The first gene therapy to be sold in Europe
comes with a perk you don’t often see in
medicine: a money-back guarantee.
The treatment, called Strimvelis, is
the first outright cure for a rare disorder to emerge from gene therapy, and its
price tag of 594,000 euros ($665,000)
makes it one of the most expensive one-time treatments ever sold by a drug firm.
It’s also the first genetic fix to come with
“The drug has to deliver what you say
or we don’t pay,” says Luca Pani, direc-
tor general of the Italian Medicines
Agency, known as AIFA, which set the
price and terms during negotiations with
GlaxoSmithKline, the company commer-
cializing Strimvelis. The treatment uses a
virus to add a missing gene to the bone
marrow of children with ADA-SCID, a
sometimes fatal inability to fight infec-
tions. In a study involving 18 children, car-
ried out at a Milan hospital, all but three
were cured outright. GSK bought rights to
the treatment in 2010 and won approval
earlier this year to sell it in Europe, but
because of its complexity the company will
offer it only in Milan, requiring families to
spend weeks there. That means the Italian
price will apply to all of Europe, says Pani.
GSK declined to comment.
By some measures, Strimvelis’s price
counts as a bargain. The cost of a bone
marrow transplant from another person—
the established way to treat ADA-SCID—
can reach $1 million. Some other patients
get treated with enzyme injections that
cost $250,000 a year. The expense of
these drugs and the care needed for a sick
child quickly add up to millions.
The idea behind gene therapy is that a
one-time correction to a patient’s DNA will
lead to a lifelong cure. Strimvelis is the first
treatment to be commercialized that lives
up to the promise. But potential cures for
hemophilia, a rare eye disease, and a fatal
brain illness could reach the market next,
and they could be similarly expensive.
The Italian agency is unusual in that
it already imposes pay-for-performance
rules on some cancer drugs. It maintains
135 patient registries to track how well
they work, and Pani says Italy has collected more than 250 million euros in
refunds. Patients receiving GSK’s gene
therapy will also be tracked in a registry, the company confirmed. Experience
gained so far suggests that GSK might
end up refunding the cost of about one in
six treatments. But the big question isn’t
whether gene therapy costs too much—it’s
whether companies can make any money
at it, especially treating ultra-rare diseases.
Only about a dozen children are born with
ADA-SCID each year in Europe. Treating
all of them would generate about $8 million—barely a blip for GSK, which sells
$30 billion worth of drugs a year.
“Treating 12 kids a year—it’s just not
commercially viable, at any price,” says
Phil Reilly, a partner at Third Rock Ventures in Boston, who invests in gene-therapy companies. “We need a new
model for ultra-rare disorders, because
we are going to develop these treatments.”
Reilly says money-back guarantees and
pay-as-you-go schemes are two ways to
make high sticker prices palatable.
GSK says it won’t make much money
off Strimvelis. Instead, it sees the treatment as a way to help patients and gain
experience with genetic treatments.
It’s also working with a small company, Adaptimmune, to genetically alter
immune cells to battle cancer. “We hope
that Strimvelis will be the first of a number of innovative gene-therapy medicines
that we will bring to patients,” says Anna
Padula, a spokesperson for GSK’s rare-diseases group. The company recognizes,
she adds, “that the industry will need to
adapt the way in which medicines are
priced and funded.” —Antonio Regalado REB
Gene-Therapy Cure Has
The most expensive drugs in history, or medicine’s biggest bargains?
Gene therapy could be both.