OBJECTIVES: Since the Act on the Reform of the Market for Medicinal Products (German: Arzneimittelmarktneuordnungsgesetz, AMNOG) has been implemented in 2011, medicinal products including orphan drugs with active ingredients or indication extension after 2011, have been undergone a benefit assessment (Rules of procedure (VerfO) of the Federal Joint Committee (FJC), chapter 5, §8). Whilst for non-orphan drugs the price is based on the comparator, the pricing of orphan drugs is unclear, since no comparative assessment is required (VerfO, chapter 5, §12). The objective of this analysis is to identify any pattern in the pricing of orphan drugs by analyzing the relationship between the price differences and potentially influencing factors from two benefit assessments of the same active ingredient.

METHODS: Therefore, all orphan drugs with at least two benefit assessments have been identified by the IMS HTA Datenbank. For orphan drugs with re-submission, a descriptive analysis regarding the change in the extent of the additional benefit has been conducted. For orphan drugs with indication extension, the relationship between the price differences in several benefit assessments and the change in possible influencing parameters has been analyzed.

RESULTS: Using a correlation method, the weighted European costs of an orphan drug was identified as the factor with the biggest influence on the percentage price change, followed by the percentage change in the size of the target population and in the annual therapy costs. The change in the extent of the additional benefit seems to have no verifiable effect.

CONCLUSIONS: For orphan drugs the relationship between the price differences from two benefit assessments and the potential influencing factors, except the weighted European costs, is difficult to identify. The pricing mechanism for orphan drugs remains non-transparent but seems to be dependent on a variety of factors and is not based on the additional benefit alone