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：
8394925
负责人：
Matthew Bernard Wiener
金额：
$ 68.93万
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 2014-06-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8394925
关键词：
Acceleration Address Advisory Committees Affect American American Cancer Society Antineoplastic Agents Budgets Businesses Cancer Etiology Cancer Patient Caring Cessation of life Characteristics Clinic Clinical Research Clinical Trials Commit Conduct Clinical Trials Consult Data Quality Diagnosis Doctor of Medicine Economics Enrollment Funding Future Gap Junctions Generations Goals Growth Head Heart Human Individual Investigational Therapies Lead Letters Life Malignant Neoplasms Manuals Medical Medicine Methodology Methods Modeling Molecular Molecular Target Oncologist Patient Care Patient Participation Patient Rights Patients Performance Pharmaceutical Preparations Pharmacologic Substance Pharmacotherapy Phase Physicians Population Positioning Attribute Productivity Quality of life Recording of previous events Research Research Infrastructure Research Personnel Resources Schedule Screening procedure Services Site Small Business Innovation Research Grant Societies Staging Standardization System Systems Development Technology Testing Therapeutic Time Training United States National Institutes of Health Validation Vendor Vision Work anticancer research base cancer care care systems commercialization cost design innovation meetings next generation oncology patient oriented product development research and development research to practice systems research technological innovation tool treatment trial web site

项目摘要

DESCRIPTION (provided by applicant): The American Cancer Society estimates that more than 1.5 million new cancer victims will be diagnosed this year in the U.S., and that ~572,000 Americans will die of cancer in 2011.1 More than 13 million Americans are living today with a history of cancer, and this number is heading toward 18 million by 2020.1 Beyond the human costs in lives, productivity, and quality of life, the NIH estimated cancer's direct medical costs t $103 billion in 2010, with total costs being $263 billion.1-2 Clearly, adequate drug therapy is not yet available, and we need better ways to rapidly develop and test new drugs. At the heart of the problem is a "one size fits all" approach to research that is not aligned to a cancer population comprised of hundreds of rare treatment indications spread among thousands of separate practices? Innovations are needed to deliver greater access to patients and to connect the right patients to the right clinical trials at the right time. We need to get next-generation cancer drugs from benchtop to the clinic-faster and at a lower cost-and the Obama administration's ambitious plans to double federal funding for cancer research within five years and to also double enrollment in U.S. cancer clinical trials is certainly in jeopardy given the current budget situation.3 Pharmatech Oncology is addressing this critical need via research system innovations that increase access to patients for participation in clinical trials. Our Just-n-Time (JIT) research system being developed in this multi- phase SBIR project is designed to closely align and coordinate U.S. clinical research with cancer patient care. Our strong Phase I results demonstrate that the feasibility of using JIT is a superior methodology for conducting clinical trials in indications where patients are potentially rare and highly embedded in a large care system. Those results set the stage for this Phase II project, which is focused upon completing the system development, optimization, standardization, and customer interactive infrastructure needed to offer the JIT research service through a website portal product, the "Pharmatech Nexus." The Pharmatech R&D team will pursue the following Phase II Aims: 1) Develop and demonstrate optimized JIT workflow and economics at research site level; 2) Develop and implement tools to standardize the JIT methodology; 3) Develop a business platform to drive scalability and expanded connectivity; and 4) Prove the integration of JIT methodology with molecular screening. Building on the concepts proven in Phases I and II, a new means of conducting clinical trials will be made broadly available. When fully developed by the expert Pharmatech team, the Pharmatech Nexus platforms will 1) make a large number of clinical trials, spanning multiple indications and treatment stages, available to oncologists for their patients; 2) provide a conduit for molecular testing to identify which targeted investigationl therapies may be appropriate; and 3) enable individual patients to enroll in the right trials very quickly and to receive research-based care on a schedule that accommodates their need to be treated. PUBLIC HEALTH RELEVANCE: As more-effective precision therapeutics become available to directly target the molecular causes of cancers, the costs and challenges of bringing a new generation of highly effective drugs from benchtop to clinic are escalating. The end goal of this multi-phase SBIR project is to establish a new, patient-centered clinical research system that is aligned with cancer patient care in the U.S. and to vastly expand patient/physician access to clinical trials as treatment options for individual patients. The Phase II work proposed here will position Pharmatech Oncology to lead a unique, collaborative marketplace initiative designed to drive cancer patient participation in clinical trials from 3% to 10% by 2020, enabling approval of next-generation cancer drugs more rapidly and at a lower cost.

