TECHNOLOGY Bloomberg Businessweek August 26, 201917PHOTOGRAPH BY ROSS MANTLE FOR BLOOMBERG BUSINESSWEEK. DATA: AMERICAN SOCIETY OF GENE AND CELL THERAPY
BATTLING THE INSURER
Newborn screening gave Sarah and Logan Stanger,
public school teachers in Monroe, Ohio, a heads-up
that their son, Duke, had SMA in the spring of 2019.
He was 3 weeks old and showed no signs of the
disease that’s typically diagnosed after a desperate
search for why a baby has stopped moving.
The Stangers and their doctors held off on giv-
ing him Biogen Inc.’s Spinraza, the only other treat-
ment option, in hopes Duke could get the one-time
gene therapy instead. Spinraza costs $750,000 for
the first year of treatment and must be given every
few months for life, adding up to $375,000 in annual
drug costs in subsequent years. Duke wasn’t able to
get into any of the early studies of Zolgensma, but his
parents cried tears of joy when the FDA approved
the gene therapy, assuming their wait was over.
The family has health insurance through the
Butler Health Plan, which covers public school
teachers and librarians in their county and is run
by Anthem Blue Cross Blue Shield. They didn’t know
that the plan specifically excluded coverage for any
type of gene therapy, regardless of disease, the
patient’s age, or condition. The Stangers fought to
overturn the plan’s gene therapy exclusion rule. But
they didn’t know when or how their plea would be
heard and were told they couldn’t attend the meet-
ing where Duke’s case would be considered.
“It’s a tortuous thing to be told your child has
this terrible genetic disorder and there’s this treat-
ment out there, but we’re not going to cover it,”
Sarah says. “It’s mind-blowing.”
The Stangers have reached out to the state health
department and lawmakers. They’ve also had col-
leagues covered by the same insurance program
write letters to the insurer. Friends suggested that
the Stangers divorce and quit their jobs so their
household incomes would drop enough for them
to apply for Medicaid for Duke. Instead, they kept
fighting, and the plan changed its policy earlier this
month, to allow coverage for gene therapy. The fam-
ily is still waiting to see if it will now cover Duke,
who’s 5 months old and hasn’t yet shown any symp-
toms. “We work hard, we pay for our premiums, and
we believe our insurance should pay for the treat-
ment our child requires,” Sarah says.CROWDFUNDING
Eliana was 8 months old when her parents, Shani
Levi Cohen and Ariel Cohen, first realized there
was a problem. “All the other kids were stand-
ing up in their cribs already and making their first
steps, standing on the couch and falling, and my
daughter was almost hardly crawling,” says Shani,
a stay-at-home mom. Eliana’s doctor told her theGene therapy is bringing out the best—and worst—in
America’s health-care system. Zolgensma, the first
systemic gene therapy of its kind that the U.S. Food
and Drug Administration has approved, appears to
cure in one shot a rare muscle-destroying disease
that can kill infants and toddlers. (The kids still carry
the gene mutation; they just don’t exhibit the even-
tually fatal symptoms.) It’s also the world’s priciest
medication. Novartis AG set the cost at $2.1 million
after the FDA approval came down on May 24, and
some families have been left scrambling for ways to
get the drug in its first several months on the market.
The medical system always faces a learning curve
with a new treatment, particularly one this revolu-
tionary, but the stakes for patients who could ben-
efit from Zolgensma have made things that much
tougher. Only 400 American babies a year are
born with spinal muscular atrophy (SMA), but left
untreated, it kills them before they turn 2 years old.
“It’s a challenge anytime you introduce a new
therapy into the marketplace to gain understand-
ing from the insurers,” says Dave Lennon, president
of the Novartis division that developed Zolgensma.
All major insurance companies cover the treatment,
he says, but one-third of them have criteria more
restrictive than the FDA’s, which says any patient
under 2 can have it. “It’s still very much an evolv-
ing marketplace,” he says. “We want to make sure
the insurance companies and the community under-
stand the gaps in coverage and help us address that.”
Novartis has been less forward about potential
problems in its Zolgensma trials. While the FDA was
reviewing the drug, the company investigated poten-
tial manipulation of data in early animal trials and
placed two top scientists involved in Zolgensma’s
development on leave. (They’ve since left the com-
pany.) Novartis told the FDA about its probe in June,
weeks after the drug hit the market. The matter has
drawn fire from lawmakers, and the FDA has said
civil and criminal penalties are possible. Novartis
says it’s preparing a response to lawmaker inquiries.
Some insurers, including UnitedHealthcare,
the nation’s largest, have changed their initial pol-
icies and now cover patients according to the FDA’s
approval. Others, including Anthem Blue Cross Blue
Shield, continue to limit access only to those who
show symptoms before they reach 6 months of age.
Here are six SMA patients whose parents have
tried different approaches to make sure they thrive
in preschool and beyond. (These patients’ insur-
ers say they carefully consider coverage decisions.)
Their experiences offer lessons for the many people
who stand to benefit from the gene therapies on the
horizon: treatments for Parkinson’s disease, sickle
cell anemia, and other more common conditions. Patgiri and Usova
with ToraPhase 3Phase2-Phase 2Phase1-Phase 119510477183○ Number of gene and
cell therapy products in
developmentEarliest phase of
developmentClosest to approval