Intractable Rare Dis Res. 2021;10(2):146-147. (DOI: 10.5582/irdr.2021.01038)

Orphan drugs in different countries and development of new drugs to treat biliary tract cancer

Cheng HH, Zhou D, Wang SH, Ding Jun, Ma F


Biliary tract cancer (BTC), which includes cholangiocarcinoma and gallbladder carcinoma, is a rare malignancy. Due to its low incidence, drugs treating these diseases are scarce, so they can be considered orphan drugs. The main treatment choice for BTC is chemotherapy with gemcitabine or cisplatin or combined use of both, but patients fail to significantly benefit from established chemotherapy. Advancements in immunotherapy and targeted therapy will shed light on ways to improve clinical outcomes for patients with BTC. In conjunction, more new drugs will come onto the market. This article compares the conditions for development of orphan drugs in different countries and it describes several types of new drugs that were recently approved to treat BTC.

KEYWORDS: biliary tract cancer, orphan drugs, durvalumab, fibroblast growth factor receptor

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