The FDA has been involved in a vast number of existing and new initiatives over the past couple of years to improve how it works with Sponsor organizations, patients and advocacy groups, data collection and analysis (internally and from pharma/device organizations), and the general public.

For example, the Oncology Center of Excellence (OCE) has several discrete initiatives underway, including:

•  Project Confirm – To promote the transparency of outcomes related to accelerated approvals for oncology indications.

•  Project Orbis – Which provides a framework for concurrent submission and review of oncology products among international regulators, which may allow patients with cancer to receive earlier access to products.
• Project Optimus – To reform the dose optimization and dose selection paradigm in oncology drug development.

Many other offices and divisions are well underway with their own substantial initiatives, making this a period of great change and evolution in the agency.

With respect to current regulatory oversight, always of great value is to assess trends in the development, approval, and commercialization of novel therapies, as well as looking across the industry at Warning Letter trends to see where deficiencies cluster.

Figure 1 shows a Pareto Chart of select 2022 Warning Letter citations while Table 1 provides a more categorical detail on the transgression criteria CFR subsection.

 

Combatting misinformation and disinformation

The FDA has recently been planting a stake in the ground to try to join the fight against misinformation and disinformation. They have created a new approach that they’ve termed “Rumor Control,” and it’s designed to help the lay public combat what misinformation they may be confronted with online in social media platforms, etc. As they note, “misinformation spreads 6x faster than the facts.” The “6x” metric is a bit of a statistical red herring (read: fallacy), but certainly the idea is well-taken, and as Mark Twain allegedly said (but didn’t really), “A lie can travel halfway around the world before the truth even puts its boots on.”* The FDA kicks off their Rumor Control website subsection with the following:

“The growing spread of rumors, misinformation and disinformation about science, medicine, and the FDA, is putting patients and consumers at risk. We’re here to provide the facts.”

I’ve included this information on the FDA’s Rumor Control initiative (see Figure 2), because at some level, almost nothing could be more critical for the agency to be involved in. People rely on the FDA (and CDC, WHO, etc.) to be consolidated, consensus sources of factual information regarding health, medicinal treatments, and strong science. Each of these three organizations have been much maligned over the past several years because of how the COVID-19 pandemic was handled, and damage control is certainly in order. Restoring credibility means confronting these epistemological challenges head-on and addressing public concerns directly.

Additional FOCI within FDA

The FDA has a new draft guidance, entitled, “Generally Accepted Scientific Knowledge in Applications for Drug and Biological Products: Nonclinical Information.” It is designed to help answer the extent to which generally accepted scientific knowledge (GASK) may be relied on for drug or biological product approval.

Another guidance document is Pediatric Drug Development Under the Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act: Scientific Considerations, wherein the purpose of the guidance is to assist industry in developing data and obtaining information needed to support approval of drug products in pediatric populations. The agency notes, “This guidance addresses selected clinical, scientific, and ethical issues regarding the development of drugs for pediatric use when such drugs are subject to the Pediatric Research Equity Act (PREA) and/or the Best Pharmaceuticals for Children Act (BPCA).”

Additionally, in March 2023, CDER and CBER published a Framework for the Use of Digital Health Technologies (DHTs) in Drug and Biological Product Development, which they describe is to help “guide the use of DHT-derived data in regulatory decision making for drugs and biological products.”

Regarding the increased use—and concern—for AI/ML, the FDA has released a discussion paper, “Using Artificial Intelligence and Machine Learning in the Development of Drug and Biological Products” to address these concerns. In 2021, more than 100 small molecule and biologic drug regulatory applications included AI/ML components, with even more in 2022.

Regulatory roundup

So, there are many facets of evolution within the FDA that are keeping the agency—and the industry—current with respect to the bi-directional exchange of ideas and information between the two entities. What is clear is that this is a period of time in which there are more active initiatives designed and designated for the forward-looking evolution of the FDA than ever before in its history. And ultimately, these changes are built for the benefit of the patient population which we all serve.

*It probably came from Irish satirist Jonathan Swift (1667-1745).

Further reading
• U.S. Food and Drug Administration. (2023). Rumor control. https://www.fda.gov/news-events/rumor-control

---

Ben Locwin is a healthcare executive and has served on scientific and regulatory committees and panels, and been involved in the design, development, and commercialization of medical therapies for over two decades.