As potentially lifesaving, but costly, therapies become available for rare genetic diseases — such as Spinraza (nusinersen) for spinal muscular atrophy (SMA) — neurologists are finding themselves in the increasingly uncomfortable position of deciding which patients are most likely to benefit, and how they can help families pay for such treatments.
In response, the Minneapolis-based American Academy of Neurology (AAN) has formed a Neurology Drug Pricing Task Force to make recommendations and provide guidance to AAN members feeling overwhelmed at these tasks.
Its goal, said Dr. Nicholas E. Johnson, a pediatric neurologist and assistant professor at the University of Utah who heads the effort, is to try to define a workable ground between “what the [treatment’s] label says” about who might receive it, and “what the insurance company says” about whom it will cover.
“It’s nearly a full-time effort for a single staff person to work through the insurance approvals,” Johnson told SMA News Today in a phone interview from Salt Lake City, where he also has a practice. “This is a national problem from the physician’s side, because these drugs provide some risk in that they have to be administered through a spinal tap, and because these drugs carry such a high cost.”
The AAN’s 18-member task force includes 16 neurologists with expertise in practice guidelines, medical economics and ethics, along with two advanced practice providers. It expects to offer guidelines in early 2018.
Spinraza received approval from the U.S. Food and Drug Administration (FDA) in December 2016 as the first disease-modifying treatment for all forms of SMA. Manufactured by Biogen, it has shown highly promising results in clinical trials in young children, but carries a high price tag. Treatment costs an average of $750,000 for the first year and $375,000 for every year after that.
“We’ve been trying to get this medicine for a few adults with SMA and have been denied coverage,” Johnson said. “Thankfully, Biogen’s Patient Assistance Program has stepped in to help these patients out. In the long run, the AAN is there to provide advocacy for both neurologists and the patients they care for, and to make sure patients are provided medications that have disease-modifying, life-altering effects in a sustainable fashion.”
Normally, doctors have some leeway in using new medications in various settings and on a variety of patients, Johnson said. But in Spinraza’s case, “even though it was approved for all ages and all types of SMA, there’s very limited data in adults who have SMA, so this is a real challenge for providers,” he added.
The task force hasn’t yet offered any recommendations, and those made will require AAN leadership approval before being publicized. Besides Spinraza, therapies it will look at include Brineura (cerliponase alfa) for Batten disease, estimated to cost $702,000 a year, and Exondys 51 (eteplirsen) for Duchenne muscular dystrophy, which costs an estimated $300,000 annually.
“Neurologists care for a number of patients with rare diseases, and many of them have a lot of the same challenges as SMA,” Johnson said. “Our goal is to evaluate the environment as it stands right now to essentially provide some guidance for neurologists, and who to prescribe medications for. We don’t really have a good sense of how nusinersen works in the adult population. Normally, a neurologist would start by prescribing the medication and see how it works.”
Johnson compared the current situation to 2006, when the FDA approved an enzyme replacement therapy to treat Pompe disease, an extremely rare hereditary metabolic disorder that strikes roughly one in 40,000 U.S. newborns a year.
“Clinical trials were conducted for the most severe form of the condition, and the drug was approved for all ages. Providers had the ability to prescribe for adults, and they saw over time that it had less benefit,” he said. “But that type of post-approval process is now limited. So what the task force seeks to do is provide guidance and recommendations for those providers who are stuck between what the label says and what the insurance company says.”
Johnson said his Salt Lake City clinic — which covers Utah as well as Idaho, Nevada, Wyoming, and western Colorado — has 130 SMA patients, half of whom are adults.
“Even though it’s a rare disease, there are quite a few patients out there with SMA,” he said. “Utah’s state Medicaid budget is around $100 million a year. If you consider the costs of this drug for our 130 SMA patients, it would easily dwarf the rest of the budget. So we regard this as a very urgent issue.”
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