背景:监管机构已经确定了小儿哮喘,临床医生,临床试验赞助商,和护理人员作为一个需要新的适合目的的临床结果评估(COA)的领域,根据美国食品和药物管理局(FDA)的监管指南,用于评估治疗试验中的临床获益。为了解决这个差距,患者报告结果(PRO)协会的小儿哮喘工作组继续开发2个COA,以评估小儿哮喘临床试验中的哮喘体征和症状,以支持疗效终点:PRO测量,8-11岁儿童的小儿哮喘日记(PAD-C)和观察者报告的结果指标,儿科哮喘日记-观察者(PAD-O)为4-11儿童的照顾者。这项定性研究旨在产生有关PAD-C和PAD-O的内容有效性的证据。
方法:对不同的美国参与者(15名8-11岁儿童和30名4-11岁儿童看护人)进行了半结构化概念启发和认知访谈。所有儿童均有临床医生诊断为轻度至重度哮喘。访谈探讨了小儿哮喘的经验,并评估了两种措施的理解和相关性。在3个迭代轮次中进行了访谈以允许修改。
结果:概念启发结果表明,在PAD-C中评估的核心体征/症状和影响概念(咳嗽,呼吸困难,上气不接下气,喘息,胸闷,和夜间觉醒/症状)和PAD-O(咳嗽,呼吸困难,呼吸短促,喘息,和夜间觉醒/体征)对应于参与者最常报告的那些;实现了概念饱和。所有PAD-C和PAD-O指令和核心项目都很好地理解并被大多数参与者认为是相关的。参与者的反馈,小儿哮喘工作组,顾问团,FDA支持对这些措施进行修改,包括在两项措施中增加1个新项目,并移除1个护理者项目。
结论:研究结果为两项措施的内容有效性提供了强有力的支持。这两种措施的横截面测量属性及其在电子格式中的用户体验和可行性将在未来的定量试点研究中通过定性退出访谈进行评估,旨在支持可靠性,构造效度,最终内容,and,最终,FDA认证的措施。
小儿哮喘是儿童最常见的慢性疾病之一。然而,存在诊断不足的问题,疾病管理不善,许多小儿哮喘患者的治疗不足,给全世界的医疗系统施压。评估哮喘症状是儿童哮喘治疗发展的重要组成部分。然而,很少有符合监管指导的临床结局评估(COA)来直接评估哮喘患儿的症状严重程度和评估新疗法的获益.在这项研究中,我们继续开发小儿哮喘日记-儿童(PAD-C)和小儿哮喘日记-观察者(PAD-O),根据监管指导,评估4至11岁儿童的哮喘体征和症状,并解决这个未满足的需求。该研究旨在探索小儿哮喘的经验,并评估其理解程度和相关措施。对15名8至11岁儿童和30名4至11岁哮喘儿童的看护人进行了三轮定性访谈。结果表明,这两种措施都得到了很好的理解,并评估了儿童和护理人员报告的小儿哮喘的相关和重要方面。研究结果提供了证据支持PAD-C和PAD-O作为症状严重程度的衡量标准及其在小儿哮喘治疗试验中的未来用途。正在进行进一步的研究,以评估其测量属性并评估用户体验和电子完成的可行性,在美国食品和药物管理局正在进行的COA认证过程中,最终支持PAD-C和PAD-O。
BACKGROUND: Pediatric asthma has been identified by regulators, clinicians, clinical trial sponsors, and caregivers as an area in need of novel fit-for-purpose clinical outcome assessments (COAs) developed in accordance with the U.S. Food and Drug Administration\'s (FDA\'s) regulatory guidance for evaluating clinical benefit in treatment trials. To address this gap, the Patient-Reported Outcome (PRO) Consortium\'s Pediatric Asthma Working Group has continued development of 2 COAs to assess asthma signs and symptoms in pediatric asthma clinical trials to support efficacy endpoints: a PRO measure, the Pediatric Asthma Diary-Child (PAD-C) for children 8-11 years old (y.o.) and an observer-reported outcome measure, the Pediatric Asthma Diary-Observer (PAD-O) for caregivers of children 4-11 y.o. This qualitative research aimed to generate evidence regarding the content validity of the PAD-C and PAD-O.
METHODS: Semi-structured combined concept elicitation and cognitive interviews were conducted with a diverse sample of U.S. participants (15 children 8-11 y.o. and 30 caregivers of children 4-11 y.o.). All children had clinician-diagnosed mild to severe asthma. Interviews explored the experience of pediatric asthma and assessed the understanding and relevance of both measures. Interviews were conducted across 3 iterative rounds to allow for modifications.
RESULTS: Concept elicitation findings demonstrated that the core sign/symptom and impact concepts assessed in the PAD-C (cough, hard to breathe, out of breath, wheezing, chest tightness, and nighttime awakenings/symptoms) and PAD-O (cough, difficulty breathing, short of breath, wheezing, and nighttime awakenings/signs) correspond to those most frequently reported by participants; concept saturation was achieved. All PAD-C and PAD-O instructions and core items were well understood and considered relevant by most participants. Feedback from participants, the Pediatric Asthma Working Group, advisory panel, and FDA supported modifications to the measures, including addition of 1 new item to both measures and removal of 1 caregiver item.
CONCLUSIONS: Findings provide strong support for the content validity of both measures. The cross-sectional measurement properties of both measures and their user experience and feasibility in electronic format will be assessed in a future quantitative pilot study with qualitative exit interviews, intended to support the reliability, construct validity, final content, and, ultimately, FDA qualification of the measures.
Pediatric asthma is one of the most common chronic diseases in children. However, there are problems of underdiagnosis, poor disease management, and undertreatment for many pediatric asthma patients, pressuring healthcare systems worldwide. Evaluating asthma symptoms is an important part of the development of treatments for pediatric asthma. However, there are few clinical outcome assessments (COAs) developed in line with regulatory guidance to directly assess symptom severity and evaluate the benefit of new treatments in children with asthma. In this study, we continued the development of the Pediatric Asthma Diary—Child (PAD-C) and the Pediatric Asthma Diary—Observer (PAD-O), according to regulatory guidance, to assess asthma signs and symptoms in children 4 through 11 years old and address this unmet need. The study aimed to explore the experience of pediatric asthma and assess how well-understood and relevant the measures are. Three rounds of qualitative interviews were conducted with 15 children 8 through 11 years old and 30 caregivers of children 4 through 11 years old with asthma. Results show that both measures are well-understood and assess the relevant and important aspects of pediatric asthma reported by children and caregivers. Findings provide evidence supporting the PAD-C and PAD-O as measures of symptom severity and their future use in pediatric asthma treatment trials. Further research is underway to evaluate their measurement properties and assess the user experience and feasibility of electronic completion, to ultimately support the PAD-C and PAD-O in an ongoing COA qualification process by the United States Food and Drug Administration.