The Effect of Market-Based Economic Factors on the Adoption of Orphan Drugs Across Multiple Countries

Orphan drugs are designed to treat rare disease. Multiple countries have approved regulations that support the creation of new orphan drugs. The regulations directly and indirectly affect orphan drug markets and contribute to classifying a country as being more or less market based. A more market-based country is recognized as functioning closest to free market principles, where the actions of supply, demand, and pricing operate with minimal government involvement. However, little information exists on how country market differences affect the adoption of these orphan drugs. This study argues that relationships exist between country market-based economic factors and the adoption of orphan drugs. This research uses a combination of qualitative and quantitative methods, drawing on orphan drug adoption of 13 marketed orphan drugs in France, Germany, Spain, the UK, and the US. Results show a statistically significant but negative relationship between the degree to which a country is market based and the adoption of orphan drugs. Results suggest that governments and regulators in less market-based countries support market factors that result in a more efficient delivery of orphan drug products to patients with rare diseases.