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

Background: It is unclear whether UK National Health Service (NHS) policies for orphan drugs, which permit funding of non-cost-effective treatments, reflect societal preferences.

Methods: 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.

Results: 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 30-38).

Discussion: The DCE indicated a greater preference for treating a common disease over a rare disease. 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.

Conclusions: The general public does not value rarity as a sufficient reason to justify special consideration for additional NHS funding of orphan drugs. This has implications regarding the appropriateness of operating higher thresholds of cost-effectiveness.