The European Medicines Agency (EMA) on Thursday opened for consultation a new guideline on the structure and data requirements for a clinical trial application for exploratory and confirmatory trials with advanced therapy investigational medicinal products (ATIMPs).
The 53-page guideline addresses development, manufacturing and quality control, featuring sections on quality documentation (including parts on the active substance and the investigational medicinal product), non-clinical documentation and clinical documentation.
According to the guideline, advanced therapy medicinal products (ATMPs) include gene therapy, somatic cell therapy medicinal products and tissue engineered products.
“In general, the development of an ATMP should follow the same general principles as other medicinal products,” EMA says. “However, it is acknowledged that the distinctive characteristics and features of ATMPs are expected to have an impact on product development.”
EMA advocates for a risk-based approach in determining the content of an investigational medicinal product dossier (IMPD), with applicants performing an initial risk analysis based on existing knowledge on the type of product and its intended use.
“Aspects to be taken into consideration include the origin of the cells, the type of vector and/or the method used for the genetic modification, the manufacturing process, the non-cellular components and the specific therapeutic use as applicable,” the guideline says.
And EMA warns that an immature quality development “may compromise the use of the study in the context of a marketing authorisation application (e.g. if the product has not been adequately characterised). A weak quality system may also compromise the approval of the clinical trial if the safety of trial subjects is at risk.”..