OBJECTIVES: Drug pricing is a complex process influenced by various factors, and understanding these determinants is crucial for informed decision-making. The purpose of this study was to investigate which characteristics of oncological drugs are associated with public prices.

METHODS: Data on oncological drugs appraised by the Haute Autorité de Santé since 2018 were obtained from the NaviHTA database. Drug price was defined as the annualized drug acquisition cost allowing for wastage, computed based on public unit price. Exploratory data analysis was performed to identify associations between prices and product characteristics such as orphan drug status, availability of treatment alternatives, improvement in health outcomes, and methods of pivotal clinical studies. Pairwise associations between the price and its potential determinants were tested using Kolmogorov-Smirnov (KS) tests. A generalized linear model with a logarithmic link function and random effects was fitted to estimate marginal effects of different variables.

RESULTS: After excluding 4 outliers with significantly higher prices, 114 drug evaluations were included. KS tests showed a significant association of the price distributions with orphan status (p-value <0.001), existence of improvement in progression, relapse, or metastasis vs. comparator (p-value = 0.004), and study design (p-value <0.001). According to the regression model, orphan drug status was associated with a 45% higher price, absence of alternatives with a 18% higher price, inclusion of the appropriate comparator in clinical studies with a 12% higher price, and the use of a randomized controlled design with an 11% lower price. No significant effect of health outcome improvement was found.

CONCLUSIONS: The prices of oncological products are mostly driven by orphan drug status and the existence of alternative treatments. The negative effect of randomized controlled design suggests that some factors associated with study design have not been captured. Further investigation of the effect of improvements in health outcomes is also recommended.