Does market exclusivity hinder the development of Follow-on Orphan Medicinal Products in Europe?

Anne Em Brabers,Sonja Van Weely,Remco La De Vrueh,Ellen Hm Moors

doi:10.1186/1750-1172-6-59

Abstract

BackgroundWe determined whether the market exclusivity incentive of the European Orphan Drug Regulation results in a market monopoly or that absence of another Orphan Medicinal Product (OMP) for the same rare disorder, a so-called follow-on OMP, is a matter of time or market size. In the interest of rare disorder patients better understanding of the effect of the market exclusivity incentive on follow-on OMP development is warranted.MethodsFirst, the impact of various market-, product- and disease-related characteristics on follow-on OMP development in the EU was determined by comparing rare disorders with an approved OMP and at least one follow-on OMP (N = 26), with rare disorders with an approved OMP and no follow-on OMP (N = 18). Next, we determined whether manufacturers continued development of a follow-on OMP upon approval of the first OMP for the intended rare disorder. Since in the EU significant benefit of an OMP has to be established, we determined for each follow-on OMP for which development was continued on what grounds significant benefit was assumed by the sponsor. Data were collected from the public domain only.ResultsThe likelihood of a rare disorder with an approved OMP to obtain at least one follow-on OMP development was strongly associated with disease prevalence, turnover of the first OMP, disease class, disease-specific scientific output and age of onset. Out of a total of 120 follow-on OMPs only one follow-on OMP could be identified for which development was discontinued upon approval of the first OMP for the same rare disorder. Only a substantial level of discontinuation of follow-on OMP development would have indicated the existence of a market monopoly. Moreover, sponsors that continued development of a follow-on OMP predominantly assumed that their product had an improved efficacy compared to the first approved OMP.ConclusionsThis study provides evidence that absence of follow-on OMP development is a matter of time or market size, rather than that the market exclusivity incentive of the European Orphan Drug Regulation creates a market monopoly.

Highlights

We determined whether the market exclusivity incentive of the European Orphan Drug Regulation results in a market monopoly or that absence of another Orphan Medicinal Product (OMP) for the same rare disorder, a so-called follow-on Orphan Medicinal Products (OMPs), is a matter of time or market size
Characterization of rare disorders with an approved OMP From the start of the European Union (EU) Regulation on Orphan Medicinal Products (April 2000) up to 31 December 2008, 58 OMPs intended for the treatment of a rare disorder obtained marketing approval in the EU [6]
Some products have been approved for more than one rare disorder, most approved OMPs are intended as treatment for only one specific rare disorder

Summary

Introduction

We determined whether the market exclusivity incentive of the European Orphan Drug Regulation results in a market monopoly or that absence of another Orphan Medicinal Product (OMP) for the same rare disorder, a so-called follow-on OMP, is a matter of time or market size. Joppi et al argued that the large number of designated orphan drugs may provide false hope to patients with a rare disorder, because only a small percentage of these designated orphan drugs have obtained marketing approval in the EU [12,13]. Both in the EU and US, certain disease classes - in particular oncology - are associated with a high number of orphan designations and approvals [9,14,15]. The development of orphan drugs is stimulated through a number of regulatory and economic incentives, of which a market exclusivity period of seven and ten years in the US and the EU, respectively, is regarded the foremost one [1,14]

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