How to democratize cell and gene therapy: A global approach

Abstract

Over the past 25 years, the field of cell and gene therapy has grown substantially, with almost 50 therapies approved in the United States to treat severe, often life-threatening diseases. Despite clinical successes, the high costs and complex manufacturing and delivery requirements of cell and gene therapies (CGTs) present significant challenges to their broad use and equitable access. Disparities in access to CGTs range from financial constraints to infrastructure limitations and regulatory hurdles. As CGT trials expand globally, innovative strategies are needed to address these inequities, including alternative manufacturing models, harmonizing regulatory requirements, and innovative payment structures to replace the high up-front cost of current treatments. Addressing barriers like lack of infrastructure-stem cell processing and specialized personnel, for example-will be necessary to increase global CGT access and requires a multi-stakeholder approach. Academic, pharmaceutical, government, and nonprofit entities must cooperate to develop a more sustainable model for CGTs. Additionally, new university-led and public-private partnerships aim to facilitate access in underserved populations. To achieve equitable global access, a comprehensive strategy must involve innovative manufacturing, education, regulatory alignment, and stakeholder engagement, while ensuring that CGTs are both accessible and affordable to those who would benefit most from them.

Keywords: CAR-T cells; HIV; accessibility; cancer; cellular therapy; gene therapy; global; hemoglobinopathies; outreach; regulatory hurdles.