Evaluating Harms in the Assessment of Net Benefit: A Framework for Newborn Screening Condition Review.
Evaluating Harms in the Assessment of Net Benefit: A Framework for Newborn Screening Condition Review.
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DOI：
10.1007/s10995-015-1869-9
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发表时间：
2016-03
影响因子：
2.3
通讯作者：
Green NS
中科院分区：
文献类型：
--
作者：
Goldenberg AJ;Comeau AM;Grosse SD;Tanksley S;Prosser LA;Ojodu J;Botkin JR;Kemper AR;Green NS
卫生与公众服务部（HHS）新生儿和儿童遗传性疾病咨询委员会（“咨询委员会”）向卫生与公众服务部部长提出关于在全国新生儿推荐统一筛查小组中增加新条件的建议。咨询委员会的决策过程包括评估提名病症筛查的净效益，由独立的病症审查工作组提供系统的证据审查。关于特定疾病筛查相关危害的证据基础往往比益处的证据基础更有限。确定新生儿筛查潜在危害的过程审查了其他公共卫生循证审查过程的框架，这些框架经过咨询委员会的投入和批准，适应了系统审查、新生儿筛查规划和生物伦理方面的专家进行的新生儿筛查。为了支持咨询委员会对提名病症的审查，工作组制定了一种标准化的方法来评估危害和证据中的相关差距。伤害类型包括对婴儿的身体负担；筛查或诊断评估给家庭带来的社会心理和后勤负担；比有临床表现的儿童更早确诊的婴儿接受医疗的风险增加；因假阴性结果而延误诊断；假阳性结果造成的社会心理伤害；临床诊断、发病年龄或临床谱不确定；以及在获得诊断或治疗方面的差异。估计处于危险中的儿童人数、危害程度、时间和可能性将纳入工作组提交咨询委员会的报告。
The Department of Health and Human Services (HHS) Advisory Committee on Heritable Disorders in Newborns and Children (“Advisory Committee”) makes recommendations to the HHS Secretary regarding addition of new conditions to the national Recommended Uniform Screening Panel for newborns. The Advisory Committee’s decision-making process includes assessing the net benefit of screening for nominated conditions, informed by systematic evidence reviews generated by an independent Condition Review Workgroup. The evidence base regarding harms associated with screening for specific conditions is often more limited than that for benefits. The process for defining potential harms from newborn screening reviewed the frameworks from other public health evidence-based review processes, adapted to newborn screening by experts in systematic review, newborn screening programs and bioethics, with input from and approval by the Advisory Committee. To support the Advisory Committee’s review of nominated conditions, the Workgroup has developed a standardized approach to evaluation of harms and relevant gaps in the evidence. Types of harms include the physical burden to infants; psychosocial and logistic burdens to families from screening or diagnostic evaluation; increased risk of medical treatment for infants diagnosed earlier than children with clinical presentation; delayed diagnosis from false negative results; psychosocial harm from false positive results; uncertainty of clinical diagnosis, age of onset or clinical spectrum; and disparities in access to diagnosis or therapy. Estimating the numbers of children at risk, the magnitude, timing and likelihood of harms will be integrated into Workgroup reports to the Advisory Committee.