Reassessment Dynamics of Orphan Drugs in Germany’s Benefit Assessment Process

OBJECTIVES: In Germany, the Federal Joint Committee (GBA) employs a special benefit assessment procedure for orphan drugs where the additional benefit is considered proven as a baseline. However, if an orphan drug's annual sales exceed €50 million, a re-assessment is triggered, requiring a full submission followed by price re-negotiations. This study aims to evaluate the reassessment outcomes of orphan drugs, examining differences in the durability of the benefits and the robustness of the initial evidence.

METHODS: The study retrospectively reviewed German records for a set of 17 approved orphan drugs, focusing on initial assessments and subsequent reassessments. Data on the submitted evidence and the extent and probability of benefits were collected and descriptively analyzed.

RESULTS: This study analyzed 24 reports spanning 33 population/subpopulation groups. Average time to resolution reassessment is 50 months. Initially, 21 outcomes were deemed non-quantifiable benefits. During reassessment, most of these were downgraded to no additional benefit proven, with only five cases maintaining their initial ratings and two showing improvement. New evidence including appropriate comparators was submitted in only two of the reassessments. One additional case involved new evidence for a meta-analysis. In the remaining cases, the original evidence was reevaluated. The downgrade in most cases was attributed to methodological limitations that failed to address the absence of appropriate comparative evidence.

CONCLUSIONS: Reassessments of orphan drugs within the GBA framework consistently indicate a decline in perceived benefits. This discrepancy between initial and subsequent assessments raises concerns about the process's validity and transparency. The benefits presumed at the time of market access for these drugs warrant a revision of their exemption from regular assessment procedures. Such a revision is crucial to identify which orphan drugs truly enhance patient care and to prevent unjust discrimination against existing treatments that might appear inferior under the current assessment system.