Changing clinical practice in rare diseases through innovative trial designs: the CAPTIVATE node

我们将做什么：我们将创建一个研究人员小组，他们将开发更好的方法，在临床试验中为患有罕见疾病的人测试新药。我们希望找到一种方法，这种方法需要尽可能少的人参与，更快，并提供当局批准一种新药所需的所有信息，以便从一次试验中为罕见病患者开出处方。这组研究人员将被称为“俘虏节点”，患有罕见疾病的人将为这项研究做出贡献。为什么我们这样做：患有罕见疾病的人希望接受基于试验证据已被批准用于NHS的治疗。很难在罕见疾病中进行试验，因为可能只有少数患有这种疾病的人能够参加。也很难期望人们在随机对照试验中服用“虚拟”药物（安慰剂）。还有一种方法叫做贝叶斯临床试验（以科学家托马斯贝叶斯命名）。这种方法可能在罕见疾病的试验中有用，因为它更灵活。该方法使我们能够通过一次分析一名患者的结果来了解试验中正在测试的药物，而不是等待试验结束。这可以帮助减少需要参与的人数。该方法还允许我们查看关于正在测试的药物的任何现有信息。我们研究试验结果时可以用这个。我们将如何做到这一点：我们将开发在单一试验中测试药物的方法（一站式研究），一次性收集批准所需的所有数据。我们将探索如何最好地包含有关已知药物的信息。通过这种方式，试验结果更有可能被医疗保健专业人员、罕见疾病患者和制定新药审批规则的当局所接受。我们将在儿童中进行试验，这对于确保有效的药物迅速到达患有罕见疾病的儿童非常重要。可能有来自成人研究的信息也可能是有用的，并包括在儿童试验中，我们将对此进行调查。我们将尝试找到一些传统的“随机对照试验”的替代方案，例如使用受罕见疾病影响的人的信息，而不是要求他们服用安慰剂。我们将如何让患者和公众参与：我们将与英国遗传联盟、英国Alstrom综合征和其他患者支持团体合作，确保罕见病患者（PLWRC）的声音被纳入研究的总体方向。我们将邀请有耐心的合作伙伴参加我们的定期项目管理会议，我们将成立一个PLWRC咨询小组，在研究开始和研究期间提供指导。解释我们的工作：CAPTIVATE节点将汇集英国领先的试验专家、在罕见病领域经验丰富的医院研究人员、行业合作伙伴、政策制定者和患者合作伙伴，制定和讨论临床试验设计，以加快罕见病药物的审批。这个专家网络将开发新的设计和指导方针，我们相信这将改善罕见疾病患者的健康状况。

What we will do:We will create a group of researchers that will develop better ways to test new medicines for people with rare diseases in clinical trials. We want to find ways that need as few people to take part as possible, that are quicker and that provide all the information needed for the authorities to approve a new medicine to be prescribed to people with rare diseases from one single trial. The group of researchers will be called 'the CAPTIVATE node' and people living with rare conditions will contribute to the research. Why we are doing this:People with rare diseases expect to receive treatments which have been approved for use in the NHS based on evidence from trials. It is hard to do trials in rare diseases because there may only be a few people affected with the disease who are able to take part. It is also hard to expect people to take a 'dummy' medicine (placebo) as part of randomised controlled trials. There is another method called Bayesian clinical trials (named after a scientist called Thomas Bayes). This method may be useful in trials in rare diseases because it is more flexible. The method allows us to learn about medicines being tested in the trial by analysing results from one patient at a time rather than waiting to the end of the trial. This can help reduce the number of people needed to take part. The method also allows us to look at any information that already exists about the medicine being tested. We can use this when we study the results of the trial. How we will do it:We will develop ways to test medicines in a single trial (a 'one-stop-study') that collects all the data needed for approval in one go. We will explore how best to include the information about a medicine that is already known. This way, trial results are more likely to be accepted by healthcare professionals, people living with rare diseases and the authorities who set the rules for approving new medicines. We will work on trials in children, where it is so important to make sure medicines that work get to children with rare diseases quickly. There may be information from studies in adults that can also be useful and included in trials for children, and we will investigate this. We will try to find some alternatives to the traditional 'randomised controlled trial' such as using information from people affected by rare diseases instead of asking them to take a placebo. How we will involve patients and the public: We will work with Genetic Alliance UK, Alstrom Syndrome UK and other patient support groups to ensure the voice of people living with rare conditions (PLWRC) is included in the overall direction of the research. There will be patient partners invited to our regular project management meetings and we will set up an PLWRC advisory group that will provide guidance at the start and during the research.Explaining our work: The CAPTIVATE node will bring together the UK's leading trial experts with hospital researchers experienced in rare diseases, industry partners, policy makers and patient partners, to develop and discuss clinical trial designs that could speed up the approval of medicines for use in rare diseases. This network of experts will develop new designs and guidelines that we believe will improve the health of people living with rare diseases.