OnCo
ideasIdea

Patient-reported side-effects (PRO-CTCAE) collected and published in every registrational trial

Doctors record only part of what patients suffer. Make it compulsory that patients report their own side-effects in every trial used to approve a drug, and that those data are published next to the doctor-graded ones.

Concordance between clinician-graded CTCAE and patient-reported symptoms is poor, with clinicians systematically under-reporting frequency and severity of fatigue, nausea, neuropathy and diarrhoea. The NCI PRO-CTCAE library provides validated items. The proposal is a regulatory requirement that registrational oncology trials collect a core PRO-CTCAE set with weekly frequency during treatment, and that results appear in the primary publication and public assessment report in a standard table.

Hypothesis
Mandated PRO-CTCAE reveals substantially higher symptomatic toxicity than CTCAE tables for most regimens and changes comparative judgements between regimens with similar efficacy.
Rationale
Selective reporting persists because it is allowed. Standardised collection with a publication requirement is the cheapest fix and has precedent in CONSORT-PRO.
What would test it
Audit the PRO-CTCAE to CTCAE discordance across the first twenty trials under the rule; survey guideline committees on whether the data changed recommendations.
Maturity
being tested at scale
Who has to act
regulator
Cost to try
Small (under $1M)
Years to first evidence
2
Bottlenecks it attacks

Key papers

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Connected

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