Clinical Trials Arena September 25, 2025
The draft guidance sets out six alternative trial designs that sponsors could adopt in CGT studies.
The US Food and Drug Administration (FDA) has released guidance about innovative trial designs for cell and gene therapy (CGT) studies.
In the eight-page draft guidance, titled ‘Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations’, the agency said the framework is a response to the Prescription Drug User Fee Act (PDUFA) VII commitment to increase efficiency in the development of CGT products. The draft guidance was released on 24 September 2025.
The draft guidance reads: “Given the urgent need for safe and effective products to treat serious and severely debilitating diseases in small populations, FDA recognises the importance...
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