Thalassemia management has reached new milestones, with new therapies promising the dawning of a new era. However, conventional and new approaches require accessibility, affordability, acceptability/adherence by patients, and medical expertise from healthcare providers. Current treatments still do not offer the expected duration and quality of life, and inequalities in patient care are almost a universal phenomenon. To understand the requirements to achieve improved care, including the adoption of new therapies, for the maximum number of the global patient population, it is necessary to recognize the weaknesses that are experienced in the present so that future corrective action can be taken. Deficits in service provision are due to poor political and financial support, lack of prioritization during resource rationing, and absence of epidemiological information for policy making. These system weaknesses require improved resource management and would benefit from patient support organizations, improved psychosocial support and patient welfare, and an increase in professional expertise through educational programs. Medical products and technology must also be made affordable and widely available, and the curative treatments and cheaper approaches to technology must be recognized as resource saving. Improvements in the access to innovative and quality care, and even a cure, require concerted actions by all stakeholders, including physicians and the patient community.

中文翻译：

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β地中海贫血：展望未来，解决未满足的需求和挑战

地中海贫血治疗已达到新的里程碑，新疗法有望开启新时代。然而，传统和新的方法需要患者的可及性、可负担性、可接受性/依从性以及医疗保健提供者的医疗专业知识。目前的治疗方法仍然无法提供预期的持续时间和生活质量，患者护理的不平等几乎是一种普遍现象。为了了解为全球最大数量的患者群体实现改善护理的要求，包括采用新疗法，有必要认识到当前所经历的弱点，以便在未来采取纠正措施。服务提供方面的赤字是由于政治和财政支持不足、资源配给期间缺乏优先顺序以及缺乏用于决策的流行病学信息造成的。这些系统的弱点需要改进资源管理，并将受益于患者支持组织、改善的社会心理支持和患者福利以及通过教育计划增加专业知识。医疗产品和技术还必须变得负担得起并广泛使用，并且必须将治疗方法和更便宜的技术方法视为资源节约型。改善获得创新和优质护理甚至治愈的机会需要包括医生和患者群体在内的所有利益相关者采取协调一致的行动。