Design, Methods, Biostatistics, and Economic Analysis Core

B.背景和意义 将这一核心纳入CEBP的理由是，在自我管理文献正文中提出了许多重要的方法学问题，表明需要应用额外的设计和分析技术，以实现推进关于这一重要主题的知识库的目标。在本节中，我们主要关注设计和分析问题，包括与经济分析有关的问题。我们讨论了与自我管理、生物行为和信息学核心中的理论框架、文化适当性和测量工具特别相关的其他方法论问题。 B1.自我管理研究的方法论问题 近年来，发表了一些关于自我管理/自我护理干预措施的系统综述和荟萃分析。这些综合突出了一些方法论问题。Chodosh等人。筛选了780项研究，其中53项进入随机效应荟萃分析(糖尿病=26项，骨关节炎=14项，高血压=13项)。[1]他们的分析得出结论，自我管理干预导致了血红蛋白A1c和血压的统计和临床意义，但只对骨关节炎的疼痛和功能有统计意义。与将设计、方法、生物统计学和经济分析核心纳入CEBP的理由主要相关的是，他们的结论是，研究的质量各不相同，“对效益负有最大责任的项目要素不能从现有数据中确定，这阻碍了对最佳效果或成本效益项目的规范。”(p.427)Warsi等人。批判性地分析了71次自我管理教育试验的结果和方法。[2]在三分之一的研究中，他们确定了与对照组相关的方法学问题，包括同时对照的方便样本(15%)和在临床或医生水平上的随机化，而不评估可能的中心效应。只有8项研究使用意向治疗分析进行，71项干预措施中只有两项是由独立于自我管理教育项目开发者的调查人员进行的。与Chodosh类似，作者也指出了对干预措施描述不足的问题。这个问题在药物依从性文献中也得到了广泛的承认。[3，4]在2005年的一篇文章中，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]具体地说，作为交付客户遵从性概况和干预的一部分 定制干预(CAP-IT)，[6]护士干预师使用家庭保健分类来记录提供给患者的定制依从性干预的各个方面(护理诊断和干预)，并记录相关的时间。这一策略使“干预剂量”的计算成为可能，并提供了实施量身定制干预的证据。 B2.自我管理研究中的经济评价 人口趋势、卫生保健技术的爆炸性增长以及不断变化的卫生保健系统都将注意力集中在卫生保健提供的成本和有效性上。尽管美国医疗保健总成本的增长速度低于过去，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]由于这一支出水平，证明干预措施对临床结果的有效性是必要的，但不再是足够的。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 在实践中被接受。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，但事实并非如此。最近，与患者自我管理干预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保健服务的利用情况(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整体质量很差。[15]这些研究人员回顾了39项已发表的经济评估，分析了自我管理研究 质量检查表改编自完善的经济方法指南。他们确定了一系列问题，使自我管理干预措施的经济分析比更传统的干预措施更具挑战性：定义不明确的比较对象(例如，“通常”护理)、安慰剂和霍桑效应、控制组污染的较高风险，以及难以确定社会对成本的看法。调查人员的结论是，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。在一篇聚焦于慢性病儿童心理教育干预的综述中，Barlow和Ellard 特别指出，缺乏关于成本效益的证据是文献中的知识差距。[8]

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]