Estimation of manufacturing development costs of cell-based therapies: a feasibility study

Abstract

Background aims: Cell-based therapies (CBTs) provide opportunities to treat rare and high-burden diseases. Manufacturing development of these innovative products is said to be complex and costly. However, little research is available providing insight into resource use and cost drivers. Therefore, this study aimed to assess the feasibility of estimating the cost of manufacturing development of two cell-based therapy case studies using a CBT cost framework specifically designed for small-scale cell-based therapies. Methods: A retrospective costing study was conducted in which the cost of developing an adoptive immunotherapy of Epstein-Barr virus-specific cytotoxic T lymphocytes (CTLs) and a pluripotent stem cell (PSC) master cell bank was estimated. Manufacturing development was defined as products advancing from technology readiness level 3 to 6. The study was conducted in a Scottish facility. Development steps were recreated via developer focus groups. Data were collected from facility administrative and financial records and developer interviews. Results: Application of the manufacturing cost framework to retrospectively estimate the manufacturing design cost of two case studies in one Scottish facility appeared feasible. Manufacturing development cost was estimated at £1,201,016 for CTLs and £494,456 for PSCs. Most costs were accrued in the facility domain (56% and 51%), followed by personnel (20% and 32%), materials (19% and 15%) and equipment (4% and 2%). Conclusions: Based on this study, it seems feasible to retrospectively estimate resources consumed in manufacturing development of cell-based therapies. This fosters inclusion of cost in the formulation and dissemination of best practices to facilitate early and sustainable patient access and inform future cost-conscious manufacturing design decisions.

Perspectives on the cost of goods for hPSC banks for manufacture of cell therapies

“…However, another study of a different T-cell adoptive therapy differed in that facility costs were highest (56% of total), with labor and materials both significantly less (i.e. ~20% each) 23 . It is important to recognize that these costs arise within the manufacture of an autologous cell product with bioprocessing far less complex than for an hPSCderivative.…”

“…the COGs for the hPSC system simply assessed costs for development of a master cell bank of an hPSC line (i.e. a starting material) and not an actual product 23 . However, this study calculated average costs for very early and late stages of preclinical development and revealed that both in the case of the hPSC bank and adoptive T cell therapy, facility costs decreased compared to labor costs as each process progressed towards clinical trial stage.…”

Current and Future Treatment of Retinitis Pigmentosa

“…Furthermore, manufacturing ATMPs requires skilled personnel and highly controlled development facilities, both of which are associated with high costs. 46 …”

“… 27 , 29 , 43 However, costs for the development of cell therapies can be driven higher by the requirements for highly specialized materials such as tissue culture, and material wastage can be high due to short expiration dates. 46 Gene therapies are also associated with additional complexities, including high development costs, requirements for genetic testing and the need for highly skilled and specially trained healthcare providers to perform the procedure, which can be associated with potential surgical complications. 27 …”

Stem Cells: Innovative Therapeutic Options for Neurodegenerative Diseases?

“…Indeed, the production phases must comply with specific GMP standards [98], which involve appropriate materials, equipment, personnel, and facilities. Specifically, a study conducted in 2020, which estimated manufacturing and development costs for cell-based therapies, pointed out that the expenses for PSCs production are around 500,000 s [112]. Moreover, pre-clinical studies and clinical trials have considerable costs too; for instance, Geron Corporation, which developed the world's first hESC product that entered clinical trials, had to invest about USD 200,000,000 in its hESC program, before FDA approval for a Phase I trial.…”