ideasIdea
Registry-based randomised trials for oncology comparative effectiveness
Use the cancer registry itself as the trial machine: randomise patients at diagnosis, then let the registry collect the outcomes for a fraction of the usual cost.
Registry-based randomised trials (R-RCTs), pioneered in Swedish cardiology, use the registry for identification, randomisation and outcome follow-up. Oncology has the registries (Nordic, England, Netherlands) but almost no R-RCTs. The proposal funds a programme of five R-RCTs on high-value comparative questions with routine endpoints (surveillance intervals, adjuvant duration, supportive care) and develops the ethics and consent templates to make the design routine.
Hypothesis
R-RCTs will answer comparative effectiveness questions with 2,000 or more patients at under 500 dollars per patient and within three years, roughly a tenth of conventional trial cost.
Rationale
TASTE randomised 7,244 patients for a small fraction of a conventional trial budget; oncology questions about duration, schedule and surveillance have equally routine endpoints.
What would test it
Run one R-RCT in a Nordic or English registry (for example, imaging surveillance every six versus twelve months after curative colorectal surgery) and report cost, enrolment and completeness of outcomes.
Maturity
early clinical
Who has to act
research
Cost to try
Medium ($1M to $50M)
Years to first evidence
4
Bottlenecks it attacks
- Weak real-world evidence and registries · We do not reliably know what happens to patients after approval, so we cannot tell which drugs deliver in practice.
- 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.
- Trials enrol too few, too slowly · Fewer than one in ten adults with cancer joins a trial. Trials close for lack of patients, not lack of ideas.