CLINICAL NEWS
with daratumumab monotherapy.
The objective response rate was 29
percent (95% CI 21-39%) with a median response duration of 7.4 months
(range = 1.2-13.1 months).
The most common treatmentrelated adverse events (occurring in
≥20% of patients) included infusion
reactions, fatigue, nausea, back pain,
pyrexia, cough, and upper respiratory tract infection. In addition, the
most common laboratory abnormalities were lymphopenia, neutropenia, anemia, and thrombocytopenia.
In its news release, the FDA
noted that blood banks should be
informed that patients are receiving daratumumab because the drug
may interfere with certain tests that
are done by blood banks (such as
antibody screening) for patients who
need a blood transfusion.
Daratumumab was approved
under the FDA’s accelerated approval program, which allows the
approval of a drug to treat a serious
or life-threatening disease based on
clinical data showing the drug has
an effect on a surrogate endpoint
reasonably likely to predict clinical
benefit to patients. As a condition of
approval, Janssen Biotech, Inc., the
drug’s manufacturer, is required by
the FDA to perform a multicenter,
randomized trial establishing the
superiority of daratumumab over
standard therapy to verify and describe the drug’s clinical benefit.
Source: U.S. FDA press release, November 16, 2015.
FDA Approves Modified Antihemophilic
Factor for the Treatment of Hemophilia A
The U.S. FDA approved antihemophilic factor (recombinant),
PEGylated for use in patients 12
years old or older with hemophilia
A. The new drug is approved for
on-demand treatment and control
of bleeding episodes, as well as to
reduce the frequency of bleeding
episodes. The drug is a modified
version of recombinant factor VIII,
with an extended circulating halflife to last longer in the blood and
potentially require less frequent
injections than unmodified antihemophilic factor when used to reduce
the frequency of bleeding.
The approval of antihemophilic
factor (recombinant), PEGylated was
based on the results of a prospective,
multicenter, open-label, non-randomized, phase III clinical trial of
137 adults and adolescents with he-
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mophilia A. Patients were assigned
to either twice-weekly prophylaxis
(40-50 IU/kg; n=120) or on-demand
treatment (10-60 IU/kg; n=17) with
the modified factor.
Previously treated patients in the
twice-weekly prophylaxis arm had 95
percent fewer annual bleeds compared
with those treated on-demand (median annual bleed rate = 1.9 vs. 41.5,
respectively). During the study, 38
percent of prophylaxis-treated patients
experienced zero bleeds. Moreover, 57
percent of patients experienced zero
joint bleeds based on six months of
prophylaxis. No safety concerns were
identified during the trial.
Source: U.S. Food and Drug Administration press release,
November 13, 2015.
Medicare Proposes
Coverage of
Hematopoietic
Cell Transplant
for Patients with
Sickle Cell Disease,
Myelofibrosis, and
Multiple Myeloma
The Centers for Medicare & Medicaid Services (CMS) released its
new proposed coverage policy for
hematopoietic cell transplantation
(HCT) among Medicare patients.
The proposal was a response to
lobbying earlier this year from the
National Marrow Donor Program
and the American Society for Blood
and Marrow Transplantation to include HSCT in the national coverage
determination for sickle cell disease
(SCD), myelofibrosis, and multiple
myeloma (MM).
Prior to this action, most Medicare services do not have a national
coverage determination for HCT
that includes SCD and myelofibrosis,
meaning patients with these conditions can be found financially liable
for HCT if a local medical director
deemed the procedure medically
unnecessary. In addition, Medicare does not currently cover HCT
among patients with MM, regardless
of the local region.
This proposed change would add
those three conditions to covered
status under the process known as
“coverage with evidence development.” This process is used by Medicare when the agency finds that the
scientific evidence of a procedure’s
efficacy is lacking in a particular
population. Medicare agrees to pay
for the service in exchange for the
services’ providers gathering data,
often using a registry.
CMS is currently seeking comments on the proposed decision.
Source: Centers for Medicaid & Medicare Services. “Proposed
decision memo for stem cell transplantation (multiple myeloma, myelofibrosis, and sickle cell disease) (CAG-00444R),”
October 29, 2015.
Medicare Releases
Final Rules on
Physician and Outpatient Hospital
Payment for 2016
CMS announced final rules for physician and outpatient hospital payments, which will go into effect on
January 1, 2016. Payment rules for
the 2016 calendar year for End-Stage
Renal Disease Prospective Payment
System, the Hospital Outpatient
Prospective Payment System, Home
Health Prospective Payment System,
and the Physician Fee Schedule were
finalized at the end of October.
This is the first year that the Medicare physician fee schedule was not
tied to the Sustainable Growth Rate
(SGR) calculation, which was replaced
by the Medicare and Children’s Health
Insurance Program (CHIP) Reauthorization Act (MACRA) in April 2015.
As a result, the overall payment rate
for Medicare in 2016 is nearly identical to the overall payment rate for the
second half of 2015.
“CMS is pleased to implement the
first fee schedule since Congress acted
to improve patient