OBJECTIVES: The additional benefit of orphan drugs (OD) assessed by the G-BA is already acknowledged by approval. However, if the annual costs for the statutory health insurance exceed 50 million euros or the OD-status is lost, a standard assessment without OD privileges is mandatory. The aim of this study was to examine the change in the extent of the added benefit, time to reassessment and change of the price discount.

METHODS: A database containing all dossiers was screened and all OD that underwent reassessment were extracted. If the G-BA assessed more than one subpopulation within the label population, the subpopulation with the highest added benefit was considered.

RESULTS: Since 2011, 24 OD underwent a reassessment. The average time to reassessment was 49 months for drugs with a lower added benefit after reassessment, 24 months for drugs with a higher added benefit, and 37 months for drugs without changes in the added benefit. The average discount on the launch price increased from 20% to 38% for drugs with a downgrade in the added benefit after reassessment, from 20% to 37% for drugs with an unchanged added benefit and remained stable at 20% for drugs with an upgrade in the added benefit.

CONCLUSIONS: In summary, there seems to be a correlation between a shorter time to reassessment and the likelihood of receiving an upgrade in the added benefit. In general, the price discount after reassessment is higher compared to the one after the initial assessment, except for drugs with an upgraded added benefit. In light of a possible lowering of the revenue threshold to 25 million euros as trigger for a standard assessment, companies launching drugs likely to exceed this threshold should be aware of the potential impact of reassessment, as only one fourth of reassessments resulted in a higher added benefit.