BackgroundTo guarantee the right to health, the health system must also ensure access to medicines. Several financial arrangements to provide these technologies are implemented and range from the direct (either total or partial) to indirect payment by the patient, being necessary to evaluate its effect on access to medicines. However, to ensure access to medicines is not just about ensuring its availability, as this only materializes in its use. Thus, evaluation studies of interventions in access to medicines have been using indicators related to the health results and use of health services as its outcomes. Furthermore, as this relationship is not direct, it is important to critically assess the adequacy of these tools to measure this phenomenon and, additionally, the ability to use it in the Brazilian scenario. Therefore, this study sought to identify, describe, and analyze the use of these indicators as medicine access outcomes, through a review of the scientific literature.MethodsAn extensive literature review was done using a bibliographic database for a systematic review. The references were selected based on inclusion and exclusion criteria, and the indicators from the papers retained were analyzed using the parameters of validity, measurability, reliability, and relevance.ResultsWe have analyzed over 12,000 references of which 30 references were included, describing the use of 49 health outcomes and health service use indicators. The majority reported the use of health service utilization measures. In our evaluation, the best indicators for assessing the effects of co-payment intervention on access are the ones aimed at specific populations or symptomatic health conditions in which the response to the therapeutic treatment is known and occurs in a short period of time. It was evident the lack of information on the indicators analyzed as well as the limitation of the Brazilian secondary databases for its calculation.ConclusionsThis research showed the variety and heterogeneity of the indicators used in scientific studies. The best indicators for access to medicines are sought to measure the use of health services for symptomatic health conditions that are quickly responsive to pharmacological treatment, while the indicators related to worker productivity loss was the most suitable for health outcomes.Electronic supplementary materialThe online version of this article (10.1186/s40985-019-0115-1) contains supplementary material, which is available to authorized users.