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We often talk about the exciting jumps in technology and innovation that improve and save lives. What is less frequently discussed is how regulation has to learn to keep pace. And in healthcare, that pace-setting is incredibly important.
To ensure that the pace can be set and maintained, regulators like the Food and Drug Administration (FDA) need to collaborate with industry and advocacy partners to stay educated, said experts on the final day of the 2025 Biotechnology Innovation Organization (BIO) International Convention in Boston.
“The one thing I know from my time at the FDA, and working with over 10,000 rare diseases, is there’s no way the FDA can be an expert in every disease,” said Adora Ndu, PharmD, J.D., Chief Regulatory Officer and Executive Vice President of Portfolio Strategy and Management BridgeBio Pharma, Inc. “And so a big part of what we do is education and really trying to share some of the knowledge, some of the science, with the agency so that they can serve their role as regulators, and they can see beyond the four corners of a review document that is submitted to them. That includes working very closely with patients and advocacy organizations to do the educating.”
As Jeremiah Kelly, Partner at Venable LLP, explained, the spirit of education is also informed by the agency’s history.
“We have to come at these things with a level of humility, because the food and drug law, over the course of its history, has developed and been propelled by very serious public health tragedies,” he said. “At the FDA, you’ve got this string of tragedy equals we have to fix something, and we also have this tension between speed and making sure we’re fully evaluating the benefit-risk profile of products.”
Adora Ndu, PharmD, J.D., Chief Regulatory Officer and EVP of Portfolio Strategy and Management BridgeBio Pharma, Inc., spoke to Bio.News in Boston
Change is the only constant
A collaborative approach to education is important for drug companies, as well, especially as they work to stay up to date on the FDA’s ever-changing policies and leadership. This fact was top of mind for panelists, given the many changes happening at the FDA today.
“Obviously, this is a watershed moment for the agency,” said Ndu. “We’ve never seen the agency in the current environment, with a significant amount of change—change in leadership, change in career staff that typically are unimpacted by new leadership at the FDA.”
“We are trying to understand how that impacts how we think about career development, how that impacts how we engage with FDA,” she continued.
What do regulatory changes mean for small biotechs?
Sixty percent of small biotechs “are just trying to stay alive for the next year,” said Cory Sago, Ph.D., CEO of Amplitude Therapeutics. “It’s harder for us to then lift our gaze even higher to, for example, four years from now, to ask, What’s that phase 2 going to look like? Let alone 10 years from now. How’s this going to get approved?”
“Failure rate for drugs is 10 failures to every one medical product,” added Kelly. “It is the highest failure rate industry that I’m aware of, and it’s incredibly expensive.”
“The predictability of the system is really essential,” Kelly said.
This need for predictability is behind some of the anxiety that the panelists expressed. With so much happening, the industry as a whole is keeping an eye on the FDA and positioning itself to respond as needed to ensure that innovative new drugs can still get to patients.
After all, as one audience member observed, the decisions and changes at the FDA don’t just affect Americans; changes and decisions at the FDA impact companies and patients worldwide.
