treatment for many patients with Type 1 and Type 2. The policy excludes patients who have tracheostomies or depend on “invasive” ventilation, and limits coverage for some using “noninvasive” ventilation—many Type 1 and Type 2 patients fit this description, she says. Davis, a UHC member, is unsure whether her son Hunter (pictured)—who has Type 1 SMA but typically uses noninvasive ventilation 8 hours at night—will be eligible for reimbursement. Hunter got three doses of nusinersen under Biogen’s compassionate use program, but the next is subject to approval from UHC.
[Updated here and below with comments from UHC] Tracey Lempner, a spokesperson for UHC, says the insurer’s commercial plans will begin covering nusinsersen starting April 1, with prior authorization, meaning there will be a wait for patients applying beforehand.
Jefferies analyst Brian Abrahams added in a research note that patients must have two copies of the SMN2 gene to get covered. That criteria could eliminate eligibility for some 25 percent of Type 1 patients, he wrote.
“The policy essentially cherry picks which SMA patients will receive coverage. It’s both maddening and comical,” Davis says. “While Anthem’s medical policy chose to cover weaker SMA patients, UHC’s seeks to cover stronger patients—while still excluding Type 4s, the strongest of all.”
“Our coverage policy is based on the drug manufacturer’s study of the effectiveness of the drug in patients 16 years or younger with Type 1, 2 or 3 [SMA],” Lempner said. “When clinical studies are conducted and the product is shown to be effective on patients with Type 4 SMA, we will review our coverage policy.” She added that coverage can vary based on a member’s health plan.
Davis is a member of the Families for Acceleration of SMA Treatments (FAST) Movement, a grassroots group of SMA families pushing for access to treatments. FAST has sent a letter to Anthem calling for the insurer to reverse its policy; it plans to send one to UHC as well. The “inclusion and exclusion criteria cross a dangerous line,” she says. “When insurance companies like UHC make changes to the FDA-approved label, based on trials conducted by doctors, and reviewed by teams of FDA doctors prior to approval, they are essentially engaging in the practice of medicine.”
The question is whether more payers will follow the lead of UHC or Anthem—particularly Medicaid, which covers an estimated two thirds of SMA patients, according to Abrahams’s research note. Initial feedback from a survey of public payers suggests Medicaid is more likely to follow Anthem and skew “towards more reimbursement for Type 1 patients,” he wrote. Coverage for Type 2 and Type 3 patients for public payers is less likely this year given “the view that Type 2/3 patient numbers in the available study are small, the [Phase 3] data are currently unpublished, and results are not specifically called out in the label—despite the label allowing for treatment across all 3 subtypes.”
But payers’ reactions to high priced rare disease treatments are not necessarily predictable anymore, now that the debate has risen to the highest levels of the federal government. Even before getting two different conclusions from Anthem and UHC based on the same data, Biogen officials were not expecting the reimbursement doors to swing wide open.
On a conference call last week, new Biogen CEO Michel Vounatsos said reimbursement challenges and capacity constraints at treatment centers have the company expecting a “gradual uptake” of the drug throughout the year. (Last month, Xconomy wrote about the difficulty one Type 3 SMA patient, Mikhail Memedovich, is having obtaining treatment.)
“We believe that over time, logic will prevail in terms of having patient access to therapy that is proven to save lives,” Vounatsos said on the call. “And the demand from the patient side is there and is strong.”
Here’s more on SMA and Biogen’s launch of nusinersen.
