Center for Comparative Effectiveness Research in Cancer Genomics - CANCERGEN

描述（由申请人提供）：许多癌症基因组应用已可用于评估疾病风险，预后和药物反应预测，提供根据患者或疾病的遗传特征量身定制的有效和高效癌症治疗的承诺-基因组学和个性化医学（GPM）。目前的监管结构侧重于分析的准确性，而不是临床有效性或效用，这造成了一种可能，即基因组测试将在没有足够证据支持其与标准护理相比的价值的情况下被引入临床实践，或者相反，提供高临床效用和价值的测试将在实践中不被利用或报销。在许多情况下，需要随机对照试验（rct）的证据来确定临床价值。为了给决策者提供有用的信息，这些试验将需要超越传统的设计，包括患者的结果和经济评估。此外，需要更多地使用更接近临床实践的试验设计，例如实用试验。美国国家癌症研究所（NCI）支持的癌症临床试验合作小组项目为开展高质量的GPM应用于癌症的前瞻性比较有效性研究（CER）提供了令人信服的基础设施。为了最大限度地发挥这一基础设施对癌症实践的影响，从多个利益相关者的角度出发，开发确定癌症基因组学中CER主题的方法也至关重要。此外，为了应对向患者提供创新的GPM技术，同时产生决策者所需的高质量证据的挑战，需要新的共享筹资机制，如证据开发覆盖（CED）。

DESCRIPTION (Provided by the applicant): A number of genomic applications for cancer have become available for assessing disease risk, prognosis, and drug response prediction, offering the promise of effective and efficient cancer care tailored to the genetic profile of the patient or disease - Genomics and Personalized Medicine (GPM). The current regulatory structure - which focuses on analytical accuracy rather than clinical validity or utility - creates a potential that genomic tests will be introduced into clinical practice without sufficient evidence supporting their value compared to standard care, or conversely that tests that provide high clinical utility and value will not be utilized or reimbursed in practice. In many cases, evidence from randomized controlled trials (RCTs) will be needed to establish clinical value. To provide useful information for decision makers, such trials will need to extend beyond traditional designs to include patient outcomes and economic evaluation. In addition, greater use of trial designs that more closely represent clinical practice, such as pragmatic trials, are needed. The National Cancer Institute (NCI)-supported cancer clinical trials Cooperative Group Program offers a compelling infrastructure for the conduct of high-quality prospective comparative effectiveness research (CER) studies of GPM applications for cancer. To maximize the impact of this infrastructure on cancer practice, it is also critical to develop methods for identifying CER topics in cancer genomics that are priorities from multiple stakeholder perspectives. In addition, to address the challenge of making innovative GPM technologies available to patients while at the same time generating high quality evidence needed by decision makers, novel shared funding mechanisms such as Coverage with Evidence Development (CED) are needed. We propose to develop a Center for Comparative Effectiveness Research in Cancer Genomics (CANCERGEN), a sustainable, multidisciplinary, collaborative consortium including the Fred Hutchinson Cancer Research Center, the Center for Medical Technology Policy, University of Washington, and the Southwest Oncology Group, one of the largest of the National Cancer Institute-supported cancer clinical trials cooperative groups in the United States. CANCERGEN will integrate expertise in decision modeling, database linkage, ethics, policy, and clinical trial design in order to leverage and build upon the existing strengths of the SWOG clinical trials network to facilitate the rapid design and implementation of prospective CER studies of GPM technologies. Moreover, CANCERGEN will create a unique and valuable partnership with the Center for Medical Technology Policy, whereby multidisciplinary stakeholders will collaborate with CANCERGEN to identify research priorities. Our vision is that CANCERGEN will overcome structural barriers that have limited the quality and timeliness of evaluations in GPM, with the result that promising technologies in the cancer genomics pipeline can move rapidly from "proof of principle" to improving the effectiveness and costeffectiveness of cancer clinical care. PUBLIC HEALTH RELEVANCE: A variety of genomic and personalized medicine applications for cancer offer the promise of improved cancer care, but the process by which genomic tests move from development to market availability rarely generates high quality evidence supporting their clinical utility or economic value. To address this problem, we will create a Center that includes researchers from the Fred Hutchinson Cancer Research Center, the Center for Medical Technology Policy, the University of Washington, and the Southwest Oncology Group, one of the largest of the National Cancer Institute-supported cancer clinical trials cooperative groups in the United States. This collaborative network will overcome barriers that have limited the quality and timeliness of evaluations in genomics and personalized medicine, with the result that promising technologies currently in the cancer genomics pipeline can move rapidly from "proof of principle" to improving the effectiveness and cost effectiveness of cancer clinical care to benefit public health.