The validation of the cost-effectiveness signal of the Dutch National Healthcare Institute as a valid method to provide a rough estimation of the cost-effectiveness of a pharmaceutical intervention.

Abstract

Background Cost-effectiveness assessments (CEAs) are a component of HTA in the Netherlands. Because they are resource consuming, the National Health Care Institute (ZIN) developed a simplified method to estimate the cost-effectiveness, called the cost-effectiveness signal (CE-signal). This study aims to investigate whether the CE-signal is a valid method to provide a conclusion on whether an intervention is likely cost-effective or cost-ineffective, and to provide suggestions for its optimization and implementation.

Methods Assessed drugs between January 1, 2019, and December 31, 2021, that included a pharmacoeconomic report were reviewed. CE-signals of the included assessments were performed, including burden of disease, effectiveness estimation and cost-effectiveness determination. Validation occurred by comparing the CE-signal outcomes with those of the corresponding CEA. After evaluating the results of this analysis, the effectiveness estimation method of the CE-signal was further optimized followed by a second analysis.

Results: A total of 28 market authorisation holder (MAH) reports were included to carry out the CE-signal. Of the completed CE-signals, a total of 21 matched the CEA outcome and seven results were incorrect. After the second analysis only one false positive result remained.

Conclusions: The results suggest that the CE-signal can be used to replace some CEAs when the drug is likely cost-effective. In addition, most drugs seem suitable for the CE-signal, excluding one-time treatments and orphan drugs due to absence of discounting and the inherent uncertainties in the data. In addition, the effect estimation method still needs to be further optimized to prevent false positives.