OBJECTIVES

Several haemophilia gene therapies are in late-stage development. However, there are issues associated with bringing these treatments to market. The aim of this research is to identify the key barriers that may prevent patient access, characterise their impact, and explore the strategies that could help bridge the gap to market.

METHODS

We surveyed the literature to identify haemophilia gene therapy assets in late-stages of development, and reviewed clinical trial designs and outcomes. We evaluated drivers and barriers to patient access, formulated strategies that may be used to optimise market access success, and validated hypotheses with payer experts in key EU markets.

RESULTS

There are 11 gene therapies for haemophilia currently in late-stage development that have demonstrated promising efficacy and safety. However, there are several concerns associated with their development: moderate unmet need due to established standard of care; uncertainty of long-term benefits due to clinical trial limitations; and affordability challenges due to potential high one-time cost.

We identified three key strategies that may help accelerate patient access. Firstly, partnering with academic institutions to develop gene therapies in specialist centres can help identify national-level unmet needs, and navigate reimbursement process. Digital health platforms can facilitate the collection of outstanding patient-relevant data, inform patients in greater need and generate data post-launch to supplement clinical trials. Lastly, innovative financial payment models such as specialist gene therapy pool funds or healthcare reinsurance may lessen the financial burden of these treatments.

CONCLUSIONS

The opportunity provided to haemophilia patients by gene therapies may be hindered by both clinical and economic concerns that delay access. Novel strategies implemented throughout late-phases of development and requiring multi-stakeholder engagement may facilitate timely access. The benefits and risks associated with each need to be further explored to understand their implementation and allow haemophilia gene therapies to benefit patients in need.