OBJECTIVES: Rare diseases, due to their low prevalence, are often overlooked. However, their economic burden can be very significant in low and middle-income countries due to their high-cost treatment. This scenario poses challenges for the elaboration of health technology assessments due to the quantity and quality of information on these diseases and their therapeutic options. The aim of this scoping review is to map out how the economic evaluations of technologies for the treatment of rare diseases are being carried out, in terms of data sources, methods, perspective of analysis and costs used in Latin America and Southeast Asia.

METHODS: We conducted a literature search using Medline, Embase, LILACS, and Scopus. Two reviewers performed the evidence screening and selection. Another reviewer conducted the data extraction process using a predesigned form and a fourth one was in charge of quality control. The findings were synthesized and summarized through a narrative description.

RESULTS: A total of 31 references were included. More information was found in Latin America (88%), specifically in Brazil (33%). The most used methods for economic evaluations were cost-effectiveness analyses (45%) and cost analysis (35%), the most used perspective of analysis was that of the public third-party payer (61%), direct medical costs were most frequently used (86%), and data from institutions of the national healthcare system (27%) and literature (21%) were the most frequently used data sources. We found limitations in the economic evaluations concerning lack of information such as epidemiological data on the rare disease, either local or worldwide.

CONCLUSIONS: There have been some efforts directed towards the conduction of economic evaluations for rare diseases in Latin American and Southeast Asian countries; however, it becomes necessary to strengthen formal research so as to overcome the limitations derived from lack of information in these therapeutic areas in both regions.