Regenerative medicine and its development and implementation: an analysis of emergent value systems and health service readiness

近年来，人们对基于“再生医学”的疗法产生了相当大的兴趣，这些疗法已经成为或可能成为现实。这是临床研究中一个重要的新兴领域，也是在英国和国际上吸引了大量投资的领域。主要开发领域包括基因治疗药物产品、细胞治疗药物产品和组织工程产品。它的重要性在于，它承诺通过体内组织的再生提供根治性的、而不是姑息的疗法，从而改变医疗保健。这本身可能会重塑医疗保健服务，尽管理解现有医疗保健系统将如何影响该领域发展的途径也同样重要。此外，该领域给监管机构带来了新的挑战，因为组织/细胞疗法的“行动模式”与监管机构必须理解和批准(或不批准)的最常见创新--新药--截然不同。简而言之，该项目寻求了解这些临床问题与更广泛的社会、组织、监管和经济问题之间的复杂关系。它通过一系列工作包(WP)做到这一点。第一张地图描绘了英国的领域(在其更广泛的国际背景下--主要是欧盟/美国)，特别是关于当前的投资模式、临床试验、产品开发、公司的知识产权战略以及塑造这些战略的具体监管程序。第二个WP审查了一系列中介机构的作用，如国家健康和临床卓越研究所(NICE)、患者团体和医疗保险公司，它们在确定什么可以被称为该领域的“医疗准备”方面发挥了作用，即该领域如何与现有实践保持一致并可以嵌入其中，以及需要做出多大程度的改变。第三个WP涉及目前从事开发/使用细胞疗法的公司和医院网站的详细案例研究，选择这些案例以反映a)按产品领域进行的不同监管分类b)按疾病密切相关的当前活动的主要领域，以及我们将访问哪些网站来跟踪产品开发。我们将使用一种名为生命科学创新系统分析(ALSIS)的建模方法，该方法捕捉不同类型的商业模式、价值链及其与更广泛的创新环境的关系。这使我们能够在决定创新规模和范围的更广泛的经济、法规、社会和政治背景下，检查将产品从概念推向市场或直接在医院应用所需的全部活动。在第四个WP中，允许我们绘制和解释创新路径的ALSIS方法将与我们对该领域潜在未来的模型进行整合并进行三角剖分，使用由国家卫生研究所地平线扫描中心的共同申请者之一开发的水平扫描技术，以询问医疗保健中可能的扩散路径，以及公共和私人行为者(包括政府和私营机构)对再生医学的期望。在整个项目中，我们将与协作方合作，包括参与的公司、医院、监管和报销机构(特别是药品和保健产品监管机构和NICE)、患者团体、英国研究理事会、技术战略委员会的细胞治疗弹射器、NIHR的i4i秘书处、科学和技术选择委员会以及跨党派议会社会科学和政策小组。

There has been considerable interest developing over recent years in regard to the therapies that have become and may become available based on what is known as 'regenerative medicine'. This is an important emerging field in clinical research, and one that has attracted considerable investment in the UK and internationally. The main areas of development include gene therapy medicinal products, cell therapy medicinal products and tissue engineered products. Its importance lies in its promise to transform healthcare by offering curative, not palliative, therapeutics through the regeneration of tissue within the body. This in itself may reshape healthcare provision though it is equally important to understand how the existing healthcare system will influence the pathways through which the field develops. In addition, the field poses new challenges for regulators because of the ways in which the 'mode of action' of tissue/cell therapies is quite different from the most common innovation the regulator has to understand and approve (or not), the new pharmaceutical drug. In short, the project seeks to understand the complex relationship between these clinical and wider social, organisational, regulatory and economic issues. It does so through a series of Workpackages (WP). The first maps the field in the UK (within its wider international - principally EU/US context), especially in regard to current patterns of investment, clinical trials, product development, the intellectual property strategies of companies and the specific regulatory processes shaping these. The second WP examines the role of a range of intermediary agencies, such as the National Institute for Health and Clinical Excellence (NICE), patient groups and health insurance companies, in determining what can be called 'healthcare readiness' for the field, that is, how the field aligns with and can be embedded in existing practice and how far changes need to be made. The third WP involves detailed case studies of firms and hospital sites currently engaged in developing/using cell therapies chosen to reflect a) different regulatory classifications by product area b) the main areas of current activity by disease that are closely linked, and the sites that we would access to track product development. We will use a methodology called Analysis of Life Science Innovation Systems (ALSIS) modelling which captures different types of business models, value-chains and their relationship to the wider innovation environment. This allows us to examine the full range of activities required to bring a product from conception to market or to direct application in a hospital within the wider economic, regulatory, societal and political contexts that play a key role in determining the scale and scope of the innovation. In the fourth WP the ALSIS method which allows us to map and explain innovation paths will be integrated with and triangulated against our modelling of the potential future of the field using horizon scanning techniques developed by one of the co-applicants based at the National Institute of Health Research Horizon Scanning Centre to interrogate likely diffusion paths within health care, and the expectations surrounding regenerative medicine among public and private actors (including government and private agencies). Throughout the project we will work with collaborating parties, including the participating companies, hospitals, regulatory and reimbursement agencies (notably the Medicines and Healthcare products Regulatory Agency and NICE), patient groups, Research Councils UK, the Technology Strategy Board's Cell Therapy Catapult, the NIHR's i4i Secretariat, the Science and Technology Select Committee and the All-Party Parliamentary Group for Social Science and Policy.