在巴西制定国家罕见病政策
Têmis Maria Félix1, Bibiana Mello de Oliveira2, Dafne Dain Gandelman Horovitz3
1Serviço de Genética Médica, Hospital de Clinicas de Porto Alegre, Rua Ramiro Barcelos 2350, Porto Alegre, RS, 90035-903, Brazil. tfelix@hcpa.edu.br.
Journal of community genetics
|September 26, 2024
概括
巴西 巴西 巴西 巴西.
科学领域:
- 公共卫生 公共卫生
- 罕见疾病 罕见疾病
- 卫生政策 卫生政策
背景情况:
- 罕见疾病 (RD) 影响着大量人口,需要针对性的医疗保健策略.
- 巴西对RD的定义影响了每10万人口中最多65人.
- 患者倡导在推动研发与开发政策制定方面至关重要.
研究的目的:
- 描述巴西关于为患有罕见疾病的人提供综合护理的国家政策 (2014) 的发展情况.
- 分析在巴西公共卫生系统内实施研发与开发护理的进展和挑战.
- 突出患者组织在倡导研发与开发政策方面的作用.
主要方法:
- 审查巴西为综合性罕见疾病护理制定政策的过程.
- 对专门的研发和开发中心的建立和认证进行分析.
- 检查基因测试和新技术在研发和开发管理中的整合.
主要成果:
- 巴西于2014年制定了国家罕见病政策,旨在减少发病率和死亡率.
- 33个专门的研发和开发中心已经获得认证,但它们在全国的分布不均.
- 遗传测试的可用性有限,ICD-10的使用妨碍了对RD的流行病学理解.
结论:
- 巴西的研发和开发政策,在十年的时间里发展了大量的患者倡导,导致了专门的中心和技术整合.
- 在公平获得基因测试,全国范围内的服务可用性和全面的流行病学数据收集方面仍然存在挑战.
- 解决高成本和确保孤儿药的可用性对于改善巴西罕见病患者的护理至关重要.
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