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Cited by 19Pancreatic function and diabetesHealth Systems, Economic Evaluations, Quality of LifeDiabetes Management and Research

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

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Wallner, Klemens, Pedroza, Rene G., Awotwe, Isaac, Piret, James M., Senior, Peter A., Shapiro, A. M. James, McCabe, Christopher·2018·BMC Endocrine Disorders
DOI 10.1186/s12902-018-0233-7PMC PMC5791348
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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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  • Development of a large-scale, cost-efficient manufacturing methodology for differentiating SC-islets will be necessary to make this therapy available to a substantial number of patients.

    “Development of a large-scale, cost-efficient manufacturing methodology for differentiating SC-islets will be necessary to make this therapy available to a substantial number of patients.174”

    From Developments in stem cell-derived islet replacement therapy for treating type 1 diabetes (2023) · cites this paper as [174]

    The Methods sections on scale of manufacturing and cost-of-goods modeling compare production at 50 and 500 doses per year and estimate lower per-dose costs at the higher volume. But the Discussion section on efficient treatment delivery cautions that high-volume production must be weighed against demand, clinical capacity, and the cells’ short shelf life. The paper does not establish that a large-scale, cost-efficient differentiation method is necessary to make SC-islets available to a substantial number of patients; it models pancreatic-progenitor implants rather than fully differentiated SC-islets. (LLM, conf=0.84)

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References 37 · Cited by 19

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Authors
Wallner, K., Pedroza, R. G., Awotwe, I., Piret, J. M., Senior, P. A., Shapiro, A. M. J., & McCabe, C.
Affiliations
  • University of Alberta (CA)
  • University of British Columbia (CA)
Corresponding author
Wallner, Klemens
ORCID
Venue
BMC Endocrine Disorders
Publisher
Springer Science and Business Media LLC
Published
2018-01-30
Volume
18
Issue
1
Pages
6-6
ISSN
1472-6823 (ISSN-L)
DOI
10.1186/s12902-018-0233-7
PMC
PMC5791348
OpenAlex
W2795465898
Cited by
19
References
37
Citations by year
2026: 2 · 2024: 2 · 2023: 2 · 2022: 3 · 2021: 4 · 2019: 3 · 2018: 3
FWCI
2.60
Type
journal article
Language
en
Open access
Licence
cc-by
Locations
  • BMC Endocrine Disorders (publishedVersion, cc-by)
  • PubMed Central (publishedVersion, cc-by)
  • PubMed (publishedVersion)
  • cIRcle (University of British Columbia) (submittedVersion, cc-by)
  • DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
Funders
  • Alberta Innovates - Health Solutions (201201154)
  • Stem Cell Network ((9/5261(PP5)), NCESCN CTRA FY17/CT5)
  • Capital Health
  • Canada Research Chairs
Topics
  • Health Sciences › Medicine › Surgery › Pancreatic function and diabetes
  • Social Sciences › Economics, Econometrics and Finance › Economics and Econometrics › Health Systems, Economic Evaluations, Quality of Life
  • Health Sciences › Medicine › Endocrinology, Diabetes and Metabolism › Diabetes Management and Research
Keywords
stem cell-derived beta cells, beta cell replacement therapy, type 1 diabetes, cost-effectiveness, pancreatic progenitor cells, islet transplantation, intensive insulin therapy, cell culture, willingness-to-pay threshold
MeSH terms
Cost-Benefit Analysis, Diabetes Mellitus, Type 1, Humans, Life Expectancy, Stem Cells, Technology Assessment, Biomedical, Islets of Langerhans Transplantation, Quality-Adjusted Life Years, Stem Cell Transplantation, Insulin-Secreting Cells

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