FDA Grand Rounds: FDA Menopausal Hormone Therapy (MHT) Labeling: Historical Context & Recent Changes
The FDA Office of Women’s Health hosted a Grand Rounds session to review recent changes to menopausal hormone therapy (MHT) labeling, tracing the evolution from early 20th‑century products to today’s boxed warnings. Speakers Dr. Nicholas Hazen and Dr. Aisha Johnson outlined how the 2002 Women’s Health Initiative (WHI) trials reshaped understanding of MHT. The large‑scale, double‑blind study of over 27,000 women revealed a 26 % relative increase in breast cancer and heightened risks of stroke, deep‑vein thrombosis, and pulmonary embolism, while showing modest protection against hip fracture and colorectal cancer. The presenters highlighted that the WHI cohort averaged 63 years—far older than typical new users—explaining why early observational data had suggested cardiovascular benefits that the trial did not confirm. The FDA’s 2003 class‑wide labeling overhaul added a black‑box warning covering thromboembolic events, myocardial infarction, invasive breast cancer, and probable dementia, prompting an 18 % drop in prescriptions within three months and a 30 % decline by year’s end. These regulatory updates force clinicians to individualize therapy, emphasizing non‑hormonal options for vasomotor symptoms when risks outweigh benefits. The shift also signals ongoing scrutiny of hormone products and underscores the need for continued post‑marketing surveillance and patient education.
Conversations on Cancer: Patient First = Project Facilitate
The Oncology Center of Excellence’s Project Facilitate, launched at ASCO in 2019, provides a centralized, patient‑first service that helps physicians navigate single‑patient INDs and expanded‑access pathways for investigational oncology therapies. Panelists explained how the program evolved from a simple...
CDER & CBER All Hands: Implementing the FDA's Plausible Mechanism Framework
The FDA held its first joint CDER‑CBER all‑hands to unveil the Plausible Mechanism Framework, a regulatory approach designed to keep pace with rapid advances in individualized medicine. Speakers highlighted the landmark case of baby KJ, whose successful gene‑editing therapy...
Advancing Real-Time Clinical Trials
The FDA announced a landmark initiative: the first ever real‑time clinical trial, where regulators can monitor safety signals and efficacy endpoints instantly via a cloud‑based dashboard. Commissioner Dr. Marty Makary framed the effort as a response to the growing "dead...
FDA Grand Rounds: Clinical Omics Biomarker Discovery and Validation in Precision Medicine
The FDA Grand Rounds session featured Dr. Richard Beger discussing clinical omics biomarker discovery and validation for precision‑medicine applications. He outlined the breadth of systems‑biology omics—genomics, transcriptomics, proteomics, metabolomics, lipidomics—and described a structured workflow that stresses early sample‑type decisions, rigorous...