Medicamentos recomendados em protocolos clínicos e diretrizes terapêuticas para doenças raras
Data
2024-08-21
Autores
Ramalho, Anna Carolina dos Santos 
Orientadores
Melo, Daniela Oliveira de
Tipo
Trabalho de conclusão de curso
Título da Revista
ISSN da Revista
Título de Volume
Resumo
Introdução: Para abordar a temática de tratamento de Doenças Raras é necessário considerar a especificidade das doenças, a individualidade dos pacientes e o funcionamento do sistema de saúde em sua totalidade. Nesse contexto, uma das respostas a movimentação social e institucional por mais atenção e cuidado integrado para pacientes com essas condições são os Protocolos Clínicos e Diretrizes Terapêuticas (PCDT) para Doenças Raras. Para além da disponibilização dos medicamentos, o cuidado aos pacientes com doenças raras precisa considerar a complexidade do tratamento e as necessidades integrais de cada um, principalmente no que diz respeito ao manejo e acompanhamento de medicamentos, exigindo clareza na descrição do tratamento indicado. Objetivo: Identificar os medicamentos recomendados nos PCDTs de Doenças Raras, bem como a situação regulatória de cada especialidade indicada, a disponibilidade de bula, a presença ou ausência dos princípios ativos recomendados na Lista de Medicamentos Referência da Anvisa bem como determinar se há padronização na nomenclatura, de acordo com as normas exigidas para descrição de Formas Farmacêuticas e Vias de Administração. Métodos: Foi realizada uma busca em dois sites oficiais (Conitec e Ministério da Saúde) para identificar apenas PCDTs com uma lista formal de medicamentos, conforme a Portaria N° 375/2009. Usou-se a plataforma RedCap® para criar um formulário de extração padronizado e coletar dados sobre PCDTs, incluindo a portaria e ano de publicação, fármacos recomendados, posologia, esquema de administração e forma farmacêutica. Após a coleta, verificou-se o status de referência dos medicamentos e informações de bulas em sites da Anvisa para confirmar registros, concentração e forma farmacêutica. Com esses dados, foram identificados os PCDTs voltados para Doenças Raras, conforme informações do Ministério da Saúde. Resultados e Discussão: Representando 42,6% (43) de todos os PCDTs publicados pelo Ministério da Saúde até o período considerado, os PCDTs de Doenças Raras são documentos que preconizam o uso de 104 princípios ativos, descrevendo-os de acordo com as orientações e diretrizes estabelecidas e com poucos erros no que tange a presença de informação de via de administração (28; 26%) e descrição da forma farmacêutica (22; 14%) que impactam no entendimento do corpo multidisciplinar que acompanha esses pacientes. Além disso, entre as 180 especialidades farmacêuticas recomendadas nos PCDTs, 16 (8,8%) apresentam situação regulatória irregular na Anvisa e 82 (45,5%) constam na Lista de Medicamentos Referência da Anvisa. Conclusão: A maioria das especialidades farmacêuticas citadas nos PCDTs de Doenças Raras contém a informação correta de via de administração e Forma Farmacêutica, de acordo com o Vocabulário Controlado elaborado pela Anvisa e isso sugere um esforço contínuo para atualização e publicação de PCDTs, especialmente para doenças raras, que apresentaram representatividade significativa de 42,6% dos PCDTs analisados, indicando atenção considerável a essas condições, considerando a escassez de informações e tratamentos específicos.
