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Tracy Beth Høeg

Tracy Beth Høeg

Acting Director, Center for Drug Evaluation and Research (CDER)

Appears in 1 story

Notable Quotes

"The Plausible Mechanism draft guidance creates a novel framework through which cutting-edge treatments tailor-made for patients with ultra-rare diseases can be used as a basis for FDA approval. We anticipate it will inspire industry to place increased focus on individualized therapies, thereby driving innovation, improving safety, lowering costs, and offering more patients with ultra-rare diseases a unique shot at a life-saving treatment." — FDA press announcement, February 2026

Stories

FDA creates new approval pathway for gene therapies tailored to individual rare disease patients

Rule Changes

Leading CDER's role in the new framework

For decades, the Food and Drug Administration (FDA) required the same basic proof for every drug: show it works in a controlled trial with enough patients to be statistically meaningful. That standard made sense for common diseases but created an impossible barrier for conditions affecting a handful of people worldwide. On February 23, 2026, the FDA issued draft guidance creating a fundamentally different standard—called the "plausible mechanism" framework—that would let developers of individualized gene-editing and ribonucleic acid (RNA) therapies win full approval by demonstrating their treatment targets the root genetic cause, successfully edits or engages the target, and improves outcomes compared to the disease's documented natural course.

Updated Feb 23