Novartis to Offer World's Most Expensive Drug for Free Via Lottery
December 19 2019 - 10:34AM
Dow Jones News
By Denise Roland
Novartis AG launched a lottery-style program to give away doses
of its pricey gene therapy for free, drawing criticism from patient
groups that say that is an inappropriate way to distribute a
lifesaving treatment aimed at babies.
Zolgensma, a one-shot cure for a deadly inherited disease whose
victims cannot control their muscles, is so far sold only in the
U.S. at a price of $2.1 million, making it the world's most
expensive drug. The so-called global managed-access program is
aimed at providing Zolgensma to a limited number of spinal muscular
atrophy patients outside the U.S.
Under the program, doctors can submit requests for the
treatment, with eligible patients entered into a draw every two
weeks for free doses. AveXis, the Novartis unit that makes the
drug, said it aimed to distribute around 100 free doses a year as
long as production capacity allows. Around 1,600 children are
thought to have the most severe form of SMA, known as Type 1, in
Europe alone.
However, patient groups criticized the move, highlighting the
thorny ethical issues that companies face when providing unapproved
treatments for free.
"It's really too crude," said Kacper Rucinski, co-founder of
U.K.-based patient-advocacy group TreatSMA. "They are making
patients compete. Which will be the lucky one? That's not
helpful."
Olga Germanenko, a board member of SMA Europe, an umbrella
organization for patient-advocacy groups from across the continent,
said the lottery made Zolgensma "look like a prize."
Patients would have instead welcomed a program that prioritized
the patients or countries with the highest needs, said Mr.
Rucinski. In some places, patients can already get a rival SMA
treatment called Spinraza, made by Biogen Inc. Novartis's program
doesn't prioritize countries where Spinraza isn't routinely
available or patients who don't respond to Spinraza.
A spokesman for Novartis said the company was advised to take
the lottery approach by a group of bioethics experts, who were
concerned that creating complicated criteria could unfairly
discriminate against some patients. "In the end we're dealing with
a difficult ethical dilemma," he said. "Unfortunately, there is no
perfect solution."
Novartis had faced high demand from patients outside the U.S. to
provide the treatment free of charge in some cases, although local
regulations may prevent some countries from adopting the
program.
Managed-access programs are common, especially for
life-threatening conditions where the treatment in question is
considered a breakthrough. However, companies typically use a
criteria based on medical need to decide who should receive the
treatment.
Novartis said it couldn't provide Zolgensma to every eligible
patient because of limited supply. The treatment is made at one
plant in the U.S., though the company hopes to open up two more
manufacturing sites within the next year.
Zolgensma is one of the first in a new wave of treatments known
as gene therapies that promise to cure certain inherited diseases
in a single treatment by providing a working copy of the faulty
gene.
It went on sale in the U.S. earlier this year and is under
review by regulators in Europe and Japan, with decisions expected
next year. Once a country or region approves Zolgensma, Novartis
will stop offering the lottery program there.
Despite its eye-catching price tag, Novartis has said Zolgensma
has sold well since its launch in late May, overcoming concerns
about whether insurers would cover the treatment as well as a
data-manipulation scandal at the unit that makes it.
Novartis has defended Zolgensma's price on the grounds it costs
less than Spinraza -- the rival treatment -- in the long term.
Spinraza is a lifelong treatment that costs $750,000 for the first
year and then $375,000 for each year thereafter.
Write to Denise Roland at Denise.Roland@wsj.com
(END) Dow Jones Newswires
December 19, 2019 10:19 ET (15:19 GMT)
Copyright (c) 2019 Dow Jones & Company, Inc.
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