Ressort Versorgung und Gesundheitsökonomie/Dept. of Health Care and Health Economics, Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWiG), Im Mediapark 8, 50670, Köln, Germany.
Eur J Health Econ. 2020 Aug;21(6):881-893. doi: 10.1007/s10198-020-01180-1. Epub 2020 Mar 31.
Dossiers submitted for early benefit assessments in Germany also provide information on the precise determination of the target population (patients eligible for a drug). The situation is complex for non-small-cell lung cancer (NSCLC) due to highly specific therapeutic indications. Our aim was to compare the different methodological steps applied to determine the target population in dossiers on drugs for NSCLC.
We analysed NSCLC dossiers assessed by the German Institute for Quality and Efficiency in Health Care (IQWiG) between 01.01.2011 and 31.12.2017. Methodological details regarding the determination of the target population were extracted and compared.
We analysed 23 NSCLC dossiers. In all dossiers, the target population was determined using the number of all patients with lung cancer as the basis for calculations. This patient population was further reduced in several successive steps by assuming proportions of patients with a specific characteristic (e.g. disease stage). The most important calculation steps were patients with NSCLC (n = 23 dossiers), with a specific disease stage (n = 23), with a specific tumour mutation (n = 14), with a specific tumour histology (n = 7), without prior treatment (n = 15), with pretreatment in second or further treatment lines (n = 17), and/or with specific pretreatments (n = 9). The proportions of patients determined within the same calculation step varied considerably between dossiers.
The calculation methods applied and the target population sizes reported in NSCLC dossiers vary considerably. A consensus with regard to the databases and calculation methods used to determine the target population in NSCLC would be helpful to reduce variations.
在德国提交的早期获益评估文件还提供了有关目标人群（有资格使用药物的患者）的精确确定的信息。由于治疗的高度特异性，非小细胞肺癌（NSCLC）的情况比较复杂。我们的目的是比较用于确定 NSCLC 药物文件中目标人群的不同方法步骤。
我们分析了德国卫生保健质量和效率研究所（IQWiG）在 2011 年 1 月 1 日至 2017 年 12 月 31 日期间评估的 23 份 NSCLC 文件。提取并比较了关于确定目标人群的方法学细节。
我们分析了 23 份 NSCLC 文件。在所有文件中，均使用所有肺癌患者的数量作为计算基础来确定目标人群。通过假设具有特定特征（例如疾病阶段）的患者比例，该患者人群在几个连续步骤中进一步减少。最重要的计算步骤是 NSCLC 患者（n=23 份文件）、特定疾病阶段（n=23）、特定肿瘤突变（n=14）、特定肿瘤组织学（n=7）、无前期治疗（n=15）、前期治疗在二线或进一步治疗线（n=17）和/或特定预处理（n=9）。在同一计算步骤中确定的患者比例在不同文件之间差异很大。
在 NSCLC 文件中应用的计算方法和报告的目标人群规模差异很大。在 NSCLC 中确定目标人群时，就使用的数据库和计算方法达成共识将有助于减少差异。
