Details
On September 30, 2022, the President signed into law the Food and Drug Administration (FDA) User Fee Reauthorization Act of 2022. This law included the sixth reauthorization of the Prescription Drug User Fee Act (PDUFA VII) and provided FDA with the necessary resources to maintain a predictable and efficient review process for human drug and biologic products. As part of this, FDA committed to the “enhancement and modernization of the FDA drug safety system,” which includes activities to optimize the Sentinel Initiative. One of the focus areas is the use of negative controls to support use of real-world evidence. This project describes the methods development activities being conducted as part of the PDUFA VII commitment on “Use of Real-World Evidence - Negative Controls.”
One project will be conducted by the Innovation Center (IC) to extend, test, and adapt the Data-driven Automated Negative Control Estimation (DANCE) algorithm to large-scale healthcare data that reflects the Sentinel data environment. Through this project, the workgroup will synthesize relevant methodological content and design simulation and empirical studies in consultation with the FDA. The studies will incorporate the complexities observed in real world data and involve test cases incorporating safety endpoints.
A parallel project will be conducted to generate a prototype tool that implements the DANCE algorithm in the target data systems and provide the code pack and corresponding technical specifications for public posting. The code will be developed in R by Mass General Brigham (MGB). The Sentinel Operations Center (SOC) will convert the code to SAS and apply it for use in the Sentinel Distributed Database system portfolio of tools.
Additional Information
Contributors
Richard Wyss, Msc, PhD; Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women’s Hospital and Harvard Medical School, Boston, MA
Xu Shi, PhD; Department of Biostatistics, University of Michigan School of Public Health, Ann Arbor, MI
Sarah Dutcher, MS, PhD; US Food and Drug Administration, Silver Spring, MD
Fatma Shebl, MD, PhD, MS; Fang Tian, PhD, MPH, MHS; Chanelle Jones, MHA, CPhT; Wei Hua, MD, PhD, MHS, MS; Yong Ma, PhD, MS; Motiur Rahman, PhD, MS, MPharm; Ryan Hickson, PharmD, MPH, PhD; Natasha Kasid, MD; US Food and Drug Administration, Silver Spring, MD
Rishi Desai, MS, PhD; Shirley Wang, PhD; Haritha Pillai, MPH; Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women’s Hospital and Harvard Medical School, Boston, MA
Darren Toh, ScD; Meighan Rogers Driscoll, MPH; Department of Population Medicine, Harvard Pilgrim Health Care Institute and Harvard Medical School, Boston, MA
Myeonghun Yu, PhD; Department of Biostatistics, University of Michigan School of Public Health, Ann Arbor, MI
Eric Tchetgen Tchetgen, PhD; Department of Statistics and Data Science, University of Pennsylvania, Philadelphia, PA
Erich Kummerfeld, PhD; Institute for Health Informatics, University of Minnesota, Minneapolis, MN