Making Advanced Therapies Affordable and Accessible: Two Strategic Approaches

This article explores two complementary strategies for addressing the affordability and access challenges facing advanced therapies. As high development costs and limited market access have led to the withdrawal of several therapies, the article examines how these barriers create 'valleys of death' that prevent innovation from reaching patients. Through the case of Glybera and other examples, it outlines a rehabilitative approach focused on reforming current systems through improved reimbursement schemes, regulatory streamlining, and more efficient manufacturing. It also presents a transformative approach that embeds affordability and access from the beginning of the research and development process, encouraging local innovation, equitable intellectual property practices, and adaptive regulatory frameworks. Lessons from the COVID-19 vaccine experience demonstrate the real-world potential of these strategies to ensure that promising therapies are not only developed, but also equitably delivered worldwide.