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Valuing Rare Pediatric Drugs: An Economics Perspective

Author

Listed:
  • Rena M. Conti
  • Jonathan Gruber
  • Daniel A. Ollendorf
  • Peter J. Neumann

Abstract

There is a coming wave of novel genetic therapies aiming to treat rare pediatric disease. A large literature investigates the valuation of new treatments, but the valuation of treatments for rare pediatric illness raises a host of unique issues. In this paper, we review the challenges of applying both the standard economic model and standard approaches to estimating cost-effectiveness using the quality-adjusted life year (QALY) to this case. We argue that there are a large number of special issues that have only been partially addressed by past work and we conclude that more data and the development of new methods are vital as innovators, health technology assessment practitioners and policymakers confront the launch of these new drugs.

Suggested Citation

  • Rena M. Conti & Jonathan Gruber & Daniel A. Ollendorf & Peter J. Neumann, 2020. "Valuing Rare Pediatric Drugs: An Economics Perspective," NBER Working Papers 27978, National Bureau of Economic Research, Inc.
    • Handle: RePEc:nbr:nberwo:27978
    Note: AG CH EH PE
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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.
    2. Gamba, Simona & Magazzini, Laura & Pertile, Paolo, 2021. "R&D and market size: Who benefits from orphan drug legislation?," Journal of Health Economics, Elsevier, vol. 80(C).

    More about this item

    JEL classification:

    • I18 - Health, Education, and Welfare - - Health - - - Government Policy; Regulation; Public Health

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