OBJECTIVES: Identify current IHC data from the unified health system (SUS) and drug products in late-stage development (DLSD) for the treatment of advanced cholangiocarcinoma with recent or near-term future potential regulatory approval in Brazil. In 2016, a national guideline was published for the management of bile duct cancers. Recommended systemic therapies include chemotherapy with cisplatin, oxaliplatin, gemcitabine, fluoropyrimidines, taxanes or irinotecan.

METHODS: Retrospective analysis of diagnoses, hospitalizations, costs, deaths, and demographic data of IHC patients, considering out and in-patient data from the SUS database between January 2018 and December 2023. A limited search was conducted on May 22, 2024, using Cortellis Competitive Intelligence and clinicaltrials.gov for DLSD for the treatment of advanced cholangiocarcinoma.

RESULTS: The number of diagnoses increased between 2018 and 2023, from 1,951 to 3,712 cases for ICD-10 code C22. For ICD-10 code C22.1, 54% of patients were women and the main age group was 65-69 years old. The number and cost of hospitalization also increased over the period, with 1,476 and R$ 2,612,153.49, respectively, in 2023. Correspondingly, the number of deaths increased 13.64% in the period. Five new drugs launched (durvalumab for locally advanced or metastatic biliary tract cancer; pembrolizumab for MSI-H or dMMR tumors including biliary cancer; pemigatinib for unresectable locally advanced or metastatic cholangiocarcinoma with a FGFR2 gene fusion or other rearrangement; futibatinib for unresectable, locally advanced or metastatic IHC harboring FGFR2 gene fusions or other rearrangements; and ivosidenib for locally advanced or metastatic cholangiocarcinoma with an isocitrate dehydrogenase-1 mutation), two in pre-registration process and ten in late-stage clinical trials were identified.

CONCLUSIONS: A progressive increase in diagnoses, number and cost of hospitalization and deaths was observed. According to the data, the availability of new therapies could change the journey of advanced IHC patients in Brazil who, until recently, were solely treated with traditional chemotherapies.