新生儿筛查现代化是一项泛欧挑战--国际德尔菲研究

IF 3.6 3区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Health Policy Pub Date : 2024-09-13 DOI:10.1016/j.healthpol.2024.105162
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引用次数: 0

摘要

新生儿筛查是一项公共卫生措施,可在新生儿出生时诊断出罕见疾病,从而将后期治疗的负面影响降至最低。基因组技术有望以低成本前所未有地扩大筛查疾病的范围,并为新生儿筛查计划带来变革潜力。然而,人们对基因组新生儿筛查的公共资助障碍知之甚少,特别是考虑到欧洲新生儿筛查的异质性。因此,本研究旨在了解国际新生儿筛查专家是否对基因组新生儿筛查的资助障碍有共同的理解。为此,我们召集了 21 位不同专业和国家背景的欧洲新生儿筛查专家进行德尔菲研究。通过 Wilcoxon 匹配对符号秩检验,我们在连续三轮调查中就所有提出的障碍达成了稳定的共识。专家们普遍认为,7 年内为所有新生儿提供基因组新生儿筛查的可能性不大,可治疗性、缺乏咨询和熟练的劳动力是公共资金的最大障碍。我们认为,重新定义罕见病治疗的价值、集中基因组专业技术和国际研究联盟是在德尔菲小组达成的共识基础上采取泛欧行动的途径。
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The modernisation of newborn screening as a pan-European challenge – An international delphi study

Newborn screening is a public health measure to diagnose rare diseases at birth, thereby minimising negative effects of late treatment. Genomic technologies promise an unprecedented expansion of screened diseases at low cost and with transformative potential for newborn screening programmes. However, barriers to the public funding of genomic newborn screening are poorly understood, particularly in light of the heterogenous European newborn screening landscape. This study therefore aims to understand whether international newborn screening experts share a common understanding of the barriers to fund genomic newborn screening. For this purpose, we convened 21 European newborn screening experts across a range of professions and national backgrounds in a Delphi study. Stable consensus, determined via the Wilcoxon matched-pairs signed-ranks test, was found via three consecutive survey rounds for all presented barriers. Experts generally judged the scenario of genomic newborn screening being available to every newborn in seven years to be unlikely, identifying treatability and the absence of counselling and a skilled workforce as the most significant barriers to public funding. We identify value re-definition for rare disease treatments, centralisation of genomic expertise, and international research consortia as avenues for pan-European actions which build on the consensus achieved by our Delphi panel.

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来源期刊
Health Policy
Health Policy 医学-卫生保健
CiteScore
6.40
自引率
6.10%
发文量
157
审稿时长
3-8 weeks
期刊介绍: Health Policy is intended to be a vehicle for the exploration and discussion of health policy and health system issues and is aimed in particular at enhancing communication between health policy and system researchers, legislators, decision-makers and professionals concerned with developing, implementing, and analysing health policy, health systems and health care reforms, primarily in high-income countries outside the U.S.A.
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