SKC presents outcomes of benefit reassessments of orphan drugs in Germany after exceedance of the 50-Million-Euro revenue threshold at the Virtual ISPOR Europe 2020 conference

For orphan drugs in Germany, an added benefit is granted by law within the early benefit assessment. However, this privilege no longer applies if the drug's turnovers exceed 50 million EUR within 12 months, leading to a benefit reassessment based on a comparison against an appropriate comparative therapy. SKC is presenting an analysis of outcomes of all German early benefit assessments of orphan drugs for which reassessment due to the exceedance of the 50-Mio.-EUR revenue threshold had started by July 2020 at the Virtual ISPOR Europe 2020 conference.

Twelve orphan drugs exceeded the 50-Mio.-EUR revenue threshold and therefore had to undergo benefit reassessment. Six out of these twelve orphan drugs (50%) are applied in the field of oncology. As defined by German law, all twelve orphan drugs that exceeded the 50-Mio.-EUR revenue threshold later on were granted an added benefit for the total overall patient population (100%) after the first benefit assessment. However, after reassessment the previously granted added benefit was lost for an average of 57% of the total patient population.

For five Orphan Drugs, net price rebates after the first benefit assessment and after reassessment due to the exceedance of the 50-Mio.-EUR revenue threshold could be analyzed. The overall mean net price rebate increased from 21.14% after the first benefit assessment to 29.38% after reassessment, which reflects an average absolute increase of 8.24% and a relative increase of 38.95%. Although in three out of the five cases analyzed the granted added benefit category was even higher after reassessment compared to the first benefit assessment, the net price rebate increased in four out of five cases. A reduction of the net rebate after reassessment, i.e. an increase of the reimbursed price, was not observed for either of the five Orphan Drugs.

Hence, even if the outcome of the benefit reassessment after exceedance of the 50-Mio.-EUR revenue threshold is better than the outcome of the first benefit assessment, there is a high risk of an increase in the negotiated net price rebate. Therefore, pharmaceutical companies should carefully assess whether a voluntary price reduction to avoid the exceedance of the 50-Mio.-EUR annual revenue threshold may be more beneficial than taking the risk of a benefit reassessment under non-orphan conditions.

For further discussion about early benefit assessment of orphan drugs in Germany, we would like to invite you to our chat at the Virtual ISPOR Europe 2020 conference on Monday, 16 November 2020, 4.30 pm – 6.00 pm