Societal Preferences for Funding Orphan Drugs in the United Kingdom: An Application of Person Trade-Off and Discrete Choice Experiment Methods

Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research
Siobhan M BourkeDyfrig A Hughes

Abstract

It is unclear whether UK National Health Service (NHS) policies for orphan drugs, which permit funding of non-cost-effective treatments, reflect societal preferences. We conducted person trade-off (PTO) and discrete choice experiment (DCE) among 3950 adults selected to be representative of the UK general population. Experimental design was informed by surveys of patients affected by rare diseases, their caregivers, health care staff, and policymakers. Societal preferences were estimated in relation to treating a common disease, increases in waiting lists, or filling of vacant NHS posts. Results of the DCE were applied to recently licensed orphan drugs. On the basis of equal cost, the majority of respondents to the PTO (54%; 95% confidence interval [CI] 50-59) chose to allocate funds equally between patients treated for rare diseases and those treated for common diseases, with 32% (95% CI 28-36) favoring treating rare diseases over treating common diseases (14%; 95% CI 11-17), which this reduced to 23% (95% CI 20-27) when rare disease treatments were 10 times more expensive. When framed differently, more respondents prioritized not increasing waiting list size (43%; 95% CI 39-48) than to treat rare disease patients (34%; 95% CI ...Continue Reading

Citations

Feb 19, 2020·The Cochrane Database of Systematic Reviews·Tara CarneyCharles Dh Parry
Mar 2, 2021·Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research·Isaac Aranda-ReneoJulio López-Bastida
Apr 28, 2021·Orphanet Journal of Rare Diseases·Fernando de Andrés-NogalesUNKNOWN FinMHU-MCDA Group
Jun 10, 2021·Journal of Medical Ethics·Andreas Albertsen
Jun 1, 2021·Frontiers in Pharmacology·Alessandra BlondaSteven Simoens
Jul 2, 2021·Orphanet Journal of Rare Diseases·Bettina M ZimmermannMatthias R Baumgartner
Sep 24, 2021·Regenerative Medicine·Karen MacphersonMelanie Calvert

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