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2021 Advancing Regulatory Science at FDA: What are the focus areas

Post thumbnail 2021 Advancing Regulatory Science at FDA: What are the focus areas

The Food and Drug Administration (FDA) conducts research in regulatory science to improve regulatory decision-making by developing new tools and approaches to assess the safety, efficacy, quality and performance of products. FDA aims at pioneering research tools to advance product development and providing innovative regulatory guidance to sponsors. Thus, FDA positions itself at the forefront of innovation.  

In particular, FDA’s Emerging Sciences Working Group scans the horizon to identify new scientific trends that may affect products coming to FDA in the future. This impacts new scientific working groups, employee recruitment and trainingand project fundingFDA published on January 11th2021 its annual report identifying areas of focus for research in regulatory science:  

  1. Public Health Emergency Preparedness and Response 
  2. Increasing Choice and Competition through Innovation 
  3. Unleashing the Power of Data 
  4. Empowering Patients and Consumers 

In this blog post, we summarize the main areas of focus presented in the report 

Public Health Emergency Preparedness and Response:

  • Development of Medical Counter Measures (MCM)  

FDA helps speed up the development and availability of potential diagnostics, therapeutics and vaccines by : 

  • Establishing clear regulatory pathways 
  • Developing standards and tools (e.g. reference materials for self-evaluation used for Covid-19 diagnostic development) 
  • Advising on clinical trials and applying innovative solutions in specific cases (e.g. if clinical trials are not feasible)  
  • Providing guidance to manufacturers 
  • Development of new technologies to reduce pathogen contamination 

Pathogen contamination is an issue especially for products that cannot be sterilized (e.g. blood, proteins). In this case, technologies should focus on pathogen detection, and contamination prevention and reduction.  

  • Substance use disorders 

FDA focuses on decreasing exposure and preventing new addiction. FDA also supports addiction treatment and will focus on stimulant use disorder for which there is still a lack of medication. Novel pain therapies should be developed as alternatives to addictive substances.  

  • Antimicrobial resistance (AMR) 

FDA has an AMR taskforce and is part of the taskforce Combating Antibiotic Resistant Bacteria (CARB). The goal is to develop approaches to detect, prevent, and limit the impact of AMR, such as diagnostic or surveillance tools and new antimicrobial products.  

  • Food safety 

FDA continues to monitor the human and animal food supply for ongoing and emerging threatsNew methods for detecting and countering these threats need to be developed and deployed.  

FDA’s Compounding Quality Center of Excellence performs research and provides education on bulk drug substances used in compoundingFDA also inspects outsourcing facilities on a voluntary basis to ensure access to high-quality compounded drugs.  

Increasing Choice and Competition through Innovation:

There is a critical need for more mechanistic understanding of diseases and improved manufacturing capabilities, especially in the case of rare diseases. FDA is exploring new technologies (omics) such as pharmacogenetics, gene therapy, bacteriophage therapy. The cloud-based community portal PrecisionFDA is aimed at sharing related tools and data 

  • Complex Innovative Trial Design (CID) 

The goal of CID is to maximize clinical trial efficiency by determining the optimal design for the question and the population of interest. The CID program offers sponsors increased interaction with FDA to discuss their proposed approach 

The microbiome research aims at investigating the impact of food & drugs on the microbiome as well as its potential use in treatments.  

  • Novel Foods and Food Ingredients 

FDA’s role is to assess the safety and labelling of novel foods or ingredients, and to develop new regulations. For example, FDA collaborates with USDA on regulating foods made from cultured animal cells.  

Regenerative medicine includes cell and gene therapy, therapeutic tissue engineering products, and combination of those. These therapies come with very specific regulatory challenges, for which there are no international standards. FDA is working on addressing the lack of international consensus standards for safety and effectiveness in regenerative medicine 

  • Advanced Manufacturing 

Advanced manufacturing includes different production technologies to improve agility, rapidity, safety. 3Dprinting can be leveraged to produce human cells and tissue implantsIn times of public health emergency, process intensification can increase the production of MCMs. Smart manufacturing includes automation and the use of artificial intelligence for process control and product releasePoint-of-care manufacturing has been developed in several hospital systems across the US.  

FDA’s goal is to develop methods and approaches for establishing the bioequivalence of generic drugs, for instance by using modelling approaches such as physiologically-based pharmacokinetic (PBPK) models 

  • Product Development Tool 

FDA constantly develops tools, methods, and models to reduce the time, complexity, or cost of developing regulated productsModelling science can be a valuable tool if translated into actionable regulatory tools. Examples include qualification processes for biomarkers, or the use of predictive technologies to replace, reduce and refine reliance on animal testing.

Unleashing the power of data

  • Diverse data sources and opportunities 

Data are collected everywhere in the healthtech industry: electronic health records, medical imaging, genomic sequencing, payer records, pharmaceutical research, Digital Health Technologies (DHT)medical devices. Opportunities are as diverse: filling knowledge gaps, reducing risks, informing decision-making, increasing patient/consumer knowledge.  

  • Focus on some data and technologies  

Among all the data and technology developments, FDA is focusing on three in particular: Artificial Intelligence (AI), DHTs and Real World Evidence (RWE). FDA is working to improve its understanding of AI opportunities and limitations with applications in learning medical devices, manufacturing automation and regulatory review resource allocationThe main challenges with DHTs are to protect data integrityimprove algorithm reliability. They can be used for example to supplement clinical trial data (e.g. FDA MyStudies mobile app). Finally, FDA is collaborating with medical device manufacturers through the National Evaluation System for health Technology (NEST) to promote the use of RWE to support medical product development and regulatory decision-making 

There is a need to identify new safety signals with more precision in decreased time. This is why FDA has created and participates in several safety surveillance platforms: FDA Sentinel SystemBiologics, Effectiveness and SafetyMedwatch 

Empowering patients and consumers 

  • Patient and Consumer Preferences and Perspectives 

The burden of disease and treatment on patients and caregivers could be better understood using a mix of qualitative and quantitative social, behavioral and declarative data. 

  • Patient-Reported Outcomes and other Clinical Outcome Assessments  

Clinical Outcomes Assessments (COAs) should matter to patients and attention should be paid to include Patient-Reported Outcomes (PROs) in clinical trials.  

  • Empowering Patients and Consumers to Make Better-Informed Decisions 

When designing communication materials, it is important to consider people’s beliefs, attitudes, feelings and motivations. FDA will leverage Social and Behavioral Science to create the most effective communication and education materials on public health topics and evaluate their impact 

In spite of the current pandemic context, FDA’s areas of focus are diversified and dedicated to innovation at every level: methods, technologies, scientific research and communication. VCLS’ expertise areas are thoroughly aligned with these priorities to support sponsors at the forefront of innovation and enable them to benefit from dedicated FDA programs.  

blog post by

Pauline Lestringant thumbnail
Pauline Lestringant
Regulatory Scientist, Ph.D.
Pauline supports clients in the design and implementation of global regulatory strategies for the development of innovative human drugs and biologics.