A proprietary digital platform for precision patient identification and enrollment of clinical trials for rare kidney diseases

用于精确识别患者和注册罕见肾脏疾病临床试验的专有数字平台

• 批准号: 10822581
• 负责人: Christopher P Larsen
• 金额: $ 97.63万
• 依托单位: NEPHROPATHOLOGY ASSOCIATES
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-09-15 至 2025-08-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10822581
• 关键词: Address; Affect; Automation; Benchmarking; Biological Markers; Biopsy; Clinical; Clinical Trials; Communication; Data; Databases; Development; Diagnostic; Disease; Effectiveness; Elements; Eligibility Determination; Engineering; Enrollment; Ensure; Evaluation; Exclusion Criteria; Geographic Locations; Goals; Improve Access; Infrastructure; Kidney; Kidney Diseases; Laboratories; Lesion; Letters; Manuals; Marketing; Methods; Nephrology; Notification; Pathologist; Pathology; Pathology Report; Pathway interactions; Patient Monitoring; Patient Recruitments; Patient Selection; Patients; Persons; Pharmaceutical Preparations; Phase; Physicians; Positioning Attribute; Process; Randomized, Controlled Trials; Rare Diseases; Regulation; Reporting; Research; Schedule; Signs and Symptoms; Small Business Innovation Research Grant; Software Validation; Standardization; Stratification; System; Technology; Telefacsimile; Testing; Text; United States; Update; Validation; clinical trial enrollment; cohort; cost; digital; digital platform; efficacy evaluation; efficacy testing; expectation; experience; field study; follow-up; improved; inclusion criteria; innovation; interest; mobile application; new technology; patient population; patient privacy; precision medicine clinical trials; programs; provider communication; rare condition; recruit; remediation; rural area; screening; success; technology development; therapeutic development; tool; trial enrollment; underserved community; validation studies

Project Summary The goal of this Direct to Phase II program is to develop and test the efficacy of a Precision Medicine Clinical Trials Enrollment Platform for kidney diseases. Approximately half of randomized controlled trials do not meet their recruiting target. Enrolling trials targeting rare kidney diseases is particularly challenging with many conditions subject to stratification and sub-typing, largely based on histopathological analysis. Due to Arkana’s position as the largest nephropathology practice in the U.S., drug developers often approach the company to assist in identifying patients with clinical indications of interest. Focusing recruitment on esoteric nephropathology is a unique and innovative approach to clinical trials enrollment, which is likely to have significant value to sponsors. Arkana is therefore in a one-of-a-kind position to support pathology-directed enrollment, with 25+ world-class renal pathologists and access to a large patient population required to form statistically valid cohorts, even for rare conditions. Arkana has already developed Arkana Connect™, a mobile app allowing clinicians to track the processing of patient biopsies and retrieve pathology reports. During a Phase I-equivalent program, the company initiated the development of key infrastructure to support pathology reporting and clinician communication on the app. Over 1,300 clinicians use the app routinely, providing a straightforward pathway for market entry of the updated enrollment app capabilities. The company has also benchmarked enrollment efficiency for multiple rare kidney diseases based on prior clinical trial enrollment projects undertaken with manual methods; we expect to at least double enrollments through development of technology that improves patient selection and the communication of trial availability with clinicians. Based on the success of Arkana Connect™, the company is eager to move forward with development and validation of enrollment functions, as well as field testing to evaluate: 1) improved enrollment efficiency using the app for rare kidney diseases; and 2) improve access to clinical trials in traditionally underserved communities including urban, exurban, and rural areas. During Phase II we will focus on optimizing the digital infrastructure of the app to ensure reliable communication and compliance with regulations regarding patient privacy. Discrete element search functions will be developed, allowing clinical trial sponsors to target narrowly defined patient populations, essential for development of therapeutics against rare diseases. Validation studies will be undertaken to finalize the commercial version of the app and a subsequent study representing 400 participating physicians will be performed to evaluate the ability of this new technology and process to improve clinical trial enrollment. Data from these evaluations will provide compelling evidence to Arkana’s customers, the sponsors of kidney disease clinical trials, that pathology-directed patient recruitment aided by technology can address the unmet need for efficient and targeted clinical trial enrollment.

项目概要 该直接进入二期项目的目标是开发和测试精准医学的功效 肾脏疾病临床试验注册平台大约有一半随机对照试验没有。 对于许多人来说，实现针对罕见肾脏疾病的招募目标尤其具有挑战性。 由于 Arkana 的原因，病情需要进行分层和分型，主要基于组织病理学分析。 作为美国最大的肾脏病理学诊所，药物开发商经常与该公司接洽 协助识别具有感兴趣的临床适应症的患者。 是一种独特且创新的临床试验注册方法，这可能对临床试验具有重大价值 因此，Arkana 处于独一无二的地位，可以支持 25 名以上的病理学指导入组。 世界一流的肾脏病理学家和接触大量患者群体所需的形成统计上有效的队列， 即使在罕见的情况下，Arkana 也已经开发了 Arkana Connect™，这是一款移动应用程序，可以让您 在 I 期等效项目中跟踪患者活检的处理并检索病理报告。 该公司启动了关键基础设施的开发，以支持病理报告和临床医生 超过 1,300 个最爱的人习惯性地使用该应用程序，为他们提供了一个简单的途径。 该公司还对更新的注册应用程序功能的市场准入进行了基准测试。 根据之前开展的临床试验入组项目，多种罕见肾脏疾病的治疗效率 手动方法；我们希望通过开发改进的技术，使注册人数至少增加一倍 基于 Arkana 的成功，患者选择和试验可用性的沟通。 Connect™，该公司渴望推进注册功能的开发和验证，因为 以及现场测试来评估：1) 使用该应用程序提高罕见肾脏疾病的登记效率；2) 改善传统上服务不足的社区（包括城市、远郊和农村）获得临床试验的机会 在第二阶段，我们将重点优化应用程序的数字基础设施，以确保可靠。 有关患者隐私的通信和合规性规定。 将被开发，允许临床试验申办者针对狭窄定义的患者群体，这对于 将进行针对罕见疾病的治疗方法的开发以最终确定。 该应用程序的商业版本以及代表 400 名参与医生的后续研究将 旨在评估这项新技术和流程改善临床试验数据的能力。 这些评估将为 Arkana 的客户（肾脏疾病的赞助者）提供令人信服的证据 临床试验表明，在技术辅助下以病理为导向的患者招募可以解决未满足的需求 高效且有针对性的临床试验入组。