OBJECTIVES:

The selection of relevant comparator therapies is a major challenge in indications with low evidence. The aim of this analysis was to use the example of German benefit assessments to identify which comparator therapies are used in indications with low evidence and which influencing factors play a role in selecting these appropriate comparator therapies.

METHODS:

In the German health technology assessment (HTA) system, the comparator therapies “treatment of physicians choice” (TPC) with or without best supportive care (BSC) are often selected by the Federal Joint Committee (G‑BA) in indications with low evidence. In order to analyse potential influencing factors, all published benefit assessments with comparator therapies TPC were extracted and analysed for differences and similarities with regard to the choice of comparator therapies.

RESULTS:

By 21^st of January 2022, a total of 44 benefit assessments with the comparator therapy TPC could be identified on the G-BA website. In 7 of the 44 assessments, BSC was included as comparator therapy. The low evidence base and the availability of several equivalent therapy options in the indication are main characteristics for benefit assessments with TPC. In addition, the analysis has shown a discrepancy between therapies approved in the indication and therapies recommended in guidelines in many cases. BSC is included as comparator therapy with TPC if no other therapy is suitable for some of the patients.

CONCLUSIONS:

In order to identify suitable comparator therapies in indications with low evidence, HTA bodies such as the G-BA aim not to exclude any therapy option with sufficient evidence to ensure that all included patients receive the optimal comparator therapy. BSC is a viable option in cases where there is a lack of approved substances with a convincing evidence basis, often this is the case for substances with a long-standing approval and no new evidence.