FEATURE
Features
In Myeloma, New Drugs,
Skyrocketing Price Tags
I
n late February, seven pharmaceutical executives were
called before the U.S. Senate Finance Committee to tes-
tify about the prices of brand-name prescription drugs,
which are often described as “skyrocketing.”
CEOs from AbbVie, AstraZeneca, Bristol-Myers
Squibb, Johnson & Johnson, Merck, Pfizer, and Sanofi
– together representing $140 billion in U.S. revenue
– defended the value of the drugs they manufacture
and cited high research-and-development costs as the
reason for continued price increases. Senators discussed
Medicare’s inability to negotiate drug prices, the use of
patents to stifle competition, drug rebates, value-based
pricing, and much more. 1
Sen. Charles E. Grassley (R-IA) set the tone for the
tense, three-hour meeting when he opened with a declara-
tion that the practices of the pharmaceutical industry
“thwart the laws and regulations designed to promote
competition,” according to The New York Times. Reining
in high prescription drug prices is an issue with bipartisan
support, as evidenced by Sen. Robert Menendez’s (D-NJ)
warning to the CEOs, “If you don’t take meaningful action
to reduce prescription drug prices, policymakers are going
to do it for you.”
This hearing was just one of almost a dozen House
and Senate committee hearings on drug costs to occur in
2019. In April, executives from pharmacy benefit manag-
ers (PBMs) took their turn testifying before Congress,
fielding questions about the secrecy of their operations
and their role in driving up drug prices. 2
Perhaps the treatment that best epitomizes the im-
pact of such pricing is that for multiple myeloma (MM).
Several agents have been approved by the U.S. Food and
Drug Administration (FDA) in recent years, leading to
a proliferation of new, multi-agent treatment combina-
tions. Patients often are treated indefinitely, so their
health-care costs continue to add up.
Despite the fervor over this issue, Ronny Gal, a
ASHClinicalNews.org
securities analyst who follows the pharmaceutical indus-
try for Sanford Bernstein & Company, said he “doubted
that drug companies would change their pricing practices
because of the hearing.” 1
Like Mr. Gal, myeloma specialist Rafael Fonseca,
MD, chair of the department of medicine at Mayo Clinic
in Phoenix, was not surprised by the lack of progress,
telling ASH Clinical News that “there is no easy answer to
this issue.”
“That’s not to say that we shouldn’t be looking for one,
just that any proposal will come down to choosing be-
tween pros and cons and making trade-offs,” Dr. Fonseca
said. (Note: Dr. Fonseca reports that he has relationships
with Amgen, Bristol-Myers Squibb, Celgene, Takeda, and
Janssen, and he, along with the Mayo Clinic, hold a patent
for prognosticating myeloma using FISH.)
ASH Clinical News spoke with Dr. Fonseca and other
myeloma and drug-pricing experts about the high costs
of diagnosing and treating myeloma, how the pharma-
ceutical industry got to this point, and whether the
skyrocketing prices are justifiable.
Putting a Price on Survival
Prior to 1995, the cornerstone of MM therapy was the
oral regimen of melphalan plus prednisone. About
50% of patients responded to this treatment, and the
five-year survival rate was about 25%. 3 Between 1995
and 1996, data emerged about the clinical efficacy of a
slightly more expensive treatment, autologous hemato-
poietic cell transplantation, which, in eligible patients,
could increase five-year survival to about 50%.
It wasn’t until 2003, nearly a decade later that the FDA
approved the next agent for MM: bortezomib (Velcade). 4
In the 16 years since bortezomib’s approval, there
have been eight new FDA-approved drugs and indica-
tions for MM.
“We have gone from having two drug classes – the
proteasome inhibitors and immunomodulatory drugs
that were introduced in the 2000s – to now having
monoclonal antibodies, histone deacetylase inhibitors,
second-generation proteasome inhibitors, and second-
and third-generation immunomodulatory drugs,” said
Saad Usmani, MD, chief of Plasma Cell Disorders and
Director of Clinical Research in Hematologic Malignan-
cies at the Levine Cancer Institute in Charlotte, North
Carolina. “This wave of new approvals in the past decade
has resulted in multiple options and multiple combina-
tions for patients, especially in the early relapsed setting.
If you’re a myeloma clinician or patient, that’s a good
problem to have,” he added.
These newly approved drugs come in four broad
categories:
• proteasome inhibitors: bortezomib (Velcade,
manufactured by Takeda), carfilzomib (Kyprolis,
Onyx Pharmaceuticals), and ixazomib (Ninlaro,
Takeda)
• immunomodulatory drugs (IMIDs): lenalidomide
(Revlimid), pomalidomide (Pomalyst), and
thalidomide (Thalomid) – all manufactured by
Celgene
• monoclonal antibodies: daratumumab (Darzalex,
Janssen) and elotuzumab (Empliciti, Bristol-Myers
Squibb)
• other: the histone deacetylase inhibitor
panobinostat (Farydak, Novartis)
“Our discussions with patients a decade ago were very
different from what they are today,” Dr. Usmani noted.
“Compared with where it was in the late 1990s, the sur-
vival for myeloma has more than quadrupled.”
ASH Clinical News
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