Background: At the beginning of 2011 the early benefit assessment of new drugs was introduced in Germany with the Act on the Reform of the Market for Medicinal Products (AMNOG). The Federal Joint Committee (G-BA) generally commissions the Institute for Quality and Efficiency in Health Care (IQWiG) with this type of assessment, which examines whether a new drug shows an added benefit over the current standard therapy on the basis of a dossier submitted by the pharmaceutical company. In this benefit assessment the costs of the therapies are not taken into account. In the case of an added benefit, the law requires this benefit to be classified as minor, considerable, or major.
Objectives: To describe and discuss the approach for assessing the extent of added benefit for binary data developed by IQWiG.
Methods: Based on the underlying law, outcomes are grouped according to their relevance as follows: 1) mortality, 2) serious (or severe) symptoms (or late complications); serious (or severe) adverse events, health-related quality of life, 3) non-serious (or non-severe) symptoms (or late complications), non-serious (or non-severe) adverse event. Thresholds for confidence intervals for the relative risk are derived by means of meta-analytic power considerations for shifted hypotheses based on suggested effects for each of the categories of added benefit for each outcome group.
Results: For the treatment effect to be classified as a minor, considerable or major added benefit, the methodological approach stipulates that the (two-sided) 95% confidence interval of the effect must exceed a specified distance to the zero effect.
Conclusions: The proposed approach provides a robust, transparent and thus predictable foundation to determine minor, considerable and major treatment effects on binary outcomes in the early benefit assessment of new drugs in Germany. After a decision on the added benefit of a new drug by G-BA (Gemeinsame Bundesausschuss), the classification of added benefit is used to inform pricing negotiations between the umbrella organization of statutory health insurance and the pharmaceutical companies.