在沙特阿拉伯制定国家罕见病定义:健康生态系统多部门研讨会成果
Ghada Mohammed Abozaid1,2, Hussain Abdulrahman Al-Omar3, Abdulaziz Alrabiah4
1Centre for Public Health, Institute of Clinical Sciences B, Royal Victoria Hospital, Dentistry and Biomedical Sciences, Queen's University Belfast School of Medicine, Belfast, United Kingdom.
Frontiers in pharmacology
|August 1, 2025
概括
沙特利益相关者赞成对罕见疾病的综合定义,结合定性和定量标准. 这旨在改善沙特阿拉伯的政策,资源配置和患者获得治疗的机会.
科学领域:
- 医学和健康科学医学和健康科学
- 公共卫生政策 公共卫生政策
- 罕见疾病研究 罕见疾病研究
背景情况:
- 罕见疾病在全球范围内在诊断,治疗和政策制定方面存在重大挑战.
- 沙特阿拉伯缺乏官方罕见病定义,阻碍了有效的政策制定和资源分配.
- 清晰的定义对于改善孤儿药物可访问性和患者护理至关重要.
研究的目的:
- 提出沙特阿拉伯罕见疾病的官方定义.
- 通过多方利益相关者的投入,就罕见疾病定义的定性和定量标准达成共识.
- 为沙特卫生保健政策提供信息,并与2030年愿景目标保持一致.
主要方法:
- 一场多方利益相关者研讨会与来自沙特阿拉伯各行各业的59名参与者举行.
- 通过Vevox®平台进行了交互式投票和调查.
- 数据分析包括描述性统计数据和定性反的主题/内容分析.
主要成果:
- 强烈的共识 (96%) 出现了一个综合定义,结合了定性和定量标准.
- 首选的关键定性术语包括"严重"和"危及生命",其中"遗传"病因非常受青 (91%).
- 流行率 (82%) 是最受欢迎的定量标准,尽管人口门产生了不同的意见.
结论:
- 该研究为沙特背景下罕见疾病提供了基础性的,基于证据的定义.
- 拟议的定义整合了以患者为中心和以资源为导向的标准,以加强公平的获取和护理.
- 建议进一步参与利益相关者,以成功整合政策并与沙特2030年愿景保持一致.
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