Using the Cost–Effectiveness of Allogeneic Islet Transplantation to Inform Induced Pluripotent Stem Cell-Derived β-Cell Therapy Reimbursement

Archibald, Peter; Williams, David J.

doi:10.2217/rme.15.59

Cited by 11 publications

(8 citation statements)

References 35 publications

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“…Stem cell-derived b-cell therapy overcomes the barrier of limited donor availability, while also possibly representing a more costeffective therapy compared to exogenous insulin. Although the future cost of stem cell-derived b-cell therapy is unknown, a speculative cost-effectiveness analysis from an early health technology assessment study (29) calculates that 8-9 years after transplantation both cadaveric islet transplantation and stem cellderived b-cell transplantation would be more cost-effective than exogenous insulin therapy. This calculation assumes that the manufacturing costs of stem cell-derived b cells will be similar to the costs necessary to isolate cadaveric islets.…”

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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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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“…Furthermore, health economic simulation based on initial clinical experience with cell therapy in Parkinson's disease supports realizable cost-saving within a decade in the treatment of early onset degenerative conditions 90,91 . Similarly, stem cellderived ß cell therapy for diabetes has been predicted to provide cost-benefit over life-long insulin therapy within an 8-year horizon 92 . Notwithstanding, health economic evaluation of advanced therapies remains limited and inconclusive [93][94][95] .…”

Section: Economic Considerationmentioning

confidence: 99%

Longevity leap: mind the healthspan gap

2021

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Life expectancy has increased by three decades since the mid-twentieth century. Parallel healthspan expansion has however not followed, largely impeded by the pandemic of chronic diseases afflicting a growing older population. The lag in quality of life is a recognized challenge that calls for prioritization of disease-free longevity. Contemporary communal, clinical and research trends aspiring to extend the health horizon are here outlined in the context of an evolving epidemiology. A shared action integrating public and societal endeavors with emerging interventions that target age-related multimorbidity and frailty is needed. A multidimensional buildout of a curative perspective, boosted by modern anti-senescent and regenerative technology with augmented decision making, would require dedicated resources and cost-effective validation to responsibly bridge the healthspan-lifespan gap for a future of equitable global wellbeing.

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“…Cost‐utility analyses of IT have shown that IT is cost‐effective, albeit at high willingness‐to‐pay thresholds (e.g. Can$100 000), and long‐term 37–39 . These studies have compared IT to insulin therapy, which are not mutually exclusive or equivalent, and will have to be updated as procedural and immunosuppression costs decrease, as reports on long‐term outcomes become more prevalent, and as stem cell therapies enter the clinic 39 .…”

Section: Indications and Procedural Considerationsmentioning

confidence: 99%

Clinical islet transplantation: Current progress and new frontiers

Marfil‐Garza

Shapiro

Kin

2021

J Hepato Biliary Pancreat

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Islet transplantation (IT) is now a robust treatment for selected patients with type 1 diabetes suffering from recurrent hypoglycemia and impaired awareness of hypoglycemia. A global soar of clinical islet transplant programs attests to the commitment of many institutions and researchers to advance IT as a potential cure for this devastating disease. However, many challenges limiting the widespread applicability of clinical IT remain. In this review, we will touch on the milestones in the history of IT and its path to clinical success, discuss the current challenges around IT, propose some possible solutions, and elaborate on the frontiers envisioned in the future of clinical IT.

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Using the Cost–Effectiveness of Allogeneic Islet Transplantation to Inform Induced Pluripotent Stem Cell-Derived β-Cell Therapy Reimbursement

Cited by 11 publications

References 35 publications

Stem Cell-Based Clinical Trials for Diabetes Mellitus

Stem Cell-Based Clinical Trials for Diabetes Mellitus

Longevity leap: mind the healthspan gap

Clinical islet transplantation: Current progress and new frontiers

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