OBJECTIVES: Annual cost of orphan therapies varies across European countries, with relatively small differences between individual therapies and significant variation between therapies within a single country. Differences in lifetime cost are still higher. We aimed to estimate the lifetime cost of a sample of therapies previously found to offer different clinical benefits.

METHODS: Systemic therapies for paediatric indications granted orphan medicinal product designation by the European Medicines Agency (EMA) between 2017 and 2020 were selected. Costing was based on prices listed in the Pricentric database for Belgium, Denmark, France, Germany, Italy, Netherlands, Poland, Spain, Sweden, and the United Kingdom. Drug dosing followed the EMA’s summary of product characteristics, with wastage accounted for. Cost was discounted at 3.5% annually over 10 and 20 years, a proxy for lifetime treatment. Variance percentage was calculated as difference between maximum and minimum price divided by minimum price.

RESULTS: Eleven therapies were identified: burosumab, cannabidiol, chenodeoxycholic acid, cerliponase alfa, dinutuximab beta, glibenclamide, metreleptin, nusinersen, tisagenlecleucel, velmanase alfa, and vestronidase alfa. Dinutuximab beta and tisagenlecleucel were the only treatments with duration limited to under 1 year. Lifetime cost was the lowest for dinutuximab beta: €175,956 both at 10 and 20 years, and the highest for cerliponase alfa: €5,027,586 at 10 years and € 8,591,737 at 20 years. Variance percentage ranged from 10.8% for dinutuximab beta to 95% for glibenclamide. Mean costs across all therapies at 10 and 20 years varied from €859,506 and €1,296,982 for Poland to €1,782,985 and €2,987,556 for Italy.

CONCLUSIONS: Lifetime treatment costs in Europe vary up to 50-fold between therapies and 2-fold between countries. Treatment duration is a major contributor to the estimate. Our results are in line with previous findings that the clinical benefit is not associated with the lifetime cost and that potentially curative therapies offer a good value to payers.