Pharmaco-economics and Optimal Strategies of Biologics Use in Inflammatory Bowel

描述(由申请人提供)：了解各种疗法在终生复杂慢性疾病中的最佳使用，对于当前最大化健康和最大限度降低成本的医疗保健使命至关重要。这份K08奖项提案概述了一项培训和研究计划，以促进我发展成为一名独立的卫生服务研究员，专注于复杂慢性病的药物经济学。我的主要职业目标是应用健康经济学和决策学为卫生政策提供各种药物疗法的最佳使用。在接下来的五年里，我将专注于炎症性肠病(IBD)(克罗恩病[CD]和溃疡性结肠炎[UC])生物疗法的药物经济学。生物制剂是目前治疗IBD最有效和最昂贵的选择。我的项目试图回答生物疗法如何影响IBD的健康和经济，以及如何更具成本效益地使用生物制品。这一K08奖项将帮助我实现以下培训和职业目标：(1)获得必要的卫生经济学和决策科学技能，以评估复杂慢性疾病的药物经济学；(2)成为一名独立的卫生服务研究员，研究临床策略的比较成本效益。为了实现这些目标，我组建了一个由主要导师Jay Bhattacharya博士、共同导师Paul Wise博士、两位顾问Margaret Brandeau博士和Jeremy Goldhaber-Fiebert博士组成的指导团队。培训将包括正式课程和个人指导。这项提案中的研究目标是由以下问题推动的：鉴于IBD患者的治疗方案多种多样，而且不同，什么是 使用生物制品维持健康和降低成本的最佳方式是什么？该项目有三个具体目标：目标1：通过确定生物制剂在CD和UC中的利用趋势和对患者预后的治疗效果，来表征CD和UC中的生物制剂的药物经济学。目的：确定生物治疗CD和UC患者的成本-效果。目的3：确定CD和UC患者在使用生物制剂时，如果采用最优的成本效益治疗策略，对卫生政策的影响。我将通过使用计量经济学方法的大型数据库分析、两个在面对面模拟中比较生物制剂的比较成本效益分析，以及两个相互竞争的风险分析来解决这些目标，如果生物制剂的成本效益使用成为标准的临床实践，这两个分析阐述了卫生政策的含义。 公共卫生相关性：我在获奖期间的K08研究计划致力于发展一种专业知识，以批判性地评估如何使用昂贵的医疗疗法来最大化健康并将患有复杂慢性病的患者的成本降至最低。我将使用炎症性肠病(IBD)作为影响儿童和成人的复杂慢性疾病的模型，通过强加终身残疾和巨大的医疗保健支出。我的研究的第一阶段使用计量经济学技术分析一个大型的国家患者数据库，以确定使用生物制剂对健康和经济的影响，生物制剂是治疗IBD的最新和最昂贵的药物疗法。我研究的第二阶段建立了决策分析模型，以确定相互竞争的生物制剂的成本效益。我的研究的最后阶段使用决策科学来比较健康政策和对社会的好处，如果生物制剂得到最佳使用。这三个项目将代表推进胃肠病学儿科和成人比较成本效益研究的开创性努力，并为其他复杂慢性疾病的类似研究铺平道路。

DESCRIPTION (provided by applicant): Understanding the optimal use of various therapies in life-long complex chronic diseases is vital to the present health care mission of maximizing health and minimizing costs. This K08 award proposal outlines a training and research plan to facilitate my development into an independent health services researcher focusing on the pharmaco-economics of complex chronic diseases. My main career goal is to apply health economics and decision science to inform health policy on the optimal use of various pharmaco-therapies. In the next five years, I will focus on the pharmaco-economics of biologic therapies in inflammatory bowel disease (IBD) (Crohn's disease [CD] and ulcerative colitis [UC]). Biologics are currently the most effective and most expensive therapeutic options in IBD. My project seeks to answer how biologic therapies have impacted the health and economics of IBD, and how biologics can be used more cost-effectively. This K08 award will help me accomplish the following training and career goals: (1) to acquire necessary skills in health economics and decision science to evaluate the pharmaco-economics of complex chronic diseases, (2) to become an independent health services researcher investigating the comparative cost-effectiveness of clinical strategies. To achieve these goals, I have assembled a mentoring team comprised of primary mentor, Dr. Jay Bhattacharya; co-mentor, Dr. Paul Wise; two advisors, Dr. Margaret Brandeau and Dr. Jeremy Goldhaber-Fiebert. Training will include formal courses and individual mentoring. The research goals in this proposal are motivated by the following question: Given the variety of therapeutic options and differences in IBD patient profiles, what is the best way to use biologics to sustain health and decrease costs? The project has three specific aims: Aim 1: To characterize the pharmaco-economics of biologics in CD and UC by determining the utilization trends and treatment effects on patient outcomes. Aim 2: To determine the cost-effectiveness of biologic therapies for biologic-naive CD and UC patients. Aim 3: To determine the health policy implications if optimal cost-effective treatment strategies were adopted in the use of biologics in CD and UC patients. I will address these aims through a large database analysis using econometric methods, two comparative cost- effectiveness analyses that compare biologics in head-to-head simulations, and two competing risk-analyses that elaborate health policy implications if cost-effective use of biologics were to become standard clinical practice. PUBLIC HEALTH RELEVANCE: My K08 research plan during the award term works toward developing an expertise in critically evaluating how expensive medical therapies can be used to maximize health and minimize costs for patients living with complex chronic diseases. I will use inflammatory bowel disease (IBD) as a model for complex chronic disease affecting children and adults by imposing lifelong disability and tremendous health care expenditures. The first phase of my study uses econometric techniques to analyze a large national patient database to determine health and economic impact of using biologic agents, the newest and most expensive medical therapy for the treatment of IBD. The second phase of my study builds decision analytic models to determine the cost-effectiveness of competing biologic agents. The final phase of my study uses decision science to compare health policies and benefits to society if biologic agents were used optimally. These three projects will represent pioneering efforts to advance pediatric and adult comparative cost-effectiveness research in gastroenterology and pave a path for similar investigations in other complex chronic diseases.