Comparative Effectiveness of Biologics for 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自身免疫性风湿病和皮肤病中生物制剂的比较有效性（CE）申请标题：生物制剂对银屑病的比较有效性银屑病是一种慢性炎症性Th-1，Th-17介导的疾病，影响超过700万美国人。银屑病，特别是当严重时，与身体和情绪健康功能的严重残疾相关，最近已被证明是心血管疾病的独立危险因素，并与过度的全因死亡率相关。银屑病的治疗最近经历了一场革命，在过去的7年中，FDA批准了至少5种生物疗法，对驱动银屑病的遗传学和免疫/炎症途径的理解取得了进展，以及针对全新途径的研究性生物制剂的开发。虽然这些新疗法在短期研究中已被证明对银屑病有效，但它们与过高的成本，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。银屑病研究中的这些巨大进步并没有反映在指导个体患者治疗决策的研究中。因此，为了做出信息性的治疗决策，提高中重度银屑病患者的护理质量，迫切需要在这一领域进行比较有效性研究。为了响应挑战赠款05-AR-101，我们建议建立一个高度创新的公私合作伙伴关系，称为CERSDN（发音为“Sirs-DEN”），这是皮肤病网络比较有效性研究的首字母缩写，以严格研究生物制剂对银屑病的比较有效性。根据《美国复苏和再投资法案》，CERDN将在生物医学研究领域创造至少3个新的就业机会，刺激费城、圣路易斯和湖城三个不同的地方经济。我们将从来自美国各地的学术实践，私人执业和国家银屑病基金会的患者成员中收集大量不同的银屑病患者人群的数据。这一目标将解决在银屑病中进行比较有效性研究的主要障碍，因为此类研究只能通过多学科协作组进行，而美国不存在皮肤病学领域的此类基础设施。通过CERSDN，我们将对皮肤科医生和患者进行调查，以确定在为患者护理提供信息所必需的比较有效性研究中应优先考虑的关键因素，并将确定银屑病患者是否愿意参与未来的纵向研究，以评估中重度银屑病治疗的比较有效性。这一目标将为规划未来的纵向比较有效性研究提供必要的数据，这些研究采用金标准设计，如大型简单试验。最后，我们将在大量不同的患者人群中进行一系列横断面研究，以评估现有治疗中重度银屑病的有效性的阶段流行率，这些治疗包括光疗、阿维A、甲氨蝶呤、环孢素、阿法西普、依那西普、阿达木单抗、英夫利昔单抗和乌司奴单抗。这一目标将确定这些疗法目前在常规临床实践中治疗的各个患者亚组中对患者（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（例如受银屑病影响的体表面积）的表现如何在给定的时间点。最终，通过挑战赠款05-AR-101的前所未有的机会，我们将进行研究，创造就业机会，刺激经济，并解决优化银屑病患者护理所需的基本知识差距。这项建议将提供必要的信息，为银屑病患者的治疗决策和未来的纵向比较有效性研究。最终，这一系列的研究将导致更多合理的治疗决策，并改善超过700万患有银屑病的美国人的患者护理。

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.