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; 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 直接进入第二阶段计划的目的是开发和测试精密医学的效率 肾脏疾病的临床试验招生平台。大约一半的随机对照试验不 达到他们的招聘目标。招募针对稀有肾脏疾病的试验尤其具有挑战性 经过分层和亚型的条件主要基于组织病理学分析。由于阿卡纳的 作为美国最大的肾病学实践的地位，药物开发人员经常接近公司 协助识别具有临床感兴趣的患者。将招募重点放在深奥的肾病学上 是一种独特而创新的临床试验入学方法，这可能具有重要的价值 赞助商。因此 世界一流的肾脏病理学家，并进入形成统计有效队列所需的大型患者人群， 即使在极少数情况下。 Arkana已经开发了Arkana Connect™，这是一种允许临床医生的移动应用程序 跟踪患者活检的处理并检索病理报告。在I阶段的等效计划中， 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 应用程序上的通信。超过1300名临床医生通常会使用该应用程序，为 更新的注册应用程序功能的市场进入。该公司还基于注册基准 基于先前的临床试验入学项目，多种罕见肾脏疾病的效率 手动方法；我们期望通过开发技术的发展至少两倍的入学率 患者的选择以及与临床医生的试验可用性的交流。基于阿卡纳的成功 Connect™，该公司渴望继续发展和验证注册职能， 以及要评估的现场测试：1）使用该应用程序来提高入学效率；和2） 改善传统服务不足社区的临床试验的进入，包括城市，外城和农村 区域。在第二阶段期间，我们将专注于优化应用程序的数字基础架构以确保可靠 沟通和遵守有关患者隐私的法规。离散元素搜索功能 将开发出来，允许临床试验发起人以狭义定义的患者人群为目标，对 开发针对罕见疾病的治疗学。将进行验证研究以最终确定 该应用程序的商业版和随后的研究代表400名参与医生 进行了评估这项新技术和过程改善临床试验入学率的能力。数据 通过这些评估将为Arkana的客户提供令人信服的证据，这是肾脏疾病的赞助商 临床试验，通过技术帮助的病理学指导的患者招募可以满足未满足的需求 有效且有针对性的临床试验入学。