OBJECTIVES

The orphan drug status in the European Union aims to promote investment by creating incentives. The primary objective of this analysis is to compare the prices of orphan drugs in France to that of four other European countries: Germany (post-amnog), Italy, Spain and the United Kingdom.

METHODS

17 orphan drugs priced in France between January 2016 and May 2021 were selected. Only products reimbursed first in an orphan indication and with public prices available were included. Daily treatment costs, first price publication date and price evolution in each country were collected. A one-to-one analysis was conducted to account for drugs not available in a country.

RESULTS

Out of the 17 products, 11 (65%) had their lowest price in France. The mean daily treatment cost in France was significantly lower than in the UK (-20,73%; P-value = 0.0123), Italy (-18,57%; P-value = 0.0109) and Spain (-15,98%; P-value = 0.0288). The difference with Germany (+4,5%) was not statistically significant (P-value = 0.579).

A trend appeared when observing the launch sequence of countries: the UK usually obtained a price first followed by other countries with a lower published price. The drop in price compared to the UK was substantial for Germany and France.

In a linear regression model, the two best factors that explained the treatment cost were the population size and level of Added Medical Value (adjusted R² = 0,7305; P-value < 0.001).

CONCLUSIONS

These findings suggest that France grants a lower listed price than its reference countries for orphan drugs. Pricing decisions seem primarily based on the number of people to treat. The new 2021 framework agreement with the CEPS could change this. Indeed, orphan drugs will access European prices with an AMV level of IV. This status should guarantee a price at least equal to the lowest price in the four countries considered.