OBJECTIVES: In case of a label extension (LE) of a pharmaceutical a separate early benefit assessment for the new indication should be conducted. LE are judged to have less high therapeutic value comparing to the first indication. The aim of this research was to analyze the distribution of different dimensions of the additional benefit (AB) in early benefit assessments (EBAs) due to LE, compare it with the distribution of AB in launch procedures and analyze the reasons for an unproven AB in these EBAs.

METHODS: The results of all completed EBAs by December 2023 due to LE in Germany were analyzed. Qualitative text analysis was used to determine the AB and the reasons for the lack of AB. The analysis was performed at the subpopulation level.

RESULTS: A total of 507 subpopulations for LE were analyzed. A major AB was identified for three subpopulations, a considerable AB for 64 subpopulations, a minor AB for 53 subpopulations, and a lesser benefit for one subpopulation. A non-quantifiable AB was found for 63 subpopulations, and no AB could be proven for 323 subpopulations. In case of no AB the most common reason was that the submitted study was not suitable for the EBA (174/323). There was a similar distribution of the AB for the subpopulations of the launch procedures and the subpopulations of the LE.

CONCLUSIONS: Despite the criticism that LE generally have lower therapeutic value compared to the initial indications, the distribution of AB among subpopulations in launch procedures and LE was found to be similar. The most common reason for the unproven AB was the unsuitability of submitted studies for EBA. The challenges facing the pharmaceutical industry in the AMNOG to provide suitable clinical evidence for the EBA become also apparent in the case of label extensions