An expert panel focused on the need for more data, standardization, and
accurate marketing during a session discussing the implications of
molecular testing on practice and policy at the National Comprehensive
Cancer Network® (NCCN®) 16th Annual
HOLLYWOOD, Fla.--(BUSINESS WIRE)--
In recent years, an increasing number of tests that use molecular
genetics to assess the potential efficacy of cancer therapies in
individual patients have become available. The introduction of these new
tests in a variety of clinical settings has brought with it a spectrum
of new challenges related to measuring the validity and value of these
tests, and translating them into clinical practice.
An expert panel at the NCCN
16th Annual Conference discussed these challenges and called
for higher standards in regulating how and where the tests are done,
better data to determine their value and cost effectiveness, and new
approaches to determining their optimal uses for today’s patient
Clifford Goodman, PhD, of the Lewin Group, moderated the panel, which
included: Scott Gottlieb, MD, American Enterprise Institute; Louis B.
Jacques, MD, Centers for Medicare & Medicaid Services; Michael
Kolodziej, MD, Innovent Oncology; Mark G. Kris, MD, Memorial
Sloan-Kettering Cancer Center; Lee N. Newcomer, MD, MHA, UnitedHealth
Group; Andrew C. von Eschenbach, MD, formerly of the National Cancer
Institute and U.S. Food and Drug Administration (FDA), currently with
Samaritan Health Initiatives, Inc.; and Elizabeth Thompson, Susan G.
Komen for the Cure®.
Dr. Goodman challenged the panel to consider the issue of molecular
testing from four perspectives; regulatory responsibility, evidence that
a test “works,” translation into everyday practice, and value.
Dr. Gottlieb pointed out that molecular tests are currently regulated by
how they are marketed, not by claims for what the test can do, and that
there is enormous heterogeneity in how and where the tests are
performed. He supports having the FDA or Clinical Laboratory Improvement
Amendments (CLIA), regulate the analytic validity of molecular testing
while leaving decisions about clinical validity to the clinical
Dr. von Eschenbach agreed that there was a great need to assure that
tests are consistent in their claims and results regardless of where
they are performed and conceded that presently, this is not always the
case. He argued for a system that would allow the FDA to develop the
infrastructure necessary to, in his words, “bring order out of chaos.”
Dr. Jacques, described the field of molecular testing as immature, and
predicted that it would take a number of years and additional experience
before it became possible to assess either the true analytic or clinical
validity of the growing number of molecular tests.
Dr. Newcomer discussed the challenge of working within a juvenile system
particularly when trying to assess the quality of the test being done or
its clinical utility. He explained that even with one of the best
established tests, the HER2 gene test for breast cancer, there is a high
percentage of inaccurate or misused tests in some settings. He added
that the system used to code procedures for insurance payments is also
outdated and does not specify what molecular test is being performed.
“The coding is so antiquated that we don’t know what we are paying for.
It just says ‘genetic test,’ which doesn’t allow us to assess either the
upfront costs or the downstream benefits that might result from this
kind of testing,” said Dr. Newcomer.
Dr. Kris, who specializes in treating lung cancer, noted that the EGFR
test that is used to predict the efficacy of a specific type of
chemotherapy in patients with advanced adenocarcinoma of the lung has
been proven to be both valid and useful. Properly done, the test can
identify patients who have the genetic mutation and the potential to
benefit from chemotherapy while sparing those who don’t from ineffective
“It is our job as clinical researchers to provide the data that
regulators and payors need to make decisions,” said Dr. Kris.
Michael Kolodziej, MD, agreed with Dr. Kris, but in his role as a
community oncologist specializing in lung cancer sees obstacles to
utilizing even established molecular tests in his practice. He noted
that some tests do not result in treatments that make a clear difference
in patient survival, and that there are often difficulties and delays in
obtaining tissue specimens needed to perform the molecular tests.
“We are aspiring to an era of personalized medicine, but we aren’t there
yet,” said Dr. Kolodziej.
From the patient perspective, Liz Thompson explained that, these tests
are “confusing and challenging.” She added that educated patients are
aware of the tests and frequently ask their doctors about them, but
still have trouble evaluating their proper use or understanding how they
apply to their specific situations.
Despite viewing the issues related to how best to use and pay for this
emerging field of medicine, the panel agreed that there was a need for
better regulation, more data, and improved methods of making the tests
accessible to patients across the spectrum of clinical settings.
Dr. von Eschenbach summarized these views stating, “These tests are
becoming mechanisms for saving money and improving outcomes for our
patients. We need to make sure we have the mechanisms in place to make
decisions about what is the right treatment, at the right dose, done for
the right reason.”
About the National Comprehensive Cancer Network
The National Comprehensive Cancer Network (NCCN), a not-for-profit
alliance of 21 of the world’s leading cancer centers, is dedicated to
improving the quality and effectiveness of care provided to patients
with cancer. Through the leadership and expertise of clinical
professionals at NCCN Member Institutions, NCCN develops resources that
present valuable information to the numerous stakeholders in the health
care delivery system. As the arbiter of high-quality cancer care, NCCN
promotes the importance of continuous quality improvement and recognizes
the significance of creating clinical practice guidelines appropriate
for use by patients, clinicians, and other health care decision-makers.
The primary goal of all NCCN initiatives is to improve the quality,
effectiveness, and efficiency of oncology practice so patients can live
The NCCN Member Institutions are: City of Hope Comprehensive Cancer
Center, Los Angeles, CA; Dana-Farber/Brigham and Women’s Cancer Center |
Massachusetts General Hospital Cancer Center, Boston, MA; Duke Cancer
Institute, Durham, NC; Fox Chase Cancer Center, Philadelphia, PA;
Huntsman Cancer Institute at the University of Utah, Salt Lake City, UT;
Fred Hutchinson Cancer Research Center/Seattle Cancer Care Alliance,
Seattle, WA; The Sidney Kimmel Comprehensive Cancer Center at Johns
Hopkins, Baltimore, MD; Robert H. Lurie Comprehensive Cancer Center of
Northwestern University, Chicago, IL; Memorial Sloan-Kettering Cancer
Center, New York, NY; H. Lee Moffitt Cancer Center & Research Institute,
Tampa, FL; The Ohio State University Comprehensive Cancer Center - James
Cancer Hospital and Solove Research Institute, Columbus, OH; Roswell
Park Cancer Institute, Buffalo, NY; Siteman Cancer Center at
Barnes-Jewish Hospital and Washington University School of Medicine, St.
Louis, MO; St. Jude Children’s Research Hospital/University of Tennessee
Cancer Institute, Memphis, TN; Stanford Comprehensive Cancer Center,
Stanford, CA; University of Alabama at Birmingham Comprehensive Cancer
Center, Birmingham, AL; UCSF Helen Diller Family Comprehensive Cancer
Center, San Francisco, CA; University of Michigan Comprehensive Cancer
Center, Ann Arbor, MI; UNMC Eppley Cancer Center at The Nebraska Medical
Center, Omaha, NE; The University of Texas MD Anderson Cancer Center,
Houston, TX; and Vanderbilt-Ingram Cancer Center, Nashville, TN.
Clinicians, visit NCCN.org.
Patients and caregivers, visit NCCN.com.
NCCNMegan Martin, 215-690-0576[email protected]
Source: National Comprehensive Cancer Network (NCCN)