FDA and Duke-Margolis Center for Health Policy to hold workshop on the use of negative controls in real-world data studies

On 8th March 2023, a public workshop is being held by the Duke-Margolis Center for Health Policy in collaboration with the FDA entitled “Understanding the Use of Negative Controls to Assess the Validity of Non-Interventional Studies of Treatment Using Real-World Evidence.”

As announced via the Federal Register, the Food and Drug Administration (FDA) is convening a new public workshop, in a cooperative agreement with the Duke-Margolis Center for Health Policy, to help stakeholders understand negative control methodologies and their use in non-interventional studies used to assess medical product safety and effectiveness. The workshop will also provide a platform for discussion around how negative controls could support new methodological approaches for causal inference in the Sentinel System, the FDA’s medical product safety surveillance system.

According to the Federal Register announcement and the Duke-Margolis Center for Health Policy website, the workshop will take place virtually on 8th March 2023 at 10am-3pm EST and provide an discussion of the topic including:

• Introduction to current negative control methods used in studies based on real-world data
• Overview of techniques related to the use of negative controls, including their strengths and limitations
• Methodological and operational considerations for both safety and effectiveness studies when utilizing negative controls as part of regulatory assessment
• Key opportunities and challenges for incorporating negative controls for their use in regulatory decision-making

The workshop will contribute to fulfilling the FDA’s commitment to “optimizing the capabilities of the Sentinel Initiative to address questions of product safety and advance the understanding of how Real-World Evidence can be used for studying effectiveness” outlined in the seventh iteration of the Prescription Drug User Fee Amendments (PDUFA VII).