人们对使用现实世界数据(RWD)来解决临床和政策相关(研究)问题越来越感兴趣,这些问题无法仅通过随机对照试验(RCT)的数据来回答。这是,例如,如肉瘤等罕见恶性肿瘤的病例,由于患者数量有限,在可行的及时性内进行随机对照试验面临挑战,可管理数量的合作者,统计力量。这篇叙述性综述探讨了RWD在肉瘤研究中产生真实世界证据(RWE)的潜力,阐明其在患者旅程的不同阶段的应用,从诊断前到随访/生存阶段。例如,检查全科医生(GP)的电子健康记录(EHR)可以探索咨询频率,并在肉瘤诊断前在初级保健中出现症状。此外,将RWD与精心设计的观察性RCT相结合的替代研究设计可能提供有关临床治疗有效性的相关信息.As,特别是在超级肉瘤的情况下,进行有效的随机前瞻性研究可能是一个极端的挑战.因此,支持适应新颖的研究设计是至关重要的。关于随访/生存阶段,检查初级和二级保健的EHR可以为确定长期随访期间治疗的短期和长期影响提供有价值的见解.RWD的使用也带来了一些挑战,包括与数据质量和隐私相关的问题,正如这项研究中所描述的。尽管面临这些挑战,这项研究强调了RWD桥梁的潜力,至少部分地,证据与实践之间的差距,并有望为改善肉瘤护理做出贡献。
There has been growing interest in the use of real-world data (RWD) to address clinically and policy-relevant (research) questions that cannot be answered with data from randomized controlled trials (RCTs) alone. This is, for example, the case in rare malignancies such as sarcomas as limited patient numbers pose challenges in conducting RCTs within feasible timeliness, a manageable number of collaborators, and statistical power. This narrative review explores the potential of RWD to generate real-world evidence (RWE) in
sarcoma research, elucidating its application across different phases of the patient journey, from prediagnosis to the follow-up/survivorship phase. For instance, examining electronic health records (EHRs) from general practitioners (GPs) enables the exploration of consultation frequency and presenting symptoms in primary care before a
sarcoma diagnosis. In addition, alternative study designs that integrate RWD with well-designed observational RCTs may offer relevant information on the effectiveness of clinical treatments. As, especially in cases of ultrarare sarcomas, it can be an extreme challenge to perform well-powered randomized prospective studies. Therefore, it is crucial to support the adaptation of novel study designs. Regarding the follow-up/survivorship phase, examining EHR from primary and secondary care can provide valuable insights into identifying the short- and long-term effects of treatment over an extended follow-up period. The utilization of RWD also comes with several challenges, including issues related to data quality and privacy, as described in this study. Notwithstanding these challenges, this study underscores the potential of RWD to bridge, at least partially, gaps between evidence and practice and holds promise in contributing to the improvement of
sarcoma care.