PharmacoEconomics & Outcomes News 804, p28 - 2 Jun 2018 Public thinks disease rarity does not justify funding orphan drugs The UK public appears to believe that disease rarity does not justify funding high-cost orphan drugs, according to findings of a study published in Value in Health. The Highly Specialised Technologies program of the National Institute for Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC) and the All Wales Medicines Strategy Group (AWMSG) use higher cost-effectiveness thresholds when appraising orphan drugs for rare diseases. Investigators conducted a person trade-off (PTO) and discrete choice experiment (DCE) in 3950 adults in the general population in the UK, using surveys in patients with rare diseases, their caregivers, healthcare professionals and policymakers to assess societal preferences with regard to treatment of rare versus common diseases, and National Health Service (NHS) funding for recently licensed orphan drugs. When drug costs were equal, 54% of respondents favoured equal funding in patients with rare diseases and those with common diseases, 32% of respondents favoured treating rare diseases, and 14% favoured treating common diseases. However, when treatments for rare diseases were ten times the cost of those for common diseases, only 23% of respondents favoured treating rare diseases over common diseases. When considering weighting lists versus treatment of rare diseases, 43% of respondents favoured not increasing the size of waiting lists, and 34% favoured treating rare diseases. "Respondents agreed with five of 12 positive appraisal recommendations for orphan drugs, even if their list price was higher, but preferred the NHS not to fund the remainder," noted the investigators. "This study shows that the UK general public does not consider rarity of a disease, in itself, as being sufficient to justify special preferential NHS funding," they said. Bourke SM, et al. Societal Preferences for Funding Orphan Drugs in the United Kingdom: An Application of Person Trade-Off and Discrete Choice Experiment Methods. Value in Health 21: 538-546, No. 5, May 2018. Available from: URL: http://doi.org/10.1016/j.jval.2017.12.026 803323496 1173-5503/18/0804-0001/$14.95 Adis © 2018 Springer International Publishing AG. All rights reserved PharmacoEconomics & Outcomes News 2 Jun 2018 No. 804
PharmacoEconomics & Outcomes News – Springer Journals
Published: Jun 2, 2018
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