CLINICAL NEWS
The agency’s decision was based
on the results of the phase III RATIFY
clinical trial, the results of which were
presented during a plenary session at
the 2015 ASH Annual Meeting. The
study found that patients treated with
midostaurin and standard induction and
consolidation chemotherapy had a significant improvement in overall survival
(OS), compared with those who received
standard induction and consolidation
chemotherapy alone (hazard ratio =
0.77; p=0.0074). The median OS for the
midostaurin treatment cohort was 74.7
months (95% CI 31.7-not reached) compared with 25.6 months for the standard
treatment group (95% CI 18.6-42.9).
No statistically significant differences
were observed between the treatment
groups in terms of grade 3 or higher adverse events. Thirty-seven patient deaths
were recorded, though no differences
were observed between patient cohorts.
Source: Novartis press release, February 19, 2016.
White House, NIH
Announce Progress
in Precision Medicine
Projects
The White House and the National
Institutes of Health (NIH) announced
that enrollment of volunteers into the
Precision Medicine Initiative (PMI)
Cohort Program has begun. The PMI
Cohort program is a long-term health
study that will examine genetics, lifestyle factors, and health in an effort to
propel precision medicine and identify
cures. The PMI Cohort Program is one
of several pilot projects announced
recently by the NIH, and the largest
component of the PMI.
The NIH wants to enroll the first cohort of 79,000 participants into the precision medicine database by the end of
2016, with a projected total enrollment of
1 million participants by 2019. The NIH
provided funding to Vanderbilt University in Nashville, Tennessee, to perform a
pilot study of direct recruitment initiatives, including a participant website and
phone line. Verily, a Google subsidy, will
provide technical help on this project.
The NIH also pledged to work with federally funded community health centers
to encourage cohort volunteers from
underserved populations.
“For most of history, medicine has
been based on trying to identify what
works for the average person,” said Francis Collins, MD, PhD, the director of
the NIH, in a press conference. “We’re all
different. This one-size-fits-all approach
is far from optimal.”
The goal of the PMI Cohort Program and recruitment initiatives is to
ASHClinicalNews.org
“empower any person, anywhere in the
United States, to raise their hand and
volunteer to participate.”
This program is projected to cost more
than $1 billion for the first four years.
Sources: NIH press release, February 25, 2016; Science, “NIH’s 1-millionvolunteer precision medicine study announces first pilot projects,”
February 25, 2016.
Report Questions
the Validity of
Rivaroxaban Trial
An investigation by the British Journal
of Medicine (BMJ) has raised questions
about the validity of the pivotal ROCKETAF trial, the results of which formed the
basis of rivaroxaban’s approval. ROCKETAF trial results were published in The New
England Journal of Medicine in 2011. The
study, which included more than 14,000
patients and took place between 2006 and
2010, found the novel oral anticoagulant
rivaroxaban to be non-inferior to warfarin
for the prevention of stroke or em &