Sandra Gillner , Gulcin Gumus , Edith Gross , Georgi Iskrov , Ralitsa Raycheva , Georgi Stefanov , Rumen Stefanov , Anne-Sophie Chalandon , Alicia Granados , Julian Nam , Andreas Clemens , Carl Rudolf Blankart
{"title":"新生儿筛查现代化是一项泛欧挑战--国际德尔菲研究","authors":"Sandra Gillner , Gulcin Gumus , Edith Gross , Georgi Iskrov , Ralitsa Raycheva , Georgi Stefanov , Rumen Stefanov , Anne-Sophie Chalandon , Alicia Granados , Julian Nam , Andreas Clemens , Carl Rudolf Blankart","doi":"10.1016/j.healthpol.2024.105162","DOIUrl":null,"url":null,"abstract":"<div><p>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.</p></div>","PeriodicalId":55067,"journal":{"name":"Health Policy","volume":"149 ","pages":"Article 105162"},"PeriodicalIF":3.6000,"publicationDate":"2024-09-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0168851024001726/pdfft?md5=611a821cac48f8e22762cd2f82d05cd1&pid=1-s2.0-S0168851024001726-main.pdf","citationCount":"0","resultStr":"{\"title\":\"The modernisation of newborn screening as a pan-European challenge – An international delphi study\",\"authors\":\"Sandra Gillner , Gulcin Gumus , Edith Gross , Georgi Iskrov , Ralitsa Raycheva , Georgi Stefanov , Rumen Stefanov , Anne-Sophie Chalandon , Alicia Granados , Julian Nam , Andreas Clemens , Carl Rudolf Blankart\",\"doi\":\"10.1016/j.healthpol.2024.105162\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><p>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.</p></div>\",\"PeriodicalId\":55067,\"journal\":{\"name\":\"Health Policy\",\"volume\":\"149 \",\"pages\":\"Article 105162\"},\"PeriodicalIF\":3.6000,\"publicationDate\":\"2024-09-13\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.sciencedirect.com/science/article/pii/S0168851024001726/pdfft?md5=611a821cac48f8e22762cd2f82d05cd1&pid=1-s2.0-S0168851024001726-main.pdf\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Health Policy\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S0168851024001726\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"HEALTH CARE SCIENCES & SERVICES\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Health Policy","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S0168851024001726","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"HEALTH CARE SCIENCES & SERVICES","Score":null,"Total":0}
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.
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.