The meeting could determine whether the two siblings would keep going blind. The doctor knew that going in, but he was feeling good. He had negotiated huge discounts before, allowing patients to get complex surgeries or budget-busting drugs they otherwise couldn’t afford. And his last two conversations with Spark Therapeutics had been promising.
Then again, at $850,000 a person, Luxturna was more budget-busting than most.
Spark had proposed a few different ways of helping insurers to cover the gene therapy — but Dr. Kevin Strauss’s patients tend not to have insurance. As medical director of the Clinic for Special Children, in Strasburg, Pa., he mostly sees members of the Plain community: Old Order Amish and Mennonite families, who believe that caring for the sick is a community’s responsibility. Relying on governments or private companies to step in is considered a serious moral breach. They don’t want medicine for free. They just want to pay the reduced prices that government and private insurers often do.
That’s what Strauss was trying to negotiate for this family. The two Amish girls have an RPE65 gene mutation, causing the specific form of retinal blindness that Spark’s therapy treats. There is no way the parents would get Luxturna for only one of them, while letting the other’s vision fade. Asking the congregation for $850,000 is a stretch; asking for $1.7 million is unimaginable.
“It puts the family in a tough position,’’ Strauss said. “They know that that is communal money, money that wouldn’t be available for the child who gets in a farming accident or needs a life-saving chemotherapy.’’
Their dilemma echoes one facing the country as a whole. Spark has none of the sneering swagger that made Martin Shkreli everyone’s favorite pharma villain. But Luxturna has the highest price per dose of any medication so far. Spark argues that gene therapies take a dizzying amount of time and money to create, and that a single injection into each eye could potentially restore vision for the rest of a patient’s life. Executives say they are doing everything possible to get the treatment to anyone who needs it.
So, on March 15, three company representatives headed out to Strauss’s clinic in the cornfields. Earlier, two Spark employees had seemed excited to provide Luxturna for less money. Now, they were bringing along someone from the financial team.
“These other two guys had set me up to believe that they were going to go back and there was going to be all this enthusiasm,’’ Strauss recalled. “And the head of commercial was like, ‘No.’ ’’ Spark said it’s working to provide some kind of discount, but can’t make any promises.
Strauss also knows there are other kids in the Plain community with the same genetic mutation. Their families are hanging back, using these two siblings as a test case.
Anger over the egregious cost of medications is one of the only things shared across party lines. During the 2016 campaign, it got Hillary Clinton promising and Donald Trump crowing.
So it might come as a surprise that families affected by inherited retinal degeneration often speak positively about Spark.
Partially, that’s because Luxturna represents a daring investment. There are more than 260 genes in which a mutation can give rise to retinal problems. About 20 of them have been linked to the rare, severe degeneration that can cause childhood blindness. Luxturna can help correct the vision loss that arises from errors in a single gene.
This specificity is the reason it works. Researchers spent decades learning to cultivate the gene RPE65 in the lab and then packaging it just so, with a virus that acts as a Trojan horse. Inject the resulting liquid into the retina, and the virus will sneak the healthy gene inside the nuclei of cells, where it can restore some ability to translate light into a chemical language that’s legible to the brain.
Advocates for other retinal disorders hope for similar new treatments.
“If you set the price low, then nobody’s going to want to invest in advancing the field. If you’re never going to recoup what you put in, then nobody’s going to come to CRB1,’’ said Kristin Smedley, the mother of two boys with CRB1-associated retinal blindness and the president of the Curing Retinal Blindness Foundation, which has received funding from Spark.
But that argument has a flip side. “There are just a few of us who can say, ‘I don’t care if my insurance will pay for it, I will find a way,’’’ Smedley said. “It’s 90 percent of the people on these online communities who are saying, ‘How on earth are we going to pay for this?’ ’’
Spark hopes to answer that question. After all, it’s gunning for the exact same outcome as the parents. The company contracts directly with insurers, to avoid any potential markups a hospital might charge. Insurers can also potentially get up to 20 percent of the price back if the treatment does not work long term, and the company can help patients cover travel and copays.
For those on government insurance, Spark is negotiating coverage as well, and has proposed stretching out the payments over years and giving bigger money-back guarantees. But as John Furey, Spark’s chief operating officer, told STAT, there is no intention to provide the treatment at a lower price.
When the therapy hit the market in March, Spark managed to realize what many patients dream about. Families got treatment without having to deal with their insurance at all. “Spark Therapeutics basically . . . worked with our insurance to get it approved. We’re not privy to all that happened, but it happened,’’ said Shawn Hogan of Fair Haven, N.J., whose son Jack was the first to get the gene therapy commercially. But others with commercial insurance are still trying to secure the therapy.
Like many other groups descended from a small number of founding families, the Plain community has a high proportion of genetic disorders, and Strauss has seen a wide array since arriving at the Clinic for Special Children in 2001. He’s cared for kids with maple syrup urine disease and Ellis-van Creveld syndrome, spinal muscular atrophy, and glutaric acidemia. But he didn’t know there was any RPE65-associated retinal blindness until he met someone working for Spark. Company representatives had reached out to him before the drug was even approved, hoping he could help plan treatment for these girls.
When they met at the clinic in October, Strauss told the two Spark liaisons that patient privacy rules prevented him from reaching out to the family. But once the father sent him an e-mail, the doctor quickly went from stranger to advocate.
It was a role Strauss had played before. His patients are among the only religious groups eligible to file IRS Form 4029, which exempts them from paying Social Security and Medicare taxes and prevents them from getting private or public insurance. Their kids are still eligible for Medicaid, but it remains taboo.
Families negotiate discounts for paying the whole cost up front. But these rebates are relatively small. Strauss thought he could do better.
“The Amish and Mennonite want to pay something, they just don’t feel it’s fair to be paying more than what Medicaid is paying, and I agree with them,’’ he said.
At some hospitals, he got his patients enrolled in preexisting charitable programs; at others he negotiated new agreements. The results are discounts of 70 to 90 percent, he said. Strauss has done similar work with drug companies. Spinraza, Biogen’s spinal muscular atrophy drug, normally costs $750,000 the first year, and then $375,000 annually. Now, 16 patients at the Clinic for Special Children get it for free.
Soon after Luxturna’s price announcement, Strauss met again with representatives of Spark. They talked about the implications of Form 4029. Strauss felt patients in the Plain community should get the drug for nothing, or at a 90 percent discount. The others, he said, seemed enthusiastic. But when they came back in mid-March they proposed enrolling the two kids in Medicaid. Strauss explained that violated their religious principles, and put the family in a near-impossible bind: choosing between an action your community sees as a moral failure, or continued deterioration of kids’ eyesight. Talks with Spark are ongoing.
Eric Boodman can be reached at eric.boodman@statnews.com.
