OBJECTIVES:

Orphan drug status offer incentives for drug development, registration and market exclusivity. The objective of this study is to determine if orphan status confers any pricing incentives in the U4 markets + the United-Kingdom (UK) and to compare the level of incentives between these countries.

METHODS:

Annual treatment costs for France, Germany, Italy, Spain and UK were calculated. A student t-test was used to compare orphan drugs commercialized between January 2015 and December 2021 versus their clinical comparators defined by the Haute Autorité de Santé (HAS). A linear regression to adjust for level of innovation, target population (both based on French evaluation by HAS) and launch date was conducted.

For country comparison, a country pricing index was developed: for each product, a score was given to a country according to its price positioning at launch vs. other markets with 1 for the highest price down to 5 for the lowest price. A mean rank was calculated.

RESULTS:

Orphan drugs benefited from significantly (p-value < 0.001) higher annual treatment costs than their clinical comparators in all the countries studied : France (179,878€ vs 26,801€), Germany (247,110€ vs 37,035€) , UK (238,173€ vs 41,067€), Italy (218,173€ vs 64,767€) and Spain (188,593€ vs 33,213€). The ratio is 6.7, 6.7, 5.8, 3.4 and 5.7 respectively. Conclusions did not change after conduction a linear regression or by including products launched before 2015.

Germany offered the highest prices for orphan drugs with an average index of 1.5, followed by the UK (2.3), Italy (2.6), Spain (3.5) and France (4.1).

CONCLUSIONS:

All five markets in the EU4+UK grant higher prices to orphan drugs versus their comparators. This incentive is not uniform as Germany and UK having the highest prices at launch and the highest absolute difference in cost between orphan and non-orphan products.