Background: Safety´s evaluation of orphan drugs is challenging for health authorities due to scarce safety data collected in clinical studies and to a lesser extent in marketing phase, since they do not allow characterizing all the risks of these medicines, making them a public health problem. Objective: To identify and map the type of evidence available and recognize knowledge gaps in safety surveillance of medicines used for orphan or rare diseases that allows the construction of a more structured safety profile. Methodology: Scoping review. Medline Ovid, Scopus, Web of Science databases were consulted. We consult databases of regulatory agencies such as FDA, EMA, ICH and thesis repositories of three universities of Colombia Selection criteria: Articles, studies and information from regulatory and technical databases that address drug safety surveillance for orphan diseases were included. Information collection: Extracted information was classified into categories. Results: 9 articles were included. Narrative reviews (n=7), retrospective cohort study (n=1) and descriptive studies (n=1). The main country to publish was Italy (n=4) and the United Kingdom (n=3). Guidelines from the FDA, EMA and ICH were used. Discussion: This review has limitations given the limited specific information available that reflects the pending challenges of the regulatory agencies and sectors involved. Conclusion Fragmented information was found that can help build a more structured profile. However, a more demanding application of good pharmacovigilance practices is necessary.
