A Patient-Centered Approach to Enrolling Rare Cancer Patients in Clinical Trials

以患者为中心的方法在临床试验中招募罕见癌症患者

基本信息

批准号：
8001922
负责人：
Matthew Bernard Wiener
金额：
$ 15.47万
依托单位：
PHARMATECH, INC.
依托单位国家：
美国
项目类别：
财政年份：
2010
资助国家：
美国
起止时间：
2010-09-03 至 2011-08-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8001922
关键词：
Acceleration Accountability Address Administrative Efficiency Adult American American Cancer Society Animals Antineoplastic Agents Arizona Arts Biological Bypass Cancer Patient Cancer Therapy Evaluation Program Charge Clinic Clinical Clinical Data Clinical Research Clinical Trials Clinical trial protocol document Communities Community Clinical Oncology Program Community Practice Computer Systems Development Consult Cost-Benefit Analysis Country Coupled Data Data Collection Data Quality Development Diagnosis Disease Drug Industry Elements Enrollment Ensure Evaluation Food Foundations Funding Gap Junctions Generations Genomics Goals Government Guidelines Hairy Cell Leukemia Hypersensitivity Hypertension Individual Industry Institutes Internet Investigational Therapies Lead Letters Life Malignant Neoplasms Measures Medical Medicine Methodology Methods Metric Modeling Molecular Molecular Abnormality National Cancer Institute Oncologist Orphan Disease Outcome Patient Care Patient Participation Patient Rights Patients Performance Pharmaceutical Preparations Pharmacologic Substance Phase Phase II Clinical Trials Physicians Population Positioning Attribute Privacy Private Practice Process Publications Qualifying Rare Diseases Relative (related person)Reporting Research Research Ethics Committees Research Institute Research Methodology Research Personnel Resources Risk Rural Community Safety Sampling Side Site Site Visit Small Business Innovation Research Grant Societies Solutions Staging System Target Populations Testing Time TimeLine Translating Underserved Population United States United States National Institutes of Health University Hospitals Validation Work anticancer research authority base cancer Biomedical Informatics Grid cancer care cancer therapy cancer type care systems clinical research site commercialization community based care comparative cost design drug development economic impact experience forging human subject improved innovation interest malignant breast neoplasm meetings member novel novel strategies novel therapeutics oncology patient oriented patient population patient safety prevent public health relevance statistics success systems research therapeutic development triple-negative invasive breast carcinoma validation studies

项目摘要

DESCRIPTION (provided by applicant): Our new president vows to address the pervasive issue of cancer. Over 1.4 million Americans will be diagnosed with cancer this year, joining over 10 million living Americans who have experienced cancer. The medical costs of cancer care were $78 billion in 2008, and the overall economic impact was estimated to be over $200 billion. The current administration plans to double federal funding for cancer research within five years, primarily at NIH and NCI, with a focus on research for rare cancers. Our expert collaborator on this project, Dr. Daniel Von Hoff, describes two classes of rare cancers. There are the truly rare diseases (e.g., hairy cell leukemia), but also common histological cancer types like breast cancer, with rare molecular subtypes such as triple negative breast cancer. Cancer profiling into these specific "rare cancer" sub-classes and customized treatment for individual patients is now clinically realistic. Challenges to the development of tomorrow's therapies include identifying and connecting the right patients with the right treatments. Today, less than 5 percent of adult cancer patients participate in clinical trials. There are multiple reasons for this, including reimbursement problems in research funding, a daunting administrative burden, and a growing shortage of clinical researchers. The Obama administration has pledged to increase cancer trial participation to 10 percent of adult patients. In addition, the NCI Director has been charged with identifying the regulatory barriers that prevent the timely completion of successful clinical research. Pharmatech Oncology has conceptualized a unique solution that identifies patients first (Just-in-Time", [JIT]), then rapidly connects patients and physicians to appropriate clinical trial opportunities. This differs from the traditional model, which focuses initially on administrative process and secondarily on patient enrollment. The traditional model can be effective for clinical trials in indications with millions of patients, but for rare cancers, it is not as effective. The goal of this Phase I SBIR project is to assess the feasibility of using the JIT approach to efficiently enroll and conduct analysis of two or more ongoing or completed clinical trials for rare cancers, compared with the traditional model. We expect to demonstrate that JIT can increase enrollment rates while maintaining patient safety and data quality, which will lead to a Phase II project designed to fully demonstrate/validate JIT across a larger number and variety of rare-cancer trials and to show how JIT streamlines the administrative workflow. We intend to demonstrate how the JIT system can be applied to community-based and institutional sites-becoming a platform for matching individual patients anywhere in the country to appropriate clinical trials without the typical and potentially harmful administrative delays. The nationwide JIT initiative will enable patient access to NCI- and privately funded clinical trials, will improve overall and representative participation, and will boost access to cancer cures and to personalized cancer treatments. PUBLIC HEALTH RELEVANCE: Modern strategies for developing new, targeted cancer therapies and testing them in clinical trials increasingly focus on highly patient-specific molecular abnormalities-turning many cancer trials into what amount to "rare cancer" trials. Currently, these developments bypass the majority (85 percent or more) of candidate cancer patients because those patients go largely undetected, embedded in a nationwide, community practice treatment system that has limited access to clinical trials of targeted therapies. The overall goal of this multi-phase SBIR project is to develop, validate and commercialize Pharmatech Oncology's novel Just-In-Time method for facilitating rapid enrollment of rare-cancer patients, reducing administrative and technical barriers, and improving patient access to state-of-the-art cancer treatment through advanced, personalized-medicine clinical trials.

