This area of research notably exercises the use of real-world evidence (RWE), complex innovative trial design (CID), modeling informed product development, product safety surveillance and digital health. Data that comprise RWE include electronic health records, administrative claims, and patient-reported data via mobile devices. CID includes complex adaptive, Bayesian, and other trial designs that are used selectively to provide substantial evidence of effectiveness in new drug applications or biologics license applications. A common feature of many CIDs is the need for simulations rather than mathematical formulae to estimate trial operating characteristics. Model-informed drug development is an approach that involves developing and applying exposure-based, biological and statistical models derived from preclinical and clinical data sources to inform drug development and decision-making. Product safety surveillance includes the use of FAERS and VAERS. Digital health includes mobile health, health information technology, wearable devices, telehealth and telemedicine, and digital personalized medicine.

This theme highlights the use of artificial intelligence (AI) and big data. AI includes machine learning, deep learning, natural language processing, robotics, image recognition, etc. Big data are regularly amassed in nonclinical and clinical settings. The breadth, depth, and diversity of big data both benefits and supports knowledge gaps.

Social and behavioral science research promotes the development of clear communication methods to diverse consumer populations and patients; apprises researchers of patient/consumer preferences and perspectives; and disseminates new knowledge about methods used by patients and consumers to report their health status through different types of clinical outcome assessments, including patient-reported outcomes. A combination of strategies are applied to inform the regulatory review process, communication strategies, and policy development. These strategies include evidence-based approaches; evaluating the impact of communication and educational materials; and understanding consumer beliefs and attitudes.

This area of research includes examining biomarkers, novel technologies (e.g., alternative methods for toxicity assessment), and advanced manufacturing. Advanced manufacturing is a collective term for medical product development and manufacturing that incorporates technology or established techniques using innovative methods with the potential to improve medical product quality, address product shortages, and speed time-to-market.

Included in this area of study are regenerative medicine, individualized and precision medicine, and examination of the microbiome/microbiota. Regenerative medicine refers to a global approach to restore, replace, or regenerate cells, tissues, or organs to treat or mitigate disease or dysfunction (e.g., cell therapy). Individualized medicine refers to the development of therapeutics to address unmet medical needs respective of unique individual maladies. Precision medicine is an emerging approach for patient treatment decisions and disease prevention that considers genomic/genetic variabilities, environment, and lifestyle. The term microbiome/microbiota refers to the community of microbial organisms linked by physical location (ecosystems), function relationships, and collective genomic potential in homeostasis or dysbiosis. There is extensive data-based evidence that human, animal and environmental microbiota play significant and varied roles in states of health and disease.

The focus of this research includes medical countermeasures and technologies to support pathogen reduction of FDA-regulated medical products. Medical countermeasures are FDA-regulated products (biologics, drugs, devices) that can be used to diagnose, prevent, protect from, or treat conditions caused by exposure to chemical, biological, radiological, nuclear threats, or emerging infectious diseases. This topic also includes the use of innovative technologies to reduce pathogen contamination and reduce or eliminate pathogens from FDA-regulated medical products to prevent transmission of infectious disease through their use.

Ensuring that the food people and animals eat is safe and protected from contamination is an essential element of promoting human and animal health. FDA ensures that the nation’s food and cosmetic supply is safe from harmful chemical, microbial (inclusive of antimicrobial resistance), and radiological contamination and other types of adulteration. FDA faces unique challenges in the oversight of human and animal food safety and cosmetic safety in the 21st century. The Agency oversees nearly 90% of the food supply for humans and animals, including domestically produced and imported products, as well as human dietary supplements and botanicals. This oversight and One Health approach includes continuous surveillance of the human and animal food supply for ongoing and emerging threats; development of methods for threat detection and quantitation; deployment of those methods to domestic and global partners; predictive toxicology and risk assessment; and strategic analysis of large amounts of data from numerous sources. Communication of these results and activities is vital to informing the public of the safety of the food supply both for them and their animals.

FDA regulates controlled substances and tobacco. The use or misuse of these products may lead to physical or psychological dependence. This topic will explore substance use and use disorders due to controlled substances, including prescription opioids, benzodiazepines, and stimulants. Substance misuse may include unintended use of products (i.e., for a different indication, through a different route of exposure) or intended use of a product that results in harm to users (e.g., tobacco). When evaluating regulated products, FDA considers product abuse liability and addiction potential. Some common measures important in determining the abuse liability of a product can include pharmacokinetics, patterns of use, and the subjective effects of that product.