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FDA Intends to Make Autosomal Recessive Carrier Screening Exempt

By Diagnostics World Staff

October 27, 2015 | Today the Food and Drug Administration announced its intent to make autosomal recessive carrier screening exempt from premarket regulatory requirements. The notice was posted today online in the Federal Register and will be open for comment until November 27.

In February, FDA approved 23andMe’s carrier screening test for Bloom Syndrome, a rare, autosomal recessive disease. FDA classified the test, and substantially equivalent devices of this generic type, as class II device under the generic name “autosomal recessive carrier screening gene mutation detection system.” Now the 23andMe Bloom Syndrome approval will serve as the de novo approval for other gene-based carrier screening tests.

FDA listed 39 conditions including Bloom Syndrome, Tay Sachs Disease, Sickle Cell Anemia, and Phenylketonuria for which genetic carrier tests would qualify for the exemption

The FDA has deemed these tests safe because carrier status in itself has no health implications for the patient, though it could, of course, mean something for the patient’s child. FDA believes the risk to patients’ health and wellbeing is extremely low for either false positive or false negative findings. True positive results are equally harmless to the patient.

There are some caveats. Test makers are required to publish details of their test’s clinical validity on their public websites through either peer reviewed journal articles, authoritative summaries of the literature, or professional society recommendations. “If there is no professional guideline recommending testing of a certain gene or variant in the indicated population, the manufacturer's Web site must warn that no such recommendation currently exists.”

Test makers are also required to disclose any limitations to coverage that would result in any carriers receiving false negative results. The notice spells out warning language that would be required of test makers to let consumers know if the disease has other genetic underpinnings that are not being tested, or if the disease is believed to have non-heritable components. And specific statements are required to explain to consumers the probability of test failure and a description of scenarios in which a test can fail.

The exemption does not extend to the collection devices—for example the spit kit that 23andMe uses to collect sample or Theranos’s nanotainer tube—and carrier tests under the exemption must use collection devices that are already cleared, approved, or classified as exempt on their own.