Presentation (CTI)

OBJECTIVES: Patients with metastatic colorectal cancer refractory to fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy (refractory mCRC) without liver metastases represent a potentially distinct subpopulation for treatment. We conducted a targeted review and gap assessment to understand the availability of epidemiological, clinical, and economic data of the approved treatments for this population.
METHODS: We searched MEDLINE (PubMed; individual searches for epidemiological, clinical, indirect treatment comparison [ITC], and economic evidence), ClinicalTrials.gov, and websites of relevant international scientific societies (including treatment guidelines) and health technology assessment (HTA) bodies (for submitted economic analyses). A structured gap assessment was conducted to identify areas with limited data on mCRC without liver metastases.
RESULTS: We identified several important literature gaps. No identified epidemiology studies reported incidence rates for refractory mCRC without liver metastases, while only one study (from the US) reported survival outcomes for this population. No observational study reported epidemiological or survival data for refractory disease. Treatment recommendations were not provided for mCRC without liver metastases in identified treatment guidelines from the US, Europe, and Japan. Identified clinical trials in refractory mCRC for approved therapies in the US and Europe did not report outcome data for patients without liver metasases, except for recent exploratory analyses from the key phase 3 trials for fruquintinib and trifluridine/tipiracil with or without bevacizumab. Consequently, none of the identified ITCs in refractory mCRC reported an analysis for this population. Similarly, no economic study or HTA submission in refractory mCRC included cost of illness, cost-effectiveness, budget impact, or utilities in refractory mCRC without liver metastases.
CONCLUSIONS: This targeted review and gap assessment identified a lack of epidemiological, comparative effectiveness, and health economic data for refractory mCRC without liver metastases. Addressing these gaps is necessary to inform research and assess unmet needs and the value of treatments under investigation for this population.