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In 2010, the Federal Parliament (Bundestag) of Germany passed a new law (Arzneimittelmarktneuordnungsgesetz, AMNOG) on the regulation of medicinal products that applies to all pharmaceutical products with active ingredients that are launched beginning January 1, 2011. The law describes the process to determine the price at which an approved new product will be reimbursed by the statutory health insurance system. The process consists of two phases. The first phase assesses the additional benefit of the new product versus an appropriate comparator (zweckmäßige Vergleichstherapie, zVT). The second phase involves price negotiation. Focusing on the first phase, this paper investigates requirements of benefit assessment of a new product under this law with special attention on the methods applied by the German authorities on issues such as the choice of the comparator, patient relevant endpoints, subgroup analyses, extent of benefit, determination of net benefit, primary and secondary endpoints, and uncertainty of the additional benefit. We propose alternative approaches to address the requirements in some cases and invite other researchers to help develop solutions in other cases. [ABSTRACT FROM AUTHOR]

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 (a positive patient-relevant treatment effect) over the current standard therapy. IQWiG is required to assess the extent of added benefit on the basis of a dossier submitted by the pharmaceutical company responsible. In this context, IQWiG was faced with the task of developing a transparent and plausible approach for operationalizing how to determine the extent of added benefit. In the case of an added benefit, the law specifies three main extent categories (minor, considerable, major). To restrict value judgements to a minimum in the first stage of the assessment process, an explicit and abstract operationalization was needed. The present paper is limited to the situation of binary data (analysis of 2 × 2 tables), using the relative risk as an effect measure. 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. In summary, we assume that our 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, the classification of added benefit is used to inform pricing negotiations between the umbrella organization of statutory health insurance and the pharmaceutical companies. [ABSTRACT FROM AUTHOR]