Decisions in the Development Lifecycle of Cell and Gene Therapies

Making better-informed decisions throughout the lifecycle of the development of cell- and gene-based therapies is crucial for their successful translation to the market. At the research and development phase, the choice of cell source can have a major impact on subsequent regulatory hurdles. The chosen indication will have substantial impact on the market size and risk–benefit profile. Each stage of the product development cycle faces its own risks and challenges, and proper management of these risks is vital for successful commercialisation. In the early phases of development, the investment risk and clinical trial uncertainties should be considered to ensure proper planning. Notably, when planning manufacturing capacity, the risks of outsourcing versus in-house manufacturing can inform major long-term decisions, such as whether or not to start building a facility. During manufacturing, risks such as batch loss to equipment or human failure should be considered to inform decision-making. The composite of these factors can impact whether or not to adopt automation strategies. Understanding the risks involved throughout the product development cycle of biomaterial allows industry practitioners to make better-informed decisions in investment, manufacturing and regulatory aspects of commercialisation of cell and gene therapies. This chapter introduces a modelling approach to tackling different decisions along the product development lifecycle and discusses various methods used in published literature.