Abstract
Cell-based therapies must achieve clinical efficacy and safety with reproducible and cost-effective manufacturing. This study addresses process development issues using the exemplar of a human pluripotent stem cell-based dopaminergic neuron cell therapy product. Early identification and correction of risks to product safety and the manufacturing process reduces the expensive and time-consuming bridging studies later in development. A New Product Introduction map was used to determine the developmental requirements specific to the product. Systematic Risk Analysis is exemplified here. Expected current value-based prioritization guides decisions about the sequence of process studies and whether and if an early abandonment of further research is appropriate. The application of the three tools enabled prioritization of the development studies.
Highlights
To ensure comparability of manufacturing process at commercial scale there must be manufacturing research sufficient to satisfy the ‘Chemical, Manufacturing and Control’ section of the regulatory application dossier. This must occur at a stage in the project cycle early enough to avoid unnecessary process re-engineering late in development
By no means all candidate products succeed at clinical trial and it is desirable to avoid spending money unnecessarily on process research for these putative products
The dilemma can be addressed through the careful use of three decision-making tools
Summary
Names shown in the second row indicate respective processes developed by the researchers detailed in Figure 4. ’–’ indicates no data available for the particular marker for that particular process. Where: C = Cost of commercialization or launch D = Cost of development ECV = Expected current calue NPV = Net present value PCS = Probability of commercial success PTS = Probability of technical success We can manage the research using simple risk models based upon the project plan This approach takes as its tenets the ideas that each study allows a decision to be made. The model returns a value of $2.020 million which, adjusted for the probability of success (30% of this arm) becomes $0.606 million In this case there is no previous decision point and the discounted cost of reaching this decision point is the sum of the first two years of research ($1,068,240) so the project as a whole has a negative value ($462,240) Figure 9
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