OBJECTIVES: To compare willingness to pay (WIP) of treatments for ultra-rare-diseases in US, Germany, France, UK and Japan

METHODS: Secondary research was conducted to identify ultra-rare diseases (affecting fewer than 1 in 50,000). Publicly available list prices for all approved treatments in the last ten years for these diseases were analysed. WIP was analysed based on factors such as patient population, disease severity, patient age (pediatric vs. adult) and gene therapies vs. non-gene therapies. A short survey was sent to payers to validate the findings.

RESULTS: WIP based on US WAC was the highest for most analogues. Elevidys (DMD) with a WAC price of $3.2 million in US and Upstaza (AADC deficiency) with a list price of €3.5million in France are amongst the highest priced treatments globally. From ultra-rare product analogues prices in Germany were approximately 40% lower (range of 25%-55%) to that in the US. Comparing France, Germany and the UK, the price were highest in France for cell and gene therapies and approximately 15-20% lower than that in US, which is not the case for small molecules and biologics. The costs were reasonably homogenous in the UK and Germany, even though the payer evaluation criteria in the UK is based on Cost-Effectiveness and in Germany on clinical-effectiveness and the quality of evidence. In Japan, prices were lower at 50% of US prices, however when compared to Germany, they were 1.35 - 0.55 times, demonstrating a wide range. In Japan, 10-25% lower than that average in Fr, De and the UK could be a good assumption for ultra rare disease treatments.

CONCLUSIONS: WIP for treatments for ultra rare diseases specifically gene therapies is very high and HTA institutions and payers have recognised the value of curative treatments reflected through positive HTA and price outcomes.