Enabling self-reported outcomes for youth with developmental disabilities: The Pediatric Evaluation of Disability Inventory - Patient Reported Outcome (PEDI-PRO) - Phase II

缺乏适合于患有神经发育障碍（DD）和相关认知障碍（包括唐氏综合征（DS））的青少年的患者报告结果测量（PROM），这对医疗保健研究和实践构成了一个重大问题。父母和其他专业人士没有确定与青少年相同的需求，并且未能让患有DD的青少年参与医疗保健评估会影响医疗保健质量和结果。鉴于DD的患病率和该人群的显著医疗保健成本，该人群对PROM的市场需求很高，其中遗传病因的最大比例是DS。在第一阶段，我们证明了我们的技术可以帮助患有DD和相关认知障碍的青少年完成PROM。PEDI-PRO原型表现出很强的内部可靠性，并从56名14-22岁的DD和认知障碍青年中获得了一致的分数。事实上，第一阶段的奖励证明了调查在智商范围为47-75的年轻人中的易用性（其中14%的样本有DS），以及在智商范围为30-110的人中的重测信度和内部效度（其中12.7%有DS）。全国的临床医生（n =14）确定了PEDI-PRO原型的可用性。本II期STTR将构建一个临床上可靠的PEDI-PRO评估软件，以解决DD（包括DS）青少年PROM的市场缺口。具体目标：1）将第一阶段PEDI-PRO概念原型转换为一个强大的，功能齐全的评估软件系统，用于康复。2)测试和优化PEDI-PRO试题库的心理测量特性，以确保有效和可靠的测量。3)设计一个临床上可解释的分数报告，准确地描述青年的功能表现。 II期研究设计遵循基于测量仪器选择标准（COSMIN）框架，以确定PEDI-PRO的有效性和可靠性。我们将继续与青年小组（n = 5）和咨询委员会（n = 8）建立合作伙伴关系。我们将设计PEDI-PRO临床管理系统（PCMS：符合HIPPA的临床医生管理仪表板），并将45个附加项目（每个领域15个，由青年小组和咨询委员会选择）纳入PEDI-PRO。其次，300名DD青年将完成PEDI-PRO，其中包括50名DS患者，因此超过了DS患者占智力残疾患者15-20%的患病率估计。我们将遵循COSMIN标准，并使用经典测试理论（CTT）和项目反应理论（IRT）来检查结构效度和内部信度。我们还将使用假设检验来建立已知组和收敛效度。第三，我们将自动评分纳入PEDI-PRO评估软件中，我们将使用混合方法设计和评估两个评分报告原型。第二阶段将使AbleLink Technologies能够在9个月的上市时间轴内积极追求商业化，并为各种平台分发PEDI-PRO评估软件。

The lack of patient reported outcome measures (PROMs) appropriate for youth with neurodevelopmental disabilities (DD) and related cognitive impairments including Down syndrome (DS) poses a significant problem for healthcare research and practice. Parents and other professionals do not identify the same needs as youth, and failure to engage youth with DD in healthcare evaluation compromises healthcare quality and outcomes. The market demand for a PROM for this population is high given the prevalence of DD and the significant healthcare costs for this population, of which the largest proportion with a genetic etiology is DS. In Phase I, we demonstrated that our technology can help youth with DD and related cognitive impairments complete PROMs. The PEDI-PRO prototype demonstrated strong internal reliability and elicited consistent scores from 56 youth ages 14-22 with DD and cognitive impairments. In fact, the Phase I award demonstrated ease-of-use of the survey in youth with an IQ range of 47-75 (of which 14% of the sample had DS), and the test-retest reliability and internal validity in those with an IQ range of 30-110 (of which 12.7% had DS). Clinicians across the country (n =14) established the usability of the PEDI-PRO prototype. This Phase II STTR will build a clinically robust PEDI-PRO Assessment Software to address the market gap in PROMs for youth with DD including DS. Specific aims: 1) Transform the Phase I PEDI-PRO concept prototype into a robust, fully functioning assessment software system for use in rehabilitation. 2) Test and optimize the psychometric properties of the PEDI-PRO item bank to ensure valid and reliable measurement. 3) Design a clinically interpretable score report that accurately characterizes youth’s functional performance. The Phase II study design adheres to the Consensus-based Standards for the Selection of Measurement Instruments (COSMIN) framework to establish the validity and reliability of the PEDI-PRO. We will continue our partnership with a Youth Panel (n = 5) and Advisory Board (n = 8). We will design the PEDI-PRO Clinical Management System (PCMS: a HIPPA compliant clinician administrative dashboard) and incorporate 45 additional items (15 per domain, selected by the Youth Panel and Advisory Board) into the PEDI-PRO. Second, 300 youth with DD will complete the PEDI-PRO, including 50 individuals with DS, thus exceeding the prevalence estimates that individuals with DS make up 15-20% of those with an intellectual disability. We will adhere to COSMIN standards and examine the structural validity and internal reliability using both classical test theory (CTT) and item response theory (IRT). We will also use hypothesis testing to establish known groups and convergent validity. Third, we will incorporate automatic scoring into the PEDI-PRO Assessment Software; we will use a mixed methods to design and evaluate two score report prototypes. This Phase II will enable AbleLink Technologies to aggressively pursue commercialization through a 9 month to market timeline and distribute the PEDI-PRO Assessment Software for a variety of platforms.