Is No Therapy a Therapy?

In Germany, the additional benefit of new drugs is evaluated during a health technology assessment, reigned by a tight regulatory framework (AMNOG). For this purpose, an appropriate comparator therapy (ACT) is determined by the German Federal Joint Committee (Gemeinsamer Bundesausschuss, G-BA). If in the respective indication no specific comparator therapy is available according to the G-BA, watchful waiting (WW) may be defined as the ACT: The drug is compared against no therapy. This analysis aims to assess the impact of WW as ACT on the outcome of the benefit assessment and subsequent price negotiations during AMNOG.

Using a database which encompasses information on benefit assessment, G-BA resolution and negotiated rebate of all AMNOG procedures ever performed, the outcomes of the benefit assessments and price negotiations of all AMNOG procedures with WW as ACT were compared to all other procedures.

In 27 out of 729 procedures (~4%) WW was appointed as ACT. In these procedures, the number of oncology indications was above average. Furthermore, the proportion of drugs that were granted an additional benefit was considerably higher (59% vs. 39%), and a higher proportion of procedures resulted in the best possible additional benefit ratings "major" (3% vs. 1%) or "considerable" (19% vs. 11%). Finally, in procedures with WW as ACT the average negotiated net annual therapy cost rebate (~12%) was lower than in procedures with other ACTs (~15%).

In procedures with WW as ACT, effect sizes are likely to be larger and therefore allow for a better benefit rating, which is the most important factor in German price negotiations. In addition, the price of the ACT plays a decisive role as a price anchor. Despite being a very cheap anchor, no negative, but rather a positive effect on rebate negotiation can be observed for procedures with WW as ACT.