Hassan Sally, Huang Hsini, Warren Kim, Mahdavi Behzad, Smith David, Jong Simcha, Farid Suzanne S
Department of Biochemical Engineering, The Advanced Centre for Biochemical Engineering, University College London, Gordon Street, London, WC1H 0AH, UK.
Graduate Institute of Public Affairs & Department of Political Science, National Taiwan University, No. 1, Sec. 4, Roosevelt Road, Taipei, 10617 Taiwan.
Regen Med. 2016 Apr;11(3):287-305. doi: 10.2217/rme-2015-0034. Epub 2016 Mar 16.
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.
MATERIALS & METHODS: 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.
一些针对大规模适应症群体需要高剂量细胞的同种异体细胞疗法，在进入市场阶段时，要求细胞扩增技术从平面培养单元转变为一次性生物反应器中的微载体培养。目的是模拟做出这种转变的最佳时机。
创建了一个开发生命周期现金流框架，以研究细胞疗法生命周期不同阶段向微载体培养转变过程所产生的影响。
在该框架所采用的假设条件下进行的分析预测，从预期的总自付成本角度来看，在开发早期进行这种转变是最佳的。从风险调整后的净现值角度来看，在一期进行转变在经济上具有竞争力，但批准后进行转变可提供最高的风险调整后净现值，因为转变成本被平面技术的初始市场渗透率所抵消。
该框架可促进工艺开发过程中的早期决策。
