OBJECTIVES: In recent years, more combination therapies emerged in the field of oncology, which contain a wide variety of drug combinations to increase effectiveness. However, this is controversially discussed – also with regard to the costs generated as a result. We aimed to conduct a systematic analysis of the oncological combination therapies investigated so far in the early benefit assessment.

METHODS: We conducted a systematic extraction of the time course and the additional benefits assigned by the pharmaceutical companies, the Institute for Quality and Efficiency in Health Care (IQWiG) and the Federal Joint Committee (G-BA). One focus was the extraction of reasons for added benefit/no-added benefit, taking methodological aspects and challenges into account. Success factors and future trends were additionally delineated.

RESULTS: 74 oncological combination therapies were evaluated since 2011 until February 28, 2023. Nivolumab and Pembrolizumab accounted for 11 of these, the highest number of assessments for one combination. Focusing on G-BA decisions, in total 135 subpopulations were assessed and predominantly showed no added benefit (41%). The awarding of an added benefit decreased, while the submission of benefit assessments increased with time. Methodological challenges concerned: the validity of progression-free survival, missing or non-interpretable data on Health-related Quality of Life (HRQoL) and non-interpretable data on adverse events (AEs). When data on HRQoL and AEs were assessed, they outweighed added benefit in 4, respectively 11 cases. Additional outcomes and subgroup analyses will be presented.

CONCLUSIONS: Oncological combination therapies are on the rise. However, added benefit decreases over time and suggests that submitted data decreases in quality and validity. In order to counteract this development, valid data, especially on HRQoL and AEs is needed. These endpoint categories may be success factors for the future. It is anticipated that costs of oncological combination therapies will continue to rise significantly, a trend that should go with convincing data.