目的:本研究旨在从不同的利益相关者中确定在糖尿病肾病(DKD)中实施精准医学的好处和障碍,并就未来的治疗方法达成共识,防止,甚至逆转这种疾病。方法:作为推动DKD实施精准医学的持续努力的一部分,在DKD,与参与药物开发和患者护理的不同利益相关者组织了为期两天的共识会议,包括患者,患者代表,制药工业,监管机构代表,卫生技术评估员,医疗保健专业人员,基础科学家,和临床学术研究人员。会议包括全体会议发言和讨论,和小组分组讨论。讨论主题是基于一个专题讨论会,焦点小组和文献检索。好处,讨论了实施精准医疗的障碍和潜在解决方案。分组会议的结果在全体会议上介绍,并构成了达成最终结论的广泛共识讨论的基础。在整个会议期间,参与者在他们的移动设备上回答了几个声明和开放式问题,使用实时在线调查工具。对陈述问题的答案进行了描述性分析。开放式调查问题的结果,对分组讨论和共识讨论进行了定性分析。结果和结论:来自26个国家的71名与会者参加了在阿姆斯特丹举行的建立共识会议,2019年4月。在第一天的开幕全体会议上,参与者同意以下观点:精准医疗是DKD的发展方向(n=57,中位数90,IQR[75-100]).缺乏有效的实施工具和生成可靠的数据被认为是重大障碍。确定的好处,例如,提高治疗的获益风险比,提供实质性激励措施,为已确定的障碍找到解决方案。更早和更多的多利益相关方合作和具体培训可以提供解决方案,以改变基于障碍和解决方案的临床和监管指南。第二天结束时,参与者对DKD中精准医学的看法更为细微(n=45,中位数83,IQR[70-92]),他们得出结论,精准医学是改善DKD患者治疗的重要途径.
Aim: This study aimed to identify from different stakeholders the benefits and obstacles of implementing precision medicine in diabetic kidney disease (DKD) and to build
consensus about a way forward in order to treat, prevent, or even reverse this disease. Methods: As part of an ongoing effort of moving implementation of precision medicine in DKD forward, a two-day consensus-building meeting was organized with different stakeholders involved in drug development and patient care in DKD, including patients, patient representatives, pharmaceutical industry, regulatory agencies representatives, health technology assessors, healthcare professionals, basic scientists, and clinical academic researchers. The meeting consisted of plenary presentations and discussions, and small group break-out sessions. Discussion topics were based on a symposium, focus groups and literature search. Benefits, obstacles and potential solutions toward implementing precision medicine were discussed. Results from the break-out sessions were presented in plenary and formed the basis of a broad
consensus discussion to reach final conclusions. Throughout the meeting, participants answered several statement and open-ended questions on their mobile device, using a real-time online survey tool. Answers to the statement questions were analyzed descriptively. Results of the open-ended survey questions, the break-out sessions and the
consensus discussion were analyzed qualitatively. Results and conclusion: Seventy-one participants from 26 countries attended the
consensus-building meeting in Amsterdam, April 2019. During the opening plenary on the first day, the participants agreed with the statement that precision medicine is the way forward in DKD (n = 57, median 90, IQR [75-100]). Lack of efficient tools for implementation in practice and generating robust data were identified as significant obstacles. The identified benefits, e.g., improvement of the benefit-risk ratio of treatment, offer substantive incentives to find solutions for the identified obstacles. Earlier and increased multi-stakeholder collaboration and specific training may provide solutions to alter clinical and regulatory
guidelines that lie at the basis of both obstacles and solutions. At the end of the second day, the opinion of the participants toward precision medicine in DKD was somewhat more nuanced (n = 45, median 83, IQR [70-92]) and they concluded that precision medicine is an important way forward in improving the treatment of patients with DKD.