Struggle for coverage of rare conditions
An insurer rejected out-of-state treatment for a 4-year-old’s rare disease five times. It relented, but why is getting an OK so hard?
Getting approval from health insurance companies for out-ofstate treatment for rare diseases can be difficult.
PAYSON, Ill. – Jaxtien Miller doesn’t seem very sick — yet.
On a recent Monday night, the 4-year-old bounced through his home in rural Payson, piecing together a giant firetruck puzzle on the kitchen floor. Then he sprang onto the couch, rolling around with his 11-year-old brother, giggling.
He then plopped all 49 pounds of his small, solid body onto his mother’s lap for a snuggle.
But Jax’s hands have started to shake, and he can no longer climb stairs on his own.
Those symptoms are just the beginning.
Jax has a rare disease called metachromatic leukodystrophy that could rob him of the ability to walk, sit and hold up his own head within the next year. Children with the disease typically die between the ages of 2 and 10.
There’s no cure for the genetic disorder. There is a treatment that may slow the disease’s progression but it cannot reverse damage that is already done nor restore abilities that are already lost, meaning time is of the essence.
Yet for most of the past two months, as his symptoms worsened, Jax and his family waited.
They waited for their Illinois Medicaid managed care insurance company, MeridianHealth, to approve treatment at UPMC Children’s Hospital of Pittsburgh.
The insurer sent the family letters denying requests for coverage five times. The insurer said the hospital was out-of-network, and Jax should get care closer to home. Those familiar with the disease, however, say the Pittsburgh hospital is one of only three centers in the country that specialize in treating children with Jax’s condition.
The insurance company relented earlier this month, agreeing to cover the treatment in Pittsburgh — after the Tribune told MeridianHealth it was writing a story about Jax’s case, and a nonprofit foundation and a state lawmaker got involved.
Still, Jax’s parents worry about the time that was lost, waiting for the OK to get on a plane to Pittsburgh. “Every second that nothing is being done is more damage,” said his mother, Mercedes Boden, 29.
‘A common situation’
No one keeps data on how many patients’ requests for outof-state care are denied, but experts say it’s not uncommon.
Typically, health insurance companies ask patients to get care at in-network hospitals — nearby facilities with which insurers have contracted rates. And that works fine for common conditions such as knee replacements, broken bones and many forms of cancer.
But when a patient has a rarer condition that in-network hospitals may not be familiar with and wants to go out-of-state for treatment by experts, insurance companies may balk.
Between 25 and 30 million Americans have rare diseases, some of whom can require outof-state care. Patients with all types of health insurance, including private insurance through their employers, can have a tough time getting their insurers to approve out-of-state care.
“It’s a very common situation when you have experts that are not within your state and those are the only ones who can treat your condition,” said Melanie Buzzelli, associate director of federal policy at the National Organization for Rare Disorders, a patient advocacy group.
Some experts say securing outof-state coverage can be especially difficult for patients on Medicaid, a state and federally funded health insurance program that serves many low-income people.
Jax’s insurer, MeridianHealth, is a Medicaid managed care organization, meaning it administers Medicaid benefits for the state. MeridianHealth is part of for-profit company WellCare Health Plans. Nearly 2.9 million people in Illinois have Medicaid, and about 2.2 million of them are part of Medicaid managed care organizations.
MeridianHealth said in a statement that privacy laws prevent it from discussing specific patients. But it said its “number-one priority is the health and well-being of our members.”
Metachromatic leukodystrophy is the result of a genetic mutation, and leads to a breakdown of the protective covering around nerve cells and nerve
fibers, known as myelin. Without that protective covering, the nerve cells and fibers become damaged.
Jax’s family wants him to get a stem cell transplant with umbilical cord blood — a procedure that doctors have found can slow the disease’s progression. Before the transplant, patients also typically undergo chemotherapy to clear out the affected bone marrow cells.
In some of the denial letters, MeridianHealth questioned the medical necessity of the evaluation and transplant. In all the denials, it said that Jax’s family and doctors had not shown it was necessary for him to be seen by an out-of-network provider when he could go to closer hospitals. The family, and a nonprofit patient advocacy organization, appealed those decisions.
In a later denial letter, which came after an external review by a physician who is board-certified in blood and bone marrow transplants, MeridianHealth said it wouldn’t cover the procedure in Pittsburgh but Lurie Children’s Hospital in Chicago could perform the transplant.
