OBJECTIVES: In the Netherlands, the Committee for Evaluating Oncological Drugs (CieBOM) assesses the clinical benefit of systemic anti-cancer treatments (SACTs). CieBOM uses different criteria for randomized controlled trials (RCTs) and non-randomized trials (NRTs). Our study aimed to evaluate whether NRTs are a viable alternative to RCTs for assessing SACTs through applying the NRT criteria to NRTs for treatments that were initially assessed based on RCTs.

METHODS: We searched for NRTs that matched the RCTs used by CieBOM for their assessments between 2015 and 2017 (n=37). The matching NRTs were then used to compare the outcome of the assessments based on the respective criteria, namely overall survival and progression-free survival (PFS) for RCTs and objective response rate (ORR) and duration of response (DoR) for NRTs. Different thresholds for ORR, DoR, and PFS were explored in scenario analyses to examine their impact on the outcome of the assessments.

RESULTS: Out of the 37 RCTs, only 13 had matching NRTs. Of these NRTs, two SACTs were assessed positively and six negatively. Five NRTs could not be assessed due to missing data on ORR and/or DoR. Only two positive RCT-based assessments aligned with positive NRT-based assessments. In one case, a negative assessment was consistent across both RCT- and NRT-based criteria, while one drug was not assessable under either set of criteria. Adding a PFS threshold of >6 months significantly reduced the number of non-assessable NRTs from five to two.

CONCLUSIONS: Limited availability of reported NRTs and limited reporting of ORR and DoR outcomes encumber the viability of assessing the clinical value of SACTs under the current NRT-related criteria used in the Netherlands. Additionally, the utilization of NRT criteria instead of RCT criteria may result in different assessment outcomes. Including PFS as an additional criterion may increase the number of assessable NRT-tested SACTs.