Sturmberg Joachim P, Kühlein Thomas
College of Health, Medicine and Wellbeing, University of Newcastle, Newcastle, AUS.
Research, Central Coast Research Institute, Gosford, AUS.
Cureus. 2025 Feb 23;17(2):e79493. doi: 10.7759/cureus.79493. eCollection 2025 Feb.
A recent science communication meeting highlighted a common pitfall in scientific communication: the failure to link the "" - the findings - to the "" - their real-world implications. The real world is complex, and exploring the complexities of "living world phenomena" requires addressing the interconnectedness and interdependencies of the many variables that shape the patterned outcomes of patient conditions we see in everyday practice. While scientific methods by necessity must simplify complexities, these simplifications should be transparently communicated to foster trust and understanding. Randomised controlled trials (RCTs) aim to eliminate contextual confounders, producing statistically significant average outcomes for a hypothetical "average" patient. While they ensure high internal validity, RCTs often lack external validity, limiting their transferability to real-world practice, where patients differ from the average trial participant. This is an inherent problem of RCTs that cannot be overcome. What is not inherent and should be changed are the outcome elements of the study design and especially their reporting. To achieve "statistical significance", trials use large sample sizes, surrogate and arbitrarily designed composite endpoints, and typically emphasise relative benefits, obscuring absolute benefits, which are often clinically marginal. Transparent reporting of absolute benefits, contextualised to patients' realities, is crucial for informed, shared decision-making. Patients and clinicians alike must weigh small disease-specific benefits against potential harms, especially when interventions compromise overall well-being or ability to manage daily life circumstances. Transparency matters, it is a moral and ethical imperative. Applied to medical sciences, it is no longer acceptable to argue that the statistical significance of research findings justifies a tacit paternalism that undermines patient autonomy. We propose a transparency framework that could enhance clear and honest communication of research findings - this is crucial to empower both clinicians and patients in making well-informed clinical or public health decisions.
未能将“发现”与“它们在现实世界中的影响”联系起来。现实世界是复杂的，探索“生命世界现象”的复杂性需要解决许多变量的相互联系和相互依存关系，这些变量塑造了我们在日常实践中看到的患者病情的模式化结果。虽然科学方法必然要简化复杂性，但这些简化应该清晰地传达，以促进信任和理解。随机对照试验（RCT）旨在消除背景混杂因素，为假设的“平均”患者产生具有统计学意义的平均结果。虽然随机对照试验确保了较高的内部效度，但它们往往缺乏外部效度，限制了其向现实世界实践的可转移性，因为现实世界中的患者与平均试验参与者不同。这是随机对照试验无法克服的固有问题。研究设计的结果要素，尤其是其报告，并非固有且应该改变。为了达到“统计学意义”，试验使用大样本量、替代指标和随意设计的复合终点，并且通常强调相对益处，掩盖了绝对益处，而绝对益处往往在临床上微不足道。根据患者的实际情况透明地报告绝对益处，对于明智的、共同的决策至关重要。患者和临床医生都必须权衡特定疾病的微小益处与潜在危害，尤其是当干预措施损害总体幸福感或日常生活管理能力时。透明度很重要，这是道德和伦理的要求。应用于医学科学时，以研究结果的统计学意义为由，默认实行损害患者自主权的家长式作风已不再被接受。我们提出了一个透明度框架，该框架可以加强对研究结果的清晰和诚实的沟通——这对于使临床医生和患者都能做出明智的临床或公共卫生决策至关重要。
