PDF(Pubmed)
Abstract：
Notwithstanding two decades of policy and legislation in Europe, aimed to foster research and development in rare conditions, only 5-6% of rare diseases have dedicated treatments. Given with the huge number of conditions classed as rare (which is increasing all the time), this equates to major unmet need for patients (over 30 million in the EU alone). Worryingly, the pace of Research and Innovation in Europe is lagging behind other regions of the world, and a seismic shift in the way in which research is planned and delivered is required, in order to remain competitive and-most importantly-bring meaningful, disease-altering treatments to those who desperately need them. The European Reference Networks (ERNs), launched in 2017, hold major potential to alleviate many of these challenges, and more, but only if adequately supported (financially, technically, and via robust policies and infrastructure) to realise that potential: and even then, only if able to forge robust collaborations harnessing the expertise, resources, knowledge and data of all stakeholders involved in rare disease, including Industry. To-date, however, ERN-Industry interactions have been largely limited, for a range of reasons (concerning barriers both tangible and perceived). This Position Statement analyses these barriers, and explains how Together4RD is seeking to move the needle here, by learning from case studies, exploring frameworks for collaboration, and launching pilots to explore how best to plan and deliver multistakeholder interactions addressing real research needs.
摘要：
尽管欧洲有二十年的政策和立法，旨在促进罕见条件下的研究和开发，只有5-6％的罕见疾病有专门的治疗方法。考虑到大量被归类为罕见的情况(这种情况一直在增加)，这相当于患者的主要未满足需求（仅欧盟就超过3000万）。令人担忧的是,欧洲的研究和创新步伐落后于世界其他地区，并且需要在计划和交付研究的方式上发生地震转变，为了保持竞争力，最重要的是带来有意义的，改变疾病的治疗那些谁迫切需要他们。欧洲参考网络(ERN)于2017年推出，具有缓解许多这些挑战的巨大潜力，还有更多,但前提是有足够的支持(财政上，技术上,并通过强有力的政策和基础设施)来实现这一潜力：即使如此，只有能够利用专业知识建立强大的合作关系，资源,涉及罕见疾病的所有利益相关者的知识和数据，包括工业。到目前为止，然而,ERN-行业互动在很大程度上受到限制，出于一系列原因(涉及有形和感知的障碍)。这份立场声明分析了这些障碍，并解释了Together4RD如何在这里移动针头，通过从案例研究中学习，探索合作框架，并启动试点，以探索如何最好地计划和交付多利益相关者的互动，以解决真正的研究需求。
