US FDA has not had a new drug application focused advisory committee meeting in more than two months, and none are currently scheduled in 2024. After a blistering pace in the first half of 2023, FDA’s sudden slowdown in expert panels is perplexing.

There is a greater commitment among larger, well-capitalized oncology companies to complete confirmatory trials, Oncology Center of Excellence Director Richard Pazdur says; smaller company approach of first getting accelerated approval and then using commercial proceeds to capitalize confirmatory trial is ‘unacceptable.’

US FDA advisory committee members questioned how potential off-target effects with Vertex’s gene therapy for sickle cell disease should be conveyed to patients and families, and whether labeling should say anything about these theoretical risks.

CBER Director Peter Marks said the US may have to consider whether to maintain its ban on the practice of creating embryos to include heritable genetic modifications if other countries allow the practice.

US FDA is emphasizing the need to assess the effects of menopause on drug safety and efficacy as a continuation of the agency’s efforts to encourage clinical trial sponsors to look more systematically for differences in clinical trial subpopulations.