gms | German Medical Science

Introduction: Within the framework of the Act on the Reform of the Market for Medicinal Products (AMNOG) in Germany, pharmaceutical entrepreneurs must submit a dossier demonstrating added benefit of a new drug compared to an appropriate comparator therapy. Underlying evidence is planned for registration purposes and therefore often does not meet the appropriate comparator therapy as defined by the Federal Joint Committee (G-BA). Therefore indirect comparisons (IC) are allowed within AMNOG to assess the extent of added benefit. The method proposed by Bucher et al. [1] has been recognized as one of the standard approaches to perform adjusted IC. Further alternative methods exist, e.g. Matching-based approaches, which aim to overcome different challenges. However, all these methods have certain limitations. This study evaluates the statistical properties of different IC methods and their limitations in practice.

Methods: We conducted a simulation study to evaluate and compare the performance of different IC approaches in different scenarios with regard to possible bias, type I error and power.

Results: Adjusted ICs are deemed to be "state of the art". Due to their requirements they are, nevertheless, often non-applicable. In most cases this is due to lack of comparability of the clinical trials, e.g. with regard to the common comparator, the study population and the study design. Different approaches aim to overcome certain challenges in situations where IC methods are needed due to the lack of direct evidence. However, every method has its limitations which need to be always discussed and considered when it comes to the interpretation of results derived by means of IC.

Conclusion: All IC approaches have in common that they imply a set of more or less feasible requirements to be able to derive valid statements. In practice, model assumptions are often very difficult to verify. However, with regard to the benefit assessment procedure and the subsequent price negotiations it would be helpful to have alternatives with acceptable properties in order to estimate the extent of added benefit. Further research and discussions on feasibility of different approaches is needed.

Der Vortrag gehört zum Workshop "Methods for Generalized Evidence Synthesis".

Organisatoren: R. Bender, K.H. Herrmann, K. Jensen, D. Hauschke, F. Leverkus & T. Friede

Die Autoren geben an, dass kein Interessenkonflikt besteht.

Die Autoren haben keine Angabe zur Beratung durch einen Ethikkommission gemacht.