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Background: Increasing pharmaceutical expenditure challenges the sustainability and accessibility of healthcare systems across Europe. Confidentiality restraints hinder assessment of actual prices of Orphan Medicinal Products (OMPs). Hence, we assessed the real prices of brand-name OMPs around market exclusivity expiry (MEE).

Objective: We aimed to explore developments in published list prices (LPs) and confidential hospital purchase prices (PPs) of brand-name OMPs relative to their market exclusivity status in Western European countries with similar GDPs.

Methods: We analyzed LPs and PPs of 13 selected OMPs purchased by university hospitals in Western European countries between 2000 and 2020. For confidentially reasons, proportions were used, with the Dutch LPs of the selected OMPs at the year of MEE serving as reference values. PPs included pre-purchase discounts. Rebates were not considered.

Results: Data were analyzed from hospitals in Denmark (DK) (n = 1), France (FR) (n = 1), Germany (DE) (n = 2), and the Netherlands (NL) (n = 1). Average LPs and PPs of included OMPs dropped gradually but limited over time, with no explicit price drop after MEE. LP levels differed more per country than PP levels: LP range before MEE was 164% (DE)-101% (FR) and after MEE was 135% (DE)-82% (FR); PP range before MEE was 150% (DE)-102% (FR) and after MEE was 107% (DE)-80% (FR). Overall differences between LPs and PPs were < 3% in all countries, except for Denmark.

Conclusion: No evident price drops of included brand-name OMPs were observed around MEE and differences in purchase prices are modest in the selected Western European countries. Results were not subject to significance testing. More robust data are needed to strengthen negotiations with suppliers.

Titel:	Prices of Orphan Drugs in Four Western European Countries Before and After Market Exclusivity Expiry: A Cross-Country Comparison of List Prices and Purchase Prices.
Autor/in / Beteiligte Person:	Dane, A ; Klein Gebbink, AS ; Brugma, JD ; Degrassat-Théas, A ; Hug, MJ ; Houlind, MB ; Paubel, P ; van der Kuy PHM ; Uyl-de Groot, CA
Link:	Zum Volltext
Zeitschrift:	Applied health economics and health policy, Jg. 21 (2023-11-01), Heft 6, S. 905-914
Veröffentlichung:	Auckland, N.Z. : Open Mind Journals Ltd., c2002-, 2023
Medientyp:	academicJournal
ISSN:	1179-1896 (electronic)
DOI:	10.1007/s40258-023-00832-6
Schlagwort:	Humans Drug Costs Europe France Orphan Drug Production Lipopolysaccharides
Sonstiges:	Nachgewiesen in: MEDLINE Sprachen: English Publication Type: Journal Article Language: English [Appl Health Econ Health Policy] 2023 Nov; Vol. 21 (6), pp. 905-914. <i>Date of Electronic Publication: </i>2023 Sep 26. MeSH Terms: Orphan Drug Production* ; Lipopolysaccharides* ; Humans ; Drug Costs ; Europe ; France References: Mestre-Ferrandiz J, Palaska C, Kelly T, Hutchings A, Parnaby A. An analysis of orphan medicine expenditure in Europe: is it sustainable? Orphanet J Rare Dis. 2019;14(article number 287). ; Simoens S. Pricing and reimbursement of orphan drugs: The need for more transparency. Orphanet J Rare Dis. 2011;6(article number 42). ; Michel M, Toumi M. Access to orphan drugs in Europe: current and future issues. Expert Rev Pharmacoecon Outcomes Res. 2012;12(1):23–9. (PMID: 10.1586/erp.11.9522280193) ; Garattini L, Padula A. HTA for pharmaceuticals in Europe: will the mountain deliver a mouse? 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Prices of Orphan Drugs in Four Western European Countries Before and After Market Exclusivity Expiry: A Cross-Country Comparison of List Prices and Purchase Prices.