Health Policies for Rare Disease Patients: A Scoping Review

Luís Carlos Lopes-Júnior (), Victor Evangelista Faria Ferraz, Regina Aparecida Garcia Lima, Sara Isabel Pimentel Carvalho Schuab, Raphael Manhães Pessanha, Geisa Santos Luz, Mariana Rabello Laignier, Karolini Zuqui Nunes, Andressa Bolsoni Lopes, Jonathan Grassi, Juliana Almeida Moreira, Fabrine Aguilar Jardim, Franciéle Marabotti Costa Leite, Paula de Souza Silva Freitas and Silvia Regina Bertolini
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Luís Carlos Lopes-Júnior: Health Sciences Center, Graduate Program in Public Health, Federal University of Espírito Santo (UFES), Vitoria 29047-105, ES, Brazil
Victor Evangelista Faria Ferraz: Ribeirão Preto Medical School, University of São Paulo (USP), Ribeirão Preto 14049-900, SP, Brazil
Regina Aparecida Garcia Lima: Ribeirão Preto College of Nursing (USP), University of São Paulo, Ribeirão Preto 14040-902, SP, Brazil
Sara Isabel Pimentel Carvalho Schuab: Nursing Department, Federal University of Espírito Santo (UFES), Vitoria 29047-105, ES, Brazil
Raphael Manhães Pessanha: Health Sciences Center, Graduate Program in Public Health, Federal University of Espírito Santo (UFES), Vitoria 29047-105, ES, Brazil
Geisa Santos Luz: Hospital de Clínicas da Faculdade de Medicina de Marília (HCFAMEMA), Marília 17519-080, SP, Brazil
Mariana Rabello Laignier: Nursing Department, Federal University of Espírito Santo (UFES), Vitoria 29047-105, ES, Brazil
Karolini Zuqui Nunes: Department of Integrated Health Education, Graduate Program in Nutrition and Health, Federal University of Espírito Santo (UFES), Vitoria 29043-213, ES, Brazil
Andressa Bolsoni Lopes: Department of Integrated Health Education, Graduate Program in Nutrition and Health, Federal University of Espírito Santo (UFES), Vitoria 29043-213, ES, Brazil
Jonathan Grassi: Health Sciences Center, Graduate Program in Public Health, Federal University of Espírito Santo (UFES), Vitoria 29047-105, ES, Brazil
Juliana Almeida Moreira: Department of Integrated Health Education, Graduate Program in Nutrition and Health, Federal University of Espírito Santo (UFES), Vitoria 29043-213, ES, Brazil
Fabrine Aguilar Jardim: Ribeirão Preto College of Nursing (USP), University of São Paulo, Ribeirão Preto 14040-902, SP, Brazil
Franciéle Marabotti Costa Leite: Health Sciences Center, Graduate Program in Public Health, Federal University of Espírito Santo (UFES), Vitoria 29047-105, ES, Brazil
Paula de Souza Silva Freitas: Nursing Department, Federal University of Espírito Santo (UFES), Vitoria 29047-105, ES, Brazil
Silvia Regina Bertolini: Department of Management and Health Care, Federal University of São Paulo (UNIFESP), São Paulo 04021-001, SP, Brazil

IJERPH, 2022, vol. 19, issue 22, 1-19

Abstract: Objective: To identify and map the available evidence on the implementation of public health policies directed at individuals with rare diseases, and to compare the implementation of these health policies between Brazil and other countries. Method: A scoping review guided by the PRISMA-ScR and JBI checklists. The search for articles was conducted in eight electronic databases, MEDLINE/Pubmed, Embase, Cochrane Library, Web of Science, Scopus, CINAHL, PsycINFO, and LILACS, using controlled descriptors, synonyms, and keywords combined with Boolean operators. All steps of this review were independently conducted by two researchers. The selected studies were classified by evidence hierarchy, and a generic quantitative tool was used for the assessment of the studies. Results: A total of 473 studies were identified, of which 13 which met all the inclusion criteria were selected and analyzed. Of these studies, 61.5% (n = 8) had final scores equal to or greater than 70%, i.e., they were classified by this tool as being well-reported. The comparative analysis of international rare diseases demonstrates that public authorities’ priorities and recommendations regarding this topic also permeate and apply to the Brazilian context. Conclusions: The evaluation and monitoring of public policies directed at rare disease patients are urgent and necessary to improve and implement such policies with less bureaucracy and more determination for this unique population that requires timely and high-quality care.

Keywords: rare diseases; public health; health policy; health management (search for similar items in EconPapers)
JEL-codes: I I1 I3 Q Q5 (search for similar items in EconPapers)
Date: 2022
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