Thanks to decisive action by state policymakers, patients with rare diseases in Maryland are one step closer to accessing cutting-edge medical treatments designed just for them. In a bipartisan show of support, Maryland legislators voted unanimously this week to advance House Bill 676 out of the House of Delegates and on to the state Senate.
Championed by Delegate Matt Morgan, and modeled after the Goldwater Institute’s Right to Try for Individualized Treatments, HB 676 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, HB 676 will protect the right of patients to pursue these customized treatments at the recommendation of their physicians and in concert with strong patient protections.
Maryland native 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.
On February 29, Rare Disease Day, Hannah provided testimony on the need for the reform in Maryland. “Under the current regulatory framework, the process for getting new treatments into patients is long, and this is time that we do not have,” she noted. “We are not asking for a handout. We are asking for laws like HB 676 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. House Bill 676 is a significant step to bringing these treatments to patients with rare diseases in Maryland.”
Maryland legislators have a long history of leadership on this important issue. In 2017, Maryland’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. HB 676 builds on this successful history and demonstrates the commitment of Maryland lawmakers to protecting and empowering patients with rare diseases.
Maryland 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 Mississippi have also introduced similar models.
Commenting on the reform, Delegate Morgan said, “I’m hopeful that the Senate will pass HB676: doing so will help citizens of Maryland who suffer from a rare disease and give them hope for a future.”
The Goldwater Institute thanks Delegate Morgan and his numerous bipartisan co-sponsors for their commitment to this critical reform and applauds the Maryland House of Delegates for its strong bipartisan support of House Bill 676.
House Bill 676 now proceeds to the Maryland Senate.
Heather Curry is the Director of Strategic Engagement at the Goldwater Institute, where she leads the Institute’s national legislative affairs strategy.
