Development of Clinical Trials Outcome Instruments for Acne Vulgaris

描述（由申请人提供）：痤疮是最常见的皮肤病，影响美国约85%的青少年人口，每年花费数十亿美元的医疗保健费用。在这个时代的变化，在经济和医疗保健实践模式的变化，有一个越来越多的需要从业人员作出循证决策有关痤疮治疗。虽然大量的临床试验已经进行了数百个可用的痤疮药物，有很少的数据，其中一个治疗可以比较到另一个。进行大型临床试验以比较痤疮疗法的资源是有限的。展望未来，痤疮领域的比较有效性研究更有可能依赖于临床试验数据的二次分析 集.在这一领域的进展已大大阻碍了痤疮临床试验，允许跨研究比较缺乏标准化的结果措施。 遵循OMERACT流变学小组的模式，一个国际多利益相关者专家小组，负责评估痤疮、护理具有不同皮肤光型的痤疮患者、社会心理学、分析痤疮试验数据、监管事务、测量患者报告的结果和患者代表，并被命名为痤疮核心结果研究网络（ACORN）。将任命一个科学专家咨询委员会，为非洲地区生态网的各项举措提供指导。该方法将完成一系列的短期目标，将作为痤疮评价的资源，同时追求长期目标，开发一个“工具箱”的验证结果的措施，用于痤疮临床试验。目前，痤疮病变计数和研究者对痤疮严重程度的总体评估是监管试验中测量的2个主要结局。此外，缺乏患者报告的躯干结局指标， 痤疮影响~一半的所有痤疮患者。短期目标包括：（1）。开发基于网络的痤疮病灶计数标准化仪器（SILCA），以改善与痤疮病灶计数相关的变异性; 2）。开发一种综合工具来评估躯干痤疮对患者生活质量的影响（CompAQ）;以及3）。生成一系列4种新技术的试验数据，以评估痤疮和/或培训痤疮评估人员。实现我们长期目标的进展将包括，就结局领域和结局指标的预期特征达成共识，完成痤疮严重程度及其影响的痤疮结局指标的系统评价，评估选定全球评估量表的可靠性和可行性，以及初步开发新的或修订的全球严重程度量表。 将采用以团队为基础的战略来同时实现项目目标。每个团队都指定了项目部分的主要责任。小组设在美国（Thiboutot/Chren博士）、英国（莱顿/Eady博士）和加拿大（Tan/Wolfe博士）。全球沟通战略将补充面对面会议，并将采用项目管理的六西格玛原则。

DESCRIPTION (provided by applicant): Acne is the most common skin condition that affects ~ 85% of the adolescent population in the United States and accounts for expenditure of billions of health care dollars annually. In this era of change in the economy and change in health care practice patterns, there is an increasing need for practitioners to make evidence-based decisions regarding acne treatment. Although large numbers of clinical trials have been conducted for the hundreds of available acne medications, there is very little data available by which one treatment can be compared to another. Resources to conduct large clinical trials to compare acne therapies are limited. Moving forward, it is more likely that comparative effectiveness research in the area of acne will rely upon secondary analysis of clinical trial data sets. Progress in this area has been greatly hampered by the lack of standardized outcomes measures in acne clinical trials that allows comparison across studies. Following the model of the OMERACT group in rheumatology, an international multi-stakeholder group of experts in evaluation of acne, care of acne patients with diverse skin phototypes, social psychology, analysis of data from acne trials, regulatory affairs, measurement of patient reported outcomes and patient representatives have been assembled and named Acne Core Outcomes Research Network (ACORN). A scientific advisory board of experts will be appointed to provide direction to ACORN initiatives. The approach will be to accomplish a series of short-term objectives that will serve as resources in acne evaluation while pursing the long-term goal to develop a "tool box" of validated outcome measures for use in acne clinical trials. At present, acne lesion counts and investigator global assessment of acne severity are the 2 major outcomes measured in regulatory trials. In addition, there is a lack of patient-reported outcomes measures of truncal acne that affects ~ half of all acne patients. Short-term objectives include: 1). Development of a web- based standardized instrument for lesion counting in acne (SILCA) to improve the variability associated with acne lesion counting; 2). Development of a comprehensive instrument to assess the impact of truncal acne on patient's quality of life (CompAQ); and 3). Generation of pilot data on a series of 4 novel technologies to assess acne and/or train acne evaluators. Progress towards our long-term objective will include, reaching consensus on outcome domains and desired features of outcome measures, completion of systematic reviews of acne outcome measures of acne severity and its impact, assessment of reliability and feasibility of select global assessment scales and preliminary development of a new or revised global severity scale(s). A team-based strategy will be used to simultaneously pursue the project aims. Each team has designated primary responsibility for portions of the project. Teams are based in the US (Drs. Thiboutot/Chren); the UK (Drs.Layton/Eady) and Canada (Drs.Tan/Wolfe). Global communication strategies will supplement face-to-face meetings and Six Sigma principles of project management will be employed.