Development of Clinical Trials Outcome Instruments for Acne Vulgaris

寻常痤疮临床试验结果仪器的开发

• 批准号: 8883116
• 负责人: DIANE M. THIBOUTOT
• 金额: $ 20.01万
• 依托单位: PENNSYLVANIA STATE UNIV HERSHEY MED CTR
• 依托单位国家: 美国
• 财政年份: 2013
• 资助国家: 美国
• 起止时间: 2013-07-15 至 2017-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8883116
• 关键词: Academia; Accounting; Acne; Acne Vulgaris; Adolescent; Adopted; Affect; Age; Agreement; Area; Boxing; Canada; Caring; Characteristics; Cicatrix; Clinical Trials; Collaborations; Communication; Computer software; Consensus; Data; Data Analyses; Data Collection; Data Set; Development; Disease; Drug Combinations; Drug Prescriptions; Eczema; Evaluation; Exercise; Expenditure; Face; Feeling; Focus Groups; Generations; Genetic Markers; Goals; Health Expenditures; Health Professional; Healthcare; Impairment; Individual; Industry; Inflammatory; International; Lead; Lesion; Maps; Measurement; Measures; Modeling; Morbidity - disease rate; Names; Office Visits; Online Systems; Outcome; Outcome Measure; Outcomes Research; Pathogenesis; Patient Outcomes Assessments; Patient Representative; Patients; Pattern; Performance; Pharmaceutical Preparations; Photography; Population; Private Practice; Process; Publications; Publishing; Quality of life; Quality-of-Life Assessment; Recording of previous events; Regulatory Affairs; Research; Research Infrastructure; Research Personnel; Resources; Rheumatology; Sample Size; Series; Serum; Severities; Silicones; Skin; Social Psychology; Standardization; Suicide attempt; Techniques; Time; TimeLine; Training; United States; Validation; Visual; Work; base; comparative effectiveness; comparative efficacy; computerized; effectiveness research; evidence base; health care delivery; improved; instrument; meetings; multidisciplinary; new technology; novel; open label; public health relevance; self esteem; sex; systematic review; tool

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.

描述（由申请人提供）：痤疮是最常见的皮肤病，影响约 85% 的美国青少年人口，每年造成数十亿美元的医疗保健支出。在这个经济变革和医疗保健实践模式变化的时代，从业者越来越需要就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，但可用于将一种治疗方法与另一种治疗方法进行比较的数据却很少。进行大型临床试验来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临床试验数据的二次分析 套。由于痤疮临床试验中缺乏允许跨研究比较的标准化结果测量，该领域的进展受到极大阻碍。 遵循风湿病学 OMERACT 小组的模式，这是一个国际多利益相关者专家小组，负责痤疮评估、不同皮肤光型痤疮患者的护理、社会心理学、痤疮试验数据分析、监管事务、患者报告结果的测量和患者代表已经聚集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顾问委员会，为 ACORN 计划提供指导。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，开发一个用于痤疮临床试验的经过验证的结果测量“工具箱”。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两个主要结果。此外，缺乏患者报告的躯干结果测量 痤疮影响约一半的痤疮患者。短期目标包括： 1)．开发基于网络的痤疮病变计数标准化仪器（SILCA），以改善与痤疮病变计数相关的变异性； 2）。开发评估躯干痤疮对患者生活质量影响的综合工具（CompAQ）；和3）。生成一系列 4 种新技术的试点数据，以评估痤疮和/或培训痤疮评估人员。实现我们的长期目标的进展将包括就结果领域和结果测量的期望特征达成共识，完成对痤疮严重程度及其影响的痤疮结果测量的系统审查，评估选定的全球评估量表的可靠性和可行性以及初步开发新的或修订的全球严重程度等级。 将采用基于团队的策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。团队位于美国（Thiboutot 博士/Chren 博士）；英国 (Drs.Layton/Eady) 和加拿大 (Drs.Tan/Wolfe)。全球沟通策略将补充面对面会议，并将采用项目管理的六西格码原则。

项目成果

Identifying What to Measure in Acne Clinical Trials: First Steps towards Development of a Core Outcome Set.

确定痤疮临床试验中要测量的内容：制定核心结果集的第一步。

• 发表时间: 2017
• 作者: Layton, Alison M;Eady, E Anne;Thiboutot, Diane M;Tan, Jerry;Acne Core Outcomes Research Network (ACORN) Outcomes Identification Group
• 通讯作者: Acne Core Outcomes Research Network (ACORN) Outcomes Identification Group

Assessing effectiveness in acne clinical trials: steps towards a core outcome measure set.

评估痤疮临床试验的有效性：制定核心结果衡量标准的步骤。

• 发表时间: 2019
• 作者: Thiboutot, D M;Layton, A M;Chren, M;Eady, E A;Tan, J
• 通讯作者: Tan, J

Identifying the Impacts of Acne and the Use of Questionnaires to Detect These Impacts: A Systematic Literature Review.

识别痤疮的影响以及使用调查问卷来检测这些影响：系统文献综述。

• 发表时间: 2021-03
• 影响因子: 7.3
• 作者: Smith, Hayley;Layton, Alison M;Thiboutot, Diane;Smith, Abbey;Whitehouse, Heather;Ghumra, Waseem;Verma, Meenakshi;Tan, Jerry;Jones, Georgina;Gilliland, Kathryn;Patel, Megha;Otchere, Elaine;Eady, Anne
• 通讯作者: Eady, Anne