OBJECTIVES

: This analysis aimed to describe Health-Technology Assessment (HTA) decisions for oncology treatments in Germany and to determine factors that may have influenced the decision of the Federal Joint Committee (G-BA) for or against a remedy (benefit versus no benefit).

METHODS

: We evaluated all oncology G-BA decisions until April 2018. Manufacturer’s dossiers and G-BA appraisals were reviewed. Additionally, all cited studies were analyzed regarding their study design. Multivariable logistic regression analysis was performed, with recommendation (benefit/no benefit) as an outcome, and considering cancer type, year of decisions, submitted study designs (randomized controlled trials (RCTs) vs. single-arm trials only), any reported overall survival (OS) improvement, and orphan status as independent factors.

RESULTS

: In total, 100 HTAs were included in this analysis. Most of the HTAs were completed within recent years (69% from 2016 onwards), and the majority dealt with treatments for lung cancer (20%), blood cancer (13%) and skin cancer (13%). Overall, 73% HTAs concluded that the assessed treatments were associated with added benefit. Cancer type and level of OS improvement did not significantly impact the recommendation of the G-BA and therefore were eliminated in a backward procedure (p>0.1). Furthermore, published year (OR=0.75, p=0.096) was not significantly impacting G-BA`s decisions. As orphan status is automatically associated with an additional benefit according to G-BA rules, it was found to be associated with a positive decision (OR=14.6, p=0.003), with a recommendation rate of 94% (p<0.001). Studies who only submitted single-arm trial studies and no RCTs had a slightly lower chance for a positive assessment (OR=0.19, p<0.076).

CONCLUSIONS

: Orphan status and evidence presented by RCTs were shown to predict positive G-BA decisions in oncology. No further contributing factors could be found. Nevertheless, HTA decisions may be driven by other undetected factors and further research is needed to identify those.