IDEAS home Printed from https://ideas.repec.org/p/hal/wpaper/hal-00752254.html
   My bibliography  Save this paper

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

Author

Listed:
  • 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
    as

    Download full text from publisher

    File URL: https://hal.science/hal-00752254v3/document
    Download Restriction: no
    ---><---

    References listed on IDEAS

    as
    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.
    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.
    Full references (including those not matched with items on IDEAS)

    Most related items

    These are the items that most often cite the same works as this one and are cited by the same works as this one.
    1. Tsung-Ming Tsao & Jing-Shiang Hwang & Sung-Tsun Lin & Charlene Wu & Ming-Jer Tsai & Ta-Chen Su, 2022. "Forest Bathing Is Better than Walking in Urban Park: Comparison of Cardiac and Vascular Function between Urban and Forest Parks," IJERPH, MDPI, vol. 19(6), pages 1-15, March.
    2. Anderson, Soren T. & Laxminarayan, Ramanan & Salant, Stephen W., 2012. "Diversify or focus? Spending to combat infectious diseases when budgets are tight," Journal of Health Economics, Elsevier, vol. 31(4), pages 658-675.
    3. Michael Grimm & Carole Treibich, 2013. "Why Do Some Bikers Wear a Helmet and Others Don't? Evidence from Delhi, India," AMSE Working Papers 1348, Aix-Marseille School of Economics, France, revised 10 Oct 2013.
    4. Christopher Fitzpatrick & Katherine Floyd, 2012. "A Systematic Review of the Cost and Cost Effectiveness of Treatment for Multidrug-Resistant Tuberculosis," PharmacoEconomics, Springer, vol. 30(1), pages 63-80, January.
    5. repec:hrv:hksfac:5341873 is not listed on IDEAS
    6. Falk, Armin & Menrath, Ingo & Verde, Pablo Emilio & Siegrist, Johannes, 2011. "Cardiovascular Consequences of Unfair Pay," IZA Discussion Papers 5720, Institute of Labor Economics (IZA).
    7. John Gibson & Steven Stillman & David McKenzie & Halahingano Rohorua, 2013. "Natural Experiment Evidence On The Effect Of Migration On Blood Pressure And Hypertension," Health Economics, John Wiley & Sons, Ltd., vol. 22(6), pages 655-672, June.
    8. Eva Deuchert, 2011. "The Virgin HIV Puzzle: Can Misreporting Account for the High Proportion of HIV Cases in Self-reported Virgins?," Journal of African Economies, Centre for the Study of African Economies, vol. 20(1), pages 60-89, January.
    9. Peter J. Rothe & Linda J. Carroll, 2009. "Hazards Faced by Young Designated Drivers: In-Car Risks of Driving Drunken Passengers," IJERPH, MDPI, vol. 6(6), pages 1-18, June.
    10. Fernando Abad-Franch & Gonçalo Ferraz & Ciro Campos & Francisco S Palomeque & Mario J Grijalva & H Marcelo Aguilar & Michael A Miles, 2010. "Modeling Disease Vector Occurrence when Detection Is Imperfect: Infestation of Amazonian Palm Trees by Triatomine Bugs at Three Spatial Scales," PLOS Neglected Tropical Diseases, Public Library of Science, vol. 4(3), pages 1-11, March.
    11. Elizabeth Kristjansson & Damian K Francis & Selma Liberato & Marik Benkhalti Jandu & Vivian Welch & Malek Batal & Trish Greenhalgh & Tamara Rader & Eamonn Noonan & Beverley Shea & Laura Janzen & Georg, 2013. "PROTOCOL: Feeding Interventions for Improving the Physical and Psychosocial Health of Disadvantaged Children Aged Three Months to Five Years: Protocol for a Systematic Review," Campbell Systematic Reviews, John Wiley & Sons, vol. 9(1), pages 1-41.
    12. Stadnik SM & Saiko OV, 2020. "Neuron-Specific Enolaza as a Marker of Lesion Cerebral Tissue in Patients with Ischemic Stroke," Biomedical Journal of Scientific & Technical Research, Biomedical Research Network+, LLC, vol. 31(1), pages 23816-23820, October.
