OBJECTIVES: Most of the world’s population resides in low- and middle-income countries (LMICs), many of which do not have orphan drug policies. Thus, access to treatments for rare diseases in LMICs represents a large global unmet need. We sought to assess orphan drug availability and pricing patterns in low-, middle-, and high-income countries.

METHODS: We identified rare-disease treatments using the US Food and Drug Administration’s Orphan Drug Designations and Approvals list. For pricing analysis, we selected treatments approved between January 1, 2012, and January 1, 2022, that were available in >20 markets, including >2 LMIC markets. We classified countries as low, middle, or high income according to the World Bank’s income classification criteria. We obtained pricing data from NAVLIN by EVERSANA^TM (a Web-based pricing subscription service). We analyzed prices by country income classification to identify potential pricing patterns between income classes and used the chi-squared test to determine statistically significant differences in prices between income groups.

RESULTS: Twelve treatments met analysis criteria. Of these, none were approved in low-income countries, whereas availability ranged from 26% in middle-income countries to 64% in high-income countries. No statistically significant differences in prices for orphan drugs between middle- and high-income countries were identified.

CONCLUSIONS: Orphan drugs have very limited presence in LMICs. When these rare-disease treatments are available in LMIC markets, prices are similar to those in high-income countries. Although the pharmaceutical industry has emphasized drug development for rare diseases, the benefits of these advancements have not yet reached many patients. The pharmaceutical industry’s technological innovation must be matched with novel pricing strategies and funding mechanisms, as well as tailored contracting approaches, to facilitate and maximize orphan drug access for patients in LMICs.