Challenges and S teps in the Development of AdvancedTherapy Medicinal Products

The introduction of advanced therapy medicinal products (ATMPs)to the global market has been revolutionizing the pharmaceuticalindus try, brings new ways for treating various incurablediseases. These products are developed in academic and/or hospital laboratories and need to be adjus ted in regards tothe s tandards of GMP manufacturing and transferred to specializedContract Manufacturing Organization or certain GMP facilities.The complexity of ATMPs brings various challenges,containing the source of s tarting and raw material, the technicalknow-how required to produce and related quality control ofthe products in the respect of regulatory and GMP compliance.Clinical development of ATMP encounters specific challengesdue to the nature of the product and the limited availability ofnon-clinical data. Validation of manufacturing and tes ting ofthese products particularly with regard to identity, purity, safetyand potency has great importance. The res tricted accessible appropriates tandards and reference material along with an inadequacyin certain guidelines are the other challenges.To translate a laboratory scale product to an indus trial scale weneed a quality management sys tem that includes Quality Assurance(QA) and Quality Control (QC) which should be appliedin all procedures in a GMP facility. The es tablishment andmaintenance of a quality management sys tem could assure thelong-term sus tainability of the product. Furthermore, qualitysys tems are necessary for the delivery of high-quality productsto the end-user.Inspection is the process of measuring, examining, and tes tingto check all characteris tics of a product or service and thecomparison of these with specified requirements to determineconformity. Products, processes, and various other results canbe inspected to make sure that the object coming off a productionline, or the service being provided, is correct and meetsspecifications.