But Jax’s family, and nonprofit organizations that support patients with the disease, argued that Lurie doesn’t have nearly as much experience treating patients with metachromatic leukodystropy as the hospital in Pittsburgh.
Lurie, one of the top children’s hospitals in the country, treats metachromatic leukodystophy and has performed two transplants on patients with the disease.
Lurie has never seen Jax and can’t comment on him, the hospital said in a statement. “However, we also respect that parents and caregivers have a very important role in the choice of where their child receives care,” Lurie said.
The Pittsburgh children’s hospital has seen more than 170 patients with metachromatic leukodystrophy, said Dr. Maria Escolar, director of the Pittsburgh hospital’s Program for the Study of Neurodevelopment in Rare Disorders. Twelve of the patients she’s overseen since 2011 received transplants in Pittsburgh.
The Pittsburgh hospital began using a less toxic form of chemotherapy on patients with metachromatic leukodystrophy and similar diseases in 2011, and since then the survival rate of those patients has been 95%, Escolar said.
Some patients that Pittsburgh treated in 2011 are still alive, walking and talking today, Escolar said.
Dean Suhr, president and co-founder of the MLD Foundation, which supports patients with the disease, said he tries to send families to the three places that specialize in its treatment, which in addition to Pittsburgh include Duke University Medical Center and University of Minnesota Medical Center. The foundation has worked to help Jax and his family in recent months.
“The people that have seen a lot of MLD patients know what to look for, and how to be proactive in their responses as they’re managing these patients,” Suhr said. Two of Suhr’s three daughters have been affected by the disease. His daughter Darcee died at age 10 in 1995, shortly after receiving a bone marrow transplant, and his daughter Lindy is 39, nonverbal and in a wheelchair.
Maria Kefalas, co-founder of Cure MLD, another group that has worked with Jax’s family in recent months, agreed with Suhr’s assessment. Kafalas’ daughter Calliope also has the disease, and at age 9, she has lived longer than the doctors predicted. But she’s nonverbal, eats through a feeding tube and needs 96 hours a week of skilled nursing care, Kefalas said. She wasn’t a candidate for a transplant like Jax.
She called Jax’s case of denial after denial by a Medicaid managed care company one of the worst she’s seen. “If you’re poor, if you’re Medicaid-dependent, your child’s life expectancy should not be dictated by whether or not they have Medicaid,” she said.
Finding a balance
Federal regulations say Medicaid must pay for outof-state care under certain conditions, such as in a medical emergency or if the services are “more readily available” in another state.
But part of the challenge for Medicaid programs is that they work with limited public dollars, said Matt Salo, executive director of the National Association of Medicaid Directors. They have to find a balance between providing the best care possible for patients and spending money responsibly.
Umbilical cord blood transplants, along with the months of care required after a transplant, can cost $100,000 to more than $300,000, according to the U.S. Department of Health and Human Services.
“On some level, you’re going to want to say, I want the best of the best. I want to go to the Mayo Clinic. I want to go to the Cleveland Clinic. I want to go to Cedars-Sinai,” Salo said, naming some of the most prestigious hospitals in the country. “When you’ve got the best of the best and you’ve got everybody wanting the best of the best, you can’t physically do that.”
In many cases, out-ofstate care is justified, Salo said. But it’s up to individual Medicaid programs and plans to figure out when.
The Illinois Department of Healthcare and Family Services, which oversees Medicaid in Illinois, said, in a statement that “Health plans must provide care for covered Medicaid services, whether these are offered in Illinois or out of state.”
The department said it monitors health plans to ensure that care is being provided, and has an appeals process for members who feel they’ve been wrongly denied care.
Many states have moved to using Medicaid managed care organizations, and last year, then-Gov. Bruce Rauner expanded and revamped Illinois’ Medicaid managed care program in hopes of improving care for patients and saving money. Critics of the system, however, have complained about late and missing payments from insurers to hospitals, and have said they worry about putting state and federal dollars meant for patient care into the hands of for-profit companies.
States typically pay Medicaid managed care organizations, such as MeridianHealth, flat monthly fees per member. The idea is to encourage the managed care groups to use the money in the most efficient way possible, such as by keeping members healthier and better coordinating care.
