BresMed Health Solutions, Steel City House, West Street, Sheffield, UK.
Department of Economics, University of Sheffield, Sheffield, UK.
Stat Methods Med Res. 2020 Jun;29(6):1563-1572. doi: 10.1177/0962280219826609. Epub 2019 Jan 30.
Where medical interventions are licensed based on only uncontrolled study data (for example a single-arm trial), a common approach for estimating the incremental benefit is to compare the treatment to a 'historical control'; data collected from patients who did not receive the intervention. We illustrate with motivating examples two methods for the creation of historical controls where disease progression and overall survival are typically the key clinically meaningful endpoints. The first method utilises information routinely collected in a clinical trial programme: patients' time to disease progression on their previous line of treatment against which outcomes can be compared. The second uses published clinical outcomes for the prior line of treatment which can be extrapolated to estimate outcomes at the next line. As examples we use two pharmaceuticals licensed on the basis of uncontrolled clinical studies - idelalisib for double-refractory follicular lymphoma and ofatumumab for double-refractory chronic lymphocytic leukemia. Although subject to limitations that should be considered on a case-by-case basis, the methods may be appropriate when trying to quantify the clinical benefit of treatment based on limited and uncontrolled trial data. As a result, the methods can be used to inform health technology adoption decisions.
在仅基于非对照研究数据（例如单臂试验）许可医疗干预措施的情况下，估计增量效益的一种常见方法是将治疗方法与“历史对照”进行比较；这些数据是从未接受干预的患者中收集的。我们用两个有说服力的例子来说明在疾病进展和总生存期通常是关键临床有意义终点的情况下创建历史对照的两种方法。第一种方法利用临床试验计划中常规收集的信息：患者在前一线治疗中的疾病进展时间，可据此比较结果。第二种方法使用已发表的前一线治疗的临床结果，可以外推到估计下一线的结果。作为例子，我们使用两种基于非对照临床试验许可的药物 -idelalisib 用于双重难治性滤泡性淋巴瘤和 ofatumumab 用于双重难治性慢性淋巴细胞白血病。尽管存在应根据具体情况考虑的限制，但当试图根据有限的和不受控制的试验数据来量化治疗的临床效益时，这些方法可能是合适的。因此，这些方法可用于为医疗技术采用决策提供信息。
