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The impact of new (orphan) drug approvals on premature mortality from rare diseases in the United States and France, 1999–2007

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  • Frank Lichtenberg

Abstract

This paper investigates the impact of the introduction of new orphan drugs on premature mortality from rare diseases using longitudinal, disease-level data obtained from a number of major databases. The analysis is performed using data from two countries: the United States (during the period 1999–2006) and France (during the period 2000–2007). For both countries, we estimate models using two alternative definitions of premature mortality, several alternative criteria for inclusion in the set of rare diseases, and several values of the potential lag between new drug approvals and premature mortality reduction. Both the United States and French estimates indicate that, overall, premature mortality from rare diseases is unrelated to the cumulative number of drugs approved 0–2 years earlier but is significantly inversely related to the cumulative number of drugs approved 3–4 years earlier. This delay is not surprising, since most patients probably do not have access to a drug until several years after it has been launched. Although the estimates for the two countries are qualitatively similar, the estimated magnitudes of the US coefficients are about four times as large as the magnitudes of the French coefficients. This may be partly due to greater errors in measuring dates of drug introduction in France. Also, access to new drugs may be more restricted in France than it is in the United States. Our estimates indicate that, in the United States, potential years of life lost to rare diseases before age 65 (PYLL65) declined at an average annual rate of 3.3% and that, in the absence of lagged new drug approvals, PYLL65 would have increased at a rate of 0.9%. Since the US population aged 0–64 was increasing at the rate of 1.0% per year, this means that PYLL65 per person under 65 would have remained approximately constant. The reduction in the US growth rate of PYLL65 attributable to lagged new drug approvals was 4.2%. In France, PYLL65 declined at an average annual rate of 1.8%. The estimates imply that, in the absence of lagged new drug approvals, it would have declined at a rate of 0.6%. The reduction in the French growth rate of PYLL65 attributable to lagged new drug approvals was 1.1%. Earlier access to orphan drugs could result in earlier reductions in premature mortality from rare diseases. Copyright Springer-Verlag (outside the USA) 2013

Suggested Citation

  • 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.
  • Handle: RePEc:spr:eujhec:v:14:y:2013:i:1:p:41-56
    DOI: 10.1007/s10198-011-0349-4
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    References listed on IDEAS

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    1. Frank Lichtenberg, 2009. "The effect of new cancer drug approvals on the life expectancy of American cancer patients, 1978-2004," Economics of Innovation and New Technology, Taylor & Francis Journals, vol. 18(5), pages 407-428.
    2. Frank R. Lichtenberg, 2009. "Have newer cardiovascular drugs reduced hospitalization? Evidence from longitudinal country‐level data on 20 OECD countries, 1995–2003," Health Economics, John Wiley & Sons, Ltd., vol. 18(5), pages 519-534, May.
    3. Frank R. Lichtenberg, 2005. "Pharmaceutical Knowledge-Capital Accumulation and Longevity," NBER Chapters, in: Measuring Capital in the New Economy, pages 237-274, National Bureau of Economic Research, Inc.
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    Cited by:

    1. Frank R. Lichtenberg, 2015. "Pharmaceutical Innovation, Longevity, and Medical Expenditure in Greece, 1995-2010," International Journal of the Economics of Business, Taylor & Francis Journals, vol. 22(2), pages 277-299, July.
    2. Ana Beatriz D′Avó Luís & Mikyung Kelly Seo, 2021. "Has the development of cancer biomarkers to guide treatment improved health outcomes?," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 22(5), pages 789-810, July.
    3. 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.

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    More about this item

    Keywords

    Orphan drugs; Orphan diseases; Rare diseases; Mortality; Innovation; I1; J1; L65; O3;
    All these keywords.

    JEL classification:

    • I1 - Health, Education, and Welfare - - Health
    • J1 - Labor and Demographic Economics - - Demographic Economics
    • L65 - Industrial Organization - - Industry Studies: Manufacturing - - - Chemicals; Rubber; Drugs; Biotechnology; Plastics
    • O3 - Economic Development, Innovation, Technological Change, and Growth - - Innovation; Research and Development; Technological Change; Intellectual Property Rights

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