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Abstract：
Adeno-associated virus-based gene therapy points to a coming transformation in the treatment of people living with haemophilia, promising sustained bleed control and potential improvement in quality of life. Nevertheless, the consequences of introducing new genetic material are not trivial. The perceived benefits should not minimise the challenges facing patients in understanding the long-term risks and providing a valid and meaningful informed consent, whether in a research or clinical setting. Informed consent is a fundamentally important doctrine in both medical ethics and health law, upholding an individual\'s right to define their personal goals and make their own autonomous choices. Patients should be enabled to recognise their clinical situation, understand the implications of treatment and integrate every facet of their life into their decision. This review describes informed consent processes for haemophilia gene therapy clinical trials, factors affecting patients\' decision making and the availability of patient-centred decision support interventions, to ensure that patients\' interests are being protected. Regulatory guidance has been published for physicians and manufacturers in haemophilia on informed consent, including for gene therapy, while best-practice recommendations for patient-physician discussions are available. In all settings, however, communicating and presenting highly technical and complex therapeutic information is challenging, especially where multiple barriers to scientific knowledge and health literacy exist. We propose several evidence-informed strategies to enhance the consent procedure, such as utilising validated literacy and knowledge assessment tools as well as participatory learning environments over an extended period, to ensure that patients are fully cognisant of the consent they give or deny. Further research is needed to define new, creative approaches for patient education and the upholding of ethical values in the informed consent process for gene therapy. The lessons learnt and approaches developed within haemophilia could set the gold standard for good practice in ensuring ethical preparedness amidst advances in genetic therapies.
UNASSIGNED: Improving the informed consent process for people living with haemophilia considering gene therapy. Gene therapy is the process of replacing faulty genes with healthy ones. In haemophilia, gene therapy involves introducing a working copy of the gene for the clotting factor that patients are missing. Following treatment, patients should begin producing their own clotting factor normally. However, people living with haemophilia (PwH) need to be fully informed regarding the potential benefits and risks of gene therapy and what this means for them, whether as part of a research study or routine medical care.Patients must be respected and supported to make decisions about their own health and wellbeing, recognising their legal and moral right to set personal goals and make treatment choices. For this to happen in practice, patients should be aware of their individual health needs, understand the effects of treatment and consider lifestyle preferences in relation to their decisions. This article attempts to describe how informed consent is obtained in haemophilia gene therapy clinical trials, what affects a patient\'s ability to make decisions and the availability of information and support to respect and protect the interests of PwH.Regulators responsible for approving medical products have published guidance on informed consent for physicians and pharmaceutical manufacturers in haemophilia, including for gene therapy. Recommendations have been made about the best ways for PwH to discuss gene therapy with their physicians. Yet, poor communication of complex topics, such as gene therapy, can be problematic, especially if patients lack the skills and confidence to understand and discuss the science, or for physicians with limited time in clinic.We propose strategies to improve the consent process, so patients can feel more able to make informed decisions about new treatments. Further research is needed to find new, creative approaches for educating patients and ensuring that the informed consent process for gene therapy in haemophilia is ethical.
UNASSIGNED: Improving the informed consent process for people living with haemophilia considering gene therapy. Gene therapy is the process of replacing faulty genes with healthy ones. In haemophilia, gene therapy involves introducing a working copy of the gene for the clotting factor that patients are missing. Following treatment, patients should begin producing their own clotting factor normally. However, people living with haemophilia (PwH) need to be fully informed regarding the potential benefits and risks of gene therapy and what this means for them, whether as part of a research study or routine medical care.Patients must be respected and supported to make decisions about their own health and wellbeing, recognising their legal and moral right to set personal goals and make treatment choices. For this to happen in practice, patients should be aware of their individual health needs, understand the effects of treatment and consider lifestyle preferences in relation to their decisions. This article attempts to describe how informed consent is obtained in haemophilia gene therapy clinical trials, what affects a patient\'s ability to make decisions and the availability of information and support to respect and protect the interests of PwH.Regulators responsible for approving medical products have published guidance on informed consent for physicians and pharmaceutical manufacturers in haemophilia, including for gene therapy. Recommendations have been made about the best ways for PwH to discuss gene therapy with their physicians. Yet, poor communication of complex topics, such as gene therapy, can be problematic, especially if patients lack the skills and confidence to understand and discuss the science, or for physicians with limited time in clinic.We propose strategies to improve the consent process, so patients can feel more able to make informed decisions about new treatments. Further research is needed to find new, creative approaches for educating patients and ensuring that the informed consent process for gene therapy in haemophilia is ethical.
