitment of Mississippi lawmakers to protecting and empowering patients with rare diseases.
Hannah Lowe is the mother of a young boy who is suffering from a rare and fatal form of childhood muscular dystrophy. She and her family know firsthand the anguish of having no hope—and the dire need for medical breakthroughs.
“Under the current regulatory framework, the process for getting new treatments into patients is long, and this is time that we do not have,” Hannah noted recently. “We are not asking for a handout. We are asking for laws…that would allow medical treatments to be developed faster with appropriate safety and efficacy measures, and under physician care and guidance for people for whom there are no treatments and no cures.”
“Americans are in the midst of a medical revolution where new treatments are discovered almost daily,” Goldwater Institute Senior Fellow Naomi Lopez said. “Some rare genetic diseases that used to be a death sentence are now treatable using the patient’s unique and personal genetic information. Senate Bill 2858 is a significant step to bringing these treatments to patients with rare diseases in Mississippi.”
Mississippi is not alone in pursuing this important reform. In 2022, Arizona became the first state to enact the Right to Try for Individualized Treatments, with Nevada following close behind in 2023. This session, California, Iowa, and Maryland have also introduced similar models, also with strong bipartisan support.
“We can’t let red tape stop children from getting lifesaving care,” said Mississippi Senator Josh Harkins. “Other countries are using individualized treatments to offer hope to patients with cancer and rare diseases. Two states have already passed this legislation: Arizona and Nevada. We owe it to the children and parents of Mississippi to get this done.”
The Goldwater Institute thanks Sen. Harkins, Sen. Hob Bryan, and Lt. Gov. Delbert Hosemann for their dedication to this reform, and commends the Mississippi Senate for standing up for the state’s most vulnerable patients.
Senate Bill 2858 awaits further action in the Mississippi House.
Heather Curry is the Director of Strategic Engagement at the Goldwater Institute, where she leads the Institute’s national legislative affairs strategy.
