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

• 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
• 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

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140万美国人被诊断出患有癌症，并加入了经历过癌症的1000万活着的美国人。 癌症护理的医疗费用在2008年为780亿美元，估计总体经济影响超过2000亿美元。当前的政府计划在五年内（主要在NIH和NCI）内为癌症研究提供一倍的资金，重点是对稀有癌症的研究。 我们在该项目的专家合作者Daniel von Hoff博士描述了两类罕见的癌症。确实有罕见的疾病（例如，毛状细胞白血病），还有常见的组织学癌症，例如乳腺癌，具有罕见的分子亚型，例如三重阴性乳腺癌。癌症分析为这些特定的“稀有癌症”子类和针对个别患者的定制治疗的癌症现象是现实的。 明天疗法发展的挑战包括识别和将合适的患者与合适的治疗联系起来。如今，不到5％的成年癌症患者参加了临床试验。造成这种情况的原因有很多，包括研究资金中的报销问题，令人生畏的行政负担以及越来越多的临床研究人员。奥巴马政府已承诺将癌症试验参与增加到10％的成年患者。此外，NCI董事还负责确定及时完成成功临床研究的监管障碍。 PharmAtech肿瘤学已经概念化了一种独特的解决方案，该解决方案首先识别患者（即将到来”，[JIT]），然后迅速将患者和医生与适当的临床试验机会联系起来。这与传统模型不同，最初侧重于行政过程，其次是针对患者的招生。传统模型可以在临床上有效，但对于数百万的患者而言，这是有效的，而不是为了有效的canscore，它是有效的。是为了评估使用JIT方法有效地招募和进行稀有癌症的临床试验的可行性，与传统模型相比，我们期望JIT可以提高入学率，同时保持患者的安全性和数据质量，这将导致II期项目旨在在跨越较大的JIT和稀有数字和较大数字的JIT和CARMAL BROVELS和VARLED CALLSL和CALLSLECERLESLESLESLESLESLESLESLECERLECERLE-CARMEL和CALLSLE-CANDLS和CARMEL cANDLS CALTLS和CALTLES cANTLS cANDLS和CALTINL。我们打算说明如何将JIT系统应用于社区和机构站点，以使该平台与该国任何地方的各个患者相匹配，以适当进行适当的临床试验，而无需典型且可能有害的行政延迟。全国JIT倡议将使患者获得NCI-和私人资助的临床试验，将改​​善整体和代表性的参与，并将提高对癌症治疗和个性化癌症治疗的机会。 公共卫生相关性：开发新的，有针对性的癌症疗法并在临床试验中对其进行测试的现代策略，越来越关注高度患者特异性的分子异常，将许多癌症试验转变为“稀有癌症”试验的量。目前，这些发展绕过了大多数（85％或更多）候选癌症患者，因为这些患者在很大程度上未被发现，嵌入了全国社区实践治疗系统中，该治疗系统有限地获得了目标疗法的临床试验。这个多相SBIR项目的总体目标是开发，验证和商业化Pharmatech肿瘤学的新型即时方法，用于促进稀有癌症患者快速入学，减少行政和技术障碍，并通过高级，个性化的药物临床试验来改善患者进入先进的癌症治疗。