Design, Methods, Biostatistics, and Economic Analysis Core

B。背景和意义 将该核心纳入CEBP的理由是，在自我管理文献的主体中存在许多重要的方法问题，这表明需要应用额外的设计和分析技术，以实现推进这一重要主题的知识基础的目标。在本节中，我们主要关注设计和分析问题，包括与经济分析相关的问题。我们讨论了其他方法论问题，特别是有关的理论框架，文化的适当性，并在自我管理，生物行为和信息学的核心测量工具。 B1.自我管理研究的方法论问题 近年来，发表了一些系统性综述和荟萃分析，重点是自我管理/自我护理干预措施。这些综合报告突出了一些方法问题。Chodosh等人筛选了780项研究，并将53项纳入其随机效应荟萃分析（糖尿病= 26，骨关节炎= 14，高血压= 13）。[1]他们的分析得出结论，自我管理干预导致血红蛋白A1c和血压的统计学和临床意义，但仅对骨关节炎的疼痛和功能具有统计学意义。在CEBP中纳入设计、方法、生物统计学和经济分析核心的基本原理的主要相关性是他们的结论，即研究的质量可变，并且"从现有数据中无法确定对受益最负责任的项目要素，这阻碍了最佳有效或成本效益项目的规范。Warsi等人批判性地分析了71项自我管理教育试验的结果和方法。[2]他们确定了与对照组相关的方法学问题，包括同期对照的便利样本（15%）和在三分之一的研究中未评估可能的中心效应的诊所或医生水平的随机化。只有8项研究使用意向治疗分析进行，71项干预措施中只有2项是由独立于自我管理教育项目开发人员的调查人员进行的。与Chodosh相似，作者也发现了干预描述不充分的问题。这个问题在药物依从性文献中也得到了广泛的认可。[3，4]在2005年的出版物中，Bakken（CEBP PI）描述了一种基于标准化护理术语的方法来解决这个问题。[5]具体而言，作为客户依从性分析和干预交付的一部分， 定制干预（CAP-IT），[6]护士干预师使用家庭医疗保健分类来记录向患者提供的定制依从性干预（护理诊断和干预）的各个方面，并记录相关时间。这一策略使得能够计算"干预剂量"，并提供证据表明提供了量身定制的干预。 B2.自我管理研究中的经济评价 人口趋势、医疗保健技术的爆炸式发展以及不断变化的医疗保健系统使人们关注医疗保健服务的成本和有效性。尽管美国医疗保健总费用的增长速度比过去有所下降，但预计到2016年，全国医疗保健支出将达到国内生产总值的20%（4.1万亿美元）。[9]由于这种支出水平，有必要但不再足以证明干预措施对临床结果的有效性。越来越多的卫生政策决策者要求在新的干预措施或卫生保健提供模式之前进行经济分析 被接受到实践中。虽然有些人可能认为，成本效益分析只适用于高技术、高成本的干预措施，但情况并非如此。最近，与患者自我管理干预措施相关的经济评估的必要性已经变得明显，一些研究人员已经开始解决这个问题。[10 - 12]与自我管理相关的经济分析的第一步是评估卫生服务利用率（例如，[13，14]）。然而，正如对支持自我管理的干预措施的成本效益进行的系统审查所指出的那样，这一领域的经济分析的总体质量很差。[15]这些研究人员回顾了39份已发表的经济评估报告，分析了自我管理研究， 质量检查表改编自完善的经济方法准则。他们发现了一系列问题，这些问题使得对自我管理干预措施的经济分析比对更传统的干预措施的经济分析更具挑战性："常规"护理）、安慰剂和霍桑效应、对照组污染的更高风险以及难以确定社会对成本的看法。调查人员得出结论，尽管大多数研究声称干预措施具有成本效益或节省成本，但缺乏严格的经济方法，这限制了这些分析影响政策的能力。 在一篇关于慢性病儿童心理教育干预的综述中， 特别指出，缺乏关于成本效益的证据，这是文献中的一个知识差距。[八]《中国日报》

B. Background and Significance The rationale for inclusion of this Core in CEBP is the number of significant methodological issues in the body of self-management literature suggesting the need for application of additional designs and analytic techniques toward the goal of advancing the knowledge base on this important topic. In this section, we focus primarily on design and analytic issues including those related to economic analyses. We discuss other methodological issues specifically related to theoretical frameworks, cultural-appropriateness, and measurement instruments in the Self-Management, Biobehavioral, and Informatics Core. B1. Methodological Issues Related to Self-Management Research In recent years, a number of systematic reviews and meta-analyses focused on self-management/self-care interventions have been published. These syntheses highlight a number of methodological issues. Chodosh et al. screened 780 studies and included 53 in their random-effects meta-analysis (diabetes=26, osteoarthritis=14, hypertension=13). [1] Their analysis concluded that self-management interventions led to statistical and clinical significance for hemoglobinA1c and blood pressure, but only statistical significance for pain and function in osteoarthritis. Of primary relevance to the rationale for inclusion of the Design, Methods, Biostatistics, and Economic Analysis Core in CEBP, are their conclusions that studies had variable quality and that "the elements of programs most responsible for benefits cannot be determined from existing data, and this inhibits specification of optimally effective or cost-effective programs." (p.427) Warsi et al. critically analyzed the outcomes and methods for 71 trials of self-management education. [2] They identified methodological issues related to comparison groups including convenience samples of concurrent controls (15%) and