Bringing Stem Cell‐Based Therapies for Type 1 Diabetes to the Clinic: Early Insights from Bioprocess Economics and Cost‐Effectiveness Analysis

Abstract

Differentiation of pluripotent stem cells (PSCs) into β cells could provide insulin independence for type 1 diabetes (T1D) patients. This approach would reduce the clinical complications that most patients managed on intensive insulin therapy (IIT) face. However, bottlenecks of PSC manufacturing and limited engraftment of encapsulated cells hinder the long‐term effectiveness of these therapies. A bioprocess decision‐support tool is combined with a disease state‐transition model to evaluate the cost‐effectiveness of the stem cell‐based therapy against IIT. Clinical effectiveness is assessed in quality‐adjusted life years (QALYs). Manufacturing costs per patient reduce from $430 000 to $160 000 with optimization of batch size and annual demand. For 96% of the patients, cell therapy improves the quality of life compared to IIT. Cost savings are achieved for 2% of the population through prevention of renal disease. The therapy is cost‐effective for 3.4% of patients when a willingness to pay (WTP) of up to $150 000 per QALY is considered. A 75% cost reduction in the cell therapy price increases cost‐effectiveness likelihood to 51% at $100 000 per QALY. This study highlights the need for scalable manufacturing platforms for stem cell therapies, as well as to prioritizing access to the therapy to patients with an increased likelihood of costly complications.

Stem Cell-Based Clinical Trials for Diabetes Mellitus

“…Emerging technologies ]such as stirred suspension bioreactor culture, wave bag bioreactor culture, multiplate culture, and roller bottle culture (32)] may eventually allow for mass production of stem cell-derived b cells and islet-like organoids. If a manufacturing expenses can be reduced, early health technology assessment studies demonstrated that stem cell-derived b-cell therapy will be a cost-effective (33,34).…”

Human pluripotent stem cell-derived insulin-producing cells: A regenerative medicine perspective

“…To achieve cost-effective manufacturing using industrial processes, synthetic small molecules are likely to replace purified proteins (Kieffer, 2016). Recent reports modeling the cost-effectiveness of hPSC-based therapy for T1D compared to intensive insulin therapy have highlighted the need for scalable manufacturing platforms for stem cell therapies (Bandeiras et al, 2019;Wallner et al, 2016).…”

The Feasibility and Applicability of Stem Cell Therapy for the Cure of Type 1 Diabetes

“…The encapsulation of SC-islet cells prior to transplantation allows them to detect blood glucose levels and secrete insulin while remaining isolated from the immune system of the recipient [ 101 ]. Bandeiras et al provided a cost-effectiveness analysis of encapsulated SC-islet cell transplantation that considered the cost of SC-islet cell production [ 102 ]. In their paper, the operational efficiency of the machine, labour, equipment, and reagent costs were taken into account assuming a demand of 50 patients per year, and the simulation resulted in a manufacturing cost per patient of about $80,000, which was higher than that for cadaveric islets [ 103 ].…”

“…However, the cost of reagents, especially those related to the differentiation stage, will continue to be relatively large. The results of a bioprocess model, which assumed a strategy of manufacturing products for 50 patients per year and 10 patients per machine, also assumed a total final cost of $650,000 per patient for the transplantation [ 102 ]. This model estimated that, compared with IIT, SC-islet cells transplantation would increase the quality-adjusted life years (QALYs) in 96.4% of the patients by an average of 3.73 years per patient over a 20-year patient period.…”