Health & Economic Impact Program

Gene, cell and tissue therapies (CGTx) offer the potential to dramatically change treatment options, patient outcomes and healthcare costs for a multitude of diseases. Many of these therapies are one-time, single administration treatments that have the potential to cure or significantly improve health for patients presenting with the most intractable – and costly — conditions. If similar results are achievable across multiple disease categories, the accelerated introduction and broader access to these therapies will have a huge impact on the healthcare economy.

But how can we quantify it?

The Foundation recognizes that presently there is no framework commonly accepted among health technology assessment agencies (“HTAs”), payers, and other stakeholders for modeling the burden of disease and clinical value as it relates to the therapeutic potential of cell and gene medicine. Further, many disease groups and companies are challenged by the need to develop their own disease models without clear guidance as to the type of model or framework that would most accurately reflect the relevant elements and metrics of disease burden and potentially corresponding CGTx value.

The HEI Program Steering Committee believes it is possible to create a consensus framework for modeling burden of disease that could be addressed by CGTx using modeling tools accepted by the HTAs and validated through a series of pilot projects under the auspices of the HEI Program. To that end, the Steering Committee has worked with Evidera, the leading pharmacoeconomic modeler in North America and Europe, to develop a modeling method and platform