Descriptive Landscape for Precision Medicines in Second-Line Biliary Tract Cancer: A Targeted Literature Review


Su W1, Sabater J2, Fan L1, Mettam S3, Breislin E4, Millward E4, Roiz J4, Smith M4, Paisley S4, Bridgewater J5
1Jazz Pharmaceuticals, Philadelphia, PA, USA, 2Jazz Pharmaceuticals, Oxford, OX4 2RW, UK, 3Jazz Pharmaceuticals, Bramley, SRY, UK, 4Lumanity, London, UK, 5UCL Cancer Institute, London, WC1E 6DD, UK

OBJECTIVES: Biliary tract cancer (BTC) is a rare group of malignancies, including gallbladder cancer, intrahepatic and extrahepatic cholangiocarcinoma, representing less than 1% of adult cancers. BTC often harbors multiple clinically actionable molecular alterations, with expression varying by subtype. Currently, FDA/EMA-approved precision medicines in BTC are only available for FGFR2 and IDH1. As such, a targeted literature review was conducted to define current standard practice in second-line (2L) BTC and the level of utilization of precision medicines in the context of their efficacy.

METHODS: MEDLINE, TRIP, WHO, GIN, and NCCN databases were searched in August 2023 for literature published since 2018. Documents underwent screening/extraction following Cochrane guidance; pre-specified prioritization factors guided selection. Supplementary hand-searches supported and contextualized findings.

RESULTS: 322 clinical guidelines and 423 articles were identified, with 4 guidelines and 26 articles selected for extraction and reporting. For 2L therapy, NCCN and ESMO guidelines recommend FOLFOX as the preferred and standard regimen, respectively, while considering targeted and immuno-therapies linked to actionable alterations and checkpoint blockade therapy. Consistently, FOLFOX/FOLFIRI were the most frequently mentioned 2L treatment options (12 articles; 46%); whereas 6 articles (23%) stated no SOC. NCCN and ESMO guidelines recommend advanced BTC patients receive comprehensive molecular profiling for genetic alterations. Among articles reviewed, HER2, FGFR2, KRAS/MAPK, IDH1/IDH2, BRAF, BRCA, and microsatellite instability were the most discussed actionable alterations, for which immunohistochemistry tests and next-generation sequencing have an important role. Testing for other targets is important, particularly HER2, where several therapies are under clinical investigation.

CONCLUSIONS: In 2L BTC, FOLFOX/FOLFIRI were the most frequently mentioned treatment options, while HER2, FGFR2, KRAS/MAPK, IDH1/IDH2, BRAF, BRCA, and microsatellite instability were the most discussed actionable alterations. Ongoing clinical investigations may cause a shift in the treatment paradigm from chemotherapy to targeted therapies in the context of the efficacy, tolerability, and potential economic value of the latter.

Conference/Value in Health Info

2024-05, ISPOR 2024, Atlanta, GA, USA

Value in Health, Volume 27, Issue 6, S1 (June 2024)




No Additional Disease & Conditions/Specialized Treatment Areas, Oncology

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