IDEAS home Printed from https://ideas.repec.org/a/spr/pharme/v38y2020i4d10.1007_s40273-019-00872-8.html
   My bibliography  Save this article

Determinants of Orphan Drug Prices in Germany

Author

Listed:
  • Franziska Worm

    (University Duisburg-Essen)

  • Charalabos-Markos Dintsios

    (Heinrich-Heine-University Düsseldorf)

Abstract

Background and Objective Legislation introduced in 2011 in Germany has instituted an early benefit assessment of newly licensed pharmaceuticals with a subsequent price negotiation. For orphan drugs (ODs) a special legal framework applies, which accounts for the fact that ODs do not have to prove an added benefit over an appropriate comparative therapy previously determined by the decision maker. As, in addition, the content of negotiations between pharmaceutical companies and the payer is confidential, the aim of this study was to identify factors influencing the negotiated prices of ODs. Methods Twelve hypotheses on factors influencing the negotiated OD price were derived based on the existing literature and framework agreement between payers and pharmaceutical unions according to German social legislation. Univariate analyses were applied to detect statistically significant correlations between annual therapeutic costs of ODs and the hypothesized factors. Bivariate analyses were used to determine confounding factors. In addition, a multiple ordinary least squares (OLS) regression with backward selection was conducted. Finally, sensitivity analyses assessed the robustness of the results. Results Thirty-five ODs were included in the analysis. The univariate analyses and subsequent sensitivity analyses validated five of the 12 hypotheses formulated. Univariate analyses suggest a statistically significant association between the OD price and the (i) therapeutic area; (ii) approval for pediatric care; (iii) treatment population size; (iv) cost of comparative therapies; and (v) European prices. The OLS regression identified European prices as the variable with the strongest association with the negotiated prices. Conclusion We show that German OD pricing is a multivariate phenomenon. However, due to interdependencies, these results must be treated with caution.

Suggested Citation

  • Franziska Worm & Charalabos-Markos Dintsios, 2020. "Determinants of Orphan Drug Prices in Germany," PharmacoEconomics, Springer, vol. 38(4), pages 397-411, April.
    • Handle: RePEc:spr:pharme:v:38:y:2020:i:4:d:10.1007_s40273-019-00872-8
    DOI: 10.1007/s40273-019-00872-8
    as

    Download full text from publisher

    File URL: http://link.springer.com/10.1007/s40273-019-00872-8
    File Function: Abstract
    Download Restriction: Access to the full text of the articles in this series is restricted.
    File URL: https://libkey.io/10.1007/s40273-019-00872-8?utm_source=ideas
    LibKey link: if access is restricted and if your library uses this service, LibKey will redirect you to where you can use your library subscription to access this item
    ---><---

    As the access to this document is restricted, you may want to search for a different version of it.

    References listed on IDEAS

    as
    1. Javier Orofino & Javier Soto & Miguel Casado & Itziar Oyagüez, 2010. "Global spending on orphan drugs in France, Germany, the UK, Italy and Spain during 2007," Applied Health Economics and Health Policy, Springer, vol. 8(5), pages 301-315, September.
    2. Jörg Ruof & Thomas Staab & Charalabos-Markos Dintsios & Jakob Schröter & Friedrich Wilhelm Schwartz, 2016. "Comparison of post-authorisation measures from regulatory authorities with additional evidence requirements from the HTA body in Germany – are additional data requirements by the Federal Joint Committ," Health Economics Review, Springer, vol. 6(1), pages 1-11, December.
    3. Henschke, Cornelia & Sundmacher, Leonie & Busse, Reinhard, 2013. "Structural changes in the German pharmaceutical market: Price setting mechanisms based on the early benefit evaluation," Health Policy, Elsevier, vol. 109(3), pages 263-269.
    Full references (including those not matched with items on IDEAS)

    Citations

    Citations are extracted by the CitEc Project, subscribe to its RSS feed for this item.
    as


    Cited by:

    1. Wettstein, Dominik J. & Boes, Stefan, 2022. "How value-based policy interventions influence price negotiations for new medicines: An experimental approach and initial evidence," Health Policy, Elsevier, vol. 126(2), pages 112-121.

