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FDA Draft Guidance on Expedited Access PMA Outlines Opportunities for Genetic Tests, Companion Dx

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NEW YORK (GenomeWeb) ─ The US Food and Drug Administration this week issued a draft guidance proposing a program through which device manufacturers could gain expedited review and market entry through the agency for high risk medical devices that treat patients with needs unmet by current technologies.

By filing a so-called Expedited Access Premarket Approval application for tests for unmet medical needs or life threatening conditions, device manufacturers could have earlier and more interactive engagement with FDA officials and together develop a plan for gathering the clinical evidence supporting market approval of their tests. The agency in the draft guidance offers as examples certain types of genetic tests and companion diagnostics that might be eligible to be reviewed under an Expedited Access PMA.

"EAP is not a new pathway to market, but rather a collaborative approach to facilitate product development under the agency’s existing regulatory authorities," the agency stated in a release discussing the draft guidance. "While other existing device programs have focused on reducing the time for the premarket review, EAP also seeks to reduce the time associated with product development."

According to the draft document, manufacturers can submit EAPs for devices intended to treat a life threatening condition or a debilitating disease, and after the data development plan for a particular device has been approved by the FDA. Furthermore, the test has to meet one of the following criteria: address a condition or disease setting where no alternative treatment or diagnostic exists; operate based on a "breakthrough technology" that offers meaningful advantages over existing devices; and offer clinically meaningful advantages over other approved options. Finally, expedited review and market entry of the device must be deemed by the FDA to be in the best interest of patients.

In the draft guidance, the FDA offers an example of a breakthrough technology that can potentially be approved through an EAP: "A genetic test capable of identifying DNA variants using blood from patients may be less sensitive than standard testing of surgically removed tumor, or bone marrow, but offers patients a convenient, non-invasive sampling method."

In the draft guidance, the FDA also suggests that it is willing to be flexible in the EAP data requirements for certain types of devices, for example a genetic test that gauges common and uncommon variants. "A genetic test for both common and uncommon variants could be a candidate for the Expedited Access PMA if, for example, common variants are supported by robust, prospective clinical evaluation and uncommon variants are supported by initial clinical data with clinical justification for the expected performance range (e.g., confidence interval of positive predictive value)," the agency states in the guidance. "The post-approval study should collect additional data on the uncommon variants to provide greater clarity with respect to analytical and clinical performance."

Finally, companion diagnostics may also, under certain circumstances be eligible for EAPs. "It should be noted that certain companion diagnostics, when appropriate, and with consultation from [Center for Drug Evaluation and Research] or [Center for Biologics Evaluation and Research], may be considered for the Expedited Access PMA," the agency said. "For example, if a drug is reviewed via the accelerated drug approval pathway based on a surrogate endpoint, the companion diagnostic may be considered for the Expedited Access PMA."

The agency's guidance on EAPs is borne out of its experience with the Innovation Pathway pilot launched in 2011 and the FDA's accelerated approval and breakthrough therapy programs aimed at speeding development of innovative drugs to market. "When utilizing the EAP program, the FDA will continue to apply the current approval standard of demonstrating a reasonable assurance of safety and efficacy," the agency stated.

The agency is currently accepting public comments on the draft document.

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