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 populations.
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 community.
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 therapy.
“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.”
Source: National Comprehensive Cancer Network