The FDA plays an incredibly important role in the process of developing, distributing, and monitoring therapeutic products. So when something changes at the FDA, the entire life sciences community sits up and takes notice.
In recent months, those changes have come fast and furious as the new administration seeks to put its priorities into action. It can be difficult to keep track of how efforts to streamline, modernize, and reorganize the agency will impact drug approvals and marketing activities, especially as regulators contend with strongly competing political, socioeconomic, and scientific ideas.
With pharma companies doing their best to reduce risks and maintain sustainability in a time of ongoing regulatory uncertainty, we sat down with COTA’s Senior Statistical Director (and former FDA insider), Laura Fernandes, to discuss what trends pharma companies should watch over the next few months.
Shifting perspectives on data diversity
Data is the lifeblood of medical research, and scientists agree that the highest quality data is diverse, representative, and inclusive of populations that are likely to use the products resulting from their R&D efforts.
While the FDA has championed data diversity in the past, such as by offering insights to industry on ensuring real-world data (RWD) and real-world evidence (RWE) are appropriately collected and equitably used, recent actions to redefine the concept of representation have changed how the agency communicates around the topic.
For example, draft guidance on diversity action plans for clinical trial enrollment was removed from the FDA website before the restoration of the information in late January, 2025. The document’s homepage now includes a disclaimer that the information does not reflect the current administration’s views on gender ideology.
At the same time, however, this administration has been widely promoting an “America first” perspective across many facets of economic and social policy, which could lead to a greater emphasis on US-based clinical trials that feature primarily or exclusively US-based patient populations.
There may be both advantages and disadvantages to this approach, said Fernandes.
“Accurate representation of US populations and direct involvement of US care delivery systems are very important when designing products for the American market,” she said. “Yet it may not be possible to complete some clinical trials for rare diseases with US patients alone, potentially delaying access to care for patients in need.
Pharma companies will need to stay informed on the latest thinking from the FDA on how to best balance scientifically sound, appropriately diverse real-world data with the administration’s viewpoints on who, what, and where to prioritize in clinical research,” she suggested.
Monitoring the risks of slowdowns, delays, and bottlenecks
The FDA is undergoing a massive restructuring, including the termination of thousands of employees who are among the 20,000 HHS workers cut from payrolls this year so far. At the same time, the government has paused or cancelled at least $1.9 billion in medical research grants, 61% of which were directly supporting R&D projects, creating uncertainty in timelines for everything from completing clinical trials to gaining approvals to bring new therapies to market.
Planned tariffs on pharmaceutical products are also expected to prompt pharma companies to rethink their supply chains, which could make it difficult to adhere to speedy timelines for producing and distributing products after they are approved.
While anecdotal evidence from COTA’s industry insiders at ASCO this June indicated that delays had not yet become a major issue, that was before the most significant round of FDA layoffs occurred in late July. Now, news outlets are reporting increasing concern around how a slimmed down agency will handle the volume of pending requests.
“Companies will need to zero in on how to create submissions that are most likely to meet FDA requirements with as little back-and-forth as possible with agency representatives,” Fernandes advised. “RWD and RWE are becoming much more important in this task, since they can provide regulatory-grade evidence that is increasingly accepted by FDA officials for submissions.”
Pharma manufacturers can also consider taking advantage of innovative programs like FDA PreCheck, which streamlines review of domestic pharmaceutical manufacturing and may offer some incentive for companies to invest more heavily in US-based operations.
A focus on efficiency and insight through AI and novel data sources
The FDA is joining the rest of the life sciences industry in embracing artificial intelligence to improve efficiency, reduce burdens, and speed up critical processes.
In June, for example, the agency announced the launch of Elsa, a generative AI tool to modernize agency functions and help a reduced workforce stay on top of their duties. The tool can understand and summarize documents, including identifying adverse events from large volumes of data to support safety assessments. It can also perform faster label comparisons and generate code for non-clinical database applications.
The agency hopes the tool will be the first in a long line of AI solutions to enhance efficiency internally and across the industry.
AI tools are rolling out in the life sciences community at the same time as companies seek to mine valuable insights from a wider variety of novel data sources, including wearable devices, electronic patient-reported outcomes (ePROs), and deeper clinical and administrative data sources.
“AI will be crucial to help aggregate, curate, and present insights from these real-world data sources, especially as the FDA continues to work with device manufacturers and data companies to best leverage these technologies,” predicted Fernandes.
“Pharma companies that work quickly to explore how AI can improve efficiency and assist with analyzing rich, comprehensive RWD sources are likely to gain an edge over their competitors while keeping pace with what’s happening on the federal level to enhance and streamline operations.”
