Clinical Outcome Assessments for Acute Pain Therapeutics in Infants and young Children (COA APTIC)

摘要 确定、开发和监管批准治疗婴幼儿急性疼痛的疗法的一个主要障碍是缺乏用于临床试验的标准化、高质量的措施和终点。为了满足这一需求，齐默尔曼博士和里夫博士将利用儿科试验网络和杜克健康测量中心的现有基础设施。这一合作伙伴关系利用了儿科药物开发、儿科临床试验的设计和成功执行、儿童止痛剂安全性和有效性评估以及高质量临床结果评估(COA)设计和评估方面的强大专业知识。齐默尔曼博士和里夫博士将带领一支具有独特资质的研究团队，确定高质量的COA和终点，用于婴幼儿急性疼痛疗法的开发，主要关注这0-2岁。具体地说，研究团队将实现以下目标：1)进行形成性研究，以确定对患者、照顾者和临床医生最重要的急性止痛药试验的重要结果；2)确定每个结果的现有COA，并确定每个COA适合急性疼痛试验的用途或对新COA的需求；3)在一项针对婴幼儿的前瞻性试验中，收集临床医生、观察者和患者报告的COA的心理测量学特性的进一步证据。在多元化和全面的外部技术咨询委员会的投入下，完成这些目标将有助于促进以患者为中心的急性止痛药试验，并减轻儿科药物开发的投资负担。

ABSTRACT A major barrier to the identification, development, and regulatory approval of therapeutics for treatment of acute pain in infants and young children is the lack of standardized, high-quality measures and endpoints for use in clinical trials. To address this need, Drs. Zimmerman and Reeve will leverage existing infrastructure of the Pediatric Trials Network and the Duke Center for Health Measurement. This partnership capitalizes on strong expertise in pediatric drug development, design and successful execution of pediatric clinical trials, evaluation of analgesic safety and efficacy in children, and design and evaluation of high-quality clinical outcome assessments (COAs). Drs. Zimmerman and Reeve will lead a team of uniquely-qualified investigators in the identification of high quality COAs and endpoints for use in the development of acute pain therapeutics in infants and young children, with a primary focus on those 0-2 years. Specifically, the research team will accomplish the following goals: 1) conduct formative research to identify important outcomes for acute pain drug trials that matter most to patients, caregivers, and clinicians; 2) identify existing COAs for each outcome and determine each COA’s fit-for-purpose for acute pain trials or the need for new COAs; and 3) gather further evidence of the psychometric properties of clinician-, observer-, and patient-reported COAs in a prospective trial among infants and young children. With input from a diverse and well-rounded external technical advisory committee, completion of these goals will help facilitate patient-centered acute pain drug trials and ease the burden of investment in pediatric drug development.