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Gene therapies have the potential to cure some diseases, but they're very expensive, and only specialized hospitals offer them. That means many patients in need are going untreated. NPR's Sydney Lupkin reports.
SYDNEY LUPKIN, BYLINE: Dustin Vidrine found out he has a rare eye disease when he was a teen. It's a genetic disease that people are born with called retinitis pigmentosa. His grandfather had it too. The 34-year-old from Lafayette, Louisiana, has already lost a lot of his peripheral vision to the illness, which has left him legally blind. Reading from a computer or tablet became nearly impossible.
DUSTIN VIDRINE: The bigger the screen, the harder it was to see because I'm only seeing one portion of detail at a time. I would have to read, like, one letter at a time.
LUPKIN: Having kids presented all new challenges, like reading the tiny markings on a baby bottle to measure out formula and water.
VIDRINE: There's just so much limitations. It's like, you know, throwing the ball with my son or whatever. And he'll say little things like, Daddy, I wish you had your eyes and stuff like that, you know? But I still do my best.
LUPKIN: Then a doctor told him about gene therapies. These are usually one-time treatments that break into cells and modify the disease-causing genes. Dr. Will Shrank is a former Harvard Medical School professor who's also worked for insurance companies like Humana and CVS Health.
WILL SHRANK: These therapies are incredible. 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.
LUPKIN: But they can cost a few million dollars for just one patient.
SHRANK: 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.
LUPKIN: Shrank recently founded a company called Aradigm to make gene therapy more widely available. As for Vidrine, there is already a gene therapy for retinitis pigmentosa on the market, 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 hourslong workup.
VIDRINE: 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.
LUPKIN: He said the doctors in Louisiana didn't really have those resources. For the first time in a long time, Vidrine had hope for preserving his vision.
VIDRINE: 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.
LUPKIN: 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. I asked if he considered moving to Texas.
VIDRINE: 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. Secondly, being a blind individual, like, that's a breach of vulnerability.
LUPKIN: Vidrine is on a United Health insurance plan for people with both Medicare and Medicaid coverage. After NPR reached out to ask about Vidrine's coverage, United Health 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.
Depending on the gene therapy, some states don't have any providers available, according to the American Society of Gene and Cell Therapy. And the exact number of patients who've received these therapies is hard to pin down because there's no central reporting system. But an NPR analysis of Medicaid data offers a snapshot that shows that where someone lives in the U.S. does seem to matter. State Medicaid programs in the South and Midwest paid for relatively few gene therapies with some exceptions like Texas, and states on the coast paid for more. I showed NPR's 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.
AMEET SARPATWARI: 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.
LUPKIN: He says now the challenge will be finding ways to address those access disparities. As for Dustin Vidrine in Louisiana, he says he's still waiting for his insurance to authorize that Texas appointment.
Sydney Lupkin, NPR News. Transcript provided by NPR, Copyright NPR.
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