FUTURE TARGETED HEALTHCARE MANUFACTURING HUB

到2025年，个性化和分层的靶向生物药物将改变医疗处方的精确度，改善患者护理和生活质量。如果要实现这一点，就必须创造新的制造解决方案。这是我们要应对的独特挑战。目前“一刀切”的药物开发方法正受到挑战，因为越来越多的人有能力只针对那些最有可能反应良好的患者进行治疗(分层药物)，甚至为每个人创造治疗方法(个性化药物)。在过去的十年里，遗传学和分子生物学的革命性进步改变了我们对疾病本质的理解。针对特定生物标记物的药物治疗将越来越多地只针对被确认为具有这些生物标记物的患者群体，使用配套的诊断或基因筛查测试；因此，分层医学成为可能。对于某些适应症，工程细胞和基因疗法提供了真正个性化医学的希望，其中治疗本身至少部分来自于个体患者。未来需要供应更多的药物产品，每种产品都针对相对较少的患者群体。目前，缺乏现有的技术和基础设施来做到这一点，目前的方法将是不可持续的。这些和其他新兴的先进疗法将在精准靶向药物的新时代发挥关键作用。对于处于极端财务压力下的医疗体系来说，所有这些都将不得不在经济上实现。这对健康和英国社会福祉的影响是深远的。市场上已经有少量靶向疗法，包括针对带有HER2受体的乳腺癌患者的赫赛汀，以及针对急性淋巴细胞白血病的转基因T细胞疗法。在接下来的十年里，将开发更多的靶向疗法，其中一些针对目前无法治愈的疾病。为了应对，该行业将需要创建更智能的系统，用于生产和供应日益分散的市场，并向其他行业学习。概念将需要解决复杂产品、能够小规模制造的工艺和设施以及具有应对波动需求和高度不确定性的敏捷性的供应链带来的具体挑战。创新的生物处理模式目前不适用于大规模制造，可能会取代分层药物的传统制造路线，同时减少工艺开发时间。减少开发成本和时间、提高制造效率和控制供应成本的压力将是巨大的，并可能成为商业化的差异化因素。我们将创造所需的技术、技能和训练有素的人员，使英国制造商能够实现先进的医疗精度和患者筛查的承诺。未来目标医疗制造中心及其研究和平移轮辐将与工业用户联网，以创造和应用必要的新工艺开发和制造方法。Hub Tools将改变供应链经济学，以实现有针对性的医疗保健，并为分层和个性化药物提供新的制造、配方和控制技术。该中心将预示着制造实践的转变，提供可持续医疗所需的工程基础设施。英国经济和社会福祉将从增强的国际竞争力中受益。

By 2025 targeted biological medicines, personalised and stratified, will transform the precision of healthcare prescription, improve patient care and quality of life. Novel manufacturing solutions have to be created if this is to happen. This is the unique challenge we shall tackle. The current "one-size-fits-all" approach to drug development is being challenged by the growing ability to target therapies to only those patients most likely to respond well (stratified medicines), and to even create therapies for each individual (personalised medicines). Over the last ten years our understanding of the nature of disease has been transformed by revolutionary advances in genetics and molecular biology.Increasingly, treatment with drugs that are targeted to specific biomarkers, will be given only to patient populations identified as having those biomarkers, using companion diagnostic or genetic screening tests; thus enabling stratified medicine. For some indications, engineered cell and gene therapies are offering the promise of truly personalised medicine, where the therapy itself is derived at least partly from the individual patient. In the future the need will be to supply many more drug products, each targeted to relatively small patient populations. Presently there is a lack of existing technology and infrastructure to do this, and current methods will be unsustainable. These and other emerging advanced therapies will have a critical role in a new era of precision targeted-medicines. All will have to be made economically for healthcare systems under extreme financial pressure. The implications for health and UK society well-being are profoundThere are already a small number of targeted therapies on the market including Herceptin for breast cancer patients with the HER2 receptor and engineered T-cell therapies for acute lymphoblastic leukaemia. A much greater number of targeted therapies will be developed in the next decade, with some addressing diseases for which there is not currently a cure. To cope, the industry will need to create smarter systems for production and supply to increasingly fragmented markets, and to learn from other sectors. Concepts will need to address specific challenges presented by complex products, of processes and facilities capable of manufacture at smaller scales, and supply chains with the agility to cope with fluctuating demands and high levels of uncertainty.Innovative bioprocessing modes, not currently feasible for large-scale manufacturing, could potentially replace traditional manufacturing routes for stratified medicines, while simultaneously reducing process development time. Pressure to reduce development costs and time, to improve manufacturing efficiency, and to control the costs of supply, will be significant and will likely become the differentiating factor for commercialisation.We will create the technologies, skill-sets and trained personnel needed to enable UK manufacturers to deliver the promise of advanced medical precision and patient screening. The Future Targeted Healthcare Manufacturing Hub and its research and translational spokes will network with industrial users to create and apply the necessary novel methods of process development and manufacture. Hub tools will transform supply chain economics for targeted healthcare, and novel manufacturing, formulation and control technologies for stratified and personalised medicines. The Hub will herald a shift in manufacturing practice, provide the engineering infrastructure needed for sustainable healthcare. The UK economy and Society Wellbeing will gain from enhanced international competitiveness.