Jana Gundy and Amanda Chaffin live within two hours of each other in Oklahoma. Each has a child with the same devastating disease, one that robs them of muscle strength, affecting their ability to sit, stand or even breathe.
So both families were ecstatic when the Food and Drug Administration approved the first treatment for the genetic condition — known as spinal muscular atrophy (SMA) — two days before Christmas 2016. It seemed the gift they had been waiting for — a chance to slow the heartbreaking decline of their young sons.
But that common hope has taken them down different paths: In April, Gundy’s child, who is on private insurance, began getting the drug Spinraza, which costs $750,000 for the initial year of treatment. Chaffin’s child — a Medicaid enrollee — was not receiving the drug, as his state regulators debated whether to offer it to children like him who use ventilators to breathe. […]
The stress is unbearable for families trying to obtain treatment for sick children — caught in the arcane world of coverage decisions and businesses’ price calculations. […]
Spinraza isn’t a cure for SMA, which affects 10,000 people in the U.S., but clinical tests show it holds promise for some. Scientific discoveries by researchers at the University of Massachusetts Medical School in the early 2000 — partly funded by grants from the National Institutes of Health and donations from patient-advocacy groups — helped pave the way for Spinraza.
The drug was granted “orphan” status, which provides tax credits for research and helped speed the review process. It won approval in five years after the start of clinical trials, based on results of a few small studies. The FDA cited interim results from one of those studies in giving its OK in which 40 percent of the patients given the drug showed improvement, compared with none in the control group. Final results came later, showing 51 percent of treated children demonstrated improvement. It was tested only on children, most under age 2, though it was approved for pediatric and adult use.
The above article links to another KHN article (below) looking at the use of Spinraza in adults:
Denial, Appeal, Approval … An Adult’s Thorny Path To Spinraza Coverage
Julie Appleby, Kaiser Health News: August 2, 2017
Tammi Bradley jumped on her computer the minute she heard the news about the Food and Drug Administration’s approval of a drug targeting spinal muscular atrophy (SMA), a progressive muscle-weakening condition she’s had since childhood. […]
But there’s a catch for older patients like Bradley: It was not tested in adults in the clinical trials leading to approval. As a result, some insurers won’t authorize it for adults.
In Bradley’s case, it led to a tug of war with her health plan, which says it covers Spinraza “when determined to be medically necessary.”
Initially, coverage was denied. “As of today, there is no medical indication of Spinraza on patients over the age of 15,” said the June 2 letter she received from Kaiser Permanente. (Kaiser Health News is not affiliated with Kaiser Permanente.) […]
She appealed the decision to an outside reviewing agency overseen by the state, and it overturned Kaiser’s denial about two weeks later. […]
Biogen, the maker of Spinraza, and patient-advocacy groups like Cure SMA believe the drug should work similarly in all patients.
Nevertheless, it’s more difficult to measure improvement in adults because they are past the developmental milestones, which provide straightforward markers to assess the progress of infants and toddlers taking the drug.
Asking insurers to pay for a drug without proof that it works is becoming a harder sell.
“Insurers in the past have said where there’s a rare disease, even if the drug was expensive, they wouldn’t blink,” said Diana Zuckerman, president of the National Center for Health Research, a nonprofit that reviews health research. “But obviously they can’t keep doing that if there is a pattern of FDA approving drugs based on zero evidence for some groups and companies charge these outrageous amounts of money for drugs that don’t cure.”
Read the first article here
Read the second article here