传导的想法“site-less” or “virtual”临床试验已经存在了一段时间,并且像许多新的见解一样,已经引起了人们的怀疑。但是,业界最近开始意识到实现此方法的可能性以及它可以为患者和赞助者带来的好处。 《自然药物发现》的专栏the month of May have dedicated two pages to the topic of 虚拟 trials, ie clinical trials conducted limiting or eliminating the need to make patients recruited at research Centres. The attention is also high from a 监管机构 point of view: companies must be sure to completely adhere to laws and guidelines requests; in this sense, both the GCPs in the second revision (validation of computerized systems, risk analysis) and FDA guidelines were implemented. The American agency seems to be ahead of EMA on this point to date.

虚拟试用

Virtual clinical trials are a relatively new method of conducting a clinical trial to collect data on the safety and efficacy of a molecule or medical device. These trials take full advantage of technology (apps, monitoring devices, etc.) and inclusion platforms (recruitment, informed consent, counselling, measurement of endpoints and any adverse reactions) to allow the patient to be homebased at every stage of clinical trial. By relying on the new possibilities offered by technology, many argue that 虚拟ly conducted clinical trials offer an opportunity for a more patient-centered approach.

Pioneeristic 虚拟 studies

第一个部分虚拟的研究可以追溯到2001年,当时礼来公司(Eli Lilly)除了参加对参考中心的访问之外,还对男性进行了他达拉非(tadalafil)药物测试以对抗勃起功能障碍。十年后是辉瑞’的时间,为REMOTE(膀胱过度活动症治疗经验的电子监测研究)的第一个完全虚拟的临床试验模型铺平了道路。该研究是第一个随机临床试验,通过网络和智能手机招募和收集患者数据。主要目标之一是将虚拟方法与常规IV期临床研究进行比较,以确定虚拟实验项目是否可以用作将来研究的有用模型。不幸的是,辉瑞公司进行的REMOTE研究面临许多挑战,尤其是招募患者的问题(目标群体的大多数成员都是老年人,因此使用技术的能力有限)。

Probably, as long as the use of technology and this new way of conducting clinical trials will not capture the trust of patients, the right approach is a step by step one, in which 虚拟 study is not the only possibility offered, but starts to be presented among the possible options. A so-called hybrid approach, as Genentech has carried out with its head to head trial on rituximab and mycophenolate mofetil in patients with pemphigus vulgaris, a rare autoimmune skin disease. Recruitment through the “virtual”渠道(尤其是通过Facebook和Google AdWords渠道)比标准渠道(包括21个研究中心)更快。去年再次展示了这种速度,当时 科学37 completed a 372-person, placebo-controlled trial of a topical probiotic spray for mild-to-moderate acne on behalf of AOBiome. Todd Krueger, AOBiome’s CEO, says that the study lasted less than 12 months, much faster than anticipated. In March of this year, Novartis announced that, with an agreement with 科学37, it will launch up to 10 虚拟 clinical trials in the next three years.

好处

The number of recruited patients increases considerably if the barriers linked to travel distances are broken down to reach the research centers. Furthermore, by reducing the discomfort associated with travel and simplifying the collection of information, it is hypothesized that 虚拟 trials can reduce the current high drop-out rate of patients involved in phase III studies (equal to 40% today). «This is a very important and interesting point – confirms 洛伦佐·科蒂尼(Lorenzo Cottini),AFI(Associazione Farmaceutici industriali)临床试验研究小组副协调员,高级研究CRO总裁。已经有足够的技术来尽可能避免患者运动,从而提高研究的依从性。你可以做“remote”拜访是针对那些无法移动但技术水平最低(有时甚至只有平板电脑)或看护者可以帮助他们的患者。为了不选择病人而只招募最多的人“technological” ones, this type of view could be added to the traditional ones – as said the hybrid approach». About the use of technology, electronic medical records can help to identify subjects with specific targeted characteristics and to increase awareness of experimentations and recruit subjects directly. From an economic point of view, although this new approach requires strong 技术的 support and the movement of health personnel, it is believed that savings can be achieved both because time is reduced and because the traditional set-up of multiple study sites is eliminated. Virtual testing also offers the possibility of reducing risks in the drug development process. The “live”借助监控装置进行数据收集,研究人员可以更轻松地进行校准,修改甚至中断研究。这有利于患者安全,允许进行更具体的研究设计,并在研究失败的情况下限制成本。最后,虚拟实验设计可以使感兴趣的团体(投资者,医生,政府机构,患者权益团体,甚至患者本身)有更多机会在研究中发挥积极作用。

怀疑主义

正如我们在《自然药物发现》中读到的那样,从法律的角度来看,虚拟试验令人恐惧。一方面,我们必须保证所招募患者的隐私,另一方面,我们必须确保所收集数据的真实性,否则会损害研究结果。这也许是该行业的主要沉默寡言。另一个困惑在于与病人接触的临床医生人数很少:虚拟研究必须保证与标准研究相同的质量标准。为了增加注册人数并限制放弃虚拟试验,就像REMOTE研究中发生的那样,提出了奖励参与者的想法。所谓的虚拟游戏,例如由EmpiraMed测试,在这种情况下,在提出的特定情况下,患者获得的每个目标积分(注册,募集等)可用于礼品卡或捐赠给协会。 “这种方法– said Cottini –可能会引入道德评估,但这是要考虑的一点:我们在意大利的立法目前不允许这样做。在西奈山伊坎医学院研究患者参与度的Jason Bobe认为,主要问题是“行业仍然倾向于将个别患者视为案例研究,而不是积极参与者”。

FDA的观点

尽管FDA表示他们认为在临床试验中适当使用技术会有好处,但他们仍在学习虚拟临床试验,自备设备(BYOD)供应模型以及其他方面今天’s tech-enabled research environment. In particular, the agency is looking for input on four specific issues: how the FDA could encourage the adoption of 技术的 tools in clinical trials; what are the barriers; how the new research models will influence patients and the eventual limiting action of regulatory requirements. Not surprisingly, the FDA is looking for input, as supporting and encouraging innovation is the agency’的工作。为此,FDA发布的指导文件“在临床研究中使用电子知情同意书 ”解释了联邦监管机构如何允许公司使用电子媒体(例如交互式网站)来促进知情同意程序。这无疑将帮助公司进行虚拟临床试验。例如,FDA一直支持虚拟测试Transparency Life Sciences,这是一家位于波士顿的生物技术初创企业,最近在30天之内就获得了FDA批准的完全远程监控的测试协议,并受到鼓励在此方面进行更多的创新。虚拟实验的方向。

未来

展望未来,不同的情况似乎是合理的。也许虚拟研究会增加,而不是取代传统的研究实践和工作流程。研究’在需要时可以利用虚拟化方面的功能– similar to how 远程 monitoring streams are being adopted by the study supervision teams today. On-site monitoring is still a pillar of the study, for example, but much of the data is monitored 远程ly, depending on the case «CRO, emphasizes Cottini, and companies are already equipped to reduce (maybe not to zero) the monitoring at the experimental centers for a “physical”检查文件夹,报告和同意书(所谓的“source data”适用于GCP)»。也许虚拟临床试验将用于传统模型失败的地区(例如,针对地理上分散的群体或罕见疾病人群)。有趣的是,赛诺菲VERKKO的研究并未测试药物,而是专注于无线血糖仪。也许虚拟工作室将很适合评估传感器和诊断等医疗设备,随着技术的发展,其重要性将继续提高。作为辉瑞’经验告诉我们,这不是一条容易的路,但它可能为某些研究和选定的患者群体带来巨大的好处。

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