Access to Gene Therapy Hinges on Location, Leaving Some Patients Behind
Dustin Vidrine’s vision is fading due to retinitis pigmentosa, a rare inherited eye disease. While gene therapy offers a potential path to preserving his sight, accessing this cutting-edge treatment has proven challenging due to geographic limitations and insurance complexities.
A Father’s Struggle with Vision Loss
Dustin Vidrine, 34, of Lafayette, Louisiana, began noticing vision narrowing in his 20s. The difficulty was particularly pronounced when viewing screens. “The bigger the screen, the harder it was to witness due to the fact that I’m only seeing one portion of detail at a time,” Vidrine explained. Fatherhood added new hurdles, such as accurately measuring baby formula due to his impaired vision, requiring him to use a marker to create visible markings on bottles. Now legally blind, Vidrine expresses a simple wish: “He’ll say little things like, Dad, ‘I wish you had your eyes’ and stuff like that, you know? But I still do my best.”
The Promise and Cost of Gene Therapy
Gene therapies, which modify disease-causing genes within cells, represent a significant advancement in medicine. “These therapies are incredible,” says Dr. Will Shrank, a former Harvard Medical School professor who has also worked for insurance companies such as Humana and CVS Health. “They can absolutely massively impact the lives of patients with terrible conditions that, when I was in medical school, none of us could imagine were curable or addressable.” However, these treatments can be extraordinarily expensive, costing several million dollars per patient.
Roadblocks to Treatment
Vidrine learned of a gene therapy, Luxturna, for retinitis pigmentosa, but it is not effective for his specific genetic mutation. He hoped to participate in a clinical trial for an alternative therapy, but faced obstacles. His retinal specialist in Louisiana couldn’t assist with finding a trial, suggesting specialists in Texas might be able to aid. However, his insurance initially denied coverage because he was not a Texas resident.
Vidrine, currently unemployed, struggled to afford the appointment out-of-pocket, and a GoFundMe campaign did not generate sufficient funds.
After NPR inquired about Vidrine’s coverage, UnitedHealthcare stated that residency is not a requirement for care in another state and that out-of-network providers are covered in certain situations. The company is now working with Vidrine to facilitate his care.
Gene Therapy “Deserts” and Access Disparities
Access to gene therapy is not uniform across the United States. An NPR analysis of 2024 Medicaid data reveals significant disparities in access, with states like California, Indiana, Massachusetts, and Texas providing more gene therapies than states like Nebraska, North and South Dakota, and Oklahoma. This disparity often correlates with the presence of academic medical centers equipped to offer these specialized treatments.
Ameet Sarpatwari, a professor of population medicine at Harvard who studies drug policy, noted that states with fewer academic medical centers tend to have limited access to gene therapy. While state Medicaid programs are theoretically obligated to cover out-of-state care when a treatment isn’t available locally, Sarpatwari’s analysis suggests this isn’t consistently happening, which he calls “troubling.”
Sarah Kikkert, a spokesperson for the American Society of Gene and Cell Therapy, confirms that location is a major barrier to access, as not all hospitals offer gene therapy. Forty-four states have at least one facility offering gene therapy, but over half of those facilities only deliver one type of gene therapy.
Looking Ahead
Efforts are underway to address these access disparities, including solutions offered by companies like Aradigm and models being tested by the Center for Medicare & Medicaid Innovation. As for Vidrine, he is awaiting insurance authorization for his appointment in Texas.