Janice Morales-Ferrer is a planner. It’s helped her get ahead in her career and in raising three young kids. But it’s hard to plan when you’ve got a rare disease, especially one with no standard treatment regimen or predictable outcome.
“I’m a data-driven person,” said the 46-year-old Boston area resident. “It’s frustrating for me to not be able to plan how long I can live for my kids. There’s no data. You don’t know what your prognosis is. You keep going and you hope for the best.”
Morales-Ferrer has focal segmental glomerulosclerosis, a rare kidney disease that results in scarring in the parts of the kidneys that filter blood. Even with treatment, many FSGS patients eventually progress to kidney failure and need dialysis or transplants. Recurrence is common after transplants.
Until a few weeks ago, things were looking even more dire. Morales-Ferrer’s insurance company was refusing to pay for the same infusion medication, Rituxan, that it had covered during her first bout of FSGS, one that her doctor credited with sending her disease into remission for years. She wrangled with them for weeks, facing down three separate denials, one of which forced her to cancel an important infusion. Her story offers a window into the confounding, seemingly irrational world of insurance coverage, one that’s given rise to a network of billing gurus dedicated to helping patients fight denials.
“The medication is right there,” she said. “My doctor is ready to offer it to me, but they’re blocking it. It’s mind-boggling.”
Two days after STAT inquired with her insurer, Blue Cross Blue Shield of Massachusetts, the company said that it had decided to cover the drug after all. While it doesn’t normally cover rituximab [Rituxan] for FSGS patients, BCBSMA said, in this specific case, there was enough evidence to support its use. Coverage was approved for Ruxience, a biosimilar version of Rituxan.
“An individual exception was granted after extensive research found clinical literature supporting the off-label use of rituximab in some cases of FSGS,” Ashley Yeats, BCBSMA’s vice president of medical operations, said in a Feb. 15 statement to STAT.
When Morales-Ferrer was first diagnosed with FSGS in 2014, she was a healthy 38-year-old who had just finished her third triathlon. But she developed a cough, high blood pressure, and strange bloating in her face, thighs, and abdomen. A blood test revealed protein in her urine, a sign of kidney damage. A biopsy confirmed FSGS.
The timing couldn’t have been worse. She and her husband, Jorge, were raising three young kids. Just two years earlier, they adopted their son, Gabriel, one month after Morales-Ferrer had given birth to their other son, Diego. Her daughter, Natalia, was a toddler. After the diagnosis, Morales-Ferrer left her job at a small web commerce company and moved the family to Puerto Rico to be closer to family. She grew up in the small town of Moca on the island’s west side. They returned to the Boston area in 2019.
At that time, Morales-Ferrer had the same health insurance she does currently: Blue Cross Blue Shield of Massachusetts. Rituximab, a monoclonal antibody medication typically used to treat certain types of cancer, was covered with no issues; she didn’t even have a copay.
This time around, it’s been a completely different story.
The first denial came on Feb. 6, a week after her doctor submitted the request. BCBSMA said there was no documentation that Morales-Ferrer had tried either of the two preferred biosimilar drugs, Riabni or Ruxience. She canceled a Feb. 8 infusion appointment, and her doctor submitted a request for Ruxience.
On Feb. 8, BCBSMA issued its second denial, this time for Ruxience. The company said the drug is not FDA approved for FSGS and, as such, did not meet its medical necessity criteria. Morales-Ferrer’s doctor asked the company to reconsider. On Feb. 9, BCBSMA sent its third denial letter, repeating the rationale from the second letter.
Without any coverage, Morales-Ferrer said she faced paying almost $5,000 per infusion visit, including $4,200 for the cost of Pfizer’s Ruxience alone. The FDA approved Ruxience as a Rituxan biosimilar in 2019. In the two years prior, Rituxan saw one of the largest increases in U.S. spending — $549 million — due to price hikes unsupported by new clinical evidence, according to a report from the Institute for Clinical and Economic Review. In that time, Rituxan’s manufacturer, Genentech, increased its net price by 14%.
