Introduction:
This article discusses the necessity for cell and gene therapy companies to not only enhance the efficiency of their manufacturing processes but also to effectively communicate the therapeutic value of their products to healthcare systems. Andrew Olaye from Orchard Therapeutics emphasizes the importance of considering both manufacturing costs and treatment efficacy when assessing the overall value of advanced therapies.
- Cell and gene therapy companies must establish the value of their therapies to healthcare systems while striving for improved manufacturing efficiency.
- Orchard Therapeutics, through its lead product Lenmeldy™, aims to treat a rare and severe disease, highlighting the challenges posed by low patient numbers in establishing cost-efficiency.
- Olaye underscores the transition from adherent-based to suspension-based cell cultures and automating drug manufacturing as key strategies to reduce costs.
- Despite cost reductions, the rarity of diseases treated limits the potential for economies of scale, necessitating a broader understanding of therapy value.
- Orchard Therapeutics is involved in newborn screening initiatives to identify eligible patients earlier, which supports their efforts to integrate treatments into national health programs.
Conclusion:
The challenges presented by rare diseases necessitate an evolving understanding of value in advanced therapies, which encompasses not only manufacturing efficiency but also presenting robust evidence of treatment benefits. Future strategies should emphasize the socio-economic impact of therapies to stakeholders within healthcare systems, ensuring a viable path for patient access and treatment implementation.
