Latest & Greatest
National Institutes of Health Awards
$55 Million to Launch Million-Person
Precision Medicine Study
The National Institutes of Health (NIH) awarded $55 million in funding for fiscal year 2016 to launch the Cohort Program of President
Obama’s Precision Medicine Initiative (PMI).
The awards will support partnerships for:
• Data and Research Support Center: Vanderbilt University Medical Center in Nashville, Tennessee, and the Broad Institute in
Cambridge, Massachusetts, will work with Verily Life Sciences
(formerly Google Life Sciences) to acquire, organize, and provide
secure access to the precision medicine research dataset. They will
also provide research support for the scientific data and analysis
tools for the program.
• Participant Technologies Center: Scripps Research Institute in San
Diego, California, and Vibrent Health in Fairfax, Virginia, will
develop, test, maintain, and upgrade the PMI Cohort Program mobile applications, which will be used to enroll, consent, collect data
from, and communicate with PMI Cohort Program participants.
• Healthcare Provider Organizations (HPOs): NIH will build a network of HPOs (including regional and national medical centers,
community health centers, and Veterans Affairs medical centers)
to ensure that PMI participants represent the geographic, ethnic,
racial, and socioeconomic diversity of the country. The regional
medical centers include:
• Columbia University Medical Center in New York City, NY
• Northwestern University in Chicago, IL
• University of Arizona in Tucson, AZ
• University of Pittsburgh in Pittsburgh, PA
In addition, six Federally Qualified Health Centers (FQHCs; community-based HPOs that reach underserved areas and populations) have
been selected for a pilot program to determine infrastructure needs.
Recipients include:
• Cherokee Health Systems in Knoxville, TN
• Community Health Center, Inc., in Middletown, CT
• Eau Claire Cooperative Health Center in Columbia, SC
• HRHCare, Peekskill in New York, NY
• Jackson-Hinds Comprehensive Health Center, Jackson, Mississippi
• San Ysidro Health Center in San Ysidro, CA
Earlier this year, the Mayo Clinic in Rochester, Minnesota, was tasked
with building the PMI Cohort Program Biobank, which will support
the collection, analyses, storage, and distribution for research use of
biospecimens.
With these partnerships, NIH expects to begin initial enrollment for
the PMI Cohort Program in 2016, with the goal of enrolling ≥1 million
U.S. participants by 2020.
FDA Issues New Draft
Guidance for the
“Appearance” of
Conflicts of Interest
The U.S. Food and Drug Administration (FDA) issued draft guidance for the
appearance of conflicts of interest with
regard to serving on one of its expert
advisory panels.
“Advisory committees provide
independent, expert advice to the FDA
on a range of issues affecting the public
health,” authors of the document wrote.
“To protect the credibility and integrity
of advisory committee advice, the FDA
screens advisory committee members
for current financial interests that may
create a recusal obligation under federal
conflict of interest laws.” This new draft
guidance covers “other interests and
relationships that do not create a recusal
obligation under federal conflict of interest laws but that may create the appearance that a member lacks impartiality,
known as ‘appearance issues.’”
Some new conflicts of interest contained in the guidance include:
• The panel member is a dean of a
medical school at a large university,
which receives a multi-year grant
from the drug manufacturer whose
product is under review. Regardless
of whether the grant is related to the
drug under review, “this is an interest or relationship that could cause
a reasonable person to question the
member’s impartiality.”
• The panel member has a relative or
someone living in the same household who has a relationship with a
person or entity appearing before
the panel.
• Those enforcing the guidance may
also look into past financial conflicts
rather than just current conflicts.
Of note, the draft guidance does not address “intellectual bias,” used to describe
a panel member who has a strong point
of view about a medical product.
FDA Grants Breakthrough Therapy
Designation for
Daratumumab for the
Treatment of Multiple
Myeloma
The U.S. Food and Drug Administration
(FDA) granted breakthrough therapy
designation for daratumumab in combination with lenalidomide and dexamethasone or bortezomib and dexamethasone
for the treatment of multiple myeloma in
patients who have received at least one
prior therapy.
The FDA’s decision was based on
the results of two phase III studies:
CASTOR and POLLUX. The CASTOR
trial evaluated daratumumab in combination with bortezomib and dexamethasone compared with bortezomib and
dexamethasone alone. Adding daratumumab to treatment resulted in a 61
percent reduction in the risk of disease
progression or death (hazard ratio [HR]
= 0.39; 95% CI 0.28-0.53; p<0.0001); the
median progression-free survival (PFS)
was not reached in the daratumumab
combination cohort, compared with 7.6
months in the bortezomib and dexamethasone alone cohort. The addition
of daratumumab also resulted in an increased overall response rate (ORR): 83
percent versus 63 percent (p<0.0001).
The POLLUX trial evaluated daratumumab in combination with lenalidomide and dexamethasone compared
with lenalidomide and dexamethasone.
The daratumumab combination therapy
resulted in a 63 percent reduction in
the risk of disease progression or death
compared with lenalidomide and dexamethasone (HR=0.37; 95% CI 0.27-0.52;
p<0.0001); the median PFS was not
reached in the daratumumab combination cohort compared with 18.4 months
for those who received lenalidomide and
dexamethasone alone. The addition of
daratumumab also significantly increased
ORR: 93 percent versus 76 percent
(p<0.0001).
Source: FDA news release, July 25, 2016.
Source: U.S. FDA, “Procedures for Evaluating Appearance Issues and Granting Authorizations for Participation in FDA Advisory Committees,” June 2016.
Source: National Institutes of Health news release, July 6, 2016.
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ASH Clinical News
September 2016