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. 2021 Oct 19;16(1):439.
doi: 10.1186/s13023-021-02022-w.

Variables affecting pricing of orphan drugs: the Italian case

Claudio Jommi  1 , Elisabetta Listorti  2 , Federico Villa  3   4 , Simone Ghislandi  2   5 , Armando Genazzani  4 , Agnese Cangini  3 , Francesco Trotta  3
Affiliations

Variables affecting pricing of orphan drugs: the Italian case

Claudio Jommi et al. Orphanet J Rare Dis. .

Abstract

Background and aim: Evidence on determinants of prices for orphan medicines is scarce and not available for Italy. The aim of this paper is to provide an evidence on variables affecting the annual treatment cost of orphan drugs in Italy, testing the hypothesis of a negative correlation with the dimension of the target population and a positive correlation with the added therapeutic value of the drug and the quality of the evidence of pivotal studies.

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 annual treatment cost and, as explanatory variables, the dimension of the target population, the existence of Randomized Clinical Trials 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 annual treatment cost when the target population is very small.

Conclusions: Our results suggest that value arguments and sustainability (dimension of the target population and its impact on budget impact) issues are considered for orphan drugs pricing: the role played by sustainability is systematically supported by our results. A more transparent and reproducible price negotiation process for orphan drugs is needed in Italy. This paper has contributed to highlight the implicit drivers of this process.

Keywords: Health technology assessment; Italy; Orphan drugs; Pricing; Rare diseases.

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Conflict of interest statement

CJ has no conflict of interest linked with this research. He received research grants from Abbvie, Amgen, Astrazeneca, Bayer, Biogen, BMS, Boehringer I, Celgene, EuroQoL Foundation, FSK, Janssen C, MSD, Novartis, Roche, Pfizer, Sandoz, Sanofi, Takeda, Teva, Zambon for his research activity and acted as a speaker and or consultant for Amgen, Astrazeneca, BMS, Celgene, Dephaforum, Gilead, Incyte, MA Provider, MSD, Roche, Sanofi, Takeda, Wellmera (now Alira Health). EL has nothing to disclose. FV has nothing to disclose. SG has nothing to disclose. AG has nothing to disclose. AC has nothing to disclose. FT has nothing to disclose.

Figures

Fig. 1
Fig. 1
Distribution of annual treatment cost (thousands euros)
Fig. 2
Fig. 2
Correlation between the independent variables and the annual treatment cost (thousand euros). In bold, correlations significant at 10%. ASMR Amélioration du Service Médical Rendu, RCT Randomized Clinical Trial
Fig. 3
Fig. 3
Costs, prevalence and RCT. Prevalence 0/1 = below/above the median value; RCT 0/1 = RCT No/Yes
See this image and copyright information in PMC

References

    1. Jommi C, Minghetti P. Pharmaceutical pricing policies in Italy. In: Ud-Din B, editor. Pharmaceutical prices in the 21st century. London: Springer; 2015. pp. 131–151.
    1. Villa F, Tutone M, Altamura G, et al. Determinants of price negotiations for new drugs. The experience of the Italian Medicine Agency. Health Policy. 2019;123(6):595–600. doi: 10.1016/j.healthpol.2019.03.009. - DOI - PubMed
    1. AIFA Agenzia Italiana del Farmaco. Criteri per la classificazione dei farmaci innovativi e dei farmaci oncologici innovativi. https://www.aifa.gov.it/-/aifa-criteri-per-la-classificazione-dei-farmac.... Accessed 01 Jan 2021.
    1. Atkins D, Best D, Briss PA, et al. Grading quality of evidence and strength of recommendations. BMJ. 2004;328(7454):1490. doi: 10.1136/bmj.328.7454.1490. - DOI - PMC - PubMed
    1. AIFA. Farmaci innovativi. Report di valutazione dell'innovatività per indicazione terapeutica. https://www.aifa.gov.it/farmaci-innovativi. Accessed 01 Jan 2021.