Comparative Effectiveness of Biologics for Psoriasis

生物制剂治疗牛皮癣的疗效比较

• 批准号: 7815007
• 负责人: JOEL M GELFAND
• 金额: $ 50万
• 依托单位: UNIVERSITY OF PENNSYLVANIA
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-09-24 至 2011-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7815007
• 关键词: Address; Adverse effects; Affect; Algorithms; American; Area; Autoimmune Process; Biological Response Modifier Therapy; Biomedical Research; Body Surface Area; Cardiovascular Diseases; Chronic; Cities; Cross-Sectional Studies; Cyclosporine; Cyclosporins; Data; Data Sources; Databases; Dermatologic; Dermatologist; Dermatology; Development; Disease; Effectiveness; Elements; Emotional; Epidemiology; Etanercept; Evaluation; Foundations; Future; Genetic; Gold; Grant; Health; Immune; Individual; Inflammatory; Knowledge; Lead; Longitudinal Studies; Measures; Mediating; Methotrexate; Occupations; Outcome; Pathway interactions; Patient Care; Patient Outcomes Assessments; Patients; Philadelphia; Phototherapy; Physicians; Population Heterogeneity; Prevalence; Principal Investigator; Private Practice; Psoriasis; Quality of Care; Recovery; Reporting; Research; Research Infrastructure; Research Personnel; Risk; Risk Factors; Series; Sodium Chloride; Structure; Subgroup; Surveys; Time; Time Study; United States; acronyms; adalimumab; alefacept; base; clinical practice; clinically relevant; comparative effectiveness; cost; design; disability; economic impact; effectiveness research; health related quality of life; improved; infliximab; innovation; member; mortality; novel; patient population; programs; public-private partnership; response; satisfaction; skin disorder; willingness

DESCRIPTION (provided by applicant): Gelfand, Joel M Research Area: (05) Comparative Effectiveness Research, 05-AR-101 Comparative Effectiveness (CE) of Biologics in Autoimmune Rheumatic and Skin Diseases Title of application: Comparative Effectiveness of Biologics for Psoriasis Psoriasis is a chronic, inflammatory Th-1, Th-17 mediated disease that affects over 7 million Americans. Psoriasis, particularly when severe, is associated with serious disability in physical and emotional health function, has been recently demonstrated to be an independent risk factor for cardiovascular disease, and is associated with excess all-cause mortality. The treatment of psoriasis has recently undergone a revolution, with the FDA approval of at least 5 biologic therapies in the last 7 years, advances in understanding of the genetics and the immune/inflammatory pathways that drive psoriasis, as well as the development of investigational biologics that target completely novel pathways. Although these new therapies have been proven efficacious for psoriasis in short term studies, they are associated with excessive costs, risks of serious side effects that are still being defined, and diminished efficacy with long term treatment. These large strides in psoriasis research have not been mirrored in research that would guide treatment decisions for individual patients. Thus, to make informative treatment decisions and improve the quality of care of moderate to severe psoriasis patients, it is urgent that comparative effectiveness studies be conducted in this area. In response to Challenge grant 05-AR-101, we propose the creation of a highly innovative public-private partnership called CERSDN (pronounced "Sirs-DEN"), the acronym for the Comparative Effectiveness Research in Skin Disease Network, to rigorously study the comparative effectiveness of biologics for psoriasis. Consistent with the American Recovery and Reinvestment Act, CERSDN will create a minimum of 3 new jobs in biomedical research stimulating three different local economies in Philadelphia, St. Louis, and Salt Lake City. We will capture data from a large and diverse population of psoriasis patients from academic practice, private practice, and patient members of the National Psoriasis Foundation from across the United States. This aim will address a major barrier to the conduct of comparative effectiveness studies in psoriasis as such studies can only be conducted through multi-disciplinary collaborative groups, and such an infrastructure in the dermatology field does not exist in United States. Through CERSDN we will conduct surveys of dermatologists and patients in order to determine the key elements that should be prioritized in comparative effectiveness research necessary to inform patient care and will determine the willingness of psoriasis patients to participate in future longitudinal studies evaluating the comparative effectiveness of therapies for moderate to severe psoriasis. This aim will provide essential data for planning future, longitudinal, comparative effectiveness studies using gold standard designs such as large simple trials. Finally, we will perform a series of cross-sectional studies in a large and diverse patient population to evaluate the period prevalence of comparative effectiveness of existing therapies for moderate to severe psoriasis such as phototherapy, acitretin, methotrexate, cyclosporine, alefacept, etanercept, adalimumab, infliximab, and ustekinumab. This aim will determine how these therapies currently perform with respect to patient (e.g. health related quality of life, economic impact, and treatment satisfaction) and physician reported outcomes (such as body surface area affected by psoriasis) in various subgroups of patients treated in routine clinical practice at a given point in time. Ultimately, through the unprecedented opportunity of Challenge grant 05-AR-101 we will conduct research that will create jobs, stimulate the economy, and address essential knowledge gaps required to optimize care of patients with psoriasis. This proposal will provide information essential to informing treatment decisions for patients with psoriasis and future, longitudinal comparative effectiveness studies. Ultimately, this line of research will lead to more rationale treatment decisions and improved patient care for the > 7 million Americans who suffer from psoriasis.

