Determination of the target population in early benefit assessments in Germany: challenges for non-small-cell lung cancer.

OBJECTIVES: 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. METHODS: 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. RESULTS: 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. DISCUSSION: 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.

[Cost of illness of age-related macular degeneration. Systematic review on the development of a costs diary]. / Krankheitskosten der altersbedingten Makuladegeneration. Systematischer Review zur Entwicklung eines Kostentagebuchs.

Due to demographic changes in the German population a significant rise in incident cases of age-related macular degeneration (AMD) is to be expected in the coming years. Also the corresponding increase in costs of the illness requires appropriate survey instruments to allow accurate estimations of costs. The cost diary for patients with AMD developed in this study is based on a previously established systematic review. The seven studies that were included were evaluated regarding their quality in order to then extract the listed cost items. The result is a prospective survey instrument with six different cost sectors of AMD.