Priority setting for orphan drugs: an international comparison

To describe the process of priority setting for two orphan drugs - Cerezyme and Fabrazyme - in Canada, Australia and Israel, in order to understand and improve the process based on stakeholder perspectives.

We conducted qualitative case studies of how three independent drug advisory committees made decisions relating to the funding of Cerezyme and Fabrazyme. Interviews were conducted with 22 informants, including committee members, patient groups and industry representatives.

(1) DESCRIPTION: Orphan drugs reimbursement recommendations by expert panels were based on clinical evidence, cost and cost-effectiveness analysis. (2) EVALUATION: Committee members expressed an overall preference for the current drug review process used by their own committee, but were concerned with the fairness of the process particularly for orphan drugs. Other informants suggested the inclusion of other relevant values (e.g. lack of alternative treatments) in order to improve the priority setting process. Some patient groups suggested the use of an alternative funding mechanism for orphan drugs.

Priority setting for drugs is not solely a technical process (involving cost-effective analysis, evidence-based medicine, etc.). Understanding the process by which reimbursement decisions are made for orphan drugs may help improve the system for future orphan drugs.