    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. Leopold, C. & Vogler, S. & Habl, C. & Mantel-Teeuwisse, A.K. & Espin, J., 2013. "Personalised medicine as a challenge for public pricing and reimbursement authorities – A survey among 27 European countries on the example of trastuzumab," Health Policy, Elsevier, vol. 113(3), pages 313-322.
    2. Olberg, Britta & Perleth, Matthias & Busse, Reinhard, 2014. "The new regulation to investigate potentially beneficial diagnostic and therapeutic methods in Germany: Up to international standard?," Health Policy, Elsevier, vol. 117(2), pages 135-145.
    3. 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.
    4. Vogler, Sabine & Zimmermann, Nina & de Joncheere, Kees, 2016. "Policy interventions related to medicines: Survey of measures taken in European countries during 2010–2015," Health Policy, Elsevier, vol. 120(12), pages 1363-1377.
    5. Degtiar, Irina, 2017. "A review of international coverage and pricing strategies for personalized medicine and orphan drugs," Health Policy, Elsevier, vol. 121(12), pages 1240-1248.
    6. Veronika Kalouguina & Joël Wagner, 2020. "Challenges and Solutions for Integrating and Financing Personalized Medicine in Healthcare Systems: A Systematic Literature Review," JRFM, MDPI, vol. 13(11), pages 1-22, November.
    7. Christine Blome & Matthias Augustin & Hidayet Metin & David Lohrberg, 2017. "Four years of early benefit assessment of new drugs in Germany: a qualitative study on methodological requirements for quality of life data," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 18(2), pages 181-193, March.
    8. Monika Wagner & Hanane Khoury & Jacob Willet & Donna Rindress & Mireille Goetghebeur, 2016. "Can the EVIDEM Framework Tackle Issues Raised by Evaluating Treatments for Rare Diseases: Analysis of Issues and Policies, and Context-Specific Adaptation," PharmacoEconomics, Springer, vol. 34(3), pages 285-301, March.
    9. Monika Wagner & Hanane Khoury & Jacob Willet & Donna Rindress & Mireille Goetghebeur, 2016. "Can the EVIDEM Framework Tackle Issues Raised by Evaluating Treatments for Rare Diseases: Analysis of Issues and Policies, and Context-Specific Adaptation," PharmacoEconomics, Springer, vol. 34(3), pages 285-301, March.
    10. Katharina E. Fischer & Tom Stargardt, 2014. "Early Benefit Assessment of Pharmaceuticals in Germany," Medical Decision Making, , vol. 34(8), pages 1030-1047, November.
    11. Fischer, Katharina Elisabeth & Heisser, Thomas & Stargardt, Tom, 2016. "Health benefit assessment of pharmaceuticals: An international comparison of decisions from Germany, England, Scotland and Australia," Health Policy, Elsevier, vol. 120(10), pages 1115-1122.
    12. Alain Denis & Lut Mergaert & Christel Fostier & Irina Cleemput & Steven Simoens, 2010. "Issues surrounding orphan disease and orphan drug policies in Europe," Applied Health Economics and Health Policy, Springer, vol. 8(5), pages 343-350, September.
    13. Stephan Eger & Jörg Mahlich, 2014. "Pharmaceutical regulation in Europe and its impact on corporate R&D," Health Economics Review, Springer, vol. 4(1), pages 1-9, December.
    14. Małgorzata Skweres-Kuchta & Iwona Czerska & Elżbieta Szaruga, 2023. "Literature Review on Health Emigration in Rare Diseases—A Machine Learning Perspective," IJERPH, MDPI, vol. 20(3), pages 1-31, January.
    15. Ulrike Theidel & J-Matthias Graf von der Schulenburg, 2016. "Benefit assessment in Germany: implications for price discounts," Health Economics Review, Springer, vol. 6(1), pages 1-12, December.
    16. Hörn, Helmut & Nink, Katrin & McGauran, Natalie & Wieseler, Beate, 2014. "Early benefit assessment of new drugs in Germany – Results from 2011 to 2012," Health Policy, Elsevier, vol. 116(2), pages 147-153.

    More about this item

    Statistics

    Access and download statistics

    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:spr:pharme:v:38:y:2020:i:4:d:10.1007_s40273-019-00872-8. 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: Sonal Shukla or Springer Nature Abstracting and Indexing (email available below). General contact details of provider: http://www.springer.com .

    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.