OBJECTIVES

Gene therapies promise a potentially life-long cure for many cancers and rare diseases. However, the path to market access success is challenging. This study evaluated the preparedness of several European countries (the European Union 5 [EU5], the Netherlands, Sweden, and Poland) for gene therapies based on three components: budget and infrastructure; finance, reimbursement and implementation; and health technology assessment (HTA) and pricing.

METHODS

The research was organised into three main components, comprising 22 indicators of gene therapy preparedness in total. Literature searches (including health policy documents and academic literature) and secondary research were conducted. Two reviewers independently scored indicators with any disagreement resolved by discussion with a third author. Indicators were weighted according to relevance. For each component, countries were categorised into one of four groups: low, moderate, high, and very high.

RESULTS

The UK and Germany scored highly for all three components, with 91 and 86 out of a possible total score of 120, indicating high to very high preparedness for gene therapies. Overall, Poland generated the lowest score, 48 out of 120. This was largely driven by a low score in the budget and infrastructure component as a result of a relatively low healthcare spend and a limited number of centres currently running gene therapy trials or manufacturing gene therapies. Across the countries studied, higher healthcare spending tended to correlate with more elements required to assess and reward the value of gene therapies and support their implementation.

CONCLUSIONS

This study highlighted that there are geographic differences in preparedness for gene therapies across Europe with disparities between countries’ ability to assess, afford/reimburse and implement such high-cost therapies.