Presentation (CTI)

OBJECTIVES: The EU HTA Regulation requires a JCA to report the degree of certainty of relative effects. This analysis asked whether the first orphan oncology JCA (tovorafenib for paediatric low-grade glioma) actually delivered that, or stopped at documenting uncertainty.
METHODS: A methodological case analysis examined how the published JCA report treated certainty and uncertainty, comparing its characterisation of limitations with the Regulation's requirement (Article 9) for an explicit statement of the degree of certainty, and considering the interpretive position of national bodies receiving the report.
RESULTS: The report examined uncertainty in detail — confounding, comparison validity, sample size, outcome definitions — but did not translate that analysis into an explicit, structured statement of how much confidence could be placed in the estimated effects. The result is an interpretive gap: national appraisal bodies receive a thorough account of what is uncertain without a calibrated certainty conclusion to anchor their decisions. Because no internationally recognised certainty framework was applied to convert documented uncertainty into a graded statement, two readers could reasonably draw different conclusions from the same report. The omission is consequential precisely because the certainty statement is the bridge between a factual assessment and a national value judgement.
CONCLUSIONS: Documenting uncertainty is not the same as stating certainty. The framework needs a transparent, structured route from listed limitations to a graded certainty conclusion — otherwise the central deliverable of the JCA is left for each national body to reconstruct.