By Denise Roland and Peter Loftus
The Food and Drug Administration approved a first-of-its kind
cancer therapy aimed at bolstering a patient's own immune cells,
while the drugmaker behind the treatment attempted to allay worry
over the high cost of the procedure.
Swiss pharmaceuticals giant Novartis AG said it would charge
$475,000 for the treatment, which involves extracting a patients'
disease-fighting blood cells, modifying them to attack cancer cells
more vigorously and then reinjecting them in the patient. The
$475,000 price tag was significantly cheaper than many analysts had
expected.
The procedure can only be undertaken at a limited number of
facilities in the U.S. It is highly tailored to individual patients
and can take the better part of a month to complete. Those
logistical hurdles and its expected high price had cast a shadow
over what has otherwise been seen as a groundbreaking
treatment.
The treatment, called CLT019 but re-christened by Novartis after
the FDA approval as Kymriah, has been shown to dramatically raise
the chances of survival for children and young people with an
aggressive form of leukemia, who don't respond to standard
treatment.
In a bid to blunt criticism over the price, Novartis said it
would only take payment for patients covered by Medicaid if they
respond to Kymriah within a month of treatment. The company also
said it would offer financial assistance -- such as help with
copay, travel and accommodation costs -- to privately insured
patients.
"We have taken a very responsible approach" to pricing, said
Bill Hinshaw, Novartis's U.S. oncology head. He cited independent
cost-effectiveness estimates that showed Kymriah could command a
price of $600,000 to $750,000.
He said that because patients will be children or young adults
-- who typically fall under their parents' or caregivers' private
insurance plans, Medicaid or other federal plans aimed specifically
at children -- few patients would likely end up paying near the
full price out of pocket. That cost will fall to the employers,
insurers and taxpayer-funded government programs that fund health
care costs.
Novartis Chief Executive Joe Jimenez said the company may charge
a lower price in cancer types where the benefit is less dramatic.
Novartis is testing Kymriah in adults with diffuse large B cell
lymphoma, another form of blood cancer, but the remission rates are
lower than in childhood leukemia.
Still, the company has already faced backlash over the price.
"While Novartis's decision to set a price at $475,000 per treatment
may be seen by some as restraint, we believe it is excessive," said
David Mitchell, president of Patients for Affordable Drugs, a
campaign group backed by the Laura and John Arnold Foundation which
supports efforts to make prescription drugs more affordable in the
U.S. "Novartis should not get credit for bringing a $475,000 drug
to market and claiming they could have charged people a lot
more."
Steve Pearson, head of the Institute for Clinical and Economic
Review, which studies the cost-benefit of drugs, said that while
the price is lower than analysts expected, it should be judged on
whether it reflects the benefit to patients.
"It will leave patients and others wondering" why $475,000 is
the right price "instead of 175 [thousand], 600, 750 or any other
number," Dr. Pearson said. "And questions will remain about how
this price will affect patients' ability to access the drug, and
how it will be used to set a benchmark for other uses of this drug
and perhaps other cancer drugs in the future."
ICER is currently conducting a cost-benefit analysis of Kymriah
that it plans to report early next year.
On Wednesday, the FDA approved Kymriah for children and young
adults up to 25 years old, who suffer from acute lymphoblastic
leukemia and who have not responded to standard therapy. The
decision was expected, following the backing of an FDA advisory
committee of experts, who voted unanimously in favor of approval in
July.
Kymriah is a form of personalized immunotherapy known as CAR-T,
or chimeric antigen receptor T-cell therapy. A patient's T-cells,
which are a type of white blood cells, are removed and then sent to
a manufacturing center where they are genetically programmed to
target leukemia cells. The cells are then infused back into the
patient to kill cancer cells.
In the early days of CAR-T research in the late 1990s, "no one
ever thought it would be possible to commercialize a therapy like
this," said Carl June, a professor of immunotherapy at the
University of Pennsylvania, whose research led to the development
of Kymriah. "I think the whole cancer world's going to be changed
forever," referring to the potential of CAR-T to treat more types
of cancer.
So far, it has only shown promise in a few blood cancers, but
Novartis is in the early stages of testing CAR-T in some forms of
lung and brain cancer. Dr. June said that some of the first
leukemia patients to receive the CAR-T treatments about seven years
ago remain cancer-free, but it will take longer-term follow-up to
see if the therapy completely eradicates cancer in these patients.
Dr. June will receive royalties from the product's sales; he said
he couldn't disclose specifics.
Acute lymphoblastic leukemia affects bone marrow and blood and
is the most common childhood cancer in the U.S. There are about
3,100 patients ages 20 and younger diagnosed with the disease each
year, according to the National Cancer Institute. The roughly 600
of these who don't respond to standard treatment would be eligible
for Kymriah.
The new treatment field is growing quickly, with the FDA saying
it has approved 76 applications by companies and researchers to
test CAR-T treatments. "This may turn out to be more broadly
applicable," especially in treating other types of blood cancer,
said Peter Marks, director of the FDA's Center for Biologics
Evaluation and Research. This week, Gilead Sciences Inc. agreed to
pay about $11 billion for Kite Pharma Inc., in an ambitious bet on
the field.
Write to Denise Roland at Denise.Roland@wsj.com and Peter Loftus
at peter.loftus@wsj.com
(END) Dow Jones Newswires
August 30, 2017 16:03 ET (20:03 GMT)
Copyright (c) 2017 Dow Jones & Company, Inc.
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