优先考虑与罕见疾病利益相关者相关的研究:系统审查和专题分析
Soho Yoon1, Minjee Lee2, Hoi-In Jung3
1Asian Institute for Bioethics and Health Law, Yonsei University, Seodaemun-gu, Seoul, Republic of Korea.
Orphanet journal of rare diseases
|November 24, 2023
概括
关于罕见疾病 (RD) 利益相关者的研究突出了未得到满足的需求和挑战. 提高公众意识和支持对于增加投资和治疗罕见疾病的发展至关重要.
科学领域:
- 医学研究 医学研究
- 公共卫生政策 公共卫生政策
- 利益相关者参与 利益相关者参与
背景情况:
- 由于证据有限,罕见疾病 (RD) 研究政策的制定具有挑战性.
- 有效的研发和开发研究需要广泛的利益相关者参与,以提高认识和获得政治支持.
- 确定研究趋势和利益相关者主题对于政策制定至关重要.
研究的目的:
- 系统地审查和分析有关研发和开发调查的现有文献.
- 确定参与罕见病研究的利益相关者.
- 为研发和发展研究提出研究重点和政策倡议.
主要方法:
- 115项研究的系统审查和专题分析.
- 从五个电子数据库 (PubMed,EMBASE,Cochrane Central,科学网,CINHAL) 中提取的数据.
主要成果:
- 患者/护理人员 (67.0%) 和卫生专业人员 (15.7%) 是主要参与者.
- 研究重点是一般的 RDs (40.0%) 和内分泌,营养和代谢疾病 (17.4%).
- 大多数研究 (64.3%) 检查了 RD 经验,主要使用调查 (99.1%) 并在高收入国家 (80.0%) 进行.
结论:
- 利益相关方的研究揭示了在管理罕见疾病方面存在重大未满足的需求和挑战.
- 公共意识和支持对于政治可行性和增加对 RDs 的投资至关重要.
- 为了治疗罕见疾病,需要加强国家投资和产品开发.
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