Presentation (CTI)

OBJECTIVES: To assess how prognostic factors (PFs) and treatment effect modifiers (TEMs) are identified, used, and critiqued in National Institute for Health and Care Excellence (NICE) technology appraisals (TAs), focusing on oncology and rare diseases (RDs) as examples of disease areas differing in available evidence quantity. Emerging Joint Clinical Assessment (JCA) guidance introduces more prescriptive expectations, including systematic literature reviews to identify covariates, making it important to understand current practice across these domains.
METHODS: TAs published between 1 May 2025 and 2026 on the NICE website were considered. TAs were categorised as oncology, RD, rare oncology (RO), or “other”. The most recent TAs were selected, comprising 7 oncology, 7 RD, and 2 RO. Relevant data for methods of PF and TEM identification, utilisation, and evidence assessment group (EAG) critique were extracted.
RESULTS: Published literature contributed to identification of PFs/TEMs in all TAs (100%), comprising prior clinical trials only in RD, and a broader mix in oncology (57% using network meta-analyses [NMAs] and targeted literature reviews). Other identification methods included clinical expert input (62.5%), subgroup analyses (43.8%), and prior health technology assessment (HTA) submissions (12.5%). Oncology TAs more often used multiple sources (≥2: 71%) than RD (14%). Oncology TAs primarily used PFs/TEMs to support indirect treatment comparisons (ITCs), with EAG critiques focusing on adjustment, subgroup strategy, and robustness. In contrast, RD TAs used PFs/TEMs to interpret trials, with EAG critiques highlighting absent ITCs, limited TEM consideration, and prognostic imbalances. RO TAs broadly aligned with RD, reflecting a reliance on trial-based interpretation, plus EAG concerns around matching-adjusted indirect comparison assumptions, though conclusions are constrained by the small sample (n=2).
CONCLUSIONS: Use of PFs/TEMs differs by disease area, with limited adoption of formal identification methods across all types assessed. As JCA raises expectations, identifying PFs/TEMs will be critical to avoid challenge and ensure decision relevance.