Process change evaluation framework for allogeneic cell therapies: impact on drug development and commercialization

• Published in: Regenerative medicine 2016-04, Vol.11 (3), p.287-305
• Main Authors: Hassan, Sally, Huang, Hsini, Warren, Kim, Mahdavi, Behzad, Smith, David, Jong, Simcha, Farid, Suzanne S
• Format: Article
• Language: English
• Subjects: allogeneic cell therapy manufacture; Allografts; Cell- and Tissue-Based Therapy - economics; cost of goods; Costs and Cost Analysis; drug development costs and cash flow; Drug Evaluation, Preclinical - economics; Humans; Models, Economic; process change; Stem Cell Transplantation - economics; Stem Cells
• ISSN: 1746-0751; 1746-076X
• DOI: 10.2217/rme-2015-0034

Some allogeneic cell therapies requiring a high dose of cells for large indication groups demand a change in cell expansion technology, from planar units to microcarriers in single-use bioreactors for the market phase. The aim was to model the optimal timing for making this change. A development lifecycle cash flow framework was created to examine the implications of process changes to microcarrier cultures at different stages of a cell therapy's lifecycle. The analysis performed under assumptions used in the framework predicted that making this switch earlier in development is optimal from a total expected out-of-pocket cost perspective. From a risk-adjusted net present value view, switching at Phase I is economically competitive but a post-approval switch can offer the highest risk-adjusted net present value as the cost of switching is offset by initial market penetration with planar technologies. The framework can facilitate early decision-making during process development.