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Open Access 01-12-2018 | Research article

Stem cells and beta cell replacement therapy: a prospective health technology assessment study

Authors: Klemens Wallner, Rene G. Pedroza, Isaac Awotwe, James M. Piret, Peter A. Senior, A. M. James Shapiro, Christopher McCabe

Published in: BMC Endocrine Disorders | Issue 1/2018

Abstract

Background

Although current beta cell replacement therapy is effective in stabilizing glycemic control in highly selected patients with refractory type 1 diabetes, many hurdles are inherent to this and other donor-based transplantation methods. One solution could be moving to stem cell-derived transplant tissue. This study investigates a novel stem cell-derived graft and implant technology and explores the circumstances of its cost-effectiveness compared to intensive insulin therapy.

Methods

We used a manufacturing optimization model based on work by Simaria et al. to model cost of the stem cell-based transplant doses and integrated its results into a cost-effectiveness model of diabetes treatments. The disease model simulated marginal differences in clinical effects and costs between the new technology and our comparator intensive insulin therapy. The form of beta cell replacement therapy was as a series of retrievable subcutaneous implant devices which protect the enclosed pancreatic progenitors cells from the immune system. This approach was presumed to be as effective as state of the art islet transplantation, aside from immunosuppression drawbacks. We investigated two different cell culture methods and several production and delivery scenarios.

Results

We found the likely range of treatment costs for this form of graft tissue for beta cell replacement therapy. Additionally our results show this technology could be cost-effective compared to intensive insulin therapy, at a willingness-to-pay threshold of $100,000 per quality-adjusted life year. However, results also indicate that mass production has by far the best chance of providing affordable graft tissue, while overall there seems to be considerable room for cost reductions.

Conclusions

Such a technology can improve treatment access and quality of life for patients through increased graft supply and protection. Stem cell-based implants can be a feasible way of treating a wide range of patients with type 1 diabetes.

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Title: Stem cells and beta cell replacement therapy: a prospective health technology assessment study
Authors: Klemens Wallner
Rene G. Pedroza
Isaac Awotwe
James M. Piret
Peter A. Senior
A. M. James Shapiro
Christopher McCabe
Publication date: 01-12-2018
Publisher: BioMed Central
Published in: BMC Endocrine Disorders / Issue 1/2018
Electronic ISSN: 1472-6823
DOI: https://doi.org/10.1186/s12902-018-0233-7

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