Implementation of risk sharing models in access to treatment of rare diseases: an integrative review

The objective of this integrative review was to identify the implementation of risk-sharing models in access to treatment for rare diseases in Brazil and the world. The chosen databases were: BVS - Biblioteca Virtual em Saúde, Embase, PubMed, and Scopus. After exclusions, 18 studies were included. The high cost of treatment and uncertainty regarding clinical evidence were observed as the primary barriers to access. Risk-sharing models are generally divided into financially-based models and performance-based models. It was noted that the implementation of these mechanisms widens access to technology that would probably not be available otherwise and highlights significant insights, such as the lack of transparency and standardization in implementation, resulting in inequity of access to orphan drugs. It is concluded that the implementation of these models minimizes financial and performance barriers, potentially expanding access to treatment for rare diseases. Transparency and the exchange of experiences between countries are crucial for the creation of policies and guidelines that make the process more effective in expanding access.