OBJECTIVES: Fabry disease is a rare, progressive, and multisystemic lysosomal storage disease. While there are treatment options available, none are curative solutions. This research aimed to gather perceptions of European payer experts and clinicians regarding the unmet needs in disease management, and the comparative attributes of existing therapies that drive clinical and payer value.

METHODS: This research study covered 120 quantitative surveys and 30 qualitative interviews in France, Germany, Italy, Poland, Portugal, Spain, Sweden, and the United Kingdom. Quantitative surveys comprised of ten payer experts and five clinicians per country, while the qualitative interviews covered three payer experts and one clinician per country. Participants were asked to score different attributes based on their importance and provide qualitative insights.

RESULTS: Both stakeholder groups characterized Fabry disease as a serious clinical burden. Payer experts noted that patients have reduced quality of life, , and reduced life expectancy. The overall cost of treatment to society was considered limited, due to disease rarity. Both stakeholder groups scored reduction of renal, cerebrovascular, and cardiac events as the greatest unmet needs, while renal and cardiac efficacy were the highest rated value drivers for Fabry disease treatments. Reduction in pain and improvement in quality of life were also rated highly. Clinicians valued oral routes of administration due to their convenience, while payer experts in Italy and Poland placed value on oral treatments due to potential cost offsets. Both stakeholder groups called out the importance of real-world evidence to support future value determination.

CONCLUSIONS: To our knowledge, this is the first comprehensive study with European payer experts and clinicians to be presented on value drivers in Fabry disease, and how current therapies meet these needs. The analysis reveals that while some needs are being addressed, there are opportunities for further improvement, as innovation moves towards the long-term goal of curative therapies.