Same patient, same drug, same insurer — coverage denied

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From STATNews

By Tara Bannow March 2, 2023

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.”

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My son’s time is running out due to a rare disease. The FDA needs to add more clinical trial flexibility

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By Judy Stecker

Feb. 28, 2023

My toddler, Wheeler, will probably not live to adulthood. Juvenile Batten disease — he has the type known as CLN3 — is stealing his childhood. And then this rare disease will steal my child.

Wheeler is missing the DNA needed to recycle a waste product called lipofuscin that his cells naturally produce. As lipofuscin builds up, his condition will get worse, robbing him of his eyesight, his speech, his mobility, and ultimately his life.

Our only hope is finding a treatment to cure, or at least a way to slow down this unrelenting disease. At age 3, Wheeler already has sleep and behavior problems, as well as delays in his speech and fine motor development. He recently began moving closer to the TV and rubbing his eyes, signals that in a matter of months or weeks he may soon live the rest of his life blind.

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Haystack Responds to CMS’ National Provider Directory RFI

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See our full comments here

If CMS determines to develop the NDH, we ask that it do so in consultation with patient groups, including rare and ultra-rare disease advocacy organizations, so that the end product is useful, accessible, and efficient for patients and their caregivers. Haystack expects that the types of information that will be most helpful to patients includes, at a minimum:

  • Provider name and contact information, including all practice locations

  • Complete licensure information o This should include all states in which a provider is licensed regardless of physical location, as recent advances in telehealth allow providers to treat across state lines

  • All networks and plans to which a provider is enrolled (and their status, i.e., participating in a plan, “accepting” reimbursement as payment in full, out-of-network provider willing to submit claims, etc.) o This should include Medicare, Medicare Advantage, Medicaid fee for service (and states for which Medicaid is accepted), Medicaid managed care (including Medicaid plans in other states) and all private insurance

  • Provider specialties and sub-specialties

  • Years in practice

  • Hospital affiliation • Whether new patients are being accepted

  • Average wait time for both new and existing patient appointments

  • The availability of telehealth services

  • Languages spoken

FDA needs to be more flexible assessing rare

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The FDA needs to be more flexible in assessing treatments forrare diseases, like the one that seemed to help my son

By Karen Quandt Sept. 7, 2022 Maria Fabrizio for STAT

Every time I read about clinical trials testing possible treatments for rare diseases, I think of my son, Ty, whose brief but successful foray into such atrial highlights their value and their devastating limitations. Ty was born in Pocatello, Idaho, in October 1996 with green eyes, a big personality, a great sense of humor, and — as we finally learned eight yearslater — a rare and fatal disease called Niemann-Pick type C. He appeared to be healthy at birth, and hit all of his first developmental milestones of walking and talking and growing. Yet we realized that he hadtrouble moving his eyes upward, and had to move his whole head to look up. Then his fine motor skills and balance began to lag, and he had difficulty learning. An enlarged spleen complicated the picture.Ty was eventually diagnosed with Niemann-Pick type C.

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