Regulation of disease-modifying treatments for Alzheimer’s disease

a comparative study between the United States and Brazil

Abstract

Background: Alzheimer’s disease is one of the greatest public health challenges of the 21st century due to population aging and the increasing prevalence of dementia worldwide. The introduction of disease-modifying therapies, particularly anti-amyloid monoclonal antibodies, has reignited debates on regulatory criteria, cost-effectiveness, and access. Problem: Although recent clinical results show promising advances, uncertainties remain regarding the magnitude of benefits, adverse effects, and, above all, the differences between the regulatory models adopted by the United States and Brazil. Objectives: This study aims to comparatively analyze the roles of the FDA and CMS, on the one hand, and Anvisa, Conitec, and ANS, on the other, highlighting approval timelines and criteria, evidentiary requirements, financial coverage policies, and access challenges. Methods: The research adopts a deductive approach, based on bibliographic review and analysis of national and international legislation, doctrine, and jurisprudence. Results: The analysis showed that the United States relies on accelerated FDA approval pathways, later converted into traditional approval with clinical confirmation, while the CMS conditions coverage on continuous evidence development, ensuring integration between regulation and financing. In Brazil, although Anvisa recently approved donanemab, the incorporation process within SUS and ANS remains under evaluation, characterized by cost-effectiveness concerns, increasing judicialization, and structural limitations in diagnostic infrastructure. Conclusions: It is concluded that the U.S. model ensures greater speed and integration between approval and public coverage, while Brazil shows regulatory progress but still faces barriers to broad and equitable access.