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.
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
A Doctoral Thesis. Submitted in partial fulfilment of the requirements for the award of Doctor of Philosophy of Loughborough University.