OBJECTIVES:

Orphan drugs for rare diseases tend to be high cost treatments. Current practice by UK health technology assessment organisations suggests that rarity in itself may be justification for exceeding standard thresholds of cost effectiveness. This study aims to examine whether or not there is support among the UK general public for prioritising treatments of patients with rare diseases.

METHODS:

Using PTO design, the preferences of 1000 people for allocating fixed funds between a drug treatment for patients with rare versus common diseases were determined. Further samples (n=500 each) were asked to allocate funding between a drug treatment for rare disease and i) increase waiting list or ii) reduced NHS staffing. Costs and treatment benefits were varied in each scenario.

RESULTS:

The general population generally preferred to distribute funds equally between patients with a common disease and those with a rare disease. Distribution of preferences for treating patients with: (i) rare, (ii) common disease, or (iii) equal allocation of funds, were: 32%, 14%, 54%. When cost of rare disease treatment increased, we observed less support for treating rare disease patients (19%, 54%, 27%). However, this was reversed if the drug also offered greater treatment benefits (71%, 50%, 44%). When asked to trade off between the alternative framing scenarios, respondents preferred to not increase waiting lists (45%, 9%, 46%, equal cost scenario) to fund treatments for rare diseases. However, respondents preferred replacing vacant NHS staff posts when treatment cost increased (31.2%, 34.4%, 34.4%).

CONCLUSIONS:

There is little support among the UK general public for prioritising treatments on the basis of disease rarity alone; although there was a preference observed when treatment benefit was greater, despite the increase in cost. This suggests that there may be a preference for funding high cost treatments for rare disease patients but only if treatment benefit was high.