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Is Orphan Designation a Driver of Orphan Drug Pricing?

Degrassat-Théas, Albane; Le Pen, Claude; Parent de Curzon, Olivier; Paubel, Pascal; Poisson, N.; Sinègre, Martine (2012), Is Orphan Designation a Driver of Orphan Drug Pricing?, Value in Health, 15, 7, p. A302. http://dx.doi.org/10.1016/j.jval.2012.08.617

Type
Article accepté pour publication ou publié
Date
2012
Journal name
Value in Health
Volume
15
Number
7
Publisher
Elsevier
Pages
A302
Publication identifier
http://dx.doi.org/10.1016/j.jval.2012.08.617
Metadata
Show full item record
Author(s)
Degrassat-Théas, Albane
Laboratoire d'Economie de Dauphine [LEDa]
Le Pen, Claude
Laboratoire d'Economie de Dauphine [LEDa]
Parent de Curzon, Olivier

Paubel, Pascal

Poisson, N.

Sinègre, Martine
Abstract (EN)
ObjectivesPolicy makers pay more and more attention to the orphan drug market. Pharmaceutical companies are suspected to take advantage of the legislation. Moreover, granted an orphan designation (OD) could be associated with higher prices. The aim of this work is to assess the impact of the current European orphan drug legislation on prices within the rare disease market of the largest University Medical Center in France (AP-HP).MethodsWe included all drugs with exclusively rare disease indications, with or without (control group) European OD prior to European marketing authorization. We collected 2011 AP-HP prices and additional characteristics: prevalence of rare diseases; alternative drug existence and improvement in medical benefit (ASMR) assessed by the French National Authority for Health (HAS).ResultsFrom drugs indicated for rare diseases available in AP-HP, we retrieved those for which all additional characteristics were documented: 41 drugs with OD, 17 without OD. The contribution to the growth of the overall drug expenditures in AP-HP reached 1.16 points out of 3.40 points for OD drugs (99% of growth was explained by 3 drugs) and 0.39 for non OD drugs. No statistically significant difference in the price distribution was found between OD drugs (median: €180.4; interquartile range: €679.9) and non OD drugs (median: €443.9; interquartile range: €1433.1) (p=0.19). The price distributions were scattered (standard deviation: 1040.2 and 3512.1 respectively). Other characteristics between the two groups were not statistically different.ConclusionsConcerns on the growing budgetary pressure of orphan drugs may affect social solidarity, and yet this study underlines the heterogeneity of the rare disease market. From these study results, OD approval should not be considered as the only price inflation cause. If the legislation should be adapted to limit windfall effects for pharmaceutical companies, the support of innovation in a fairness and egalitarian way is needed.
Subjects / Keywords
rare disease market; orphan drugs; cost
JEL
I11 - Analysis of Health Care Markets

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