Regional disparities in access to gene therapies in the European Union, the United States, Japan, and China.

Abstract

Understanding the regional differences in approved gene therapies, clinical trial development, and regulatory frameworks is crucial for ensuring equitable access and addressing justice issues in advanced therapeutics. This review aimed to evaluate the differences between the US, the EU, Japan, and China and offer policy recommendations to promote harmonization between these countries and regions. Gene therapy approvals show significant regional disparities, with the US leading with 23 approved therapies, followed by the EU with 16. Few products are accessible worldwide reflecting challenges in obtaining cross-border approvals. Moreover, access is uneven within regions like the EU, with high-income countries having better accessibility. High costs and complex reimbursement processes exacerbate these issues, with some products being withdrawn from the market due to pricing disputes. Regulatory differences, such as differing data needs, further delay access in countries, like Japan, where gene therapy products are unavailable until years after a product is ready for approval. Clinical trial activity mirrors these disparities, with China's growing number of trials potentially reshaping the landscape. Harmonizing regulations across regions could streamline the approval process for therapies, making them more efficient and reducing disparities. Furthermore, key solutions include incentivizing cost reductions, adopting innovative payment models, and aligning evidence/reimbursement requirements.