Different interpretation of additional evidence for HTA by the commissioned HTA body and the commissioning decision maker in Germany: whenever IQWiG and Federal Joint Committee disagree

Background The purpose of this study was to analyse the impact of commissioned addenda by the Federal Joint Committee (FJC) to the HTA body (IQWiG) and their agreement with FJC decisions and to identify potential additional decisive factors of FJC. Methods All available relevant documents up to end of 2017 were screened and essential content extracted. Next to descriptive statistics, differences between IQWiG and FJC were tested and explored by agreement statistics (Cohen’s kappa and Fleiss’ kappa) and ordinal logistic regression. Results Most of the 90 addenda concerned oncological products. In all contingent comparisons, positive changes in added benefit or evidence level on a subpopulation basis (n = 124) prevailed negative ones. Fleiss’ ordinal kappa for agreement of assessments, addenda, and appraisals reached a moderate strength for added benefit (0.474, 95%-CI, 0.408–0.540). Overall agreement between addenda and appraisals on a binary nominal basis is poor for added benefit (Cohen’s kappa 0.183; 95%-CI: 0.010–0.357) ranging from “less than by chance” (respiratory diseases) to “perfect” (neurological diseases). The OR of the selected regression model showed that i) mortality, ii) unmet need, the positions of iii) the physicians’ drug commission and iv) medical societies, and v) the annual therapeutic costs of the appropriate comparative therapy had a high influence on FJC’s appraisals deviating from IQWiG’s addenda recommendation. Conclusions IQWiG’s addenda have a high impact on decision-maker’s appraisals offering additional analyses of supplementary evidence submitted by the manufacturers. Nevertheless, the agreement between addenda and appraisals varies, highlighting different decisive factors between IQWiG and FJC.

INDUSTRY'S EXPERIENCES WITH THE SCIENTIFIC ADVICE OFFERED BY THE FEDERAL JOINT COMMITTEE WITHIN THE EARLY BENEFIT ASSESSMENT OF PHARMACEUTICALS IN GERMANY

Objectives: Optional scientific advice (SA) for the early benefit assessment of pharmaceuticals is offered by the German decision maker, the Federal Joint Committee (FJC). The aim of this study was to elicit manufacturers’ experiences with the SA procedures offered by the FJC to date.Methods: A preliminary survey on a small sample size was conducted. Subsequently, a questionnaire comprising eight items, which was developed on the basis of that survey, was used. Data were analyzed using qualitative and quantitative approaches.Results: The elicitation, including a sample of 25 percent of the completed advice, highlighted the following, regarding the process as well as to the content shortcomings of the SA procedures from an industrial perspective: inconsistencies, FJC's lack of expertise in conducting clinical trials, partially incomplete answers. and a low willingness of the FJC to engage in dialogue with industry were criticized. On the other hand, the majority of respondents expressed a positive attitude concerning unambiguousness, completeness, traceability, discussion atmosphere, and the protocol of the advice. Early SA, before pivotal trials start, showed a significantly higher completeness compared with late SA with respect to endpoints and study duration. Within 4 years the quality of FJC's propositions on some topics improved significantly.Conclusions: Only a few statistically significant differences were detectable between early versus late SA. A positive trend in industry's perception of the SA can be observed over time. A more active involvement of additional stakeholders and the incorporation of procedural elements from other healthcare systems could improve the quality of the SA offered by the FJC.