在指南制定者中进行的一项定性研究揭示了罕见病指南制定方面的挑战和策略
Mirthe J Klein Haneveld1, Willemijn F E Irvine2, Martina C Cornel3
1Amsterdam UMC, University of Amsterdam, Emma Children's Hospital, Amsterdam Reproduction & Development, Amsterdam Public Health, Meibergdreef 9, PO Box 22660, Amsterdam 1100 DD, The Netherlands; European Reference Network on Rare Congenital Malformations and Rare Intellectual Disability ERN-ITHACA, Clinical Genetics Department, Robert Debré University Hospital, 48 Boulevard Serurier, Paris 75935, France.
为罕见疾病制定临床实践指南面临着独特的挑战. 这项研究确定了开发人员使用的关键方法问题和策略,以改善未来的罕见病指南创建.
科学领域:
- 医学研究 医学研究
- 临床实践指南 临床实践指南
- 罕见疾病 罕见疾病
背景情况:
- 对罕见疾病的临床实践指南 (CPG) 开发受到有限的证据,小型专家团队和资源限制的阻碍.
- 现有的罕见病CPG表现出不同的方法质量,需要改进.
研究的目的:
- 探索罕见病指南制定者的经验.
- 确定罕见病CPG开发中的方法论挑战和成功策略.
- 为开发罕见病CPG的方法指南提供信息.
主要方法:
- 在全球范围内与15个指导方针开发者进行半结构化采访.
- 包括来自不同医学领域的专家,具有罕见疾病CPG开发经验.
- 分析数据使用结合的演和归纳方法,基于GIN-McMaster指南开发清单.
主要成果:
- 小专家组面临着利益冲突,有限的专业知识和代表性挑战.
- 开发人员使用实用方法,将稀缺的直接证据与间接证据,注册表数据和机械推理相结合.
- 评价GRADE方法的透明度,但认为并不总是可行的;更广泛的问题,如文件类型的选择也被考虑.
结论:
- 获得了关于罕见病CPG开发的关键方法问题的见解.
- 强调需要加强罕见病指南的指导和方法开发.
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