PUBLIC POLICIES AND THE CHALLENGE OF ACCESS TO TREATMENT FOR PATIENTS WITH RARE DISEASES

Abstract

Introduction: Rare diseases are characterized by their low prevalence in the population, but they present significant challenges for patients, families, and the health system. These individuals often face considerable barriers in accessing specialized medical care, including difficulties in accessing appropriate treatments and surgical procedures, when indicated. Objective: to describe the challenges encountered and the implementation of public policies to guarantee access to surgeries for patients with rare diseases. Method: This is a literature review carried out in the electronic databases of Latin American and Caribbean Literature on Health Sciences (LILACS), Medical Literature Analysis and Retrieval System Online (MEDLINE/PubMed) and Scientific Electronic Library Online (SciELO). The inclusion criteria were complete articles, with text available online, published between 2014 and 2024. The search for studies was carried out between the months of May and July 2024. Results: Regarding the main results, it was found that patients with rare diseases face considerable difficulties in accessing treatments, especially those of a surgical nature. One of the main obstacles is the shortage of specialized professionals, which, combined with the high cost of treatments, makes access difficult. Conclusion: The complexity of access to treatment and surgery for patients with rare diseases is a significant problem, exacerbated by the absence of clear clinical protocols and the high cost of treatments. Public policies aimed at rare diseases are fundamental and need to be expanded to ensure improvements in access to treatment.