Introduction Drugs for rare diseases are often associated with extremely high costs which can be a barrier to patients' accessibility. Due to scarce healthcare resources, funding for expensive orphan therapies is a predicament for policy-makers and patients. In Canada, funding decisions for drugs for rare diseases are made at the provincial and regional level primarily on an individual basis, and are typically made on the grounds of historical and political factors. In many cases, limited reflection is given to the collective costs or the alternative applications of these resources. To ensure that equitable decisions are made, formal and transparent processes for the reimbursement of orphan drugs are needed.

Objectives Given the unique economic and ethical challenges associated with orphan drugs, the applicability of existing approaches used for the priority setting of health care resources may be limited. This study will consider existing priority setting frameworks in order to identify the decision criteria that can be applied for the funding of drugs for rare diseases.

Methods A systematic review will be conducted to identify the available frameworks used when making healthcare resource allocation decisions. A case study of an orphan drug therapy will be used to assess the varying funding decisions that may result from applying different frameworks and decision criteria.

Results Work in progress.

Conclusions This research will help establish an understanding of the available priority setting frameworks and the decision criteria that can be applied when making reasonable conclusions related to the reimbursement of orphan drugs. An in depth understanding of the factors to consider when making priority setting decisions may help in the development of a standardized framework for the funding of drugs for rare diseases.