Texans with rare diseases are one step closer to accessing cutting-edge treatments after legislators voted unanimously today to advance House Bill 4059 out of the Texas state House. Modeled after the Goldwater Institute’s Right to Try for Individualized Treatments, HB 4059 will empower 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.
Why does this matter?
About 30 million Americans suffer from rare diseases for which there are no treatments or cures, and half of those are children. Texas—the second-most-populous state in the nation—is home to countless people who are searching for hope. In 2015, Texas legislators approved Goldwater’s Right to Try reform, landmark legislation that protects the right of terminally ill patients to access investigational treatments advancing through the U.S. Food and Drug Administration’s approval process. While the original Right to Try is already working for many patients in Texas, there is still work to be done. For Americans with rare or ultra-rare diseases, where treatments are increasingly designed for an individual patient using his or her own genetics, the existing pathway is not broad enough. House Bill 4059 will extend the benefits of Right to Try by creating a pathway that allows patients with rare diseases to access treatments on the cutting edge of medicine.
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. That’s why she is advocating for the Right to Try for Individualized Treatments.
“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 told the Texas Legislature. “We are not asking for a handout. We are asking for laws like HB 4059 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.”
Championed by Representative Ken King, HB 4059 has garnered bipartisan support in the Texas House, as well as the support of earlier advocates of Right to Try, including Representative Kyle Kacal, who carried the 2015 House version. Additionally, companion legislation has been introduced by Senator Paul Bettencourt, sponsor of the 2015 Senate Right to Try bill.
“Americans are in the midst of a medical revolution where new treatments are discovered almost daily,” Goldwater Institute Vice President for Healthcare Policy 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 4059 is a significant step to bringing these treatments to Texans with rare diseases.”
Texas is not alone in pursuing this important reform. Arizona became the first state to enact the Right to Try for Individualized Treatments last year, Nevada and Iowa have both introduced measures to adopt this Right to Try expansion in 2023, and more states are expected to follow suit.
The Goldwater Institute commends the Texas House of Representatives for advancing House Bill 4059, and applauds the Texas Legislature for considering this important reform.
Heather Curry is the Director of Strategic Engagement at the Goldwater Institute.
