Societal preferences for granting orphan drugs special status in reimbursement decisions

Background

Orphan drugs, for patients with a rare disease, are increasingly available but often do not meet standard cost-effectiveness criteria for reimbursement. Consequently, policymakers regularly face the dilemma whether to relax these criteria for reimbursing orphan drugs. We examined whether—and why—there would be societal support for such differential treatment of orphan drugs.

Methods

We conducted a labelled discrete choice experiment in a sample of the adult population (n = 1,172) in the Netherlands. Respondents were presented with ten choices on whether to reimburse an orphan drug given that a non-orphan drug with similar characteristics would not be reimbursed, because it was not cost-effective, and asked to explain their choices. We used random-intercept logit regression models and inductive coding for analysing the quantitative and qualitative data.

Results

Of the respondents, 36.4% consistently chose for reimbursing the orphan drug, mostly because “everyone is entitled to live a healthy life and good quality healthcare”, and 17.3% consistently for not reimbursing the orphan drug, mostly because “[this] is unfair to patients with a common disease”. The remaining 46.3% made alternating choices and were more likely to choose for reimbursing orphan drugs when patients were aged between 1 and 70 years, had moderate disease severity, and considerable health gain from treatment.

Conclusions

This study finds considerable support but also strong preference heterogeneity amongst members of the public in the Netherlands for differential treatment of orphan drugs in reimbursement decisions, when these drugs do not meet common cost-effectiveness criteria. However, a substantial minority opposes differential treatment, mostly on moral grounds.