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

描述（由申请人提供）：了解各种治疗方法在终身复杂慢性疾病中的最佳使用，对于当前最大限度地提高健康水平和降低成本的医疗保健使命至关重要。这个K 08奖项的建议概述了培训和研究计划，以促进我发展成为一个独立的卫生服务研究员，专注于复杂的慢性疾病的药物经济学。我的主要职业目标是应用卫生经济学和决策科学，为各种药物疗法的最佳使用提供卫生政策。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IBD最有效和最昂贵的治疗选择。我的项目旨在回答生物疗法如何影响IBD的健康和经济，以及如何更经济有效地使用生物制剂。这个K 08奖将帮助我完成以下培训和职业目标：（1）获得必要的技能，在卫生经济学和决策科学，以评估复杂的慢性疾病的药物经济学，（2）成为一个独立的卫生服务研究员调查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辅导。本提案中的研究目标是由以下问题激发的：鉴于IBD患者特征的各种治疗选择和差异， 使用生物制剂来维持健康和降低成本的最佳方法是什么？该项目有三个具体目标：目标1：通过确定利用趋势和对患者结局的治疗效果，表征CD和UC生物制剂的药物经济学。目的2：确定生物治疗初治CD和UC患者的成本效益。目标3：确定在CD和UC患者中使用生物制剂时是否采用最佳成本效益治疗策略的卫生政策影响。我将通过使用计量经济学方法的大型数据库分析来解决这些目标，两个比较成本效益分析，比较生物制剂的头对头模拟，以及两个竞争的风险分析，阐述卫生政策的影响，如果生物制剂的成本效益使用成为标准的临床实践。

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.