Advancing Regulatory Science at FDA

FDA's 2021 Focus Areas of Regulatory Science

Regulatory science research plays an important role for the U.S. Food and Drug Administration (FDA) as it enables the agency to enhance its regulatory mission. The recently published Advancing Regulatory Science at FDA: Focus Areas of Regulatory Science (FARS) report outlines topics the agency has identified as needing continued development and investment. Learn more about the different strategic initiatives and their focus in this article.

Public Health Preparedness and Response

The FDA is responsible for protecting public health in the United States. For this reason, it controls the safety and efficacy of human and veterinary drugs, biological products and vaccines, medical devices, food, radiation-emitting devices, tobacco products and cosmetics. This applies to products manufactured in the U.S. as well as imported products.

To counteract naturally occurring diseases, such as Covid-19 and influenza, so-called medical countermeasures (MCMs) must be initiated. MCMs describe the products that can be used to diagnose, prevent or treat conditions. The FDA is responsible for reviewing the safety, effectiveness, and quality of MCMs, which include biologics, drugs and devices. However, developing MCMs often presents unique challenges. The lack of incentive in commercial markets or poor feasibility of clinical trials, are just some of the factors that could compound the development of MCMs. For this reason, the FDA responds to these challenges by applying innovative science and developing standards, tools, and strategic approaches to support safe and effective MCMs.

FDA actions include:

  • The development of reference materials to facilitate evaluation of specific diagnostic devices for emergent viruses
  • Development and evaluation a variety of micro-physiological systems to use as tools to support the creation of MCMs for acute radiation syndrome (radiation sickness) and COVID-19


Increasing Choice and Competition through Innovation

Scientific and technological innovations play a critical role in making regulatory decisions to meet public health needs. The FDA's Emerging Sciences Working Group deals with the identification of new scientific trends that may affect products coming to the FDA in the future. For this reason, the FDA adapts and responds by actively promoting scientific and technological innovations. This plays a particularly important role when it comes to developing individualized therapeutics, each targeted at one to a few individuals to address unmet health needs. The FDA is exploring new technologies to address critical needs for greater mechanistic understanding and improved manufacturing capabilities.

Scientific and technological innovations also play an important role when it comes to improving production processes. Advanced manufacturing is a collective term for innovatively applied or new medical product manufacturing technologies and processes that can improve quality, increase efficiency, eliminate medical device bottlenecks, or accelerate time to market.

FDA actions include:

  • Advanced manufacturing techniques can apply smart concepts that use automation, digitalization, and artificial intelligence to streamline production methods, collect more process control data, and ultimately use a smart algorithm to adaptively control or make decisions about production or release


Unleashing the Power of Data

Data is a critical resource for all of the FDA's work. For this reason, the FDA focuses on identifying and using reliable data sources. To control and improve the quality and integrity of FDA-regulated products throughout the product lifecycle, the FDA uses healthcare data and analytics. The faster data can be collected and analyzed, the better the FDA can fill knowledge gaps and support decision-making. To make the most efficient use of the data, the FDA relies on high-performance computing environments and new computational tools based on machine learning (ML) and AI.

The FDA is responsible for monitoring and ensuring the safety of FDA-regulated products. However, due to limitations in clinical trials, safety data often needs to be continuously collected post-market. If adverse events occur after launch, they may influence regulatory actions such as labeling changes and issuance of safety notices. To protect the public's health, it is important for the FDA to identify potential safety signals as early as possible through post-market surveillance. Therefore, the FDA is working to improve ways to identify new safety signals with greater precision in less time. Some of the approaches being evaluated for feasibility are based on incorporating AI, real-world evidence (RWE), and the use of data from a combination of active and passive safety monitoring systems.

FDA actions include:

  • MedWatch is the FDA’s medical product safety reporting program for health professionals, patients and consumers. MedWatch receives reports from the public and when appropriate, publishes safety alerts for FDA-regulated products, including over-the-counter medicines, biologics, medical devices


Empowering Patients and Consumers

The better the FDA knows consumer preferences and perspectives, the more effectively the FDA can develop relevant and easy-to-understand communication and resources. In doing so, the agency aims to better inform consumers, patients and healthcare professionals about FDA-regulated products so they can live healthier and more productive lives.

FDA actions include:

  • Conducting of small, informal, non-regulatory, non-public Patient Listening Sessions, where patients directly share their experience with a disease or condition with FDA staff
  • Collaboration with the Medical Device Innovation Consortium, a public-private partnership, to explore the impact of patient selection methods

To read the full report click here.