OBJECTIVES: As increasing oncology health expenditures call for a maximization of the value-per-dollar spent, health technology assessments (HTA) agencies, such as the G-BA in Germany, are requesting long-term, mature comparative clinical data. The aim of this research was to identify additional data requests by G-BA in oncology diseases that relate to uncertainties in the clinical evidence package at launch and, subsequently, in the value-per-dollar spent.

METHODS: We selected two hematological (chronic lymphocytic leukemia [CLL], multiple myeloma [MM]) and two solid (ovarian cancer [OC], triple-negative breast cancer [TNBC]) oncology disorders. We identified all assessments conducted by G-BA until December 31, 2022, on targeted therapies for these disorders and analyzed the data-driven reassessments requests and time-limits impositions.

RESULTS: We identified a total of 44 HTA appraisals, of which seven (OC: n=2; CLL: n=1; MM: n=4) were reassessments. Most reassessments (5/7) related to clinical uncertainties at the initial assessment, with the remaining (2/7) being related to pricing. Additionally, 10 out of the 44 HTA appraisals (OC: n=3; TNBC: n=1; CLL: n=1; MM: n=7) involved time-limit resolutions related to clinical uncertainties at the initial assessment. Additionally, none of these time-limit resolutions were imposed in the reassessments. Many reassessments (6/7) and some time-limit impositions (7/12) involved resolutions published after 2019.

CONCLUSIONS: As pressures to control expenditure increases, so does the need to provide clinical data that supports health investments and offers clinically meaningful evidence directly relevant to patient experience and clinical practice. This research shows how reassessments and time-limits are increasing in Germany as the demand for value increases. Early payer engagement that allows the understanding of evidentiary expectations together with robust at-launch and post-launch evidence generation packages can help mitigate the access risks posed by clinical uncertainties.