Despite widespread concern about political disruption at the FDA, key metrics for innovation in new drug approvals—such as first-in-class drugs and new targets—were almost completely flat in 2025 compared to previous years. This suggests the core regulatory engine has remained consistent, for now.
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While political drama at the top of the FDA captures headlines, the agency's rank-and-file reviewers are largely maintaining operational continuity. Many drug programs are still receiving necessary feedback within expected timeframes, suggesting the core machinery of the FDA is resilient.
Despite massive turnover and internal dysfunction at the FDA, biotech investors have largely shrugged off the regulatory uncertainty. This disconnect suggests the market believes the negative impacts, like drug review delays, are a lagging indicator that won't materialize immediately, creating a potential future risk for the sector.
Amidst growing turmoil at the FDA, a viable strategy is to "invest around" the risk. This involves prioritizing companies whose drugs show clear data on well-understood, validated endpoints, as these are most likely to navigate the current political environment successfully, regardless of leadership changes.
Unicure's setback with its Huntington's gene therapy demonstrates a new political risk at the FDA. A prior agreement on a trial's design can be overturned by new leadership, especially if the data is not overwhelmingly definitive. This makes past regulatory alignment a less reliable indicator of future approval.
A key trend in 2025's drug approvals is that "best-in-class" therapies are distinguished not just by efficacy, but by innovations in formulation and delivery that improve the patient experience. Examples include subcutaneous versions of IV drugs and new delivery methods that expand patient access.
The key risk facing biomedical innovation is not just policy chaos, but the normalization of political and ideological influences on science-based regulation. This includes CEOs negotiating prices with the president and FDA enforcing pricing policies, breaking long-standing norms that separated science from politics.
The biotech industry is entering a paradoxical period. Financial markets show signs of recovery with rising follow-ons and potential IPOs, suggesting a bear market end. However, this optimism is contrasted by significant uncertainty and political turmoil at key US agencies like the FDA and NIH, creating a challenging operating environment for innovation.
The current disconnect between the FDA leadership's public calls for flexibility and its divisions' strict actions is not new. For decades, the agency's hierarchy has acted as a promotional arm to encourage industry, while the review divisions have maintained a more conservative, old-school approach to rigor. This historical pattern is often overlooked.
The FDA's current leadership appears to be raising the bar for approvals based on single-arm studies. Especially in slowly progressing diseases with variable endpoints, the agency now requires an effect so dramatic it's akin to a parachute's benefit—unmistakable and not subject to interpretation against historical data.
Industry sentiment on the FDA is not monolithic. A recent survey reveals that while biotechs largely maintain confidence in the agency's hardworking staff and their day-to-day interactions, there is deep concern and a lack of trust in the agency's top leadership. This nuanced view highlights that the perceived problems are rooted in politicization and leadership competence, not frontline operations.
