University of Groningen, Groningen Research Institute of Pharmacy, Groningen, The Netherlands.
Department of Health Sciences, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands.
Expert Opin Pharmacother. 2021 May;22(7):923-935. doi: 10.1080/14656566.2021.1873953. Epub 2021 Jan 13.
When estimating the cost-effectiveness or budget impact of chronic obstructive pulmonary disease (COPD) medication, it is common practice to use trial data for clinical inputs. However, such inputs do not always reflect the real-world situation. Previous reviews recognized the need for taking real-world data (medication adherence, comorbidity and adverse drug reactions [ADRs]) into account. Whether recent cost-effectiveness analyses of COPD medication implemented those recommendations is unknown.
The authors reviewed recent economic evaluations of COPD-maintenance treatments focusing on medication adherence, comorbidity and ADRs.
In most registration trials of COPD treatment, strict inclusion and exclusion criteria are applied. During trials, patient monitoring is well controlled. As such, medication adherence is often higher than seen in less controlled, real-world environments with more heterogeneous characteristics. Additionally, safety data collected in trials may not be widely generalizable due to more comorbidity and polypharmacy in the real-world. Consequently, when merely relying on trial data, the impact of adherence, comorbidity and ADRs on the cost-effectiveness can be underestimated. To overcome these real-world data gaps, use of pragmatic trials and observational studies in addition to strictly controlled trial data is recommended. To catalyze implementation of these real-world issues, reporting checklists should be updated.
在评估慢性阻塞性肺疾病（COPD）药物的成本效益或预算影响时，通常使用临床试验数据来估算临床参数。然而，这些输入并不总是反映真实世界的情况。先前的综述认识到需要考虑真实世界的数据（药物依从性、合并症和药物不良反应 [ADR]）。最近的 COPD 药物成本效益分析是否实施了这些建议尚不清楚。
作者回顾了最近关于 COPD 维持治疗的经济评估，重点关注药物依从性、合并症和 ADR。
在 COPD 治疗的大多数注册试验中，应用了严格的纳入和排除标准。在试验期间，患者监测得到很好的控制。因此，药物依从性往往高于在控制较差、特征更加异质的真实世界环境中观察到的水平。此外，由于真实世界中合并症更多且用药更复杂，试验中收集的安全性数据可能无法广泛推广。因此，如果仅仅依赖试验数据，可能会低估药物依从性、合并症和 ADR 对成本效益的影响。为了克服这些真实世界数据的差距，建议除了严格控制的试验数据外，还使用实用试验和观察性研究。为了推动这些真实世界问题的实施，应更新报告清单。
