A young Navy pilot’s life was hanging in the balance. But then, his future changed for the better, all because of a federal law that turns five years old today. That law is Right to Try, and it’s a story every American needs to hear.
Naval aviator Matt Bellina had already been grounded due to loss of coordination by the time he received his preliminary diagnosis of amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, in 2013. He was 30 years old when doctors confirmed the diagnosis the following year. There was no U.S. Food and Drug Administration-approved treatment that had the potential to stall or cure this disease. There were some treatments undergoing clinical evaluation that might be able to help, but they were still years away from full approval.
Stories like Matt’s are precisely why the Goldwater Institute created the Right to Try. This landmark law, which Goldwater first passed in 41 states and then at the federal level in 2018, makes it legal for patients to seek out innovative, not-yet-approved treatments when under a doctor’s care and with safeguards for patient consent. It puts patients first, removing the bureaucratic obstacles that can often subjugate the doctor’s expertise and a patient’s medical autonomy.
So it’s fitting that Matt was one of the first beneficiaries of the federal law that carries his and a handful of other patient advocates’ names. In 2019, he began receiving an investigational treatment under the law, and within weeks, he experienced improvements in his breathing and physical strength. Matt, a husband and father of young sons, has now significantly outlived his expected prognosis of just two to five years.
“I became a Right to Try advocate because my experience taught me that the existing access programs were broken and would never help people like me,” Matt said. “But under the Right to Try law, I was able to access a groundbreaking treatment that likely added years to my life.”
Patients like Matt don’t have time to wait months, years, or even decades for the FDA to approve a treatment that could help them. While most of us mark the passing years by milestones—birthdays, New Year’s celebrations, the starts and ends of school years—some facing debilitating disease mark time by mobility, or the ability to verbalize, or the capacity to breathe on one’s own. Goldwater created the Right to Try so that these patients, who are out of options and desperate for hope, might have the opportunity to pursue investigational, yet promising, treatment pathways without first begging the federal government for permission.
Today, Matt and his wife, Caitlin, continue to advocate for treatment pathways and raise money for medical breakthroughs. And they will continue to do so until every patient can access the treatment they need. Most important, Matt’s three active and growing boys are making memories with their father, experiencing things like baseball, scouts, and the neighborhood kids’ shenanigans.
It’s not just Matt. Right to Try is saving countless lives around the nation today, with pharmaceutical companies using the federal law to treat entire groups of patients in promising new ways. But more treatment pathways are needed, particularly for the 3o million Americans, half of them children, who are facing rare- and ultra-rare disease diagnoses. For many, there is no treatment on the horizon, there aren’t enough patients for a clinical trial, and, even if there were, there aren’t strong prospects for commercialized treatments down the road. The promise of 21st-century medicine is here with cutting-edge treatments like gene therapy, but our decades-old regulatory system is leaving too many of these patients behind.
Goldwater’s Right to Try for Individualized Treatments, or Right to Try 2.0, provides this needed pathway. This reform does not alter the functioning or future success of the original Right to Try law. Instead, it establishes a new, secure, and physician-directed pathway for patients with rare and ultra-diseases who lack treatment options in clinical trials and whose doctors are recommending a personalized treatment. Goldwater enacted this law in Arizona last year, and it’s now under consideration in a growing number of states across the country.
In late March, the FDA announced that it would hold an Advisory Committee meeting to consider the same treatment Matt first received in 2019. A date has not yet been announced. While there is no cure for ALS yet, it is important to remember that an estimated 100,000 ALS individuals have passed away since Matt’s first treatment under the Right to Try law.
Critically ill Americans simply don’t have the time to wait on the FDA. Right to Try and its natural next step, Right to Try 2.0, offer a better way to get the right treatment, to the right patient, at the right time.
Naomi Lopez is the Vice President of Healthcare Policy at the Goldwater Institute.
