TY - JOUR
T1 - Estimation of manufacturing development costs of cell-based therapies
T2 - a feasibility study
AU - ten Ham, Renske M.T.
AU - Nievaart, Julianne C.
AU - Hoekman, Jarno
AU - Cooper, Rachel S.
AU - Frederix, Geert W.J.
AU - Leufkens, Hubert G.M.
AU - Klungel, Olaf H.
AU - Ovelgönne, Hans
AU - Hoefnagel, Marcel H.N.
AU - Turner, Marc L.
AU - Mountford, Joanne C.
N1 - Funding Information:
No funding was received. The authors have no commercial, proprietary or financial interest in the products or companies described in this article. Conception and design of the study: RtH, JH, GF, OH, HO, MH, JM and MT. Acquisition of data: JN, RC, JM, MT and JN. Analysis and interpretation of data: JN, RC, JM, MT and JN. Drafting or revising the manuscript: RtH, JN, RC, JH, GF, OH, HO, JM and MT. All authors have approved the final article.
Publisher Copyright:
© 2021 International Society for Cell & Gene Therapy
PY - 2021/8
Y1 - 2021/8
N2 - 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.
AB - 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.
KW - cell therapies
KW - cell-based therapies
KW - costs
KW - development
KW - manufacturing
UR - http://www.scopus.com/inward/record.url?scp=85100990219&partnerID=8YFLogxK
U2 - 10.1016/j.jcyt.2020.12.014
DO - 10.1016/j.jcyt.2020.12.014
M3 - Article
C2 - 33593688
AN - SCOPUS:85100990219
SN - 1465-3249
VL - 23
SP - 730
EP - 739
JO - Cytotherapy
JF - Cytotherapy
IS - 8
ER -