[Challenges of Determining the Prevalence and Incidence of Urothelial Carcinoma in Early Benefit Assessment].

Since the introduction of the Medicines Market Reorganization Act (AMNOG) in 2011, there have been some structural changes for pharmaceutical manufacturers, due to the early assessment of benefits in the market launch of innovative drugs. This means that the manufacturer is constantly faced with challenges in determining the target population by using prevalence and incidence. Even given the same indication, there are often different approaches and models used for estimating the target population. In bladder carcinoma, the fourth most common tumor in men, there have been many advances in the field of immunotherapy for the treatment of locally advanced or metastatic carcinomas. These can be used to compare existing dossiers. The aim of this study was to evaluate the quality of the prevalence and incidence calculations of the dossiers in the indication urothelial carcinoma and to identify the recommendations for action that can be derived for their future determination. This problem was examined based on the methods paper of the Institute for Quality and Efficiency in Health Care and the benefit assessments of the decisions on additional benefits. Our comparison showed similarities in the use of epidemiological measures, but identified mainly differences in the calculation steps of the target population. As a result, the target population was mostly underestimated or overestimated. The quality of the dossiers differed mostly in the traceability of the calculations and the origin of data. With regard to external validity and generalizability to the German health care system, there is still room for improvement.
Competing Interests: Dr. Daniel Wirth ist Mitarbeiter von Janssen Pharmaceutical Companies of Johnson & Johnson.
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