Hundreds of industry leaders have signed a letter to FDA Director Marty Makary, M.D., about the importance of having a predictable regulatory agency, citing a survey in which 82% of biopharma respondents said they were concerned about the FDA’s ability to function.
Overall, CEOs worry that added volatility in an already high-risk industry will discourage overall investment in biotech innovation or will drive it to other countries, according to the Nov. 20 letter posted by the nonprofit group No Patient Left Behind. The signatories include prominent industry leaders such as Alnylam Pharmaceuticals' founding CEO John Maraganore, Ph.D., and Nkarta Therapeutics CEO Paul Hastings, among many others.
The survey was disseminated among biopharma CEOs and regulatory affairs leaders and focused on recent experiences with the FDA. Notably, the survey was collected before Richard Pazdur, M.D., was tapped to lead the Center for Drug Evaluation and Research, an appointment which the authors expressed strong support for.
While some respondents said their interactions with the FDA were satisfactory or “business as usual,” the survey revealed that confidence in the agency has been shaken by inconsistent and unpredictable decisions, with most leaders reporting concerns about the FDA’s abilities.
CEOs highlighted the persistent gaps in institutional memory and accountability, “where alignment no longer guarantees a stable regulatory framework for drug development,” according to the letter.
“In one instance the agency had not done a substantive review of our package and put forth their policy perspective and a sort of a take-it or leave-it approach,” one unnamed biopharma leader said in the survey.
Some respondents pointed out the increasing frequency of missed deadlines and canceled meetings, including advisory committee meetings. Over the last 10 months, the agency has pulled meetings with biotechs to discuss trial plans, delayed multiple approval decisions and thrown up new obstacles for treatments it previously showed support for.
Industry leaders also expressed concern about the continued exodus of experienced FDA employees, which has affected the regulator’s engagement with sponsors given the increased workload and knowledge gaps among remaining staffers, according to the letter.
“Not one reviewer has any clinical experience in our therapeutic category (a large category),” one leader responded.
This spring, under the freshly elected Trump administration, the federal government launched mass layoffs, which were expected to affect around 3,500 FDA workers. In April, No Patient Left Behind penned a letter voicing concerns about the agency’s capacity after the layoffs. At least one past FDA official has warned against the cuts, noting that the changes could disrupt the drug review process.
Some efforts have since been made to rehire laid-off workers, and the FDA this week said it's hiring more than 1,000 people, though no job postings are publicly available.
Industry concerns also stem from the agency’s “failure to stand up for established vaccine science and important mRNA research, and an unwillingness to address misinformation,” according to the letter.
These points have prompted numerous other challenges, according to survey respondents. Several leaders cited difficulty planning and starting clinical trials plus hardships maintaining investor support.
Meanwhile, regulatory delays and higher development costs are increasingly encouraging sponsors to consider options outside of the U.S.
“For our ongoing operations, we have largely gone ex-U.S. where we have better scientific engagement and greater speed,” one CEO said.
While the authors applauded the agency’s efforts to improve upon the previous status quo, the writers said the U.S. has to “avoid erecting needless, new barriers to groundbreaking drug development at a time when visionary leadership, experience, flexibility and creativity are needed most at FDA, and when global competition is at its fiercest.”
“FDA must be an effective institution that can be counted on to say what it means and mean what it says, even when personnel change at the top, in the middle, or on the front lines,” the letter continues.
Institutional expertise is particularly significant to small, early-stage biotechs, which account for most new drug and biologic applications submitted to the FDA, the authors said.
“As a nation, we must not erode our regulatory infrastructure, but leverage new thinking and technologies to modernize it,” the authors write.
“We should not accept increased development costs that do not serve the patient, lapsed commitments, and unfounded disdain for scientific experts,” the writers said in the letter to Makary. “We should not accept second-best, shifting regulatory standards that undermine American biomedical superiority. And we should not tolerate FDA volatility driving companies to take their investment resources and clinical trials overseas.”
When asked about the survey findings, a spokesperson for the Department of Health and Human Services told Fierce the “FDA decisions reflect evolving scientific evidence and regulatory standards.”
“What some view as shifting expectations is often the natural result of new data or updated insights,” the spokesperson continued. “The agency’s strength lies in a rigorous, evidence-driven review process that must remain flexible to keep pace with novel therapies and emerging technologies.”
“Under Commissioner Makary’s leadership, no FDA in history has produced so much regulatory innovation in such a short span,” the agency representative said.
The spokesperson did not elaborate further on that statement.