Gene therapies have the potential to provide transformative treatments for monogenic conditions, but they often come with extremely high price tags. This creates significant challenges in gaining reimbursement approval from payers and health technology assessment bodies.
Key takeaways
- Patient population heterogeneity matters. Companies should identify subgroups where the proposed pricing aligns with the costs of current standard of care.
- Early incorporation of health economics and outcomes research (HEOR) is critical. Proactively addressing evidence needs for cost-effectiveness and value demonstration can bridge the gap with payers.
- Real-world evidence, translatable endpoints, and demonstrating durability help build payer confidence in the therapy's value proposition.
- Creative reimbursement models like value-based agreements may be needed for broader patient populations.
Developing strategies that integrate clinical, economic, and payer evidence needs from the start is crucial for gene therapy developers to maximize patient access and recoup their significant investment costs. Early HEOR planning can make or break a therapy's commercial success.
For more information about addressing payer considerations and incorporating health economics into gene therapy development strategies, read the full article.
