Delivering externally manufactured cell and gene therapy products to patients: perspectives from the academic center experience.

Abstract The recent success of the commercialization of CAR-T and other immune effector cells has led to the rapid expansion of clinical trials using cellular therapy products. The expansion of both investigational and commercially available cell therapies has been driven largely by products that are manufactured outside the point-of-care medical center by industry partners or other third parties. The delivery of externally manufactured products to patients requires a coordinated effort with the medical center, as it may be responsible for collection/processing of starting material, shipping, receipt, storage and release for administration of the drug product. As medical centers are grappling with increasing demands for supporting externally manufactured products, they have been forced to modify their processes to handle this demand in reactive rather than proactive fashion. The cell processing facility (CPF) plays a critical role to ensure proper handling and safety of the product as it is transported from the medical center to the manufacturer and back to the patient for infusion. In this mini-series, we have invited several CPFs from medical centers to share their experience, including how they have implemented processes and procedures to successfully ensure product integrity as they cooperate with industry/third parties to deliver novel cell therapy products. For the purpose of this mini-series, we focus on externally manufactured products that fall into the category of biologics that comprise human cells, tissues or cellular and tissue-based products (HCT/Ps) and are regulated under section 351 of the Food and Drug Administration's Public Health Service Act, or advanced therapy medicinal products (ATMPs) governed by regulatory bodies such as the European Medicines Agency. The goal of this collection of articles is to engage professionals in the discussion about issues related to externally manufactured products and, together, define best practices and potential standards for the cell therapy community to streamline the safety and delivery of externally manufactured products to our patients.