Morales-Ferrer and her husband, who works at a medical device company, have good jobs, but paying for the treatments out-of-pocket would have meant significant financial hardship, maybe even bankruptcy. She went ahead with the first infusion on Feb. 13, agreeing to cover the full cost if her insurer didn’t. In the meantime, she entered a grievance process with BCBSMA that allows her doctor to speak with a doctor at the insurer about her case.
Insurers often initially deny coverage for drugs like rituximab, said Priya Verghese, division head of pediatric nephrology at Ann & Robert H. Lurie Children’s Hospital of Chicago and a pediatric nephrology professor at Northwestern University’s Feinberg School of Medicine. But when she explains the clinical justification to the insurers’ doctors, they almost always approve the drug in the end, she said. Verghese is not involved in Morales-Ferrer’s treatment.
“I feel like they make you jump through hoops in the hopes that some people will give up along the way,” Verghese said. “I always get the drugs I need approved for my kids.”
Most doctors would prescribe rituximab again for FSGS patients who responded well in the past when they have a recurrence, Verghese said. A small 2022 study in the journal Kidney International Reports found previous response to rituximab is the main determinant of response to further treatment with the drug.
Some people develop FSGS because of infections, drug use, other medical conditions like diabetes, or genetic factors. For others, like Morales-Ferrer, there’s no known cause. When it’s not clear why the patient developed FSGS it can be especially difficult to treat, Verghese said.
“There is no protocol,” she said. “There are no guidelines. There are no norms.”
Most doctors start with steroids in both adult and pediatric patients, Verghese said. But those are temporary because the side effects are “abysmal,” with the potential to cause cataracts and bone loss.
After that, Verghese said doctors typically try calcineurin inhibitors like tacrolimus or cyclosporine, which suppress the immune system, and then rituximab. Even though it’s used off-label, Verghese said the data clearly show the drug is effective in a subset of FSGS patients who responded to steroids and are experiencing relapses.
“We are all using it,” she said.
Jing Miao, an assistant professor of medicine in Mayo Clinic’s nephrology department, agreed that the first thing to know is whether the patient responded to steroids. If they did, there’s a good chance the patient will also benefit from rituximab. Miao said rituximab is also good to try because, unlike steroids, it’s relatively safe with minimal side effects. Miao is not involved in Morales-Ferrer’s treatment.
After her initial diagnosis, Morales-Ferrer’s doctor at Massachusetts General Hospital started her on steroids. While helpful in treating her FSGS, the side effects were predictably bad: constant headaches, mood swings, hot flashes, and trouble sleeping. After that, she briefly went on a drug called cyclophosphamide that’s a form of chemotherapy. Finally, her doctor prescribed rituximab, which is delivered through 4-hour infusions. At first, the infusions happen every two weeks, then they’re more spaced out.
The rituximab was working so well, Morales-Ferrer’s doctor raised the idea of stopping treatment altogether. Best case scenario: The disease might not return. They decided to try it. For almost four years, the disease stayed at bay.
Then, in December, Morales-Ferrer noticed foam in her urine, a sign that her kidneys weren’t properly filtering protein. Her doctor, who declined to be interviewed, confirmed the FSGS had returned at a checkup in January. The news brought tears, but also a sense of resignation.
“I was extremely sad, but they always told me it was probably going to come back at some point,” Morales-Ferrer said. “I was just enjoying my healthy years.”
Fortunately for Morales-Ferrer, the frustrating insurance coverage predicament ultimately worked out in her favor.
The reason the drug was covered in 2014 is because BCBSMA didn’t require prior authorization around the use of rituximab for most conditions at the time, Yeats said. Prior authorization means the insurer has to do a clinical review to see whether medical evidence supports coverage. Since then, the company has added specific indications for which it doesn’t think there’s enough evidence to warrant coverage, FSGS being one of them.
BCBSMA informed Morales-Ferrer of its change of heart in a Feb. 17 approval letter. The letter says the approval is valid for one year. Morales-Ferrer said she’s concerned about having to go through the same process all over again in future years. She said she’ll likely need the treatment for several years, if not for the rest of her life.
She hopes BCBSMA will improve its transparency. And she worries about people who are older or sicker than her and don’t have the time or energy to challenge the denials.
“People will just give up,” she said. “They won’t fight it.”
Correction: An earlier version of this article misstated Morales-Ferrer’s age at the time of her first diagnosis.
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