The Food and Drug Administration's role in supporting therapeutic development to address unmet needs: the National Academies report and beyond.

Despite substantial progress, many serious diseases continue to lack safe and effective therapies. Building on a recent National Academies consensus study report that offers several recommendations to better align investments in therapeutic development with unmet needs, in this commentary, we focus on the Food and Drug Administration's role in advancing this goal. Historically, FDA has encouraged investment where market incentives fall short, undertaken efforts to address broad scientific challenges, and administered programs to expedite development and review of drugs targeting unmet needs. Under the second Trump administration, FDA has launched several initiatives that continue these key themes, although some have been controversial, primarily due to concerns about resource sufficiency and the potential for political interference. Regarding other domains addressed by the National Academies report, FDA has recently given mixed signals on its commitment to upholding rigorous approval standards; paid limited attention to noted gaps, including improving rigorous postmarket evidence generation and validating the surrogate markers often used to support product approval; and failed to ensure adequate financial and human resources to support the agency's critical work. Engagement with the National Academies report's recommendations can help ensure that FDA continues to advance progress toward addressing unmet medical needs.