Gene therapies have the potential to deliver transformative improvement in patient outcomes but challenge the status quo of value assessment by HTA bodies and affordability for health care systems.

In the value assessment of gene therapies, which typically target severe diseases, various challenges are uniquely combined. For example, for diseases with early-onset and a slow progression such as inherited retinal disease (IRD) X-linked retinitis pigmentosa, multiple issues exist in measuring and quantifying the quality of life over a long time horizon. Furthermore, while some gene therapies may have relatively large upfront costs, their potentially large long-term benefits for patients, informal carers, and families are uncertain. Their expected value is also affected significantly by the various discounting approaches used internationally. Understanding and, where possible, overcoming these and other value assessment challenges for gene therapies like IRD X-linked retinitis pigmentosa is critical for all concerned with value-driven innovation, payment, and access models.

This educational session will be focused primarily on those within the HEOR community that aim to expand their knowledge base regarding the challenges faced by gene therapies. It will provide an introductory overview of how traditional value assessment is challenged when evaluating gene therapies, using IRD X-linked retinitis pigmentosa as one example, and what solutions may exist based on learnings from various countries and/or other technologies. These challenges and solutions will be discussed by representatives from a patient organisation, an HTA-organisation, and industry. A moderated debate between the speakers and the audience will conclude the session.