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Les médicaments orphelins : rétrospective et perspectives du marché européen Orphan drugs : retrospective and prospective analysis of the european market

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  • Khristina Fauvelle

    (Chercheur indépendant, ISC Paris - Institut Supérieur du Commerce de Paris)

Abstract

L'industrie pharmaceutique, en mutation, est en quête de relais de croissance. Les médicaments orphelins sont destinés à traiter les maladies rares, dont la prévalence statistique est inférieure à 1 personne sur 2000. Les milliers de maladies rares affectent dans leur ensemble des millions d'européens. Souvent mortelles, elles constituent un grave enjeu de santé publique. La politique européenne incite les industriels à développer des médicaments orphelins dans un cadre harmonisé, mais chaque État demeure souverain concernant les prix et remboursements, ce qui peut induire des disparités intracommunautaires. Par ailleurs, la législation européenne octroie aux promoteurs de médicaments orphelins une exclusivité commerciale, soupçonnée d'induire une situation monopolistique et des prix excessifs. Les firmes pharmaceutiques manifestent un engouement croissant pour les médicaments orphelins, qui engrangent un chiffre d'affaire en croissance rapide. Cette tendance semble répondre simultanément à l'enjeu sociétal et à la quête de relais de croissance des industriels. Cependant, l'augmentation des dépenses de santé affectées aux médicaments orphelins provoque l'inquiétude des autorités. Les dernières prévisions économiques publiées en juillet 2012 sont pessimistes. Sous cet éclairage conjoncturel, quelle est la vision rétrospective et prospective du marché des médicaments orphelins en Europe (histoire, règlementation, épidémiologie, modèle économique, monopole, prix, surface / couverture / limites du marché) ? The constantly evolving pharmaceutical industry is seeking growth levers. Orphan medicinal products, so-called orphan drugs, are designed to treat rare diseases, with a statistical prevalence of less than 1 in 2,000 people. The thousands of rare diseases that exist affect millions of Europeans. Often fatal, they represent a serious public health problem. European policy encourages manufacturers to develop orphan drugs within a harmonised framework but each member state retains control of prices and reimbursement levels, which can lead to disparities within the EU. In addition, European legislation grants orphan drug sponsors commercial exclusivity, something that is suspected of creating a monopolistic situation and excessive prices. Orphan drugs are becoming increasingly popular with big pharmaceutical companies, leading to a fast-growing turnover. This trend appears to simultaneously address a societal issue and manufacturers' quest for growth levers. However, the increase in health spending allocated to orphan drugs is prompting concern on the part of the authorities. The latest economic forecasts published in July 2012 are pessimistic. In the light of this economic context, what is the retrospective and prospective view of the orphan drugs market in Europe (history, regulations, epidemiology, economic model, monopoly, pricing, size / cover / limits of the market) ?

Suggested Citation

  • Khristina Fauvelle, 2012. "Les médicaments orphelins : rétrospective et perspectives du marché européen Orphan drugs : retrospective and prospective analysis of the european market," Working Papers hal-00752254, HAL.
    • Handle: RePEc:hal:wpaper:hal-00752254
    Note: View the original document on HAL open archive server: https://hal.science/hal-00752254v3
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    References listed on IDEAS

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    1. Alan D. Lopez & Colin D. Mathers & Majid Ezzati & Dean T. Jamison & Christopher J. L. Murray, 2006. "Global Burden of Disease and Risk Factors," World Bank Publications - Books, The World Bank Group, number 7039, December.
    2. Eline Picavet & Marc Dooms & David Cassiman & Steven Simoens, 2011. "Drugs for rare diseases: Influence of orphan designation status on price," Applied Health Economics and Health Policy, Springer, vol. 9(4), pages 275-279, July.
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