描述（由申请人提供）：美国癌症协会估计，今年美国将有超过 150 万新癌症患者被诊断出来，2011 年约有 572,000 名美国人将死于癌症。1 如今，超过 1300 万美国人生活在癌症中。癌症史，到 2020 年，这一数字将接近 1800 万。1 除了人类在生命、生产力和生活质量方面付出的代价之外，NIH 估计癌症对人类的直接影响2010 年医疗费用为 1,030 亿美元，总费用为 2,630 亿美元。1-2 显然，充分的药物治疗并不但我们需要更好的方法来快速开发和测试新药。问题的核心是“一刀切”的研究方法与癌症人群不相符，该人群由分布在数千个不同实践中的数百种罕见治疗适应症组成？需要创新来为患者提供更多机会，并在正确的时间将正确的患者与正确的临床试验联系起来。我们需要以更快的速度和更低的成本将下一代癌症药物从实验室推向临床，而奥巴马政府雄心勃勃的计划是在五年内将联邦癌症研究经费增加一倍，并将美国癌症临床试验的注册人数增加一倍，这无疑是一项雄心勃勃的计划。鉴于当前的预算状况，该技术处于危险之中。3 Pharmatech Oncology 正在通过研究系统创新来满足这一迫切需求，这些创新增加了患者参与临床试验的机会。我们在这个多阶段 SBIR 项目中开发的准时 (JIT) 研究系统旨在密切协调和协调美国临床研究与癌症患者护理。我们强有力的 I 期结果表明，使用 JIT 的可行性是一种针对患者可能很少且高度嵌入大型护理系统的适应症进行临床试验的优越方法。这些结果为第二阶段项目奠定了基础，该项目的重点是完成通过网站门户产品“Pharmatech Nexus”提供 JIT 研究服务所需的系统开发、优化、标准化和客户交互基础设施。 Pharmatech 研发团队将追求以下第二阶段目标： 1) 在研究现场层面开发并展示优化的 JIT 工作流程和经济性； 2) 开发并实施工具来标准化 JIT 方法； 3) 开发一个业务平台来推动可扩展性和扩展连接性； 4) 证明 JIT 方法与分子筛选的整合。基于第一阶段和第二阶段验证的概念，将广泛提供一种进行临床试验的新方法。当 Pharmatech 专家团队全面开发后，Pharmatech Nexus 平台将 1) 为肿瘤学家为患者进行大量临床试验，涵盖多个适应症和治疗阶段； 2）提供分子测试的渠道，以确定哪些靶向研究疗法可能是合适的； 3) 使个体患者能够非常快速地参加正确的试验，并按照适合其治疗需要的时间表接受基于研究的护理。公共健康相关性：随着更有效的精准疗法可直接针对癌症的分子原因，将新一代高效药物从实验室推向临床的成本和挑战正在不断升级。这个多阶段 SBIR 项目的最终目标是建立一个新的、以患者为中心的临床研究系统，该系统与美国癌症患者护理相一致，并极大地扩大患者/医生参与临床试验的机会，作为个体患者的治疗选择。这里提出的 II 期工作将使 Pharmatech Oncology 领导一项独特的协作市场计划，旨在到 2020 年将癌症患者参与临床试验的比例从 3% 提高到 10%，从而更快、更低地批准下一代癌症药物成本。