Putting a price tag on novel autologous cellular therapies

Abstract

Cell therapies, especially autologous therapies, pose significant challenges to researchers who wish to move from small, probably academic, methods of manufacture to full commercial scale. There is a dearth of reliable information about the costs of operation, and this makes it difficult to predict with confidence the investment needed to translate the innovations to the clinic, other than as small-scale, clinician-led prescriptions. Here, we provide an example of the results of a cost model that takes into account the fixed and variable costs of manufacture of one such therapy. We also highlight the different factors that influence the product final pricing strategy. Our findings illustrate the need for cooperative and collective action by the research community in pre-competitive research to generate the operational models that are much needed to increase confidence in process development for these advanced products. Key Words:Immunotherapy, Adoptive T Cell Therapy, Reimbursement, Commercialization, Regulation, Market Adoption, Cost of Goods, Good Manufacturing Practice, Scale-Up, Price Word Count: 2900 2In the past decade, there has been a rapid increase in the development of autologous cell therapies, with several investigational products demonstrating encouraging clinical outcomes, especially in immunotherapies. It has been recognized, for instance, that adoptive transfer of in vitro expanded virus-specific T cells can prevent and also effectively treat viral infectious complications in immunocompromised patients after solid organs (SOT) or hematopoietic stem-cell transplantation (HSCT) (1-4). Infectious complications that arise due to immunosuppression, that organ recipients need for the lifetime of the transplanted organ to prevent rejection, are mainly caused by the cytomegalovirus (CMV), BK virus, and the Epstein -Barr virus (EBV) (5). Although the adoption of universal antiviral prophylactic strategies has significantly reduced the incidence of CMV infection and disease, the development of drugresistant and late-onset CMV disease after discontinuation of these prophylactic antivirals is prone to high risk of malignancy, graft loss and mortality (6), and associated with a significant rise in treatment costs (7). Additionally, other serious adverse events such as nephrotoxicity and neutropenia can also result from the administration of antiviral agents (8). Thus, adoptive immunotherapies associated with lower toxicities for the prevention and treatment of CMV infection and disease are highly needed and may also produce overall cost savings in posttransplant patient care. Indeed, a recent study has suggested that even if the prevention capabilities of antiviral donor-derived cytotoxic T lymphocytes (CTL) in HSCT, which cost $10,000 to manufacture, would only be 50% effective at avoiding the need for antiviral treatment, it is still considered the less expensive option compared to the cost of antiviral treatment and associated hospital care of more than $50,000 per patient (9). Researchers working ...

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