Regulatory Aspect of Pre-clinical Studies for Regenerative Medicine

Abstract

Regenerative cell therapy is a discipline in youth, with only limited experience in clinical practice. Once manufacturing protocols of cells have been established in vitro studies, the manufactured cells were assigned as candidates for cell-based medicinal products. To judge whether the cell-based medicinal products manufactured according to the established protocols should be valuable for further development for clinical practice, pre-clinical studies shall be conducted. In construction of a pre-clinical study package for cell-based medicinal products, the preclinical study package used for chemical compounds will help a lot. The requirements and contents of the pre-clinical studies are classified into toxicity studies (general toxicity studies and special toxicity studies), pharmacological studies (primary pharmacodynamics studies and safety pharmacological studies), pharmacokinetic studies, formulation studies, and others. The pre-clinical studies for the cell-based medicinal products are conducted to evaluate and qualify the efficacy and safety of the candidates. No wonder there is an argument about the selection of assessment parameters in toxicity, pharmacological and pharmacokinetics studies for cell-based medicinal product, because it is well-known that the cell-based medicinal products are different in properties from chemical compounds. Besides parameters used in these studies, the assessment issues may be an assumable minimum consensus, and others should be added on a case-by-case basis, depending on the cell manufacturing processes. In anticipation of the approval as the cell-based medicinal products, the possible requirements should be discussed here in the development process of such products from the standpoint of regulatory science, especially focusing on those in pre-clinical studies.