Scott Gottlieb, MD, asked the participating panelists their opinion on whether FDA regulation of laboratory diagnostics tests (LDTs) would impact payer response or consumers adoption.
John Fox, MD, MHA, believes payers are concerned with the clinical utility of the test rather than the approval process itself. “If the FDA says that a company cannot market a test without FDA approval, it’s a done deal, we will not cover the test,” he said. Dr Fox explained that multiple tests further complicate decisions. If there are 3 different manufacturers of tests that look at risk of recurrent breast cancer that have different genes, and data on analytic and clinical validity, “how do we decide, how do consumers decide, how do providers decide which ones are more useful in improving patient outcomes?”
He added that loopholes in the law allow academic institutions and hospitals to provide LDTs as a service to their patients.
Dr Gottlieb then drew the panel to discuss multi-varied tests that generate genomic and proteomic profiles, which can then be correlated with outcomes such as response to chemotherapy. Indicating that several people believe these tests that are hard for physicians to decouple and difficult to replicate, fall “outside of the CLIA framework.”
Dr Patel agreed. When CLIA was launched in 1988, the administrations had instilled hope in the predictive abilities of these tests, especially for cancer. “So falling outside of this is exactly the kind of tension spot, which is why I mentioned cancer testing, autism spectrum disorder testing which rely on those variables.” She firmly believes the FDA will have to decide on the impact of their regulatory reach on the marketplace.
Some of these tests, with proven utility, have a consumer reach because they are outside of FDA regulation and do not incur administrative costs. “So, I think we are talking about attention to how fast, how soon to market, and what really is the market and what is the reach for the administration,” Dr Patel said. She believes the FDA will be pressured to act on the report that they released end of 2015 that underscores the need to broaden their regulatory reach to LDTs.
Establishing analytic and clinical validity is much easier with single gene assays rather than multiple gene assays, which require whole-exome sequencing or whole genome sequencing, said Dr Fox. However, “How do you establish clinical validity when you have no idea what these mutations signify?” Developing a database that will help us identify rare, novel mutations that are associated with disease would be the way to go, he believes, in order to make sense of the tremendous amount of genomic and proteomic data that the tests are expected to generate.
Dr Gottlieb clarified the FDA’s stance, saying they do not want to regulate decisions on the clinical utility of a test. “The FDA will only certify that the test reports to measure a certain output.” Decisions on clinical utility will be left to the prescribing physician, Dr Gottlieb added.
Alluding to President Obama’s Precision Medicine Initiative, which has a goal of creating a library of a million different genome patterns, Dr Fox thinks the data would still fall short. “I think payers like the fact that it’s in the market because there are a lot of claims being made, but I think the biggest challenge will be how we establish the clinical utility and validity of these new findings.”