描述（由申请人提供）：我们的新总统发誓要解决普遍存在的癌症问题。今年将有超过 140 万美国人被诊断患有癌症，加入到超过 1000 万在世美国人患有癌症的行列。 2008 年癌症治疗的医疗费用为 780 亿美元，总体经济影响估计超过 2000 亿美元。现任政府计划在五年内将联邦癌症研究经费增加一倍，主要是在 NIH 和 NCI，重点是罕见癌症的研究。我们该项目的专家合作者 Daniel Von Hoff 博士描述了两类罕见癌症。有真正罕见的疾病（例如毛细胞白血病），但也有常见的组织学癌症类型，如乳腺癌，以及罕见的分子亚型，如三阴性乳腺癌。现在，将癌症分析为这些特定的“罕见癌症”亚类并为个体患者提供定制治疗在临床上是现实的。未来疗法开发面临的挑战包括识别正确的患者并将其与正确的治疗联系起来。如今，只有不到 5% 的成年癌症患者参与临床试验。造成这种情况的原因有很多，包括研究经费的报销问题、繁重的行政负担以及临床研究人员的日益短缺。奥巴马政府承诺将癌症试验参与率提高到 10% 的成年患者。此外，NCI 主任还负责查明阻碍及时成功完成临床研究的监管障碍。 Pharmatech Oncology 构思了一种独特的解决方案，首先识别患者（准时制”，[JIT]），然后快速将患者和医生与适当的临床试验机会联系起来。这与传统模式不同，传统模式最初侧重于管理流程和其次是患者入组。传统模式对于数百万患者的适应症临床试验可能有效，但对于罕见癌症，该 I 期 SBIR 项目的目标是评估使用 JIT 方法的可行性。有效地与传统模型相比，注册和分析两个或多个正在进行或已完成的罕见癌症临床试验，我们希望证明 JIT 可以提高注册率，同时保持患者安全和数据质量，这将导致设计的 II 期项目。在大量和多种罕见癌症试验中充分展示/验证 JIT，并展示 JIT 如何简化管理工作流程。我们打算展示 JIT 系统如何应用于基于社区和机构场所，成为一个平台，用于将全国任何地方的个体患者与适当的临床试验相匹配，而不会出现典型的和潜在有害的行政延误。全国性的 JIT 计划将使患者能够参与 NCI 和私人资助的临床试验，提高整体和代表性的参与度，并将促进癌症治愈和个性化癌症治疗的获得。公共健康相关性：开发新的靶向癌症疗法并在临床试验中对其进行测试的现代策略越来越关注患者高度特异性的分子异常，从而将许多癌症试验转变为“罕见癌症”试验。目前，这些进展绕过了大多数（85％或更多）候选癌症患者，因为这些患者基本上未被发现，被纳入全国范围内的社区实践治疗系统，而该系统对靶向治疗的临床试验的访问机会有限。这个多阶段 SBIR 项目的总体目标是开发、验证和商业化 Pharmatech Oncology 的新型即时方法，以促进罕见癌症患者的快速入组，减少管理和技术障碍，并改善患者获得最新状态的机会-通过先进的个性化医学临床试验提供最先进的癌症治疗。