Cell therapy manufacturing value systems and cost analysis

Cell Therapies are promising clinical instruments with significant therapeutic potential and commercial promise. However, the industry engaged in their commercial and clinical development faces significant financial, technical, regulatory and market challenges. These challenges are compounded by an understanding gap in the cell therapy industry. Commercial failures and financial difficulties have forced the industry to address the need to provide value and estimate and control costs early in the development timeline. The problem is that this issue is not being systematically or thoroughly addressed in the academic community while they pursue potential future treatments. Articles that highlight the need to understand costs and value are appearing with increasing frequency highlighting a growing consensus that work needs to be carried out in this area. However examples of models and tools to predict or estimate or even calculate costs in developing and producing a product do not exist in the literature. This work consists of three parts. Part one entails a new model of the characteristics observed in cell therapy new product development. This model is an evolution of an activity based dependency structure matrix (DSM). Result from the model suggests that some favoured development strategies (such as applying for an orphan indication status) provide less financial benefit than is commonly expected. The ability to scale manufacturing levels between clinical trial phases is also a pressing problem. 3 Part two presents a model to predict the cost of manufacturing and delivering a cell therapy product. This cost of good supplied (COGS) model combines both rules and predictive activity based costing across multiple manufacturing platforms, cell types and supply chain configurations. This model highlights the significant cost burden of validating both single and, more markedly, multiple sites of manufacture. The model also examines the potential for economies of scale when using different production technology in the manufacture of human Mesenchymal Stem Cells. Based in part on the results and knowledge gleaned in parts one and two, part three outlines the development of a novel, scalable expansion system developed to enable lower cost, controlled manufacture of adherent cell populations. While still at an early stage of development the technology has demonstrated the ability to maintain cells in a high rate of growth for a longer period than traditional culture techniques. This allows for the creation of a manufacturing technology with a higher expansion ratio than manufacturing systems on the market today.