McIntosh, Avery, Sverdlov, Alex, Yu, Li and Kaufmann, Petra (2021) Clinical design and analysis strategies for development of cell and gene therapies: quantitative drug development in the age of genetic medicine. Clinical pharmacology and therapeutics. pp. 1-9.

Abstract

Cell and gene therapies have shown enormous promise across a range of diseases in recent years. Numerous adoptive cell therapy modalities as well as systemic and direct to target tissue gene transfer administrations are currently in clinical development. The clinical trial design, development, analysis, and reporting of novel cell and gene therapies can differ significantly from established practices for small molecule drugs and biologics. Here we discuss important quantitative considerations and key competencies for drug developers in the preclinical, trial design, and lifecycle planning for gene therapies. We argue that the unique development path of gene therapies requires practicing quantitative drug developers—statisticians, pharmacometricians, pharamcokineticists, and medical and operational leads—to exercise active collaboration and cross-functional learning across development stages.

Item Type:	Article
Date Deposited:	04 Apr 2021 00:45
Last Modified:	04 Apr 2021 00:45
URI:	https://oak.novartis.com/id/eprint/44246