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NCI Trims Operating
Expenses for 2019
Despite receiving a larger budget for fiscal year
2019 – a $179-million increase over the previ-
ous year – the National Cancer Institute (NCI)
plans to cut its operating expenses by 5 percent.
The agency’s budget was approved at $5.74
billion for 2019, representing the fourth largest
annual increase after more than a decade of
stagnation. However, NCI Director Norman
“Ned” Sharpless, MD, explained to Science
that various research priorities (such as the
National Cancer Moonshot Initiative) and an
ever-increasing number of grant applications
have stretched the budget.
For example, only $79 million of the 2019
increase went to NCI’s base operating budget,
while $100 million was set aside for the Moon-
shot Initiative.
younger than age 1 whose disease has had an in-
sufficient response to corticosteroids, immuno-
globulins, or splenectomy. Romiplostim initially
was approved for the treatment of adult patients
with ITP.
The decision to expand the drug’s indica-
tion was based on findings from two double-
blind, placebo-controlled clinical trials in
pediatric patients. In the first, patients with
refractory or relapsed ITP were randomized
2:1 to receive romiplostim (n=42) or placebo
(n=20). A greater proportion of romiplostim-
treated patients than placebo recipients
achieved a durable platelet response (at least 6
weekly platelet counts ≥50×10 9 /L during weeks
18 through 25 of treatment): 22 patients (52%)
vs. two patients (10%; p<0.05). Romiplostim
treatment also led to a greater number of and
more durable platelet responses, compared
with placebo.
In the second study, patients were ran-
domized 3:1 to receive romiplostim (n=17) or
placebo (n=5). Fifteen romiplostim-treated
participants (88%) achieved a platelet count
≥50x10 9 /L and an increase in platelet count
of ≥20×10 9 /L above baseline that lasted for
two consecutive weeks. No patient receiving
placebo achieved either endpoint.
In both trials, the most common adverse
events (AEs) in romiplostim-treated patients
included contusion, upper respiratory tract
infection, and oropharyngeal pain.
Romiplostim was approved with an
orphan-product designation.
Results from a randomized trial demonstrated
that calaspargase pegol-mknl administered
every three weeks had a similar safety
profile to pegaspargase administered every
two weeks.
Calaspargase pegol-mknl was approved
with orphan-product designation.
Source: FDA news release, December 20, 2018.
Cigna and Express Scripts
Seal $54 Billion Merger
Health insurer Cigna completed its $54 billion
acquisition of Express Scripts, which manages
the prescription plans of more than 80 million
Americans. The announcement continues a
trend of large health-care mergers and acquisi-
tions, following CVS Health’s takeover of Aetna
and Anthem’s plan to build its own pharmacy
benefits manager.
According to The Wall Street Journal,
Cigna CEO David Cordani said new initia-
tives will take advantage of the two compa-
nies’ combined access to medical and phar-
maceutical data. He also stated that, with the
new merger, the company will focus on cost
saving, transparency, and “alignment with
customers and other patients.” Prior to the
merger’s approval, Express Scripts unveiled
a plan that involved passing all rebates and
other payments from drug companies directly
to employers. Mr. Cordani remarked that the
arrangement was “indicative of what we’ll be
doing more together.”
Various research
priorities and a
growing number of
grant applications
have stretched the
National Cancer
Institute’s operating
FDA Approves
budget.
Calaspargase Pegol-mknl Cigna’s acquisition
of Express Scripts
In the past six years, Dr. Sharpless added, re-
for
Acute
Lymphocytic
search proposals sent to the NCI have increased
continues a trend
by 46 percent – faster than proposals at the Na-
Leukemia
tional Institutes of Health overall, which only saw
of large health-care
an 11-percent increase. He attributed this rise in
The FDA approved calaspargase pegol-mknl,
part to “a very exciting time” in cancer research.
an asparagine-specific enzyme, for the treat-
mergers in the U.S.,
All of NCI’s grant programs and intra-
ment of pediatric and young adult patients
mural laboratories – except its cancer centers,
(ages 1 month to 21 years) with acute lympho-
moonshot grants, and training awards – will
cytic leukemia (ALL). The agent was approved
which have been
be subject to cuts. Despite the changes, NCI
as part of a multi-agent chemotherapeutic
plans to continue its policy of prioritizing
regimen.
met with mixed
research by early-stage investigators.
The approval was based on pharmaco-
kinetic data demonstrating that calaspargase
reviews.
pegol-mknl achieved and maintained a nadir
Source: FDA news release, December 14, 2018.
Source: Science, December 7, 2018; Joint Meeting of the NCI Board of Scientific
Advisors (BSA) and the National Cancer Advisory Board, December 4, 2018.
FDA Expands
Romiplostim Indication
to Pediatric ITP
The U.S. Food and Drug Administration (FDA)
approved romiplostim for the treatment of
immune thrombocytopenia (ITP) in children
ASHClinicalNews.org
serum asparaginase activity above the level
of 0.1 U/mL in 124 patients with B-cell ALL.
The agent was delivered intravenously at doses
of 2,500 U/m 2 every three weeks. Compared
with other available pegaspargase products,
calaspargase pegol-mknl allows for a longer
interval between doses.
The most common grade ≥3 AEs
(occurring in ≥10% of participants) included
elevated transaminase, increased bilirubin,
pancreatitis, and abnormal clotting studies.
Takeovers like the Cigna-Express Scripts
deal have been met with mixed reviews. A
survey from the National Business Group on
Health earlier this year found that only 26
percent of employers were optimistic about the
mergers’ success, 56 percent were skeptical they
would lead to improvement, and 18 percent
worried they would raise costs. ●
Source: The Wall Street Journal, December 20, 2018.
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