Economics of Beta-Cell Replacement Therapy

Bandeiras, Cátia; Hwa, Albert; Ferreira, Frederico Castelo; Finkelstein, Stan N.; Gabbay, Robert A.

doi:10.1007/s11892-019-1203-9

Cited by 16 publications

(12 citation statements)

References 47 publications

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“…While their study suggested that the most cost‐effective method to produce human PSC‐derived pancreatic progenitors could be achieved with high volume suspension cultures, 1 the recommendation/pricing will change depending on ongoing breakthroughs of scale‐up technologies available. Similar bioprocess cost analyses have been performed by others 96‐98 . Bandeiras et al reported a comprehensive bioprocessing cost model for manufacturing beta cells from PSCs taking into account numerous variables such as the number of batches produced per year, the number of patients that could be implanted from cells generated per batch, and the number of patients that could implanted with the cells per year 96 .…”

Section: Demonstration Of Scalable Production Of Human Psc‐derived Pancreatic Progenitorsmentioning

confidence: 76%

“…Similar bioprocess cost analyses have been performed by others. 96 , 97 , 98 Bandeiras et al reported a comprehensive bioprocessing cost model for manufacturing beta cells from PSCs taking into account numerous variables such as the number of batches produced per year, the number of patients that could be implanted from cells generated per batch, and the number of patients that could implanted with the cells per year. 96 Based on their model, the projected cost of manufacturing beta cells for one patient could be as high as $427,231.…”

Section: Demonstration Of Scalable Production Of Human Psc ‐Derived Pancreatic Progenitorsmentioning

confidence: 99%

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Process Parameter Development for the Scaled Generation of Stem Cell-Derived Pancreatic Endocrine Cells

Iworima

Rieck²,

Kieffer

2021

Stem Cells Translational Medicine

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Section: Demonstration Of Scalable Production Of Human Psc‐derived Pancreatic Progenitorsmentioning

confidence: 76%

Section: Demonstration Of Scalable Production Of Human Psc ‐Derived Pancreatic Progenitorsmentioning

confidence: 99%

Process Parameter Development for the Scaled Generation of Stem Cell-Derived Pancreatic Endocrine Cells

Iworima

Rieck²,

Kieffer

2021

Stem Cells Translational Medicine

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“…In both cases, successful long-term engraftment is essential for the therapies to become profitable (30). The cost of stem cell-derived b-cell therapy will depend on a number of variables, including the requirement of immunosuppression, the duration of graft survival, and, most importantly, the optimization of the manufacturing process (31). While avoidance of autoimmune rejection will reduce the price, higher upfront costs are needed for the development of a scalable manufacturing process and the creation of stem cell banks for clinical use.…”

Section: Why the Need For Stem-cell Based Therapy In Diabetes?mentioning

confidence: 99%

Stem Cell-Based Clinical Trials for Diabetes Mellitus

Klerk

Hebrok

2021

Front. Endocrinol.

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Since its introduction more than twenty years ago, intraportal allogeneic cadaveric islet transplantation has been shown to be a promising therapy for patients with Type I Diabetes (T1D). Despite its positive outcome, the impact of islet transplantation has been limited due to a number of confounding issues, including the limited availability of cadaveric islets, the typically lifelong dependence of immunosuppressive drugs, and the lack of coverage of transplant costs by health insurance companies in some countries. Despite improvements in the immunosuppressive regimen, the number of required islets remains high, with two or more donors per patient often needed. Insulin independence is typically achieved upon islet transplantation, but on average just 25% of patients do not require exogenous insulin injections five years after. For these reasons, implementation of islet transplantation has been restricted almost exclusively to patients with brittle T1D who cannot avoid hypoglycemic events despite optimized insulin therapy. To improve C-peptide levels in patients with both T1 and T2 Diabetes, numerous clinical trials have explored the efficacy of mesenchymal stem cells (MSCs), both as supporting cells to protect existing β cells, and as source for newly generated β cells. Transplantation of MSCs is found to be effective for T2D patients, but its efficacy in T1D is controversial, as the ability of MSCs to differentiate into functional β cells in vitro is poor, and transdifferentiation in vivo does not seem to occur. Instead, to address limitations related to supply, human embryonic stem cell (hESC)-derived β cells are being explored as surrogates for cadaveric islets. Transplantation of allogeneic hESC-derived insulin-producing organoids has recently entered Phase I and Phase II clinical trials. Stem cell replacement therapies overcome the barrier of finite availability, but they still face immune rejection. Immune protective strategies, including coupling hESC-derived insulin-producing organoids with macroencapsulation devices and microencapsulation technologies, are being tested to balance the necessity of immune protection with the need for vascularization. Here, we compare the diverse human stem cell approaches and outcomes of recently completed and ongoing clinical trials, and discuss innovative strategies developed to overcome the most significant challenges remaining for transplanting stem cell-derived β cells.

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“…As time goes by, improvements in all facets of islet Tx procedure would lead to reduced costs and better outcomes meaning even more cost-effectiveness [94]. Studies such as these must be dependent on local costs and stakeholders willingness to pay the costs which explains their different findings [95]. Interesting to note, despite islet Tx not being found to be cost-effective in the Alberta study, allogeneic islet Tx is still offered to patients with the appropriate medical indication because it can provide better health outcomes to recipients regardless of cost.…”

Section: Willingness To Paymentioning

confidence: 99%

The Future of Clinical Islet Transplantation in the United States

Knöll¹,

Knoll²,

Bottino³

et al. 2021

OBM Transplantation

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Clinical islet transplantation was first realized over four decades ago at the University of Minnesota. Autologous islet transplantation is now widely recognized as a treatment to prevent diabetes in patients after pancreas excision and is offered at major transplant centers throughout the United States and the world. Type 1 diabetes represents a much larger demographic in which islet transplantation may benefit patients. Allogeneic islet transplantation can now offer similar outcomes to pancreas transplantation in a subset of patients with labile type 1 diabetes with less risk than whole organ transplantation. It is recognized as a standard of care in nations around the world but not in the United States, despite the important developmental role US scientists and physicians have played. Early reports of islet transplantation focused on insulin independence that proved to diminish over time. However, regardless of insulin status, islet transplantation provides benefits ranging from improved quality of life to reduction in diabetic complications. A National Institutes of Health sponsored multi-center Phase 3 Clinical Trial (CIT-07) demonstrated safety and efficacy, although the Food and Drug Administration chose to consider islets as a biologic that requires licensure, which makes offering the procedure in the clinic very challenging. Until regulations can be brought into communion with international standards, allogeneic islet transplantation in the United States is unlikely to match international levels of success and once promising programs are left to wither on the vine. Food and Drug Administration approval would open the door for third party medical reimbursement and allow many patients the opportunity to enjoy better health and quality of life. Establishment of clinical islet transplantation for type 1 diabetes would lead to optimizations in procedures making it more efficacious and cost effective while offering support for ongoing islet xenotransplantation studies that could bring islet transplantation to even more patients.

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Economics of Beta-Cell Replacement Therapy

Cited by 16 publications

References 47 publications

Process Parameter Development for the Scaled Generation of Stem Cell-Derived Pancreatic Endocrine Cells

Process Parameter Development for the Scaled Generation of Stem Cell-Derived Pancreatic Endocrine Cells

Stem Cell-Based Clinical Trials for Diabetes Mellitus

The Future of Clinical Islet Transplantation in the United States

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