National policy for rare diseases: challenges on implementation and Health Technology Assessment in the Brazilian Unified Health System (SUS)

Abstract

The recent publication of the “National Policy on Comprehensive Care for People with Rare Disesses and the Guidelines for Comprehensive Care for People with Rare Diseases within the National Health System”, published via Ordinance 199/2014, has been demonstrated for all actors involved in the political process scheduling, design, implementation, and actually, monitoring, an important advance toward a health care system will be increasingly egalitarian and for all Brazilians. However, it is possible to discuss some structural issues relating to the implementation of this policy process, especially regarding the incorporation of technology in the NHS, the impact, especially the budget to be generated in the system and access challenges imposed on patients and their caregivers. Thus, the aim of this paper is to explain and discuss the scenario of rare diseases in Brazil - especially after the publication of the National Policy on this topic - and the world from the perspective of Health Technology Assessment , highlighting the process obstacles given the need to incorporate specific treatments for these diseases in the Brazilian Unified Health System.