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Appraising Ultra-Orphan Drugs: Is Cost-Per-QALY Appropriate? A Review of the Evidence

Author

Listed:
  • Towse, A.
  • Garau, M.

Abstract

The report addresses the implications of NICE appraising treatments for very rare diseases using a cost-per-QALY gained decision rule of the type used by NICE in its Technology Appraisal Programme to appraise therapies for more common conditions. Given the importance of non-QALY elements in the assessment of HSTs, such as treatment impact on the process of care and on the patients' or their carers' ability to go to school or to work respectively, and issues in measuring quality of life when the population affected are infants or young children, it is inappropriate to focus the appraisal of treatments for very rare diseases solely on a cost-per-QALY measure. Given the lack of empirical basis, the new £100,000 cost per QALY threshold and its further possible uplift up by a factor of three seem arbitrary.

Suggested Citation

  • Towse, A. & Garau, M., 2018. "Appraising Ultra-Orphan Drugs: Is Cost-Per-QALY Appropriate? A Review of the Evidence," Consulting Reports 001978, Office of Health Economics.
  • Handle: RePEc:ohe:conrep:001978
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    File URL: https://www.ohe.org/system/files/private/publications/468%20-%20Appraising%20ultra-orphan%20drugs.pdf
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    References listed on IDEAS

    as
    1. Paul Dolan & Rebecca Shaw & Aki Tsuchiya & Alan Williams, 2005. "QALY maximisation and people's preferences: a methodological review of the literature," Health Economics, John Wiley & Sons, Ltd., vol. 14(2), pages 197-208.
    2. Jose-Maria Abellan-Perpiñan & Jose-Luis Pinto-Prades, 1999. "Health state after treatment: a reason for discrimination?," Health Economics, John Wiley & Sons, Ltd., vol. 8(8), pages 701-707.
    3. repec:spr:aphecp:v:15:y:2017:i:2:d:10.1007_s40258-017-0310-5 is not listed on IDEAS
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    More about this item

    Keywords

    Judging value for money and improving decision making;

    JEL classification:

    • I1 - Health, Education, and Welfare - - Health

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