September 5, 2018

Media Inquiries

  Alison Hunt
  240-402-0764

“The FDA’s gold standard for product review strives to maximize benefits, and minimize risks and significant uncertainties in meeting our principal obligation to make sure that new products are safe and effective,” said FDA Commissioner Scott Gottlieb, M.D. “But we can never have absolute certainty about all aspects of how a new product will perform. No reasonably sized premarket trial can ever be expected to reveal everything that could eventually become known about a novel medical device. Along with other recent FDA initiatives, the draft guidance issued today advances a rigorous, methodical and science-based approach for how the FDA considers uncertainty in benefit-risk determinations to support medical device premarket decisions that are based on the totality of scientific evidence available at the time of market entry. By providing a more systematic review to how we evaluate and address uncertainty – and how we define an acceptable extent of uncertainty – we believe that this sort of transparency can help improve device development programs, especially when it comes to innovation in treatment options for small patient populations, such as medical devices targeted to rare conditions or pediatric patients. Considerations around acceptable uncertainty are already a feature of all product review programs. The goal of this guidance is to provide a transparent, and more systematic accounting of how these considerations are made. By providing a written framework for how we evaluate and address varying uncertainty in our benefit-risk determinations, and how these principles factor into our premarket decisions, we hope to advance the development of more products targeted to unmet needs.”

The U.S. Food and Drug Administration today issued the draft guidance, “Consideration of Uncertainty in Making Benefit-Risk Determinations in Medical Device Premarket Approvals, De Novo Classifications, and Humanitarian Device Exemptions” to clarify the FDA’s policy with regard to considering and evaluating uncertainty as part of the FDA’s overall assessment of whether a novel device meets the regulatory standard for approval.

Building on work that the FDA has already done to bring clarity and consistency to premarket benefit-risk decisions, the draft guidance describes the factors that the agency may consider when evaluating uncertainty as part of benefit-risk determinations. These factors include but are not limited to: the public health need that a product intends to serve, the availability of alternative treatments and the ability to appropriately reduce or resolve uncertainty through robust data collection in the post-market phase.

The benefit-risk balance is already incorporated into premarket review, and the draft guidance is designed to make these considerations more transparent, consistent and objectively-defined for the review of medical devices. It provides illustrative examples of how these different factors could be applied for certain Breakthrough Devices or devices intended to treat or diagnose relatively small patient populations, such as pediatric patients. The benefit-risk based policies described in the draft guidance can help facilitate timely patient access to high quality, safe, and effective medical devices. It further demonstrates the FDA’s commitment to advancing modern regulatory policies that are designed to promote medical device innovation, while continuing to assure our gold standard for safety and effectiveness.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.