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: 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
• 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

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，今年将在美国诊断出150万新的癌症受害者，约有572,000名美国人将在2011年死于癌症。11.1今天有1300万美国人生活在癌症的历史上，这一数字持续了1800万，到2020年，到2020年。到2020年。 2630亿美元。1-2清楚，足够的药物治疗不是 但是可用，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“一种适合所有规模”的研究方法，该方法与数百种罕见的治疗迹象组成的癌症人群不符，数以千计的单独实践中扩散了？需要创新以提供更多与患者的机会，并在正确的时间将正确的患者连接到正确的临床试验。我们需要将下一代癌症药物从台式台阶到诊所 - 成本较低，而奥巴马政府的雄心勃勃的计划在五年内将联邦政府的资金加倍，并且在美国癌症临床试验中的注册肯定是危险的，鉴于当前的预算情况，这是危险的。3PharmAtech Oncology通过研究临床进行了临床Innerov的访问，以提高该临床的访问。我们在这个多期SBIR项目中开发的正式n--N（JIT）研究系统旨在与癌症患者护理紧密结合和协调美国的临床研究。我们强大的I期结果表明，使用JIT的可行性是在患者可能罕见并且高度嵌入大型护理系统中进行临床试验的卓越方法。这些结果为该II阶段项目奠定了基础，该项目的重点是完成系统开发，优化，标准化和客户互动基础架构，以通过网站门户网站产品“ Pharmatech Nexus”提供JIT研究服务。 Pharmatech研发团队将追求以下第二阶段的目标：1）在研究站点层面开发和展示优化的JIT工作流程和经济学； 2）开发和实施工具以标准化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。在第I和II阶段证明的概念的基础上，将广泛使用一种新的进行临床试验的方法。当专家Pharmatech团队完全开发时，Pharmatech Nexus平台将1）进行大量临床试验，涵盖多个指示和治疗阶段，可供肿瘤学家为患者提供； 2）提供用于分子测试的管道，以确定可能适当的靶向研究疗法； 3）使各个患者能够非常快速参加正确的试验，并按照适应其需要接受治疗的时间表接受基于研究的护理。 公共卫生相关性：随着更有效的精确治疗可直接针对癌症的分子原因，将新一代高效药物从卧床台上带到诊所的成本和挑战正在上升。这个多相SBIR项目的最终目标是建立一种以患者为中心的新的，以患者为中心的临床研究系统，该系统与美国的癌症患者护理保持一致，并大大扩展患者/医师获得临床试验作为单个患者的治疗选择。此处提出的第二阶段工作将使Pharmatech肿瘤学领导一项独特的，协作的市场计划，旨在使患者参与到2020年的临床试验从3％到10％，从而使下一代癌症药物更快地批准以较低的成本批准。