TESSEE – Tool for Early Stem Cells Economic Evaluation

Abstract

Stem cell therapies are promising for diverse indications. However, there are manufacturing and reimbursement challenges that must be addressed toward widespread adoption. Relying on bioprocess and/or health economics models, early health technology assessment (eHTA) has provided insights on manufacturing innovations towards reducing the manufacturing cost of goods (CoG), and the long-term value that prospective stem cell therapies must provide to secure reimbursement. These economics models have focused on commercial tools or proprietary code. In order to increase awareness to the usefulness of eHTA in stem cell engineering, this thesis presents a new open-source tool, TESSEE (Tool for Early Stem Cells Economic Evaluation). Three relevant case studies for mesenchymal stem/stromal cell (MSC) and pluripotent stem cell (PSC) based therapies are presented as applications of TESSEE for decision-making in the implementation of stem cell engineering innovations. A study on the choice of a culture media supplement for autologous MSC therapy manufacturing determined that human platelet lysate (hPL) reduces CoG, in comparison to fetal bovine serum (FBS), for 97% of donors. An expansion system focused case study showed that the implementation of a new vertical wheel reactor (VWR) for the microcarrier-based culture of MSC increases the number of cells per batch and reduces costs of goods per dose by up to 48% from typical two-dimensional flasks for expansion. eHTA of devices containing PSC-derived beta cells for treatment of type 1 diabetes patients showed that the transplantation of the devices could be very effective at improving the quality of life. However, a price reduction, accompanied by reduction of manufacturing costs, is required to achieve widespread cost-effectiveness. This thesis highlights the versatility of TESSEE and builds evidence for eHTA for rational implementation of innovations in stem cell engineering, towards more cost-effective stem cell therapies.