OBJECTIVES

This study approaches aspects of Brazilian public policies for high cost drugs for rare genetic diseases. Rare genetic diseases were taken as an example because of their rarity and the international trend for the development of new drugs for their treatment, all at high costs. During the last years, new drugs for these diseases were introduced to the Brazilian public health system (SUS) by the Ministry of Health. The inclusion process demands that a specific commission (CONITEC) that uses HTA tools, among other principles, perform analysis.

The objective of the study was to define which criteria and HTA tools were applied in the decision-process on the health technology assessment process for high-cost drugs for rare genetic diseases and compare the Brazilian policies in the area with European Countries policies.

METHODS

All the processes carried out by CONITEC since its foundation in 2011 were evaluated those with medicines for rare genetic diseases selected, the outcomes of these processes (included in SUS or not included) and HTA tools applied.

RESULTS

Cost-benefit analysis and cost-effectiveness were the most frequent analysis applied. The indirect costs were not applied in the analysis and epidemiology was not central for the ‘inclusion or not’ decision. There were important differences between the evaluation processes in Brazil and in European countries.

CONCLUSIONS

Despite the heterogeneity difficulties in establishing a health policy for each rare disease, it is important to create rational models to deal with this challenge. Definition of criteria and thresholds could be beneficial to the process.