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. 2022 Feb 17:9:820757.
doi: 10.3389/fmed.2022.820757. eCollection 2022.

Orphan Drug Prices and Epidemiology of Rare Diseases: A Cross-Sectional Study in Italy in the Years 2014-2019

Federico Villa  1   2 , Aurora Di Filippo  2 , Andrea Pierantozzi  2 , Armando Genazzani  1 , Antonio Addis  3 , Gianluca Trifirò  4 , Agnese Cangini  2 , Giovanni Tafuri  2   5 , Daniela Settesoldi  2 , Francesco Trotta  2
Affiliations
Free PMC article

Orphan Drug Prices and Epidemiology of Rare Diseases: A Cross-Sectional Study in Italy in the Years 2014-2019

Federico Villa et al. Front Med (Lausanne). .
Free PMC article

Abstract

Introduction: It is well acknowledged that the price of orphan drugs is normally higher than that resulting from the value-based pricing. A correlation between the cost of therapy for orphan drugs and the epidemiology (prevalence and incidence) of the related rare disease can be hypothesized.

Methods: This analysis includes all approved orphan drugs by European Medicines Agency whose reimbursement was granted for the first therapeutic indication in the years 2014-2019 in Italy. Regression and correlation analyses were performed to analyze the possible correlations between the logarithm of the annual therapy cost and the epidemiology of the rare diseases, between orphan drugs consumption and epidemiology of related rare disease and between therapy cost and the consumption.

Results: The regression analysis between the annual cost of therapy estimated on the published ex-factory price and the prevalence showed a slightly decreasing, not statistically significant, trend (coefficient: -0.10, p-value: 0.41). The results were similar when using the price resulting from the application of Managed Entry Agreements (coefficient: -0.11, p-value: 0.40). The regression analysis between sales volume and prevalence showed a positive slope without an acceptable level of significance (p-value: 0.04). The correlation analysis between the therapy cost and the sales volume highlighted again an absence of significant association, similarly if considering only ATC L orphan drugs, or the incidence.

Discussion: The definition of the price of an orphan drug seems not to depend on the rarity of the disease, and sales volumes do not correlate with the epidemiology of the rare disease and with the annual cost of therapy.

Keywords: AIFA; HTA; Italy; drug coverage; drugs pricing; market access; orphan drugs; rare disease.

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Conflict of interest statement

FV, ADF, AP, AC, DS, and FT were employed by Agenzia Italiana del Farmaco (AIFA). GT was employed by Zorginstituut Nederland. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Figures

Figure 1
Figure 1
Orphan drugs that applied for P&R in Italy in the period 2014–2019. Legend: A Class: Medicines reimbursed by the NHS. H Class: Medicines reimbursed by the NHS for inpatient use. C Class: Medicines not reimbursed by the NHS. C(nn) Class: Medicines waiting for P&R process, not reimbursed by the NHS.
Figure 2
Figure 2
Correlation analysis between the log of the annual therapy cost (calculated on the ex-factory price) and the prevalence of the disease.
Figure 3
Figure 3
Correlation analysis between the log of the annual therapy cost (calculated on the Final Price) and the prevalence of the disease.
Figure 4
Figure 4
Correlation analysis between the sales volume in the first year of commercialization in Italy and the prevalence of the related rare diseases.
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