    13. Pinna Pintor, Matteo & Fumagalli, Elena & Suhrcke, Marc, 2024. "The impact of health on labour market outcomes: A rapid systematic review," Health Policy, Elsevier, vol. 143(C).
    14. Feyza G. Sahinyazan & Marie‐Ève Rancourt & Vedat Verter, 2021. "Food Aid Modality Selection Problem," Production and Operations Management, Production and Operations Management Society, vol. 30(4), pages 965-983, April.
    15. La Torre, Davide & Liuzzi, Danilo & Marsiglio, Simone, 2021. "Epidemics and macroeconomic outcomes: Social distancing intensity and duration," Journal of Mathematical Economics, Elsevier, vol. 93(C).
    16. Danielle N. Medgyesi & Heather A. Holmes & Jeff E. Angermann, 2017. "Investigation of Acute Pulmonary Deficits Associated with Biomass Fuel Cookstove Emissions in Rural Bangladesh," IJERPH, MDPI, vol. 14(6), pages 1-15, June.
    17. Terje A Eikemo & Rasmus Hoffmann & Margarete C Kulik & Ivana Kulhánová & Marlen Toch-Marquardt & Gwenn Menvielle & Caspar Looman & Domantas Jasilionis & Pekka Martikainen & Olle Lundberg & Johan P Mac, 2014. "How Can Inequalities in Mortality Be Reduced? A Quantitative Analysis of 6 Risk Factors in 21 European Populations," PLOS ONE, Public Library of Science, vol. 9(11), pages 1-1, November.
    18. Jari Lahti & Marius Lahti & Anu-Katriina Pesonen & Kati Heinonen & Eero Kajantie & Tom Forsén & Kristian Wahlbeck & Clive Osmond & David J P Barker & Johan G Eriksson & Katri Räikkönen, 2014. "Prenatal and Childhood Growth, and Hospitalization for Alcohol Use Disorders in Adulthood: The Helsinki Birth Cohort Study," PLOS ONE, Public Library of Science, vol. 9(1), pages 1-8, January.
    19. Pierre Kopp & Marysia Ogrodnik, 2017. "The social cost of drugs in France in 2010," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 18(7), pages 883-892, September.
    20. Tsung-Ming Tsao & Ming-Jer Tsai & Ya-Nan Wang & Heng-Lun Lin & Chang-Fu Wu & Jing-Shiang Hwang & Sandy-HJ Hsu & Hsing Chao & Kai-Jen Chuang & Charles- CK Chou & Ta-Chen Su, 2014. "The Health Effects of a Forest Environment on Subclinical Cardiovascular Disease and Heath-Related Quality of Life," PLOS ONE, Public Library of Science, vol. 9(7), pages 1-8, July.
    21. Zhiwei Xu & Perry E. Sheffield & Wenbiao Hu & Hong Su & Weiwei Yu & Xin Qi & Shilu Tong, 2012. "Climate Change and Children’s Health—A Call for Research on What Works to Protect Children," IJERPH, MDPI, vol. 9(9), pages 1-19, September.

    Corrections

    All material on this site has been provided by the respective publishers and authors. You can help correct errors and omissions. When requesting a correction, please mention this item's handle: RePEc:hal:wpaper:hal-00752254. See general information about how to correct material in RePEc.

    If you have authored this item and are not yet registered with RePEc, we encourage you to do it here. This allows to link your profile to this item. It also allows you to accept potential citations to this item that we are uncertain about.

    If CitEc recognized a bibliographic reference but did not link an item in RePEc to it, you can help with this form .

    If you know of missing items citing this one, you can help us creating those links by adding the relevant references in the same way as above, for each refering item. If you are a registered author of this item, you may also want to check the "citations" tab in your RePEc Author Service profile, as there may be some citations waiting for confirmation.

    For technical questions regarding this item, or to correct its authors, title, abstract, bibliographic or download information, contact: CCSD (email available below). General contact details of provider: https://hal.archives-ouvertes.fr/ .

    Please note that corrections may take a couple of weeks to filter through the various RePEc services.

    IDEAS is a RePEc service. RePEc uses bibliographic data supplied by the respective publishers.