BACKGROUND: In 2023, seven gene therapies were FDA approved, setting a record for the highest annual number in the past decade. Expectations for 2024 remain high, with an expanding clinical pipeline and pending regulatory approvals.

OBJECTIVES: There is growing uncertainty regarding the affordability and impact of funding increasing numbers of high-cost gene therapies. This study provides a thematic analysis on the short and long-term impacts of the current gene therapy pipeline on healthcare systems and payers.

METHODS: Cogentia analyzed 124 gene therapies in clinical development or approved between June 2023 and June 2024. Developments were categorized as positive (e.g. clinical advancement), limited/no development (e.g. no further news) or negative (e.g. discontinuation). Qualitative mixed methods were used to identify key themes and their implications for payers, and a matrix of factors impacting HTA was developed for five near-term gene therapies, including an assessment of expected budget impact.

RESULTS: Our analysis revealed positive developments for 52 of the 124 gene therapy assets, limited/no developments for 48 and negative developments for 24. Key themes include a shift towards targeting more prevalent indications, a desire for improved safety and administration regimes, and the de-prioritization of assets due to competitive dynamics. Analysis of five near-term gene therapies supports the hypothesis that budget impact concerns will continue to be of concern to payers, which in turn may support restricted recommendations.

CONCLUSIONS: Rare indications still dominate the pipeline, with an ever increasing price- Lenmeldy recently launched at $4.25m in the US- posing ongoing affordability and access challenges. However, the emergence of gene therapies targeting indications with larger prevalences, such as diabetes and Parkinson’s disease, alongside technological advancements (e.g. gene editing and re-dosing) may shift the gene therapy reimbursement and pricing expectations in the future.