ArticleOrphanet journal of rare diseases2021
Variables affecting pricing of orphan drugs: the Italian case.
Article in Orphanet journal of rare diseases, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.
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9 citing papers in PubMed, 12 citations in OpenAlex.
- Determinants of the Financial Impact of Orphan Drugs in Italy: Differences Between Expected and Observed Pharmaceutical Expenditure.Drugs - real world outcomes · 2025Article
- Determinants of orphan drug health technology assessment in South Korea: an empirical analysis.Frontiers in pharmacology · 2025Article
- The impact of level of documentation on the accessibility and affordability of new drugs in Norway.Frontiers in pharmacology · 2024Article
- Variables affecting new drug prices in South Korea's pricing system.Frontiers in pharmacology · 2024Article
- Role of Economic Evaluations on Pricing of Medicines Reimbursed by the Italian National Health Service.PharmacoEconomics · 2023Article
- Assessment of the availability of repurposed orphan drugs in India.PLOS global public health · 2023Article
- Real-world use of orphan medicinal products (OMPs) in rare disease (RD) patients: A population-based registry study.Frontiers in pharmacology · 2022Article
- Gene Therapy for Neuromuscular Diseases: Health Economic Challenges and Future Perspectives.Journal of neuromuscular diseases · 2022Review
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Authors and funding
7 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
BACKGROUND AND
aimEvidence 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.
methodsDrugs 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.
resultsIn 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.
conclusionsOur 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.
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