Health technology assessment strategies for incorporating technologies for rare diseases into health systems: scoping review protocol

Health technology assessment for rare diseases is surrounded by challenges related to few incentives for research and development, a small number of patients and scarce therapeutic options. Methodological barriers affect the certainty of evidence on the safety and efficacy of interventions. In addition, the sustainability of healthcare systems can be negatively affected by the high costs of technologies for rare diseases. The decision-making process for the healthcare of rare diseases may also be influenced by the lack of standardized clinical guidelines. Strategies to help overcome these challenges need to be identified to support the decision-making process of technology assessment agencies. This review aims to identify these strategies to help overcome these challenges and thus support the decision-making process of technology agencies in the context of rare diseases.