By Liz Szabo August
23, 2017
When doctors talk
about a new leukemia drug from Novartis, they ooze enthusiasm, using words like
“breakthrough,” “revolutionary” and “a watershed moment.”
But when they think
about how much the therapy is likely to cost, their tone turns alarmist.
“It’s going to cost a
fortune,” said Dr. Ivan Borrello at Johns Hopkins Sidney Kimmel Comprehensive
Cancer Center in Baltimore.
“From what we’re
hearing, this will be a quantum leap more expensive than other cancer drugs,”
said Leonard Saltz, chief of gastrointestinal oncology at Memorial Sloan
Kettering Cancer Center in New York.
Switzerland-based
Novartis hasn’t announced a price for the medicine, but British health
authorities have said a price of $649,000 for a
one-time treatment would be justified given the significant benefits.
The cancer therapy was
unanimously approved by a Food and Drug Administration advisory committee in
July, and its approval seems all but certain.
The treatment, CTL019,
belongs to a new class of medications called CAR T-cell therapies, which
involve harvesting patients’ immune cells and genetically altering them to kill
cancer. It’s been tested in patients whose leukemia has relapsed in spite of
the best chemotherapy or a bone-marrow transplant.
The prognosis for
these patients is normally bleak. But in a clinical trial, 83
percent of those treated with CAR T-cell therapy — described as a “living drug” because
it derives from a patient’s own cells — have gone into remission.
CAR T cells have been
successful only in a limited number of cancers, however, and are being
suggested for use as a last resort when all else has failed. As a result, only
a few hundred patients a year would be eligible for them, at least initially,
said Dr. J. Leonard Lichtenfeld, deputy chief medical officer for the American
Cancer Society.
The FDA is scheduled
to decide on approval by Oct. 3. The agency is also considering a CAR T-cell
therapy from Kite Pharma.
A third company, Juno
Therapeutics, halted the development of one its
CAR T-cell therapies after five patients died from
complications of the treatment.
Rather than wait for
Novartis to announce a price, an advocacy group called Patients for Affordable Drugs has
launched a pre-emptive strike, asking to meet with company officials to discuss
a “fair” price for the therapy. The Novartis drug has the potential to be one
of the most expensive drugs ever sold, said David Mitchell, the patients
group’s president, who has been treated for multiple myeloma, a blood cancer,
since 2010. (The Laura and John Arnold Foundation, which provides some funding
for Kaiser Health News, supports Patients for Affordable Drugs.)
“Many people with
cancer look forward with great hope to the potential of your new drug,”
Mitchell wrote in a letter to Novartis. “But
drugs don’t work if patients can’t afford them.”
Cancer drugs today
routinely cost more than $100,000 a year. A combination therapy for melanoma
sells for $250,000. Such prices are particularly outrageous, given that
taxpayers fund many drugs’ early research, Mitchell said.
The federal government
spent more than $200 million over
two decades to support the basic research into CAR T-cell therapy, long before
Novartis bought the rights.
The patients group
urged Novartis to charge no more for the drug in the U.S. than in other
developed countries.
Novartis has agreed to
meet with the patients group. In a statement, Novartis said the company is
“carefully considering the appropriate price for CTL019, taking into
consideration the value that this treatment represents for patients, society
and the healthcare system, both near-term and long-term.”
Novartis made a
significant investment in CAR T-cell therapy, according to the statement.
“We employ hundreds of
people around the world who work on CAR-Ts, we are conducting ongoing U.S. and
global clinical trials, and have developed a sophisticated, FDA-validated
manufacturing site and process for this personalized therapy.”
Soaring prices for
cancer drugs have led many patients to cut back on treatment or skip pills, a
recent Kaiser Health News analysis showed.
The effect of CAR
T-cell therapies on overall health costs would initially be relatively small,
because it would be used by relatively few people, Lichtenfeld said.
Health systems and
insurers may struggle to pay for the treatment, however, if the FDA approves it
for wider use, Lichtenfeld said. Researchers are studying CAR T-cells in a
number of cancers. So far, the technology seems more effective in blood
cancers, such as leukemias and lymphomas.
Hidden costs could
further add to patients’ financial burdens, Borrello said.
Beyond the cost of the
procedure, patients would need to pay for traditional chemotherapy, which is
given before CAR T cell therapy to improve its odds of success. They would also
have to foot the bill for travel and lodging to one of the 30 to 35
hospitals in the country equipped to provide the high-tech treatment, said Dr.
Prakash Satwani, a pediatric hematologist at New York-Presbyterian/Columbia
University Medical Center, which plans to offer the therapy.
Because patients can
develop life-threatening side effects weeks after the procedure, doctors will
ask patients to stay within two hours of the hospital for up to a month. In New
York, even budget hotels cost more than $200 a night — an expense not typically
covered by insurance. Patients who develop a dangerous complication, in which
the immune system overreacts and attacks vital organs, might need coverage for
emergency room care, as well as lengthy stays in the intensive care unit,
Satwani said.
Doctors don’t yet know
what the full range of long-term side effects will be. CAR T-cell therapies can
damage healthy immune cells, including the cells that produce the antibodies
that fight disease. Some patients will need long-term treatments with a product
called intravenous immunoglobulin, which provides the antibodies that patients
need to prevent infection, Lichtenfeld said.
Saltz, an oncologist
who has long spoken out about high drug prices, said he applauded the patients
group’s efforts. But he said he doubts their efforts will persuade Novartis to
set a reasonably affordable price.
“I’m not optimistic
that this will have much effect on the company,” said Saltz. “There’s no market
pressure for the company to respond to.”
High drug prices don’t
just hurt patients, Saltz said. They also drive up insurance premiums for
everyone.
“They affect each and
every one of us,” he said, “because these costs will be paid by anyone who has
any kind of insurance coverage.”
KHN’s coverage of
prescription drug development, costs and pricing is supported in part by
the Laura and John Arnold Foundation.
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