Evidence on determinants of prices for orphan medicines is scarce and not available for Italy. The aim of this paper is to fill this literature gap. Methods: Drugs with a European orphan designation reimbursed in Italy in the last 6 years (2014-2019) were considered. Univariate, cluster analysis and multiple regression models were used to investigate the correlation between the yearly unit cost per patient and, as explanatory variables, the dimension of the target population, the existence of Randomized Clinical Trials (RCT) as a proxy of the quality of the pivotal studies, the added therapeutic value. Results: In the univariate analysis prevalence and added therapeutic value, as expected, have a negative and positive correlation with cost, respectively. The correlation with RCT is not significant. In the multivariate model, coefficients for prevalence and added value are confirmed but for the latter are not significant anymore. We also found, through an interaction analysis, that the existence of an RCT has a positive impact on prices when the target population is very small. Conclusions: Our evidence suggests that value arguments and sustainability issues are considered for orphan drugs pricing: the role played by sustainability is systematically supported by the statistical evidence. A more transparent and reproducible price negotiation framework for orphan drugs is needed in Italy. This paper has contributed to highlight the implicit drivers of this framework.
The paper has been accepted for publication on the 20th of September 2021 in Orphanet Journal of Rare Diseases.
Authors: Claudio Jommi*, Elisabetta Listorti*, Federico Villa^, Simone Ghislandi*§, Armando Genazzani#, Agnese Cangini^, Francesco Trotta^
*Cergas (Centre for Research on Health and Social Care Management), SDA Bocconi School of Management, Bocconi University, Milan, Italy
^Aifa (Italian Medicines Agency), Rome, Italy (FV is not affiliated with Aifa anymore; his present affiliation is Astrazeneca, Milan, Italy)
*§Department of Social and Political Sciences, Bocconi University, Milan, Italy
#Department of Pharmaceutical Science, Università del Piemonte Orientale, Novara, Italy
Speaker Claudio Jommi, SDA Bocconi, Link bio