CDER

Jacqueline Corrigan-Curay, the FDA's acting director of the Center for Drug Evaluation and Research, announced her retirement in July after more than eight years at the agency.

The FDA approved 18 new personalized medicines in 2024, representing 38% of all newly approved therapeutic molecular entities across multiple treatment areas including cancer and Alzheimer's disease.

• Recent FDA staffing cuts affecting 19% of the agency's workforce (3,500 employees) and leadership departures create significant uncertainty for pharmaceutical and biotechnology product development pipelines. • Drug developers are advised to pursue specialized regulatory pathways like fast track and breakthrough designations to ensure continued FDA engagement, while creating strategic teams to navigate regulatory decisions when guidance is unavailable. • Companies developing rare disease treatments, cell and gene therapies, and combination products face particular challenges as these areas typically require close FDA collaboration and communication.

The rare disease drug market shows significant expansion with FDA and EMA approving numerous treatments in 2024, offering hope to over 66 million patients in the US and EU combined.

The FDA's CDRH has initiated a pilot program mirroring the CDER's drug trials snapshots to enhance transparency in medical device clinical trials.

The SCOPE Summit 2024 in Orlando emphasized patient-centricity as the core focus for clinical trials, with industry leaders advocating for bringing trials to patients rather than expecting patients to seek trials.

The FDA is actively supporting the integration of artificial intelligence in clinical trials, with officials addressing industry concerns through newly released guidance and discussion papers.

A systematic review of FDA approval documents from 2019-2021 found that 85% of new drug and biologic approvals incorporated real-world evidence (RWE) in some form, with the proportion increasing from 75% in 2019 to 96% in the first half of 2021.