FDA Issues Final Guidance on Benefit-Risk Assessment for Clinical Studies of Devices

Wednesday, January 25, 2017

Source: AAMI

A new guidance document from the Food and Drug Administration (FDA) seeks to clarify the agency’s assessment of benefits and risks for medical device applications involving human clinical studies.

Applications for what is called an investigational device exemption or IDE are generally required to determine the safety of a “significant risk” medical device before it can enter human clinical trials, according to the FDA.

The final guidance is intended to improve “predictability, consistency, and transparency” for the IDE review process, the FDA wrote in the document, Factors to Consider When Making Benefit-Risk Determinations for Medical Device Investigational Device Exemptions. “FDA believes use of this benefit-risk framework will facilitate the incorporation of evidence and knowledge from different domains—clinical, nonclinical, and patient—to support a comprehensive, balanced decision-making approach.”

The FDA explained that its assessments are tailored to the stage of the device’s development. Devices that are earlier in development are likely to contain more uncertainty, or less evidence, compared to devices that are further along. For early-stage devices, the FDA focuses more on whether appropriate risk control measures are in place to account for both anticipated and unanticipated risks, as well as to assess safety and proof of concept. Studies for later stages devices are typically evaluated to determine device safety and outcomes for the proposed population.

“IDE benefit-risk assessments should take into consideration whether the level of uncertainty is appropriate to the stage of development for the investigational device,” the FDA wrote. “For IDE benefit-risk determinations throughout all stages of device development, it is also important to recognize that non-clinical and prior clinical data play a critical role.”

When applying a benefit-risk framework to IDE application decision, the FDA’s assessment considers both anticipated benefits for patients as well as the benefits to society. The FDA recommended that IDE sponsors use an accepted method of risk assessment, such as ANSI/AAMI/ISO 14971: Medical Devices - Application of risk management to medical devices, to analyze the increased risks to study subjects. In addition, the guidance outlines best practices for informed consent and taking into account a patient’s preferences.

The final document includes changes from the draft guidance, published in June of 2015. It seeks to clarify what the FDA considers to be a “well-designed study,” which was flagged as ambiguous in a comment from medical technology association AdvaMed. The final guidance defines a well-defined study as “more likely to produce important knowledge about a device or disease.”

In its comments on the initial draft, AdvaMed wrote that it “strongly supports FDA’s efforts to provide greater clarity for sponsors, FDA staff and sponsor-investigators about the principle factors FDA considers when determining the benefits and risks of IDE applications as well as FDA’s efforts to strengthen and streamline the U.S. clinical trial enterprise.”

JDRF, an advocacy organization that funds type-1 diabetes research, also provided support to the draft guidance, and lauded a focus on the patient’s perspective.

“It is essential to incorporate patient perspective throughout the life cycle of medical devices. And, acknowledging the place that this holds in benefit-risk determinations for IDEs underscores that fact,” the organization wrote in a comment. “This guidance, once finalized, will certainly bring safer and more effective patient-driven products to market and increase transparency of the regulatory process.”

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