randomization at the level of the clinic or physician without assessment of a possible center effect in one third of the studies. Only 8 studies were conducted using intent-to-treat analyses and only two of the 71 interventions were conducted by investigators independent of the self-management education program developer. Similar to Chodosh, the authors also identified the issue of inadequate description of the intervention. This issue has been widely acknowledged in medication adherence literature as well. [3, 4] In a 2005 publication, Bakken (CEBP PI) described an approach based upon standardized nursing terminology to address the issue. [5] Specifically, as part of the delivery of the Client Adherence Profiling and Intervention Tailoring Intervention (CAP-IT), [6] the nurse interventionist used the Home Health Care Classification to document the aspects of the tailored adherence intervention (nursing diagnoses and interventions) that were delivered to the patient and also documented the associated time. This strategy enabled calculation of an "intervention dose" and provided evidence that a tailored intervention was delivered. B2. Economic Evaluations in Self Management Research Demographic trends, the explosion in health care technology, and a changing health care system have focused attention on both the cost and the effectiveness of health care delivery. Although total U.S. health care costs are rising at a decreased rate than in the past, national health care spending is projected to reach 20% of the gross domestic product by 2016 ($4.1 trillion). [9] Because of this level of spending, it is necessary, but no longer sufficient, to demonstrate the efficacy of an intervention on clinical outcomes. Increasingly, health policy decision-makers request economic analyses before new interventions or models of health care delivery are accepted into practice. While some may suggest that cost-effectiveness analyses are only applicable for high technology high cost interventions, this is not the case. More recently, the need for economic evaluations related to patient self-management interventions has become apparent and some researchers have begun to address this topic. [10-12] A first step in economic analysis related to self-management is assessing health services utilization (e.g., [13, 14]). However, as noted in a systematic review of cost-effectiveness of interventions to support self-management, the overall quality of economic analyses in this field is poor. [15] These investigators reviewed 39 published economic evaluations analyzing self-management studies using a quality checklist adapted from well-developed guidelines for economic methods. They identified a set of issues that make economic analyses of self-management interventions more challenging than those of more conventional interventions: ill-defined comparator (e.g., "usual" care), placebo and Hawthorne effects, higher risk of control group contamination, and difficulty of ascertaining a societal perspective on costs. The investigators concluded that although the majority of studies claimed that interventions were cost-effective or cost-saving, rigorous economic methods were lacking, which limits the ability of these analyses to affect policy. In a review focused on psycho-educational interventions for children with chronic disease, Barlow and Ellard specifically identified the lack of evidence regarding cost-effectiveness as a knowledge gap in the body of literature. [8]