According to the Institute for Quality and Efficiency in Health Care (Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, IQWiG, orphan drugs should undergo a regular early benefit assessment at market entry.
Currently, orphan drugs are exempt from the early benefit assessment requirement (they are assumed to offer additional benefit) and undergo a simplified evaluation process except where their annual sales exceed €50 million. However, according to analysis by IQWiG, in more than half of the cases in which a regular benefit assessment of orphan drugs was carried out, additional benefit was not confirmed. For the purpose of its analysis, IQWiG looked at 41 orphan drug assessments for assessment have been conducted since 2011. The analysis showed that in 22 of the 41 assessments (54%), no additional benefit could be determined in the regular benefit assessment.
IQWiG goes on to comment, “If in more than half of the cases in which a regular benefit assessment of orphan drugs was carried out, the originally stated additional benefit is not confirmed and orphan drugs, which represent real added value, can no longer be recognized as such from the start, there is a need for action: Ten years after the law on early benefit assessment came into force, it is time to abolish the privilege of additional benefits for orphan drugs! Drugs for orphan diseases should also undergo a regular – early – benefit assessment compared to the appropriate comparator therapy by IQWiG and G-BA [Federal Joint Committee, Gemeinsamer Bundesausschuss] when they enter the market.”
www.iqwig.de, “Orphan Drugs: Privileg des „fiktiven“ Zusatznutzens nicht gerechtfertigt”, 12th January 2022