Firth, I., Schirrmacher, H., Zhang, K., Towse, A. and Hampson, G.
Gene therapies represent a paradigm shift in medicine, with the potential to address the root causes of chronic diseases. They offer one-time treatment regimens and, in some cases, potentially a cure. As a result, they offer transformative value for patients, physicians, health systems and society. However, with the prospect of more gene therapy approvals there is concern in Europe that these technologies could threaten the financial sustainability of health systems.
In this paper we explore whether gene therapies can be sustainable for health systems, drawing from a literature review and a series of expert interviews. We review the value that gene therapies generate for patients and for health systems. To understand the variation in economic value that gene therapies offer, we present a categorisation that broadly groups therapies depending on whether they generate health gain primarily through length of life or quality of life and whether they generate cost offsets for the health system or wider society. We then discuss the short-term challenges of affordability and uncertainty in value for money of gene therapies and discuss how Managed Entry Agreements can be used to address these challenges. Finally we explore the factors that influence the financial sustainability of gene therapies: R&D pipeline, pricing and competition. We conclude that further research is needed to understand the practical barriers to the implementation of Managed Entry Agreements and that policy makers could support financial sustainability in future by supporting on-patent and off-patent competition.