DETERMINE
DETERMINE is the United Kingdom's version of TAPUR: people with rare cancers whose tumours carry a change that an already licensed drug targets in another cancer receive that drug through the NHS in a trial, and if a group does well the results are designed to go straight to the bodies that decide NHS funding.
Overview
DETERMINE opened in March 2023, sponsored by Cancer Research UK, run from the Christie and the University of Manchester with Matthew Krebs as chief investigator, and delivered through Cancer Research UK's network of experimental cancer medicine centres. It is open to adults, teenagers and children, which is unusual for a precision medicine platform, and takes patients with a rare cancer, or a rare molecular alteration in a common cancer, for which a licensed drug has a plausible target. Roche supplied the first treatment arms, including alectinib, atezolizumab, entrectinib, trastuzumab with pertuzumab, vemurafenib with cobimetinib and vismodegib, and Novartis joined with further arms.
Each arm is a phase 2/3 with a pre-set response threshold; arms that clear it can expand, and the trial was designed with NHS England so that positive arms produce evidence in a form the health service and its appraisers can act on, including the possibility of routine NHS access for the new indication. Genomic results come from NHS Genomic Medicine Service testing or from research programmes such as TARGET National, so the platform rides on the country's existing sequencing rather than building its own.
Nothing has been reported yet; recruitment is under way toward a planned 825 participants. What DETERMINE is meant to change is the fate of small positive signals: in TAPUR and NCI-MATCH they informed guidelines but rarely changed labels or funding, and DETERMINE's wager is that a national single-payer system can turn them into access directly.
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