TAPUR (Targeted Agent and Profiling Utilization Registry)

TAPUR opened in March 2016 as the first clinical trial run by the American Society of Clinical Oncology. Oncologists at more than a hundred community and academic sites order genomic testing as they normally would; if a patient's tumour has an alteration that one of the participating companies' approved drugs targets, and the match is on the protocol's list or is approved by a molecular tumour board, the patient receives the drug at no cost and is followed for response, progression and toxicity. Drugs have come from AstraZeneca, Bayer, Boehringer Ingelheim, Bristol Myers Squibb, Genentech, Lilly, Merck, Pfizer, Seagen and Taiho, including pembrolizumab, nivolumab with ipilimumab, palbociclib, trastuzumab with pertuzumab, cetuximab, olaparib, talazoparib, vemurafenib with cobimetinib, sunitinib, axitinib, regorafenib, dasatinib, bosutinib, crizotinib and erlotinib. Each drug-by-tumour cohort follows a two-stage design: ten patients, expanding to 28 if enough show benefit.

Cohorts report continuously in JCO Precision Oncology and the Journal of Clinical Oncology. Positive cohorts include pembrolizumab in breast and other cancers with high tumour mutational burden, trastuzumab plus pertuzumab in colorectal cancer with ERBB2 amplification, and several immunotherapy cohorts; negative cohorts include palbociclib in non-small-cell lung cancer with CDKN2A loss, and cohorts of cetuximab, temsirolimus and other drugs where the target proved a poor predictor. A 2024 analysis reported high agreement between the trial's molecular tumour board reviewers, evidence that curated matching can be standardised.

What TAPUR changed is the treatment of off-label targeted therapy as data rather than anecdote, at a cost far below a conventional trial, and it seeded sister programmes: the Netherlands' DRUP (2016) and Canada's CAPTUR use the same design and share results. What remains open is whether TAPUR-style evidence, which lacks a control arm, can support label changes or reimbursement; so far it has informed guidelines and directed drug companies toward or away from confirmatory trials.