PERSONALIZED DRUG RESPONSE: IMPLEMENTATION and EVALUATION in CKD

慢性肾脏病(CKD)是高收入国家的主要死亡原因之一，其患病率正在迅速增长。治疗慢性肾脏病的新疗法在人群水平上显示出明显的疗效。然而，个别患者的反应不同，许多患者表现出不佳的疗效。这表明“一刀切”的做法不再可持续。目前，在慢性肾脏病中还没有有效的药效学生物标记物来帮助指导个体患者的最佳治疗。此外，在日常临床实践中，生物标记物的研究与基于生物标记物的护理的实际实施之间仍有很大差距。因此，迫切需要验证和翻译生物标志物作为日常临床实践的工具。Prime-CKD在应对这一挑战方面具有独特的优势，因为它建立在最近发现的用于CKD治疗的药效生物标记物的基础上，并可以访问大量的临床数据和生物样本。Prime CKD将：1)通过药效学生物标志物的验证和实施，为CKD的药物治疗提供突破，这将促进个性化药物的使用。2)科学验证可供研究人员、诊断行业和监管部门使用的创新临床试验要素、管道和资格程序。3)建立利益相关者网络，真正让患者、临床医生和其他利益相关者参与个性化药物的实施4)制定生物标记物在日常临床实践中使用的路线图，以实现CKD的最佳治疗。这些结果将使临床医生能够在尽可能短的时间内将CKD患者与最有效的可用药物相匹配，从而减少疾病负担和疾病进展，提高生活质量和成本效益。Prime-CKD的结果将释放生物标记物在医疗保健中的潜力，并缩小生物标记物研究和临床应用在日常实践中的差距

Chronic Kidney Disease (CKD) is one of the major causes of death in high income countries and its prevalence is growing rapidly. New therapies for the treatment of CKD have shown clear efficacy at a population level. Yet, individual patients respond differently with many patients showing suboptimal efficacy. This indicates that a ‘one size fits all’ approach is no longer sustainable. Currently there are no validated pharmacodynamic biomarkers in CKD that aid in guiding optimal therapy for individual patients. Furthermore, there remains a large gap between biomarker research and actual implementation of biomarker based care in daily clinical practice. Thus, there is an urgent need to validate and translate biomarkers as tools for daily clinical practice. PRIME-CKD has a unique advantage to address this challenge as it builds on recently discovered pharmacodynamic biomarkers for CKD treatment, and has access to a vast amount of clinical data, bio-samples. PRIME CKD will: 1) Provide a breakthrough in the pharmacotherapy of CKD with the validation and implementation of pharmacodynamic biomarkers which will advance personalized medicine. 2) Scientifically validate innovative clinical trial elements, pipelines and qualification procedures that can be used by researchers, diagnostics industries and regulatory authorities. 3) Establish a Stakeholder Network to truly engage patients, clinicians and other stakeholders in implementation of personalized medicines 4) Develop a roadmap for biomarker use in daily clinical practice for the optimal treatment of CKD. These results will enable clinicians to match patients with CKD with the most effective available drug within the shortest possible timeframe leading to less disease burden and progression of disease, enhanced quality of life and increased cost-efficiency. PRIME-CKD results will thus unlock the potential of biomarkers in healthcare and close the gap between biomarker research and clinical use in daily practice