Presentation (CTI)

OBJECTIVES: Developers may review a draft JCA only for factual accuracy — not challenge its reasoning. The first orphan oncology JCA (tovorafenib for paediatric low-grade glioma) shows why that distinction matters. This analysis examined the consequences of a review right that stops at facts.
METHODS: A procedural case analysis examined the factual-accuracy stage in the published JCA report and record, focusing on the boundary between contesting a factual error and contesting the assessment team's methodological reasoning, and on the disputes that fell across that line.
RESULTS: The review stage confines the developer to flagging factual or technical inaccuracies and bars comment on the assessment's results or reasoning. In practice the boundary proved unstable: a key dispute — whether the developer had comprehensively identified prognostic variables and effect modifiers — is simultaneously factual and methodological, yet could not be reopened as reasoning. The effect is that the assessment team's analytical conclusions are, in practical terms, beyond challenge within the procedure, however consequential they are for the outcome. For a first-in-class orphan case where methodology drove the result, foreclosing methodological reply concentrates decisive influence in one stage with no internal route to test it.
CONCLUSIONS: A factual check cannot reach the reasoning that decides the outcome. Where methodological judgement determines assessability, developers need a bounded, documented way to contest that reasoning — not only to correct numerical or typographical errors.