The Art Of Successful Gene Therapy Launches

The cellular and gene therapy development pipeline anticipates major transformations with hundreds of products moving forward toward approval. The 40 gene therapies projected to launch by the end of 2025¹ will shape the future treatment landscape for life-threatening disorders as well as common conditions. Navigating the evolving and complex healthcare data, policy, and regulatory requirements provides exciting opportunities and presents new challenges to patients’ access to treatment.

Patients who have or will receive human gene therapy products may need to be followed for up to 15 years to effectively evaluate potential long-term safety effects, due to the product design and how it modifies the host genome². With current gene therapies on the market targeted for rare and orphan diseases, and future approvals from regulators for more common conditions such as osteoarthritis and wet age-related macular degeneration, pressure will be on payers and policymakers to develop innovative payment models that can accommodate the associated high-costs.

Below are some important considerations for gene therapy manufacturers when approaching filing and commercialization.