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FDA Outlines Regulatory Science Efforts for Medical Devices

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Originally published Oct. 3.

A year after launching an initiative to improve its policies and practices through regulatory science, the US Food and Drug Administration has issued a report outlining its efforts to use this approach to improve the safety and effectiveness of medical devices, including genomic advances that are helping to personalize healthcare.

The FDA is currently in the process of instituting regulatory science initiatives throughout its various divisions to ensure that it can establish the safety and efficacy of technological advances that bring new innovations to healthcare but also present new oversight challenges. The agency outlined its agency-wide plan for implementing regulatory science — defined as "the science of developing new tools, standards, and approaches to assess the safety, efficacy, quality, and performance of FDA-regulated products" — in a report last year (PGx Reporter 10/6/2010).

Since her confirmation last year, FDA Commissioner Margaret Hamburg has publicly stated on several occasions that she intends to improve the agency's scientific and regulatory expertise to usher in more innovative treatments and advanced technologies.

"New trends in clinical care will create markets for new technologies," the FDA states in the latest report, "Regulatory Sciences in FDA's Center for Devices and Radiological Health."

It adds that "as personalized medicine continues to advance, new assessment tools will be needed, including biomarkers, genomic and proteomic detection arrays, computer-assisted diagnostic tools, and computational models like the virtual patient."

Genomic advances were listed alongside other types of device innovations, such as percutaneously deliverable implants, robotic surgical systems, and advanced imaging methods. "It will be vital for us to keep up with these kinds of changes, and to develop the needed assessment methods," the agency noted. "This will enable the deployment of important new technologies quickly and cost-effectively" and will also "give the public access to new technologies."

In applying regulatory science initiatives, the agency hopes to improve device quality and manufacturing; better analyze medical device performance; bolster capabilities to address new safety questions and stop device-related adverse events before they occur; develop new ways to use clinical data to evaluate medical devices; protect people against emerging infectious diseases and bioterrorism; and address the needs of pediatric and other special populations.

In the personalized medicine field, the agency has recently issued a number of new guidelines and is planning others as it anticipates greater integration of molecular diagnostics within the drug approval process.

The FDA recently issued draft guidelines on the marketing of tests labeled research-use-only and another draft document outlining regulatory considerations when developing companion diagnostics (PGx Reporter 6/08/2011; 7/13/2011). Additionally, the agency plans to release three guidance documents explaining its regulatory plans for laboratory-developed tests (PGx Reporter 7/20/2011).

On the regulatory science front, one of the major challenges the agency will face is developing procedures for reviewing and approving diagnostics based on whole-genome sequencing technologies.

Although most readily used as a research tool, the FDA acknowledges in the new report that whole-genome sequencing is "nearing clinical use." Since "technologies for accessing a patient’s full genomic sequence are now under intensive development," this "necessitate[s] the development and validation of reliable tools to characterize these products and assure that they are accurate and appropriate," the report states.

According to the report, the agency is collaborating with other government agencies, academia, and industry to advance its understanding of WGS. Toward this end, the FDA earlier this year held a one-day workshop to discuss next-generation sequencing in clinical diagnostic applications, but has set no timeline for when — or whether — it intends to issue guidance on the matter (CSN 6/29/2011).

Short of whole-genome sequencing, the FDA is somewhat further along in developing validation methods other types of complex molecular diagnostics, such as microarray-based technologies. The agency has "evaluated the ability of manufacturers to validate the performance of their microarray technologies in treating breast cancer, multiple myeloma, and neuroblastoma," the agency said in the report. "These efforts have identified ways to sharpen the evaluation of microarray technologies, which should increase the reliability of this new medical tool."

Molecularly guided patient stratification to identify best responder population for drugs for serious illnesses is another area where the FDA is focusing on improving its regulatory capabilities. "Current methods for measuring the effect of chemotherapy on breast cancer tissue have often been inadequate to predict how the tumor will respond," the agency states in the report. "To help address this problem, we’re developing new analytical tools using multiplex protein arrays that can rapidly monitor tumor response."

Such analytical tools will "aid in selecting the right drug for the right patient," the agency added. "Our goal is to better personalize breast cancer treatment and reduce the patient’s exposure to ineffective and potentially toxic therapies."

The agency is also working on developing a so-called Library of Models, which will house publicly accessible, interoperable computer simulations of human physiology in different disease states. Such computer modeling and simulation is critical to advancing personalized medicine, according to the FDA.

"Personalized medicine requires more than a personalized genome, it requires personalized functional anatomy," the agency states in the report. "We plan to make this Virtual Physiological Patient accessible to researchers and medical device developers for testing new device designs and applying for device clearance and approval."

Lastly, biomarker development will also be a key part of improving regulatory efforts at the agency for devices.

"Biomarker development is needed to: detect damage to organs; develop approaches to predict the toxicity of compounds released from devices; evaluate the interactive effects of compounds released from devices; and characterize the exposure of patients to compounds released from medical devices," the FDA notes in the report. "As CDRH continues to qualify the use of biomarkers in clinical settings, we will also strive to develop new biomarkers to aid in determining the safety and effectiveness of medical devices."

In line with FDA's efforts to improve its knowledge of advanced, emerging technologies, the agency is seeking public input on a plan to create a network of outside scientific experts who would provide FDA staff with "specialized knowledge about emerging technology." Members of the public can comment on the plan for developing this Network of Experts until Oct. 28. CDRH is conducting a 12-week pilot of the network from Sept. 30 to Dec. 30.

Unlike experts on FDA's advisory committees, members of CDRH's Network of Experts will not advise the agency on policy matters. "Instead, network members will share their particular expertise on specific topics to help center staff form their own conclusions," the agency said in a statement.

In a document outlining the ways in which the experts may be used by the agency, the agency cites "current technical limitations in whole genome sequencing technology," as one of the potential topics on which network members may be asked to provide input.

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