OBJECTIVES: Submissions of Health technology assessments (HTA) with single-armed registration trials risk rejection by HTA agencies due to uncertainty of relative effect compared to current standard of care. The objective of this study was to identify and compare relevant parameters for Sweden and Denmark that influence the decision outcome.

METHODS: Decisions and recommendations for products with single-arm trials were retrieved from the public databases of the respective authorities - the Danish Medicines Council (DMC), the Swedish Dental and Pharmaceutical Benefits Agency (TLV) and the Swedish New Therapies (NT) Council, between January 2017 and April 2022.

Parameters of relevance were identified, examples being disease area, severity, prevalence, clinical value, and cost per quality adjusted life years. The decisions were categorised by the recommendation outcome and parameters were quantitatively assessed through a logistic regression.

RESULTS: 17 individual drugs across Sweden and Denmark were identified in the following disease categories, 60% in oncology, 10% genetic, 15% neurological and 15% others. In Sweden the received outcomes were, 47% positive, 37% negative and 16% still waiting. In Denmark the outcomes were, 37% positive, 37% negative, 10% still ongoing, 11% not yet submitted and 1 (5%) withdrawn.

The results from the logistics regression did not provide statistically significant results at a significance level of 5%, the closest occurring at p=0.0684 for effect size of disease severity for Sweden. The severity and oncology indications would be the most likely to lead to a positive recommendation in both Sweden and Denmark. Comparing Sweden and Denmark, there were cases where an assessment was not made in Denmark following an earlier negative recommendation for the same assessed indication in Sweden.

CONCLUSIONS: Both Sweden and Denmark exhibited unpredictability in estimating the outcome of the assessment process, with Sweden appearing more accessible in its evaluations, and Denmark presenting a more apparent conservative approach.