FDA Approvals on Track Despite Turbulent Year, Rare Disease Focus Continues

The pharmaceutical industry has weathered a tumultuous year marked by leadership changes and policy shifts at the FDA, yet drug approvals remain consistent with recent trends. As the fourth quarter begins, a comprehensive analysis reveals that the regulatory landscape, while chaotic, has not significantly impacted the approval process for new medications.

Approval Numbers Hold Steady Amid FDA Upheaval

Despite the exodus of senior leadership and the elimination of 3,500 staff positions at the FDA, drug approvals for the first three quarters of 2025 are in line with previous years. According to a Jefferies analysis, 28 approvals were recorded by late August, with projections suggesting a year-end total of 43—well within the range of 37-68 approvals seen over the past five years.

"Fears of a dysfunctional FDA may be overblown," Jefferies analysts reported to investors. "We think the FDA's actions YTD have not signaled a more stringent or irrational agency."

The Center for Drug Evaluation and Research (CDER) is on track to issue fewer Complete Response Letters (CRLs) compared to the past five years, while the Center for Biologics Evaluation and Research (CBER) is trending towards more CRLs.

Rare Disease Initiatives Show Progress

FDA Commissioner Marty Makary's prioritization of rare disease therapies is bearing fruit, with 14 novel treatments for rare conditions approved this year. These include therapies for alkaptonuria, recessive dystrophic epidermolysis bullosa, and phenylketonuria.

The agency has introduced new frameworks to expedite approvals for ultra-rare diseases, including the Rare Disease Evidence Principles. However, the path hasn't been smooth for all rare disease treatments, with seven receiving CRLs, some unexpectedly.

Oncology leads in novel drug approvals, followed by treatments for infectious diseases and autoimmune/immunological conditions. The FDA's commitment to rare diseases is evident, with Jefferies analyst Andrew Tsai noting, "They seem to be following it through with what they want to achieve, especially within the rare disease side, and expediting drugs and so forth."

Political Transitions and Regulatory Consistency

The transition to a new administration has not significantly altered the FDA's approval patterns, a trend consistent with historical data. Steven Grossman, a policy and regulatory consultant, explains, "You would not expect a flood of new drug approvals in the inaugural year of a new administration."

The current U.S. government shutdown adds complexity to the regulatory landscape, with the FDA unable to accept new drug applications until the situation resolves. This temporary halt in new submissions may impact the final approval tally for 2025.

As the industry navigates these challenges, the FDA's ability to maintain consistent approval rates amidst internal changes and external pressures demonstrates the resilience of the drug review process. The focus on rare diseases and the steady pace of approvals suggest that despite the turmoil, the agency continues to fulfill its critical role in bringing new therapies to patients.