Three strategic initiatives form the backbone of the regulatory science areas of focus for the US Food and Drug Administration in 2021, according to a new report.
The Focus Areas of Regulatory Science (FARS) issued on Monday “outlines topics that the FDA has identified as needing continued targeted investment in regulatory science research to facilitate the development of innovative products, provide data and methodology to inform regulatory decision-making and improve guidance to sponsors,” according to FDA Chief Scientist Denise Hinton.
The first of the three broad themes, outlined at the beginning of 2020 by FDA Commissioner Stephen Hahn, MD, was dubbed “Unleashing the Power of Data.” The second theme focuses on using innovation to increase choice and competition, while the third is aimed at empowering patients and consumers.
“The focus areas are not intended to be a comprehensive list of all the FDA’s research needs, but generally encompasses research affecting more than one center or office,” said Hinton in a press release announcing the availability of the report. “The areas identified have been designed to be flexible so they can be updated as needed to address evolving regulatory needs.”
Public health preparedness
The coronavirus pandemic, unforeseen at the start of the new decade, also led Hahn to include public health emergency preparedness and responsiveness in the FARS report. The report details some of the regulatory considerations involved in details of emergency preparedness such as speedy development of medical countermeasures and technologies to reduce pathogen contamination. The report also touches on non-pandemic related public health preparedness to address the opioid crisis, the ongoing serious problem of antimicrobial resistance, food safety, and ensuring the quality of compounded drugs.
The power of data
The theme centered around bringing the power of data to bear on FDA’s regulatory work includes product safety surveillance activities. FDA’s Sentinel system and the Biologics, Effectiveness and Safety (BEST) systems are examples of active surveillance systems currently in use by the agency; Sentinel and BEST are both key data-gathering tools to track post-authorization safety of COVID-19 vaccines, for example.
FDA is both using artificial intelligence and machine learning techniques to support its activities while also exploring how, for example, artificial intelligence “can combine diverse data so clinical trial results can be analyzed in a more comprehensive and expeditious way,” according to the report.
Digital health technologies such as wearables and apps also fall under the “Harnessing the Power of Data” area of focus. Here, FDA’s Digital Health Center of Excellence comes into play as the agency works to keep pace with consumer technology in this rapidly growing field.
The final data-centric area of focus is the increasing importance of real-world evidence (RWE) in the conduct of clinical trials and postmarketing review. Following the establishment of the RWE program under the 21st Century Cures Act, “FDA is committed to exploring the use of RWE in regulatory decision-making, including its ability to provide fit-for-purpose clinically meaningful information about the safety and effectiveness of medical products,” according to the FARS report.
Innovation in the service of choice and competition
The broad theme of harnessing innovation to boost choice and competition draws on the work of FDA’s Emerging Sciences Working Group, which “scans the horizon to identify new scientific trends that may affect products coming to FDA in the future,” according to the report. Areas of interest under this umbrella include individualized therapeutics and precision medicine, where “FDA sees a critical need for more mechanistic understanding, improved manufacturing capabilities, and additional tools.”
Another key area for regulatory science exploration is complex innovative trial designs, such as complex adaptive and Bayesian trials. FDA’s programmatic work in this area also fulfills an agreed-upon performance goal under the Prescription Drug User Fee Act VI (PDUFA VI). Microbiome research, regenerative medicine, and novel foods and food ingredients – such as laboratory-cultured “meat” sources – also fall under this rubric.
Advanced manufacturing techniques ranging from 3D printing to production methods controlled by artificial intelligence are also addressed in the innovation theme. In the report, FDA also cites its ongoing work to ease the approval path for generic alternatives to complex drugs by using innovative quantitative methods and modeling.
Reducing reliance on animal testing and refining the ability of non-clinical studies to predict or replicate clinical results is an additional area of focus, as is making product development tools publicly available. In a related area, FDA continues to support model-informed product development, another activity undertaken in partial fulfillment of PDUFA VI goals.
Considering the view of the patient
Patient and consumer empowerment is another area of focus for FDA in 2021. Understanding patient and consumer preferences and perspectives is a key part of this focus; patient listening sessions and patient-focused drug development meetings are two examples of concrete actions FDA is taking in this area.
Patient-reported outcomes are an increasingly important part of FDA’s assessment processes. “Strengthening FDA’s ability to use patient-focused methodology to inform regulatory decision-making is specified in FDA user fee agreements for medical product development and the Cures Act,” notes the report. Ongoing collaboration with patient groups, academia, and professional organizations seeks to broaden ways in which outcomes important to patients and their caregivers are included in regulatory decision-making.
Another aspect of patient and consumer empowerment is giving individuals the best information possible for making decisions about FDA-regulated products. Here, social and behavioral science researchers at FDA investigate how labeling statements and other claims made by marketers of drugs and devices affect patient decisions. In another impact area, FDA addresses how to improve messaging about the risks of tobacco use. As another example, FDA points to its Project Patient Voice website that collates patient-reported outcomes data from some oncology clinical trials.
The FARS report also details the funding sources and research capabilities and tools FDA has available to conduct its regulatory science research work, reminding the reader of the many public-private partnerships and inter- and intra-agency collaborations that support FDA’s efforts.
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