From Pipeline to Pricing & Reimbursement: Economic Impact and Access Challenges of Upcoming Gene Therapies

Author(s)

Cooke E1, Orchard M2
1Cogentia, Cambridge, Cambridgeshire, UK, 2Cogentia, Cambridge, CAM, UK

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.

Conference/Value in Health Info

2024-11, ISPOR Europe 2024, Barcelona, Spain

Value in Health, Volume 27, Issue 12, S2 (December 2024)

Code

EE732

Topic

Economic Evaluation

Topic Subcategory

Budget Impact Analysis

Disease

Genetic, Regenerative & Curative Therapies, Rare & Orphan Diseases

Explore Related HEOR by Topic


Your browser is out-of-date

ISPOR recommends that you update your browser for more security, speed and the best experience on ispor.org. Update my browser now

×