The pharmaceutical industry's focus on rare diseases has intensified, with 57% of all novel drugs approved in 2025 designated as orphan treatments. This is a continued increase from prior years, indicating a strategic shift towards smaller patient populations with high unmet needs, as exemplified by three different drugs for Hereditary Angioedema (HAE) being approved within ten weeks.

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.

In a striking reversal from 2024, large European pharmaceutical companies secured 13 FDA approvals while their eight largest US counterparts received only four. No single US company had more than one new drug approval, highlighting a significant performance gap and a potential shift in R&D pipeline productivity between the two regions.

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.

The FDA is abandoning rigid, fixed-length clinical trials for a "continuous" model. Using AI and Bayesian statistics, regulators can monitor data in real-time and approve a drug the moment efficacy is proven, rather than waiting for an arbitrary end date, accelerating access for patients.

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.

Richard Pazdur's immediate goal as the new CDER director is to restore stability and integrity at the FDA. His initial focus will be on rebuilding the team by recruiting, retaining, and empowering staff—deferring major policy shifts like accelerated approval reform until the agency's morale and operational capacity are restored.

By using big data for continuous, real-time post-market surveillance, the FDA can identify safety signals almost instantly. This robust safety net after a drug is launched paradoxically allows the agency to lower the evidence threshold required for initial approval, accelerating access to new cures.

The resignation of key figures like Peter Marks triggered a cascade of departures, leaving the FDA with a significant loss of long-term institutional knowledge. This creates uncertainty around the execution of new policies and guidance for the biopharma industry.