RE:FDA offers clinical trial design on Accelerated ApprovalSeptember 29, 2023 - FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies
This pilot program is further to the current FDA Accelerated Approval program and is intended for gene or cellular therapy ( like CAR-T therapy) to address an unmet medical need as a treatment for a rare disease or serious condition, which is likely to lead to significant disability or death within the first decade of life, such as rare neurodegenerative conditions, including those of rare genetic metabolic type.
https://www.fda.gov/news-events/press-announcements/fda-launches-pilot-program-help-further-accelerate-development-rare-disease-therapies