Avaliação da Política Nacional de Atenção Integral às Pessoas com Doenças Raras para esclerose múltipla no Ceará à luz da judicialização da saúde

Abstract

This dissertation examines how Brazil’s National Policy for Comprehensive Care for People with Rare Diseases (PNAIPDR) has been implemented in Ceará for individuals with Multiple Sclerosis (MS). It starts from the hypothesis that the recurring resort of MS patients to the courts signals that the policy has yet to meet its objectives fully. The study is qualitative, descriptive, framed as an instrumental case study and grounded in systematic documentary analysis. The corpus comprises PNAIPDR’s normative acts and 601 appellate rulings retrieved from the online repositories of the TJCE, TRF-5, STJ and STF using the keywords <esclerose= and<múltipla=; the decisions span 20193 2024. All documents were systematized and submitted to thematic content analysis inspired by Fourth-Generation Evaluation. Findings show that, despite normative advances, everyday implementation faces obstacles such as unclear care pathways, patchy availability of high-cost tests and medicines, service concentration in the state capital, and a shortage of specialized professionals. In this context, litigation becomes a frequent alternative for people with MS. Most rulings concern provision of non- standardized drugs, with the remainder covering continuous-care supplies or access to specialized therapies. The high rate of provisional relief granted underscores the clinical urgency of these cases and shifts unforeseen costs onto the Unified Health System (SUS) budget. Overall, the effectiveness of the PNAIPDR in Ceará remains fragile. While court action is essential to safeguard patients’ rights, it does not address4and may sometimes exacerbate4the structural factors that drive lawsuits. The study urges policymakers and stakeholders to narrow the gap between law and practice, highlighting the need to revise clinical protocols, decentralize reference centers, provide ongoing training for MS care teams, and adopt transparent criteria for incorporating orphan drugs into the SUS.