Societal Preferences For Funding Orphan Drugs In The UK: An Application Of Value Based Pricing Using Discrete Choice Experiment Methods

Abstract

Orphan drugs tend to be expensive and many may not provide value for money by standard measures of cost-effectiveness. Their general availability by the National Health Service (NHS) suggests that special criteria apply to rare diseases for OD reimbursement. The aim of the present study is to identify characteristics that may influence policy and to determine a value based price (VBP) for orphan drugs based on public preferences. Participants (n=2000) who completed a discrete choice experiment (DCE) made choices between hypothetical drugs described by varying disease and drug characteristics. The coefficients from the DCE were used to estimate a utility model based on reimbursement evidence and thus the VBP of evaluated orphan drugs. Rarity -0.888 (95% CI -0.9439, -0.8323) and the availability of other drug treatments -0.0756 (95% CI -0.1511, -0.0272) was unlikely to influence the general populations decision when determining which treatment the NHS should provide. However, the results showed a strong preference of the general public to fund drugs that had large treatment benefits 0.8536 (95% CI 0.8044 to 0.9027) and improvements to every day life 0.6589 (95% CI 0.6261, 0.6918), regardless of the prevalence of the disease. According to the utility model, 6 of 12 NHS approved orphan drugs passed the reimbursement preference threshold of the general population. Respondents would not prefer NHS funding for 5 of the remainder (VBP <£0 per patient per year), while one would be acceptable with a 9-fold reduction in the list price. Estimation of the VBP suggests that the treatment benefit of a drug is the primary driver when determining the value of a drug. Based on our model, there does seem to be a preference from the general population to fund high cost treatments regardless of disease prevalence.