Article type
Abstract
"Background: Rare Diseases (RD) affect up to 65 people out of 100,000 individuals. In Brazil, approximately 13 million people have RD (6% of Brazilian population). Clinical practice guidelines (CPG) are especially important to guide diagnoses and treatment of RD patients. National Committee for Health Technology Incorporation (Conitec) advises Brazilian Ministry of Health in CPG development.
Objective: To present rare diseases CPG published by Brazilian Ministry of Health and developed in Brazil public health context, in the last twelve years.
Methods: Descriptive qualitative study about rare diseases CPG development and update by Conitec in Brazilian Unified Health System context. Data collection and analyses used Excel 2010 software. Variables considered were update or new CPG and year of publication (2012 to 2023).
Result: Brazilian Ministry of Health has 63 rare diseases CPG published, such as Cystic Fibrosis, Miastenia Gravis, Spinal Muscular Atrophy and Amyotrophic Lateral Sclerosis. In 2012-2023 period, 89 rare diseases CPG were published: 31 (31,9%) new CPG, 72 (74,2%) updates and 6 (6,2%) revoked. The annual average for rare diseases developing and updating guidelines was 8.1 (standard deviation ± 3.5) guidelines.
The historical series of CPG revealed that the largest number of CPG (n=13) were published in 2018 and the smallest number (n=1) in 2023. The new guidelines were mostly published in 2019. The new CPG were published in 2016 and 2017. CPG revocations occurred in 2018 (n=1), 2021 (n=1) and 2022 (n=4).
Conclusions: Brazilian Ministry of Health has been investing in production and updating CPG with a view to qualifying the health care provided to people with rare diseases throughout the country. Developing and updating of CPG, based on the best scientific evidence available, contributes to to better health outcomes in these population.
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Objective: To present rare diseases CPG published by Brazilian Ministry of Health and developed in Brazil public health context, in the last twelve years.
Methods: Descriptive qualitative study about rare diseases CPG development and update by Conitec in Brazilian Unified Health System context. Data collection and analyses used Excel 2010 software. Variables considered were update or new CPG and year of publication (2012 to 2023).
Result: Brazilian Ministry of Health has 63 rare diseases CPG published, such as Cystic Fibrosis, Miastenia Gravis, Spinal Muscular Atrophy and Amyotrophic Lateral Sclerosis. In 2012-2023 period, 89 rare diseases CPG were published: 31 (31,9%) new CPG, 72 (74,2%) updates and 6 (6,2%) revoked. The annual average for rare diseases developing and updating guidelines was 8.1 (standard deviation ± 3.5) guidelines.
The historical series of CPG revealed that the largest number of CPG (n=13) were published in 2018 and the smallest number (n=1) in 2023. The new guidelines were mostly published in 2019. The new CPG were published in 2016 and 2017. CPG revocations occurred in 2018 (n=1), 2021 (n=1) and 2022 (n=4).
Conclusions: Brazilian Ministry of Health has been investing in production and updating CPG with a view to qualifying the health care provided to people with rare diseases throughout the country. Developing and updating of CPG, based on the best scientific evidence available, contributes to to better health outcomes in these population.
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