OBJECTIVES: To describe alternative methodologies implemented by health technology assessment (HTA) agencies, as the inclusion of social values criteria or patient’s associations’ opinion in the decision process to evaluate health technologies for rare/orphan diseases.

METHODS: We conducted a scoping review, following the Joanna Briggs Institute methodology. The scoping review, consisted of seven phases: (1) identifying the research question; (2) searching for relevant studies; (3) selecting studies; (4) charting, extracting and tabulating data; (5) analyzing data; (6) consulting relevant experts; and (7) presenting the results.

RESULTS: The review process resulted in the inclusion of 19 articles and documents. We identified five agencies (NICE, IQWiG, CVOR, SMC and LFN) implementing alternative methodologies for HTA. The exceptional decision criteria implemented by the agencies were: quality of life (QOL), patient benefit, ethical and social considerations, degree of innovation, uncovered needs, burden of disease, cost-effectiveness analysis, severity of the disease, rarity of the disease and therapeutic effects.

CONCLUSIONS: This study provides evidence on the alternative methodologies implemented by HTA agencies for orphan drugs. Our study did not identify processes of implementing alternative HTA methodologies in Latin America. There remains a need for studies in Latin America, particularly of methodologies implemented by HTA local agencies. Future evidence may substantially impact upon the presented results and may help to develop new methods applicable in the Latin American context.