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R&D and market size: who benefits from orphan drug regulation?

Author

Listed:
  • Simona Gamba

    (Department of Economics (University of Verona))

  • Laura Magazzini

    (Department of Economics (University of Verona))

  • Paolo Pertile

    (Department of Economics (University of Verona))

Abstract

Since the early 80s, orphan drug regulations have been introduced to stimulate R&D for rare diseases. We develop a theoretical model to study the heterogeneous impact on optimal R&D decisions of the incentives for diseases with different levels of prevalence. We show the mechanisms through which the type of incentives deployed by orphan drug regulations may stimulate R&D more for orphan diseases with comparatively high prevalence, thus increasing inequality within the class of orphan diseases. Using data from the Food and Drug Administration on the number of orphan designations, our empirical analysis shows that, while R&D has increased over time for all orphan diseases, the increase has been much greater for the less rare. According to our baseline specification, the difference between the predicted number of orphan designations for a disease belonging to the highest and the lowest class of prevalence is 5.6 times larger after 2008 than it was in 1983. Our findings support the idea that the type of incentives in place may be responsible for this increase in inequality within orphan diseases.

Suggested Citation

  • Simona Gamba & Laura Magazzini & Paolo Pertile, 2019. "R&D and market size: who benefits from orphan drug regulation?," Working Papers 09/2019, University of Verona, Department of Economics.
  • Handle: RePEc:ver:wpaper:09/2019
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    References listed on IDEAS

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    1. DiMasi, Joseph A. & Grabowski, Henry G. & Hansen, Ronald W., 2016. "Innovation in the pharmaceutical industry: New estimates of R&D costs," Journal of Health Economics, Elsevier, vol. 47(C), pages 20-33.
    2. Pierre Dubois & Olivier de Mouzon & Fiona Scott-Morton & Paul Seabright, 2015. "Market size and pharmaceutical innovation," RAND Journal of Economics, RAND Corporation, vol. 46(4), pages 844-871, October.
    3. Wolfgang Keller, 2002. "Geographic Localization of International Technology Diffusion," American Economic Review, American Economic Association, vol. 92(1), pages 120-142, March.
    4. G. M.P. Swann, 2009. "The Economics of Innovation," Books, Edward Elgar Publishing, number 13211.
    5. Yin, Wesley, 2009. "R&D policy, agency costs and innovation in personalized medicine," Journal of Health Economics, Elsevier, vol. 28(5), pages 950-962, September.
    6. Frank Lichtenberg, 2013. "The impact of new (orphan) drug approvals on premature mortality from rare diseases in the United States and France, 1999–2007," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 14(1), pages 41-56, February.
    7. Pavitt, Keith, 1984. "Sectoral patterns of technical change: Towards a taxonomy and a theory," Research Policy, Elsevier, vol. 13(6), pages 343-373, December.
    8. Wolfgang Keller, 2004. "International Technology Diffusion," Journal of Economic Literature, American Economic Association, vol. 42(3), pages 752-782, September.
    9. Greene, William, 2010. "Testing hypotheses about interaction terms in nonlinear models," Economics Letters, Elsevier, vol. 107(2), pages 291-296, May.
    10. Eliana Barrenho & Marisa Miraldo & Peter C. Smith, 2019. "Does global drug innovation correspond to burden of disease? The neglected diseases in developed and developing countries," Health Economics, John Wiley & Sons, Ltd., vol. 28(1), pages 123-143, January.
    11. Daron Acemoglu & Joshua Linn, 2004. "Market Size in Innovation: Theory and Evidence from the Pharmaceutical Industry," The Quarterly Journal of Economics, Oxford University Press, vol. 119(3), pages 1049-1090.
    12. Rainer Winkelmann, 2008. "Econometric Analysis of Count Data," Springer Books, Springer, edition 0, number 978-3-540-78389-3, January.
    13. Jobjörnsson, Sebastian & Forster, Martin & Pertile, Paolo & Burman, Carl-Fredrik, 2016. "Late-stage pharmaceutical R&D and pricing policies under two-stage regulation," Journal of Health Economics, Elsevier, vol. 50(C), pages 298-311.
    14. Laura Magazzini & Fabio Pammolli & Massimo Riccaboni, 2009. "Nuove politiche per l'innovazione nel settore delle scienze della vita," Working Papers CERM 03-2009, Competitività, Regole, Mercati (CERM).
    15. Mansfield, Edwin, 1995. "Academic Research Underlying Industrial Innovations:," The Review of Economics and Statistics, MIT Press, vol. 77(1), pages 55-65, February.
    16. Matthew Herder, 2017. "What Is the Purpose of the Orphan Drug Act?," PLOS Medicine, Public Library of Science, vol. 14(1), pages 1-4, January.
    17. Yin, Wesley, 2008. "Market incentives and pharmaceutical innovation," Journal of Health Economics, Elsevier, vol. 27(4), pages 1060-1077, July.
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    Cited by:

    1. Massimo Florio & Simona Gamba, 2021. "Biomed Europa: After the coronavirus, a public infrastructure to overcome the pharmaceutical oligopoly," Annals of Public and Cooperative Economics, Wiley Blackwell, vol. 92(3), pages 387-409, September.

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    More about this item

    Keywords

    pharmaceuticals; innovation; orphan regulations; market size; inequality;
    All these keywords.

    JEL classification:

    • I14 - Health, Education, and Welfare - - Health - - - Health and Inequality
    • I18 - Health, Education, and Welfare - - Health - - - Government Policy; Regulation; Public Health
    • O31 - Economic Development, Innovation, Technological Change, and Growth - - Innovation; Research and Development; Technological Change; Intellectual Property Rights - - - Innovation and Invention: Processes and Incentives
    • O38 - Economic Development, Innovation, Technological Change, and Growth - - Innovation; Research and Development; Technological Change; Intellectual Property Rights - - - Government Policy
    • C35 - Mathematical and Quantitative Methods - - Multiple or Simultaneous Equation Models; Multiple Variables - - - Discrete Regression and Qualitative Choice Models; Discrete Regressors; Proportions

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