In March 2024, the FDA approved a groundbreaking gene therapy for children with a specific form of leukodystrophy, offering hope to those with no current care options. However, the approval came with a hefty price tag, making headlines as the most expensive medicine in the world at $3,500,000 per patient. With the cost of innovation so high, what can we do to de-risk development, ensure affordability and achieve equitable access to these potentially life-changing therapies for the long term?

Nick Kenny, CSO for Syneos Health and host for this episode, is joined by colleagues Abhi Gupta, Head of Genetic Medicine, and Dr. Prateet Minhas, Managing Director, Value & Access Consulting, to explore the evolution of genetic medicines, from the early days of clinical trials fraught with challenges to the recent surge in approvals and the promise of curative potential. Abhi and Prateet discuss the landscape for genetic medicines today, touching on topics such as manufacturing challenges, regulatory considerations, and the importance of demonstrating durability of response in clinical development.

As they navigate the complexities of the field, they address key questions surrounding the value proposition of genetic medicines, including their potential to transform patient outcomes and the challenges of reimbursement and access. They also examine the role of collaboration and innovation in driving progress in the field, highlighting the need for a balanced approach that prioritizes both scientific advancement and real-world impact.

For more from our cell & gene therapy experts, check out these insights:

BLOG: Three Lessons Learned in Adoptive Cell Therapy Trials: A Look at the Road Ahead for Immunology

BLOG: A Holistic Approach to Genetic Medicines is Next for the Life Science Industry

Exploring an Expedited Regulatory Pathway for Cell and Gene Therapies

BLOG: Cell and Gene Therapy Sponsors Must Overcome Unique Hurdles to Realize Promise to Patients 

The views expressed in this podcast belong solely to the speakers and do not represent those of their organization.

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