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Aug. 24 — The FDA wants to finalize guidances on advanced genetic screening tests “quickly and ideally some time next year,” the agency’s personalized medicine director said Aug. 24.
“But I can’t promise that,” Elizabeth Mansfield, a director in the Food and Drug Administration’s device center, quickly added during a summit on next-generation diagnostics. “In the meantime, if you want to bring in NGS [next-generation sequencing] tests, it would just be through the usual process.”
Mansfield was referring to two guidance documents the FDA Center for Devices and Radiological Health released in July that together represent how the agency proposes to oversee NGS tests, or advanced diagnostic tests that allow rapid sequencing of large segments of an individual’s DNA and even an individual’s entire genome (10 LSLR 14, 7/8/16).
These sophisticated tests, which are evolving rapidly, are considered critical to advancing the White House’s initiative on precision medicine, an emerging approach for disease treatment and prevention that seeks to maximize effectiveness by taking into account individual differences and lifestyle, environment and genes.
“The guidances are intended to encourage innovation by allowing the test developer to incorporate new things and test new clinical information rapidly while at the same time assuring the quality and reliability of the test,” Mansfield told audience members at the Next Generation DX Summit. “And we’d like to promote adoption of these tests into the community if they are good tests because we think this will really support the advance of precision medicine.”
The first draft guidance, Use of Standards in FDA Regulatory Oversight of Next Generation Sequencing (NGS)-Based In Vitro Diagnostics (IVDs) Used for Diagnosing Germline Diseases , provides recommendations for designing, developing and validating NGS-based tests for hereditary diseases.
“The draft guidance only applies to this particular intended use right now, but the principles will probably be more broadly applicable to other uses in the future,” she said.
The draft guidance describes specific approaches to test design, something the FDA doesn't always include in its guidance documents because design is usually covered under systems regulation, Mansfield said.
“But we really want people to think through as they’re designing these tests what they’re designing them for and how they’re going to validate them,” she said. “So we do describe the approach We believe that our approach accommodates a lot of different test designs.”
The draft guidance also addresses how FDA-recognized standards could be used to demonstrate analytical validity, and how the FDA would like a widely recognized body such as the International Organization for Standardization to develop standards that the agency can recognize.
“As science changes, as technology changes and so on, you need to change your recommendations over time to keep up with it,” Mansfield said. “FDA’s ability to do that through regulation is actually kind of slow and administratively intense.”
Because the rulemaking process takes a long time, sometimes the final rule is “not so relevant anymore” by the time the FDA releases it, she said. “Standards organizations can do this much more quickly.”
Another important quality about moving through a standard-developing organization is that the organization must issue standards based on consensus, she said.
“It can’t just represent one sector. It has to be everybody coming together,” Mansfield said. “FDA can recognize that standard as, ‘We agree with that.'”
The second draft guidance, Use of Public Human Genetic Variant Databases to Support Clinical Validity for Next Generation Sequencing (NGS)-Based In Vitro Diagnostics , would allow test developers to cite valid scientific evidence from FDA-recognized databases.
This database draft guidance is a little more straightforward than the standards one, she said. “What we're trying to do—to use a buzzword—is to crowdsource evidence,” Mansfield said.
“We should be moving more in this direction as a society where we’re aggregating evidence,” Mansfield said. “The advantage of this is that the evidence is generated by multiple parties. And as we aggregate evidence, we get a stronger evidence base.”
Mansfield said comments on both draft guidance documents are due Oct. 6. The FDA also is holding a public workshop Sept. 23 on adapting regulatory oversight of NGS-based tests in Bethesda, Md.
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