描述（由申请人提供）： Gelfand，Joel M研究领域：（05）比较有效性研究，05-AR-101生物制剂的比较有效性（CE）在自身免疫性风湿性和皮肤疾病中的应用标题：生物学对牛皮癣的比较有效性，对牛皮癣的比较有效性是一种长期炎症性，炎症性TH-17，TH-17，TH-1，TH-1，TH-17介导的疾病，影响了700亿美国人。牛皮癣，尤其是在严重的情况下与身体和情绪健康功能严重的残疾有关，最近已被证明是心血管疾病的独立危险因素，并且与过多的全因死亡率有关。牛皮癣的治疗最近发生了一场革命，在过去的7年中，FDA批准了至少5种生物疗法，了解遗传学和驱动牛皮癣的免疫/炎症途径的进步以及对完全新颖途径的研究生物学的发展。尽管这些新疗法在短期研究中已被证明对牛皮癣有效，但它们与成本过高，仍在定义的严重副作用的风险以及长期治疗的疗效降低有关。牛皮癣研究中的这些大步很大，在研究中尚未反映出可以指导个别患者的治疗决策。因此，为了做出信息性的治疗决策并提高中度至重度牛皮癣患者的护理质量，迫切需要在该领域进行比较有效性研究。为了回应挑战101年5月5日，我们提出了一种称为CERSDN的高度创新性的公私合作伙伴关系（发音为“ Sirs-den”），这是皮肤病网络中比较有效性研究的首字母缩写，以严格研究生物学对牛皮癣的比较有效性。与《美国恢复和再投资法》一致，CERSDN将在生物医学研究中至少创造3个新工作，从而刺激费城，圣路易斯和盐湖城的三个不同地方经济体。我们将从美国各地的国家牛皮癣基金会的学术实践，私人执业和患者的大量牛皮癣患者中获取数据。该目标将解决牛皮癣进行比较有效性研究的主要障碍，因为这样的研究只能通过多学科的协作群体进行，并且在美国不存在皮肤病学领域的基础设施。通过CERSDN，我们将对皮肤科医生和患者进行调查，以确定应在对患者护理的比较有效性研究中优先确定的关键要素，并确定牛皮癣患者参与评估对中度至严重牛皮癣治疗疗法的比较有效性的未来纵向研究的意愿。该目标将为计划未来，纵向，比较有效性研究提供基本数据，使用黄金标准设计，例如大型简单试验。最后，我们将对大型且多样化的患者人群进行一系列横断面研究，以评估现有疗法对中度至重度牛皮癣的比较疗法的患病率，例如光疗，丙替型，丙酰蛋白，甲基甲氨蝶呤，环孢菌素，alefacept，Aletacept，aleefacept，etanercept，etanercept，adalimumab，adalimumab，fillimimumab，filliximab，usiximab，ustekinab，ustekinab。该目标将决定这些疗法目前在患者（例如与健康相关的生活质量，经济影响和治疗满意度）和医生报告的结果（例如受牛皮癣影响的身体表面积）的医生中如何进行的，在给定时间点在常规临床实践中接受治疗的患者中的结果。最终，通过挑战01年5月5日的前所未有的机会，我们将进行研究，以创造就业机会，刺激经济，并解决优化牛皮癣患者护理所需的基本知识差距。该提案将为牛皮癣和未来的纵向比较有效性研究提供为治疗决策提供信息至关重要的信息。最终，这项研究将导致更多的理由治疗决策，并改善患有牛皮癣的700万美国人的患者护理。