Decentralising precision medicine patient recruitment and clinical trial research to accelerate drug development

分散精准医学患者招募和临床试验研究以加速药物开发

• 批准号: 10034737
• 金额: $ 41.69万
• 依托单位: SANO GENETICS LIMITED
• 依托单位国家: 英国
• 项目类别: Collaborative R&D
• 财政年份: 2022
• 资助国家: 英国
• 起止时间: 2022 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=10034737
• 关键词: Decentralising; precision; medicine; patient; recruitment

Traditionally outsourced to Contract research organisations ("CROs"), and run through in-person visits to the clinical trial sites, the clinical trial process has not changed since the 1980s.Over the past ten years, there has been a monumental shift in our ability to collect and analyse genomic data due to continued innovation and reduced DNA sequencing costs. By the nature of personalised medicine, finding patients is complex, with very selective inclusion/exclusion criteria to enrol participants into trials. The CROs have a cost-plus model where they rely on bricks-and-mortar sites for patient recruitment, which often do not have adequate infrastructure or patient access. This is particularly hard given the fragmentation of genomic data across government, academia, consumer platforms and healthcare providers, many of which do not meet the high quality of information required for gene therapy trials. This combination results in significant time and cost inefficiencies and a high failure rate for patient enrolment.Clinical trials in genetic neurodegenerative diseases, and other common complex diseases in particular take a significant amount of time, resources, and funding to recruit the right patients at the right stage of disease, with many failing due to poor recruitment and engagement. Taking up to 15 years and ~£119m to bring to market, potential therapies often show promise in Phase 2 trial results, then prove to be ineffective or face enrolment challenges in the subsequent, larger-scale Phase 3 trials.The focus of this innovation is to develop a fully decentralised (at-home and digital first) genetic testing platform that combines:Genetic counsellingNon-invasive sample collectionBioinformaticsReturn of resultsIn one platform, available in multiple languages across the UK and EU.The output of the innovation will be an end-to-end platform for providing non-invasive sample collection, proprietary bioinformatics analysis, genetic counselling, and return of results that can be provisioned on independent cloud infrastructure in order to enable the operation of decentralised genetic testing studies across a range of jurisdictions with different data privacy regulations. The solution will address rare and common genetic diseases across three major customer segments: biopharma, biotechnology, and population genomics.

传统上外包给合同研究组织（“CRO”），并通过亲自访问临床试验地点，临床试验过程自20世纪80年代以来一直没有改变。在过去的十年中，由于持续的创新和DNA测序成本的降低，我们收集和分析基因组数据的能力发生了巨大的变化。由于个性化医疗的性质，寻找患者是复杂的，具有非常选择性的入选/排除标准来招募参与者参加试验。CRO有一个成本加成模式，他们依靠实体网站进行患者招募，这些网站通常没有足够的基础设施或患者通道。考虑到政府、学术界、消费者平台和医疗保健提供商的基因组数据碎片化，这一点尤其困难，其中许多数据无法满足基因治疗试验所需的高质量信息。遗传性神经退行性疾病和其他常见的复杂疾病的临床试验需要大量的时间、资源和资金来招募处于疾病正确阶段的正确患者，其中许多由于招募和参与不足而失败。潜在的疗法通常在2期试验结果中显示出希望，然后被证明无效或在随后的更大规模的3期试验中面临招募挑战。（在家和数字化第一）基因检测平台，结合了：遗传咨询非侵入性样本收集生物信息学结果返回在一个平台上，在英国和欧盟提供多种语言。创新的成果将是一个端到端的平台，用于提供非侵入性样本收集，专有的生物信息学分析，遗传咨询，以及可以在独立的云基础设施上提供的结果返回，以便在具有不同数据隐私法规的一系列司法管辖区内进行分散的基因检测研究。该解决方案将解决三个主要客户领域的罕见和常见遗传疾病：生物制药，生物技术和人口基因组学。