Report gallbladder cancer as its own population in every biliary trial
Every drug gallbladder cancer patients receive was tested in mixed bile duct trials where they were a subgroup. Requiring trials to pre-specify and publish gallbladder results, with a minimum number enrolled, would give the disease its own evidence at almost no cost.
Overview
ABC-02, BILCAP, ABC-06, TOPAZ-1 and KEYNOTE-966 enrolled gallbladder cancer alongside intrahepatic and extrahepatic cholangiocarcinoma and reported it, if at all, in forest plots. Yet the diseases differ: HER2 positivity runs at 9 to 20 percent in gallbladder and extrahepatic cancers against 5 percent intrahepatic; immune profiles differ by population; and the UK CAPBIL cohort saw no adjuvant benefit in a mostly gallbladder population. A reporting standard (pre-specified gallbladder stratum, minimum enrolment, mandatory subgroup publication) could be asked of sponsors by regulators and journals and would cost the trials almost nothing.
- Rare and paediatric cancers without markets · Taken together rare cancers are a fifth of all cancers, but each one alone is too small for a company to invest in.
- Trial design, endpoints and cost · A phase 3 trial takes years and hundreds of millions of dollars, and often answers a question that has already moved on.
- Failures are hidden · Negative trials, failed drugs and abandoned programmes are rarely published, so the same mistakes are repeated.
The adjuvant finding sits uneasily beside BILCAP, on which NHS adjuvant capecitabine rests; in a mostly gallbladder population the benefit is not visible. ACTICCA-1 and ARTEMIDE-Biliary01 are the trials that can settle it.
Evidence that the immune environment of gallbladder cancer differs by population even when the mutations do not; a reason to report gallbladder cancer and its regions separately in immunotherapy trials rather than as one biliary subgroup.
The prevalence estimate that made HER2 the first gallbladder-relevant target; it argues for testing every advanced gallbladder cancer, since one in five may qualify for zanidatamab or trastuzumab deruxtecan.
Similar pages
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