FEATURE
PhRMA, only about two-thirds (63%) of a branded drug’s
list price is retained by the companies, with one-third
going back to payers, the government, or other stakehold-
ers. 13 Of that revenue, about 20% is then re-invested in
research and development for new treatments.
“The biotech market is considered a high-risk, high-
reward area of investment,” Dr. Fonseca noted. Economic
trade-offs exist between cancer drug pricing and innova-
tion, he continued, with several studies having shown
that a reduction in drug profitability stifles innovation
and investment. 14
So, while lower prices could increase access to drugs,
they may reduce access to innovative therapies in the
future.
Dr. Rajkumar, however, takes the opposite stance:
“The fact that pharmaceutical companies can price a drug
high regardless of whether or not it is innovative or just
a modification, like an analog rather than a new class of
drugs, is the enemy of innovation.”
What a Life Is Worth
Commenting on the new treatment options for myeloma,
Dr. Usmani said that, “All of these developments are won-
derful for patients, but the important question is, ‘Can
we continue to pay for all these drugs?’ What does the
introduction of so many myeloma therapies mean for our
patients who are dealing with copays and out-of-pocket
expenses for their cancer care?”
Dr. Fonseca, for one, believes that the extended sur-
vival seen with newer treatment approaches is worth the
high costs. “We would not have all of this progress absent
these expensive drugs,” he said.
However, Dr. Carlson and members of the Institute
of Clinical and Economic Review (ICER) disagree. The
organization recently looked at the cost-effectiveness
of drugs used to treat relapsed or refractory MM in
the U.S., evaluating carfilzomib, elotuzumab, ixazomib,
daratumumab, and panobinostat in combination with
lenalidomide, or bortezomib plus dexamethasone as
second- or thirdline therapy. 15 The introduction of these
regimens appeared to provide clinical benefit by length-
ening progression-free and overall survival and improv-
ing quality of life. However, using a cost-effectiveness
threshold of $150,000 per quality-adjusted life-year,
ICER found that “only the addition of daratumumab or
panobinostat may be considered cost-effective options.”
The authors concluded that “achieving levels of value more
closely aligned with patient benefit would require substan-
tial discounts from the remaining agents evaluated.”
“Critics will say that quality-adjusted life-years have
some limitations, and that is true,” Dr. Carlson told ASH
Clinical News, “but it is the best measure we have and has
been widely adopted.”
A similar study comparing carfilzomib, daratumumab,
and pomalidomide plus low-dose dexamethasone in re-
lapsed or refractory MM suggested that the pomalidomide-
dexamethasone combination may be the most cost-effective
option. 16 Assuming equal efficacy of the regimens, the
doublet regimen resulted in a cost-savings of about $12,000
over the two other agents.
Because it is not practical to have direct comparisons
for all regimens, creative cross-trial comparisons may
be useful. Despite the results reported in these studies,
the nature of MM is that most patients likely will require
sequential treatment with a majority of the available agents
to achieve the best possible outcomes.
Dr. Carlson encouraged clinicians to consider cost-
effectiveness during treatment selection, no matter what
patients are willing to pay.
“At some point, people need to understand that when
we pay for medical treatments, we have to give up paying
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for other things… like education or infrastructure.”
Dr. Fonseca again noted the trade-offs that come
with demanding lower prices for novel therapeutics. For
example, more drugs are available in the U.S. and become
available more quickly than in other countries.
“In the U.S., we are
blessed that we have
access to drugs for
our patients with
myeloma, but at the
same time, we are a
little frivolous with
the spending part of
it. There needs to be
some reconciliation.”
—SAAD USMANI, MD
“The reality is that access to drugs in countries like
the U.K. occurs at a much later time than it does in the
U.S.,” Dr. Fonseca said. “In turn, patients have inferior
outcomes. It is a trade-off of spending less and having less
access or spending more with earlier access.”
“In the U.S., we are blessed that we have access to drugs
for our patients with myeloma, but at the same time, we are
a little frivolous with the spending part of it,” Dr. Usmani
noted. “There needs to be some reconciliation.”
Solutions in Sight?
A variety of solutions have been proposed to begin to rein
in drug prices, at all levels of the drug-pricing system,
but finding real solutions will require the participation of
all stakeholders, Dr. Usmani added. “This issue has not
been looked at systemically,” he said. “Physicians have
been talking about it from their perspective, and patients
have been talking about it from theirs. We need to have
broader discussions about myeloma treatment costs that
culminate in solutions.”
One concept is value-based reimbursement and pric-
ing, which would allow the Centers for Medicare and
Medicaid Services (CMS) or other agencies to negotiate
the sale price of a drug based on the incremental value
provided by that drug.
“Developed countries except the U.S. have a system
where approval of a drug is only the first step,” Dr.
Rajkumar said. “The second step is a negotiation of the
price based on the value that new drug will bring.”
Dr. Rajkumar also supported an increase to com-
petition by allowing for easier entry for generics and
biosimilars into the market and patent reform. Legislative
efforts have stalled in the past, but they may have received
renewed interest recently, thanks to the introduction of
biosimilars.
Earlier this year, Sen. Patrick Leahy (D-VT), re-
introduced the Creating and Restoring Equal Access To
Equivalent Samples (CREATES) Act, which – among other
policies – would allow a biosimilar or generic developer
to bring a civil action against an innovator drug company
if the latter refuses to make available enough samples of a
product for testing. 17 The CREATES Act was first intro-
duced in 2016 and has enjoyed broad bipartisan support
but has not been voted on since its introduction.
“The more generics that come into the market, the
more likely prices are to fall,” he said. “We’ve seen it hap-
pen with imatinib and many other prescription drugs.”
These suggested solutions aren’t new, and few – if any
– have gained significant traction, but hematologists and
oncologists should still be paying attention.
“Several parties are involved in the pricing of and pay-
ment for myeloma treatment,” Dr. Usmani said. “It’s not
just the pharmaceutical industry or the insurance industry
or the health-care organization or the patient advocacy
groups or the treating physician – it’s all of us together.
We need to find a way to bring all of these stakeholders
together to actually start finding a solution.”
“We need to step up and we need allies. Big organi-
zations need to step up, too, and forget about what big
pharma brings them,” Dr. Rajkumar added. “We need to
call people to task. If we don’t, who will?” ●
—By Leah Lawrence
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