Introduction: To address the issue of treating Rare Diseases, it is necessary to consider the specificity of the diseases, the individuality of patients and the functioning of the health system as a whole. In this context, one of the responses to the social and institutional movement for more attention and integrated care for patients with these conditions are the Clinical Protocols and Therapeutic Guidelines (PCDT) for Rare Diseases. In addition to the availability of medicines, care for patients with rare diseases needs to consider the complexity of the treatment and the comprehensive needs of each person, especially with regard to the management and monitoring of medicines, requiring clarity in the description of the recommended treatment. Objective: To identify the medications recommended in the PCDTs for Rare Diseases, as well as the regulatory status of each specialty indicated, the availability of leaflets, the presence or absence of active ingredients recommended in the Anvisa Reference Medicines List, as well as to determine whether there is standardization in the nomenclature, in accordance with the standards required for the description of Pharmaceutical Forms and Routes of Administration. Methods: A search was conducted on two official websites (Conitec and the Ministry of Health) to identify only PCDTs with a formal list of medications, in accordance with Ordinance No. 375/2009. The RedCap® platform was used to create a standardized extraction form and collect data on PCDTs, including the ordinance and year of publication, recommended drugs, dosage, administration regimen, and pharmaceutical form. After collection, the reference status of the medications and information on package inserts were verified on Anvisa websites to confirm registrations, concentration, and pharmaceutical form. With this data, PCDTs aimed at Rare Diseases were identified, according to information from the Ministry of Health. Results and Discussion: Representing 42.6% (43) of all PCDTs published by the Ministry of Health up to the period considered, the Rare Diseases PCDTs are documents that advocate the use of 104 active ingredients, describing them in accordance with established guidelines and guidelines and with few errors regarding the presence of information on the route of administration (28; 26%) and description of the pharmaceutical form (22; 14%) that impact the understanding of the multidisciplinary team that monitors these patients. Furthermore, among the 180 pharmaceutical specialties recommended in the PCDTs, 16 (8.8%) have an irregular regulatory status at Anvisa and 82 (45.5%) are included in Anvisa's List of Reference Medicines. Conclusion: The majority of pharmaceutical specialties mentioned in the PCDTs for Rare Diseases contain the correct information on the route of administration and Pharmaceutical Form, according to the Controlled Vocabulary prepared by Anvisa and this suggests a continuous effort to update and publish PCDTs, especially for diseases rare, which represented a significant 42.6% of the PCDTs analyzed, indicating considerable attention to these conditions, considering the scarcity of information and specific treatments.
Introduction: To address the issue of treating Rare Diseases, it is necessary to consider the specificity of the diseases, the individuality of patients and the functioning of the health system as a whole. In this context, one of the responses to the social and institutional movement for more attention and integrated care for patients with these conditions are the Clinical Protocols and Therapeutic Guidelines (PCDT) for Rare Diseases. In addition to the availability of medicines, care for patients with rare diseases needs to consider the complexity of the treatment and the comprehensive needs of each person, especially with regard to the management and monitoring of medicines, requiring clarity in the description of the recommended treatment. Objective: To identify the medications recommended in the PCDTs for Rare Diseases, as well as the regulatory status of each specialty indicated, the availability of leaflets, the presence or absence of active ingredients recommended in the Anvisa Reference Medicines List, as well as to determine whether there is standardization in the nomenclature, in accordance with the standards required for the description of Pharmaceutical Forms and Routes of Administration. Methods: A search was conducted on two official websites (Conitec and the Ministry of Health) to identify only PCDTs with a formal list of medications, in accordance with Ordinance No. 375/2009. The RedCap® platform was used to create a standardized extraction form and collect data on PCDTs, including the ordinance and year of publication, recommended drugs, dosage, administration regimen, and pharmaceutical form. After collection, the reference status of the medications and information on package inserts were verified on Anvisa websites to confirm registrations, concentration, and pharmaceutical form. With this data, PCDTs aimed at Rare Diseases were identified, according to information from the Ministry of Health. Results and Discussion: Representing 42.6% (43) of all PCDTs published by the Ministry of Health up to the period considered, the Rare Diseases PCDTs are documents that advocate the use of 104 active ingredients, describing them in accordance with established guidelines and guidelines and with few errors regarding the presence of information on the route of administration (28; 26%) and description of the pharmaceutical form (22; 14%) that impact the understanding of the multidisciplinary team that monitors these patients. Furthermore, among the 180 pharmaceutical specialties recommended in the PCDTs, 16 (8.8%) have an irregular regulatory status at Anvisa and 82 (45.5%) are included in Anvisa's List of Reference Medicines. Conclusion: The majority of pharmaceutical specialties mentioned in the PCDTs for Rare Diseases contain the correct information on the route of administration and Pharmaceutical Form, according to the Controlled Vocabulary prepared by Anvisa and this suggests a continuous effort to update and publish PCDTs, especially for diseases rare, which represented a significant 42.6% of the PCDTs analyzed, indicating considerable attention to these conditions, considering the scarcity of information and specific treatments.