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FDA to Release Several Guidance Documents Aimed at Streamlining Dx, PGx-Based Theranostic Development


The US Food and Drug Administration plans to release several guidance documents over the next few months in order to help streamline the process of developing molecular diagnostics and pharmacogenomic-based companion products.

The FDA said it has been seeing an increase in the number of requests from sponsors seeking pre-submission meetings and advice on the best ways to test technologies such as genetic and proteomic diagnostics and therapeutics, the FDA Center for Devices and Radiological Health said in a statement this week. The center hopes to address its part of that need.

In an attempt to answer some of these questions and eventually help pave the way for speedier and less costly diagnostic review procedures, the agency has created what it calls the Medical Device Innovation Initiative. CDRH said the initiative aims to "promote scientific innovation" in product development, "focus device research on cutting-edge science," and "modernize" device review.

The effort involves the release of several guidance documents this year from CDRH and other centers, but the FDA is not ready to release tentative titles or release dates, according to a center spokesperson. One such guidance released this week lays out the use of Bayesian statistical analysis methods for clinical trials. These methods should allow developers to make "use [of] existing information in an efficient and scientifically rigorous manner."

In a statement last week, CDRH said the initiative aims to "promote scientific innovation" in product development, "focus device research on cutting-edge science," and "modernize" device review.

Clarifying PGx, Theranostics, ASRs

As part of the initiative, CDRH expects to produce a guidance document on genetic and pharmacogenomic tests this year, in addition to documents related to more traditional medical devices.

The center also plans to release an intercenter guidance document that will aim to "outline scientific, clinical and technical issues that should be considered early in the development process" for combination products. This document would aim to explain how sponsors should collect, analyze, and submit information on drugs, devices, or biological constituents, and how to ensure that they are safe and effective.

Additionally, CDRH and other unnamed FDA centers plan to release several documents to give product developers "regulatory clarity" and flexibility. For example, CDRH plans to release a guidance aiming to clarify regulatory requirements for analyte specific reagents.

CDRH also said it plans to help the agency's Critical Path initiative by identifying "barriers" to biomarker development for diagnostics and pharmacogenomic devices.

Lastly, CDRH said it is "stepping up" its interactions with industry, patients, and clinicians to fuel innovation, partly through workshops and advisory panel meetings. The center did not specify what sort of innovation it was hoping to spur.

Updating Device Review

To be sure, it was not immediately clear exactly how CDRH plans to accelerate the clearance of biomarker-based diagnostic technologies. But in examples outlined in the Medical Device Initiative statement, its scientists have in the past helped device developers by suggesting approaches that require cost- and time-saving testing for safety and effectiveness. In one example, FDA scientists suggested the use of a fluid-flow model to bypass the need for animal testing of a redesigned stent, but the center intends to apply new approaches to a broad spectrum of areas.

As part of the Medical Device Initiative, CDRH said it will examine its scientific review process in order to help its staff hasten diagnostic clearance, as well as help developers understand what CDRH requires for new products. So far, the center has conducted reviews in biocompatibility, sterilization, and statistical analysis, and it plans to approach software validation next.

In premarket submission reviewing, CDRH has begun a system based on milestones to ensure "predictability and accountability."

With its in-house information technology, the center plans to spot potential adverse events and warn doctors and patients quickly, as well as continue to gather information about approved devices, including molecular diagnostics. One of its IT initiatives involves putting in place a tracking system to monitor workload and delegate scientific expertise. Also, the center will put in place electronic submission procedures for IVDs, as well as a web-based program to allow information sharing and communication for team members working on PMAs.

The initiative's device review section includes the guidance on Bayesian statistical approaches to clinical trial design.

— Chris Womack ([email protected])

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