Pharmaceutical Statistics
Quantitative Dose Optimization for Gene Therapy Trials: Implications for Project Optimus and Early‐Phase Design
ABSTRACT Quantitative dose optimization in early phase clinical trials for investigational new drugs has been expanding across drug modalities and disease indications in response to the limitations of non‐quantitative or algorithmic methods of dose progression. In the context of the FDA's Project Optimus Initiative, which emphasizes dose optimization rather than reliance on the maximum tolerated dose, these challenges motivate the development of alternative quantitative frameworks tailored to gene therapies. In thi …