MIT SCM Symposium 2024

Cell and Gene Therapy (CGT) treatment require significant upfront R&D investments coupled with lower patient demand volumes compared to traditional blockbuster drugs while not benefiting from the economies of scale principles for cost reduction. Therefore, ensuring the economic viability of a CGT treatment is one of the most pivotal considerations for pharmaceutical companies to balance between bringing life-saving innovation to the market and ensuring a sustainable business. In this work, we developed an integrated framework for early assessments of the economic viability of CGTs consisting of a high-level, manufacturer neutral cost model combined with a region and treatment/indication specific market demand pricing. In the context of specific oncology cancer indications in the US market, the integrated framework provides valuable economic viability assessment results coupled with the identification of key levers that influence product profitability within the cost model (batch, dose and donor yield) and market demand pricing model (standard of care, recurrence rate and survival rate). The proposed integrated framework enhances strategic decision making by providing early insights in the economic consequences of development- and commercialization scenarios to be used by pharmaceutical companies to bring life-saving innovative treatments to patients.