Where patients live matters for access to gene therapy

Dustin Vidrine’s sight is disappearing — shrinking, really.

He has retinitis pigmentosa, a rare, inherited eye disease that runs in his family.

By the time he was in his 20s, he noticed that his vision had narrowed. It was especially apparent when he was looking at a computer or TV.

“The bigger the screen, the harder it was to see because I’m only seeing one portion of detail at a time,” says Dustin, 34, of Lafayette, La. “I would have to read like one letter at a time, whether I’m zooming in or not.”

Then Vidrine had kids. Fatherhood brought new challenges.

Mixing baby formula was tricky because he couldn’t see the tiny markings on the bottles to measure how much water to add. He wound up using a thick marker to make a bold line he could see.

“It’s just so much limitations,” he says. He wishes he could throw a ball with his son, but he’s now legally blind. “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.”

After a doctor suggested gene therapy might help, Vidrine was intrigued.

But getting access to a cutting-edge treatment that could preserve his vision has proven tricky. There are no options for the specialized care he needs in southern Louisiana, where he lives.

Promise of gene therapy can be hard to access  

Gene therapies are usually one-time treatments that break into cells and modify the disease-causing genes.

“These therapies are incredible,” says Dr. Will Shrank, a former Harvard Medical School professor who’s 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.”

But they can cost a few million dollars for just one patient.

“The fragmented way that we pay for and deliver care in this country is perfectly misaligned with equitably delivering those therapies to patients who can benefit,” says Shrank.

Shrank recently founded a company called Aradigm to help make gene therapy more widely available.

Vidrine hit a roadblock

As for Vidrine, there is already a gene therapy for retinitis pigmentosa on the market, called Luxturna, but it doesn’t work for the genetic mutation he has. So he’s hoping to get into a clinical trial for one of the other therapies being developed.

His retinal specialist in Louisiana told him he couldn’t help with that, but there were specialists in Texas who could. The first step would involve a complicated hours-long workup.

“That let me know that, hey, that’s something that can potentially change my life because I never had — it never took that long to do scans before,” he says, explaining that his doctors in Louisiana didn’t really have those resources.

For the first time in a long time, Vidrine had hope for preserving his vision.

“I made the appointment in Texas and everything like that, had my transportation set up only to find out that my insurance wouldn’t cover it because I’m not a resident of Texas,” he says.

He tried to figure out how to pay for the appointment himself, but right now he’s unemployed and can’t really afford it. And a GoFundMe fundraiser he tried never really took off.

NPR asked if he considered moving to Texas.

“A lot of people in my situation doesn’t just have a savings account where they can just pull out of it and just start moving or making a move,” says Dustin. Being blind, he says, makes him feel especially vulnerable about moving.

Vidrine has a UnitedHealthcare insurance plan for people with both Medicare and Medicaid coverage. After NPR reached out to ask about Vidrine’s coverage, UnitedHealthcare said residency isn’t a requirement to receive care in another state and that it does cover out-of-network providers in certain situations.

The company said it is working with Vidrine to help him get care.

The Centers for Medicare and Medicaid Services said in an emailed statement to NPR that it couldn’t comment on Vidrine’s specific case, but “generally, for people who are dually eligible and enrolled in a Medicare Advantage (MA) Dual Special Needs Plan (D-SNP), coverage can depend not just on medical necessity but on whether the specialist is in the plan’s network and service area. Meanwhile, Medicaid is state-based, and out-of-state non-emergency care can require prior authorization and the appropriate provider enrollment/arrangements.” There are additional steps and rules for participating in clinical trials.

The Louisiana Department of Health, which is responsible for the state’s Medicaid program, did not respond to requests for comment.

Gene-therapy deserts leave people without options

The exact number of patients who’ve received these kinds of treatments is hard to pin down because there’s no central reporting system, says Sarah Kikkert, a spokesperson for the American Society of Gene and Cell Therapy.

But an NPR analysis of Medicaid data offers a snapshot that shows location matters. The data is from 2024, the most recent full year available, and while it doesn’t include the total number of patients who benefited, it shows the number of different kinds of therapies each state program paid for that year.

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State Medicaid programs in Nebraska, North and South Dakota and Oklahoma paid for relatively few gene therapies, for example. Meanwhile, states including California, Indiana, Massachusetts and Texas paid for more.

NPR showed its analysis to Ameet Sarpatwari, a professor of population medicine at Harvard who studies drug policy. He says states that paid for more gene therapies tended to have more academic medical centers.

“Where you’re seeing deserts, so to speak, are areas in which it is likely the case that you don’t have these centers that have the sort of scientific know-how, have the capability of offering these therapies,” he says.

In theory, even if someone’s state doesn’t have a hospital that offers gene therapy, the state Medicaid program is supposed to pay for that person to get it in another state, Sarpatwari says. But NPR’s analysis shows that isn’t happening, he says, “and that’s troubling.”

Kikkert of the American Society of Gene and Cell Therapy agreed that where someone lives can be a major hurdle in getting access to gene therapy because not all hospitals provide them. “Depending on the therapy, some states have zero locations in their borders,” she says.

Forty-four states have at least one facility that offers gene therapy, Kikkert says. But more than half of all the facilities that her organization is aware of only deliver one type of gene therapy.

Now, the challenge will be finding ways to address those access disparities.

There are companies like Shrank’s Aradigm, which offer solutions for health plans. And there are models currently being tested by the Center for Medicare & Medicaid Innovation on how to make these treatments more accessible.

As for Vidrine, he says he’s still waiting for his insurance to authorize that Texas appointment.