MAIN COMPONENTS OF PUBLIC POLICIES AND FINANCING STRATEGIES ADOPTED BY OECD COUNTRIES TO IMPROVE ACCESS TO DRUGS FOR RARE DISEASES: A SCOPING REVIEW

BACKGROUND: Since the Orphan Drug Act in United States in 1983, several countries have been implementing courses of action to improve access to Drugs for Rare Diseases (DRD). Despite of the growing body of literature available to describe these efforts, until now, there's no systematic research in place to characterize and compare the wide range of OECD countrie activities to accomplish their task. OBJECTIVES: Describe the main components of public policies and financing strategies adopted by OECD countries to improve access to DRD. METHODS: We carried out a scoping review through its five first stages. Search strategy considered different sources, as electronic databases (Medline, Embase, Cochrane, CINAHL, LILACS, ECONLIT, Web of Science), key journals (Orphanet Journal of Rare Diseases, and Value in Health), grey literature from different institutions (we consult ISPOR Health Authority by Country inventory to identify their web sites in each OECD countries), and reference lists. The search algorithm was buid using the work of the ISPOR Rare Diseases Groups of Interest on terms and definitions. Categorization and result analysis were performed using the analytical-descriptive method, considering 3 conceptual frameworks: public policy definition (Subirats, 2008), financing function (Kutzin, 2000), and fourth hurdle systems (Hutton, 2006). RESULTS: 85 articles were included in this review, most of them referred to R+D activities in the form of incentives to preclinical and clinical research. More than 50% of the articles detailed variations in coverage decision making process, specifically quality of evidence needed to prove incremental benefits DRD. The European Union and the United States have the highest number of documents describing their policy. CONCLUSIONS: There is only a few of countries that actually have adopted policies to improve patients access to DRD (United States, Italy and France). Even in this group there's still work to do to influence the whole pharmaceutical system, therefore improve access.