By Diagnostics World Staff
April 13, 2018 | Yesterday, FDA Commissioner Scott Gottlieb announced via Twitter new policies for the Administration he says are designed to “keep pace with fast-moving [next-generation sequencing] technologies.” This announcement coincides with a speech Gottlieb gave earlier that day at the 2018 Community Oncology Conference.
“When it comes to complex, serious, and life-threatening diseases like cancer, promoting and protecting public health requires the FDA to adopt policies to ensure that regulation serves as a bridge to this oncology innovation, and not a barrier to it,” Gottlieb said in his prepared remarks.
Three guidances being released today, according to Gottlieb, focus solely on NGS and in vitro diagnostic development. Gottlieb is optimistic that these guidances will “encourage further development of these powerful diagnostic platforms by enabling more efficient regulatory review.”
The first guidance, titled “Considerations for Design, Development, and Analytical Validation of NGS-Based In Vitro Diagnostics Intended to Aid in the Diagnosis of Suspected Germline Diseases,” will focus on the analytic validation of NGS-based tests designed to help diagnose suspected germline diseases.
“The FDA believes that the analytical validation recommendations laid out in this guidance could spur the creation of consensus standards for NGS-based tests that will be developed by the community and potentially recognized by the FDA,” Gottlieb said.
The second guidance is called “Use of Public Human Genetic Variant Databases to Support Clinical Validity for Genetic and Genomic-Based In Vitro Diagnostics,” describing the Administration’s considerations for genetic variant databases that are publicly accessible as valid scientific evidence to support clinical validity during premarket review.
Gottlieb cites the NIH’s ClinGen as an example of such a database, saying that ClinGen works to standardize a genomic variant’s clinical annotation and interpretation through evidence-based expert consensus.
“Eventually, we believe that this guidance will encourage expert-based crowd sourcing of NGS evidence generation, curating, and data sharing – which can all advance the development of high quality precision medicine treatments and diagnostics,” said Gottlieb.
The third proposed guidance is a draft guidance, Gottlieb said, titled, “Investigational In Vitro Diagnostics in Oncology Trials: Streamlined Submission Process for Study Risk Determination.” This guidance is directed towards certain oncology trial sponsors, offering them a streamlined submission process related to “determining whether use of an investigational in vitro diagnostic (IVD) -- including IVDs that incorporate NGS technology -- in a trial of investigational cancer drug or biological products is considered significant risk, nonsignificant risk, or exempt from review.”
This final guidance is, Gottlieb says, a move toward the FDA’s goal of having a common filing for a drug and diagnostic system where the drug is co-developed with a diagnostic test.
“Taken together, these guidances pave the way for possible down classification and even exemption from premarket review of NGS-based tests that demonstrate conformity with future FDA-recognized standards and tests that rely on demonstrating their validity against FDA-recognized databases,” Gottlieb said. “Most importantly, it will give patients, payers, researchers, and clinicians greater confidence that NGS platforms can be used to inform treatment decisions and improve patient outcomes.”