Cancer Research UK Clinical Trials Unit, University of Birmingham, Birmingham, UK.
Cancer Research UK Clinical Trials Unit, University of Birmingham, Birmingham, UK.
Lancet Oncol. 2016 Feb;17(2):e70-e80. doi: 10.1016/S1470-2045(15)00396-4.
Rare cancers are a growing group as a result of reclassification of common cancers by molecular markers. There is therefore an increasing need to identify methods to assess interventions that are sufficiently robust to potentially affect clinical practice in this setting. Methods advocated for clinical trials in rare diseases are not necessarily applicable in rare cancers. This Series paper describes research methods that are relevant for rare cancers in relation to the range of incidence levels. Strategies that maximise recruitment, minimise sample size, or maximise the usefulness of the evidence could enable the application of conventional clinical trial design to rare cancer populations. Alternative designs that address specific challenges for rare cancers with the aim of potentially changing clinical practice include Bayesian designs, uncontrolled n-of-1 trials, and umbrella and basket trials. Pragmatic solutions must be sought to enable some level of evidence-based health care for patients with rare cancers.
由于常见癌症通过分子标志物进行重新分类，因此罕见癌症的数量不断增加。因此，需要确定方法来评估干预措施，这些措施必须足够稳健，有可能影响这一领域的临床实践。在罕见疾病临床试验中提倡的方法不一定适用于罕见癌症。本系列论文描述了与发病率水平范围相关的罕见癌症的相关研究方法。旨在最大限度地提高招募效率、最小化样本量或最大限度地提高证据有用性的策略，可以使传统临床试验设计应用于罕见癌症人群。旨在潜在改变临床实践的、针对罕见癌症特定挑战的替代设计包括贝叶斯设计、非对照的 n-of-1 试验以及伞式和篮子试验。必须寻求实用的解决方案，以使罕见癌症患者能够获得某种基于证据的医疗保健。
