It has been a tumultuous few months for the US Food and Drug Administration (FDA), with the unexpected exit and swift return of the agency’s biologics lead, Dr Vinay Prasad, sending shockwaves through the agency.
Throughout Prasad’s fragmented reign as the head of the Center for Biologics Evaluation and Research (CBER), there has also been a range of unexpected drug application rejections – some of which were directly communicated by Prasad himself.
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A prominent example of this is the very public back-and-forth between the CBER lead and Sarepta Therapeutics, which saw Prasad face backlash for his request to remove Duchenne muscular dystrophy (DMD) gene therapy, Elevidys, from the market after the report of patient deaths associated with the drug.
Bold moves such as this led Becky Wood, ex-chief counsel of the FDA, to state that the FDA’s leaders – including Prasad – are “acting like political figures”.
At the Sidley Healthcare Investor conference in London, Wood described the FDA’s current dynamic as “odd”, noting that the FDA’s commissioner Marty Makary, as well as Prasad and Center for Drug Evaluation and Research (CDER) head, George Tidmarsh, are creating a “very different environment from what was historically seen with previous commissioners”.
The FDA echoed this sentiment in a statement to Pharmaceutical Technology, noting: “FDA leadership is challenging the status quo to deliver tangible results for the American people through gold-standard science and common sense.”
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By GlobalData“Under their direction, the agency is advancing radical transparency and prioritising the interests of Americans over special interests.”
She also noted that the new FDA leadership has seen a range of companies receive last-minute reach-downs to resettle expectations, after they thought they had a “regular order with the review division”.
The FDA did not provide comment on the recently distributed CRLs.
FDA interactions ‘normal’ for most
Though some biotech companies have run into last-minute roadblocks with the FDA in recent months, Wood notes that these issues are the exception rather than the rule.
She stated: “From time to time, we are seeing some unpleasant reach-downs, but 99% of the interactions we’re seeing with the FDA are very normal.
“This year, the agency has already approved 31 new drugs across oncology, rare disease and neurology, meaning it is on track to grant the same number of approvals seen year over year for the last several years.”
Despite FDA crackdowns being in the minority, Wood notes that companies attempting to commercialise drugs of specific interest to the President, Robert F. Kennedy Jr and the FDA leadership, such as vaccines or drugs for rare diseases, may be more strongly impacted by these changes.
For those operating in these niches, she advises that companies implement a “360 strategy early”.
Wood commented: “If you think there is a chance you might be the subject of an unpleasant reach down, I recommend that you are ready to reach out to government officials – sometimes as high as the White House – as well as the committees in oversight of the FDA and the press about your case.”
When making this point, she referred to the Sarepta example, which garnered significant attention from both the media and Congress.
Ex-FDA staff prepare for big changes
While she caveated that the FDA is not currently approving less drugs, Wood also noted that, in private practice, a fair few former FDA staff members are forming a shadow FDA to prepare for “big structural changes”.
This includes former FDA commissioner Dr Scott Gottlieb and former deputy commissioner and CMO Dr Janet Woodcock.
“I think former FDA-ers are concerned that, with all the reductions in force and the political reach downs, we may reach a tipping point where we are seeing more complete response letters (CRLs), fewer approvals and further disruption,” Wood said.
This led her to theorise that, if the industry ever did reach this tipping point, these initiatives could promote some normalcy within the pharma regulatory space.
As one of these initiatives, Wood discussed the possibility of making the FDA an independent agency: “This has been on the table for years, as this would make the FDA truly independent and potentially more science-backed.”
She also contemplated the benefits of Senate confirmation for Prasad and Tidmarsh, due to their current political standing: “If an individual is Senate approved, they then have to sit in the hot seat and answer questions from Congress, which can help balance things if they are viewed as disruptive.”
Though she notes that big reform could come, if necessary, Wood states that this is not the case yet, as CRLs and disruptions are not yet impacting the space to a concerning degree.
