Rigor of Multi-Registry CER Studies: Towards Patient-Centered Treatment Planning

描述（由申请人提供）：这个K18职业提升奖的目的是提供指导的研究经验，职业提升和教育活动，为申请人过渡到比较有效性（CE）和以患者为中心的结果研究（PCOR）在儿科慢性疾病作为当地和国家的方法学专家。申请人是一位成熟的独立统计方法研究者和成功的生物统计学家，具有多年的临床试验经验，并在儿科炎症性肠病（IBD）（包括儿科克罗恩病（CD））方面进行了广泛的合作。她正处于这一转变的风口浪尖，以实现她的职业目标， 通过在人群和个体患者水平上加强CER的定量分析，提高证据信息的质量。这一职业目标源于她在儿科IBD方面的合作，缺乏CE证据导致护理和管理实践存在巨大差异，尽管治疗取得了进展，但在过去四十年中观察到的结果几乎没有改善。与许多儿科慢性疾病一样，儿科IBD符合NIH对罕见疾病的定义，合作研究网络及其注册数据在其中发挥着关键作用。合并多个登记研究的队列将增加统计学功效和外部有效性。然而，登记研究彼此不同，如果不充分解决这些差异，可能会威胁研究的有效性。申请人提出了CER/PCOR的基本培训/教育活动，以及旨在通过合并两个儿科IBD网络登记系统来回答儿科发病CD中生物制剂CE问题的研究项目。这两个登记系统在患者/提供者人群基础以及收集的数据的广度和深度方面相互对比，这带来了一系列方法学挑战。拟议研究的开展将由一个强大的指导团队进行监测，该团队包括一名注册相关的以患者为中心的结局研究专家、一名临床内容专家和一名既定的流行病学专家，并将对申请人进行有关多注册CE研究的适当培训。生物制剂引入时间的研究问题是医学研究所（IOM）CER的前25个优先事项之一。本研究将提供目前缺乏的生物制剂CE信息，使所有利益相关者能够更客观地评估治疗的成本与受益或有效性与安全性的权衡，并促进决策。利用K18提供的知识，技能和专业提升，候选人将做好准备，担任本地和全国的CER/PCOR方法专家，特别是在儿科慢性疾病领域。

DESCRIPTION (provided by applicant): The purpose of this K18 career enhancement award is to provide mentored research experience, and career enhancement and education activities necessary for the applicant's transition to comparative effectiveness (CE) and patient-centered outcomes research (PCOR) in pediatric chronic disease as a local and national methodology expert. The applicant is an established independent statistical method researcher and successful biostatistician with many years of clinical trial experience, and has extensively collaborated in pediatric inflammatory bowel disease (IBD) including pediatric onset Crohn's disease (CD). She is at the cusp of this transition in order to attain her career goal of improving the quality of evidentiary information by enhancing the quantitative analysis of CER at both population and individual patient levels. This career goal arose from her collaborations in pediatric IBD, where the lack of CE evidence leads to a huge variation in care and management practice and little or no improvement in outcomes has observed over the past four decades despite therapeutic advances. Pediatric IBD, like many pediatric chronic conditions, meets the NIH definition for a rare disease for which collaborative research networks and their registry data play a critical role. Pooling the cohorts of multiple registries will increase statistical powr and external validity. However, registries differ from one another and the discrepancies, if not sufficiently addressed, may threaten the validities of studies. The applicant proposes training/education activities fundamental to CER/PCOR and a research project that aims to answer the timing of biologics CE question in pediatric onset CD by combining two pediatric IBD network registries. The two registries contrast each other in their patient/provider population bases, and breadth and depth of data they collect, which poses an array of methodological challenges. The conduct of the proposed research will be monitored by a strong mentoring team that includes a registry related patient centered outcomes research expert, a clinical content expert and an established epidemiology and will properly train the applicant about multi-registry CE studies. The research question of timing of biologics introduction is one of the Institute of Medicine (IOM)'s top 25 priorities for CER. This study will provide the currently lacking CE information of biologics, which will enable all stakeholders to more objectively assess the tradeoffs of cost versus benefit or effectiveness versus safety of the therapies, and facilitate th decision making. Leveraging the knowledge, skills and professional enhancement afforded by this K18, the candidate will be well poised to serve as a CER/PCOR method expert both locally and nationally, particularly in pediatrics chronic disease areas.