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.

项目摘要 这一直接到第二阶段计划的目标是开发和测试一种精密医学的疗效 肾脏疾病临床试验征集平台。大约一半的随机对照试验没有 达到他们的招聘目标。招募针对罕见肾脏疾病的试验尤其具有挑战性，因为许多 病情需要分层和分型，主要基于组织病理学分析。由于阿卡纳的 作为美国最大的肾脏病理诊所，药物开发商经常与该公司接洽 协助确定有相关临床指征的患者。聚焦深奥肾脏病理征集 是临床试验登记的一种独特和创新的方法，这可能对 赞助商。因此，Arkana处于独一无二的地位，支持以病理学为导向的注册，有25+ 世界一流的肾脏病理学家和接触到大量患者群体所需的统计有效队列， 即使在罕见的情况下也是如此。Arkana已经开发了Arkana Connect™，这是一款移动应用程序，允许临床医生 跟踪患者活组织检查的过程并检索病理报告。在阶段I-等效计划期间， 该公司启动了支持病理报告和临床医生的关键基础设施的开发 在应用程序上进行交流。超过1300名临床医生经常使用这款应用程序，为 更新后的招生APP功能进入市场。该公司还对注册人数进行了基准测试 基于先前开展的临床试验登记项目对多种罕见肾脏疾病的有效性 手工方式；我们希望通过改进技术的开发至少将招生人数增加一倍 患者选择和与临床医生沟通试验可用性。基于Arkana的成功 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，以评估：1)使用APP提高了罕见肾脏疾病的登记效率；以及2) 在城市、郊区和农村等传统服务不足的社区改善临床试验的可及性 区域。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 沟通和遵守有关患者隐私的规定。离散元素搜索函数 将被开发，允许临床试验发起人针对狭义的患者群体，对于 罕见病治疗方法的发展。将进行验证研究，以最终确定 该应用程序的商业版本和代表400名参与医生的后续研究将是 以评估这一新技术和流程改善临床试验登记的能力。数据 这些评估将为Arkana的客户提供令人信服的证据，这些客户是肾脏疾病的赞助商 临床试验，在技术的帮助下，以病理为导向的患者招募可以解决未得到满足的 高效、有针对性的临床试验招募。