PHP281 - THE METHODOLOGICAL DIFFERENCES OF EUROPEAN JOINT ASSESSMENT AND GERMAN HTA: AN EMPIRICAL APPROACH

Abstract

With the legislatory endorsement of European Joint Assessments for pharmaceuticals (JAs) the HTA landscape in Europe is approaching fundamental structural change. Since 2016, only two pharmaceuticals (both in oncology) have undergone both European JA and national assessment in Germany. This is the first time that the methodology applied in the different assessments is compared based on concrete examples. Differences between the JA and the corresponding German assessment report were analyzed for Midostaurin in the indication of FLT3-positive acute myeloid leukemia (JA published November 2017) and Alectinib in the indication of ALK-positive advanced non-small cell lung cancer (JA published January 2018). The comparison features five key items previously identified: body of evidence, comparators, endpoints, evidence synthesis, and subgroups. While in Germany only RCT data with a specific comparator was accepted in each case, non-RCTs and multiple comparators were included in the JA leading to a larger body of evidence (1 study vs. 3 studies in each example). Indirect comparisons were considered only in the JAs (e.g. Alectinib vs. Ceritinib). Furthermore, some endpoints not regarded as relevant in Germany, such as progression- and disease-free survival, were presented within the JAs. The methodology used to evaluate the certainty of results differed significantly. However, there were also similarities between JA and the German assessment: both approaches follow the principles of evidence-based medicine; central endpoints were overall survival, health-related quality of life, and safety; subgroups investigated were consistent. The examples reviewed confirm that the outcome of European JAs and their German equivalent can be expected to differ for methodological reasons. In general, JAs offer a broader spectrum of possibilities in terms of suitable evidence and analytical methods. Nevertheless, a high degree of consensus was observed in the final evaluations suggesting a strong potential for future harmonization of the different procedures.