Research efforts focus on predictive toxicology, precision medicine, biomarker development, drug safety, and drug repositioning. Most research projects are in collaboration with scientists within NCTR, across FDA product centers, and in the larger scientific community. One key endeavor of the branch is to construct knowledge bases in the specific areas of FDA’s responsibility to provide a data-driven decision-making environment for enhanced safety evaluation and precision medicine.

Conducts peer-reviewed research of statistical methods to analyze toxicological and molecular data as well as data-mining techniques for pattern identification and signal detection. The branch also provides statistical support related to FDA’s mission to protect and promote public health.

Provides critical support and enhancement to infrastructure in the areas of software and database development for research support and research management, high performance computing, systems integration, and information system-asset management and procurement.

Strengthens the division, focuses on “knowledge uptake” of the Division’s research products for regulatory application, and enables “data liberation” of regulatory data from the FDA product centers, thus facilitating regulatory-science research in the Division and increasing NCTR's linkages with FDA product centers.

Regulatory Applications and Support

Collaborating with CDER to develop and support:

• Data Analysis and Search Host (DASH)
• Breakthrough Therapy Designation (BTD)
• Safety Policy and Research Team (SPRT)
• Smart Template System (STS)
• FDA drug labeling documents (FDALabel)
• Supporting ORA by developing Automated Laboratory Information System (ALIS)
• Collaborating with CTP to create ASSIST4Tobacco 

Alternative Methods and Knowledge Bases

Developing the following: 

• Advanced DILI prediction models for FDA-regulated products like drugs and supplements.
• Opioid Agonists/Antagonists Knowledgebase (OAK) for better management of opioid-use disorder.
• Molecules with Androgenic Activity Resource (MAAR) open-access platform for assessing the safety profile for chemicals in various food, supplement, or cosmetic products that may affect the endocrine system and cause hormonal disfunction.

Precision Medicine and Therapeutics

• Conducting studies under the ongoing project, Sequencing Quality Control Phase 2 (SEQC2), an NCTR-led consortium effort to assess technical performance and application of emerging technologies for safety evaluation and clinical application.

Artificial Intelligence and Machine Learning (AI/ML)

• Conducting research to harness AI in mining FDA documents for relevant information to enhance regulatory operations and applications.
• Accessing the safety profile of prospective drugs in Investigational New Drug (IND) applications to assist FDA reviewers.
• Developing explainable AI (XAI) models that will facilitate the agency’s adoption and application of AI technologies in evaluating and regulating AI-based medical products.

Real-World Data and Real-World Evidence

• Investigating racial disparities in patients with heart failure admitted to critical care and its subsequent impact on their health.
• Analyzing safety profiles of various prescription NSAIDs to understand the difference in patient sexes (male vs. female) across the U.S. population.
• Developing an integrative model for assessing DILI risks for various drugs and supplements.