Health and Economic Outcomes for Newborn Screening

新生儿筛查的健康和经济成果

• 批准号: 8337630
• 负责人: Lisa A. Prosser
• 金额: $ 48.72万
• 依托单位: UNIVERSITY OF MICHIGAN AT ANN ARBOR
• 依托单位国家: 美国
• 财政年份: 2011
• 资助国家: 美国
• 起止时间: 2011-09-30 至 2016-09-29
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8337630
• 关键词: Health; Economic; Outcomes; Newborn; Screening

DESCRIPTION (provided by applicant): Recent enhancements in screening technology have allowed for newborn screening for many additional disorders with little increase in testing cost. At the same time, there is little information on long-term outcomes for conditions that can be detected by newborn screening. The Secretary's Advisory Committee on Heritable Disorders in Newborns and Children was chartered by the Health Resources and Services Administration to evaluate new conditions nominated for inclusion on the recommended uniform newborn screening panel using a systematic review process. Several conditions have been nominated but not recommended for uniform screening, primarily due to a lack of evidence. This research project will build on the evidence reviews conducted on behalf of the Advisory Committee for Heritable Disorders in Newborns and Children. Due to technical and ethical challenges that preclude the use of traditional clinical trials in this area, this research project will use computer modeling to simulate clinical trials of newborn screening programs compared to no screening (clinical identification). By combining information from systematic reviews, published and unpublished data, and expert opinion, we will project long-term health and economic outcomes for conditions that can be detected by newborn screening. This research study will also conduct a survey of public values for newborn screening programs. The project team will include investigators from a wide range of disciplines who have substantial expertise in evidence synthesis and policy analysis of newborn screening programs as well as clinical expertise. Research questions for the study will be guided by an Advisory Committee whose members represent the many relevant viewpoints when considering the expansion of newborn screening programs. This proposed research project focuses on the evidence gap identified by the Secretary's Advisory Committee on Heritable Disorders in Newborns and Children and focuses on children, an identified AHRQ priority population. Results on health and economic outcomes paired with public preference data will provide the necessary information needed for policy decisions regarding the selection of conditions for newborn screening panels.

描述（由申请人提供）：筛查技术的最新改进允许新生儿筛查许多其他疾病，而测试成本几乎没有增加。与此同时，关于新生儿筛查可以检测到的疾病的长期结果的信息很少。新生儿和儿童遗传性疾病秘书咨询委员会由卫生资源和服务管理局特许，使用系统性审查程序评估提名列入推荐的统一新生儿筛查小组的新条件。有几个条件已被提名，但不建议统一筛选，主要是由于缺乏证据。该研究项目将建立在代表新生儿和儿童遗传性疾病咨询委员会进行的证据审查的基础上。由于技术和伦理方面的挑战，排除了在这一领域使用传统的临床试验，该研究项目将使用计算机建模来模拟新生儿筛查计划的临床试验相比，没有筛选（临床鉴定）。通过结合来自系统评价、已发表和未发表的数据以及专家意见的信息，我们将预测新生儿筛查可检测到的疾病的长期健康和经济结果。这项研究还将对新生儿筛查计划的公众价值进行调查。该项目团队将包括来自广泛学科的研究人员，他们在新生儿筛查计划的证据合成和政策分析以及临床专业知识方面具有丰富的专业知识。该研究的研究问题将由一个咨询委员会指导，该委员会的成员在考虑扩大新生儿筛查计划时代表了许多相关观点。这个拟议的研究项目的重点是由秘书的咨询委员会在新生儿和儿童遗传性疾病确定的证据差距，并侧重于儿童，确定AHRQ的优先人群。健康和经济结果的结果与公众偏好数据相结合，将为新生儿筛查小组选择条件的政策决定提供必要的信息。