But those flat monthly payments also may prompt the companies to deny services that are expensive, said MaryBeth Musumeci, associate director at the Kaiser Family Foundation’s program on Medicaid and the uninsured.
“If you’re looking at a set amount of money at the beginning of the month, there can be this perverse incentive to want to ration that,” she said.
The Office of Inspector General at the U.S. Department of Health and Human Services has said it plans to look into the issue, reviewing the extent to which Medicaid managed care organizations denied services or payments only to have those decisions overturned on appeal. It said, “Payment models in managed care may create an incentive for MCOs (managed care organizations) to inappropriately limit or deny access to covered services to increase profits.”
The race to help Jax
Jax has been on Medicaid his whole life. His dad, Damien Miller, works at an automotive shop, and his mother is a stay-at-home mom.
Though Miller gets health insurance through his employer, he can’t afford to extend that coverage to the entire family.
That wasn’t a problem until about a year ago. Jax’s right eye started crossing inward. An ophthalmologist recommended he wear glasses for three months.
But when his eye didn’t improve, the doctor referred him to a specialist at SSM Health Cardinal Glennon Children’s Hospital in St. Louis, about a two-hour drive from Jax’s home.
The specialist observed that Jax’s nerves were raised behind his eyes and recommend an MRI. The test showed abnormalities, at which point Jax was referred to a pediatric neurologist. After further testing, he was diagnosed with metachromatic leukodystrophy in July of this year.
That’s when the race began to help Jax before he deteriorated further.
Boden and Miller applied for Jax to take part in two clinical trials of treatments for the disease. But he didn’t qualify for either trial, his parents said.
Jax’s family did all it could while waiting for approval from MeridianHealth.
They installed additional handrails to help Jax use the stairs at home. They set up a GoFundMe page and sold “Team Jaxtien” bracelets to raise money for his medical expenses. The family distributed “Team Jaxtien” canisters to local businesses, which put them atop counters.
Boden describes her hometown — where the only businesses are a gas station and a dollar store — as a place where “neighbors look after neighbors.”
Jax’s parents also enrolled him in physical therapy in nearby Quincy, in hopes of keeping his muscles as strong as possible. During a recent appointment, Jax happily waded through a colorful ball pit, an exercise designed to help him with his full-body strength.
The physical therapist eventually coaxed him out of the ball pit for another exercise. She set up hurdles, a couple of inches off the ground, for him to step over while wearing ankle weights.
“Why?” Jax asked in a high-pitched voice, as she affixed the weights to his ankles.
He was growing tired from the exercises. He was losing his balance more easily and experiencing tremors.
“Why are we putting these on your legs?” the therapist responded. “These are going to make your legs stronger.”
Changing the law
The two months that Jax’s family spent trying to get insurance approval for the transplant were hard on the family.
“I’m trying to do everything I possibly can, and doing nothing is making me feel helpless,” said Miller, 31, before Jax received MeridianHealth’s approval.
But once Jax got the OK from the insurance company, the family wasted no time. Within days of getting the call from MeridianHealth, they were on a plane to Pittsburgh.
But it shouldn’t have been so difficult to gain that approval, said Suhr, with the MLD Foundation. The office of state Sen. Jil Tracy, R-Quincy, also got involved, contacting the state agency that oversees Medicaid, after Jax’s grandmother reached out to Tracy’s office.
Some groups have been working to try to make it easier for children on Medicaid to get coverage for out-of-state treatments. The Children’s Hospital Association pushed for federal legislation that was signed into law in April that helps that cause in cases of medically complex cases.
That law goes into effect in 2022. It will be up to states whether to opt into it.
Boden likes the idea of working to change federal law to make it easier for other sick children on Medicaid to get care out-of-state. “I fear for other children in the future who are going to have to go through the same thing,” she said.
But for now, she’s focused on Jax. The 4-year-old spent this past week in Pittsburgh undergoing testing, in hopes of starting the transplant process soon. Doctors have to determine if he’s still a good candidate for a transplant. After the testing, his parents will have to decide whether to move forward with the transplant, which carries risks such as death and disability.
If Jax undergoes the transplant, he’ll likely have to spend three to six months in Pittsburgh recovering. There’s no telling exactly how long a transplant might extend his life or quality of life.
But for the first time in months, Jax’s family is moving forward.