Economic Aspects of Reimbursement of Drugs for Rare Diseases

The reimbursement of drugs that treat rare diseases, also known as orphan drugs, have been the topic of much debate in the last few years presenting a problem to decision makers who are challenged by increasing drug budgets. This thesis covers three main objectives: 1. Are the Incremental Cost Effectiveness Ratios (ICERs) for rare diseases different from ICERs for non-rare diseases? Although it is believed that ICERs for drugs assessing rare diseases are higher than those of drugs assessing common diseases, no studies to date have formally examined the evidence for this belief. Our objective in this chapter was to assess whether there are differences in ICERs for rare and common cancers using published data. 2. What is the estimated cost of drug development for orphan drugs? Research and Development (R) costs associated with orphan drug development have not been well characterized in the published literature. The objective of this chapter was to identify the differences in clinical trial characteristics between orphan and non-orphan drugs and use that information to estimate the clinical R costs for both groups using publicly available data. 3. What country-specific economic and institutional factors can explain the variability in the reimbursement of orphan drugs in OECD (Organization for Economic Co-operation and Development) countries? It is known that there are differences in the approval and access of drugs across countries. Country-specific factors that lead to these variances have not been studied for orphan drugs. This chapter aims to better understand the differences in the approvals, launch and reimbursement of orphan drugs among 20 OECD countries and determine which economic and institutional factors can explain these differences. The results from these three chapters shed light on three different aspects of reimbursement of orphan drugs including cost-effectiveness, cost of drug development and access to orphan drugs. This research can be essential when developing orphan drug related policies across the world.Ph.D