Development of Clinical Trials Outcome Instruments for Acne Vulgaris

描述(申请人提供)：痤疮是最常见的皮肤疾病，影响到美国约85%的青少年人口，每年花费数十亿美元的医疗保健费用。在这个经济变化和卫生保健实践模式变化的时代，从业者越来越需要对痤疮治疗做出基于证据的决定。尽管已经对数百种可用的痤疮药物进行了大量的临床试验，但可以用来比较一种治疗方法和另一种治疗方法的数据很少。用于进行大型临床试验以比较痤疮疗法的资源有限。展望未来，痤疮领域的比较有效性研究更有可能依赖于对临床试验数据的二次分析 布景。由于在痤疮临床试验中缺乏标准化的结果衡量标准，允许跨研究进行比较，这大大阻碍了这一领域的进展。遵循OMERACT风湿病学小组的模式，一个由评估痤疮、护理不同皮肤类型的痤疮患者、社会心理学、痤疮试验数据分析、法规事务、患者报告结果测量和患者代表组成的国际多方利益相关者专家小组已经组建，并命名为痤疮核心结果研究网络(ACORN)。将任命一个由专家组成的科学咨询委员会，为ACORN倡议提供指导。该方法将实现一系列短期目标，作为痤疮评估的资源，同时追求长期目标，开发用于痤疮临床试验的有效结果测量的“工具箱”。目前，痤疮皮损计数和研究人员对痤疮严重程度的全球评估是监管试验中衡量的两个主要结果。此外，缺乏患者报告的躯干预后衡量标准。 粉刺影响了大约一半的粉刺患者。短期目标包括：1)。开发基于网络的痤疮皮损计数标准化仪器(SILCA)，以改善与痤疮皮损计数相关的变异性；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项新技术的试点数据，以评估痤疮和/或培训痤疮评估员。朝着我们的长期目标取得的进展将包括：就结果领域和结果措施的期望特征达成共识，完成对痤疮严重程度及其影响的结果措施的系统审查，评估选定的全球评估量表的可靠性和可行性，以及初步制定新的或修订的全球严重程度量表(S)。以团队为基础的战略将被用于同时追求项目目标。每个团队都指定了项目部分的主要责任。团队的总部设在美国(ThiBoutot/Chren博士)、英国(Layton/Eady博士)和加拿大(Tan/Wolfe博士)。全球沟通战略将补充面对面的会议，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.