摘要：
基于腺相关病毒的基因治疗表明血友病患者的治疗即将发生转变，有希望的持续出血控制和生活质量的潜在改善。然而，引入新的遗传物质的后果并不微不足道。感知的益处不应最小化患者在理解长期风险和提供有效和有意义的知情同意方面面临的挑战。无论是在研究还是临床环境中。知情同意是医学伦理学和卫生法中的一个根本重要学说,维护个人定义个人目标和自主选择的权利。患者应该能够认识到他们的临床情况，了解治疗的影响，并将他们生活的方方面面融入他们的决定。这篇综述描述了血友病基因治疗临床试验的知情同意过程，影响患者决策的因素和以患者为中心的决策支持干预措施的可用性，确保患者的利益得到保护。在知情同意的情况下，已经为血友病的医生和制造商发布了监管指南，包括基因疗法,而患者-医生讨论的最佳实践建议是可用的。在所有设置中，然而,交流和呈现高度技术性和复杂的治疗信息是具有挑战性的，尤其是在科学知识和健康素养存在多重障碍的地方。我们提出了几种循证策略来加强同意程序，例如在很长一段时间内利用经过验证的识字和知识评估工具以及参与式学习环境，以确保患者完全了解他们给予或拒绝的同意。需要进一步的研究来定义新的，在基因治疗的知情同意过程中，对患者进行教育和维护道德价值观的创造性方法。在血友病中吸取的教训和发展的方法可以为良好做法设定黄金标准，以确保在基因疗法的进步中做好道德准备。
■改善考虑基因治疗的血友病患者的知情同意程序。基因治疗是用健康的基因取代有缺陷的基因的过程。在血友病中，基因治疗包括为患者缺失的凝血因子引入基因的工作拷贝。治疗后,患者应该开始正常生产自己的凝血因子。然而,血友病（PwH）患者需要充分了解基因治疗的潜在益处和风险，以及这对他们意味着什么，无论是作为研究研究还是常规医疗的一部分。必须尊重和支持患者对自己的健康和福祉做出决定，承认他们设定个人目标和做出治疗选择的法律和道德权利。为了在实践中发生这种情况，患者应该意识到他们个人的健康需求，了解治疗的效果，并考虑与他们的决定相关的生活方式偏好。本文试图描述如何在血友病基因治疗临床试验中获得知情同意，是什么影响患者的决策能力以及信息和支持的可用性，以尊重和保护PwH的利益。负责批准医疗产品的监管机构已经发布了关于血友病医生和药品制造商知情同意的指南，包括基因治疗.已经就PwH与他们的医生讨论基因治疗的最佳方法提出了建议。然而，对复杂主题的沟通不畅，比如基因治疗，可能会有问题，特别是如果患者缺乏理解和讨论科学的技能和信心，或在临床上时间有限的医生。我们提出改善同意程序的策略，这样患者就能更有能力对新疗法做出明智的决定。需要进一步的研究来找到新的，对患者进行教育的创造性方法，并确保血友病基因治疗的知情同意过程符合道德。
■改善考虑基因治疗的血友病患者的知情同意程序。基因治疗是用健康的基因取代有缺陷的基因的过程。在血友病中，基因治疗包括为患者缺失的凝血因子引入基因的工作拷贝。治疗后,患者应该开始正常生产自己的凝血因子。然而,血友病（PwH）患者需要充分了解基因治疗的潜在益处和风险，以及这对他们意味着什么，无论是作为研究研究还是常规医疗的一部分。必须尊重和支持患者对自己的健康和福祉做出决定，承认他们设定个人目标和做出治疗选择的法律和道德权利。为了在实践中发生这种情况，患者应该意识到他们个人的健康需求，了解治疗的效果，并考虑与他们的决定相关的生活方式偏好。本文试图描述如何在血友病基因治疗临床试验中获得知情同意，是什么影响患者的决策能力以及信息和支持的可用性，以尊重和保护PwH的利益。负责批准医疗产品的监管机构已经发布了关于血友病医生和药品制造商知情同意的指南，包括基因治疗.已经就PwH与他们的医生讨论基因治疗的最佳方法提出了建议。然而，对复杂主题的沟通不畅，比如基因治疗，可能会有问题，特别是如果患者缺乏理解和讨论科学的技能和信心，或在临床上时间有限的医生。我们提出改善同意程序的策略，这样患者就能更有能力对新疗法做出明智的决定。需要进一步的研究来找到新的，对患者进行教育的创造性方法，并确保血友病基因治疗的知情同意过程符合道德。
