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Early Check: A Collaborative Innovation to Facilitate Pre-Symptomatic Clinical Trials in Newborns

早期检查：促进新生儿症状前临床试验的协作创新

基本信息

批准号：
9975249
负责人：
Donald B Bailey
金额：
$ 155.7万
依托单位：
RESEARCH TRIANGLE INSTITUTE
依托单位国家：
美国
项目类别：
财政年份：
2016
资助国家：
美国
起止时间：
2016-09-15 至 2022-06-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/9975249
关键词：
Advocate Affect Aftercare Authorization documentation Award Birth Child Child Support Clinical Services Clinical Trials Collection Consent Counseling Data Decision Making Disease Early Intervention Ensure Evaluation Family Foundations Fragile X Syndrome Funding Future General Population Goals Health Health Communication Health Services Research Industry Infant Informatics Infrastructure Intellectual functioning disability International Laboratories Learning Life Modeling Motor Neuron Disease Neonatal Screening Newborn Infant North Carolina Outcome Policies Policy Maker Population Positioning Attribute Process Public Health Public Health Informatics Rare Diseases Registries Research Research Personnel Science Secure Source Spinal Muscular Atrophy Symptoms Testing Time Translational Research Universities Work base caregiving design effective therapy experience experimental study family support field study flexibility follow-up forest improved innovation medical schools patient advocacy group programs psychosocial public-private partnership rare condition recruit screening screening panel screening policy screening program success symptom treatment translational medicine

项目摘要

PROJECT SUMMARY/ABSTRACT Newborn screening (NBS) is designed for pre-symptomatic identification of serious conditions for which there are effective treatments that must begin early. Central to NBS policy is evidence that pre-symptomatic treatment is more effective than treatment after symptoms appear. Unfortunately, such evidence is difficult to amass because most nominated conditions are rare and the effort required to identify pre-symptomatic infants for clinical trials is substantial. Researchers and advocates find themselves in a classic “Catch 22” situation—NBS cannot happen without sufficient evidence, but gathering this evidence necessarily requires large-scale population screening. This problem is such a formidable barrier to translational research that many disorders will never have the evidence needed to justify inclusion in NBS programs. We propose to develop and implement Early Check—a research program in which voluntary screening for a panel of conditions is offered on a statewide basis. Early Check would allow rapid screening for new candidate conditions, advance understanding of early disease, and facilitate registry and clinical trial recruitment. We will build and implement an experimental research program with an ongoing evaluation component in which we revise and improve the program as we learn from our implementation experiences and engagement with the general public and families directly affected by screening. Once we have finalized all aspects of the program, we will offer screening for a gradually expanding set of conditions to all 120,000 birthing families per year in North Carolina. Our first condition offered for screening will be spinal muscular atrophy, a life-threatening degenerative motor neuron disorder. We will determine participation rates; conduct screening; return results; provide counseling and clinical services; support families in caregiving decisions; inform families of ongoing clinical trials; provide support for families in deciding whether they want to participate in a clinical trial; and follow children and families over time to study benefits, harms, and psychosocial outcomes of screening. We will seek external funds to expand Early Check to other candidate disorders, such as fragile X syndrome. Implementation data will be used to refine the process, inform replication, and establish an infrastructure for testing other candidate conditions. To achieve long-term viability, we will develop a model of public-private partnerships based on collaborative engagement with federal agencies, foundations, patient advocacy groups, and industry.

项目总结/文摘