Health Technology Assessment (HTA) strategies for drugs in rare diseases: a rapid scope review

Abstract

The Health Technology Assessment HTA considers evidence regarding clinical benefits epidemiological profile innovation costeffectiveness ethics and equity in its assessment process to support managers decisions In the context of drugs in rare diseases the work of the ATS is challenging given the low availability of evidence and the high cost of technologies The objective of the review was to analyze the available strategies for evaluating the demands for incorporating drugs for the treatment of rare diseases in health systems A rapid review was performed with a structured search in the MEDLINE database via PubMed the Cochrane Library and Health Systems Evidence Studies on strategies for evaluating drugs used to treat rare diseases were included and additionally searches were carried out in ATS Agencies in Brazil Australia New Zealand Canada United Kingdom France United States and Germany The synthesis of the results was qualitative grouping the major ones into thematic axes Safety and effectiveness Costeffectiveness Budgetary impact and Societys perspective 267 publications were identified 16 selected from indexed databases and 7 from gray literature With the analysis of the documents it can be concluded that the adoption of specific criteria harmonized with the current ATS model is a possible path to be followed in the context of drugs for rare diseases At the same time approaches to encourage research and the creation of specific committees to deal with the issue in HTA agencies would complement actions towards the consolidation of this work