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close the program at the end of December. Hunter’s next dose is scheduled for Jan. 18, so he’s now “at the mercy of [Davis’s insurer] United Healthcare” to pay for treatment.
“We do not have unlimited resources, and cannot afford $125,000 a vial for [nusinersen],” she says, adding that she’s “especially concerned” about patients who have yet to start treatment, particularly those who only have Medicaid, which insures roughly 77 million Americans. While the fate of government insurance programs is uncertain, President-elect Donald Trump and the GOP-controlled Congress are likely to make big changes that could roll back Medicaid expansion and give each state a “block grant” for Medicaid, which might mean less funds. (Here’s more in the New York Times.)
“We want to see this amazing treatment covered for every patient with SMA,” Davis says.
When asked about Davis’s concerns, Fearer said Biogen can’t comment on individual insurance plans. But the company and clinicians will help patients currently on its compassionate use program transition to approved therapy with nusinersen, he says.
Drug prices to treat rare diseases and more common ones, like cancer and deadly allergic reactions, have become a national debate. Even if the treatments, like nusinersen, are for deadly diseases with no alternatives, the prices can induce sticker shock.
Whether insurers and other payers push back against Biogen’s price remains to be seen, though nusinersen has a good case to make—it showed a clear benefit in placebo-controlled trials. One recent point of comparison is eteplirsen (Exondys 51), which in September became the first ever approved drug for Duchenne muscular dystrophy, a crippling, fatal genetic disease. The developer, Sarepta Therapeutics (NASDAQ: SRPT), set a price of $300,000 per patient, per year. But the drug was approved only for a tiny subset of Duchenne’s patients with a specific genetic malfunction, and, in a controversy that rocked the FDA, it only got the nod when top officials overruled their staff’s negative review. Sarepta did not definitively prove in clinical testing that the drug actually worked; the drug was approved because it was “reasonably likely” to benefit patients.
Many observers felt insurers would accept the price, fearing a backlash of denying a drug to a few thousand dying kids. But some insurers, like Anthem and Humana, have either denied coverage or placed restrictions on eteplirsen’s use. A recent survey by Jefferies analyst Gena Wang found several other instances of pushback from national and regional managed care organizations.
Shares of Sarepta skyrocketed after eteplirsen was approved in September, but have fallen all the way back to levels they were at before the decision due to these types of struggles.
[Updated with analyst comments] Nusinersen looks to have a better case to make to payers. Even so, in a research note Wednesday morning, Leerink Partners analyst Geoffrey Porges wrote that the drug’s high price tag is “likely to invite a storm of criticism, up to and including Presidential tweets” and may lead to backlash from insurers. “At the very least…the price is going to force payers to closely scrutinize which patients receive access and limit the overall access provided,” he wrote, adding that it “seems certain” that older patients with type 3 or type 4 SMA and milder symptoms will “find it difficult to obtain treatment.”
Jefferies analyst Abrahams believes the drug can bring Biogen as much as $1.6 billion a year. (Ionis gets royalties on sales via a partnership deal.)