Discussion on Threshold of Pharmacoeconomics for Orphan Drugs

Corresponding author: Wu Jing, jingwu@tju.edu.cn

DOI: 10.12201/bmr.202110.00039

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  Abstract: Objective: To adjust the method of setting threshold for orphan drugs that can greatly improve the health of patients with severe and rare diseases and improve the rationality of pharmacoeconomics. Methods: This article analyzes theory of setting threshold for pharmacoeconomics, and discuss the theoretical shortcoming of setting threshold from the perspective of health utility theory, and explore the theoretical solution of threshold adjustment for orphan drugs. Literature retrieval and summary of international experience were carried out to verify the theoretical solution. Results: Cost-utility analysis considers that health utility corresponding to one QALY remains unchanged, which underestimates the value of orphan drugs which can improve health of patients with severe and rare diseases. For patients with severe and rare diseases of almost zero QALYs, marginal health utility of extra one QALY will increase as QALYs increasing, until patients can participate various social activities normally, then the marginal health utility will decrease. Based on the principle of paying for utility, if orphan drugs can greatly improve health of patients with severe and rare diseases, the threshold of each QALY should be higher that of general new drugs which can slightly improve health. Conclusions: Traditional constant threshold should be adjusted according to health improvement to evaluate economics of orphan drugs scientifically.

  Key words: Orphan drugs; Pharmacoeconomics; Threshold; Quality-Adjusted Life Years; Health utility

  Submit time: 21 December 2021

  Copyright: The copyright holder for this preprint is the author/funder, who has granted biomedRxiv a license to display the preprint in perpetuity.
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