OBJECTIVES: To investigate the impact of introducing the Act on the Reform of the Market for Medical Products (AMNOG), which aims to contain cost of pharmaceuticals in Germany, on orphan drug (OD) costs in Germany, compared with France.

METHODS: All ODs authorized before November 2017 in Europe were identified using the European Medicines Agency’s database, including drugs which orphan designation has expired or been withdrawn. Only the first indication was considered for assessment of annual cost of drugs with multiple approved indications. Ex-factory unit prices were obtained from AMELI and Thériaque databases for France and Orphan POLI Global for Germany. Annual drug costs were calculated based on unit prices and approved regimens for two time periods – before the introduction of AMNOG in 2011 and post-AMNOG introduction. The relationship between annual drug costs in France and Germany before and after AMNOG introduction was evaluated using regression analysis.

RESULTS: ODs for which prices were available in both France and Germany were identified (pre-AMNOG: 36, post-AMNOG: 19). The average annual cost of an OD pre-AMNOG was 97,571€ in France and 107,486€ in Germany. After the introduction of AMNOG in 2011, annual average costs seemed to increase in both countries (118,581€ in France; 153,473€ before HTA and 125,602€ after HTA in Germany). A strong price correlation relationship between France (x axis) and Germany (y axis) was observed before 2011 (correlation coefficient: 0.99, regression equation: y=1.06x + 4052). After 2011, the regression equation was estimated as y=0.967x + 10900, indicating that AMNOG succeeded in containing the annual cost of ODs in Germany for products that cost approximately 74,000€ or more in France.

CONCLUSIONS: ODs prices tended to increase over time. The presence of AMNOG seemed to contribute to containing the annual cost of ODs in Germany, despite a favourable pricing policy.